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Combined Stem Cell and Gene Therapy Trial Safe for Humans

In the latest news on stem cell research, Cedars-Sinai researchers have developed an investigational therapy using support cells and a protective protein that can be delivered past the blood-brain barrier.

“Using stem cells is a powerful way to deliver important proteins to the brain or spinal cord that can’t otherwise get through the blood-brain barrier,” said Dr Clive Svendsen, PhD, professor of Biomedical Sciences and Medicine and executive director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute.

This combined stem cell and gene therapy can potentially protect diseased motor neurons in the spinal cord of patients with amyotrophic lateral sclerosis, a fatal neurological disorder known as ALS or Lou Gehrig’s disease

“We were able to show that the engineered stem cell product can be safely transplanted in the human spinal cord. And after a one-time treatment, these cells can survive and produce an important protein for over three years that is known to protect motor neurons that die in ALS.”

Aimed at preserving leg function in patients with ALS, the engineered cells are potentially a powerful therapeutic option for this disease that causes progressive muscle paralysis, robbing people of their ability to move, speak and breathe. 

None of the 18 patients treated with the therapy—developed by Cedars-Sinai scientists—had serious side effects after the transplantation, according to the data.

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