A new therapeutic agent could be effective in treating cancer with certain gene mutations

Researchers from Mount Sinai have developed a therapeutic agent that shows high efficacy in vitro in disrupting the biological pathway that helps cancer survive, according to a paper published in Cancer detection, Journal of the American Association for Cancer Research, in July.

Therapy is a constructed molecule, called MS21, that causes the breakdown of AKT, an enzyme that is overactive in many cancers. This study presented evidence that the pharmacological degradation of AKT is a viable treatment for cancers with mutations in certain genes.

AKT is a cancer gene that encodes an enzyme that is often abnormally activated in cancer cells to stimulate tumor growth. Degradation of AKT reverses these processes and inhibits tumor growth.

“Our research lays a solid foundation for the clinical development of ACT degradation for the treatment of human cancer with certain gene mutations,” Dr. Sc. Ramon Parsons, director of the Tisch Cancer Institute and Ward-Coleman, chairman of cancer research and the Department of Oncology at Icahn Medical School on Mount Sinai. “A study of 44,000 human cancers found that 19 percent of tumors have at least one of these mutations, suggesting that a large population of cancer patients could benefit from ACT degradation therapy such as MS21.”

MS21 has been tested on cell lines derived from human cancer, and these are models used in laboratories to study the effectiveness of cancer therapies. Mount Sinai wants to develop MS21 with an industrial partner to open clinical trials for patients.

Translating these findings into effective cancer therapies for patients is a high priority because the mutations and resulting pathways to cancer presented in this study are probably the most frequently activated pathways in human cancer, but this effort has proven particularly challenging. We look forward to the opportunity to develop this molecule into a therapy that is ready to be studied in clinical trials. “

Dr. Jian Jin, Professor of Mount Sinai Therapy Discovery and Director of Mount Sinai Therapy Discovery Center on Mount Sinai Icahn


Sinai Mountain Health System

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