Study Determines Efficacy of Two Drugs to Treat a Form of Leukemia

Researchers have determined that two Phase 1 drugs (CX-4945 and JQ1) can work together to efficiently kill T-cell acute lymphoblastic leukemia cells while having minimal impact on normal blood cells.

Although both drugs were previously tested as single agents in clinical trials to treat cancers, the success of the combined actions on cancer cells was previously unknown until now. The findings appear in the journal Haematologica.

Acute lymphoblastic leukemia, also known as acute lymphocytic leukemia or acute lymphoid leukemia, is a form of cancer of the white blood cells, characterized by the overproduction and accumulation of cancerous, immature white blood cells, known as lymphoblasts. Despite treatment improvement, T-cell leukemia remains fatal in 20 percent of pediatric and 50 percent of adult patients. Both CX-4945 and JQ1 are in clinical trials now as single agents to treat solid and hematological cancers.

“Previous studies provided us a rationale to test the combination of CX-4945 and JQ1 on refractory/relapsed T-cell leukemia,” said corresponding author Hui Feng, MD, PhD, assistant professor of pharmacology & experimental therapeutics. “Our findings suggest that the combination treatment of CX-4945 and JQ1 could be an effective strategy to target refractory/relapsed T-cell leukemia,” she added.

According to the researchers the efficacy of using a combination of JQ1 and CX-4945 in treating other cancers should also be investigated.

This research was led by Haiwei Lian, a co-trained PhD candidate in Feng’s laboratory and Dr. Hui Fu’s laboratory at Wuhan University (China), through a close collaboration with Dr. Esther Landesman and the late Dr. David Seldin at BUSM.

Funding for this study was provided by Boston University, the National Institutes of Health, the Leukemia Research Foundation, the St. Baldrick Foundation, the Rally Foundation and the Alex Lemonade Stand Foundation.