Multiple Myeloma Archives

Plasma cells are cells in the immune system that make antibodies, which help the body fight infection and disease. Multiple myeloma cells are abnormal plasma cells (a type of white blood cell) that build up in the bone marrow and form tumors in many bones of the body.

More resources for Multiple Myeloma from Patient Empowerment Network.

Fertility Preservation in People with Cancer

This podcast was originally published by Cornell Weill Cancer Cast, on March 22, 2019, here.

Myeloma Patient Cafe® – Genetic Testing from A Myeloma Patient Perspective

Myeloma Patient Cafe® – Genetic Testing from A Myeloma Patient Perspective from Patient Empowerment Network on Vimeo.

PEN Board Member, Jack Aiello, leads a myeloma patient panel discussion on genetic testing.

See More From The Myeloma Patient Cafe®


Transcript:

Jack:

Thanks for joining us for this Patient Empowerment Network Myeloma Patient Café. I’m Jack Aiello. I’ve been living with myeloma since 1995, and the world has changed a lot since then, including the introduction of genetic testing. That’s gonna be our topic of discussion today. I personally have never had genetic testing because it wasn’t done back then, so I’m looking forward to learning from you all, our patient panel, who have been diagnosed more recently than I have.

We’ll talk exactly what genetic testing is about, why you might get genetic testing done, and more, but we won’t really go into the science of it. Instead, this is gonna be a conversation among patients and caregivers and serve off as a jumping point to pique your interest in genetic testing, and have a discussion with your doctor about it if you desire.

Before we dive in, I wanna meet our panel, and I’m gonna ask each of you to introduce yourself. Tell me when you were diagnosed and the treatments you’ve gone through, and I will start with Doug.

Doug:

I’m Doug Kenaley. I was diagnosed in 2015, and my initial induction treatment is a little different than most. It was really only – it turned out to be Velcade and dex, and it got me down to the level where I could have a stem cell transplant, so then, I had an auto stem cell transplant. And then, about five months after that, I joined the elotuzumab maintenance trial, so I’ve been on elotuzumab and Revlimid since that point.

Jack:

Okay. Peggy, tell us about yourself.

Peggy Lindley:

My name is Peggy Lindley, and I was diagnosed with this lovely disease on Valentine’s Day of 2019, and it was just from my regular doctor. I go every year for my bloodwork, and he found something with me, and he found it only – he was aware of it because his mother was diagnosed a couple of years before that, so he’s the one that got me there because I would have never thought that. He asked me, “Do you have any bone pain or anything?” I said, “Well, just my back,” and that was it.

Anyways, he told me what I had. Then, I had a bone marrow biopsy, and that showed it. So, I went through five rounds of the Revlimid, dex, and Velcade, and then, in July of last year, I had my stem cell transplant, and I got my stem cells back on July 12th, and then, in November of last year, I started with the maintenance therapy, which is elotuzumab with Revlimid, so I do that every 28 days now. It was a little bit sooner, and you start one week – it was a progression, so now, I go once every 28 days.

Jack:

Got it. Nancy, tell us about yourself.

Nancy:

My name is Nancy Raimondi, and I was initially diagnosed in 2006 with smoldering multiple myeloma, and I was followed over the next nine years – I just continued to smolder until 2015. I developed a plasma cytoma, and that got biopsied, and it was 60 percent myeloma cells, so I needed treatment, so I started treatment July of 2015, I was diagnosed as high risk, so I was put in a clinical trial that included carfilzomib.

I had five rounds of chemo, did tandem stem cell transplants, and finished everything about seven and a half months later. Went in maintenance therapy the first year, was when Ninlaro was just released, so I was on Ninlaro, Revlimid, and dex for a year, and then, that got changed to daratumumab, Revlimid, and dex, and I was on that for another year. And then, in December 2017, I was MRD-negative, and I’ve not been on any treatment for myeloma since then.

Jack:

We’ll talk more about MRD-negative because that’s important to this discussion. George, how about yourself?

George:

My name is George Burrell. I was diagnosed in April of 2011. Ironically, the day that I was diagnosed was Easter Sunday and my wife and I’s anniversary. The – doctor told us we had multiple myeloma, and that we needed to get the numbers down so that he could put me in a stem cell transplant. I’ve had two of those, and I’m currently on a three-stage regimen of Cytoxan, dexamethasone, and Kyprolis, and it seems to be working quite well. My numbers are down, and have been holding pretty steady for about four or five months now, so we’re really happy.

Jack:

I thought it was interesting, Peggy, how George introduced the fact that “we” were diagnosed with myeloma, so maybe you can talk about what that experience was like for you.

Peg Burrell:

Well, definitely, it is a journey of “we,” and it was very frightening. I’d only heard the word “multiple myeloma” one time, with a colleague from work whose father was much older, who’d had multiple myeloma. And, George’s symptom was low iron anemia, and he’d been sent to an oncologist for iron infusions, but he never presented any other symptoms.

The doctor would say, “How are you?”, and he would say, “I’m fine,” and a year later, he was rushed to the emergency room with bleeding ulcers, and that’s when the oncologist just happened to be in the ER, and they thought George was having a heart attack, his blood count was so low, so they did a CT scan, and his oncologist came in and said, “This is multiple myeloma, I’m pretty sure.”

So, it was devastating, very frightening, but once we had a game plan – and, the one thing that George told me – he says, “Stop treating me like I’m dead,” and I was running over curbs taking him to appointments, and I was just a wreck. He was like, “You’re gonna kill me.” But, it is quite a journey, and I’m happy that I’ve been able to be there with him.

Jack:

Good. Since this Patient Café is to focus on genetic testing, let’s first get agreement what genetic testing is, which is basically looking at potential mutations in your myeloma cells. So, with that in mind, other than me, who’s never had genetic testing, has every patient here had genetic testing?

Doug:

Yup.

Peggy Lindley:

I have.

Jack:

Probably, right? Because you begin maybe with a FISH and cytogenetics testing. Doug, when you had that, did that yield anything interesting for you?

Doug:

Mine was a bit interesting because I went to a local oncologist, even though I was here in Houston, who’s close to me, and he had done a stint at MD Anderson. And so, he presented it to me when I was diagnosed – “You should have genetic testing right away.” So, I looked into it and thought it was a good idea, even though four years ago, even, there wasn’t a whole lot more – you have a test, but then what? That kind of thing.

This emphasizes why a lot of times, you wanna go to a specialty place like MD Anderson, because they did the bone marrow biopsy, and the tech put it in the wrong solution, and it destroyed the sample. But they were gonna hold off my induction. So, the doctor was pretty mad, but my first attempt was a failure. But then, he said, “Well, ultimately, you’ll probably go for a stem cell transplant. We’re gonna hook you up with MD Anderson right away, even during your induction.”

And, the first thing they do here is genetic testing. So, at that point, I got a genetic test – successful genetic test – and it was interesting because the results came in, I got the labs, and I’ve done science – I’m a scientist, I’m a geologist – but it’s just a lot of alphabets, and it’s very complicated. They’re worse in the summaries. It said, “No deletions found, no translocations found,” things like that, but you really couldn’t understand the rest of what was in there, and you kind of suspect there was something hidden in there.

But I sat down with the doctor here, and he went over it. It said basically, I was a standard-risk patient, and my FISH and cytogenetics showed that I had tetrasomies – so, four versions of the genes instead of the normal two. And, he says, “So, if you wanna look at it, that’s kind of a good news thing because we have drugs that target certain things, you have lots of those things to target – multiple copies of those things,” so that kind of relaxed me a little bit. I think it actually impacted my standard of care a little bit, and certainly, my quality of life, because I think the doctors relaxed a little bit too. They wanna get ahead of it if you’re high-risk.

Jack:

So, Peggy, when Doug mentions he got a report from FISH and cytogenetics, which is essentially gobbledygook –

Peggy Lindley:

It is.

Jack:

What did you do when you got that?

Peggy Lindley:

They told me right off the bat that I had myeloma, and that I had an aggressive form. So, I went through the rounds, and I responded very well to induction therapy.

Jack:

And, by “aggressive form” – how did they find that?

Peggy Lindley:

They just said it was aggressive. They didn’t really – they said the FISH test – it was still Greek to me. So, now, two years later, I’m understanding it more and more, but what it was was the translocation of the 4-14. So, I find that, and I ask doctors about that, and they say, “Yes, it is aggressive, it’s on the aggressive form, but it’s still on the intermediate side.” So, I’m not as concerned, but at least the doctors know, and they’re aware.

Jack:

And, “4-14” means that chromosome 4 and chromosome 14 pieces have been swapped places?

Peggy Lindley:

I don’t really understand that yet, but I’m learning. That’s good, very good. See? I’ve learned something more.

Jack:

There you go. And, Nancy, you’ve been at this for a little while, so you probably understand a little bit more about genetic testing. What’s the impact been on you?

Nancy:

Well, I’m getting there, but it is – it’s a lot of alphabet soup. It’s hard to retain. But, yeah, I had genetic testing done right away once the myeloma became active, and I also had aggressive highrisk. I had abnormal female karyotype, monosomy 13, the P-53, and a translocation – but I forget which one. And, what was interesting is in my initial appointment with my oncologist, he thought I was low-risk and talked about treatment, but when all the final results came back, turned out I was high-risk, which meant completely different treatment. So, that was a shocker.

Jack:

So, expand on that a bit. How did that high risk change your treatment?

Nancy:

He recommended a clinical trial instead of what they were gonna put me in, which included being treated with carfilzomib, which, at the time – this was 2015 – carfilzomib was being used mostly for people who had relapsed, and they were doing a clinical trial to see about treating patients up front with it that are high-risk. Why wait until they relapse? So, I had that in addition to the PACE cocktail with thalidomide, something else – there were seven different chemos.

Jack:

So, that was important that that high risk for you helped determine a change for the treatment, but you got into that clinical trial, and that was an effective trial, by the way, so that’s good.

Nancy:

Yes. It was definitely effective for me.

Jack:

Good. And, George, when you did – or, you did genetic testing, I presume, and did it show anything?

George:

Yes. The first time we did it was to run tests to get ready for the first stem cell transplant, and at that time, I didn’t understand the importance of all that. The oncologist that I was working with at the time did explain as much as he could, and in layman’s terms as best he could, but it still mostly went over my head. I was more thinking about the actual transplant itself than anything else. But, when I came to MD Anderson and got ready for – I was getting ready to try one of their clinical trials, they ran some more tests then, just to see how things had progressed through that number of years, and so, I’ve actually had partially two of them.

Jack:

So, I was gonna ask – have any of you had subsequent genetic testing where results have changed after your treatment for myeloma? You’re nodding your head, Nancy.

Nancy:

Yeah. Over a year ago now, I had my genetics repeated, and all the abnormal stuff went away, so that was pretty exciting, because I was now MRD-negative, so that was very reassuring. And, I actually just had another bone marrow about 10 days ago now, so I’m still waiting for those results to see what’s happened.

Jack:

And, are they gonna test that bone marrow for genetics as well?

Nancy:

Yes.

Jack:

Because you might find there are changes. You might find there’s a translocation where there wasn’t one before, you might find there’s a deletion where there wasn’t one before, because this myeloma is a fairly tricky disease, and we talk about the myeloma clone as made up of a percentage of different mutations, some of which get cured by treatment, and others of which expand because they were not affected by the treatment. It’s pretty interesting, in a lousy sort of way. Anything else, Doug, that you thought was interesting that came out of your genetic testing?

