What MDS Patients Should Know About Clinical Trials | Aplastic Anemia and MDS International Foundation

What MDS Patients Should Know About Clinical Trials

Dr. Sekeres is Professor of Medicine, Director of the Leukemia Program, and Vice Chair for Clinical Research at the Cleveland Clinic Taussig Cancer Institute.  He earned his medical degree and a master’s degree in clinical epidemiology from the University of Pennsylvania School of Medicine. Dr. Sekeres completed his postgraduate training at Harvard University, finishing an internal medicine residency at Massachusetts General Hospital and a fellowship in hematology-oncology at the Dana-Farber Cancer Institute in Boston.

Do you find that some patients don’t understand what clinical trials are?

There’s a wide range of patient knowledge and opinions about clinical trials. I have some patients who ask me about them because they want to be part of the latest research and latest opportunities to try new drugs or drug combinations for MDS. I also have patients who want nothing to do with them, saying in effect, “I don’t want to be a guinea pig,” – not wanting to be part of any kind of experimental study. 

Do some patients feel when clinical trials are recommended that this is a ‘last resort’ treatment option?

There are a wide variety of clinical trials, and it also depends on what the patient’s philosophy is on engaging in them.  Some reserve clinical trials for when all other options are exhausted –when there’s nothing else available for them. This isn’t really a last resort, though – I consider this another option when available therapies either aren’t appropriate or haven’t worked, after which there’s nothing but blood and platelet transfusions to turn to.

At present we have only a limited number of FDA approved drugs available for people with MDS. These are lenalidomide (Revlimid®), azacitidine (Vidaza®), and decitabine (Dacogen®). We use a few other drugs off-label, such as erythropoiesis stimulating agents (ESAs) like erythropoietin or darbepoeitin, or immunosuppressants like anti-thymoctye globulin, (ATG).  My approach is that we’ll always have those three approved drugs to fall back on. But if we have a trial that is available and right for the patient, let’s try that first and if there’s no success we can always go back to those available therapies.

Can you describe some current areas of MDS clinical research?

Clinical research runs the spectrum of a person’s individual experience with MDS. I try to think about research from a patient’s perspective –how we can improve this person’s experience from the very moment he or she is diagnosed. In a way, clinical trials are designed to answer these intrinsic questions. How can we improve a person’s quality of life? For this, we engage in research about quality of life issues. How can we help patients minimize the number of blood or platelet transfusions they receive? Here, we conduct research that looks at supportive care issues. How can we improve treatments for a patient’s lower-risk or higher -risk MDS?  Should we look at developing drugs specifically for those conditions? How can we develop drugs for those who have been exposed to other therapies that didn’t’ work for them? We call this refractory MDS, where there was no improvement after 4 or 6 months, or recurrent/relapsed MDS, where there is initial improvement, but then MDS returns to its original state. Ideas for clinical trials are built around areas of inquiry like these.

Who are the primary sponsors of clinical trials?

Trials can be sponsored by a number of different sources. A trial can be born in the institution where the patient is receiving care. The primary investigator may be the patient’s doctor, or perhaps this doctor wrote the trial. It could be for a drug that was developed in the cancer center that is finally reaching MDS patients. The National Institutes of Health (NIH) can also be a sponsor. Here at Cleveland Clinic, we just  last year completed a randomized study called the North American Intergroup study where trial participants -- people with higher risk MDS -- received either azacitidine alone, azacitidine and lenalidomide, or azacitidine combined with vorinostat.  I wrote this study under the auspices of the Southwest Oncology Group, which is one of the National Cancer Institute’s (NCI) cooperative groups.  We participated in this trial along with the Eastern Cooperative Oncology Group, the Alliance Oncology Group (which are all sponsored by NCI), and the National Cancer Institute of Canada. So, in this case, four government-sponsored cooperative groups participated in one study funded by the NIH.

Another common source of sponsorship are the drug companies themselves. Many drugs are discovered or developed by these companies, and they will conduct trials to see if the drug is safe and effective enough to be approved by the FDA – the majority of drugs have this point of origin.

What can an MDS patient in a clinical trial expect to learn?

Every clinical trial that is conducted in the US is registered in the clinicaltrials.gov domain, so the results will be reported there. It may take years before a clinical trial is finished, but the final results are always reported and those results are intended to be publically available. So patients have a great resource to learn about many clinical trials – their purpose and their actual progress.

Why is it important for MDS patients to consider participating in a clinical trial?

There are different reasons. My patients tell me one reason they will participate is the strict, rigorous schedule that is adhered to. Everyone has access to the standard level of care, but some are receiving care far beyond current standards. They may have access to a drug that works for them years before it’s approved by the FDA and widely available. Some participate for completely altruistic reasons – they really do want to help the next generation of MDS patients. But all patients know that there’s a chance they can’t be included in the trial and that even if they are, they may not benefit from the drug regimen being tested.

It’s an exciting time for MDS research, but we need MDS patients for the many clinical trials that are being planned or are in process. That’s the only way new treatments can result from the progress being made in basic research.
Interviewee: 

Mikkael Sekeres, MD, MS

Lead Photo
Position / Title: 
Director, Leukemia Program, Department of Hematologic Oncology and Blood Disorders
Institution: 
Cleveland Clinic Taussig Cancer Institute

Mikkael A. Sekeres, MD, MS has been Medical Advisory Board Co-Chair since 2004. As a professor of medicine and director of the leukemia program, Dr. Sekeres is also Vice Chair for Clinical Research at the Cleveland Clinic Taussig Cancer Institute in Ohio. He earned his medical degree and a master’s degree in clinical epidemiology from the University of Pennsylvania School of Medicine. Dr. Sekeres completed his postgraduate training at Harvard University, finishing an internal medicine residency at Massachusetts General Hospital and a fellowship in hematology-oncology at the Dana-Farber Cancer Institute in Boston. He chaired the Oncologic Drugs Advisory Committee of the FDA.

An invited speaker at numerous meetings, grand rounds, and conferences, Dr. Sekeres is a member of the American Society of Hematology, the American Society of Clinical Oncology, and the Southwest Oncology Group—Leukemia Committee. His current research focuses on patients with MDS and older adults with acute myeloid leukemia, and he has been the national primary study investigator on several phase I/II trials. He is the author or co-author of over 230 articles and over 250 abstracts published in leading journals such as Blood, Journal of Clinical Oncology, Nature Genetics, Journal of the National Cancer Institute, PLoS One, Cancer, Haematologica, and Leukemia. He is also the co-author of 6 books; the editor-in-chief of the ASH Clinical News magazine; he is on the editorial board of several journals; and is an essayist for The New York Times and Huffington Post.