New study found that New Gene Therapy Developed That Could Be Effective Against Many Types of Cancer

Topic: New study found that New Gene Therapy Developed That Could Be Effective Against Many Types of Cancer

By Mount Sinai School of Medicine

According to a paper published in July in Cancer Discovery, a journal of the American Association for Cancer Research, researchers at Mount Sinai have developed a therapeutic agent that shows high effectiveness in vitro in inhibiting a biological pathway that helps prevent cancer survival. helps in.

The therapy is an engineered molecule, named MS21, that causes the degradation of AKT, an enzyme that is highly active in several cancers. This study provided evidence that 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. Depletion of AKT reverses these processes and inhibits tumor growth.

“Our study lays a solid foundation for the clinical development of an AKT degrader to treat human cancers with certain gene mutations,” said Ramon Parsons, MD, PhD, director of the Tisch Cancer Institute and Ward-Coleman in Cancer Research. Chair of Oncological Sciences at the Icahn School of Medicine at Mount Sinai. “An investigation of 44,000 human cancers showed that 19 percent of tumors contained at least one of these mutations, which suggests that a large population of cancer patients may benefit from therapy with AKT degraders such as MS21. Is.”

MS21 was tested in human cancer-derived cell lines, which are models used in laboratories to study the efficacy of cancer therapies. Mount Sinai is looking to develop MS21 with an industry partner to begin clinical trials for patients.

“Translating these findings into effective cancer therapies for patients is a high priority because the mutations and the resulting cancer-driving pathways that we determine in this study are arguably the most active pathways in human cancer, but this effort is particularly has proven to be challenging,” said Jian Jin, PhD, Mount Sinai Professor of Therapeutics Discovery and director of the Mount Sinai Center for Therapeutics Discovery at Icahn Mount Sinai. “We look forward to the opportunity to develop this molecule into a therapy that is ready for study in clinical trials.”