A cell therapy intended to boost myelin regeneration — Q-Cells by Q Therapeutics — has received a green light from the U.S. Food and Drug Administration to proceed with a clinical trial in patients with transverse myelitis (TM), a disease that like multiple sclerosis is characterized by myelin damage.
FDA approval of the company’s Investigational New Drug (IND) application allows researchers to start a Phase 1/2 clinical trial in which nine patients 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 instance, the myelin-producing oligodendrocytes in multiple sclerosis.
Q-Cells are what researchers call glial-restricted progenitor cells. In plain language, it means that they 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.
“This is another milestone in our quest to bring effective treatments for devastating CNS [central nervous system] diseases and injuries to the clinic,” Steven Borst, CEO and chairman of Q Therapeutics, said in a press release.
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.
Interestingly, the study reported that benefits did not correlate in time with the formation of new myelin. Animals became better before new myelin was fully formed, suggesting that the treatment might have additional beneficial effects.
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.
The company is also planning to launch a trial in ALS patients.
“The FDA’s clearance of this IND is yet another validation of our collaborative and purposeful approach to move Q-Cells into the clinic,” added James Campanelli, PhD, vice president of Research and Development at Q Therapeutics. “We are eager to move forward with this trial and optimistic that Q-Cells will prove effective in treating human CNS injury and disease.”