Wondering how YOU can advance MPN research?

September is Blood Cancer Awareness Month and we’ve spent the month focusing on ways you can become a more empowered patient. If you missed our recent webinar: What YOU can do to advance MPN Research, the replay is now available. As always, we’d love to hear from you about ways you’ve empowered yourself!

Living Well with Multiple Myeloma – Friendly Dieting

Friendly Dieting – Sifting Through Science vs. Hype

Living Well With Multiple Myeloma – Friendly Dieting from Patient Empowerment Network on Vimeo.

In this myeloma-friendly dieting webinar with Julie Langford of Cancer Dietitian (Cancer Services) and Danny Parker of MultipleMyelomaBlog.com , they lead a discussion focused on how to eat when you are living with myeloma. Both help us understand what “bad and good foods” are and what overall lifestyle changes may be helpful in allowing you to live a full life with myeloma.

12 Keys to Finding, Growing, and Nurturing Your Online Community

“If you want to go fast, go alone; if you want to go far, go together” – African Proverb

Before the Internet connected people from every corner of the globe, many patients experienced their illness in isolation.  The Internet (and social media in particular) has lessened our sense of isolation, helping us feel more connected to others who are going through the same experiences.   Online communities may be virtual, but they are no less real in terms of support and influence. We see just how much people are willing to reach out to others to provide advice and support – even to strangers online.

At the heart of a high-functioning social network is a strong sense of community.  Social media has accelerated the growth of patient and caregiver communities, allowing people to come together around shared experiences, regardless of time or place. For many of us, finding our online community has made a significant difference to how we cope with our illnesses.  From helping us to uncover a diagnosis and finding the right doctors and treatments, to learning about everyday coping tips, turning to our online community can make all the difference.

Your online community can be your eyes and ears, helping you find something you may have missed or not known about.  Isabel Jordan, the mother of a son with a rare disease, turned to her online community to help find a diagnosis for her son.  “Connecting the dots by seeing them in someone else let me provide valuable clues to our own clinician researchers and now we’re heading down a new diagnostic path”, she says. “Would I have seen them anyway? I don’t know. But I credit my connections on social media for helping me keep my eyes open to new ideas”.  Katherine Leon, an SCAD (spontaneous coronary artery dissection) patient, leveraged the power of her online community to find the cause of her rare heart disease, and prevent it from happening to others. At the time of her diagnosis, SCAD was a poorly understood and under-researched condition. Physicians had no clinical studies on which to base treatment plans. Katherine connected with fellow SCAD survivors through social media and used their collective voice to do what hospitals couldn’t – to launch research at the Mayo Clinic.


Five Ways to Find Your Online Community

Are you a newly diagnosed patient or a caregiver wondering where to find your own online community? Here are five practical ways to get started.

(1)  Find People to Follow on Twitter

Start by following the Twitter accounts of organizations and groups related to your interest. Go to their website and click on the Twitter follow button if they have one. Once you start following individuals and organizations, Twitter will automatically populate your account with suggestions of similar accounts to follow. You can also view my list of patient advocates on Twitter and add your own name to this list if you wish.

(2) Build Twitter Lists

Twitter can be a little overwhelming to new users. To help you keep track of conversations, it’s helpful to organize your followers into lists; e.g. “organizations”, “researchers”, “patient advocates”, “hospitals”. You can create your own lists or subscribe to lists created by others. Christina Lizaso, a moderator of the #gyncsm Twitter chat, has created several public lists worth subscribing to. See also this comprehensive list of patient chat community members created by Team Intake.Me. 

(3) Follow Relevant Conversations

The easiest way to find conversations of interest is to click the native “Search” facility at the top of your Twitter screen and enter your keyword or hashtag, for example #cancer. Hashtags are a useful way to search for health related conversations.  Jo Taylor, a moderator of the UK-based breast cancer Twitter chat #BCCWW, explains that “finding disease hashtags opens up connections. If you connect with others you will be able to meet others easily online and you will build and learn from there.”

(4) Join Twitter Chats

A Twitter Chat is a public Twitter conversation around one unique hashtag. This hashtag allows you to follow the discussion and participate in it. Twitter chats can be one-off events, but more usually are recurring weekly chats to regularly connect people, for example #PatientChat held every other Friday at 10:00 am Pacific/1:00 pm Eastern. The chat will be hosted and the host will ask questions along the way to stimulate discussion and sharing of ideas. There are chats for most disease topics and a full list can be found by searching the database of the Healthcare Hashtag Project. This is also a useful resource to find Twitter users to follow. In addition you will find past transcripts of chats on the website so you can familiarize yourself with the chat and its norms before taking part. And “if you can’t find a tweet chat you enjoy,” recommends patient advocate, Annette McKinnon, “start a new one, register it @symplur and build a new community.”

(5) Join Facebook, Google+ and LinkedIn Groups

On Facebook, Google+ and LinkedIn you can connect with other patients and join groups related to your disease or condition. Many organizations have a Facebook and LinkedIn presence and by following their pages you can keep informed of their activities and find other patients to connect with.

When you’ve identified some groups which interest you, don’t feel you have to join in straight away. Take a little time to learn if the group is the right fit for you. Does it appear to be a welcoming and safe space? Are the discussions and norms of the group respectful and in line with your interests and values? “Patients and caregivers seeking to join an online support community should ensure that they feel comfortable browsing a new online group before posting,” according to John Novack, Communications Director for Inspire, a healthcare social network with more than one million registered members. “Be an active lurker in a new group,” he advises, “and if possible, search for the topics most discussed there, because that will give you a sense of the overall focus of that community.”   Jo Taylor agrees: “Watch and lurk (i.e. you don’t have to contribute – just read what is said) or join in.  It’s up to you,” she says. “Don’t feel pressured to talk, but also don’t feel that you would say anything wrong.  It’s a friendly place.  Join in and meet others.” You might even find these online connections become friends offline too.  “I have met people from all over the world,” says Jo, “but some are in my own town and I see them regularly.  Whether you want online connections or face to face, both can happen due to the power of social media.”


7 Ways to Nurture Your Community

If you are moving towards creating an online community, here are seven ways for you to develop your community and help it flourish.

(1) Grow Your Community

A community is grown over time, not built overnight.  I reached out to Julia, also a moderator of #BCCWW, to ask about her experience of growing a Twitter chat. She explained that the community will evolve by trial and error, “but it’s important to know what it is you are aiming to achieve and why. If you can get that clear”, she says, “It will follow from there”.

Don’t get hung up on follower numbers. It’s the quality of your interactions and your ability to cultivate meaningful relationships that is key to building a successful online community.  As Erin Gilmer, founder of The Research Loop, points out, sometimes it may seem like there are only two people in a Twitter chat, but it turns out to be more, because many patients “lurk” but don’t feel comfortable tweeting openly. “Even if it’s just two of you,” she remarks, “it’s still a community.”

However, if your community is new you will want to build up your initial numbers to get started. Go through your e-mail contacts list and invite relevant people to join your community. Do the same with your followers on Twitter, Facebook, LinkedIn and any other social networks you are active on. Ask existing members to invite their friends.  When choosing which social network to communicate on, go for the platform your audience is most familiar and comfortable with.

(2) Provide Valuable Information

Building a sustainable community requires you to provide value and be responsive to the needs of that community. Think about how your group will become the go-to information resource for your members. This means you will need to create and share information on a consistent basis. Don’t automatically assume you know what the group need. Ask questions to better understand their issues and concerns. Invite researchers, physicians and other healthcare professionals to answer questions for your community through Facebook, LinkedIn, Twitter and blogs.

(3) Connect Members to Each Other

As humans we have an innate desire to feel connected with others who are going through the same experiences we are. Clay Shirky, author of Here Comes Everybody: How Change Happens when People Come Together, holds that “the desire to be part of a group that shares, cooperates, or acts in concert is a basic human instinct.” In the future new online tools will come and go, but our innate desire to reach out, to connect, and to help one another will remain.  “People seek community online to connect with other people”, says Colleen Young, Director of Community, Mayo Clinic Connect at Mayo Clinic. “If you want to build a thriving community, focus on the people, help them connect and get them talking.”  Introduce members to each other and connect like-minded people.  Think about how you might create common bonds, cultivate a sense of belonging, and build strong relationship among members.

(4) Listen: Don’t Judge

Do listen to your community and try to address their legitimate concerns. You may not agree with everyone in your group, but try to understand where they are coming from. Determine whether negative comments have any merit. This doesn’t mean you have to engage with trolls or unwarranted criticism. Sometimes people just want to cause drama or discord, so it’s important to put clear policies in place which protect you and your community from abuse.

