Investigators led by Eva Feldman, a neurologist at the University of Michigan, have been given the green light to take another step forward in a small but very closely watched clinical trial of a new stem cell treatment for Lou Gehrig's disease.
After successfully injecting stem cells into the lower spines of 12 patients with ALS, with no significant safety issues raising a red flag, the FDA has given the go-ahead on injections for the upper spine in a select group of six patients, Neuralstem ($CUR) announced. While not powered to provide solid efficacy data, on an anecdotal basis the therapy has provided some additional proof of concept.
"Our quantitative clinical assessments showed no evidence of acceleration of disease following stem cell injections, meeting our stated goal of proving safety for this Phase I trial," said Feldman and two of her colleagues in a prepared statement. "We have cautious optimism that a few of the patients may have slowed in their progression of lower extremity weakness, and one patient may have improved."
One patient who had required a cane to walk and often experienced trouble breathing told CNN recently that the treatment had produced "miraculous" results. Regulators, though, will require much better data than that before even beginning to consider an approval for Neuralstem's treatment.
"The goal of our cell therapy program is to create therapies that will slow down, stabilize or reverse functional deficits in central nervous system (CNS) diseases," said Neuralstem CSO Karl Johe. "By moving the cell delivery to cervical spinal cord--the first time the FDA has approved intraspinal injections in this region--we will demonstrate that we can deliver our cells safely and routinely to all parts of the spinal cord."
- here's the press release
- get the story from AnnArbor.com