Disease Focus: Amyotrophic Lateral Sclerosis
CNS10-NPC-GDNF delivered into the motor cortex for the treatment of ALS
Therapeutic Candidate or Device CNS10-NPC-GDNF – a neural progenitor cell secreting GDNF Indication Amyotrophic Lateral Sclerosis (ALS) Therapeutic Mechanism This therapy will replace damaged astrocytes. The new astrocytes will release paracrine factors. As the cells have been modified to release GDNF they will also provide this neuroprotective factor to dying motor neurons in the motor […]
A Phase 3, Randomized, Placebo-controlled Multicenter Study to Evaluate Efficacy & Safety of Repeated Administrations of NurOwn® in Patients with ALS
Therapeutic Candidate or Device A cell therapy that delivers high levels of neurotrophic factors to the CNS Indication Amyotrophic lateral sclerosis (ALS) or Lou Gehrig Disease Therapeutic Mechanism The Cell therapy is aimed at providing high levels of neurotrophic factors directly to the CNS, to support the dying neurons Unmet Medical Need Amyotrophic lateral sclerosis […]
Human Neural Progenitors Secreting Glial Cell Line-Derived Neurotrophic Factor (CNS10-NPC-GDNF) for the Treatment of Amyotrophic Lateral Sclerosis
Therapeutic Candidate or Device CNS10-NPC-GDNF – a neural progenitor cell secreting GDNF Indication ALS Therapeutic Mechanism This therapy will replace damaged astrocytes. The new astrocytes will release paracrine factors. As the cells have been modified to release GDNF they will also provide this factor to dying motor neurons. Unmet Medical Need There is no treatment […]
Manufacturing of AS-202, an Antisense oligonucleotides for a Phase 1/2 Clinical Trial for Amyotrophic Lateral Sclerosis
Therapeutic Candidate or Device AS-202, an antisense oligonucleotide Indication Amyotrophic Lateral Sclerosis Therapeutic Mechanism AS-202 targets PIKFYVE, a lipid kinase. By inhibiting PIKFYVE activity, it induces exosomal secretion, which robustly clears misfolded proteins including C9ORF72 dipeptide repeat proteins (DPRs), TDP-43 and tau from neurons. Unmet Medical Need To date, therapeutic options for ALS have been […]
Facebook Live: Stem Cells and ALS
This Facebook Live event, “Ask the Stem Cell Team about ALS”, aired on July 31st, 2018 and featured a conversation between Drs. Clive Svendsen, Robert Baloh from Cedars-Sinai, Dr. Ralph Kern, the Chief Operating Officer and Chief Medical Officer of Brainstorm Therapeutics, and CIRM Senior Science Officer Dr. Lila Collins. Make sure to “like” CIRM’s […]
Stem Cells in Your Face: Treating ALS with a Disease in a Dish
To a stem cell scientist, “Disease in a Dish” describes a powerful way to study human disease in the lab using a Nobel Prize winning technique. But to a non-scientist it sounds more like a scene from some disgusting sci-fi horror cooking show. Our latest video takes a lighthearted approach to help clear up any […]
Google Hangout: Progress Toward Stem Cell Therapies for ALS
On July 3rd, CIRM hosted a Google Hangout to discuss progress toward stem cell therapies for Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s Disease. Dr. Clive Svendsen, a CIRM grantee and director of the Cedar Sinai Regenerative Medicine Institute, joined the hangout to describe his efforts to bring a stem cell based ALS […]
Webinar: Injecting Neural Stem Cells into ALS Patients: Results of a Phase 1 Trial | Jonathan Glass
Title: Lumbar Intraspinal Injection of Neural Stem Cells in Patients with ALS: Results of a Phase I Trial in 12 Patients Speaker: Jonathan D. Glass, M.D. Professor, Neurology and Pathology, Emory University School of Medicine Presented April 15, 2013 This presentation was part of the webinar event, “Clinical Trials: Moving Stem Cell based Therapies to […]
$18 Million ALS Research Grant for Stem Cell Therapy Applauded by ALS Advocates
On July 26th 2012, the governing board of California’s Stem Cell Agency approved an $18 million Disease Team research grant that aims to bring a stem cell-based ALS treatment to clinical trials. The project team is led by Clive Svendsen, PhD, director of the Cedars-Sinai Regenerative Medicine Institute and chair of the California ALS Research […]
Lou Gehrig’s Disease (ALS): Progress and Promise in Stem Cell Research
CIRM has funded the ALS Disease Team led by scientists at who aim to bring a human embryonic stem cell based ALS therapy to clinical trials within four years. Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s Disease, is a progressive motor neuron disorder. Most people with ALS die within 3 to 5 years […]