(5) Reach Out and Support the Community

Collaboration, not control is at the heart of a successful community.  Reach out to your members and find out how you can help and support them. Find answers to their questions, retweet, favorite and share their content with others.  Equally, don’t try to do everything yourself. Co-create content with your members and ask for help when you need answers and support too.

(6) Nurture Your Community

When you nurture relationships in a human way, they flourish like friendships in our personal lives. Take time each day to interact personally by welcoming new followers, answering questions, acknowledging comments, addressing members by their name and thanking people for contributing to the conversation.  Also, take time to acknowledge birthdays, milestones and other achievements.

(7) Be Open, Honest and Transparent

Be open and transparent in all your online activities. Without honesty, you have no trust or credibility. Model the behavior you wish to see in the community. Be willing to self-disclose and encourage self-disclosure in others by creating a safe space for members that welcomes open and honest discussions.

Bonus Tip: Broaden Your Reach

If your goal in creating a community online is to influence policy or improve communication with a wider healthcare audience, you will need to broaden your reach to create impact. “Patient advocates who lead successful online groups and chats have to establish credibility with all stakeholders in a particular therapeutic area, if the advocates want to broaden beyond establishing groups of only patients,” says John Novack. “Some Twitter chat communities like #BCSM, #LCSM and #GYNCSM are powerful”, he notes, “because caregivers, clinicians, and technology leaders are regularly involved.”

Building a community is an ongoing process; it requires an investment of time, and according to Annette McKinnon, “a core group of committed and persistent people.” It’s about building trust, connecting people, and providing valuable information and support over the long-term. Your community is always about something greater than yourself. The best communities will provide a safe space to support each other, mentor and help each other grow. Whether you are joining a group for the first time or starting your own online community, consider how you might contribute your unique experience and expertise to make the group a more connected and inclusive space.  Finally, it’s important to have realistic expectations. A community requires “balance, and equanimity; a generosity of spirit; an expectation of complexity; a tolerance of frustration; a desire to listen, and to give,” says Andrew Spong, Lead, Health Equals.   “The truth may be that communities are less cohesive than they appear,” Spong reflects, “but they are still the best tools we have to create bonds with others of like minds and experience.”

Related Reading

#PatientChat transcript on online communities

Notable News

Remember last month when we told you about the successful immunotherapy test trial known as CAR T therapy using the patient’s own immune cells to fight leukemia? There was a big push for the U.S. Food and Drug Administration (FDA) to approve the therapy and they did! According to NPR.org the approval of the first cell-based gene therapy in the United States is being called a historic action. The drug called Kymriah was developed by Novartis. It has been approved to treat a form of acute lymphoblastic leukemia in children and young adults up to age 25 who have not responded to other treatments or who have experienced relapse. In the test trials, 83 percent of 63 patients were in remission within three months of undergoing the therapy. While very successful, the treatment does come with serious risks including a potentially life-threatening immune system response. Therefore, the FDA is requiring strong warnings and, for now, the treatment is only available at 32 specially trained hospitals and clinics. The treatment is also very expensive — $475,000 for the one-time treatment. Patients who do not respond within one month will not be charged. You can read more details here and if you missed the information we shared about CAR T therapy last month you can find that here.

This month fda.org announced another first in cancer treatment. A biosimilar drug, which is an almost identical copy of an original drug made by another company, was approved for the treatment of several types of cancer. Mvasi, a biosimilar to Avastin, is the first biosimilar approved for cancer treatment in the United States. This is significant because biosimilar drugs encourage competition among companies and help keep healthcare costs down for patients. Mvasi is approved to treat certain colorectal, lung, brain, kidney and cervical cancers in adult patients, but it could still be a couple years before it is available. More specifics about Mvasi, the cancers it is used to treat, and the side effects can be found here. Also, an interesting article on reuters.com about the impact biosimilars will have on healthcare costs as they become more widely available can be found here.

It’s hard to believe, but a virus known to cause brain defects could eventually be used to heal the brain. That’s what sciencedaily.com is reporting based on a study by researchers from Washington University School of Medicine in St. Louis and the University of California, San Diego. The study involves the Zika virus and glioblastoma, the most common form of brain cancer. The Zika virus targets immature and growing brain cells. That’s what makes it so dangerous to pregnant women and fetuses. However, in laboratory testing the researchers found that in an adult patient with glioblastoma, the Zika virus may bypass the normal brain cells to target the cancerous cells. It’s still too early to tell if the virus is a viable option for cancer treatment and there are other health concerns to consider, but this encouraging study opens the door for more promising research. Learn more about the Zika virus study here.

Finally this month, a patient story that is going viral. A woman in Minneapolis with stage 4 breast cancer mentioned to her mail carrier how overwhelmed she felt by her diagnosis and treatment. The mail carrier, also battling cancer, organized a very heart-felt expression of hope in the form of 101 heart-shaped balloons planted in the woman’s front yard. See video and learn more about these two empowered patients here and make sure you stay in touch with what’s going on at PEN here and look for more Notable News coming next month.

Patient Profile: Elizabeth Carswell

She was 25 weeks pregnant and felt like she had the flu. It was early July 2015 and she made her way to her obstetrician’s office because the antibiotics she’d taken over the Fourth of July holiday hadn’t made her feel better. A couple hours later Elizabeth Carswell was in the hospital being diagnosed with Acute Myeloid Leukemia. She was 33 years old.

“Obviously, the scariest part was the first two months when I was still pregnant,” says Carswell, known as Liz to her friends. She was immediately transferred to Jackson Memorial Hospital in Miami and began chemotherapy. “It was a very tough decision to be treated, but I wanted to meet my baby.”

The first two rounds of chemo were unsuccessful and her doctors wanted her to undergo a more aggressive treatment. That meant it was time to deliver the baby. Liz was 32 weeks along and the surgery was extremely risky. Her blood counts and platelets were considered too low for surgery, but despite it all, baby Hudson was born weighing in at 4 pounds. Liz continued her treatment in the hospital while Hudson was admitted to the NICU for five weeks.

When Hudson was released from the hospital, Liz was not. Her boyfriend, Hudson’s father, Frankie Lightfoot had to return home to work, so Liz’s father and her sister moved to Miami, rented an apartment near the hospital and cared for the baby. Liz was never alone. Her father and sister alternated caring for the baby and spending the night in the hospital with her. Her friends showered her with gifts for the baby and travelled to her bedside to decorate her hospital room. Meanwhile, her doctors were trying to ready Liz for a bone marrow transplant, but could never get her levels where they needed to be and eventually her Jackson Memorial team told her it was time to go home and spend whatever time she had left with her baby.

That’s when a friend connected Liz with the Leukemia and Lymphoma Society which led her to a clinical trial at the City of Hope Cancer Research Hospital in Duarte, California under the care of Stephen J. Forman, M.D., F.A.C.P. She went the next day. The trial involved very aggressive full-body radiation and a stem cell transplant. Her brother, a perfect 10 match, was her donor.

The transplant put her into remission. It was November 2015. Then, in August of 2016, she found some lumps in her breast. The leukemia had returned in mass form. More radiation and seven more rounds of chemo later she is technically in remission. But, due to a gene mutation that makes her highly likely to relapse, Liz continues with regular rounds of chemo for maintenance.

“My doctor expects recurrence,” she says. That’s why she will undergo another stem cell transplant within the next couple months. This time, from an unrelated donor so that a new immune system can be introduced. She will spend another six weeks in the hospital, but she believes this is what she has to do. “I know it’s needed,” she said. “I trust it’s needed. I trust my doctor. He’s pretty amazing.”

Liz is undaunted by what lies ahead. “There are lots of ups and downs, but a positive attitude is a must if you’re going to survive,” she says. Though she credits Hudson, now two, as her main motivation. Early on she felt it was Hudson growing inside her who was keeping her alive and now being his mother keeps her going. “I’m not scared to go through it again,” she says of her upcoming transplant. “I’m excited to do it again. I’ll do anything that will give me a chance to watch Hudson grow up.”

Liz has kept a record of her journey on her Facebook page, Prayers for Elizabeth. She continues to keep her page updated and in addition to helping her, she hopes that sharing her story can help others. You can keep up with Liz here.

Living Well with MPNs – What YOU Can Do to Advance MPN Research

What YOU Can Do to Advance MPN Research

Living Well With MPNs – What YOU Can Do to Advance MPN Research from Patient Empowerment Network on Vimeo.

