Researchers at the Mayo Clinic hope that a Phase I clinical trial will enable them to guide newly grown stem cells to become protective of neuromuscular function and that it will shed light on ways to halt or slow the progression of amyotrophic lateral sclerosis.
“We use fat-derived mesenchymal stem cells from the patient’s own body. These cells are modified in the laboratory and delivered through a spinal tap into the fluid around the patient’s nervous system to promote neuron survival,” Anthony Windebank, MD, deputy director for discovery in the Center for Regenerative Medicine, at Mayo Clinic, in Rochester, Minn., stated in a press release from the Mayo Clinic. “We hope that the growth factors that they are producing will help protect and promote the survival of nerve cells and therefore slow down or arrest the progression of ALS [amyotrophic lateral sclerosis]. If we can halt an ALS patient’s loss of cells at 20 to 30 percent, that person’s function would be well-preserved.”
Windebank and colleagues are looking at the safety and efficacy of the stem cell treatment in the FDA-controlled trial. If they find it is safe to inject patients with ALS with stem cells harvested from the patients’ own fat cells, the second phase of the trial will begin, according to the release.
Phase II, a randomized, double-blind, placebo-controlled trial, would allow further study of safety and efficacy of this approach in a greater number of patients.
According to the release, the FDA also recently approved Mayo Clinic’s participation in the BrainStorm Phase II trial, which is focused on whether stem cells actually replace neurons damaged by ALS.