Seventy-five years after baseball player Lou Gehrig was diagnosed with ALS, Mayo Clinic researchers have begun a human study hoping to slow progression of the devastating ailment.
ALS, or amyotrophic lateral sclerosis, has become widely known as Lou Gehrig's disease.
The catastrophic diagnosis carries a crushing prognosis for both the patient and those around him.
Gehrig, for instance, only lived two years after he was diagnosed. But the new treatment offers the hope of extending the life of those with ALS.
"Lifespan after diagnosis is roughly two to three years, but there's a huge range," said Mayo neurologist Dr. Nathan Staff. Affected individuals can survive more than a decade after diagnosis, or die within six months.
Brian McCoy, a Rochester carpenter, is one of the first 25 people ina Phase I ALS stem-cell clinical trial at Mayo in Rochester.
"I have nothing to lose," said McCoy, who was diagnosed in October, 2012."I don't think it will help me. But, down the road, it might help somebody."
The trial is a safety study to determine if it's safe to give ALS patients injections of stem cells grown from samples of their own fat tissue.
If the process is indeed safe, the study will be expanded to more patients. That will allow researchers to further study both safety and effectiveness in a Phase II randomized, double-blind, placebo-controlled clinical trial.
"We don't know if it will work or not," said Dr. Anthony Windebank, deputy director for discovery in the Center for Regenerative Medicine at Mayo. "It may even do harm."
If the treatment is both safe and effective and has a marked impact on survival, it would be the first body-heal-thyself treatment designed specifically to slow ALS progression.
More than 700 people with ALS have expressed interest in joining one of the Mayo studies, illustrating the tragic reach of the disease.
Currently there's only one main drug that extends the life of ALS patients. But ion average, it only adds about two months to lifespan.
And that short time, McCoy says, just isn't enough.
Mayo researchers dream of adding a new treatment tool to fight the disease directly, instead of fighting symptoms.
The ALS Association says "progressive degeneration of the motor neurons in ALS eventually leads to their death."
When the neurons die, "the ability of the brain to initiate and control muscle movement is lost. With voluntary muscle action progressively affected, patients in the later stages of the disease may become totally paralyzed."
The chance to actually make a difference in survival and quality of life for people who otherwise have no options, Windebank said,is "what really make one want to get up in the morning and go to work — you're working on something that has the potential to make a world of difference."
Health reporter Jeff Hansel writes the Pulse on Health column every Monday. Follow him on Twitter @JeffHansel.
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