An audience of MPN patients and their caregivers joined us online or on the phone as experts discussed what patients can do to advance research and to raise awareness for MPNs.



It is Blood Cancer Awareness Month so our webinar is what YOU can do to advance MPN research. I’m Beth Probert. I am a polycythemia vera patient and advocate.

I was diagnosed with PV in April, 2016 and I had about 12 months of treatment which included a few phlebotomies and interferon, Pegasys. I reacted very well to Pegasys and I am now in remission. I get my care at the University of Southern California Norris Cancer Research Center in Los Angeles. I’m coming to you live from Oxnard, California, which is just north of Los Angeles on the central coast.

I would like to start off by thanking our Patient Empowerment Network for their support, and the MPN Research Foundation for their continued partnership.

I’d like to welcome our guests today. We’ll start off with Dr. Verstovsek, who is a renowned MPN expert from The University of Texas MD Anderson Cancer Center. Thank you for joining us today, Doctor.

Dr. Verstovsek:

It’s my pleasure. Thank you very much for having me on the program.


And I’d also like to introduce you to Lindsey Whyte, from the MPN Research Foundation. Lindsey, thank you for taking the time to join us today.


Thank you so much for having me. I look forward to enjoying this esteemed panel.


Thank you. We have two patient panelists today joining us. Both have been in clinical trials and both run support groups in their area. Our first patient panelist is Nick, and he’s coming to us from central Florida. Thanks for joining us, Nick.


Thank you very much, Beth. Appreciate it, glad to be here.


Great. And I’d love to introduce Andrea, our other patient panelist. And she’s coming to us today from Dallas, Texas. Thanks for joining us, Andrea.


Hi everybody, it’s great to be here. Thank you.


Great. I’d like to start off our program today just getting to know a little bit more about Nick and Andrea. Nick, I’m going to start off with you. You were diagnosed with myelofibrosis in February, 2016. Let’s talk about when you were first diagnosed. What was your first move after diagnosis?


Definitely I was quite shocked. I was asymptomatic, and the doctor who presented me with the diagnosis really didn’t give me a whole lot of information; basically a Google printout that said I had one to three years to live. So, I traveled throughout the country, hooked up fortunately with Patient Power and MPN advocacy; went to a seminar in Stanford, outside of San Francisco with a wonderful group of doctors there to really learn about the disease.

Ultimately went to May Clinic, Dr. Tefari; Hutchinson Clinic, Dr. Mesa in Scottsdale, Arizona; and finally spent time with Dr. Pemmaraju who works with Dr. V. at MD Anderson. I felt it was important to try to learn as much as we can about the disease to try to help other people behind us, so I signed up for a trial with azacitidine and Jakafi at the MD Anderson hospital, which was phenomenal. They did a wonderful job but unfortunately my blasts had spiked up and I had to go to transplant, which I did a transplant on January 15 of this year.


Wow, you have really been through quite a lot in such a short period of time. We’re definitely going to come back to you and talk about some of those details you gave us more specifically. Thank you.


You’re welcome.


Andrea, I’d like to go to you, now. You were initially diagnosed with essential thrombocythemia about 19 years ago, and then you were later diagnosed with myelofibrosis about nine years ago. So, you’ve certainly had quite the journey. I’d like to hear a little bit about how you reacted when you were first diagnosed.


Sure. My primary care doctor was really great in seeing that my platelets were rising. And while they weren’t that high, she still sent me to a hematologist just in case; a local person here. He did know about it but wasn’t too versed in the MPN world, and we worked together and I was on anagrelide and a couple of drugs and I was doing great, no problems. My platelets were good; everything was great for ten years. And then, things started to change. I started getting tired. And the first thing he said to me was, I think we ought to send you to MD Anderson.

When I had essential thrombocytosis, I felt fine. Emotionally I was fine because I had no symptoms. But when it started to convert, when things started to change and my lifestyle changed and I got much more tired and of course anemic, that was emotionally difficult. But then I went to see Dr. Kantarjian, and soon Dr. Verstovsek, who started me on different trials, I said I am not going to sit here and die. Because I got the same thing; five to seven years, not from any of the doctors at MD Anderson, let me clarify. But I knew that it was at that point kind of a death sentence, not to be dramatic about it.

But I was not going to sit back and let that happen. So I said, what can we do? And was told let’s start you on a trial. I’ve been on five. Three of them didn’t work. One emotionally almost killed me; it was not fun at all.

But the last two, I guess I could say I was in remission. I never heard the word from Dr. V, but I felt great. My life was going along fine. The last maybe six or eight months, the drug seems to not be working as well and we’re looking for something else, possibly stem cell transplant.


You’ve had quite a journey over about 19 years, so I’m guessing you’re really a guru to a lot of us; myself, being newly diagnosed compared to you. And I know that both you and Nick run support groups. And if I can ask you, since we’re talking right now, can you tell me a little bit about your support group and then Nick, I’ll go to you in just a few minutes. Your support group is in the Dallas area. Can you give me a little feedback about it?


Sure. It was actually started by someone, Karen Stern, a good while ago; I don’t remember exactly when. Unfortunately, Karen got very sick very suddenly and is no longer with us.

She had asked when she was ill if I could take it over for her. I said absolutely, I’d be happy to do that. So, we meet quarterly. We have anywhere from 15 to maybe 30 people that come. We talk about what’s new with us, what’s new with our symptoms, what reading we’ve done, how we have learned, what we’ve seen in all the different conferences, and we share a lot of information. We eat, we have a glass of wine, and it’s great fun.

It’s wonderful to be hooked up because as much as I think I try to keep up with the diseases, I learn something from people at our support group. What’s hard is when people don’t come anymore for reasons that they’re sick or other reasons, and that’s tough on the group. But it’s all part of the process, I believe.

We have a lot of communication; we talk to each other. We use Facebook and we learn.


Wow, and it sounds like you guys have a really personal group there and happy; that in-person connection sounds wonderful. Thank you. Nick, tell us a little bit about your support group in central Florida.


It’s definitely a little looser organization. We have probably an email list of about 15 people. We keep adding just about one person every other month or so. Unfortunately for me, we got the group together and I started just doing the emails, sharing information with people, guiding them to Patient Power, guiding them to MPN. A lot of them are trying to find doctors in smaller towns in Florida where they don’t have access to people like Dr. V, and so trying to steer them to some of the better doctors in the area.

Unfortunately then I was going through a transplant so I was kind of out of pocket. So we’ve not had a chance to physically meet. We are kind of spread out; we have people in Jacksonville, Orlando, Tampa, Miami, and Naples.

So just with my regrouping here after the transplant, we’re hoping to probably in the next month or two have our first face-to-face meeting and get some folks together, probably in Orlando, and try to get the group together. But so far, it’s just been a matter of emails; folks emailing each other back and forth. We do have a lot of it seems like mostly PV patients, and they will share different techniques and just like Andre was sharing, some of the things she’s been through. I definitely feel that being around other people who are in the same boat is so much more powerful because it’s hard sometimes to talk to your family members or friends or people at work because they just don’t get it.

They don’t understand what you’re going through and how scary it is. They can’t really give advice. So we’ve found that the support group has really been – it helps me as much as being a part of it is feeling better about what’s going on. It is kind of sad, too, as Andrea said as you see some of the folks take a turn, and you’ve got to kind of rally everybody together because it is – unfortunately, this group, it’s folks who are sick and they’re all going to have their ups and downs and we have to kind of be there for each other. So, it is one of the tougher parts of being a part of a support group as well; I agree with Andrea.


I can definitely imagine. Just hearing from both of you and my own experience being in a support group, whether it’s in person or whether it’s online or through email, they’re just all equally effective and really sometimes the best medicine for us; a certain part of our care. I’d like to shift gears a little bit, and Lindsey I’d like to talk to you a little bit with your expertise in the field. Patients are newly diagnosed and you know, how do you convey to them the importance of educating themselves and their family and their friends? What would your message be?


The MPN Research Foundation has extensive information on our website, and also when somebody registers through our online system.

And I’m not talking about the registry; just contacts us initially, we send out packets with information and we will customize those packets according to a specific patient’s need. We also do or best to hook people up with local support groups or many times people come to us looking for a doctor or a specialist, and we’ll point them in the direction of online resources or others who may be able to help them locally. We have lots of resources available to patients through our organization, and also on our website and to the extent that we can help with a specific question, then we usually try and point someone in the direction of someone else who can.


I have to say that that’s exactly what I did. I found your website when I was newly diagnosed and I registered. I got my packet. I was so excited, it had a lot of great information for me. I also got some wrist bands, and I just felt connected.

