New cancer drug tested in mice may benefit certain leukaemia patients

Almost 6,000 new cases of acute lymphoblastic leukemia, or ALL, are expected to be diagnosed this year in the United States. Up to 30 percent of adult acute lymphoblastic leukemia or ALL patients have Philadelphia chromosome, where two segments of chromosomes have aberrantly fused together. Adult ALL patients often see high relapse rates, and treatment-related deaths remain high. Researchers now report on a study that could provide better therapeutic options for patients. In the Leukemia study, HCI scientists learned how to inhibit and override the negative activity of this chromosome. The findings could possibly lead to future novel drug treatments. The Philadelphia chromosome promotes repair through numerous proteins. But putting together a cocktail of drugs to inhibit them all would likely be too toxic and affect normal cells. So, Srividya Bhaskara, HCI investigator and assistant professor of radiation oncology at the University of Utah, focused on two specific proteins she found directly involved in DNA repair, called histone deacetylases (HDAC) 1 and 2. She has now collaborated with a company to make a drug that inhibits HDAC1,2 activity. The HDAC1,2 inhibitor drug has been tested in patient samples and mice, showing encouraging results, either alone or in combination with a chemotherapy drug called doxorubicin.

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