Doug:

It looked pretty standard and fairly boring to people who liked exciting genetic testing. I did have two, so I had one – so, my original doctor says, “We like to get patients early to get an original profile.” That’s kind of like your baseline. And, I also had one right before my stem cell transplant because they like to check to see if anything happened, but the doctor says the chemo messes with myeloma – obviously, that’s why you have chemo – and he says, “You’ll probably see some differences, but that’s why we like an original one, too.”

So, I compared the two, and really, there were no extra risks – high risks or anything – that appeared. The only thing that popped up was instead of tetrasomies, I had trisomies also, but that was pretty much it. So, it didn’t really change anything in terms of treatment in terms of work that was being planned.

Jack:

And, tetra- and trisomies are basically quadruple and triple duplications of your chromosome. So, I’m wondering, both Peg and George, have you had a second MRD testing, and why did you end up doing that?

George:

I don’t know that we’ve had a second one. Have we?

Peg Burrell:

Yes.

George:

We have?

Jack:

Oh, you had MRD testing?

Peg Burrell:

Yes, I’m pretty sure we had. He was in a clinical trial in 2018 at MD Anderson, and I’m sure they did it then. They also did some very unusual – not normal, but they were genetic tests that they ran as part of the – at the beginning of this study so they could get a baseline, or find out what other things might be going on.

Jack:

And, he did this MRD trial testing to determine if he had a significant number of cells with this BCMA antigen in order to qualify for this CAR-T trial?

George:

Probably.

Peg Burrell:

In the beginning, yes. That was in 2012. And then, he had his – he was in a second trial that was called Amgen 224. That’s all we know. It’s a mystery. And, it worked for him for about a year. It brought his cancer numbers back down, and there was a lot of genetic testing for that particular trial.

Jack:

So, I think just about any of these new trials that are coming onboard these days incorporate MRD testing. As we all heard earlier and we know, MRD is a really good prognosticating factor in terms of if a patient becomes MRD-negative, they show that they have better progression-free survival and overall survival. It’s not really used to change or determine treatment, but those trials are going on as well, so I think that’s really important.

And, there’s so many other avenues – again, back to this genetic testing, I always wonder, well, suppose I’m MRD-negative, but I’m high-risk, versus I’m MRD-positive but I’m standard-risk. Which is better? I don’t know. I don’t think the community knows, and I think it may be individualized as well. What do you all think about that? Any feelings?

George:

For me, I think that’s probably what is gonna be revealed to us as we move forward with this because the genetic testing idea is fairly new, at least to the patient. I’m sure that the doctors could pull each and every one of our files and show us all sorts of information that they just haven’t shared with us because of – it can be kind of complicated and hard to understand. Sometimes, I think that they try not to give us too much information because then, we have a tendency to think we can get on the computer and try to diagnose ourselves or find something.

Jack:

Little Rock, Arkansas was kind of the pioneer in what’s called gene expression profiling. And, we all have 25,000 genes, let’s say, and Arkansas kind of developed a test which showed that there were about 70 genes that were very distinctive in resulting in high-risk myeloma, except they were distinctive across, say, 50 percent of patients, but not the other 50 percent of patients. And then, they tried to get it down to even 15 or 25 genes, let’s say, and therefore, it was less accurate.

So, I think you’re right, George. I think there’s still a lot of work that’s gonna be done in this area to make it something that really can be useful in terms of having the best treatment for patients. There’s an interesting trial going on right now that’s looking at treating myeloma patients according to a mutation. If we have a certain mutation and we have a drug to treat that mutation – it could be for a different cancer – then that patient will be given a baseline of treatment plus that drug to try to increase the amount of precision therapy that’s given for given patients.

So, this whole area of genetic testing, as I see it, is really fascinating, complex, difficult to understand at the patient level, but can mean a lot for us as we go forward. What do you think? Peggy, I think you’re the most newly diagnosed patient here. What does all this mean for you?

Peggy Lindley:

I think his analogy of the alphabet soup is exactly right because when I looked at mine, I was like, “Those are words? Yeah, no.” But, I tried not to worry about it because I figured I was going to the best when I came to MD Anderson, so I really didn’t worry about it too much because I figured it’s gonna be what it’s gonna be, and I wanna – the quality of life is what I’m looking for.

Jack:

Well, I think you’re really correct there. The fact that you’re going to MD Anderson, the fact that we are getting second opinions from myeloma specialists who have a much better shot at understanding this stuff than we do is really key to long-term treatment success for us. There are a lot of drugs out there. In fact, I’ll often tell patients when I was diagnosed in ’95, there weren’t many treatment options. Today, the good news is there are lots of treatment options, but the bad news is there are lots of treatment options. You really don’t know what’s best for you, and that’s why it’s so important to have a myeloma specialist on your side.

George:

Well, with that, the idea of being able to target certain things within myeloma is gonna be a big step forward, I think, because it’ll help eliminate some of the things – the trials that we might try, or have to make a decision – “Do we try this or not?” We’ll be able to say, “This didn’t work, so this will – let’s try this.”

Jack:

Yeah. There’s a drug called venetoclax, which has been shown to be effective in myeloma patients with a certain mutation – 11-14 – and it’s in trials now to hopefully, one day, get approved for that class of patients.

Doug:

One of the things I’d probably add to the discussion is there’s a lot of talk about patient advocacy, and if you follow any of the myeloma discussions, it is almost all genetics now. That’s kind of where cancer research has gone, even in other cancers. But, one of the things that I see genetic testing is doing is my ability to help the doctor help me.

So, if it was more difficult to get genetic testing – maybe not local to a major facility or something – I would still encourage it because that’s helping the doctor see your specific disease, and maybe helping them modify what you have as a standard treatment in terms of what you need instead of the standard treatment. Plus, you have it in the bank then. You have your test, and if something is discovered a year from now, that this particular drug works with this particular genetic profile, you can go back, and look, and say, “Do I have that? Is that something I should consider?”

Jack:

Good point. Nancy, how important do you think it is for patients to 1) Insist that they get some type of genetic testing, and 2) to try to understand what’s going on?

Nancy:

Well, I think it’s extremely important. For me, it was a major change in treatment. Without genetic testing, I doubt I would be MRD-negative right now because my treatment path went along a completely different way. So, I think it’s extremely important. What was the second part of your question?

Jack:

How important it is for the patient to understand it.

Nancy:

I think everybody has a different level of what they can understand, and that it’s important for your oncologist to give you that information in language that you can understand, and to the level that you want. A lot of that’s gonna depend on your background, your education, what makes sense to you. I came from a medical background, so I wanted a little more knowledge, and my doctor was great in giving that to me.

Jack:

Patients need to ask questions.

Nancy:

Yes, they definitely need to ask questions, and then, the physician needs to communicate in a way that the patient’s gonna understand because it is a lot of gobbledygook, and I often – I have a hard time understanding it with having a medical background, and I often wonder how you make sense of this without having a background. It’s difficult.

Jack:

This has been a good discussion, and I think we’ll wrap it up. Peg, maybe I’ll start with you. Folks listening to this discussion – what do you think they should take away from it?

Peg Burrell:

Well, definitely, talk your physician, learn as much as you can. Support group for us has been very beneficial and helpful. Our support group brings in different people in the medical profession and has explained a lot of the things and given us knowledge we wouldn’t have had otherwise. And then, working with – sometimes, insurance may not wanna pay for certain tests. I’ve found that in working with MD Anderson, their financial people – we had some tests that were gonna be – I think they said “non-concerted.” I’d not heard that before. It basically meant they were questioning the test and whether or not it was necessary. So, MD Anderson was very helpful with that.

Jack:

George, can you add on to what your better half says?

George:

For me, it’s been working closely with Dr. Patel and her team, both when I was part of the clinical trial and even now, with the three-track regimen that they have me on. Again, ask questions, try to understand as much as you can, and I, too, support the idea of working with a support group and sharing information with each other because you find out so much more of what someone else heard through their doctor and their team because we all do have different doctors.

Jack:

Nancy?

Nancy:

I think it’s real important for people to go to a center of excellence, at least for a second opinion, if not for your treatment. They are the cutting-edge people that are gonna be able to treat you the best, and you can just google “center of excellence, multiple myeloma,” and you’ll get a list of all the centers all across the United States. I think it’s made a huge difference. I was treated at UAMS in Little Rock, and I wouldn’t have had it any other way. I was fortunate to be able to go there.

Jack:

Good. Peggy?

Peggy Lindley:

I think as patients, we all need to be as informed as you can, and work with your doctor, and get confidence in your doctor. If that doctor doesn’t do it for you, find another one, but be confident in your doctor that they’re gonna do what’s right for you, but you have to be educated as well.

Jack:

I heartily agree. Doug?

Doug:

I’d stress the same as everyone else, and also recommend definitely having genetic testing. One of the things that are kind of an intangible benefit is even your own stress level. You would think that, for instance, if you’re tested and you find out you’re not high-risk, you’re standard-risk, that’d be the end of it, but it turns out, for instance, even with me, my doctors will actually modify – have modified my treatments, even my maintenance treatments, because I’m not high-risk and I have very stable myeloma.

So, they’ll say, “Well, we’re going to de-escalate. We’re gonna take you off all these drugs. You don’t need all of them, so we don’t wanna over-treat, either.” Nobody wants to be over-treated with all the symptoms and things like that. When they initially said that, I was like, “Wait a minute, I’d rather just start adding drugs. Let’s just kill this thing.” But, that’s right, and I think the fact that I can go back to genetic testing and look at what he was saying about stability over a period of years and things like that just gives me more of a comfort level that that’s probably the right answer, and I don’t need to be taking all these drugs if they’re not gonna benefit me in the long term, or I could switch drugs if I need to.

Jack:

So, I guess I’d summarize it by thanking you all. You’re all terrific examples of being your own best patient advocate. If we aren’t advocating for ourselves, who else should? It’s really up to us, and the good news is there are so many resources for good information out there.


Please remember the opinions expressed on Patient Empowerment Netowrk (PEN) are not necessarily the views of our sponsors, contributors, partners or PEN. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.

Connect with Myeloma Groups

This resource was originally published by Myeloma Crowd here.

CONNECT WITH THE MYELOMA CROWD

Myeloma Crowd is a division of the CrowdCare Foundation and is a registered 501(c)3 non-profit, tax ID 45-5354811. Contact us at info@crowdcare.org. For donations, please donate online to the MCRI project here or mail checks to:

CrowdCare Foundation
PO Box 1286
Draper, UT 84020

 

JOIN MYELOMA FACEBOOK GROUPS

MULTIPLE MYELOMA SPANISH SITE

Financial Assistance Programs

These resources were originally published by Myeloma Crowd here.

Financial Resources

Before starting treatment, patients and their caregivers should meet with the financial counselor at the facility where they will be treated. Each hospital will have its own financial counselor within each specific clinic (like the myeloma clinic or myeloma/lymphoma clinic). The counselor will help determine coverage and estimate out of pocket costs. There may be restrictions on treatment options covered by Medicare and Medicaid. After you have met with the counselor, the resources below can help bridge financial gaps.