It was one of the first organizations I found and it just really gave me a sense of really being connected. So, Dr. Verstovsek, I would like to talk to you a little bit about how do you advise your new patients about identifying trustworthy info as far as… we could all go on the internet and go willy-nilly. I was convinced I was dying after two minutes on the internet. But you must get a lot of that. You must get patients asking you what do I do, how do I get information; how do you guide them?

Dr. Verstovsek:

Just towards my endorsement of what we have discussed so far in terms of education and the patients’ engagement, I have seen some of you in my own clinic and you know very well that to every new patient, because this is a chronic disease or diseases, the patients have to be engaged.

And I always endorse them to become a partner in what we are trying to do together to control the disease and eliminate the problems, and make people enjoy life fully for as long as possible with a good control in signs and symptoms. And therefore, engagement and partnership with your doctor and self education through support groups, through symposia, through pamphlets through web is increasingly important. Because the decision-maker is the patient, after all. The doctors are here to support.

And if they can partner together throughout their lives and become good friends, and I always seem to joke about it; we become good friends for the rest of the time. We actually do, and we engage together and we try to educate each other. I learn from the patients, patients learn from me and we go through life together. And so an educated patient is the one who is the best patient because we can participate together. And the sources are increasingly available to all of us.

What we are doing her today, Patient Power on the web, MPN Research Foundation through the web, through symposia, through pamphletsthrough educational material, participating in patient symposia through educational foundations; those are also available apart from MPN Foundation. And then going to the academic centers and their websites; MD Anderson, Mayo Clinic, University of New York City. There are a number of very well established academic centers that have very nice academically-driven websites for education of the patients.

It’s not necessarily to engage at the professional level where the doctors would go and educate themselves about these rare conditions, but also the patient side of the academic sites are very well informative for the patients. This is where you get up-to-date information from very well established professionals that are engaging in education of the patients.

Plus, what you have described, Nick and Andrea, engagement at the personal patient level; support groups aerospace increasingly important for the understanding of the complexities that we are facing and understanding the diseases, and understanding the therapies and the different outcomes. Look at yourselves in this panel. We have a patient, Beth, who is an example of an extraordinarily response to a therapy in complete remission.

Nick had a progressive disease, ended up having a transplant; and Andrea has lived so many years with the condition and went through the different therapies with different outcomes and is living now way more than what her first doctor said about myelofibrosis; nine years and going very well. So, people are different. Things are improving markedly and we need to work together, and education is the primary source of that effort.


That is fabulous. I could really see how your really personal approach to caring and educating your patients really allays their fears. I wore those shoes where I was scared, and I really admire the way that you help your patients understand what is out there for them and to see the positive approach. Dr. Verstovsek, why do you feel an MPN specialist is so important when we have patients like Nick and Andrea and myself and others out there, as opposed to just kind of avoiding the specialists? What are the things that we should be really aware of and why we should go to an MPN specialist?

Dr. Verstovsek:

MPN diseases are rare diseases. And in many circles in academia, particularly essential thrombocythemia, polycythemia vera, ET, MPV are considered benign. Yet, we know that patients can change. Andrea has changed. There might be complications that can affect not just the quality of life, but the life duration as well.

Myelofibrosis is certainly much more aggressive and progressive, and can shorten life a lot but is the rarest of the free. So, myelofibrosis being so rare, people in a community setting, in smaller academic centers don’t have much experience. ET and PV, so-called benign conditions but not to me so benign, there is a need for education not just at the patient level but also at the level of physicians. There is much effort to educate professionals about the new developments in diagnosis, in therapy, and in prognosis of these conditions.

And therefore, a second opinion is always good to get. Wherever you are, even if you come to me as a first doctor, I encourage people, patients who come to me as a first doctor to seek a second opinion if it’s necessary to fulfill that educational potential that professionals can give so the patient is fully aware of what’s happening, and to live with that condition for the duration of time that there is.

The professional impact on the patient’s life is enormous because these are conditions that people live with. And therefore, proper indication and seeking a second opinion, and perhaps visiting an MPN specialist is highly encouraged


Doctor, how do you coordinate care from afar? If it’s just not geographically… someone doesn’t have an MPN specialist near them, can you tell us a little bit about how you would coordinate that?

Dr. Verstovsek:

This is an excellent question because the specialists are around the United States and as you aid, not everybody can be local or come very often. And therefore, communication with the local doctor and education through the email or the phone is vital. We try to form a team. Patients at my center know that we are not here just to provide one-time opinion; it is continuous engagement with the local doctor.

And the patients are always encouraged to come back at the frequency that can be absorbed by their social and financial status; every six months, every 12 months if possible with more benign conditions. Or, with more aggressive conditions more often; engage in what they do, developing new medications or advising on the proper use of standard therapies. Because it is not easy even for a patient in a community setting to apply standard medications properly because of the rarity of these conditions, particularly myelofibrosis.

So, a team effort with the specialist, with the local doctor, and the third most important is the patient themselves; fully engaged in these three triangular here will provide the most benefit for the patient’s outcome overall. And that can be done as necessary. But one thing is that sometimes I should say expectations from the MPN specialist perhaps are overshadowing what actually the expert may do.

For example, myself, I am not able to monitor patients from a distance; it’s not advisable. Therefore, the local doctor must be part of the team with the interaction through the ways that I described for the best outcome of the patients. But not one doctor that can be supervising patients wherever they are.


And that’s wonderful to know that this option is there for them. Because we do hear from patients who are in more remote areas and feel that perhaps are not getting the care for someone that’s really more renowned in their field. So it’s so wonderful to hear about those types of patient’s and how it can really benefit the patients. Thank you.

Lindsey, I’d like to ask you a few questions about the goal of raising awareness. Why is raising awareness essential to move forward, to really move research forward?


That’s a great question. You know, as most people on the phone are probably already aware, the MPN Research Foundation is very involved in funding research and trying to help direct research for MPNs. One of the things that we really need to try and understand is the course of the disease and how we can help to affect the quickest results for the patients to help them to feel better. I don’t know if that answers your question, but that’s one of the things that we focus the most on, and that’s where our projects are directed.


That’s great information. You know, I think people tend to forget that if we are such a rare group of diseases, the MPNs, and if we’re not raising awareness, we’re missing out. And we’re missing out in  moving that research forward.

Nick and Andrea, Andrea I’ll start with you; could you just give me some ideas, maybe? Has your support group or you, yourself personally, thought of different ways to get involved to raise awareness?


Well, one of the things we’re trying to do is invite speakers to the meeting. And my hematologist came once, and it was on a Sunday; I thought that was a very noble thing to do. People got great information. No holds barred, he answered questions and it was great. And hopefully we’re going to have someone from Cancer Care so that questions can arise, and then people can go and ask about them and check. How to raise awareness, I think it’s talking to people, I think it’s participating in maybe the LISTSERVs. It’s participating in things like this where they tune in and hear things and ask their questions.

I think it’s part of it on Facebook, encouraging people to go to meetings and to get more educated.


Those are all great ideas. Nick, do you have a few ways or things that you’ve done either individually or through your support group that you try to raise awareness?


Yes. I was kind of fortunate in that several years ago I was president of a company headquartered here in Tampa called Beef ‘O’ Brady’sWe had 270 sports pubs, kind of like a Buffalo Wild Wings. And then more recently I have a company called Little Greek, and we have 33 restaurants. We have five or six in Dallas and 17 here in Tampa. So fortunately the local media kind of got behind me and they did several articles about my disease, talking about myelofibrosis, trying to help us find – we signed up 700 Be the Match folks. Because I was trying to talk about the importance of getting people in this Be the Match database.

And then my wife did a blog going through the transplant process; she had over 10,000 hits and we actually set it up where it’s in book form where you can order it on Amazon and we’ve given it out to folks as well. So, we’ve definitely been pretty proactive here in the Tampa market with the Tampa Bay Times and Tampa Bay Business Journal and the Gulf Course Business Journal. They’ve been very nice about covering my experience and some of the ups and downs, and going into transplant when you don’t know if you’re going to make it, and how do you set up your business.

So we’ve been pretty fortunate. But it is such a rare disease, I have to explain myelofibrosis. They say what’s that, and I say it’s kind of like leukemia but red blood cells. I know my primary doctor said Nick, in 17 years you’re my only myelofibrosis patient and probably before I retire, you’ll still be my only myelofibrosis patient.

Fortunately, people like Dr. V. out there, his videos were so helpful for me early on, and I really do appreciate the senior physicians in this field. They’ve been so gracious with their time to get out there and help educate us. It really blew me away to have access to those types of materials because there’s so many questions.

