Leukogene Therapeutics receives funding to develop compound for resistant multiple myeloma

A $2 million phase 2 Small Business Technology Transfer (STTR) grant to optimize a promising new compound that has shown efficacy in preclinical studies against treatment-resistant multiple myeloma has been awarded to a Medical University of South Carolina (MUSC) startup company, Leukogene Therapeutics, Inc., in collaboration with MUSC researcher and company founder, Nathan G. Dolloff, Ph.D. Dolloff is an assistant professor in the Department of Cell and Molecular Pharmacology & Experimental Therapeutics at MUSC and a member of MUSC Hollings Cancer Center. Dolloff and his team will use the STTR award to further develop the new compound into a drug that could be used with proteasome inhibitors in treatment-resistant multiple myeloma.

Proteasome inhibitors have contributed to the dramatic improvement in multiple myeloma treatment and outcomes over the past 15 years. They disrupt the normal ebb and flow of protein synthesis and breakdown in cells by blocking the activity of the proteasome, which is the cell’s major protein degradation machinery. This causes excess proteins to accumulate, which is highly toxic to some cancer cell types. Multiple myeloma is a cancer of plasma cells, a type of white blood cells that normally help fight off infection by producing a large quantity of proteins called antibodies. Because these cells produce a great deal of protein, they are prime targets for proteasome inhibitor treatment.

Although proteasome inhibitors work really well up front, patients eventually become resistant to the treatment. The compound Dolloff is developing is intended to provide patients with resistant multiple myeloma a new therapeutic avenue.

“Nearly all myeloma patients eventually reach that stage when their physician tells them that they have explored all the options and that there’s nothing else,” says Dolloff. “Our goal has always been to develop that next treatment option and get it to patients as quickly as possible.”

Read more: https://www.eurekalert.org/pub_releases/2018-08/muos-ltr083018.php