ROCHESTER, Minn. — Seventy-five years ago, on July 4th 1939, baseball legend Lou Gehrig delivered the famous speech bidding farewell to the ballpark and his fans. Two weeks before Gehrig had been diagnosed with amyotrophic lateral sclerosis (ALS) at Mayo Clinic in Rochester, Minnesota. Accompanied by his wife, Eleanor, Lou left Mayo Clinic with the devastating diagnosis on June 20th 1939, a day after his 36th birthday. He died in June two years later, not quite 38 years old, of the rare neurological disease that would come to bear his name.
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ALS is a type of progressive motor neuron disease that typically strikes at middle to later life and causes nerve cells in spinal cord, brain stem and brain to gradually break down and die. These nerve cells are responsible for muscle function so eventually, ALS can affect the ability to control the muscles needed to move, speak, eat and breathe.
While ALS still evades cure and effective treatment, researchers at Mayo Clinic are conducting Phase I clinical trial in the hope that they can guide newly grown stem cells to become protective of neuromuscular function.
“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,” explains neurologist Anthony Windebank, M.D, deputy director for discovery in the Center for Regenerative Medicine at Mayo Clinic in Rochester. “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. If we can halt an ALS patient's loss of cells at 20 to 30 percent, that person’s function would be well-preserved," says Dr. Windebank.
In the current phase of the FDA-controlled trial, Dr. Windebank and his team are studying the safety and efficacy of the treatment. If injecting ALS patients with stem cells grown from samples of their own fat tissue is found to be safe, the research would move to a Phase II, randomized, double blind, placebo-controlled trial to allow further study of safety and efficacy on a greater number of patients.
The FDA just approved another clinical trial in which Mayo Clinic will take part. The BrainStorm Phase II trial will look into whether stem cells can be used to actually replace the neurons that have been destroyed by ALS.
About Mayo Clinic
Recognizing 150 years of serving humanity in 2014, Mayo Clinic is a nonprofit worldwide leader in medical care, research and education for people from all walks of life. For more information, visit 150years.mayoclinic.org, www.mayoclinic.org and newsnetwork.mayoclinic.org.
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