I really appreciate him even being on here today; it’s so impressive.


Great. Wow, Nick, that’s so encouraging to hear about how you and your wife got the community involved and really brought awareness to such a rare disease. Dr. Verstovsek, how broadly within the oncology community, how aware are they of MPNs? There seems to be other cancers and such that have a lot more attention.

Dr. Verstovsek:

Over the last ten years, much has been done on the awareness of these conditions for the physicians themselves. You may know that for example, one of the major meetings, the professional meetings in the United States is American Society of Hematology meeting that is always done at the beginning of December. And I tell you that ten years ago, there was no session on myeloproliferative neoplasms or MPN.


No educational sessions, no scientific sessions of significance of all; it was neglected completely. We changed the field completely from 2004 on by the discovery about what is problematic, the JAK-Stat pathway abnormalities, these biological abnormalities in all the patients and all the associated genetic abnormalities that leads to progressive disease. We changed the understanding of the disease. That led to development of new medications, developing new prognosis coding systems, improvements in our ability to manage patients. And now, I should be proud probably that it is not as fast as we would like, the MPN is at the same level as any other more serious conditions like acute myeloid leukemia. We have full fledged scientific sessions on MPN on its own, presentations, oral presentations.

I would say that people in the field, because this is relatively new, still, are hungry for information; the education sessions are full. Much more can be done on education of the physicians. So it is coming there, but it is a slow process, educational process on the patient side is in parallel being done to educational professionals.


Wow, that is great. And it’s encouraging to know that the MPNs are getting the attention that they should be getting. Lindsey, I wanted to ask you, do you feel that focus on MPNs are being overlooked because of more prominent advocacy with these other conditions?


I would say that it’s similar to what Dr. Verstovsek was saying. I’ve personally been at the American Society of Hematology meeting myself, and I’ve been at a lot of other – participated in some other meetings recently.

I’ve started to look more – obviously, given what I’m doing now with the registry project in the media, there’s definitely a lot more focus on MPNs than there probably once was. I also participate in some activities in Washington, D.C. having to do with the rare disease organizations there; there’s a few different rare disease organizations. So the MPN Research Foundation is trying to participate in activities like that to make sure there’s continued focus on MPNs there.

But you know, I think that we can never do enough, really, when it comes to a rare disease. We all have to play our part and keep it at the forefront of people’s radar because until there are better therapies out there, we can never stop.


Wow, and that is great to know that the MPN Foundation is really getting more focus with the rare diseases and the work you’re doing in Washington. In fact now, I’d like to show a quick little video. There are a lot of people in the MPN community around the world that support MPN awareness and they’re doing great things. This short video will give you an idea of who those people are out there. So, I hope you enjoy this video for a few moments.


Alright, so did you guys catch Nick in there? We’re hoping to hear from now we’re going to run out of slides because there are so many folks out in the community doing such great things, and that was real cool, Nick. Lindsey, I’m dying to ask you this question. myMPN Registry; we are all really super excited about this from the MPN Research Foundation. Can you talk a little bit and tell us what it is and what the goal is?


Sure, I’d love to, thank you. So, as we’ve talked about on this webinar, there’s a lot of different experiences by each patient with an MPN.

Some people live with it for a long time, some people get a diagnosis that comes out of nowhere and it advances quickly. There’s lots of different paths that can be followed by an MPN patient. And one of the things that we’re most focused on, as I’ve stated previously, is to try and get some therapies that can help the patients regardless of where they are on that pathway. We said to ourselves, how can we better understand the pathway itself because each person’s experience is different.

People have different symptoms, they have… some of their symptoms are in their blood counts, some symptoms are more depression or they may have itching, they may have some other brain fogginess; different things. Everyone has different combinations.

So we wanted to understand that, and what we decided to do was put together what’s called myMPN. It’s a registry for patients to go in and share their experience. And in doing so, they can help to change the prognosis of all patients. Actually in the slides that you ran, there was a quote from Helen Keller and it talked about it’s not just one major mover or shaker; it’s actually everybody in the process along the way. That really encapsulates what is going on with myMPN.

We need patients each to go in and share their individual experience. We would love it if they would not do it just once but many times; keep coming back to the registry. So, the registry is structured so that we gather the information in a primary survey that has some history, background about an individual, their diagnosis, a little bit about their history and treatment, the drugs or therapies that they’re using.

But then there are additional surveys that we invite the patients to come back and fill out again and again, which collect information about what’s changing. So, there might be an event that has affected their health. Maybe they had some sort of a thrombosis, or a pregnancy, or it could be something related to their MPN or something unrelated. Then there’s another third survey called How Do You Feel Today, and that’s where we really want to understand the patient’s whole experience with the disease; how did they feel today versus how will they feel next week. Are they getting sleep, are they eating? Are they having a lot of fatigue, are they itching?

The idea is that for the many patients that we gather this information over a long period of time, then that will start to help us to understand the diseases better and over time, hopefully we can figure out if there are triggers to the disease advancing from one stage to another; trying to help to put together some of the pieces of the puzzle that currently aren’t available to researchers in the labs and in the hospitals.

We’re hoping to augment the research that’s already going on by helping to provide those researchers and doctors with day-to-day experiences of patients. So it’s a really great way to involve the patients directly in the direction that research is having.


I think this is fabulous. Is this up and running now? Is it accessible?


Yes. We launched actually earlier this month in connection with Blood Cancer Awareness Month. I’m humbled by the response; it’s just been fantastic to see how many people have really been involved and engaged and coming back to the registry.


That is wonderful. How does someone get involved? How do they sign onto this?


We have a dedicated website for the registry; it’s www.mympn.org. And on that web page they will see the link through to begin the registry process. The first thing that a user would do is set up their privacy settings. We liked the platform for this particular registry setup because it enables each patient to customize their privacy settings.

Some patients are very comfortable sharing the information about their health history and background, helping to contribute that information toward the scientific process. Some people just want to record it for their own future reference, and that’s perfectly fine.

Either way, we’re just glad that people are getting engaged. The system, myMPN system, actually does have that flexibility so the patients can determine what information, if any, they’d like to share with the researchers and the research process. That’s the first step; you set up your profile for your privacy settings, and then as I said, you start to go into the survey where you talk about your history with the disease. And then we encourage you to come back and fill out the other surveys on an ongoing basis.


Great. Well, I signed up so I’m really excited that I can be a part. Dr. Verstovsek, I wanted to ask you, you’re on the steering committee of myMPN, one of the doctor’s registry.

What kind of impact do you think this is going to have for you and the way you practice medicine and make decisions and deal with patients over the next few years?

Dr. Verstovsek:

This is a very significant step forward. Look, what we know about the current conditions, like for example polycythemia vera. Let’s talk about polycythemia vera. You have in the literature assessment of the outcome of the patients that were referred to tertiary centers in the consultations. So they would come to Mayo Clinic, or to MD Anderson, or Moffitt or any other large academic centers. And there would be possibly let’s say 500 patients with polycythemia vera that were seen over the last 20 years.

Academicians would analyze them from the time they arrived and what happened with them with therapies, and you would come up with some knowledge. But that is such a small, small part of the larger community of polycythemia vera patients. There are possibly about 100-150,000 polycythemia vera patients living with the condition here in the United States.

And what does it mean to analyze the outcome of the 500 patients that were referred to you in academic centers? It means a lot, but not too much in the larger picture. So if we have a way of having a registry where multiple, multiple people, patients from – many patients from a larger group of patients, not just those that are seen in academic centers, can participate. And we can learn how they were diagnosed in a community setting; what did the local doctor do?

What symptoms did they acquire during their lives? What therapies did they receive, and why and what happened with those therapies? Any complications with the thrombosis? Any complications through the pregnancy? Those are the issues that are mentioned already. What led them to be referred to academic centers?

We can enlarge our knowledge, better our knowledge about the disease conditions; time to diagnosis, time to progression, management in community setting and the relevant new interventions and new ways of assessing needs of the patients, and understanding life with PV, or life with ET, or life with myelofibrosis on a larger scale and see where to intervene and how in the future.


And in some ways, this really seems to overlap personalized medicine. We’re now not looking at the one scenario for PV or myelofibrosis or ET. We are looking at hopefully a huge group of MPN patients and putting those pieces in the puzzle, as we said Lindsey.

Dr. Verstovsek:

And let me add – this is really good. This is an excellent comment. Because we these days think a lot about the genetic complexity. There are patients with myelofibrosis or any other conditions that differ based on genetics, as I mentioned earlier on. But there are patients with myelofibrosis that present with anemia only, patients that prevent with very big spleen and poor quality of life.

