AML Treatments and Clinical Trials

When it comes to treatment, AML patients and their care partners have much to consider. There are often many options available, each with advantages and disadvantages. Some people may seek clinical trials, others may have few feasible options. Understanding treatment options, goals, and what to expect are vital to achieving the best possible outcome for you.

More resources for AML Treatments and Clinical Trials from Patient Empowerment Network.

How is Acute Myeloid Leukemia (AML) Treated?

How is Acute Myeloid Leukemia (AML) Treated? from Patient Empowerment Network on Vimeo.

 When diagnosed with Acute Myeloid Leukemia (AML), understanding available treatment options can be overwhelming. Dr. Alice Mims, an AML specialist, provides an overview of AML therapies and discusses factors to consider when deciding on an appropriate therapy with your healthcare team.

Dr. Alice Mims is a hematologist specializing in acute and chronic myeloid conditions. She serves as the Acute Leukemia Clinical Research Director at The Ohio State University Comprehensive Cancer Center – James.

See More From The Pro-Active AML Patient Toolkit


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Effective AML Combination Treatment: Pairing Old and New Therapies

 

AML Treatment Options: What’s Available?


Transcript:

For the past 30 years, we’ve had the same treatment options, which have been standard intensive induction chemotherapy that weren’t really tailored to individual patients and had significant toxicity. And not necessarily effective for all AML genomic subtypes.

Now we have quite a bit added to the treatment arsenal for AML, including continuing intensive induction chemotherapy for patients who are appropriate. There’s also been the addition for newly diagnosed patients for hypomethylating agents and a new BCL-2 inhibitor called Venetoclax. IDH inhibitors for patients with IDH1 and IDH2 mutations. The addition of FLT3 inhibitors for patients either newly diagnosed or with relapse or refractory disease.

And liposomal daunorubicin and cytarabine in for patients with AML with MDS related changes or therapy related AML that are newly diagnosed. Lastly, there’s also a hedgehog inhibitor, glasdegib, that’s been approved for newly diagnosed AML patients in combination with low dose cytarabine.  

So, when working with patients, there are multiple factors that we take into consideration when coming up with a treatment decision together and it really should be a team approach. But one of the most important things is trying to understand the patient’s goals of care.

Because different treatments have different expectations, side effects, toxicities that we want to be sure we’re all aligned when we’re making a treatment decision together. Also, other features that we take into account can be age. Other comorbidities, including other diagnosis such as cardiovascular disease, diabetes and other medical issues patients may have.

So, for roles that patients have in making these decisions, they should know that they’re their own best advocate. And so, as you’re getting to learn your oncologist who’s helping you make these treatment decisions, it’s very important that you talk about things that are important to you in regards to quality of life, overall goals for your life. Ask questions in regard to side effects and expectations for outcomes for potential treatment. Whether they’re curative or more palliative, which can extend life. And for quality of life, it may not be curative for AML.  

So, AML really was considered a single disease 30, 20 years ago. Now we really know it’s likely dozens of diseases based off of looking at molecular features of an individual patient’s AML. So, it’s very important to try to understand what genomic features your AML may have, meaning DNA mutations that are just present in the leukemia cells. Chromosomal changes as well. And then understanding if, based off that information, that that may afford you additional treatment options other than the current standards of care.  

Effective AML Combination Treatment

Pairing Old and New Therapies

Effective AML Combination Treatment: Pairing Old and New Therapies from Patient Empowerment Network on Vimeo.

With advances in AML research and a number of new treatments, can older therapy types still play a role in care? Dr. Alice Mims discusses pairing early AML treatments with new agents to boost their effectiveness.

Dr. Alice Mims is a hematologist specializing in acute and chronic myeloid conditions. She serves as the Acute Leukemia Clinical Research Director at The Ohio State University Comprehensive Cancer Center – James.

See More From The Pro-Active AML Patient Toolkit


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Key Genetic Testing After an AML Diagnosis

 

How is Acute Myeloid Leukemia (AML) Treated?

 

AML Treatment Advances: What’s  New for YOU?


Transcript:

So, in regards to older treatments and being effective, seven plus three, which is an intensive chemotherapy, is still the standard of care treatment for patients with favorable risk AML, if they’re candidates for intensive treatments because it is potentially curative. And 7 + 3 is also the backbone for newly diagnosis for patients with FLT3 mutations, we add a FLT3 inhibitor called Midostaurin onto that, as it’s shows to improve overall survival with the addition of that compared to just the chemotherapy alone.

And also, hypomethylating agents, which are a less intensive treatment, were the standard of care for patients who couldn’t tolerate intensive chemotherapy.

And now we’re seeing the addition of other agents being added to this, like the BCL2 inhibitor of Venetoclax.

And recent data in phase 3 trial comparing the hypomethylating agent alone versus adding that agent did show an overall survival advantage. And so, these are definitely evolving, and I think as we are learning more about targeted therapies and how they can best be used in combination with chemotherapy other than single. Agent. But you give two targeted therapies together and having even better outcomes. We hope we continue to make improvements from where we were even just five years ago and do a better job for.

Confused About Immunotherapy and Its Side Effects? You Aren’t Alone

“You don’t look like you have cancer.”

More than one patient undergoing immunotherapy to treat cancer has reported hearing statements like that. Immunotherapy is one of the recent advances in cancer treatment that belie the stereotypes about the effects of cancer treatment. 

The side effects of immunotherapy are different from those associated with chemotherapy and radiation. However, that does not mean immunotherapy does not have side effects. Patients and care partners need to be aware of these potential side effects and to be vigilant in addressing them with their oncologists because they can signal more serious complications if left untreated.

What is Immunotherapy?

