Funded in partnership with the Dolphins Cancer Challenge (DCC)
Chronic myelomonocytic leukemia (CMML) is a form of cancer of the blood in which malignant cells multiply very fast and accumulate, leaving no room for healthy cells to grow. This rare form of cancer does not respond well to current available treatments, leaving the majority of patients with very few options to find a cure. Even for patients who do respond to the treatments, the disease quickly comes back so that, overall, less than 20% of patients survive more than 5 years after the disease is first found. In our recent work, we studied a large group of CMML patients and analyzed their disease. We found that patients who did not respond to treatment all shared the presence of a gene known as PRAME, yet patients who did better never had this gene present. Very little is known about PRAME’s role in cancer cells in general and CMML in particular. Recently, new treatments have been developed that specifically kill cells with PRAME. While these new treatments are being studied in other cancers, their impact on CMML is unknown. Therefore, we will test whether these new approaches may be used for patients with this aggressive form of leukemia.