There are different ways of people progressing or presenting with a condition not only based on genetics. What is the experience of patients that have only low blood cell count versus those that have a very big spleen; how did they fare? It’s clinical assessment and quality of life assessment and blood cell count assessment. We don’t really need to engage in extraordinary tools to learn. Genetic is one part of it, but it is much more we can learn from our on practice on the larger scale.


Dr. Verstovsek, is there really a difference between this and an observational study?

Dr. Verstovsek:

There is some difference in that observational studies are usually much more focused in a shorter time period in a selective group of patients, in academic centers usually, with much more scrutiny of the detail. So, if you have an observational study in polycythemia vera, since we’re talking about polycythemia vera, there is such a study; it’s called the REVEAL study. There is also observational study for patients with essential thrombocythemia and early stage myelofibrosis called the MOSS Study.

So, a limited number of patients with very dedicated focus to see patients periodically, collect all the data of what happens to them all the time, because they are followed by clinicians and the researchers and nurses; this is a full fledged clinical study. Without intervention, just to see what happens with them; what symptoms develop, what complications they may have with other medical problems, what happens when they’re hospitalized and why, what the reasons are for intervention for their disease.

What is the effect on their work, ability to communicate with the family, engagement in social encounters. So it’s much more focused, perhaps more in detail and in a short period of time. But it does very well complement the registry, which is much broader and for a much longer period of time. So, I encourage patients to participate in both efforts.

There is complementation between the two, and certainly encouragement from the academic and local doctors to learn from these efforts.


Doctor, what is the criteria to participate in an observational study?

Dr. Verstovsek:

Unlike the registry where really we are trying to include everybody who has the disease at different stages to see what is happening with them, registries are usually focused on the patients like the Moss Study; I mentioned it’s for patients who have essential thrombocythemia that requires therapy; and for patients with myelofibrosis that do not require therapy. So it’s a concerted effort to understand much better in detail particular groups of the patients. So there is eligibility criteria for participation in some of these observational studies, unlike the registry where we really like to have everybody.


Alright. And of course now this leads me to clinical trial. We hear a lot about clinical trials. Could you briefly describe for us the difference between a clinical trial and an observational study?

Dr. Verstovsek:

Absolutely. And see, while I was giving an example of an historical analysis of patients in academic centers that leads to information how to manage patients and what to do about them. But these are the patients who are referred to academic centers, usually – most of the time – because they are not doing well and there is a need for intervention. And most of the time, we talk about treating patients that need to have something corrected.

They suffer from anemia, they suffer from a blood clot and this is where we intervene, and this is where most of the work in academia is focused on. This is where we do clinical studies. Clinical studies in MPN, most of the time and in the United States in particular, are focused on correcting something that is wrong; improving quality of life, decreasing the spleen, improving the anemia in ET or PV.

We would like to treat people for what they suffer from; high platelets, high red blood cell count, big spleen, symptoms. Intervention studies in myelofibrosis for example are needed as we try to prevent another clot in patients with ET and PV. So we are moving from interventional studies to prevention studies. And to get proper assessment of those patients in need, observational studies are needed to learn about the experiences.

A registry is needed to learn about a wide spectrum of patient experiences for us to identify groups that would, for example, benefit from prevention rather than waiting for them to suffer and then prescribe something to correct it. So prevention studies will be major developments, in my view, from observational studies to see where we need to intervene once we observe.


Very interesting. I had not even heard of that. That is fabulous.

Both Nick and Andrea, you have both participated in trials. And Andrea, I would like to go to you first. You’ve been in several clinical trials. What was your journey, and what was beneficial in participating in those?


Well, frankly the whole point of my participation was not only to feel better and maybe arrest the disease to an extent, but I felt since it was such a rare disease I had an obligation to try to advance research by using me as a live guinea pig. Instead of donating my body to science later; let’s try to do something now. Of course I had selfish reasons, as well. What happened with the early studies which were done nine years ago or so, several of them just were very toxic for me, and for different reasons.

But different things happened; a couple landed me in the hospital. But I knew there was something out there, working with Dr. Verstovsek, working with my local hematologist; I knew that if I didn’t try and didn’t do things, that I certainly wasn’t going to get any better. So, when we hit on CYT387, which I bugged Dr. Verstovsek about because I read about it, and I said it sounds right for me; he said yeah, I’m gonna get it, I’m gonna get it.

He finally did. It worked for five years. The company was bought out by another company. I have a similar drug; it doesn’t seemed to have worked as well but for five years I’ve been transfusion independent, which is huge.

While my anemia has not been anywhere near normal, it’s certainly functional and I’ve been great. Now we’re looking for something else. I’m a perseverant person, and the first couple of trials didn’t work but I didn’t die, and I didn’t get any worse. And the fourth and fifth trial did work, so I think I have to encourage people to not give up. We’re all different, and we talk about that in our support group. Everybody is different. We are so individualistic in this disease that we can listen to other people, but we have to really listen to our bodies and ourselves.

So now, it’s that period of kind of treading water; what do we do next? There are some things that Dr. V has mentioned, there are some things that I’ve been reading about that I’m going to quiz on him when I see him next. And so it’s a little discomforting right now because something has to happen.

 But I’m confident and positive that there will be something out there, but if I don’t try, we’ll never know and a patient behind me – we have people in our group who are 20 years old and 30 years old. They’re panic-stricken. So hopefully I can help them, maybe; if I don’t someone else does.


You know, Andrea, you’re very inspiring. Me kind of being new to the game, too, it’s very inspirational to hear about your perseverance and your attitude. I love the idea that you’re also there for the younger people in your group to show that this is the journey but it’s working, and there’s hope on the horizon. Nick, if you could tell us a few things, a few thoughts about your journey through your clinical trial and if there was a benefit?


Yeah, sure. It’s kind of interesting for me initially when I was diagnosed, the local doctor said well, you’re so lucky; you have Moffitt right here in your backyard. So I went to Moffitt Cancer Center, and they’re very good but the hematologist said basically the same thing that the initial doctor who did the diagnosis; well, you’re a good candidate for transplant, just wait a year, watch and wait a year and then go to transplant and so be it. But you do the research, and transplant is a pretty tough deal.

When I went to Moffitt, basically that’s what the doctor said, is we’ll see you once a month or every other month and sometime next year you’ll go to transplant. I said doctor, I can’t just sit around and wait and just sit back and placidly have this ticking time bomb hanging over me without trying to do something. And like Andrea, I also felt I owed it to the other patients that maybe being part of a trial that we’ll learn something.

I was hoping – my goal was to try to postpone my transplant for several years. But I said being part of the trial, maybe they’ll learn something good or bad that will help other patients as well. And that’s where I was very fortunate to land at MD Anderson with Dr. Pemmaraju.

They set me up on azacitidine and Jakafi. Moffit’s pharmacy doesn’t even carry Jakafi. So, the No. 1 tool in the toolbox for MPN patients, they don’t even have it there. That’s where we did the trial, and I think with the trial the one thing that people need to know, you’re under a much higher level of scrutiny. Dr. Pemmaraju at Moffitt, they’re having me come in every 30 days for blood work, 60 days to visit with the doctor. Pemmaraju, he’s seeing my stuff every week; they’re looking at my numbers. I go fly out there once a month to meet with them.

And so you have an extra layer of coverage. And ultimately, it was Dr. Pemmaraju who said hey, Nick – by the way, CAL-R, JAK2 was my mutation, which also people need to know who’s doing what. But in October, Dr. Pemmaraju noticed my BLAST had spiked from 1 to 5. He said Nick, you’ve got to go for transplant because if it turns into AML, your prognosis really gets a lot worse.

And I’ll always remember the look on his face. He just looked at me and said, time to go. That’s why I think his higher level of watching me gave me that sense because there is some controversy out there as to when to go to transplant. When do you go? Do you wait? Is there a certain number you have to hit?

I felt that his advice, based on all the doctors I had met with, was very right on point and I appreciated their – there’s a great group of folks there, and I certainly think that my success – and I don’t know if Dr. V. agrees, but me being on Jakafi, actually they kept me on it through the transplant; I think that helped me because I’ve had a fairly good transition through the transplant process. And I think it was based on the work that Dr. Pemmaraju did.


Great. Nick, you’re very encouraging. Dr. V, since Nick brought that up, how do you feel about that, that he was kept on Jakafi?