Despite the increase of immunotherapy treatment options in recent years and considerble media attention paid to advancements in this field, there remains confusion about immunotherapy and its side effects. Many cancer patients are unaware of whether immunotherapy treatments are available for their specific diagnosis. Others don’t know that genetic profiling of their tumors is usually required to determine if immunotherapy is an option and not all treatment centers routinely conduct genetic profiles of tumors. A  survey by The Cancer Support Community found that the majority of patients who received immunotherapy knew little to nothing about it prior to treatment and were unfamiliar with what to expect.

Immunotherapy works by manipulating the patient’s immune system to attack cancer cells. It is perceived as gentler and more natural than chemotherapy and radiation, without the same destructive effect on the body’s healthy tissues.  This, combined with a lack of prior understanding of immunotherapy, can lead patients and care partners ill-prepared for possible side effects.

Furthermore, immunotherapy is a category of therapies, not a single type of treatment. There are a variety of immunotherapy drugs, most of which are administered via infusion.  Side effects will vary by drug, the cancer and its location, treatment dose, and the patient’s overall health.

The following are the most common types of immunotherapy.

  • Checkpoint inhibitors use drugs to block proteins in the patient’s immune system that would otherwise restrain the immune system, often referred to as taking the “brakes” off the immune system.
  • CAR-T therapy modifies the patient’s T-cells in a lab to enhance their ability to bind to cancer cells and attack and kill them.
  • Oncolytic virus therapy uses genetically modified viruses to kill cancer cells.
  • Another therapy uses cytokines (small proteins that carry messages between cells) to stimulate the immune cells to attack cancer.

Immunotherapy can be part of combination therapy. It might be combined with chemotherapy. It might be used to shrink a tumor that is then surgically removed.  Or multiple immunotherapy drugs might be used simultaneously.

What Are The Side Effects?

With immunotherapies, side effects typically occur when the immune system gets too revved up from the treatment. The most common side effects for immunotherapy treatments are fatigue, headache, and fever with flu-like symptoms. Some people also experience general inflammation often in the form of a rash. Many melanoma patients report blotchy skin discoloration, called vitiligo, during treatment. These milder side effects can usually be managed with over-the-counter remedies and adjustments to daily activities.

For checkpoint inhibitors, the fastest growing segment of immunotherapy treatments, mild side effects occur in 30% – 50% of patients. Serious side effects typically occur in less than 5% of patients. (See “Understanding Immunotherapy Side Effects” from the National Comprehensive Cancer Network and the American Society of Clinical Oncology.)

Less common side effects are blisters, joint pain, thyroid inflammation, and colitis (inflamed colon resulting in diarrhea with cramping). Some patients who receive CAR T-cell therapy develop a condition known as cytokine release syndrome, which causes fever, elevated heart rate, low blood pressure, and rash. 

In rare cases, immunotherapy has resulted in lung inflammation, hepatitis, inflammation of the pituitary, and detrimental effects on the nervous and endocrine systems. In most cases, the conditions clear up when treatment ends.  However, there have been outcomes in which immunotherapy caused diabetes or tuberculosis.

“Overall there are fewer side effects [with immunotherapy],” explained Dr. Justin Gainor, a lung and esophageal cancer specialist at Mass General during an Immunotherapy Patient Summit hosted by the Cancer Research Institute. “But the immune system can affect anything from the top of the head down to the toes. Any organ has the potential to be affected.”

As the application of immunotherapy has expanded, so has our understanding of the potential side effects. Like most medical treatments, how one person responds to immunotherapy can be different from another even when the cancer diagnosis and drug therapy are the same.

The essential thing patients and care partners need to know about side effects is they should always be reported to their oncologist or nurse oncologist.

Why Patients Should Talk to Their Provider About Immunotherapy Side Effects

Because immunotherapy has created newer therapy options, there isn’t the volume of experiences as with older treatments. The infinite number of variables that patients provide once a treatment moves beyond clinical trials and into the general patient population generate more diverse outcomes.  And, as most therapies are less than 10 years old, there hasn’t been an opportunity to study the long-term effect of these therapies. This is why oncologists advise patients and their caregivers to be extra vigilant in noting any changes experienced during and after treatment.

Many side effects are easy to treat but medical providers want patients to be forthcoming in discussing any and all side effects. This is in part to improve understanding of side effects, but also because a mild cough or a case of diarrhea might be harbingers of a more systemic issue that will grow worse if left untreated.

Patients should not be hesitant to discuss side effects because they fear they will be taken off immunotherapy.  Sometimes a pause in treatment might be necessary, but the earlier the oncologist is made aware of a side effect, the less likely that will be necessary.

In addition, patients undergoing immunotherapy should always take the name(s) of their immunotherapy drugs and the name of their oncologist when seeing medical professionals outside of their cancer treatment team. This is especially important when visiting the ER.  Because immunotherapy drugs are newer and highly targeted to certain cancers, many medical professionals remain unfamiliar with drug interactions and treating related side effects.

Immunotherapy On The Rise

Immunotherapy treatments have resulted in reports of remission in cases that would’ve been deemed hopeless just five or 10 years ago.  The Federal Drug Administration (FDA) has approved various immunotherapy treatments for melanoma, lung cancer, head and neck cancer, bladder cancer, cervical cancer, liver cancer, stomach cancer, lymphoma, breast cancer, and most recently bladder cancer.  (Here is a list of  immunotherapies by cancer type from the Cancer Research Institute.)

“It’s revolutionized how we treat our patients,” says Dr. Gainor of Mass General about immunotherapy’s impact on lung and esophageal cancer.

Advances in immunotherapy research and trials continue to generate optimism and excitement. A clinical study in Houston is looking at using immunotherapy to prevent a recurrence. Researchers in Britain recently announced a discovery that might lead to advances in immunotherapy treatments to a much broader array of cancers.

