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Cell Therapy Trial Approved, Aimed to Restore Myelin

July 31, 2017

A cell therapy intended to boost myelin regeneration, Q-Cells by Q Therapeutics, has been approved by the U.S. Food and Drug Administration to proceed with clinical trial in patients with transverse myelitis (TM). TM is a disease, like multiple sclerosis (MS), which is characterized by myelin damage. The company’s Investigational New Drug (IND) can now be studied in their Phase ½ clinical trial. The trial will consist of nine patients, who will receive increasing doses of the treatment. Q Therapeutics hopes trial results will support further development of Q-Cells to treat other diseases that lack healthy glial cells. For example, myelin-producing oligodendrocytes in MS.

Q-Cells are stem cell-like cells that are destined to become glial cells, which in the brain and spinal cord perform numerous and indispensable functions, including myelin formation. In the study, “Transplanted human glial-restricted progenitors can rescue the survival of dysmyelinated mice independent of the production of mature, compact myelin,” published in the journal Experimental Neurology, Piotr Walczak and colleagues at Johns Hopkins University showed that mice without myelin survived for longer when treated with the cell therapy. “We have long believed that Q-Cells’ unique ability to repair and support CNS nerve cells is fundamental to treating many CNS disorders. The ability of these cells to replicate once injected, migrate, differentiate into mature glial cells and repair myelin, as demonstrated by Dr. Walczak’s lab, further highlights the power of this therapeutic approach,” said Mahendra Rao, MD, PhD, Q Therapeutics’ scientific co-founder and chief strategy officer.

The study also reported that benefits did not correlate in time with the formation of new myelin. Animals became better before new myelin was fully formed. Other data from animal studies also support the idea that brain and spinal cord disease or injury can be treated by delivering healthy glial cells. “Our approach uses the glial cell’s natural ability to repair and support nerve cells in the CNS. Q-Cells hold great promise not only for those people with rare diseases such as TM and ALS [amyotrophic lateral sclerosis], but for the many people worldwide who live with MS [multiple sclerosis], spinal cord injury, and stroke,” Borst said.