Targeting ‘undruggable’ leukemia proteins

Ani Deshpande, PhD at Sanford Burnham Prebys Medical Discovery Institute in California is working to target previously ‘undruggable’ proteins in acute lymphoblastic leukemia.

The problem:

Research helps kids like Liam, who is fighting ALL. Click to read "Leukemia and Lightsabers - Liam's Story."

Some types of childhood leukemia have two genes that fuse together to form a cancer-causing fusion protein. Some of these fusions proteins make the leukemia extremely difficult to treat. “The gene fusions that we study are associated with dismal survival outcomes,” said Dr. Deshpande. “There are no clinically effective targeted therapies for children with these fusions.” The mandated use of extremely aggressive non-specific therapies leads to significant toxic side effects. Many of these patients immediately undergo high-risk bone marrow transplants. Originally thought of as a rare mutation, it is increasingly coming to light that fusions of the AF10 gene happen in a relatively large number of patients.

How his research will help:

Dr. Deshpande’s team is aiming to continue to understand how AF10 fusions work and to find a drug to target these genes.

”There are two things we hope to achieve: (1) to create another treatment option where one does not exist, and (2) to make therapies which are less toxic and more efficacious,” he said. First, they want to study how leukemia with an AF10  initiates and maintains itself. Then, the key is approaching the fused genes with the right drug, which is what his lab will be spending their CCRF funding to determine.

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