Amyotrophic Lateral Sclerosis (ALS) Fact Sheet

CIRM funds many projects seeking to better understand ALS and to translate those discoveries into new therapies.

Description

About 6,000 people are diagnosed with ALS (also known as Lou Gehrig’s disease) each year in the U.S., and the average survival time is two to five years. The disease results when the cells in the brain or spinal cord that instruct muscles to move—called motor neurons—die off. People with the disease lose the ability to move their muscles and, over time, the muscles atrophy and people become paralyzed and eventually die. There is no effective therapy for the disease.

California’s stem cell agency has funded several research projects that could help people with ALS (the full list of CIRM awards in this disease is below). Some of those projects are very basic—researchers are trying to understand the origin of the disease and what causes the motor neurons to die. These are the kinds of questions researchers need to understand if they are going to develop the most effective therapies.

With CIRM funding, researchers have made progress understanding which cells are responsible for damaging the motor neurons. It turns out that the cells surrounding those neurons—called astrocytes—are secreting a chemical that damages the neurons. They’ve also learned how to take certain kinds of stem cells and turn them into motor neurons and astrocytes and this might help us better understand the relationship of these cells and even one day prove useful in developing new ways to treat people with ALS.

We also fund projects that are in the later stages of research leading up to and in some cases including clinical trials. These projects involve teams of researchers who carry out the experiments that are required before the U.S. Food and Drug Administration will allow the potential therapy to be tested in people. Recently, CIRM has funded research into ALS that has advanced into clinical trials. You can read more about these trials below.

Clinical Stage Programs

Cedars-Sinai Medical Center

This team of researchers plans to protect surviving neurons in people diagnosed with ALS from further degeneration. They will implant middle-man “progenitor” cells made by maturing stem cells from fetal tissue down a path destined to become astrocytes, the brain cells that protect nerves and that become defective in ALS. Those cells will be boosted with genes for a growth factor that when the cells release it after transplantation, will have an added protective effect on nerves. This approach recently received approval to treat ALS patients in a CIRM-funded clinical trial (read here). A feature story on this work appeared in The Stem Cellar blog in 2017.

Brainstorm Cell Therapeutics

BrainStorm is using mesenchymal stem cells that are taken from the patient’s own bone marrow to treat patients with ALS. These stem cells are then modified to boost their production of neurotrophic factors, which are known to help support and protect neurons, the cells destroyed by the disease. The CIRM funding will enable the company to test this therapy, called NurOwn, in a Phase 3 trial involving around 200 patients.