Dr. Verstovsek:

I would say that both Andrea and Nick have amazing stories to say and to tell us their experience and to learn, and really inspiring stories. I’m glad that there was a time period to enjoy some good quality of life and control of the disease for Nick. But in this tough disease, myelofibrosis, it can be controlled for a long period of time but it does change as the medications really don’t work forever. And sometimes it is necessary to go to transplant and we treat patients up to the transplant with Jakafi to maintain the benefit that might still be there.

After the transplant it’s not necessary anymore, of course. But that is one of the experiences that people need to understand that there is a potential for medications to control the disease, either through clinical studies and the first option may not work, the second may not work and there are always – and we try to have those here – multiple other options through conventional medications or through investigation medications to help patients. And if the transplant is necessary, we will do it when the time comes and it is a personal decision most of the time.

It’s not really one number; it has to be between the physician and the patient.


Absolutely, absolutely. Lindsey, I’d like to ask you the last question, and we do have a few questions from our viewers. We’re talking so much about clinical trials and raising awareness, how do we do that, and how does raising awareness affect the clinical trials? I know like barely anything about clinical trials.


Sure, that’s a great question. In fact, one of the things that we try to do at the MPN Research Foundation is to let people know about clinical trials that are going on so that to the extent that someone is looking for something better, kind of like Andrea was or has been. Maybe they haven’t been made aware by their own doctor of an ongoing clinical trial.

And by using our social media and our website, etc., we can make people aware of that. I think that’s – it’s a great way for people who are patients but also I think that it’s important for patients to understand the message that Andrea delivered that I think was so eloquent. Which is unfortunately, drugs don’t come onto the market without brave patients like Andrea and Nick who are willing to put themselves out there and take something that is yet to be approved by the FDA. Because the FDA has very specific standards and they scrutinize the trials very carefully. But at the end of the day, we need patients to volunteer for these trials.

I think it’s wonderful when patients do that, and it’s even better when the results are great, obviously. But I think from an educational standpoint, we as an organization and I know others try and help people to understand what their options are, what the process of a trial is. We’ve actually in the past put together graphics to help people understand how trials progress. We actually have been involved ourselves in discussions with the FDA on how trials can be more focused on the patient experience.

So I think that things are definitely going to improve, and hopefully we’ll see more drugs that are available for patients that will provide more relief very soon.


That’s very encouraging. I absolutely agree with both of you how people like Nick and Andrea, and could be myself as well one day in the future; we can’t move forward. We don’t want to be stuck and not be able to have those results. I’m going to shift gears a little bit. We’re ready to take a question. Dr. Verstovsek, this question I believe is geared for you, and I’m going to go ahead and read it. Dolf from Holland is watching.

How is research internationally organized? Dolf goes on to say lots of trials with MPN hematologist around the world, and we can find them on clinicaltrials.gov. But is there also some specific research in some countries that’s not registered at that site? Is that something you can address?

Dr. Verstovsek:

That’s a very good question. There is an attempt by clinicaltrials.gov, which is actually the federal site, to collect all the clinical trials that are being conducted not just in MPN, obviously, but in any other condition. And the effort is really significant with the prospect of utilizing that for patients as well to identify the sources, if not only for the physicians. It is really the professional side but the patients can search that as well. Perhaps it’s too cumbersome sometimes to find information, but underlying the attempt is very well received at least in the professional circles.

If there are any other studies in other countries that are not registered, of course it’s a possibility this is the Federal Government of the United States. I am not aware, didn’t really look into that, whether other governments in Europe – and I would say some they do, maintain something similar for their own countries like in Germany or France.

This is, after all, one of the best sources of information, clinicaltrials.gov, for clinical studies.


Great, thank you so much. We’re going to take one more question; unfortunately we’re almost out of time. But we have another event coming up October 7, and we will definitely answer many of these questions that have come through tonight. And in fact, Dr. Verstovsek will be on that panel as well, on the upcoming one. I do have another question for you, Dr. V. It’s from Julie. Do you have any tips for patients to educate their primary care provider about MPNs? And she said more specifically, PV.

Dr. Verstovsek:

The information that is available to the patients through the MPN Educational Foundation or through the information that can be gathered from the academic websites for the patients typically is organized very well for anybody to understand it.

I have patients that are engaged with us together, as we said, in that educational effort for all participants to be at the same level. That means not just us in a referral center or patients, but also referring doctors. So the best information to a primary care doctor, and I assume that was what the question was about, the local oncologist; that is delivered by us as experts in MPN and by the patients.

By bringing that information to the attention to a local doctor is valuable. There is rarely any doctor that would not appreciate so much the involvement of the patients on the part of education, and bringing the new information to the doctor. Look, the local hematologists and oncologists, that’s a hero for me.

That doctor, a female or a male, has to take care of not just the PV; it has to take care of patients in brain cancer and kidney cancer and lung cancer; both of the perspective of life and professional commitment in front of you for that particular doctor. That is the doctor that is doing all of this at the same time, and has to catch up. So we have to also realize that it’s really difficult to follow all the updates and all new developments in the field for PV or any other MPN. So, I know that doctors appreciate when the patient is engaged and brings information to them.


Excellent advice. Julie, when you asked about their primary care providers as well, I’m sure Dr. V. feels the same way; we can take a look at Andrew Schorr and his wife Esther who created and head up Patient Power. Esther is very vocal about being a very educated care provider.

And through everything we’ve talked about today with myMPN registry or the MPN Research Foundation, or all the things that Dr. Verstovsek brought up about how he educates his patients, the primary care providers definitely should be in that conversation. Those things are all welcome.

Well, we are just about to wrap up. We’ve had a really great discussion here today, and I would like to get some final thoughts before we say goodbye. We are a little pressed for time. Nick, if you can give us a few quick, final thoughts and we’ll just go around after that.


Well first, Beth, great job on the hosting. It was very impressive, and glad to be able to participate. And certainly want to thank all the panelists to try to keep moving folks forward. And hopefully some day the people behind us won’t have to worry about the same issues we’re dealing with today. And appreciate Dr. V. taking the time especially, and all the great work that his team is doing, too. So, wish everybody the very best, and thanks, and great job, Beth.


Thank you, Nick, and right back at you; it’s been a pleasure having you. Andrea, some final thoughts from you?


Yes, thank you everyone for participating, it’s been great. It’s my first time. I would like to say that one thing I found successful was to print some pamphlets up and to distribute to my doctor and leave them out so people are aware of our meetings. And also, to really encourage people to look very seriously into clinical trials and going to an institution like MD Anderson that specializes in their disease so that they can get the best care and educate themselves the best.


Thank you, Andrea. Those are great words of wisdom and we appreciate all of your feedback, having been in this journey for almost 20 years. Lindsey, can you give us a few final thoughts?


Sure. Thank you very much for including me in this conversation; it’s been wonderful to join Dr. Verstovsek and Andrea and Nick.

I encourage anyone who has not yet visited myMPN to please visit us at www.mympn.org. If you have any concerns or questions, please don’t hesitate to reach out to me. I encourage everyone, like Andrea said, to try and get involved in your care, whether that’s through myMPN, through joining a clinical trial, and certainly just being an advocate for your own health and going to go visit those doctors armed with the information that you can.


Thank you. Great words of wisdom. And again, we are very excited about the myMPN registry, so thank you for sharing that today. Dr. Verstovsek, we appreciate you being here. Some final words that you can give to our audience?

Dr. Verstovsek:

I really appreciate you having me on the panel; it was a wonderful experience.

And certainly what we have achieved together, I’m sure for many patients is to engage, engage and be educated, learn about what you have, learn about the abilities to help you if you are not already. Be an active participant because these conditions are there to stay with you; we don’t have a cure yet. We are working toward it. But life can be good, life can be controlled well. We have means and we are trying to do our best. So be an active participant with the local doctor, with expert. Make the most out of it and if you need a second opinion, search for it. Basically, do not give up. Be your own advocate, if you like.


Wonderful, and I couldn’t agree with you more. It’s just very inspirational and those words will resonate with many of our viewers, I know.

Thank you, I’ve enjoyed speaking with such experts on this panel today, and thank you for the words of wisdom and best of good luck and health to all of our viewers, and Nick and Andrea. Thank you.

Health Cost Literacy: “How much is that?”

The title of this post asks the $3.5 trillion-with-a-T question in American healthcare: how much is that? It often feels like healthcare is split into two camps, with one side working away feverishly to find more cures for life-threatening conditions like cancer and ALS, while the other side is working at an equally feverish pace to figure out just how many millions of dollars they can make of the latest breakthrough.

