Early stage research from Spain could lead to a treatment for amyotrophic lateral sclerosis (ALS) using adult stem cells from bone marrow to stop progressive muscle paralysis.
Researchers from the Neuroscience Institute at the Universidad Miguel Hernández (UMH) in Elche, Alicante, and the Spanish National Research Council (CSIC) aim to test a stem cell treatment for ALS in a group of 100 patients after obtaining encouraging results in mice.
The treatment development follows the discovery that bone marrow cells transplanted to a mouse’s muscles can stop the deadly progressive muscular deterioration caused by ALS. Adult stem cells from the bone marrow may then prevent the death of motor neurons, nerve cells involved in movement. Taking stem cells from a patient’s bone marrow has the advantage that there is less risk of an immune response against the patient’s own cells.
Importantly, the treatment method allows for the amount of transplanted cells and number of transplants to be adjusted according to the size of the muscle receiving the stem cells. Additionally, the treatment is non-invasive as it does not require surgery and can be administered with only a local anesthetic.
The researchers plan to transplant bone marrow cells into the tibialis anterior muscle, which is near the shin, as well as into the hand’s muscles — two areas that are particularly affected by ALS.
The Spanish researchers are now seeking funding to start running a clinical trial for their stem cell treatment. Once approval is obtained from the Spanish Medicine Agency (AEMPS), the treatment could be tested on other muscles commonly affected by ALS, such as the diaphragm.
ALS affects around 2 out of every 100,000 people in North America and Europe. There is no known cure and the causes of ALS are still not fully understood. The need for effective treatments is great: to date, there are only two FDA-approved drugs and the average survival time for ALS is only 3 years.
Other efforts towards combating ALS include developing cell and gene therapies, monoclonal antibodies and identifying new drug candidates using AI. Additionally, CRISPR technology has been used by researchers at Stanford University to identify genes that influenced the severity of ALS, as well as identifying compounds that modify these genes and thereby improved the survival of motor neurons.
Nonetheless, the research remains in very early stages and the technology is still far from the market. Without a partner in the biotech or pharma industries, it is difficult for academic research to live up to its full clinical potential.
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