An experimental therapy developed by researchers at Cedars-Sinai Center in Los Angeles, California (USA) has shown that stem cell transplant Possible and safe for patients suffering from amyotrophic lateral sclerosis (ALS). The results are published in the peer-reviewed scientific journal nature medicine,

By combining gene and cell therapy, scientists have shown that these cells can cross the blood-brain barrier and release a protective protein from the motor neurons of the spinal cord. This could potentially prevent its worsening and paralysis of muscle functions that characterize this neurodegenerative disease.

Stem cells are a powerful method for Transport of important proteins in the brain or spinal cord which otherwise could not cross the blood-brain barrier, explains Clive Svendsen, MD, director of medicine and biomedical sciences at the Cedars-Sinai Institute for Regenerative Medicine and senior author of the current study.

We have been able to show that these laboratory-modified stem cells can be safely transplanted into the spinal cord. And, after a one-time treatment, these cells survive and Protein production for more than three years What we know protects neurons that die as ALS progresses, Svendson continues.

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These cells were intended maintain leg function In patients, one of the first manifestations of paralysis is associated with amyotrophic lateral sclerosis. Subsequently, the disease automatically leads to the inability to walk, speak, and finally breathe. The trial involved 18 volunteers, and none of them experienced serious side effects.

The modified cells in Svendsons lab were designed to produce GDNF. a protein called, stands for glial cell line-derived neurotrophic factor. These glial cells are affected by ALS and stop protecting the neurons responsible for passing the nerve signals that enable muscle function and movement, causing them to deteriorate.

GDNF alone cannot cross the blood-brain barrier But stem cell transplantation is a method that manages to move the protein to where it should be, explains Dr. Pablo Avalos, co-author of this work. In this first therapeutic approach, the technique was to demonstrate that that it did not cause adverse in patients.

ALS often results in a loss of strength in both legs simultaneously, so researchers applied therapy. only one of them Through the half of the cord that controls it, so that the results can be compared with the other untreated leg. After transplantation, patients were followed for one year. During this period, it was found that the implant did not negatively affect the muscle strength of the operated leg.

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One of the minor side effects, however, manifested itself when the transplanted cells went to higher levels of the marrow, affecting sensory area and causing instances of pain. there were also cases of benign growth, As Svendson points out, these phenomena should be studied in future trials with a deeper focus and a different surgical approach.

The next trial, he explains, will enroll more patients with earlier ALS cases, to verify the long-term effects. This time they will focus on the lower part of the spine. In addition, these cells will be tested Direct implants in the cerebral motor cortex In hopes of better preserving the neurons responsible for the use of the hand.

We are very grateful to the study participants, Svendsson says. ALS is a very difficult disease to treat, and this research a new Hope To get closer to a way to delay the progression of this disease through therapy.

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New Hope Against ALS: They Manage To Transplant Stem Cells That Could Prevent Paralysis - Nation World News