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People Suffering From ALS Or Lou Gehrig’s Disease Soon May Get A New Therapy

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Cedars-Sinai researchers have developed an investigational therapy that uses support cells and a protective protein that can cross the blood-brain barrier.

This combination of stem cell and gene treatment has the ability to protect damaged motor neurons in the spinal cord of individuals with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease.

The Cedars-Sinai researchers demonstrated that the delivery of this combined medication is safe in humans in the first experiment of its kind.

The findings were published in the peer-reviewed journal Nature Medicine today.

“Using stem cells,” according to senior and corresponding author Clive Svendsen, “is a powerful way to deliver important proteins to the brain or spinal cord that can’t otherwise get through the blood-brain barrier.” 

In the study, they “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.”

The modified cells, which are aimed at retaining limb function in ALS patients, could be a strong therapeutic alternative for this condition, which causes progressive muscle paralysis, robbing people of their abilities to move, speak, and breathe.

According to the statistics, none of the 18 patients who received the therapy—which was created by Cedars-Sinai researchers—had any severe negative effects following the transplant.

The research utilized stem cells created in Svendsen’s lab to make glial cell line-derived neurotrophic factor, a protein (GDNF). This protein can help motor neurons survive, which are the cells that send messages from the brain or spinal cord to muscles to allow them to move.

In patients with amyotrophic lateral sclerosis (ALS), dysfunctional glial cells can become less supportive of motor neurons, leading to progressive motor neuron degeneration and paralysis.

In the central nervous system, where the damaged motor neurons are located, the engineered protein-producing stem cells can be implanted. There, these stem cells can differentiate into new supportive glial cells and release the protective protein GDNF, both of which contribute to the survival of the motor neurons.

“GDNF on its own can’t get through the blood-brain barrier,” as explained by co-lead author Pablo Avalos, “so transplanting stem cells releasing GDNF is a new method to help get the protein to where it needs to go to help protect the motor neurons.” 

As “they are engineered to release GDNF,” the co-author added, “we get a ‘double whammy’ approach where both the new cells and the protein could help dying motor neurons survive better in this disease.”

The trial

The trial’s main objective was to make sure that delivering the GDNF-releasing cells to the spinal cord didn’t cause any safety problems or impair limb function.

Researchers only implanted the stem cell-gene product into one side of the spinal cord since ALS patients often lose strength in both legs at a comparable rate. This allowed them to compare the therapeutic effect on the treated limb to the untreated leg.

To safely administer the stem cell-gene product, known as CNS10-NPC-GDNF, to patients’ spinal cords, the scientists created a unique injectable device.

Patients were monitored for a year after the transplant so the researchers could compare the strength of their treated and untreated legs. The purpose of the trial was to see if it was safe, which was proven by the fact that the cell transplant had no negative effect on muscle strength in the treated leg compared to the untreated leg.

“We’re excited that we proved safety of this approach, but we need more patients to really evaluate efficacy, which is part of the next phase of the study,” added Johnson, who is also the vice chair of Neurosurgery at Cedars-Sinai. “Proving that we have cells that can survive a long time and are safe in the patient is a key part in moving forward with this experimental treatment.”

Even though there were no major side effects, the team found that in some patients, the cells went too high in the spinal cord and ended up in pain-sensitive areas. In several cases, they also observed benign growths connected to cell transplantation. Deeper targeting and a different surgical technique will be used to solve this in future experiments, according to Svendsen.

Researchers want to begin a new trial with more participants very soon. To maximize the likelihood of observing the impacts of the cells on ALS progression, they will target lower in the spinal cord and enrol individuals at an earlier stage of the illness.

Svendsen expressed his gratitude to all of the study participants. This research provides us optimism that we are coming closer to discovering strategies to slow down this disease, even though ALS is a very difficult condition to treat.

The Cedars-Sinai team is also utilizing GDNF-secreting stem cells in a different ALS clinical study by introducing the cells into the motor cortex, an area of the brain that regulates the start of hand movement. In the new study, they have just finished treating the first 16 people. The main goal of the study is to show that it is safe, but they are also looking to see if there are any long-term effects on how people use their hands.

Image Credit: Getty

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