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Cedars-Sinai researchers have developed an investigational therapy that uses Sertoli cells and a protective protein that can cross the blood-brain barrier
In the first trial of this type, the Cedars-Sinai team showed that the combination treatment is safe in humans
"Using stem cells is a powerful way to deliver important proteins to the brain or spinal cord that would otherwise not cross the blood-brain barrier," said senior corresponding author Clive Svendsen, Ph.
To preserve leg function in ALS patients, the engineered cells could be a powerful option for treating ALS
The study used stem cells originally engineered in Svendsen's lab to produce a protein called glial lineage-derived neurotrophic factor (GDNF)
"GDNF itself cannot cross the blood-brain barrier, so transplanting GDNF-releasing stem cells is a new way to help get the protein where it needs to go, helping to protect motor neurons," said co-lead author of the paper, Regenerative Medicine said Pablo Avalos, MD, associate director of translational medicine at the institute
Safety test
The primary goal of the trial was to ensure that delivering GDNF-releasing cells to the spinal cord would not have any safety concerns or negatively impact leg function
After the transplant, the team followed the patients for a year in order to measure the strength of the treated and untreated legs
The cells can survive long periods of time in patients and be safe, a key part of advancing this experimental treatment
The researchers expect to launch a new study with more patients soon
"We are very grateful to all who participated in this study," Svendsen said
The Cedars-Sinai team also used GDNF-secreting stem cells in another clinical trial for ALS, transplanting the cells into a specific brain region, called the motor cortex, that controls the initiation of hand movements
Transplantation of human neural progenitor cells secreting GDNF into the spinal cord of patients with ALS: a phase 1/2a trial