While there is excitement around the field of immunotherapy and it has resulted in unprecedented success in treating some previously hard-to-treat cancers, it remains an option for a minority of cancer diagnoses.  It works best on solid tumors with more mutations, often referred to as having a high-mutational load or microsatellite instability (MSI) high. And it is not universally successful for every patient.

With hundreds of clinical trials involving immunotherapy alone or in combination with other therapies, it is certain more treatment options are on the horizon. As more therapies are developed and more patients with a greater variety of conditions undergo immunotherapy, we will also increase our understanding of potential side effects.

Side effects should not dissuade patients and care partners from considering immunotherapy if it is available or from advocating for genetic tests to deteimine if it is an option. Many patients undergoing immunotherapy have previously undergone chemotherapy and report that the side effects are fewer and milder by comparison.  The important thing is that patients and their partners know what to expect and communicate with their treatment team.

If the next 10 years in immunotherapy research and development are anything link eth elast 10, we can expect more exciting advancements in the battle against cancer. For more perspective on what’s ahead for immunotherapy see the Cancer Research Institute’s article: Cancer Immunotherapy in 2020 and Beyond.

Understanding Clinical Trials: A Jargon Buster Guide

When it comes to cancer treatment you or a loved one may be considering participating in a clinical trial as a treatment option.  Clinical trials are designed to evaluate the safety and effectiveness of a treatment. They may involve researchers administering drugs, taking blood or tissue samples, or checking the progress of patients as they take a treatment according to a study’s protocol.

Learning about clinical trials can be a steep learning curve – not least because the process comes with a lot of new terms, acronyms and jargon.  To help you, I’ve put together this list of the most common terms you will find when you are researching clinical trial information. This is not an exhaustive list but it is a helpful starting point. At the end of this article you will see links to find more information.

Adverse Effects (AE)

Also called Adverse Events, or Adverse Drug Reaction, AEs are any harmful event experienced by a person while they are having a drug or any other treatment or intervention. In clinical trials, researchers must always report adverse events, regardless of whether or not the event is suspected to be related to or caused by the drug, treatment or intervention.

Arm

Subsection of people within a study who have a particular intervention.

Bias

Bias is an error that distorts the objectivity of a study. It can arise if a researcher doesn’t adhere to rigorous standards in designing the study, selecting the subjects, administering the treatments, analysing the data, or reporting and interpreting the study results. It can also result from circumstances beyond a researcher’s control, as when there is an uneven distribution of some characteristic between groups as a result of randomization.

Blinding

Blinding is a method of controlling for bias in a study by ensuring that those involved are unable to tell if they are in an intervention or control group so they cannot influence the results. In a single-blind study, patients do not know whether they are receiving the active drug or a placebo. In a double-blind study, neither the patients nor the persons administering the treatments know which patients are receiving the active drug. In a triple-blind study, the patients, clinicians/researchers and the persons evaluating the results do not know which treatment patients had. Whenever blinding is used, there will always be a method in which the treatment can be unblinded in the event that information is required for safety.

Comparator

When a treatment for a specific medical condition already exists, it would be unethical to do a randomized controlled trial that would require some participants to be given an ineffective substitute. In this case, new treatments are tested against the best existing treatment, (i.e. a comparator). The comparator can also be no intervention (for example, best supportive care).

Completed

A trial is considered completed when trial participants are no longer being examined or treated (i.e. no longer in follow-up); the database has been ‘locked’ and records have been archived.

Control

A group of people in a study who do not have the intervention or test being studied. Instead, they may have the standard intervention (sometimes called ‘usual care’) or a dummy intervention (placebo). The results for the control group are compared with those for a group having the intervention being tested. The aim is to check for any differences. The people in the control group should be as similar as possible to those in the intervention group, to make it as easy as possible to detect any effects due to the intervention.

Efficacy

How beneficial a treatment is under ideal conditions (for example, in a laboratory), compared with doing nothing or opting for another type of care. A drug passes efficacy trials if it is effective at the dose tested and against the illness for which it is prescribed.

Eligibility Criteria/ Inclusion and Exclusion Criteria

Eligibility criteria ensures patients enrolling in a clinical trial share similar characteristics (e.g. gender, age, medications, disease type and status) so that the results of the study are more likely due to the treatment received rather than other factors.

Follow-up

Observation over a period of time of participants enrolled in a trial to observe changes in health status.

Informed Consent

A process (by means of a written informed consent form) by which a participant voluntarily agrees to take part in a trial, having been informed of the possible benefits, risks and side effects associated with participating in the study.

Intervention

The treatment (e.g., a drug, surgical procedure, or diagnostic test) being researched. The intervention group consists of the study participants that have been randomly assigned to receive the treatment.

Investigator

A person responsible for the conduct of the clinical trial at a trial site. If a trial is conducted by a team of individuals at a trial site, the investigator is the responsible leader of the team and may be called the principal investigator (PI).

Multicentre Trial

A clinical trial conducted according to a single protocol but at more than one site, and therefore, carried out by more than one investigator.

Number needed to treat (NNT)

The average number of patients who need to receive the treatment or other intervention for one of them to get the positive outcome in the time specified.

Outcome Measures

The impact that a test, treatment, or other intervention has on a person, group or population.

Phase I, II, III and IV Studies

Once the safety of a new drug has been demonstrated in tests on animals, it goes through a multi-phase testing process to determine its safety and efficacy in treating human patients. If a drug shows success in one phase, the evaluation moves to the next phase

  • Phase 1 tests a drug on a very small number of healthy volunteers to establish overall safety, identify side effects, and determine the dose levels that are safe and tolerable for humans.
  • Phase II trials test a drug on a small number of people who have the condition the drug is designed to treat. These trials are done to establish what dose range is most effective, and to observe any safety concerns that might arise.
  • Phase III trials test a drug on a large number of people who have the condition the drug is designed to treat. Successful completion of Phase III is the point where the drug is considered ready to be marketed.
  • Phase IV trials can investigate uses of the drug for other conditions, on a broader patient base or for longer term use.