Myeloma advocate and financial planner Diahanna Valentine helped compile this list of helpful financial resources. To contact Diahanna for professional assistance, email her at dtvallentine@gmail.com

Pharmaceutical Company Patient Assistance Programs

Amgen Assist 360  (1-800-28-AMGEN) (Kyprolis, Xgeva, Neupogen, Neulasta)

BMS Access Support (1-800-861-0048) (Empliciti)

Celgene Patient Support (1-800-931-8691, EXT 4077(Revlimid, Pomalyst, Thalomid)

Genentech  (1-888-941-3331) (Venclexta)

(800-609-1083) (Doxil, Darzalex)

Novartis Patient Assistance (1-800-245-5356) (Farydak, Zometa)

Takeda Oncology (1-844-817-6468) (Ninlaro, Velcade)

Other Patient Assistance Programs

Be The Match (Post-transplant financial assistance)

CancerCare

CephalonCares Foundation

HealthWell Foundation

Help with Paying Bills

The Leukemia & Lymphoma Society’s Co-pay Assistance Program

Patient Financial Aid

The Max Foundation

National Cancer Institute

National Organization for Rare Disorders

NeedyMeds

Patient Access Network Foundation

Patient Advocate Foundation

Patient Advocate Foundation Co-Pay Relief Program

Ronald McDonald House Charities

Basic Living Expenses

American Childhood Cancer Organization

American Life Fund
The Bone Marrow Foundation

The CHAIN Fund Inc

Child Care Expenses

Family Reach Foundation

The Bone Marrow Foundation

CancerCare

Equipment /Supplies Expenses

American Kidney Fund

CancerCare

Lymphoma Research Foundation

Stupid Cancer

Government Assistance

Benefits.gov

Centers for Medicare & Medicaid Services

Eligibility.com (Medicare Resources)

Legal Services Corporation

Social Security Administration

Social Security Disability Resource Center

U.S. Department of Veterans Affairs

Home Health Care Assistance

AVON Foundation for Women

The Bone Marrow Foundation

CancerCare

Brenda Mehling Cancer Fund

Stupid Cancer

Housing During Treatment

American Cancer Society (Hope Lodges)

The Bone Marrow Foundation

Healthcare Hospitality Network Inc

Hospitality Homes

Joe’s House

Lymphoma Research Foundation

Insurance Premium Expenses

American Kidney Fund (dialysis patients)

American Life Fund

The Bone Marrow Foundation

CancerCare Co-Payment Assistance Foundation

The CHAIN Fund Inc

Fifth Season Financial Assistance

HealthWell Foundation (diagnosis-specific)

Patient Advocate Foundation Co-Pay Relief

Patient Services, Inc

Stupid Cancer

Legal Issues

American Bar Association

Cancer and Careers

Cancer Legal Resource Center

LawHelp.org

Legal Services Corporation

National Coalition for Cancer Survivorship

National Health Law Program(links to assistance programs)

Patient Advocate Foundation

Social Security Disability Resource Center

Medical Care Expenses

American Life Fund

Be the Match

The Bone Marrow Foundation

CancerCare

Cancer Warrior, Inc

Fifth Season Financial Assistance

Foundation for Health Coverage Education

Leukemia and Lymphoma Society

Patient Access Network Foundation

Patient Advocate Foundation

Post Treatment Financial Needs

Be The Match

Stupid Cancer

Prescription Expenses

The Bone Marrow Foundation

CancerCare Co-Payment Assistance Foundation

Cancer Financial Assistance Coalition

The CHAIN Fund Inc

Foundation for Health Coverage Education

GoodDays

HealthWell Foundation

Leukemia and Lymphoma Society

Needy Meds

Patient Access Network Foundation

Patient Advocate Foundation Co-Pay Relief

RxAssist

RxHope

RxOutreach

Together Rx Access

Transportation and Travel Resources

Angel Flight

Air Charity Network

American Cancer Society (Road to Recovery)

Angel Airline Samaritans

Angel Airlines for Cancer Patientsrg

Angel Flight Central

CancerCare

Chai Lifeline

Corporate Angel Network

Family Reach Foundation

Lifeline Pilots

Lymphoma Research Foundation

Mercy Medical Angels

National Patient Travel Center

Veterans Airlife Command

Co-pay Assistance Programs & Concerns – Myeloma

This video was originally published by Myeloma.org on Mar 31, 2017, here.

Recently, the International Myeloma Foundation’s InfoLine has been flooded with calls from patients and caregivers facing insurmountable financial challenges due to the high cost of drugs and increases in insurance copays. In the past, several services have been available to provide financial assistance to patients. We understand that funds for co-pay assistance are difficult to come by, and available funding will likely dry up quickly.

DOWNLOAD SLIDES

New Developments in Myeloma Therapy

This podcast was originally published by The Cancer Cast with Weill Cornell here.

 

 

Adriana Rossi, MD – Speaker Bio

Know What Your Doctors Know: Multiple Myeloma

This video was originally published by the National Comprehensive Cancer Network on April 15, 2019, here.

Experts discussed updates in the treatment of multiple myeloma during live webinars hosted by the National Comprehensive Cancer Network (NCCN) on April 15 and 16, 2019.

Essential Tests for Diagnosis and Monitoring

This resource was originally published by International Myeloma Foundation here.

Multiple myeloma tests are diverse and complex. Tests are used throughout the course of your disease — for initial diagnosis, discovering the type of myeloma you have, staging the disease, and for monitoring your response to treatment.

What Can Tests Tell Us?

Tests results are the most important tools that your doctor will use to:

  • Diagnose monoclonal gammopathy of undetermined significance (MGUS), smoldering multiple myeloma (SMM), and active myeloma.
  • Assess the risk of progression of MGUS or SMM to active myeloma.
  • Assess the stage of your myeloma.
  • Assess your genetic risk factors.
  • Evaluate your response to treatment.
  • Monitor remission periods and determine when to start treatment again.
  • Monitor for disease-related and treatment-related side effects.

Tests for multiple myeloma patients fall into three major categories:

  • Blood and urine tests
  • Bone marrow tests
  • Imaging studies

No Single Test Tells the Whole Story

Each test can be thought of a piece of puzzle. Only when the pieces are assembled together can a patient and their doctor make their proper conclusions and decisions. Myeloma is unique in each patient; therefore, it’s important not to compare your test results with other myeloma patients.

We encourage you to discuss all your test results with the doctor who is treating you. Your doctor will be able to put all the puzzle pieces together to put your results in context. The patterns of results viewed over time are more meaningful than any single test itself.

Save Copies of Your Test and Lab Results

It is important to request, print out, and save copies of your results. Bring a summary of results with you when you to all oncology visits or when getting a second opinion.

Variables That May Affect Your Test Results

Be aware that your lab results can be affected by many variables, including:

  • other medications and supplements that you may be taking
  • the amount and type of fluids you have consumed
  • whether you have eaten before the test

Before undergoing tests, consult with your doctor to make sure there are no special instructions about taking certain medications, supplements, food, or drinks.

Deciphering My Lab Results

This resource was originally published by Myeloma Crowd here.

We thought it might be helpful to have a document that describes your multiple myeloma test lab results and helps identify the important markers. This document contains a diagnosis summary checklist, a treatment summary form and each lab result described and color-coded for relevance.

 

Download It Here:

How is Multiple Myeloma Diagnosed? Symptoms, Prognosis, Causes, Treatment, and More

This resource was originally published by Everyday Health by Pamela Kaufman here.

Last Updated: 10/11/2018

Multiple myeloma is a rare cancer that affects plasma cells.

Plasma cells, a type of white blood cell, are found primarily in bone marrow — the soft, inner tissue of bones.

Plasma cells are an important part of the immune system. They produce proteins called antibodies that help the body fight infection by attacking and killing germs.

When plasma cells grow out of control and begin to collect in bone marrow in different parts of the body, the result is multiple myeloma.

Multiple Myeloma Complications

Multiple myeloma can cause a number of complications, some of which may be very serious. These include:

  • Low blood counts As myeloma cells multiply in bone marrow, they crowd out red blood cells, white blood cells, and platelets. Low red blood cell levels (anemia) can result in fatigue and weakness. A drop in white blood cell count can decrease resistance to infection. Lack of platelets can lead to significant bruising and bleeding even from minor scrapes or cuts.
  • Bone and calcium issues Myeloma cells produce substances that speed the breakdown of old bone and slow the buildup of new bone. This leads to holes in bones and osteoporosis (low bone density), raising the risk of fractures. Even normal activities like coughing and walking may lead to a broken bone.
  • Myeloma cells not only reduce the number of infection-fighting white blood cells, they produce a single type of abnormal antibody that does not fight infection. This antibody is referred to by a number of names: monoclonal immunoglobulin, monoclonal protein, M protein, M-spike, and paraprotein.
  • Kidney damage The antibody produced by myeloma cells can build up in the blood and urine, leading to kidney problems. (1)

How Many People Get Multiple Myeloma?

According to the most recent statistics from the National Cancer Institute, almost 31,000 Americans will receive a multiple myeloma diagnosis in 2018.

That makes multiple myeloma relatively rare, accounting for 1.8 percent of all new cancer cases.

Around 0.8 percent of men and women in the U.S. will be diagnosed with multiple myeloma at some point during their lives.

The number of people diagnosed with multiple myeloma has climbed an average of 0.9 percent each year over the course of a decade, according to the most recent data.

But death rates from multiple myeloma have decreased 0.5 percent per year, reflecting improvements in treatment. (2)

What Causes Multiple Myeloma and What Are the Risk Factors?

Scientists still don’t know what causes multiple myeloma or how to prevent it. For most people who develop the disease there are no clear reasons why.

Researchers have made progress understanding the DNA mutations (changes) that turn healthy plasma cells cancerous. This is vital work that many hope will point the way to improved treatment.

Researchers have identified a number of factors associated with increased multiple-myeloma risk.

These include:

  • Age Multiple myeloma is most often diagnosed in adults over the age of 65.
  • Gender Men are at a slightly higher risk than women.
  • Race African-Americans are almost twice as likely to get multiple myeloma as white Americans.
  • Family history Having a close relative with multiple myeloma increases a person’s odds of developing it. Is multiple myeloma hereditary? Sometimes. But most people with multiple myeloma have no family history of the disease.
  • Obesity People who are overweight or obese are at higher risk.
  • Other plasma cell diseases Men and women with a relatively benign condition called MGUS (monoclonal gammopathy of undetermined significance) should be vigilant: Every year 1 percent of these people go on to develop multiple myeloma. Patients with a type of blood cancer called solitary plasmacytoma also have a greater chance of developing multiple myeloma. (1)

The Signs and Symptoms of Multiple Myeloma

When symptoms of multiple myeloma do occur, they may include:

  • Bone pain, most often in the back or ribs (although pain can occur in any bone)
  • Bone fractures
  • Fatigue
  • Weakness
  • Frequent infections
  • Numbness, tingling, burning, or pain in the hands and feet
  • Excessive thirst and urination
  • Constipation
  • Abnormal bleeding
  • Headaches
  • Chest pain
  • Decreased alertness
  • Shortness of breath

Doctors sometimes use the acronym CRAB to describe myeloma symptoms:

  • C — calcium elevation (high levels of calcium in the blood)
  • R — renal insufficiency (poor kidney function)
  • A — anemia (low red blood cell count)
  • B — bone abnormalities (lesions) (3)

 

Learn More About the Symptoms of Multiple Myeloma

How Multiple Myeloma Is Detected and Diagnosed

In order to make a multiple myeloma diagnosis, a medical team has a lot to consider: the patient’s symptoms, the results of a physical exam, and the findings of a battery of tests. Doctors look for telltale substances in blood and urine (calcium and certain kinds of proteins and antibodies), analyze samples of bone marrow and bone, and search for signs of disease in imaging tests such as X-raysCT scans, and MRIs. Whatever else doctors find, a myeloma diagnosis will always involve a tumor made up of myeloma cells or at least a high concentration (at least 10 percent) of these cells in the bone marrow.

