Those living with amyotrophic lateral sclerosis — also known as Lou Gehrig’s Disease — typically lose their lives within three to five years of being diagnosed as they eventually lose control of the muscles needed to move, speak and breathe. Treatment options are often scare.
However, new research at the University is aiming to correct the deficit of treatment options. Following approval by an independent ethics review committee, University researchers will begin to conduct a clinical trial using direct injection of stem cells into the spinal cord of ALS patients.
Principal investigator Eva Feldman, director of the A. Alfred Taubman Medical Research Institute, said this trial could possibly result in new options for treatment.
“Stem cells provide a new treatment avenue in a disease where there are few other viable options,” Feldman said.
Lisa Bardach, a speech pathologist at ALS of Michigan, said Michigan has a higher incidence of ALS than other states, adding that the community is enthusiastic about the potential research.
“People who have ALS couldn’t care less about those politics. It’s very exciting research because it’s something that gives us hope, and anything that gives us hope is a wonderful thing,” Bardach said.
The disease involves the degeneration of motor neuron — cells that convey impulses from the brain to muscles. Four percent of patients live longer than 10 years and most die of respiratory failure within three to five years.
ALS has only one treatment approved by the Food and Drug Administration, a drug called Rilutek, which slows the disease’s progression by three to five months.
The trial’s critics are disturbed by the source of the stem cells — the spinal cord of an 8-week-old fetus.
Feldman said the cells were provided by trial sponsor Neuralstem Inc., a Maryland-based biotech company. The mother gave consent to the usage of the fetus’ cells a decade ago and the cells will be used under the guidelines of the FDA, the National Institutes of Health and an outside independent review board.
Feldman said the research, and its use of stem cells, has received an “overwhelmingly positive” community response.
Ed Rivet, legislative director of Right to Life, a group that advocates against abortion, said the organization has received an unusual amount of press attention concerning its response to the University research.
“It’s utilitarianism to the worst degree,” Rivet said. “It’s one thing to say, ‘You know, I can donate blood that can help another person.’ But this is destroying a developing human fetus and using that as medical treatment. It’s just unethical.”
Rivet said this was the first time a Michigan university is involved with a large-scale stem cell research project. However, Right to Life, whose activities mainly focus on advocacy and education, is not currently organizing any legislative action to counter the trials.
LSA junior Victoria Criswell, president of Students for Life, said her group will also not be taking action, aside from the possibility of hosting a debate on stem-cell research.
She added that Students for Life supports stem cells taken from umbilical cords or bone marrow.
This trial will deal with developing new treatments. The first phase of the trial concerned safety and took place at Emory University School of Medicine. Out of 18 patients, one regained use of his legs. Feldman said, to her knowledge, such results have not been reported with other therapies.
The FDA approved expansion into the next phase on April 15.
“In a Phase Two trial, you get to expand your approach,” Feldman said. “You’re not only looking at safety but the efficacy of the product you are giving to the patient.”
A custom-built apparatus injects stem cells directly into the patient’s spinal cord, which is exposed after the removal of some of the patient’s vertebrae. The Phase One trial involved 500,000 to one million stem cells injected in the patient, while Phase Two will involve two to 16 million.
Follow Rachel Premack on Twitter at @rr_premack.