Placebo

A fake (or dummy) treatment given to patients in the control group of a clinical trial.  Placebos are indistinguishable from the actual treatment and used so that the subjects in the control group are unable to tell who is receiving the active drug or treatment. Using placebos prevents bias in judging the effects of the medical intervention being tested.

Population

A group of people with a common link, such as the same medical condition or living in the same area or sharing the same characteristics. The population for a clinical trial is all the people the test or treatment is designed to help.

Protocol

A plan or set of steps that defines how something will be done. Before carrying out a research study, for example, the research protocol sets out what question is to be answered and how information will be collected and analysed.

Randomized Controlled Trial (RCT)

A study in which a number of similar people are randomly assigned to 2 (or more) groups to test a specific drug, treatment or other intervention. One group has the intervention being tested; the other (the comparison or control group) has an alternative intervention, a placebo, or no intervention at all. Participants are assigned to different groups without taking any similarities or differences between them into account. For example, it could involve using a computer-generated random sequence. RCTs are considered the most unbiased way of assessing the outcome of an intervention because each individual has the same chance of having the intervention.

Reliability

The ability to get the same or similar result each time a study is repeated with a different population or group.

Sample

People in a study recruited from part of the study’s target population. If they are recruited in an unbiased way, the results from the sample can be generalised to the target population as a whole.

Subjects

In clinical trials, the people selected to take part are called subjects. The term applies to both those participants receiving the treatment being investigated and to those receiving a placebo or alternate treatment.

Trial Site

The location where trial-related activities are conducted.


References

The Canadian Institutes of Health Research (CIHR)

TROG Cancer Research

ICH.org

NICE

Further Resources

American Society of Clinical Oncology’s Cancer.Net trials site

National Cancer Institute (NCI) Clinical Trials lists open and closed cancer clinical trials sponsored or supported by NCI. 

ClinicalTrials.gov database of privately and publicly funded clinical studies

CenterWatch Clinical Trials Listing

Does Cannabis Oil Have a Role in Cancer Treatment?

Does Cannabis Oil Have a Role in Cancer Treatment? from Patient Empowerment Network on Vimeo.

Is it just a trend or could cannabis oil truly have a role in cancer care and treatment? Dr. Sangmin Lee share his perspective.

Dr. Sangmin Lee is a hematologist-oncologist specializing in blood disorders and blood cancers at Weill Cornell Medicine and New York Presbyterian Hospital. More about Dr. Lee here.

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Transcript:

Patricia:

The use of cannabis oil is becoming prevalent. Does this have a role in cancer care and treatment?

Dr. Lee:

Absolutely. So, we use it for a lot of side effect management. So, cannabis can be helpful, in terms of appetite and nausea, for example. So, we often use it in conjunction to manage some of the side effects that patients can have throughout their treatment.

You should consult with your medical team, and of course, I should say that laws differ state by state, so it doesn’t apply to every state. But when it’s available, it can be a valuable addition.

Patricia:

Sure. Discuss that with your physician.

Are Clinical Trial Participants Monitored More Closely?

Are Clinical Trial Participants Monitored More Closely? from Patient Empowerment Network on Vimeo.

Dr. Sangmin Lee discusses the monitoring of clinical trial participants and the measures taken for patient safety.

Dr. Sangmin Lee is a hematologist-oncologist specializing in blood disorders and blood cancers at Weill Cornell Medicine and New York Presbyterian Hospital. More about Dr. Lee here.

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Transcript:

Patricia:

How about this next one? I am monitored more closely in a clinical trial.

Dr. Lee:

In some cases, it’s true. Clinical trials do have certain monitoring visits, in terms of doctor’s visits, laboratory tests, and physical exams.

The purpose of that is to make sure that it is safe. So, the purpose of monitoring closely, in a lot of cases, is for the patient’s safety. We are testing drugs in a lot of clinical trials, for which the complete safety profile, as well as efficacy profile, is not known. So, the purpose of closer monitoring is to make sure whatever we’re doing is safe, and if there are any unexpected side effects, then it allows us to address the side effects, as well. So, it’s mainly for patients’ safety.

Will Clinical Trials Cost You? The Facts.

Will Clinical Trials Cost You? The Facts. from Patient Empowerment Network on Vimeo.

Dr. Sangmin Lee reviews the financial impact associated with clinical trials, including a discussion of what expenses are covered for participants.

Dr. Sangmin Lee is a hematologist-oncologist specializing in blood disorders and blood cancers at Weill Cornell Medicine and New York Presbyterian Hospital. More about Dr. Lee here.

See More From The Fact or Fiction? AML Series


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Transcript:

Patricia:

All right, how about this one: I may have unexpected costs if I join a clinical trial.

Dr. Lee:

So, typically, that’s actually, usually not true, because how it works is that the clinical trial drugs, and that there may be extra procedures or visits associated with clinical trials.

And what usually happens is that the sponsor of the clinical trial provides the cost of the drug, intervention, and anything extra that are required for the clinical trial. So, in the end, the cost of participating in a clinical trial should not be any more than receiving standard care treatment.

In some rare cases, there may be stipends associated with the clinical trial, especially with travel. So, if you participate in a clinical trial, and you live far away, then you should ask to see if there is any stipends available, especially for travel.

The Truth About Clinical Trials in AML

The Truth About Clinical Trials in AML from Patient Empowerment Network on Vimeo.

Dr. Sangmin Lee reviews common misconceptions about clinical trials and shares examples of how these studies are changing the landscape of AML treatment. Want to learn more? Download the Program Resource Guide here.

