How regenerative medicine is advancing ALS research
This neurodegenerative disorder causes nerve cells in the brain and spinal cord to die, blocking signals to the muscles. That results in paralysis that robs a person of the ability to walk, talk and breathe. Because there is no cure, ALS eventually leads to respiratory weakness and death. Roughly 1 in 10 cases of ALS are the inherited type that are linked to genetic mutations. However, 90% are a sporadic type that are without an obvious cause and presumed to be due to interactions between a person's genetics and environmental exposures. People over 40, smokers and men appear to be at a higher risk for the disease. This means the disease seems to occur at random with no clearly associated risk factors and no family history of the disease.
New options are needed, because the drugs approved by the Food and Drug Administration (FDA) to treat ALS are not effective enough at slowing the course of the disease. A transformation is underway in health care that is shifting the focus of medicine from fighting disease to rebuilding health. Investigators are probing whether mesenchymal stem cells derived from adipose, or fat, tissue could provide growth factors and anti-inflammatory properties to protect nerve cells from dying, thereby halting disease progression.
Results of a phase 1 clinical trial at Mayo Clinic found mesenchymal stem cell therapy is safe and warrants further study. Building on the phase 1 clinical trial, Mayo Clinic researchers are testing stem cell therapy in a larger group of patients for a longer period of time. The study seeks to answer whether stem cells can slow the debilitating effects of ALS, and if so, why stem cell therapy works for some but not others.
"We are trying to understand what is driving the different responses. Is it the patient's genetics? Is it the immune system? Is it possibly the cells themselves and how they're reacting to the therapeutic environment? Our current studies try to understand the different factors that cause people to respond or not respond to mesenchymal stem cell therapy," says Dr. Staff, principal investigator of the phase 2 clinical trial for ALS.
The research team is optimistic that clinical trials at Mayo and around the world will someday bring new answers and new options for patients. In parallel, the discovery of genetic mutations also has spawned gene therapy clinical trials for patients with the inherited form of the disease.
https://regenerativemedicineblog.mayoclinic.org/2021/05/07/how-regenerative-medicine-is-advancing-als-research/
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