In about a fifth of cases, doctors detect multiple myeloma during routine physical exams in patients without any outward signs of the disease. (5)

A diagnosis of asymptomatic multiple myeloma, also called smoldering multiple myeloma, does not typically lead to immediate treatment. Instead physicians often choose to take a watch-and-wait approach, performing regular blood and urine tests and offering medication or other therapies only when the cancer advances. (5)

Learn More About Diagnosing Multiple Myeloma

The Stages of Multiple Myeloma and What They Mean

Staging multiple myeloma is the method doctors use to assess how much cancer is in the body and where it is. Doctors stage the disease in order to come up with a treatment plan and a prognosis (a prediction of the course of the illness).

Staging may initially involve categorizing the cancer as symptomatic or asymptomatic, since patients in the first category generally don’t receive treatment and can remain stable for many years. Doctors may also categorize the cancer as stage 1, stage 2 or stage 3 using the Revised International Staging System (RISS). The RISS looks at a number of factors, such as blood levels of beta-2 microglobulin — a protein produced by myeloma cells. (6)

Learn More About the Stages of Multiple Myeloma

Is Multiple Myeloma Curable?

Multiple myeloma has long been considered an incurable disease.

Throughout the 1990s, the average life expectancy for people with multiple myeloma was three years. Advances in medicine since then have increased median survival to more than five years, while some patients are pushing that number to upward of 10 years. (7)

A small subset of patients have been known to live in remission for decades. Are they “cured”? Doctors say it might be possible, although they remain highly cautious about making more definitive claims.

Meanwhile, new treatments are coming down the pike that hold promise to anyone facing a multiple myeloma diagnosis. These treatments aim to relieve symptoms, provide prolonged remissions, and increase the multiple myeloma survival rate.

Multiple Myeloma Treatments: Drug Therapy, Stem Cell Transplants, and More

Oncologists have many weapons in the fight against multiple myeloma. Treatment options include:

  • Chemotherapy and drug therapy, including targeted drugs that home in on specific genes or proteins in the cancer
  • Stem cell transplantation (in which cancerous cells are replaced with healthy cells) combined with high-dose chemotherapy
  • Radiation therapy
  • Supportive care to relieve symptoms
  • Clinical trials of new drugs, drug combinations, or stem cell transplant approaches

The rapid pace of research means that more and more treatments are in the pipeline. Among the most promising new treatments under investigation are those involving a type of immunotherapy called CAR T-cell therapy. CAR T-cell treatments involve genetically engineering a patient’s own T cells (a type of white blood cell) to allow them to recognize and attack cancer cells.

For every type of approach, outcomes for people with multiple myeloma depend on a number of factors, including overall health and ability to tolerate treatment.

Quality of care also makes a difference. The Leukemia and Lymphoma Society emphasizes the importance of seeking out a doctor with experience treating multiple myeloma or one who will work in concert with a specialist. These specialists are usually called hematologist oncologists. (8)

Learn More About Treatment for Multiple Myeloma

Prognosis: What to Expect After Being Diagnosed

The median five-year survival rate (the percentage of people who live at least five years after diagnosis) for multiple myeloma is a little more than 50 percent. (9) But that doesn’t tell the whole story of what a person with this rare cancer of the plasma cells may experience. Life expectancy and quality of life depend on a number of factors, including a person’s age and overall health. The type of multiple myeloma also makes a difference. People with the early kind called smoldering multiple myeloma may remain symptom-free for many years before the disease begins to take a toll. Conversely, patients who have a form of myeloma with a high-risk genetic feature may have an especially poor prognosis.

Learn More About Prognosis for Multiple Myeloma

Life After Multiple Myeloma Treatment

With multiple myeloma there’s no simple way to define “survivorship.” While this type of cancer is considered incurable, some patients consider themselves to be survivors if they have no signs of disease after treatment. Others call themselves survivors because they’re committed to pursuing treatment over the long term and doing whatever they can to prevent recurrence.

Managing a mix of powerful emotions — fear, hope, guilt, joy — is part of life as a multiple myeloma survivor. An in-person support group, online community, and individual counseling can help patients understand complicated feelings.

For patients who regard their illness as motivation to make positive changes in their life, survivorship can involve eating well, not smoking, limiting alcohol and managing stress. It can also entail returning for regular medical checkups and tests as well as services such as physical therapy, nutritional planning, and pain management. (10)

Additional Resources for Multiple Myeloma

If you’ve been diagnosed with multiple myeloma, there are a number of excellent resources that can help you understand and cope with your condition. Click on the link below for more information on organizations, blogs, and websites that provide a wealth of information as well as medical, emotional, and financial support for people with multiple myeloma.

Learn More About Resources for Multiple Myeloma

 

.

Multiple Myeloma

This podcast was originally published by City of Hope Radio here.

Gargi Upadhyaya, MD, FACP – Speaker Bio
  • Topic Info: Myeloma is the second most common type of blood cancer, accounting for around one percent of blood cancer cases. It develops in plasma cells, white blood cells that grow in bone marrow. Myeloma most often affects the aged — most cases are diagnosed in people age 65 and older. Although myeloma grows within bone, it is not considered bone cancer.

    Listen as Gargi Upadhyaya, MD discusses multiple myeloma and the treatment options at City of Hope.

Resources For Survivors

This resource was originally published on Bone Marrow and Cancer Foundation here.

The Journey Continues

The Bone Marrow & Cancer Foundation’s Survivorship Program provides resources that can address the needs of all bone marrow, stem cell, and cord blood transplant survivors, their families, and caregivers. Our goal is to provide education and support for people coping with the physical and emotional challenges of transplantation. Web accessibility to many of these resources means that no matter if you are at home, at a treatment center, or staying in out-patient lodging immediately following discharge, you are not alone; the survivor community is at your fingertips. The website will be an interactive community that serves as a meeting place and a shared resource for those who have survived a transplant and their families.

Transplant survivors tell us that while they felt well-prepared for transplant, many were very isolated in the days, weeks, and even months following transplant. The return to “normal” life takes a different path for each person; yet the shared common experiences can provide significant support and encouragement during the process. The Bone Marrow & Cancer Foundation’s Survivorship Program will address the ongoing need for emotional and social support, provide education about transplant and side effect related issues, host online discussion forums about social, physical, and psychological concerns, and help you create a healthy new life.

Survivor Telephone Support Group

Survivor Telephone Support Group staffed by oncology social workers, provides bone marrow, stem cell and cord blood transplant survivors with a weekly scheduled telephone conference support group to share experiences and draw support from others. For patients one year or more post-transplant. For more information or to register, contact the Bone Marrow & Cancer Foundation at patientservices@bonemarrow.org or 1-800-365-1336.

Resources for Patients and Families

The Foundation offers several programs, such as Ask the Expert and SupportLine to help patients and their families make the connections they need and resources to find information to help allay their fears and better understand the challenges they face.

Financial Assistance: Filling in the gaps

This blog was originally published on Bone Marrow & Cancer Foundation here.

Lifeline Fund

The Lifeline Fund provides support to patients who often lack the financial resources necessary to afford the often overlooked living and ancillary expenses that can present considerable financial obstacles—or prohibit the transplant altogether. Funding from the Bone Marrow & Cancer Foundation’s Lifeline Fund helps to cover the myriad costs associated with transplants, such as donor searches, compatibility testing, bone marrow harvesting, medications, home and childcare services, medical equipment, transportation, cord blood banking, housing, and other expenses associated with the transplant. Health insurance often does not cover these vital support services, and many patients cannot afford them on their own.

Open Homes Medical Stays

Our partnership with Airbnb’s Open Homes Medical Stays program provides free temporary accommodations to patients diagnosed with any form of cancer or undergoing a hematopoietic stem cell transplant, as well as housing for their caregivers, family members, and donors.

One-to-One Funds

A One-to-One Fund is a personal fund created for a specific patient. It is a simple and effective way for a patient’s family, friends, and community to raise money on a patient’s behalf with all money raised going directly to their benefit. All donations are tax-deductible. The Bone Marrow & Cancer Foundation administers and maintains the fund, and can offer support with fundraising ideas.

Scholarship Grants

Scholarship Grants help make educational aspirations a reality for bone marrow, stem cell and cord blood transplant survivors. Transplants affect all aspects of life and often prohibit the pursuit of educational opportunities. To address this, the Bone Marrow & Cancer Foundation offers survivors support towards an academic future so their hopes and dreams remain intact. Scholarship Grants support students of all ages as they pursue their educational goals.

Get The Best Myeloma Care NOW: A Physician’s View

Get The Best Myeloma Care NOW: A Physician’s View from Patient Empowerment Network on Vimeo.

Advocating for yourself is critical when diagnosed with multiple myeloma. Dr. Peter Forsberg details the value of collaborating with your healthcare team on treatment decisions.

Dr. Peter Forsberg is assistant professor of medicine at the University of Colorado School of Medicine and is a specialist in multiple myeloma. More about Dr. Forsberg here.

See More From The Pro-Active Myeloma Patient Toolkit

Related Resources

Overwhelmed By a Myeloma Diagnosis? The Key Steps to Take

Should You Consider a Second Opinion? Advice from a Myeloma Advocate 

Find Your Voice Myeloma Resource Guide

Transcript:

Patient education and self-advocacy I think are critical in multiple myeloma. Myeloma is a complicated disease. Getting your head around it can be challenging. Beyond that we have more and more treatments. Treatments are fairly complex. Our goals can be pretty different patient to patient. So really, patient education can be a key to understanding that and removing layers of complexity from something that can be a little challenging to get into.

I think self-advocacy is also really important in that, sometimes you can feel swept up into a wave of what the next treatments are gonna be, what the next steps are. So, making sure you’re taking time to voice your opinions or concerns for yourself, to make sure that you’re not leaving stones unturned in terms of what your best options are, what the best next steps are, what treatments or testing might be available.

I think myeloma, maybe more so than even some other diseases because it’s such a unique type of cancer, one where patients are often dealing with it for many years… Making sure that there’s a good level of education that evolves over time can help make sure that the patients get the best out of their treatments; to make sure that they’re able to have the most fulfilling experience dealing with their cancer and with their cancer team, and making sure that they’re advocating to get all options available to them in the mix potentially.

I think patients are often very thoughtful about knowing that providers are busy and that clinic can be kind of fast-paced, but I want to make sure that they know that the last thing that they’re ever doing is bothering me or other members of my team when they ask questions. I think one of the keys to making sure that everybody is comfortable with the steps we’re taking with their myeloma is to recognize that it’s a team. And the patients and myself and other members of my team, you know I think that the goal is for all of us to be on the same page and to understand what we’re working towards.