Dr. Sangmin Lee is a hematologist-oncologist specializing in blood disorders and blood cancers at Weill Cornell Medicine and New York Presbyterian Hospital. More about Dr. Lee here.

See More From The Fact or Fiction? AML Series


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Why Speaking Up Matters: Tips from an AML Advocate

Transcript:

Patricia:                      

I’ll tell you a few things that we’ve heard from AML patients, and you tell me if this is fact or fiction. Okay, Dr. Lee?

Dr. Lee:

Yeah.

Patricia:

Clinical trials are the last resort treatment option.

Dr. Lee:

That’s actually not true in a lot of cases, because, yes, there are a lot of clinical trials after you have tried all of the standard therapy. But then, standard therapy in AML, like any other condition, are not perfect. So, there are many clinical trials where, even if you’re diagnosed with new AML, because standard care is not perfect, there are trials to try to improve upon the standard of care.

So, there may be clinical trials when you’re first diagnosed with AML, as well.

Patricia:

Yeah. How about this one: I feel like a guinea pig.

Dr. Lee:

Well, the clinical trial is to test drugs in humans. So, in a way, you are a test subject. But then, you have to remember that all of the drugs that we are testing have a rationale.

They all show promise, in terms of laboratory testing to kill leukemia cells in the test tube. And the problem is that, just because they are killed leukemia cells in the test tube, or in an animal model, doesn’t actually mean that it works in humans, or we know the safety profile. So, we need to do these testings to demonstrate that these drugs, which seem promising, actually work in humans.

Patricia:

Right. Well, then, that’s a good segue to this thing we’ve heard: Treatments being studied today may be the future standard of care.

Dr. Lee:

That is absolutely true, because all of the new developments that have come out, including Venetoclax, or IDH inhibitors, or other inhibitors, that are approved today, came through the clinical trial process. One example I like to include is a patient of mine, who, five years ago, had very, very aggressive leukemia, and she happened to have an IDH2 mutation.

It was four or five years ago. And she has a very refractory, aggressive leukemia, and it was life-threatening. And she had an IDH2 mutation. And we enrolled her in a clinical trial involving ivosidenib, which was in clinical trial at the time.

Ever since then, she became – she went into remission, and she has a normal blood count. And, to this day, she’s on this medicine, which is now approved, and she remains healthy with a normal blood count, in remission. So, yes. Clinical trials do include promising drugs, and if they show really good efficacy and promise, they will become standard of care down the road.

The advantage of clinical trial is that you may get early access to drugs that may become standard care down the road. So, it’s a way to get early access to potentially promising drugs.

Patricia:                      

How do you counsel your patients about joining clinical trials? What are you thinking about when you’re talking with them?

Dr. Lee:                       

So, in terms of clinical trials, we all look at clinical trials, and they exist for a reason, because we think that an intervention or drug can do better than standard of care. So, how I approach it is that, depending on the situation, if we can improve upon what is available, or if there are no other options, then it definitely is a great option to improve upon what would otherwise be standard.

Patricia:                      

I’ve got one more. Once I enroll, I am locked into the trial, and I can’t change course.

Dr. Lee: 

Absolutely not true. So, clinical trial participation is always voluntary. So, if you sign a clinical – So, what happens is you typically sign a consent to participate in a clinical trial.

And if you change your mind at any time, you can decide not to participate in a clinical trial. It’s not a binding agreement, so you can decide not to participate at any time.

Patricia:

Great. And that’s obviously a decision you should make with your healthcare provider before withdrawing.

Dr. Lee: 

Oh, absolutely. Absolutely, absolutely. But you should always remember that just because you sign up for a clinical trial, it’s not a binding requirement to stay on it.

Patricia:                      

Okay, okay. And let’s talk for just one moment, if you have a second again, about why patients – why it’s important for patients to participate in clinical trials.

Dr. Lee:                       

Why it’s important? It’s because the drugs we test could become the standard care in a few years. And you might have early access to a promising drug that may change treatment of AML. One prime example is Venetoclax. Venetoclax, when it was in clinical trial, was very promising, but before we started treatment, we had no idea how well it was going to work.

So, the patients receiving Venetoclax obviously benefitted from it, and they had early access to a drug that would have become standard of care a few years down the road.

Is It Safe? Breaking Down the Clinical Trial Process

Is It Safe? Breaking Down the Clinical Trial Process from Patient Empowerment Network on Vimeo.

The idea of a clinical trial can be intimidating and confusing for many patients. Dr. Sangmin Lee explains the phases of clinical trials, including the safety protocols in place to protect patients.

Dr. Sangmin Lee is a hematologist-oncologist specializing in blood disorders and blood cancers at Weill Cornell Medicine and New York Presbyterian Hospital. More about Dr. Lee here.

See More From The Fact or Fiction? AML Series


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Transcript:

Patricia:

What is the process for getting medicine to patients during clinical trials?

Dr. Lee:

So, clinical trials are basically what’s needed to prove that drugs work. So, a lot of times, we test drugs in a test tube in AML cell lines, and they show great promise. But just because a drug works in a test tube setting, doesn’t actually mean that it will work in humans, because human bodies are much more complicated. So, we need to test promising drugs in humans to make sure they are safe and effective.

And that’s what the purpose of clinical trials are. Once they demonstrate safety and efficacy, then a drug then gets to be approved, and is available commercially. So, that’s the purpose of clinical trials.

To be involved in clinical trials, what it involves is, basically, you have to meet a sort of criteria, called eligibility, because different clinical trials have different criteria for selection. So, we have to look into that. And then, once you fit an eligibility or selection criteria, then you typically undergo certain diagnostic tests to enroll on a clinical study. And then, you get whatever drug or intervention that is designed to test in that setting.

