Making Progress for Myelodysplastic Syndrome

In an interview with CURE, Rami Komrokji, M.D., a hematologist at Moffitt Cancer Center in Tampa, Florida, discusses the advances that have been made in MDS in the past few years, as well as the MDS Clinical Research Consortium and its importance.
BY ALLIE CASEY
PUBLISHED: JULY 25, 2017
Myelodysplastic syndromes (MDS) are rare, which can make them difficult to study. The biggest challenge is finding enough patients to enroll in clinical trials that could help to advance treatment. But the Aplastic Anemia and Myelodysplastic Syndrome International Foundation (AAMDSIF), a nonprofit organization, wants to change this by providing patients and patient data to support research. One of its efforts in this area is the MDS Clinical Research Consortium.

In an interview with CURE, Rami Komrokji, M.D., a hematologist at Moffitt Cancer Center in Tampa, Florida, discusses the advances that have been made in MDS in the past few years, as well as the MDS Clinical Research Consortium and its importance.

What progress has been made in the field of MDS in the last few years?

There have been several discoveries that led to better understanding of the disease and its biology. Hopefully this can translate into improving patient care and finding new treatments.

The use of molecular testing can help us in deciding, or tailoring, the treatment. There are also some new treatments on the horizon based on some of the molecular targets of testing that are being conducted. 

Advances have also been made in the biology of the disease and our understanding of MDS. We've made huge progress in understanding what occurs in MDS, what are the gene mutations that are acquired in this disease, the role of the bone marrow microenvironment and inflammation in developing MDS.

Also, risk stratification for patients with MDS is a crucial step for patient management. How do we risk stratify the disease? Once we establish a diagnosis for patients, we try to gauge the risk of the disease  because we tailor therapy based on that, such as recommending allogeneic stem cell transplant for patients. An accurate risk assessment for the disease or certification is important for the patient and their families. It gives them prognostic information. It's also important for the physicians because we decide on our therapies based on the disease risk.

What new treatments are in this area?

In MDS, there is an unmet need for patient treatments. We've made some progress with current medications like Vidaza (azacitidine), Dacogen (decitabine), Revlimid (lenalidomide). We consider transplant as a curative option for patients with high-risk disease. Those are the options we currently use. Other than transplant, most of the treatment options are not curative. They work and patients can derive benefit in terms of survival and quality of life, but at some point they stop working. We always are in a need for next treatments.

Over the last few years, we started to understand more about the biology of the disease and found certain genetic abnormalities that hopefully we can target. There are several trials with promising medications looking at this concept.

For example, there is a study that just finished, called the MEDALIST study, which tests a drug called luspatercept, which is injected every three weeks for patients with lower-risk MDS, but need transfusions on a regular basis. In the early phases, phase 1 and 2, this drug showed promising results and is now finishing the phase 3 study. If that study is positive, then this drug could be approved and available for patients. Luspatercept works in certain subsets of patients with MDS, particularly those who have ring sideroblasts or a certain gene splicing mutations, SF3B1 mutation.



Talk about this article with other patients, caregivers, and advocates in the Myelodysplastic syndrome CURE discussion group.
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