Lentigen, a biotechnology company specializing in the development and manufacture of lentiviral gene delivery products, announced that that the FDA has granted orphan drug status to LG631-CD34, P140K methylguanine methyltransferase (MGMT) transduced human CD34 cells, for bone marrow protection in the treatment of glioblastoma multiforme.

The designation qualifies Lentigen for seven years of market exclusivity following marketing approval by the FDA and provides other development-related incentives.

"LG631-CD34 consists of the patients' adult hematopoietic stem cells genetically modified with a Lentiviral vector expressing a human MGMT gene variant, which is designed to protect the cells from the toxic side-effects of Temodar, a standard of care treatment for glioblastoma multiforme," said Dr. Boro Dropulic, chief scientific officer of Lentigen. "Protection of blood-forming hematopoietic stem cells from the side-effects effects of Temodar would provide immediate benefits to patients. It potentially enables higher doses and more intensive drug treatment with reduced toxicity, resulting in improved clinical outcomes.”

LG631-CD34 is currently being evaluated in a NIH grant-funded phase I clinical trial  at University Hospitals Case Medical Center. The trial is supported by a grant from the National Cancer Institute and another grant to the Center for Stem Cell & Regenerative Medicine (CSCRM) from Ohio's Third Frontier Commission under its Research Commercialization Program.

"Glioma is such an aggressive and challenging cancer," said Dr. Stanton Gerson, MD, director of the Seidman Cancer Center at UH Case Medical Center. "When patients are diagnosed with this life threatening disease, they have an average life expectancy of less than 12 months. The medical community needs to find new treatment strategies that can improve clinical outcomes in this devastating disease. I am hopeful that LG631-CD34 can make an important contribution and help us improve the lives of our patients."

Gerson is an inventor on this mutant alkyltransferase technology. He, as well as Case Western Reserve University and University Hospitals, could gain from commercialization of the invention.