Rare Disease Report

Orphan Drug Designation Granted to Q-Cells for Treatment of Transverse Myelitis

SEPTEMBER 27, 2018
Krista Rossi
The US Food and Drug Administration (FDA) has granted an orphan drug designation to Q Therapeutics Inc.’s Q-Cells for the treatment of transverse myelitis, a rare disease caused by the inflammation of 1 level of the spinal cord.

“Orphan drug designation of Q-Cells for transverse myelitis is a great step toward our goal of advancing glial cell therapy for demyelinating and neurodegenerative diseases,” Steven Borst, MBA, president and CEO of Q Therapeutics Inc., said in a recent statement.

Q-Cells are human glial restricted progenitor cells and their progeny. In preclinical tests conducted by collaborators at Johns Hopkins University last year, normal function and life expectancy were restored in mice through the use of the therapy, which replaced the missing myelin. The preclinical data prompted the FDA’s swift clearance of Q Therapeutics’ proposed TM clinical trial.
 
“People who are hit hardest by transverse myelitis face a lifetime of paralysis and wheelchair confinement,” Borst added. “Building upon the remarkable, disease-modifying results that have been achieved in pre-clinical studies, we aim to someday reverse that paralysis, not only in these patients, but all who have disabilities that result from damaged or destroyed myelin.”

A debilitating autoimmune disease that can lead to paralysis in many affected individuals, transverse myelitis destroys key components of the spinal cord. While transverse myelitis is similar to multiple sclerosis, it can cause paralysis in patients much more rapidly—sometimes in just 24 to 36 hours. This is due to myelin sheaths that surround nerve fibers that are attacked by the immune system.

Currently, no effective therapies that can replace destroyed myelin exist.  

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