So, I think that my philosophy about how best to take care of patients tis to try to make it as collaborative as possible. To make sure people understand what we’re doing and why. And to be all on the same page I think you have to feel comfortable to take a moment to say, “Why are we doing this?” or to voice concerns about what’s going on or what the next steps might be.

Is It Difficult to Participate in a Clinical Trial?

Clinical Trial Mythbusters

 

Downloadable Program Guide

Three experts discuss the clinical trial process and the difficulty in participating in a trial. Our expert panel includes:

  • Ken Getz, MBA – Founder and Board Chair, CISCRP
  • Andy Lee – Senior Vice President and Head, Global Clinical Trial Operations, Merck
  • T.J. Sharpe – Melanoma Survivor and Patient Advocate

Transcript:

Andrew Schorr:

And greetings from Southern California. I’m Andrew Schorr from Patient Power. And welcome to this Patient Empowerment Network program, another in our series of Clinical Trials MythBusters. Our goal, of course, is to help you get the treatment for you or a loved one that you need and deserve. I want to thank the financial supporters for this program to the Patient Empowerment Network; AbbVie, Inc., Celgene Corporation. Daiichi Sankyo and Novartis for their support. They have no editorial control and we’re going to have a very freewheeling discussion today. And really what it’s about is how can a clinical trial be made easier for you to participate? Are there barriers? We’ve talked about it in previous programs. But specifically, what are the companies—the pharmaceutical industry mostly, who sponsor trials all around the world, what are they doing to make trial participation easier? For you to know about trials. For the people at your clinic to know about it and what to say and how to administer it. For you to have documents that are understandable for you and your family to know whether you want to participate. To keep you informed. And also related to the requirements of trials. How can they be relaxed a little so that there may be a trial that would benefit you, that you and your doctor agree on, and the requirements of it allow you to be in the trial. Okay, and the logistics of it are not so tough either. All right, I’ve been in two clinical trials, and I believe I’m alive today because of that. So, I’m very grateful. We have some wonderful panelists with us over the next hour. Now as you have questions, send them to questions@patientpower.info. And some of you have. So, you’ll be able to interact with us as we go along. First, I want to go to Ft. Lauderdale, Florida, and T.J Sharpe. And T.J. has been on programs with me over the years. Stage four melanoma patient having been in trials. And T. J., you would agree, you’re alive today because you were in trials, right?

T.J. Sharpe:

Absolutely, Andrew. I think both of us are very fortunate that we found a trial that was the right treatment for us and gave us the ability to combat our disease in areas may not have been available to us if we just waited for standard of care therapies.

Andrew Schorr:

Right. And here you are—we should say that you were diagnosed a number of years ago with melanoma, went through trials. And now you’ve had two years without treatment, right?

T.J. Sharpe:

Yes. It’s been five years of treatment followed by now two good years of a clean bill of health.

Andrew Schorr:

Well, great. And I should mention for our audience, many people are familiar with T.J. T.J. goes around the country, gives speeches. He’s been at many events, consults with industry that are developing trials to try to bring the patient perspective forward. So, T.J., thank you for all you do. We really appreciate it.

T.J. Sharpe:

You’re welcome. It’s my honor to be able to represent all these patients.

Andrew Schorr:
Well, most every family—certainly most have been touched by cancer. But our other guests are not cancer patients but are in national leadership programs. And so, let’s go up to Medford, Massachusetts at Tufts University outside Boston, Ken Getz. Ken, welcome to the program. Ken, ladies and gentlemen, is a true national leader when it comes to clinical trials and really helping us move forward with better processes, better understanding. Ken, tell us a little bit about your organization there, CISCRP. What does that stand for?

Ken Getz:

Thank you. And I have to say your pronunciation was nearly perfect. It’s hard to pronounce it. It’s an acronym and it stands for The Center for Information and Study on Clinical Research Participation. It’s a non-profit organization. It was founded 18 years ago. And it’s really there to help patients and their families navigate the whole clinical research progress which for many is completely unfamiliar terrain until they’ve been diagnosed with an illness or when they have exhausted all other treatment options. So, CISCRP really helps people become more educated and informed so that they can really think of the clinical research process with more confidence. And they can navigate this unknown terrain.

Andrew Schorr:

All right. I’m going to come back to you in a minute because you have such an overview, and you’re also an Associate Professor at Tufts. And so, you study all this, and you’ve written books. But I want to introduce the third guest. And that is a leader from the pharmaceutical industry and one of our most respected and venerable companies in the field, and this is Merck. So, joining us in a senior vice president of clinical operations there around the world. And that’s Andy Lee. Andy, welcome. Thank you so much for being with us.

Andy Lee:

Andrew, thank you. And pleasure to be with some prestigious panelists, both of whom I know. And I’ve met you over the last two weeks. And thank you to T.J. and yourself who have been trial participants and who are representing that part of the organization.

Andrew Schorr:

Okay, and we should mention that both T.J. and Andy are working on a couple of levels. And Ken sounds off on this too. There is a group called TransCelerate where pharmaceutical industry is working together on some of the issues they face in having the proliferation of trials. More trials sites, more accessibility, procedures for that. And then, of course, Andy has helped lead that effort at Merck related to breakthrough therapies that they have been trying to develop there in supporting patients who might be in Merck trials. So, we are going to come back to that. But I want to go to you for a second, Ken. Ken, how low is the participation among adults in clinical trials, at least in the U.S. Now, I’ve heard really low percentages. Where are we now with that?

Ken Getz:

Right, it’s a great myth for us to start with, this notion that only three to five percent of patients—eligible patients, participate in clinical research. That’s actually a statistic that was published by the National Cancer Institute in the early 1990’s. The latest research really shows that it varies widely. For example, when we look at pediatric cancers, the participation rates are extremely high, 80 to 90 percent in same cases—pediatric leukemia. In part because those communities have very engaged healthcare providers, very engaged families that really share their information. It’s just an enabled community where all of the stakeholders support participation. And then there are other areas of course. Some cancers where we do see relatively low participation rates. But I want to point out that low participation is driven by so many factors, Andy, including the strict eligibility criteria. And the demanding protocol designs which are a real burden for some people, and they choose not to participate. As well as low awareness, very low accessibility to trials among minorities and underserved communities. So, there are many factors that contribute to this variation in the participation rates.

Andrew Schorr:

Yeah, you’ve ticked off some now. T.J., in your own experience, one of the breakthrough trials you were in you had to go from Ft. Lauderdale in South Florida and move your whole family to Tampa in central Florida, right. I mean that was a big deal.

T.J. Sharpe:

Absolutely. When you have a young family and a stage four cancer diagnosis, relocating simply across the state during the holidays especially, is no big deal. We were fortunate because we had the means to be able to move there with work situation, with family. But too many people can barely go across the county, much less the state or the country to find a trial that might be the best match for them.

Andrew Schorr:

Andy, so we’ve ticked off some of the obstacles, and Ken touched on some about even the proliferation of trials. Is that a lot of what you do is how can we have trials be more accessible, be more widely distributed to a clinic near you?

Andy Lee:

Yes, let me just explain. When we look at a new cancer therapy, we look at the various cancers that may be affected. And what we do is we go for high probabilities of success. And the challenge is if you bring a new cancer agent. You normally start off in very advanced disease. So, patients would have failed multiple lines of therapy, and often it is a last gasp. And you have to show some sort of clinical efficacy. And then you move sort of backwards in the disease, and you go from sort of third-plus line, second line and first line.

And then you may work downwards into earlier stages of the disease into an adjuvant setting and maybe a neoadjuvant setting. So, as we sit down and design a trial, what we need to look at is what is the population that is most likely to show any benefit at all. And quite often when you are developing a new therapy, it’s difficult to show benefit because many of the patients are very ill. So, what we have to do is optimize the opportunity for success of a compound by going to the right target patients.

And quite often as we have learned a lot more about cancer, this does not mean we test a product broadly in anyone with cancer. We typically try and find a profile of a patient that is likely to respond. And many patients now will realize their predictor biomarkers or prognostic biomarkers. So, for example, with immunotherapies, those that work through the PD1 mechanism would probably want to have a PD1 ligand receptor positive patient who is likely to bind to the drug.

And that gives a higher probability of success. So, it sounds counterintuitive that while we want to develop therapies for all cancer patients, when we start clinical trial development, we have to show efficacy in a population that will benefit. And that’s normally predefined and makes the inclusion criteria fairly strict. As we show efficacy and as we can move into broader populations, it makes it a lot easier for us to design more liberal clinical trials. And then we can actually spread those in the geographic domains.

I could talk more about geographic allocation, but let’s hold that for the time being, and let’s see if there’s time later on.

Ken Getz:

Can I just add to what Andy said because I think it’s really important for your viewers to understand just how active drug development activity is today. We’re looking at over 4,000 pharmaceutical and biotechnology companies, some of them very, very small. But in total, we’re looking at nearly 6,000 drugs that are in active clinical trials. And to Andy’s point, many are really targeting a patient with a very specific genetic profile or a specific biomarker. But it should give anyone who believes that a clinical trial may be an important care option for them, they should recognize that there may be many, many trials out there.

In total we estimate as many as 80,000 clinical trials, nearly 50 just conducted in the U.S. alone—50,000. So, it’s just important that we keep all this activity in perspective.

Andrew Schorr:
Right. So, T.J., that’s why all of us as patients need to ask about them, right? Go to different resources, whether it’s an advocacy group that you ultimately spoke with other patients, and obviously quizzing the doctors we go to. Is there something that may line up with my situation, right T.J.?

T.J. Sharpe:

Absolutely. There is a both top down and bottom up approach here that patients as they become educated—and every patient should be the owner of their healthcare as they become educated. Hopefully they are coming across advocacy organizations, other informed patients, patient support groups—all of which will help inform them different options for disease treatment, including hopefully as Ken mentioned, clinical research as a care option. At the same time, there is certainly very much an opportunity from the top down from the sponsors who develop the trials and from the sites that execute them to educate patients as they come in.

Not just at their own site, but at any site, at any medical facility. That if you have a diagnosis and you are looking into your care options, that you should be asking the question. And we should be giving you more information on the possibility of clinical trials and where you may find clinical trials that are appropriate for you.

Andrew Schorr:

Right, the whole enchilada, if you will, of all your options. Andy, so you mentioned about trial requirements. So, first of all, what efforts either at Merck or are you aware in the industry are being made to really talk to patients early on as you are designing trials? Whether it’s the requirements—how many CT scans you’re going to have. How often you are going to have to go to the main trial site. All the different things that sometimes get in the way.

Andy Lee:

Well, firstly we start with design. And we believe in exquisite trial design, quality by design as well. So, what we want is to run the experiment once and not have a sloppy trial design. We want to make it really robust in terms of scientific integrity and operational execution. So, we have a lot of internal design committees and what we do is we co-op with many groups external to our company. So, we speak to people who run clinical trials at cancer institutes.

We speak to the doctors who manage this. We speak to the trial coordinators. We speak to people involved with the transporting and shipping of medicine how they would do that. And then we of course speak to people in the ecosystem. We quite often speak to investigational review boards before we start trials. We talk to them about our design and what would be best to protect the rights and well-being of patients. And then, of course, the patient-centric approach says that we need patient insights.

