You might say it is a case of “self-control” involving the test of a stem cell therapy for an infamous and terrible disease.
Not self-control in the usual sense, but in the sense of controls during a clinical trial for a treatment prior to its release to the general public.
The key scientist involved in the trial is Clive Svendsen at Cedars-Sinai in Los Angeles. He and his team have just received the federal go-ahead for the test of a therapy for amyotrophic lateral sclerosis, commonly known as Lou Gehrig’s disease. Approval came after a 4,600-page application was submitted.
Research leading to the trial has been heavily funded by California’s $3 billion stem cell agency. About $17 million has been awarded to Svendsen related to the project, which is tackling what is a truly frightful affliction.
As Kevin McCormack, senior communications director for the stem cell agency, put it this morning in an item on the agency’s blog:
“People with ALS lose the ability to speak, eat, move and finally, breathe. The average life expectancy after diagnosis is just 3 – 4 years. It’s considered an orphan disease because it affects only around 30,000 people in the US; but even with those relatively low numbers that means that every 90 minutes someone in the US is diagnosed with ALS, and every 90 minutes someone in the US dies of ALS.”
Normally in clinical trials, a treatment is tested on two groups of patient. One group receives the treatment. The other, control group does not,. McCormack explained how Svendsen’s trial will work.
“In this clinical trial the patients will serve as their own control group. Previous studies have shown that the rate of deterioration of muscle movement in the legs of a person with ALS is the same for both legs. So Svendsen and his team will inject specially engineered stem cells into a portion of the spine that controls movement on just one side of the body. Neither the patient nor the physician will know which side has received the cells. This enables the researchers to determine if the treated leg is deteriorating at a slower rate than the untreated leg.”
The agency, formally known as the California Institute for Regenerative Medicine (CIRM), quoted its chairman Jonathan Thomas in news release as saying,“The disease affects around 30,000 people in the US, too small a number to make it economically viable for many companies to develop a treatment. Fortunately, the people of California created CIRM to get around problems like that. Our goal is not to make money. Our goal is to save lives.”
Cedars said in its press release: “The stem cell implantations for ALS patients involved in the clinical trial could begin by the end of the year, Svendsen said. Participants will come from the ALS Clinic at Cedars-Sinai, and the trial will be led by Robert Baloh, MD, PhD, and Peggy Allred, PT, DPT. The neurosurgical team will be led by J. Patrick Johnson, MD, MS.”
Ed’s Note: David Jensen, a regular contributor to Capitol Weekly, is a retired newsman who has followed the affairs of the $3 billion California stem cell agency since 2005 via his blog, the California Stem Cell Report, where this story first appeared. He has published more than 4,000 items on California stem cell matters in the past 11 years.