Expert Mikkael Sekeres, MD, explains that the COVID-19 pandemic affected all of medicine, but in a rare disease domain that impact was even greater.
Mikkael Sekeres, MD, is not a stranger to rare disease. Although, he may not have started med school with a dream to treat patients living with myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), or paroxysmal nocturnal hemoglobinuria (PNH), he's Chief of Division of Hematology in the Sylvester Cancer Center at Miami University, and Chair of the Medical Advisory Board for the Aplastic Anemia and MDS International Foundation (AA*MDS).
In his own words, he specializes in treating people who have bone marrow disorders. More specifically, he focuses on different types of leukemia and myelodysplastic syndromes.
As a healthcare professional that treats patients with some of the rarer of the rare diseases, he gives perspective as to how prevalence rates among rare diseases can vary–even within the domain of accounting for less than 200,000 cases per year in the US.
In this interview, he shares insight to what it's been like trying to treat patients and conduct a clinical trial during the COVID-19 pandemic.
"So let's go back in time, a little bit to the beginning of 2020," he said, "when this pandemic was just hitting, and we were trying to figure out what we needed to do on behalf of our patients in real time."
Sekeres compared our knowledge, or lack thereof, at the beginning of the COVID-19 pandemic, to what's now understood about the virus.
"People sometimes forget that when you come and see me with a diagnosis of myelodysplastic syndrome, and I give advice on how to treat you, I'm basing it on very well designed studies that took years to accomplish," he said.
The pandemic had an impact on many aspects of medicine. Patient care and clinical trials had to adapt to a virally infected landscape. Many clinical trials across the board shut down entirely.
"When we were pivoting to deal with the COVID-19 pandemic we didn't have a years of well designed studies to rely on," he continued. "we were trying to manage this in real time, before the studies had matured."
Before speaking about his current clinical trial, he stated a reminder that now, there are guidelines in place for the COVID-19 pandemic because of the trials that were immature a year or so ago.
"So, I have dedicated a good portion of my career to exploring combinations of drugs to treat people who have mild dysplastic syndromes," Sekeres said.
This particular trial is aimed at examining how a combination of 2 FDA-approved drugs for MDS affect patients with lower-risk MDS. 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 explained. "So, we're trying to see if we can offer something that has a better chance of working."
The trial is open at University of Miami, and will be opening at 5 other centers around the country. All of these centers are NCI, National Cancer Institute, comprehensive cancer centers–major players in oncology, Sekeres said.
It's important to spread awareness of rare disease like myeloid dysplastic syndromes, whether it's information or a connection to an ongoing clinical trial.
At the end of the interview, Sekeres wanted to share a parting message specifically for patients with a new diagnosis of a rare disease and the significance of educating themselves because "it is true, knowledge is power".
"So, I urge them to turn to places like Aplastic Anemia and MDS Foundation which is a trusted source, as opposed to broad searches on the internet, which I don't oppose," he clarified. "I love when patients come and ask me questions from the internet so we can address misconceptions."
"But the internet is an unrefereed environment," Sekeres continued. "And you want to go to a place where the the knowledge that you're trying to gain has actually been closely vetted and curated."
Stay tuned for more from Mikkael Sekeres of AA*MDS in the Rare Disease Report podcast.