And I’ve chosen my words very carefully because the insights are really important. Not all patients—and I’m very respectful that some patients are very intelligent and actually may be involved in this. Some patients can contribute to design, not all can. And so, what we do is we take the insights and we impute those. We often have focus groups. We talk about this disease. We talk about the burden of the disease. And then we talk about how that disease is managed in an ecosystem. And quite often in different countries it’s managed differently.

And so, we have to appreciate the global clinical trials have to navigate a path that may not be a linear path as we’d see it at an exquisite elite cancer center in the United States. It’s community-based, it’s all the rest. So, we take that input, and what we try to do is unburden the trial for the patient. We say, “How can we design a trial that requires the least visits to the clinic—the hospital, the least burden for them. And how can we take some of that burden from the clinic and actually transfer that into an easier environment.

So, document reading and review. Perhaps filling in questionnaires about quality of life. These are things that don’t have to be done in the clinic itself. And then often when we work with clinics, we work with them to help them understand how we as sponsors can make their life easier. And some of those things might be simplifying the informed consent. But I want to stress just one point here is that we can do whatever we like in the design at a company.

One of the things is, the patients are not sponsor patients. Okay, we sponsor clinical trials. The patients are managed by a doctor and a professional. And underneath that principal investigator is a whole oncology team. And it involves radiology. It involves pharmacists. It revolves around a 360 multidisciplinary team. They’re exquisite. They help manage the patient, not the sponsor. We provide the enabling functions for them. And then also that the oversight of the patient’s right, safety and wellbeing is the responsibility of an institutional review board.

And while we may provide templates and simplify templates in text and language, we rely heavily on the institutional review boards to help us with things that may make things easier, such as reimbursement for parking, transport, all of these things. And by and large, the institutional review boards are very supportive of these things. But they are very difficult to quantify in exact terms because of different geographic regions and different norms in different places. So, we rely heavily on exquisitely well-trained 360 team who manages oncology patients with a great PI. They manage patients.

And we work collaboratively with the sites who work with patients on our behalf. So, I just wanted to say the myth is that sponsors interactive with patients. That’s a myth. And the truth is that we engage with clinical sites, and we try and make our design and all the elements—the enabling elements, simpler for the trial sites in order to manage the patients in a simpler way.

Andrew Schorr:

Okay. Thank you for that. So, Ken, I want your comment on that. Because okay, we are downstream patients. We have a doctor, healthcare team. And we know somewhere in the background there’s a sponsor that tried to enable good things to happen to get reliable data and hopefully a cure for us. So, how do we—what’s happening? Are we improving things there in that interaction between clinic and patient?

Ken Getz:

Yes, we absolutely are. And I’ll start by just echoing and acknowledging that Andy has really laid out just an incredible amount of input that goes into the design of a protocol. And that’s really for a really large company. We see many, many examples now of patient advocacy groups or smaller companies turning to a variety of approaches to solicit input from patients and healthcare providers. Some virtual approaches through a social media or digital community. So, there’s lots of ways that feedback is being channeled.

And that’s really important. The flip side, to really answer your question, is that our protocol designs are becoming more and more complex, more and more demanding. A much larger proportion of drugs are now targeting rare diseases that have been stricter inclusion and exclusion criteria. And the designs of the studies—the number of procedures and the number of visits. The number of investigators that are involved, all of that has also continued to grow. And as a result, we do see that our trials are taking longer.

We have yet to see a year when we actually witnessed a reduction in the cycle time to conduct a clinical trial. And we just have to figure out new ways of making the participation process less burdensome and more efficient.

Andrew Schorr:

Oh, my. So, T.J., you had been living with stage four melanoma, a life-threatening condition. We have people even on our team who are living with stage four disease. So, when Ken talks about things slowing, that’s not what we want to hear. We want to hear two things. One is, we can accelerate a development of new medicine. And ideally—because this is an issue certainly in the U.S., but I think worldwide, that by speeding the process, cutting through red tape, improving procedures and us participating, the cost can be less as well.

And when we talk about cancer, the costs are going through the roof as you know for people living with chronic cancer. And you know so well, Andy, people who are on some of the medicines that you’ve come out with at Merck. Where people used to die unfortunately in short order, are living a much longer life thanks to new medicines. We want it to happen faster and be financially achievable. Andy, any comment about the pace of science?

Andy Lee:

Yeah, I would like to make a couple of comments about that. We often hear the sort of story that 80 percent of clinical trials don’t recruit on time, et cetera. We do immense feasibility. Once we have designed a protocol, we send it out to all of the countries that could potentially work with us. We have staff in 47 countries. And they look at two areas of interest. One is the medical durability, is the comparator the one we use in our country. Is the protocol designed the way we practice clinical medicine, not clinical research medicine?

And will that enable us to recruit the patients? That’s the first level. The second level we look at is to ask the question, is this operationally feasible? Can we source the comparator? Do the clinical sites have the equipment? How would we have to ship the biological samples around the world? And based on medical durability and the operational durability, we do a site selection. And we run the indicators through a Monte Carlo simulation. And we simulate this trial. What if we took three countries out? What if we added this more sites? What if we changed this inclusion?

And we come up with a model of what the recruitment would look like. And recruit about 80 percent of our trials according to our model. So, about 80 percent of our trials recruit on our model time. And then if we look at the typical time for drug development, it has been from eight to 10 years for many years in the industry. And when we look at some of the development timelines now—the cycle times. Pembrolizumab (Keytruda), for example, from first study until first approval, was 60 percent reduction in time.

We were looking in the four-year time period. And we are looking at five or six years for many indications. And so, we’ve halved that cycle time for some of the newer oncology products. And there are a number of reasons we’ve done that. One is we have found operational efficiencies. Two is the trial design has enabled us to interim analysis with independent data monitoring committees to assist with that. I’d also like to put in a positive plug for the regulators.

I do believe—and I’ll talk specifically about the FDA, because they are the agency for the United States. They have revolutionized the way they approach the designs and the way they review the data. And they have breakthrough designation status they’ll give to compounds that are really looking like they have strong efficacy. And so, the approval process through the agency has improved remarkably. And they’re open to adaptive designs. And they are open to interim analysis. And they are open to all sorts of things.

So, I really wanted to give credit to our agency who has said, “Where there’s a need for breakthrough medications, we’ll try to find the path.” And so, I do believe there’s a real positive side to this. The challenge is the market is saturated. We have now more than 25 PD1s in development. And to put the 25th one in there, they are so far behind in development. I wonder what that does. It clogs up the system. So, when you look at how can we influence sites, at the top sites we only get one or two patients.

And we compete with 50, 60, 70, 80 other sponsors. And so, it becomes so saturated that, that site has to learn to do systems and process with 70 companies. And what they are doing is almost hedging. They are not focusing on certain things. So, in those cancer centers, they offer treatment for all lines of therapy and all types of cancer, the specialized and nonspecialized. And we are moving out of that sort of geography and moving it community-based oncology practices where it’s less saturated, and we can actually have more traction there and be able to engage more with the clinical trial enterprise for the good of the patients.

Andrew Schorr:

Ken, you write books about all kinds of issues around this. So, if we are getting—particularly in oncology to have trials offered at the community practice where those doctors work night and day—the nurses. They are really stretched. More and more cancers, genomic subtypes, most sophisticated testing. How—what would you say the patient can do. T.J. talked about it a little bit. What would you recommend to patients so that at that community oncology practice the patient and the family can kind of discover what may be available for them as Merck and other companies try to get these trials distributed?

Ken Getz:
Right, well you—talk about the whole enchilada, Andrew. You’re really touching on it. It’s also very exciting times for patients, not just cancer patients, but patients that are dealing with any chronic and severe illness today. And it’s really all about more of a partnership with the clinical care environment and clinical research. And of course, at the heart of it is the patients and their family being as informed as possible, sharing their electronic health and medical information so that they can be connected to trials that might be appropriate for them.

But it’s moving—as Andy said, away from the classic places where trials used to be conducted. And in many cases, they were at these dedicated centers that only conducted clinical trials. It’s a very competitive environment now for patients. So, many sponsor companies like Merck and others are looking at clinical care settings and moving into communities or, in some cases, large health systems where you can have clinical research professionals who will supplement and provide support to the healthcare providers, so they’re not stretched too thin.

But so that they have the clinical research capability onsite at the point of care. For patients it’s a great opportunity because now they have the opportunity to get their own healthcare or treating physician and treating nurse involved in a clinical trial as part of their overall care. And we expect to see more of that over time. We expect to see other virtual trials or opportunities for patients to participate in the comfort of their own home tied in with their clinical care setting.

And all of this is relatively new to the whole world of clinical trials and the investigation of experimental medications.

Andrew Schorr:

You touched on something I just want to follow up on. I’ve heard of this term site-less trials where you said you participate in your home. So, T.J. had to go from Ft. Lauderdale to Tampa. I had to go from Seattle to Houston. There are not—this is a big deal, especially if you have little kids as I did, he has. So—and away from work and whatever your situation is. So, is technology going to come in play so Andy can get the data he needs for the FDA, but that we can have technology help accrue that data in a more efficient way.

Ken Getz:

And I’ll say absolutely. And my colleagues here today I’m sure can comment on this as well. But absolutely. We are seeing wearable technologies and mobile applications that now have the ability measure vital signs and other important baseline information in a validated manner. There are ways that you can access a specific facility for a highly specialized test, specialized imaging for example where the technician can evaluate it remotely. Blood can be drawn at remote locations as well.

So, there are lots of places where we have sort of this more flexible environment that can cater more to the patients and less about a specific physical facility where you have to go to participate in a trial.

Andrew Schorr:
T.J., I want to talk to you about diversity. So, you and I are kind of middle-class white guys. But we want to know how new medicines work for a variety of populations, ethnically, economic groups, et cetera. And Andy needs that data. And he goes to the FDA, and the FDA says, “Well, do you have Hispanic people? Do you have Asian people? Do you have African American people?” or whatever the country is because he works globally. And they say, “We want to understand are there differences?”

How are we doing with that. How can we make a difference there so that we really know what medicines make a difference for broader and also distinct populations?

T.J. Sharpe:
I’m sure Ken can back up some of these things with more hard data than I can. I know that different populations have different levels of trust with the medical system. One thing that you and I both experienced was a lack of options—a lack of good options. And when you get into dire straits, you tend to be a little more trustful of anything that comes along. But we have serious or chronic conditions that have proven treatments that might not be the most effective for certain populations.

And we’re not able to broad the scope to these minority populations or populations that don’t have access to NCI designated cancer centers or top-notch medical facilities. They are not able to get either in a trial that is looking for a drug that would help them or even get access to medicines that have been recently approved simply because their healthcare situation doesn’t allow it. Whether that’s a lack of insurance, a lack of healthcare literacy or simply a mistrust of—there’s a lot of generational mistrust I think in some communities of the clinical trial system.

So, as an advocate, I certainly push caretakers especially—and children caregivers for older populations who are maybe first or second-generation Americans to help facilitate a conversation between the medical professional who’s trusted and a patient that might not be able to get or rely on the information they’re given. Because it really will speak to populations that don’t get the opportunities that you and I have gotten simply because they are either not aware, or there is a barrier there to get to that medical professional.

