In this MD Newsline exclusive interview with hematologist/oncologist Dr. Cheryl Mensah, we discuss sickle cell disease treatments and the latest research on these treatments.
Which treatments are used to manage sickle cell disease?
Dr. Cheryl Mensah:
“The treatments used for sickle cell disease are mainly used to prevent sickle cell disease crises and complications. There’s hydroxyurea, which is the oldest medication and was FDA-approved to prevent sickle cell crises. We’ve found over time, it also helps to prevent stroke, acute stress syndrome, and many complications of sickle cell disease.
We also have some newer medications for sickle cell disease, like L-glutamine (a protein powder) and crizanlizumab (a monoclonal antibody). These medications have been shown to reduce vaso-occlusive crises.
Also, there’s voxelotor, a medication that increases hemoglobin. It’s a great medication for patients that require a lot of transfusions or frequently have low hemoglobin.”
Is there any research that excites you or that you think is important for physicians to know related to the treatment of sickle cell disease?
Dr. Cheryl Mensah:
“There are two areas of sickle cell disease research that I’m very excited about. The first area is gene therapy, which is similar to transplant and is the only FDA-approved way to cure patients of sickle cell disease. However, in comparison to transplant, which requires a patient to have a donor (often a sibling or family member), with gene therapy, the patient is the recipient and the donor. So, it’s an exciting area of research that provides curative treatment and involves less chemotherapy than a traditional transplant.
The second area of sickle cell disease research that I’m excited about is pyruvate kinase activators. These are oral medications that affect one of the enzyme pathways involved in hemolysis. Pyruvate kinase activators are still being studied in clinical trials, and I’m excited about them because they may be able to raise hemoglobin levels and reduce sickle cell crises. These medications could help patients rely less on intravenous medications and other interventions, and could reduce sickle cell disease-related hospitalizations.”
Responses have been condensed and lightly edited.