So, there are numerous steps to actually enroll in a clinical study.

Patricia:

And like you mentioned, there’s a long way between rat studies and human trials. What are the phases of clinical trials?

Dr. Lee:

So, there are three phases for clinical trials, commonly. There’s phase one, and phase two, and phase three. Phase one is the earliest part of the clinical trial process. So, goal of a phase one study is to make sure a drug is safe in a human. So, phase one studies are usually the first time that you are testing the drug in humans, and the main purpose is to demonstrate that it’s safe. So, typically, in a phase one study, typically, you test a drug at a lower dose or dose levels to demonstrate safety. What it means is that you’re enrolling a few patients at a time.

Once a drug is proven to be safe, then you move on to phase two, which is basically testing the drug in more patients. And the purpose of phase two is to get a preliminary assessment of how effective a treatment would be.

So, typically, a phase two study involves many more patients in that setting. And then, if a phase two study shows that a drug is very promising, then the drug may move on to phase three, where, basically, in phrase three, you are comparing one intervention or a drug compared to the standard of care. And, typically, in a phase three setting, a computer decides randomly which intervention you get, whether it’s an intervention or new drug versus standard of care. And standard of care may include either placebo or chemotherapy intervention, that is standard of care. So, it’s not always placebo in phase three.

AML Treatment Advances: What’s New for YOU?

AML Treatment Advances: What’s New for YOU? from Patient Empowerment Network on Vimeo.

 AML specialist and researcher, Dr. Sangmin Lee, breaks down the recent advances in AML treatment and how targeted therapies are improving patient care.

Dr. Sangmin Lee is a hematologist-oncologist specializing in blood disorders and blood cancers at Weill Cornell Medicine and New York Presbyterian Hospital. More about Dr. Lee here.

See More From The Fact or Fiction? AML Series


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Transcript:

Patricia:

Dr. Lee, thanks so much for joining us, again. Can you please give us an overview of the field of AML research?

Dr. Lee:

So, AML research is evolving very rapidly, and there’s a lot of promising new drugs that have come out in AML. As with any other cancers, we’re getting more sophisticated in characterizing AML in terms of molecular mutations, and characterizing AML stem cells, so –

The field is moving very rapidly in that regard. There have been a number of promising and effective drugs that have been approved in the last few years, as well. For example, Venetoclax has been a game changer in treatment of AML, especially in the elderly population. And there are several targeted agents that have been FDA approved in the recent years, as well. So, definitely since about three to five years ago, there have been new drugs that have come out for AML that is very exciting for treatment of AML.

Patricia:

Let’s talk a little bit about genetic testing. How is that changing the landscape for AML patients?

Dr. Lee:

So, genetic testing has become standard in AML patients, in terms of – at their diagnosis and relapse. And part of that is, we can use that information to guide prognosis, how well or not well a patient is expected to do.

But more importantly, there are actually drugs that can target specific mutations. For example, there are new drugs that target a mutation called IDH1 and 2 that have been approved recently for patients with AML, as well as new drugs that target mutation called FLT-3, or FLT3 mutation, as well. So, genetic testing has become standard, not only to tell you prognostic information, but also used in therapy for AML patients.

Patricia:

You mentioned a few treatments that were advancing. What other therapies are showing promise for AML?

Dr. Lee:

So, there are a number of treatments that are ongoing. Venetoclax has been game-changing, and now, although Venetoclax has improved outlook, in terms of AML treatment, compared to conventional therapy, there’s still resistance to Venetoclax and the response is not durable.

So, there is research looking at resistance mechanisms to Venetoclax, for example. The other exciting field is, there are some advances in immunotherapy, with clinical trials underway. Like in other malignancies, there are clinical trials involving CAR T-cell, or other ways of engaging your own T cell immune system to approach and attack the AML.

AML Research: What’s New in Treatment?

AML Research: What’s New in Treatment? from Patient Empowerment Network on Vimeo.

 AML expert, Dr. Jessica Altman, discusses the future of AML research, and new learnings that continue to improve current treatment approaches.

Dr. Jessica Altman is Director of the Acute Leukemia Program at Robert H. Lurie Comprehensive Cancer Center of Northwestern University. More about Dr. Altman here.

See More From The Fact or Fiction? AML Series


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AML Treatment Treatment Side Effects: What’s Fact and What’s Fiction?


Transcript:

Patricia:            

Are there any new treatments on the horizon that you can talk about, Dr. Altman?

Dr. Altman: 

Absolutely. So, I love to talk about new therapies in AML. Until the last couple of years – it had been 40 years since we approved a sustained treatment in the marketplace in AML. We had been treating the disease the same. And over the last couple of years there have been a growth of therapies. We’re now trying to sort out exactly when we’re using one over another. We also have clinical trials where we’re combining novel therapies for adults with either newly diagnosed disease or relapsed and refractory disease. 

We are in an era of looking out at antibody therapy in AML – that’s one of the new waves of treatment. We are still exploring targeting therapies in the sense of inhibition of FLT3, IDH, and other mutations. So, it’s an era where there’s lots of excitement, and I’m hopeful for our patients.

Patricia:     

Yeah. Tell me what makes you most hopeful about the future of research in this area, and treatment?

Dr. Altman: 

So, I think that’s a great question. I think the fact that we now – the deeper the understanding we have of the biology of the AML, why AML happens, what mutations drive the disease, and then how to target those mutations with individual therapies is what excites me the most. So, our basic science research has exploded, and that occurs at a very quick pace, and that’s allowing us to develop therapies at a much faster rate than I would have anticipated before.

Patricia:

What a wonderful way to end our chat. Thank you so much, Dr. Altman, for taking the time to join us today.

Dr. Altman: 

It’s a pleasure to be here. Thank you so much.

