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ALS Stem Cell Treatments Found Safe, Slow Progression

Man in wheelchair


15 Dec. 2020. Results from an early clinical trial of stem cells to treat amyotrophic lateral sclerosis, or ALS, shows the therapy is safe and slows progression of the disease. The findings from a second group of patients enrolled in the trial are reported by Kadimastem Ltd., a biotechnology company in Ness Ziona, Israel.

ALS, also known as Lou Gehrig’s disease, is a progressive neurodegenerative disorder where neurons or nerve cells controlling muscles in the body begin to waste away, and can no longer send or receive signals from the brain or spinal cord. As the nerve cells stop functioning, muscles in the limbs, and later speech and breathing muscles, begin weakening and eventually stop functioning. Most people with the disease die of respiratory failure.

Kadimastem develops regenerative medicine therapies from human embryonic stem cells. The company’s technology is based on research by Michel Revel, professor emeritus of molecular genetics at the Weizmann Institute of Science and the company’s co-founder and chief scientist. For ALS, Kadimastem cultures stem cells from healthy donors to differentiate or transform into functioning astrocytes, cells in the brain that support nerve cells that send and receive signals. Healthy astrocytes are then expected to support normal nerve cell signaling in the brain, rather than degrade signaling as occurs in ALS patients.

The early-stage clinical trial is enrolling 21 patients with ALS at the Hadassah Medical Center in Jerusalem. Participants are randomly assigned to receive one of three dosage levels of Kadimastem’s AstroRx treatments as injections into the spinal column. Injected astrocytes are then expected to flow in cerebrospinal fluid from the spinal column into the brain, where they reduce oxidative stress and other toxicities, and promote production of neuroprotective proteins.

After the astrocyte injections, participants are monitored for 11 months, with the study team looking primarily for adverse effects from the treatments. The researchers are also tracking changes in patients’ functioning ability, based on measurements on a standard rating scale of ALS functioning, as well as muscle strength and scores on a quality of life questionnaire.

The new findings report on experiences of five ALS patients in the trial’s second of three groups. For those five patients, six months after the injections, results show no serious adverse events or toxicities that may limit the treatment’s dose levels. In addition, says Kadimastem, the decline in function loss by patients slowed measurably in the three months following injections, compared to the three to four month period preceding injections. And after six months, says the company, scores on the standard ALS function scale were similar to patients’ ratings before the treatments.

Kadiamastem reported similar safety and functional rating scale scores in January 2020, with the first group of five patients receiving a lower dose of the AstroRx therapy. Results from the third group of patients are expected in the first half of 2021.

“The results after six months of follow up are encouraging, as they suggest a clinically meaningful signal of effect for a period of three months by a single administration of AstroRx and confirm the safety of AstroRx,” says Haddasah Medical Center neurologist Marc Gotkine, the trial’s principal investigator in a Kadimastem statement. “These results support a further, randomized-controlled, clinical trial with repeated doses of AstroRx in patients with ALS, in order to prolong the clinical effect observed by a single dose.”

While Kadimastem’s results are encouraging, recent results from a late-stage trial shows stem cell treatments do not slow progression of ALS when compared to a placebo, as reported by Science & Enterprise in November. That trial, conducted in the U.S., tested a therapy developed by BrainStorm Cell Therapeutics Inc. in New York.

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