Ken Getz:

I appreciate, T.J., you mentioned CISCRP. That’s one of the things that we’ve focused on for 18 years is bringing clinical research education into major metropolitan areas around the U.S. and parts of northern and western Europe where we plan for several months, and then we put on what we call an Aware for All events. And we really work very hard to encourage participation by—or from patients based within minority or underserved communities.

And I’m happy to say that we’ve had a lot of success with that. These are really difficult communities to reach through a lot of the traditional approaches. We have to rely on community centers and clergy and other approaches to really help these communities, for a lot of the reasons T.J. mentioned, trust the educational information, and come out to learn more. And I’m happy to say we’re seeing more and more people of diverse backgrounds that are curious and interested in learning more about clinical research, especially knowing that representative populations provide more information that can inform treatment for different types of patient sub-populations.

Andrew Schorr:
I want to go to Andy in a second. Andy, just one second. I wanted to mention and call out—and Andy’s company has been a leader in this. He was talking about PD1 and all of that. But drugs that have been breakthrough in immunotherapy for people like T.J. where—and it’s being explored in broader cancers where otherwise life was going to be short. And how to activate the immune system and really fight the cancer in people living long term. So, the people in those trials—and certainly there were people in the melanoma trials like yourself T.J.

Lung cancer trials and increasingly now others who did get tomorrow’s medicine today. Andy talked about accelerated approval which is great. So, that’s the impetus for the patient and the family. Is there the chance to get tomorrow’s medicine today? Now the obstacles may be distrust. You talked about that, Ken. And also, is maybe accessibility. Is it as a clinic near you? And Andy you talked about pushing that out. And then sometimes it’s related to cost.

Now is there anything that sponsors can do, Andy, related to the costs that people may have in being in certain trials? Where do we stand with that?

Andy Lee:

Yeah, so I’ll just touch on the distribution first and then get into the costs because they are linked. When we prosecute global trials—we’ve had a very U.S.-centric discussion so far. But cancers present differently in different geographic regions of the world. And so, when we want speed out of our trials. You want me to shorten that timeline and get drugs to market quickly. I do it internationally and in some cancers like esophageal cancer or some of the gastrointestinal cancers, Asia has a much higher prevalence of these cancers.

And we do a greater proportion of work there. We always include multi-country studies. And U.S. may have a greater proportion in other areas. So, we balance that out to optimize speed. Of course, with clinical trials the cost structure around the globe is very different. But let’s talk about U.S. We have spoken about a saturated core of clinical trial sites that we all go to. And I speak generally now for all sponsors. And we are all looking to optimize and get great efficiency.

At the same time, we realize we have many underrepresented geographies and ethnic groups—and not just ethnic groups, but under resourced populations. And so, what we’ve been thinking about is how can we support people, and support people at all levels. And so, we start off with thinking about the cost structure, and we obviously pay clinical sites for what they do. But we will support all sorts of things. We’ve been negotiating with Uber and Lyft, so we can build that into automated transport for patients.

Again, the IRB has to approve that. We are looking at ways to augment that they are not out-of-pocket for things. And we’ve been talking a lot with a group called Lazarex Foundation who has really expanded into under resourced communities and found ways to ensure that they have daycare and different access for those patients. We have worked extensively now to look at outreach programs into communities that typically wouldn’t be in trials. We are focusing in two areas right now as we speak.

One is next generation of HIV medicines, and the other one is in prostate cancer. And we’ve got a large program rolling out in prostate cancer. So, what we are doing is going into sites and we have put together training videos and training materials. And we are looking at cultural competency. So, it starts at the site. Are they culturally competent to engage a different community? And we’ve spoken about working with the community churches, community education systems.

And so that starts with cultural competency. I have a woman, Madelyn Goday, who works on this day and night in my organization. And she’s very strong at this. It’s early days, but if we can show that it works in one or two therapeutic areas and cancer types, we’d expand it further and further. But we can’t just have a shotgun approach and just go and do 100 sites and hope it works. Hope isn’t a good strategy. We are working systematically to engage different people. And as appropriate and approved by ethics committees, we will support all of these communities and help build infrastructure and capacity.

Those are important things for us. But as I said, where appropriate and where it’s sustainable. We can’t just throw money at something in the hopes something sticks. We have to have something sustainable and it goes to what Ken says, and that’s education and providing resources and materials. And we’ve used quite a lot of Ken’s materials in multiple clinical trials. Thank you for that, Ken. It’s been really helpful for us.

Andrew Schorr:

Great. I wanted to note for your audience. If you have a question, send it to questions@patientpower.info. We have expert panelists here. And this is really—we are all in this together. I think you hear the dedication from Andy at Merck and T.J. as a patient advocate and Ken as a professor and founder of organizations devoted to this. We want obviously accelerate medicines, but have the accurate data of how it affects different people, who is it right for so that the regulators—and thank you for what you said about the FDA here in the U.S., has the information to make a decision on should this medicine be available for people with that diagnosis.

Okay, so what about staying in the trial. So, T.J., how long—let’s take with the Keytruda trial or one of them. How long were you in to for?

T.J. Sharpe:

Nearly four years. Three-and-a-half years.

Andrew Schorr:

Were there ever times when you said, “I’m done. I want to bail out.” You know.

T.J. Sharpe:

I’ll be very careful how I answer this question for Andy’s sake.

Andy Lee:

It’s okay, T.J., we’re friends.

T.J. Sharpe:

No, probably the biggest crossroads I ever came to was when one of my tumors started growing about a year into it. And we weren’t sure if the medicine stopped working or not. We didn’t know what to do. And as it turned out, it was still working. And I think was just one spot that wasn’t responding. But everything else had responded great. However, at the point, as a patient, you’re thinking about yourself first and your family first and the trial second. It’s easy to stay compliant on a trial when things are going well.

But when you’re ahead of the medicine in some ways, and I think patients with chronic illnesses or in some cases rare diseases, are almost more knowledgeable than some of their doctors or the trial protocols about when they’re stopping. They don’t have the luxury of finishing out a protocol and seeing where their disease journey takes them. And the best example I can give of this is a very passionate advocate by the name of Jack Wheelen who we unfortunately lost a couple of years ago, but whose influence has kind of dominated the patient advocacy world for the last decade or so.

And Jack was able to monitor his health almost better than a doctor. And he knew when his trials weren’t working. When we get to that point in a clinical trial setting where we know the medicine is not being effective or where a patient would be better served to move on to another treatment. That’s when we are going to take the next step in clinical research, because now we’re aligning the trial design and the trial goals with a patient and a patient’s family’s treatment goals. And as those two points merge, that’s where clinical research becomes that much more effective as a care option.

Andrew Schorr:

That was well said. And I think with all those trials, you’re right, the team—that care team, what’s right for you at that time. Obviously to get the data, but also not at all costs. In other words, if the data is showing something is no longer effective for you, is there another treatment or a trial? I’ll just share my story for a second. So, I was in a phase two trial of combination therapies—which are increasingly common certainly in oncology. And after three months—halfway in the trial, my blood was kind of cleaned up.

And I had nausea and some other side effects. And I said to the trial coordinator, “You know, I think I’d like to stop.” And she said, “You know, our belief is that you still have microscopic illness in your bone marrow—in this case with the blood cancer, and the additional three months in this protocol will make a long-term difference for you. That’s what we believe.” They didn’t have the answer, but that’s what they believed. You know what? I stuck it out. She was right. I had 17-year remission.

If I’d stopped after three months, would I have? So, it’s a dialogue with the care team Andy, right? It’s this ongoing discussion not just entering the trial, but remaining in the trial, correct?

Andy Lee:

Yes. Absolutely. And I just wanted to impress a really important thing. People talk about people dropping out of trials. In cancer trials we see extremely low drop out. I mean these are potentially lifesaving medicines for all of the companies. But what we do want to make sure about is that when there is progression of disease, and it’s shown that the drug—whichever it is, the control arm or the active arm or the new agent, where there is progression of disease that they get the best available therapy.

And so that often contaminates trials because we have the crossover effect that now they are getting maybe the experimental agent in the standard of care type of thing. But most important thing for us is to track the survival of the patient, regardless of whether they go on another therapy. And we have put a tremendous amount of effort into looking at the informed consent and making sure we work with IRB to track patients long term survival.

Because as you’ve said, you may have a short-term issue that shows that the drug may not be working short term, but long term it may have prolonged and profound effects. Positive or negative, we don’t know that. And so, what we like to do is get long term survival. And we ask patients to consider when they sign the consent for whatever trial and whichever sponsor is sponsoring this, is to consider that knowing their status throughout their treatment—whether it’s on a sponsor’s drug or another sponsor’s drug or x therapy. It is really important — and I ask people to think about that.

Because that really helps us get as much data out of the individual treatment as possible. And that may prevent nonrequired trials in the future or it may say, “Wow, that really informed.” And we’d like to inform all cancer patients. If data we generate can inform other therapies, we certainly want to do that. We do not want to do wasteful clinical trials. So, tracking patients long term or patients—the message to patients is being cognizant of letting the sponsor—and the sponsor could be an institution. Letting them know your status is really important. All they want to know is are you dead or alive.

Andrew Schorr:

In the end, just one thing is, are we partners. In the end, our viewers here, are we your partner? And can we feel that not just for their doctor but you guys behind the scenes with the labs and everything, that in the end we are partners. And unless we see it that way, we won’t get anywhere.

Andy Lee:

Absolutely. I’m glad you used the term partners. Because when we’ve done a prep for this people have said, “Are they investors in the thing?” So, yes, patients invest their time and everything, but they are partners in research. They are contributing so much. They are contributing—they are going into the absolute unknown. And there is an immense trust level that is there. And we owe that back as research professionals is to treat people with respect, dignity and as partners, to make information available, to publish our data to get it out there as quickly as possible. And to make sure we get that back into the participant’s sort of hands.

Andrew Schorr:

So, Ken, how are we doing on that because you go back over the years and people say, “I don’t want to be in a trial because I’ll be a guinea pig,” and respect was not seen as part of it.

Ken Getz:
Well, that’s also a bit of a myth, right? You had a few that claimed that they felt the process made them feel like a guinea pig. The vast majority of people, over 90% of people who participate in a trial, would do it again. So, once they get past that unfamiliar area where they’ve perhaps only heard a few case examples or a few very vocal people who had bad experiences. Once they’ve done it themselves or they’ve been able to work with a group of advocates that really help them think about this process, and they become more educated, generally they’re very impressed with the level of professionalism, the compassion that exists at all levels.

I work with so many professionals—science professionals and pharmaceutical companies and at the research centers, and they all share that kind of commitment that Andy just mentioned. There’s a real desire to partner with the patient to really inform them. I would say one place where we need to see much, much more however is in the return of clinical trial results in a plain language to people who’ve been in trials. That’s a place where as an enterprise—government, research sponsors as well as industry have not really made this a standard practice at this point. And that’s one thing that we’re really working on actively.

Andrew Schorr:

Right. Great. So, T.J., you and I are investors—and Ken used that term and Andy used it, and I’ve always believed it. We are investors of our tissue, our body, our future to help other people and hopefully help ourselves. And certainly, for profit companies that may greatly benefit if they have a blockbuster therapy. But we need to be kept informed in the long term, right T.J.? We want to know what a difference our participation made.