A recent example of this Tale of Two Healthcares was the roaring headlines about the first FDA-approved gene therapy, Kymriah (tisagenlecleucel), for leukemia. The business side of healthcare was ecstatic, pricing the drug at $475,000, which made Wall St. happy, and Novartis (the drug’s maker) ecstatic. The patient side of healthcare? Not so much.

Kymriah is an extreme example of healthcare pricing, but even trying to get a CT scan can turn into a trip down the rabbit hole, if you try to find out before the scan how much it will cost you. Asking “how much?” can seem like shouting down a well the first time you do it – you’ll hear an echo, because the person you’re asking will likely say “how much?” right back, in total shock at the question. However, asking questions is how we get answers, right?

Here are tips for asking “how much is that?” and getting meaningful answers:

  • Find out if your insurer has a cost-estimator tool. If so, use it. For everything required for your care. You’ll need the insurance billing code for the test, scan, or procedure (called the CPT code), so get that from your doctor’s billing office.
  • Use online price-check tools like Clear Health Costs or Fair Health Consumer to reality-check the pricing information you get from your insurer’s cost-estimator tool.
  • When your doctor refers you to a lab for testing, or an imaging center for scans, ask if they know what the cost is. They likely won’t at first, but the more of us who ask the question the more they’ll want to know the answer.
  • Call around to labs and imaging centers in your insurer’s network to ask about their cash price for the test or scan that’s been ordered for you. Depending on the cash price, you might be better off not using your insurance, and actually paying cash for the test or scan. If you have a high-deductible plan, you’ll need to assess which medical services are worth going off-the-books for if you haven’t yet met your annual deductible.

I know a lot about “how much is that?” because I was uninsured for five years after my own cancer treatment ended. I discovered that asking the question got me the answers I needed, and I could choose the providers that could give me a cash price for the mammograms and follow-up oncology services I needed. I’ve continued to use the simple question “how much is that?” every time a doctor has ordered tests or scans, because even with insurance, you’ll wind up with a bill for some part of the service.

If we all work together, asking “how much is that?” before receiving any medical service, we’ll start to shift the system, and the culture of healthcare. It takes a village, not just to raise a child, but also to change a status quo.

It’s your turn. Start asking.

Clinical Trials – Patients ARE the Pioneers

Editor’s Note: After a long and resilient battle with primary peritoneal cancer, Roberta Aberle, 53, of Auroro, CO passed away on November 1, 2017 with her husband David Oine at her side. Even as she battled her own cancer, Roberta was a tireless advocate for patient care, hoping to improve the lives of others also fighting life-threatening illnesses. She will be greatly missed by all who knew her.

“The pioneering spirit is less about thinking up new ideas as ridding ourselves of dogma and old habits that hold us captive in thinking.” Bertrand Piccard http://bertrandpiccard.com/home

Profound. Who wants to be captive in their thinking? No one I can name. Especially in the world of healthcare. Medical sciences are far surpassing dogma in many aspects of delivering care to patients – in technology, in documentation, in processes and structure, in diagnostics, in pharmaceuticals and other interventions such as surgical techniques all the way to preventative care. Some diseases have been eradicated entirely, others are manageable and have become chronic rather than life threatening.

It is invigorating to have even a small part in innovations with the potential to alter and improve the landscape of health and well-being. In last week’s broadcast of Mythbuster’s #3 – Do Patients Have a Voice While Participating in Clinical Trials? Our host, Andrew Schorr brought up the notion of being perceived as a pioneer in cancer treatment with each of our respective roles in clinical trials. Our panel was comprised of an oncologist and a clinical trial navigator (a resource who pairs patients to relevant trials)and myself, a patient advocate and participant in multiple clinical trials.

One trial in which I participated led to the provisional release of a therapy added to front line treatment for Ovarian, Fallopian Tube and Primary Peritoneal Cancer who test positive for either the BRCA 1 or 2 inherited genetic mutations. Addition of the oral medication researched has since shown astounding results for patients with these forms of cancer that prolongs disease free progression an average of 19.1 months. https://www.lynparza.com/ovarian-cancer-treatment.html

I personally never considered myself a pioneer, but in this context, I suppose it fits. Without patients, researchers and oncologists exhausting every resource to identify what can extend our life or achieve a cure for incurable forms of cancer; how can we validate new inroads to treatment?  Regardless of how it is termed, I take great pride in participating in clinical trials to prove efficacy for patients of today and the future. Pioneer. Forerunner. Research Subject. Terms mean less  to me than the knowledge that if just one patient considers a clinical trial as a result of seeing our webinar, it is validation enough my involvement matters.

Shortly after the broadcast, I was talking to a new acquaintance at a gathering about the program and my personal experience with various clinical trials. As I detailed my journey for her, she asked a time or two, so “ALL you ever did was take experimental medicines?” Communication breakdown. But yet, I was quite happy she expressed her question in the way she had so I could clarify. My response was swift, “No. In actuality, in only one clinical trial was the medication ‘‘experimental’. Other than one, I was enrolled in only Phase 3 or higher trials where the medications were known to be effective, they just needed more data to understand dosage, frequency and when to use in combination with existing chemotherapy drugs to amplify the power of each.”

Despite being in multiple clinical trials, I have yet to achieve remission and the elusive cure for my form of cancer; yet, I still chime praise for each trial I’ve been enrolled in. I consider each an opportunity a peek into the future of cancer management. While I cannot claim certain things regarding my cancer as a result of clinical trials, each one has earned me quality time with loved ones or has been a bridge to a new treatment option not available to me were it not for the commitment of researchers, oncologists and patients worldwide.

When you view the webinar rebroadcast or in summation, my hope is that you will be receptive to clinical trials as an additional avenue available to you. Beyond our specific topic of whether patients have a voice in clinical trials, in our segment, our message is clearly that patients do retain their voice during a clinical trial. Of equal importance, collectively our outcomes and first hand experiences are an even more powerful voice chiming their merits. In many ways the patient voice is important not just during, but extends long past the time parameters of trials.


Clinical Trial Mythbusters: Do Patients Have A Voice While Participating in a Clinical Trial?

For survivors Roberta Alberle and T.J. Sharpe and so many others, cancer was an unwelcome intruder that suddenly demanded their attention. Both became proactive and engaged, vocal patients, doing research about their treatment options and gaining access to clinical trials that made a HUGE difference.

Watch to learn:

  •       Do patients still have a voice during the clinical trial process?
  •       What is a clinical trial navigator?
  •       How can patients help their healthcare team be more effective in locating trials for them?
  •       More about clinical trial resources

Clinical Trials Mythbusters: Do Patients Have A Voice While Participating in a Clinical Trial? from Patient Empowerment Network on Vimeo.


Living Well with Multiple Myeloma – Staying in Tip Top Shape

Exercising With Multiple Myeloma – Staying in Tip Top Shape

Living Well With Multiple Myeloma from Patient Empowerment Network on Vimeo.

How do you exercise if you have myeloma? Should myeloma patients avoid exercise all together? Melanie House, a physical therapist at the University of Iowa Hospitals and Clinics specializing in prescribing exercise, shares how and when to exercise with myeloma.  Jim Bond, aka James Bond, a 25-year, stage III myeloma survivor and Matt Goldman, a 7-year myeloma survivor, shares how they stay active trough cycling and how they challenge themselves to stay in tip-top shape.

Clara Health – #PatientsHavePower

We are proud to announce that we are official participants in the Patients Have Power campaign run by Clara Health. Clara Health believes that all patients should have the power to access the most advanced healthcare available. But they understand that getting started on the journey can be overwhelming. So, they built Clara to be the simplest, fastest, most patient-friendly way to connect you to the newest treatment options.

This campaign is a Boston based initiative aligning the world’s epicenter of healthcare around one message: Patients Have Power. The movement is comprised of patients, caregivers, allies, nurses, doctors, researchers, members of the healthcare workforce and anyone and everyone who believes in patient power all around the world.

Patient Empowerment Network believe patients should be empowered with the knowledge and resources they need to hold the ultimate power in their healthcare journey and are proud to say that we live the mantra: Patients Have Power.

Interested in getting involved? Show your support by participating in the following:

  1. Declare your support: Join our #PatientsHavePower campaign on Twitter. Click to tweet.
  2. Join the Thunder: Add your voice on Thunderclap to help the message spread! Register here.
  3. Twitter Chat: Join the #PatientsHavePower chat on Thursday, September 7 at 3:00 PM EST.
  4. Patients Have Power Signs: On Thursday, September 7 take a picture of yourself holding up a Patients Have Power sign and share on social media.