Glial Cell Therapy Gets FDA's Orphan Drug Designation for Transverse Myelitis

TM is a debilitating autoimmune disease that destroys parts of the spinal cord
TM is a debilitating autoimmune disease that destroys parts of the spinal cord

Q Therapeutics announced that Q-Cells (human glial restricted progenitor cells and their progeny), an adult cell product, has been granted Orphan Drug Designation for the treatment of transverse myelitis (TM).

TM is a debilitating autoimmune disease that destroys parts of the spinal cord and may lead to total paralysis; currently, there are no treatments that restore the myelin once it has been destroyed. The Q-Cells product is intended to restore or preserve normal central nervous system (CNS) activity by supplying essential nerve cell functions. The cells are isolated and cultured from brain tissue at the precise stage of development when their ability to multiply, migrate, and differentiate into glial cells is most potent.

Preclinical data showed that Q-Cells replaced missing myelin in mice, restoring the animals to normal function and life expectancy. Other preclinical models of spinal cord injury also showed the restorative capabilities of Q-Cells as they have successfully differentiated into astrocytes and oligodendrocytes.  

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The Company has obtained Investigational New Drug (IND) clearance for Phase 1/2a trials of Q-Cells in both TM and amyotrophic lateral sclerosis (ALS). 

For more information visit Qthera.com.