T.J. Sharpe:

Certainly. And I think to echo what both Andy and Ken said is that patients do become partners. Patients who are involved in clinical research, a significant chunk become altruistically invested. I’ve heard more than once, “Even if this doesn’t help me, I’m glad I participated because it might help somebody else.” I know I’ve felt like that, and I’d venture that you’ve had some of that too, Andrew on your journey. So, it’s only—it’s at the very minimal fair, and it’s certainly very justified to expect as a co-participant in this.

And as kind of a co-creator of science with sites and sponsors that we understand what has come of our sacrifice and our time dedication to helping science out. We shouldn’t have to find it out through press releases from ASCO or hope that we hear about it on the nightly news. We deserve to hear what has happened. Not just because it can affect us as people and as patients, but that we put a lot into this too. And then we did our part to further medical research and we want to be part of the—whatever the end of the trial ends up being. We want to be aware of that. Not just for personal knowledge, but to know that it’s going to help this many other people.

Andrew Schorr:

Right, to be honored. So, Andy, at Merck you’ve established some internet platforms in particular related to keeping people informed, right?

Andy Lee:

Well, we’ve got an internet platform that people can log onto. I’m happy to share that with you; in which they can get access to a list of our trials. So, I didn’t prepare this but especially, but I did make a handmade note. And if anyone wants, it’s a very simple log on. Andrew Schorr:
You’re a great artist.

Andy Lee:

And it’s a simple one. What that will get you access to is two main important things. One is it gives access to information about clinical trials. We have a tab on there that tells everyone about the phases of clinical trials and what to expect in a trial. So, it’s an educational part. Then we have a lot of information about the Keytruda clinical trials were, are running, and they’re called keynote trials. And there you can look at the different indications. And you can look up and it has a telephone number you can call.

Now I must stress is that we run over 1,000 clinical trials in oncology. But many of them are not sponsored by us, they are investigator sponsored trials. So, you can go to clinics, and they run their own clinical trials that are not sponsor-related. And the NCI runs their clinical trials. So, there are a lot of different sources. And many companies will have clinical trials. We also have the website clinicaltrials.gov. I’ve had to use that in the last two days for a colleague.

And you can navigate that and look for different types of trials. And you can look at different products and everything. It’s not perfect. But at least it’s a place to go to. And I don’t want to sound as if I’m one sponsor centric. Many other companies have access to websites, and they really want to try and enhance and direct people to the clinical trials sites at which they are working.

Andrew Schorr:

Right, absolutely. And then you were working at the industry level with a group called TransCelerate, and I know T.J. is involved too, to try and establish common procedures as you establish trial sites, as you have communication, as you have training, right? So, that hopefully all boats will rise, right?

Andy Lee:

That’s correct. TransCelerate is a group that formed about eight or nine years ago. There were 10 initial member companies. I was a founder member of that. And we got together to say, “We have to improve operational efficiency.” So, we do not collaborate on molecular structures and those types—that’s competitive. We collaborate on what we call precompetitive, procompetitive aspects which says, “If we all work together to improve something, we’ll all get the benefit of this.” And we share it publicly.

There’s a website, you can look at it. But we’ve looked at standardizing protocols. We have a common protocol template. We’ve adopted that at our companies, so have other sponsors. The protocol can be developed in a standardized way. We’ve looked at standardizing ways where we can improve monitoring. We’re looking now at ways that we can work with investigative sites through i-platforms, shared investigative platforms. So, a clinical trial site has to provide the information for us as a sponsor and use the exact same standardized questionnaire and information for any other sponsor through a standard portal.

So, we are trying to reduce the burden on clinical trial sites. And we’ve plugged away for many years, and we are seeing greater traction there. We are seeing more efficiency, more standardization. We are seeing greater quality, less rework. And so, while it’s hard to quantify this, what we believe is that the sites are freed up of some of the more burdensome things, and they can direct their attention towards patients, patient safety, and access to clinical trials. So, the work may not be directly related to access for a cancer patient into a cancer trial, but there’s a lot of tangential spin-off of making a site more efficient so they can put their resources and energy in the right place.

Andrew Schorr:

Well, thank you for that effort and your leadership. So, Ken, you’ve been around this a long time. And you’ve deal with all the companies and the government and the various agencies. And as you know, in some quarters there’s a distrust for pharma. We mentioned cancer that you get the price tag of a drug, and it’s very expensive. And some people are struggling to pay for it. And there’s just frustration about it. And often in the news media they are the bad guys who are called out for unethical procedure or something that went awry.

So, how are we doing there in overcoming that because we talk to Andy, he seems very ethical, dedicated guy representing a company that’s been around I think well over 100 years. So, how are we doing to move clinical trials on in this area when people aren’t sure what to make of pharma.

Ken Getz:

Yeah. It’s a huge issue, Andrew. And I think part of the challenge is that all it takes is one questionable behavior, and it makes it difficult for the reputation of the entire industry. Right now, we are dealing with major pharma companies that are actually being fined for having contributed—a judgement, having contributed to the opioid crisis. And when you start looking at some companies aggressive marketing tactics, right? It really sort of sheds a darker light on a lot of the great work that companies do.

What we look at, at the Tufts Center for the Study of Drug Development at the School of Medicine. We look at the overall output, the level of innovation that’s coming from the industry today. And we look at the number of complaints that have been filed with the FDA and other regulatory agencies around the world. And what we see is tremendous growth in the innovation and the quality of the innovation—drugs like Keytruda and other cancer immunotherapies. What an exciting area.

We see that the vast majority of companies really support and live by highly ethical, highly professional, highly compassionate approaches because they all know that it takes just one questionable issue that can really tarnish the reputation of every company operating in the industry. So—again, Andy also mentioned just how regulated we are as an industry, the fact that we have ethical review committees and data safety monitoring boards and so many other external agencies that help to oversee the work that’s done here.

So, I would say for patients who are thinking about clinical trials, it’s good to know the history. It’s good to know what you need to do to protect yourself. But the vast majority find that the people they deal with are ethical, they are professional, they are compassionate. And, as I mentioned, over 90% of people who get involved in trials say that they would do it again.

Andrew Schorr:

Thank you. That was a wonderful response. Andy, you mentioned earlier about starting research with the sickest people basically, where there are no options. But one of the questions that came in is, “Are trials only for the sickest people or are there of all those trials you talked about opportunities for people who maybe are newly diagnosed or could be their fairly initial therapy?

Andy Lee:

Yeah, great question. And thank you to the person who asked that. And the answer is that we start in people—because we don’t know if our experimental agent will work. And everyone assumes that new medicines are all going to succeed. And we work in research and researcher because of that many things fail very early on. They fail in phase one before anyone hears of it. It’s normally a code number at that point. And we may just not make the drug soluble enough, or it may not be distributed enough.

So, we may have a thing that works in a test tube or a petri dish. But to get that into humans and make sure that it’s safe at the dosage we use often fails. We just don’t progress far enough. So, what we want to make sure of is that firstly the drugs are safe. And there’s a trade-off between safety and efficacy. We’re constantly trading off. And so, what we do is we look at that and say when someone has no option and we want to get an option going, that’s where we start.

We’ve actually moved down the disease scale, and we’ve come into adjuvant treatment or secondary prevention. And we’ve gone into newer adjuvant is when you have a small tumor is we pre-treat to manage that tumor before surgery is done. And post-surgery we hope that there’s limited treatment or no treatment. And we actually have removed the cancer, and there would be no evidence of disease. But. of course, using the word cured is something we try not to do, because we prefer to use no evidence of disease.

But absolutely. And the next strategy is prevention of cancer. Our company does a lot of vaccinations in women’s health. We have a product that protects against human papilloma virus which is a precursor for cervical cancer. So, people who are vaccinated with this particular product—and I’m deliberately not using brand names for obvious reasons. But when you vaccinate for HPV, you essentially are preventing the likelihood of a cervical cancer. And there are now prospects in many disease areas where either vaccination or early treatment gives you a tremendous positive prognosis of not getting the disease later on in life.

The answer to your question is yes, we are absolutely looking at ways to prevent getting to a very advanced stage which is very costly to manage and very emotional and stressful and difficult.

Andrew Schorr:

I want to thank you. I just want to get a final comment on what you would say to patients or family member. And I want to start with you, Ken. What do you want patients right now to know so that—what tips would you give them so that they’d consider being part of clinical research or stay in clinical research and the benefit it could be for them.

Ken Getz:

I will say really two things. The first is there’s just a tremendous amount of information out there, and we recommend education before participation. So, do your homework and engage family and friends and people you meet and trust to help you make the decision. And the second point comes off of that. And that is this is not a decision you make alone. Really bring in your treating physician, your nurse. Bring in your support network. And chances are you will learn a lot, and you might even find a trial that is right for you.

Andrew Schorr:

Right. And Andy, what about you? A final point—what would you say to a friend or family member or colleague related to considering trials today.

Andy Lee:

We get this question every single day. And we get it from patients in need. And my answer is we are all patients. We are all going to face this as professionals in our job or professionals outside. And so, I say community of practice. And disease hits all of levels of society in all education professions, et cetera. And so, my thing is to encourage people to do what Ken has said. Work as a team. Get multiple inputs.

And I am sponsor agnostic. Get the best therapy that is available. And that may be the best care option—as I said, the ecosystem in which you get the care is really important as well as the medicines that you get. So, have the discussion. Trust the medical professionals, they are very skilled out there. They are extremely well educated. And I just urge people, “Don’t think on two clicks on Google you are going to solve what your treatment option is.” Really discuss it with people because not all the options are public, and there is not enough information available about how to manage the whole disease through the entire enterprise. Trust the professionals.

Andrew Schorr:

Well said. And T.J., you and I are alive today because of trials. What do you want—what’s the thing you want to leave our viewers with?

T.J. Sharpe:

That they don’t have to be involved in clinical research. I think that’s an important distinction to make. And it’s going to pull together what Andy and Ken said that clinical research should not be considered a hail mary or last gasp option. If you are a patient—and we are all going to be patients as Andy mentioned. You want the best care for you. You want to be able to weigh all of your options. And if you are not considering clinical research, if you don’t know about it or aren’t able to get the information you need about it, then you are not going to be able to make the best healthcare decision long term for your health.

So, take that information that you can get. Find the trusted sources. Be able to reach out to advocates or colleagues or someone that you know that would have the disease or can connect you with good information. And be your own advocate—a little cliché, but really own that healthcare information. And once you are able to collect all of the different treatment options, then you consult with your professional medical team as to what the plan forward—the best plan forward for your individual situation would be.

Andrew Schorr:

Right. T.J., my friend, thank you. It’s a delight to see you again. Andy, with Merck, thank you so much for being with us and bringing your years of expertise. And, Ken, being at an independent non-profit center and also at Tufts University there, thank you for all the work you do. I want to thank the Patient Empowerment Network for pulling this all together. And the sponsors who supported us in this effort, AbbVie Inc., Celgene Corporation. Daiichi Sankyo and Novartis.

All these companies and I’m sure many more, working so that research can move forward. We can be true partners in it. And hopefully get tomorrow’s medicine today to make a difference for the community and live a long life, and hopefully a cure, right? I’m Andrew Schorr in California. Remember, knowledge can be the best medicine of all.


Please remember the opinions expressed on Patient Empowerment Network (PEN) are not necessarily the views of our sponsors, contributors, partners or PEN. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.