Misconceptions in Clinical Trials: What’s Fact and What’s Fiction?

Misconceptions in Clinical Trials: What’s Fact and What’s Fiction? from Patient Empowerment Network on Vimeo.

AML expert, Dr. Jessica Altman, addresses common misconceptions patients have about clinical trials regarding treatments, regulations, and standards of care. Want to learn more? Download the Program Resource Guide here.

Dr. Jessica Altman is Director of the Acute Leukemia Program at Robert H. Lurie Comprehensive Cancer Center of Northwestern University. More about Dr. Altman here.

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Transcript:

Patricia:            

What about clinical trials? What common misconceptions do patients have about enrolling in trials?

Dr. Altman: 

So, I think the misconceptions regarding clinical trials can be very masked. And I think it really depends on the intent of a clinical trial and the phase of the clinical trial. I think that a well-designed clinical trial is almost always the right choice for a patient with acute leukemia at any stage in their therapy. 

That is a bias as a clinical trialist. I think it’s the right bias, but it is still my bias. I think patients frequently worry that they’re being treated as a guinea pig, or they’re not getting an appropriate treatment. What I can tell you is the clinical trials that we and my colleagues across the country and across the world participate in are clinical trials where the patients are getting at least what we consider a standard of care for that phase of their disease, and they may be getting something in addition to that or something that is slightly different, but expected to have a similar response rate. 

We have this phrase in clinical trials, something called equipoise, that if there’s a randomization between options that we need to feel, as the practitioner and as the clinical trialist, that each option is at least as good as the other.  

Patricia:

That kind of goes back to the vetting of treatments before they go to a clinical trial. Tell me a little bit about history. How can we make patients feel more comfortable?

Dr. Altman: 

I want to make sure that I understand the question.

Patricia:

So, how thoroughly are treatments vetted before they go to a clinical trial?

Dr. Altman: 

Great. So, the way that agents get into early phase clinical trials and then later phase studies are these are compounds that have been studied in the laboratory, then studied in small animals, then larger animals. And then, frequently, a drug is started in a patient with relapsed and refractory Acute Myeloid Leukemia and found to be safe – that’s what we call a Phase I study. 

Once we know the right dose and the associated side effects from an early phase clinical trial, later phase studies – i.e. Phase II, where the goal is to determine the efficacy and response rate is conducted. And then, if that appears and looks like it’s promising, a larger, randomized, three-phase study is frequently conducted, where we compare a standard of care to the new approach. 

Patricia:

So, patients should be comfortable that the clinical trial that they’re going through has been thoroughly vetted, has gone through multiple stages before human trials occur?

Dr. Altman: 

That is accurate in terms of compounds get through animal studies, and then depending on the way that the trial is being connected, will then be studied in patients either with relapsed or refractory disease or very high-risk disease. But it’s also very important to mention that these pharmaceutical companies and physicians are not making these decisions alone. 

The clinical trials are all reviewed by scientific review committees through the cancer centers, which are other investigators making sure that everything appears appropriate. In addition, there are institutional review boards at every university whose goal it is to keep patients and research subjects in well-done clinical trials safe. That is their primary goal. And the IRBs – institutional review boards – are very involved with making sure that clinical trials are appropriate and that the conduct of clinical trials is appropriate.

Addressing Common Myths About AML Treatment

Addressing Common Myths About AML Treatment from Patient Empowerment Network on Vimeo.

AML expert, Dr. Jessica Altman, discusses common myths surrounding available AML treatment options, stem cell transplant and how leukemias are classified.

Dr. Jessica Altman is Director of the Acute Leukemia Program at Robert H. Lurie Comprehensive Cancer Center of Northwestern University. More about Dr. Altman here.

See More From The Fact or Fiction? AML Series


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Transcript:

Patricia:            

Dr. Altman, let’s talk about some AML treatment myths floating around. I’ll throw some stuff out there, you let me know if you’ve heard this. “Leukemia is one disease.”

Dr. Altman: 

So, I have heard that. Leukemia is actually a number of different diseases, and it’s very heterogenous. There are acute and chronic leukemias. The acute versus chronic really depends on a couple of factors. The biologic factor is the presence or absence of 20% loss or more in the bone marrow, but that also coincides with how patients present clinically. Acute leukemias tend to present more acutely, more rapidly. And chronic leukemias tend to be a bit more indirect. And the treatments are very different for those entities. 

There are also myeloid or lymphoid leukemias, so there’s Chronic Myeloid Leukemia and Acute Myeloid Leukemia and Chronic Lymphocytic Leukemia and Acute Lymphoblastic Leukemia. So, those are the four major categories. We’re talking about Acute Myeloid Leukemia today. Within Acute Myeloid Leukemia, there are multiple different types of Acute Myeloid Leukemia that are really now best categorized by history – patient history – and the molecular and cytogenetic abnormalities of the disease. 

Patricia:

Now, we’ve already learned about a bunch of them. So, “There are limited treatment options” is definitely a myth. Correct, Dr. Altman?

Dr. Altman: 

So, we have had a major growth of the number of treatment options available for Acute Myeloid Leukemia really in the last couple of years. It’s been a very exciting time for practitioners and for our patients that we have now a number of new therapies. So, there is not just one treatment available. In fact, the conversation regarding treatment options becomes quite extensive with patients and their families, because there are choices. And that’s why consideration of goals in the intent of treatment becomes even more important. 

Patricia:

Here’s another one: “Stem cell transplant – the only chance for cure.”

  Stem Cell Transplant, also called a bone marrow transplant, is a procedure in which healthy blood stem cells are used to replace damaged or diseased bone marrow. This procedure can be used to treat certain types of blood cancers.

