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| Motor neurons control our muscle movements. In ALS, these cells die. |
In 2012, CIRM’s Board awarded over $17 million for ALS research and clinical trials at Cedars-Sinai Medical Center.
Allied with the Department of Defense
The Department of Defense (DoD) added to that with a $2.5 million grant to the same Cedars-Sinai team for a similar amyotrophic lateral sclerosis (ALS) related research.
The military spends several million annually on ALS research because the disease affects an unusually high percentage of veterans for reasons that are not understood.
More on ALS
ALS, also known as Lou Gehrig’s disease, is a devastating condition that is usually fatal within four to five years. The disease develops when motor neurons—cells in the brain and spinal cord that control muscle movement—die off. Dr. Clive Svendsen, who leads the Cedars‑Sinai team, describes its impact:
“The disease usually begins in one limb, starting with weakness and progressing to paralysis, before spreading to other limbs and muscles throughout the body. The most common cause of death is respiratory failure when the diaphragm muscles stop working.”
The CIRM‑funded team led by Svendsen discovered that a protein called GDNF can protect motor neurons that have not yet been damaged by ALS. But delivering GDNF to the spinal cord is extremely difficult because it does not cross from the bloodstream into the spinal cord.
To solve this, Svendsen’s team engineered neural stem cells to produce GDNF. When injected into patients, these cells are expected to migrate to the sick motor neurons and deliver GDNF precisely where it is needed.
More on GDNF
The Department of Defense-funded project announced yesterday also relies on the protective effects of GDNF. As reported in a press release, which was picked up by Phys.Org, the team will deliver the protein into the leg and diaphragm muscles, where ALS often strikes. Former Svendsen lab member and current University of Wisconsin–Madison professor Dr. Masatoshi Suzuki explains the benefit of muscle delivery:
“It’s clear that GDNF has strong neuroprotective effects on motor neurons when delivered to the muscle. We think GDNF will help maintain these connections in patients and keep the motor neuron network functional.”
The DoD funding will support the animal studies needed to advance this strategy to clinical trials. Because clinical trials are unpredictable, Svendsen’s team is wise to pursue a two‑pronged approach to ALS. The press release also highlights CIRM’s Disease Team award, showing how our support helped attract additional funding. Hopefully, this momentum will lead to faster progress for people living with this devastating disease.
For more information about CIRM‑funded ALS research, visit our ALS fact sheet. You can also watch a video of a recent CIRM-hosted Google Hangout about stem cell therapies for ALS featuring Dr. Svendsen.
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