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FDA allows Q-cells for first-in-human transverse myelitis trial

The US Food and Drug Administration (FDA) has given Q Therapeutics, developer of clinical-stage cell therapies for central system disease and injury, the go ahead on a phase 1/2a clinical trial of Q-Cells in patients with transverse myelitis.

Transverse myelitis is inflammation of the spinal cord. Similar to multiple sclerosis (MS) it destroys the myelin sheath surrounding nerve fibres in the spinal cord, compromising muscle control and affecting sensation.

“This is another milestone in our quest to bring effective treatments for devastating central nervous system diseases and injuries to the clinic,” said Steven Borst, the company's CEO and Chairman. 

Clinical trials in the nine-person, dose-escalation transverse myelitis safety study will pave the way to developing Q-Cells for treatment of several debilitating disorders caused by a lack of healthy glial cells. 

“Our approach uses the glial cell's natural ability to repair and support nerve cells in the central nervous system,” explains Borst. “Q-Cells hold great promise not only for those people with rare diseases such as transverse myelitis and ALS (Amyotrophic Lateral Sclerosis), but for the many people worldwide who live with MS, spinal cord injury and stroke.”

The company also has FDA allowance to initiate a phase 1/2a clinical trial in ALS or Lou Gehrig's disease.

Q-Cells are human glial-restricted progenitor cells (GRPs) and the Company's first patented cellular therapeutic candidate. As reported in the May 2017 issue of Experimental Neurology, Piotr Walczak, MD, PhD, and his team at The Johns Hopkins University showed that Q-Cells exert a robust therapeutic effect when transplanted into the central nervous system of animals born unable to produce healthy myelin. The Q-Cells performed multiple repair and support functions resulting in myelination and restoration of animals to normal function with extended life span. These data, along with other published pre-clinical data from animal models of central nervous system disease and injury, show that delivering healthy glial cells into the brain and spinal cord can alter the course of currently incurable central nervous system conditions. 

“We have long believed that Q-Cells' unique ability to repair and support central nervous system nerve cells is fundamental to treating many central nervous system 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. “We are eager to move forward with this trial and optimistic that Q-Cells will prove effective in treating human central nervous system injury and disease.”

Date: 27/07/17

Source: MS-UK

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