Stem cell-gene therapy shows promise in ALS safety trial

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Stem cell-genetherapy shows promise in ALS safety trial NatureMedicine

In the first trial of its kind, the Cedars-Sinai team showed that delivery of this combined treatment is safe in humans. The findings were reported today in the peer-reviewed journal"Using stem cells is a powerful way to deliver important proteins to the brain or spinal cord that can't otherwise get through the," said senior and corresponding author Clive Svendsen, Ph.D.

Along with Avalos, Robert Baloh, MD, Ph.D., previously a professor of Neurology at Cedars-Sinai and now global head of neuroscience at Novartis, and J. Patrick Johnson, MD, co-medical director of the Spine Center at Cedars-Sinai, are co-lead authors on the publication.The primary goal of the trial was to ensure that delivering the cells releasing GDNF to the spinal cord did not have any safety issues or negative effects on leg function.

After the transplantation, patients were followed for a year so the team could measure the strength in the treated and untreated legs. The goal of the trial was to test for safety, which was confirmed, as there was no negative effect of the cell transplant on muscle strength in the treated leg compared to the untreated leg.

 

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NatureMedicine If it's safe, grant 'compassionate use' to ALS patients immediately. Give hope where there is none.

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