Dr. Altman: 

Okay. So, that is also a myth. There are certain types of Acute Myeloid Leukemia where stem cell transplant is the most appropriate treatment once the disease is in remission if the goal of the patient is of curative intent. Stem cell transplant is not appropriate for every individual, and for some types of Acute Myeloid Leukemia, stem cell transplant is not considered. 

Patricia:

What kinds of things do you think about when you’re considering a stem cell transplant with a patient? 

Dr. Altman: 

So, again, I go back to patient goals and understanding their goals of treatment. A stem cell transplant is among the most medically intensive procedures that we have. It is also not just a treatment that occurs over a short time. While the actual transplant is a relatively limited hospitalization and the administration and infusion of stem cells and preparative chemotherapy, it is something that can continue to have side effects and alterations in life quality that can persist for months to years afterwards. 

So, that’s one aspect of things that we talk about regarding stem cell transplant. And really understanding what the benefit of transplant is in terms of a survival advantage, versus what the risk and the cost in terms of toxicities are. And that’s the basis of a lot of the conversations we have.

Patricia:

Sure. Here’s one more: “AML patients require immediate treatment.”

Dr. Altman: 

Sometimes AML patients require immediate treatment, and sometimes they don’t. And that depends on the biology of the disease. How high is the white blood count when the patient comes in? What are the best of the blood counts? Is the patient having immediate life-threatening complications of their acute leukemia? 

And there’s some forms of acute leukemia that require immediate therapy to prevent complications, and there’s some forms of acute leukemia who present an extreme distress from their disease, but there are many patients who present with acute leukemia, and we have time to get all of the ancillary studies back – the studies of genetics and the molecular studies1 – to help further refine the conversation, and further design an appropriate treatment strategy. 

Patricia:

What else? What do you hear from your patients that you feel is maybe a misconception or something they’re not quite understanding about the AML?

Dr. Altman: 

So, I think one of the biggest things that I would like to mention is that response rate and cure are not the same. So, it is possible for one to be treated for Acute Myeloid Leukemia and the disease to enter remission, and yet still not be cured of their disease. 

Acute Myeloid Leukemia is a disease that frequently requires additional cycles of treatment or a stem cell transplant after the initial induction therapy to be able to have the best chance for a long-term cure. So, response and cure are not the same thing.

Acute Myeloid Leukemia: Your AML, Your Treatment, Your Decision

Acute Myeloid Leukemia: Your AML, Your Treatment, Your Decision from Patient Empowerment Network on Vimeo.

AML experts Dr. Pinkal Desai, Assistant Professor of Medicine at Weill Cornell Medical College and Assistant Attending Physician at the New York-Presbyterian Hospital, and Dr. Tapan M. Kadia Associate Professor, Department of Leukemia at The University of Texas MD Anderson Cancer Center, share research-based insight on how AML is diagnosed, including the symptoms and recommended tests, and disease management strategies.
 
These experts give an overview of currently approved AML therapies and share clinical trial updates on treatments in development. The panel discusses AML management and how you can ask questions and talk to your doctor to feel confident with your care. Additionally, you will hear from an AML patient who shares their experience and advice for approaching the decision-making process.
 

Understanding and Managing AML Treatment Side Effects

Understanding and Managing AML Treatment Side Effects from Patient Empowerment Network on Vimeo.

AML expert, Dr. Jessica Altman, discusses how AML affects the body, and the common side effects patients may experience during varying AML treatment phases.  

Dr. Jessica Altman is Director of the Acute Leukemia Program at Robert H. Lurie Comprehensive Cancer Center of Northwestern University. More about Dr. Altman here.

See More From The Fact or Fiction? AML Series


Related Resources

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AML Treatment and Side Effects Program Resource Guide


Transcript:

Patricia:            

Dr. Altman, let’s talk about some common AML treatment side effects. What are some of the things that patients can expect when they begin treatment?

Dr. Altman: 

So, the side effects depend in part on the actual treatment strategy that’s utilized. It’s also important to note that AML itself has symptoms, and so sometimes it’s hard to separate out the symptoms of the Acute Myeloid Leukemia and the symptoms from the treatment. Acute Myeloid Leukemia is a disease where the bone marrow is not functioning normally. The bone marrow is responsible for making healthy red blood cells, healthy white blood cells, healthy platelets, and also is very intimately involved with the immune system. 

And so, patients with Acute Myeloid Leukemia by itself without treatment are at risk for fatigue if the hemoglobin is low, bleeding and bruising when the platelet count is low, and at risk for infections. 

Also, shortness of breath and other side effects from having abnormal blood counts. In addition, the treatment frequently lowers the blood counts further, and the treatment itself increases those risks associated with low blood counts. Patients can be supported with blood transfusions. Patients are also supported with antimicrobial therapy to prevent infections, and if fever or infections occur despite that, patients receive additional antimicrobial therapy based on what the perceived organism is. 

Patients with Acute Myeloid Leukemia, when they receive chemotherapy, are also sometimes at risk for something called tumor lysis syndrome. 

That’s when we kill the leukemia cells, when the leukemia cells are killed quickly, sometimes the contents of the leukemia cells can inflame the kidneys and lead to alterations in the electrolytes and the acids and salts in the body, and that’s something that needs to be monitored for and prevented. 

Patients with Acute Myeloid Leukemia who receive chemotherapy are also at risk for organ inflammation, and that is something that is monitored with the blood counts.

Patricia:     

What can patients or their caregivers suggest to help manage some of these side effects?

Dr. Altman:    

So, I think the biggest side effect that might be the hardest for us to manage and for patients to manage is fatigue. And I’m a believer that energy begets energy, and so trying to be as active as one can throughout all phases of their treatment I think helps the most. And also, the hopeful recognition that the fatigue should be self-limited, and that with time away from treatment, the energy should improve.

I think that’s one of the biggest things I hear from my patients.