#72 - Mast Cell Disease, the Hematologist & New Treatments on the Horizon

Why talk to a hematologist about mast cell disease and systemic mastocytosis treatment?

Dr. Douglas Tremblay, a leading hematologist, joins Dr. G and Kortney to discuss the role of a hematologist-oncologist in diagnosing and treating mastocytosis. We discuss bone marrow's vital role in diagnosing and treating systemic mastocytosis. 

Dr. Tremblay discusses the latest KIT inhibitors, a promising group of medications, and explains the difference between indolent and advanced systemic mastocytosis. He clarifies how KIT inhibitors work and emphasizes the importance of managing side effects. Finally, Dr. Tremblay provides insights into the future of KIT inhibitor approval and explores the possibility of participating in a clinical trial for Bezulastinib. 

What we cover in this episode about treating systemic mastocytosis:

• What is a hematologist looking at with mast cell disease patients?
• Mast cell diseases and the role of bone marrow
  • Bone marrow biopsy for systemic mastocytosis 
  • What is a bone marrow biopsy?
  • Information bone marrow biopsy provides for diagnosis
• KIT mutation 
• KIT mutation testing in bone marrow and blood (digital droplet PCR)
• Indolent Mastocytosis versus Advanced Mastocytosis
• Current research for treating mast cell disease
• How KIT inhibitors work
• Do KIT inhibitors weaken the immune system?
• KIT Inhibitors discussed in this episode:
  • Avapritinib
  • Bezulastinib
  • Elenestinib
• Are KIT inhibitors a cure for mast cell disease?
• Determining the tolerance for side effects of medications when researching and adding new medicines for diseases 
• The meaning of ‘NIB’ in the medication names
• The timeline for the new KIT inhibitors approval
• The type of patient that can participate in the clinical trials for Bezulastinib

Resources about mast cell disease:

• Dr. Tremblay: https://profiles.mountsinai.org/douglas-a-tremblay

  • Dr. Tremblay’s email: douglas.tremblay@mssm.edu 

• Mast Cell Disease Overview: https://allergyasthmanetwork.org/health-a-z/mast-cell-diseases/

• What is Anaphylaxis? https://allergyasthmanetwork.org/anaphylaxis/

• What is Epinephrine? https://allergyasthmanetwork.org/anaphylaxis/what-is-epinephrine/

• The Mast Cell Disease Society: https://tmsforacure.org/

• Current studies enrolling patients: https://tmsforacure.org/clinical-trials/