CIRM Grants Targeting ALS

Researcher NameInstitutionGrant TitleGrant TypeAward Amount
Albert La Spada Ph.D.University of California, IrvineRole of ataxin-3 polyadenylation site selection in ALS neuron toxicity and disease pathogenesisFoundation – Discovery Stage Research Projects$1,514,416
Julia KayeGladstone Institutes, J. DavidDeveloping a Human Model of Sporadic ALS Using Machine Learning and Robotic MicroscopyFoundation – Discovery Stage Research Projects$1,406,622
Dr. Ritchie HoCedars-Sinai Medical CenterC9orf72 repeat expansion-tuned allelic suppression by CRISPRi as an ALS therapyQuest – Discovery Stage Research Projects$2,274,768
Dr. Justin K IchidaUniversity of Southern CaliforniaDevelopment of a VAV2 antisense oligonucleotide (ASO) treatment for ALSQuest – Discovery Stage Research Projects$2,072,560
Ziwei HuangUniversity of California, San DiegoDevelopment of a new therapeutic for directing target specific stem cell migration and treatmentQuest – Discovery Stage Research Projects$1,129,512
Dr. Justin K IchidaUniversity of Southern CaliforniaDevelopment of a SYF2 antisense oligonucleotide (ASO) treatment for ALSQuest – Discovery Stage Research Projects$222,300
Dr. Lawrence S. B. GoldsteinUniversity of California, San DiegoHuman Embryonic Stem Cell-Derived Neural Stem Cell Transplants in Amyotrophic Lateral SclerosisTherapeutic Translational Research Projects$1,790,000
Dr. Justin K IchidaUniversity of Southern CaliforniaThe 7th Annual California ALS research network and PAC10 meetingConference II$10,830
Prof. John M. RavitsUniversity of California, San DiegoCalifornia ALS Research Summit 2016Conference II$11,400
Clive SvendsenCedars-Sinai Medical CenterCNS10-NPC-GDNF delivered into the motor cortex for the treatment of ALSClinical Trial Stage Projects$11,990,372
Dr. Ralph KernBrainStorm Cell TherapeuticsA Phase 3, Randomized, Placebo-controlled Multicenter Study to Evaluate Efficacy & Safety of Repeated Administrations of NurOwn® in Patients with ALSClinical Trial Stage Projects$15,912,390
Professor Clive Niels SvendsenCedars-Sinai Medical CenterHuman Neural Progenitors Secreting Glial Cell Line-Derived Neurotrophic Factor (CNS10-NPC-GDNF) for the Treatment of Amyotrophic Lateral SclerosisClinical Trial Stage Projects$6,154,067
Mr. Samuel V. Alworth MS, MBAAcuraStem IncorporatedManufacturing of AS-202, an Antisense oligonucleotides for a Phase 1/2 Clinical Trial for Amyotrophic Lateral SclerosisLate Stage Preclinical Projects$0
Leif HavtonUniversity of California, Los AngelesDevelopment of a Relevant Pre-Clinical Animal Model as a Tool to Evaluate Human Stem Cell-Derived Replacement Therapies for Motor Neuron Injuries and Degenerative DiseasesTools and Technologies III$1,308,711
Prof. John M. RavitsUniversity of California, San DiegoCell Therapy for amyotrophic Lateral Sclerosis (ALS) -Testing the Limits: What should we use as preclinical standards of clinical trials?Conference$7,193
Professor Clive Niels SvendsenCedars-Sinai Medical CenterCalifornia ALS Summit 2012Conference$6,825
Dr. Martina Wiedau-PazosUniversity of California, Los AngelesCalifornia ALS Summit 2011Conference$13,300
Dr. Steve M. FinkbeinerGladstone Institutes, J. DavidTHE 5TH ANNUAL CALIFORNIA ALS PAC10 AND RESEARCH NETWORK MEETINGConference$9,529
Dr. Lawrence S. B. GoldsteinUniversity of California, San DiegoStem Cell-Derived Astrocyte Precursor Transplants in Amyotrophic Lateral SclerosisEarly Translational from Disease Team Conversion$4,139,754
Dr. Steve M. FinkbeinerGladstone Institutes, J. DavidDevelopment of Novel Autophagy Inducers to Block the Progression of and Treat Amyotrophic Lateral Sclerosis (ALS) and Other Neurodegenerative DiseasesEarly Translational IV$2,049,053
Gene Wei-Ming YeoUniversity of California, San DiegoStem cell models to analyze the role of mutated C9ORF72 in neurodegenerationBasic Biology IV$1,260,360
Professor Clive Niels SvendsenCedars-Sinai Medical CenterProgenitor Cells Secreting GDNF for the Treatment of ALSDisease Team Therapy Development – Research$16,168,464
Gene Wei-Ming YeoUniversity of California, San DiegoMolecules to Correct Aberrant RNA Signature in Human Diseased NeuronsEarly Translational III$1,532,323
Professor Clive Niels SvendsenCedars-Sinai Medical CenterStem Cells Secreting GDNF for the Treatment of ALSDisease Team Therapy Planning I$63,487
Gene Wei-Ming YeoUniversity of California, San DiegoNeural and general splicing factors control self-renewal, neural survival and differentiationBasic Biology III$1,287,619
Dr. Eric T. AhrensUniversity of California, San DiegoMolecular Imaging for Stem Cell Science and Clinical ApplicationResearch Leadership$5,680,474
Dr. Bin ChenUniversity of California, Santa CruzMolecular mechanisms of neural stem cell differentiation in the developing brainNew Faculty I$2,147,592
Dr. Ying LiuUniversity of California, San DiegoGeneration of disease models for neurodegenerative disorders in hESCs by gene targetingTools and Technologies I$709,829
Dr. Jeremy ReiterUniversity of California, San FranciscoHigh throughput modeling of human neurodegenerative diseases in embryonic stem cellsNew Faculty II$2,259,092
Dr. Jerome A. Zack Ph.D.University of California, Los AngelesGeneration of clinical grade human iPS cellsNew Cell Lines$1,341,000
Dr. Fred H GageSalk Institute for Biological StudiesDevelopment of Induced Pluripotent Stem Cells for Modeling Human DiseaseNew Cell Lines$1,737,720
Dr Hans S KeirsteadUniversity of California, IrvinehESC-Derived Motor Neurons For the Treatment of Cervical Spinal Cord InjuryComprehensive Grant$2,158,445
Dr. Fred H GageSalk Institute for Biological StudiesMolecular and Cellular Transitions from ES Cells to Mature Functioning Human NeuronsComprehensive Grant$2,749,293
Dr. Benhai Zheng Dr.University of California, San DiegoGenetic manipulation of human embryonic stem cells and its application in studying CNS development and repairSEED Grant$600,441
Prof. Samuel Lawrence PfaffSalk Institute for Biological StudiesGene regulatory mechanisms that control spinal neuron differentiation from hES cells.SEED Grant$704,543
Dr. Bin ChenUniversity of California, Santa CruzIn vitro differentiation of hESCs into corticospinal motor neuronsSEED Grant$465,624
Dr. Lawrence S. B. GoldsteinUniversity of California, San DiegoStem Cell-Derived Astrocyte Precursor Transplants in Amyotrophic Lateral SclerosisDisease Team Research I$5,694,308
Dr. Bennett G Novitch Ph.D.University of California, Los AngelesMolecular Characterization of hESC and hIPSC-Derived Spinal Motor NeuronsBasic Biology I$1,228,278
Total:
$99,812,494.55

CIRM ALS Videos

Facebook Live: Stem Cells and ALS

Stem Cells in Your Face: Treating ALS with a Disease in a Dish

Google Hangout: Progress Toward Stem Cell Therapies for ALS

Webinar: Injecting Neural Stem Cells into ALS Patients: Results of a Phase 1 Trial | Jonathan Glass

$18 Million ALS Research Grant for Stem Cell Therapy Applauded by ALS Advocates

Lou Gehrig’s Disease (ALS): Progress and Promise in Stem Cell Research

Spotlight on Disease Team Awards – ALS: Introduction

Spotlight on Disease Team Awards – ALS: Larry Goldstein

Spotlight on Disease Team Awards – ALS: Don Cleveland

Spotlight on Disease Team Awards – ALS: Mark Bonyhadi

Spotlight on Disease Team Awards – ALS: Martin Marsala

Spotlight on Disease Team Awards – ALS: Lucie Bruijn

Spotlight on Disease Team Awards – ALS: Dan Desmond

Spotlight on Disease Team Awards – ALS: Wrap Up

Stem Cell Research Today: Larry Goldstein – CIRM Science Writer’s Seminar

News and Information

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