The Rare Disease Report Podcast: Myelodysplastic Syndromes & Acute Myeloid Leukemia

Mikkael Sekeres, MD, is the chief of the Division of Hematology, Sylvester Cancer Center at Miami University. He also serves as chair of the Medical Advisory Board for the Aplastic Anemia and MDS International Foundation (AA*MDS), as well as the American Society of Hematology (ASH).

Rare diseases are a common part of life for Sekeres, as he spends his days treating patients with bone marrow disorders like myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML), among others.

A condition that affects less than 200,000 people in the US is classified as a rare disease. MDS and AML have an incidence rate of approximately 5 per 100,000, each affecting roughly 20,000 individuals.

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 his expertise on treatment regimens, overcoming barriers with clinical trials, and reliable rare disease resources for physicians and patients.

Clinical Trial Awareness is Critical

Sekeres has been leading a clinical trial that's open at University of Miami, and will be opening at 5 other centers around the country. All of these centers are National Cancer Institute (NCI) comprehensive cancer centers, "major players in oncology," he said.

"So, I have dedicated a good portion of my career to exploring combinations of drugs to treat people who have myelodysplastic syndromes," Sekeres explained.

The trial's evaluating a combination of 2 FDA-approved drugs in patients with lower-risk MDS. The drugs, Luspatercept (REBLOZYL) and Lenalidomide (Revlimid), haven't been examined in conjunction, according to Sekeres.

"This is the population where we only have a limited number of drugs available and we're starting to run out of tools in our toolkit," 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 is critical when it comes to rarer conditions for both providers and patients. Sekeres emphasized the significance of self-education because "it is true, knowledge is power".

He loves when patients ask him questions about things they found on the internet, however, misinformation can be detrimental. It's important to make sure the source of information is a reliable one like the Aplastic Anemia and MDS Foundation.

"The internet is an unrefereed environment," Sekeres said. "And you want to go to a place where the knowledge that you're trying to gain has actually been closely vetted and curated."

Sekeres said that one of the reasons he was drawn to work with 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've long believed that as people who are researchers and academics, our research needs to start with our interactions with our patients and the information our patients are getting."