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A therapeutic agent has been developed by Mount Sinai researchers!

Gene Therapy

According to a paper published in Cancer Discovery, in July, a therapeutic agent has been developed by Mount Sinai researchers and high effectiveness is shown in vitro at disrupting a biological pathway that helps cancer survive.

The therapy named MS21 is an engineered molecule, and this causes the degradation of ATK. ATK is an enzyme that is overly active in many cancers. This study found that pharmacological degradation of AKT is a viable treatment for cancers with mutations in certain genes. AKT is a cancer gene that produces an enzyme that is frequently abnormally activated in cancer cells to stimulate tumour growth. When ATK degradants, these processes are reversed and inhibits tumour growth.

According to Ramon Parsons, MD, PhD, Chair of Oncological Sciences at the Icahn School of Medicine at Mount Sinai, Director of The Tisch Cancer Institute and Ward-Coleman Chair in Cancer Research, “Our study lays a solid foundation for the clinical development of an AKT degrader for the treatment of human cancers with certain gene mutations. Examination of 44,000 human cancers identified that 19 per cent of tumours have at least one of these mutations, suggesting that a large population of cancer patients could benefit from therapy with an AKT degrader such as MS21.”

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

According to Jian Jin, PhD, Mount Sinai Professor in Therapeutics Discovery and Director of the Mount Sinai Center for Therapeutics Discovery at Icahn Mount Sinai, for the patients translating these findings into effective cancer therapies is on high priority because the resulting cancer-driving pathways and mutations that they layout in this study are arguably the most commonly activated pathways in human cancer. But this effort is very challenging. They are looking forward to an opportunity to develop this molecule into a therapy that is ready to be studied in clinical trials.

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