Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, is one of those conditions that a lot of people know about but don’t know a lot about. If they are fortunate it will stay that way. ALS is a nasty neurodegenerative disease that attacks motor neurons, the cells in the brain and spinal cord that control muscle movement. As the disease progresses the individual loses their ability to walk, talk, eat, move and eventually to breathe. There are no effective treatments and no cure. But now research out of Texas is offering at least a glimmer of hope.
Dr. Stanley Appel, a neurologist at the Houston Methodist Neurological Institute noticed that many of the ALS patients he was treating had low levels of regulatory T cells, also known as Tregs. Tregs play a key role in our immune system, suppressing the action of molecules that cause inflammation and also helping prevent autoimmune disease.
In an article on Health News Digest Appel said:
Dr. Stanley Appel: Photo courtesy Australasian MND Symposium
“We found that many of our ALS patients not only had low levels of Tregs, but also that their Tregs were not functioning properly. We believed that improving the number and function of Tregs in these patients would affect how their disease progressed.”
And so that’s what he and his team did. They worked with M.D. Anderson Cancer Center’s Stem Cell Transplantation and Cellular Therapy program on a first-in-human clinical trial. They took blood from three people with different stages of ALS, separated the red and white blood cells, and returned the red blood cells to the patient. They then separated the Tregs from the white blood cells, increased their number in the lab, and then reinfused them into the patients, in a series of eight injections over the course of several months.
Their study, which appears in the journal Neurology,® Neuroimmunology & Neuroinflammation, found that the therapy appears to be safe without any serious side effects.
Jason Thonhoff, the lead author of the study, says the therapy also appeared to help slow the progression of the disease a little.
“A person has approximately 150 million Tregs circulating in their blood at any given time. Each dose of Tregs given to the patients in this study resulted in about a 30 to 40 percent increase over normal levels. Slowing of disease progression was observed during each round of four Treg infusions.”
Once the infusions stopped the disease progression resumed so clearly this is not a cure, but it does at least suggest that keeping Tregs at a healthy, high-functioning level may help slow down ALS.
CIRM is funding two clinical trials targeting ALS. One is a Phase 1 clinical trial with Clive Svendsen’s team at Cedars-Sinai Medical Center, the other is a Phase 3 project with Brainstorm Cell Therapeutics.
The Stem Cellar 
Something I am trying to understand is, that you are telling the world all a out the breakthrougs of your findings in the stem cell field I had applied so many times and they do not call me, I am dying of Snogrens syndrome and nobody seems to care, but there are you guys bragging of your new discoveries in the stem cell field
Dear Jose, I’m so sorry to hear about your health problems and difficulty with getting on a clinical trial. We don’t mean to brag, the goal is simply to let people know about progress in stem cell research. We know that this does not always help people battling different conditions right now, but we hope it will at least convey that research is underway that will eventually help people.
My sister Nancy is still struggling in the later stages of ALS. She has lost her speech and is confined to a wheelchair. Has a stomach tube and her husband told me she will be getting hospice care soon because they can address her needs better. I realize this is not a cure but anything that would slow the disease progression would be welcomed. Hopefully participating in a clinical trial would still be an option although her breathing rate is poor.
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