Chronic Myeloid Leukemia (CML)

Investigators: Fernanda Festa, Ph.D.

Collaborators: Nathaniel Gray, Ph.D. (HMS, DFCI)

Disease Background

Who gets it?
What are the symptoms?
How do we detect it now?
How is it currently treated?
What are the current challenges?
  • 1–2 per 100,000 people worldwide get chronic myeloid leukemia (CML) per year
  • It can affect all age groups, but is more common in the middle-aged and elderly.
  • CML is a type of cancer of the blood cell. Patients are often asymptomatic.
  • Without treatment the mortality rate is extremely high.
  • CML is caused by the fusion of two chromosomes, creating a abnormal chromosome, known as Philadelphia chromosome.
  • CML is diagnosed typically by complete blood count, followed by bone marrow biopsy and identification of the fusion chromosome.
  • Several drugs are available as treatment option, including Imatinib and Desatinib.
  • Many patients become resistant to treatment
  • Often the target of the drug mutates so that drug no longer affects it
  • The treatment of each patient has to be personalized

 


Our Approach

Chronic Myeloid Leukemia is caused by a translocation in between two chromosomes that makes a fusion protein that can be specifically targeted with drugs to treat the disease. Unfortunately, mutations in this protein often occur and results in disease that is resistant original to the first generation drug regimens. We are studying these mutations and identifying which the best 2nd generation drugs to target which the different mutations to identify the appropriate drug for each patient.