Mikkael Sekeres, MD, is the chief of the division of hematology at the Sylvester Cancer Center at the University of Miami. He is also Chairman of the Medical Advisory Board of the Aplastic Anemia and MDS International Foundation (AA*MDS), as well as the American Society of Hematology (ASH).
Rare diseases are an integral part of Sekeres’ life, as he spends his days treating patients with bone marrow disorders such as myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML), among others.
A condition that affects fewer than 200,000 people in the United States is classified as a rare disease. MDS and AML have an incidence rate of approximately 5 per 100,000, each affecting approximately 20,000 people.
Sekeres explained that there are rare diseases and then there are very rare diseases in hematology.
In this interview for the Rare Disease Report podcast, Sekeres shared her expertise on drug regimens, overcoming obstacles with clinical trials and trusted rare disease resources for doctors and patients.
Awareness of clinical trials is essential
Sekeres led a clinical trial that is open at the University of Miami and will open in 5 other centers across the country. All of these centers are National Cancer Institute (NCI) comprehensive cancer centers, “major players in oncology,” he said.
“So I’ve spent a good part of my career exploring drug combinations to treat people with myelodysplastic syndromes,” Sekeres explained.
The trial is evaluating a combination of 2 FDA-approved drugs in patients with low-risk MDS. The drugs, Luspatercept (REBLOZYL) and Lenalidomide (Revlimid), were not reviewed together, according to Sekeres.
“This is the population for which we only have a limited number of drugs available and we are starting to run out of tools in our toolbox,” he said. “So we’re trying to see if we can offer something that has a better chance of working.”
“Knowledge is power”
Awareness and access to information are key when it comes to rarer diseases, for both providers and patients. Sekeres stressed the importance of self-education because “it’s true, knowledge is power”.
He loves when patients ask him about things they’ve found on the internet, however, misinformation can be detrimental. It is important to make sure the source of information is reliable, such as the Aplastic Anemia and MDS Foundation.
“The internet is an unrestricted environment,” Sekeres said. “And you want to go somewhere where the knowledge you’re trying to acquire has been carefully vetted and curated.”
Sekeres said one of the reasons he was drawn to AA*MDS 20 years ago was because the foundation had all the resources a patient needed – educational materials and toolkits, access to conferences , webinars and even support groups.
“I have long believed that as researchers and academics, our research must begin with our interactions with our patients and the information our patients obtain.”