Disease Focus: Amyotrophic Lateral Sclerosis
Center of Excellence for Stem Cell Genomics – Stanford
The Center of Excellence in Stem Cell Genomics will bring together investigators from seven major California research institutions to bridge two fields – genomics and pluripotent stem cell research. The projects will combine the strengths of the center team members, each of whom is a leader in one or both fields. The program directors have […]
Stem Cell-Derived Astrocyte Precursor Transplants in Amyotrophic Lateral Sclerosis
Development of Novel Autophagy Inducers to Block the Progression of and Treat Amyotrophic Lateral Sclerosis (ALS) and Other Neurodegenerative Diseases
ALS is a progressive neurodegenerative disease that primarily affects motor neurons (MNs). It results in paralysis and loss of control of vital functions, such as breathing, leading to premature death. Life expectancy of ALS patients averages 2–5 years from diagnosis. About 5,600 people in the U.S. are diagnosed with ALS each year, and about 30,000 […]
Stem cell models to analyze the role of mutated C9ORF72 in neurodegeneration
Amyotrophic lateral sclerosis (ALS) is an idiopathic adult-onset degenerative disease characterized by progressive weakness from loss of upper and lower motor neurons. Onset is insidious, progression is essentially linear, and death occurs within 3-5 years in 90% of patients. In the US, 5,000 deaths occur per year and in the world, 100,000. In October, 2011, […]
Progenitor Cells Secreting GDNF for the Treatment of ALS
This project aims to use a powerful combined neural progenitor cell and growth factor approach to treat patients with amyotrophic lateral sclerosis (ALS or Lou Gehrig’s Disease). ALS is a devastating disease for which there is no treatment or cure. Progression from early muscle twitches to complete paralysis and death usually happens within 4 years. […]
Molecules to Correct Aberrant RNA Signature in Human Diseased Neurons
Approximately 5,600 people in the U.S. are diagnosed with ALS each year. The incidence of ALS is two per 100,000 people, and it is estimated that as many as 30,000 Americans may have the disease at any given time. There are no effective therapies of ALS to-date. Recent genetic discoveries have pinpointed mutations that lead […]
Stem Cells Secreting GDNF for the Treatment of ALS
This project aims to use a powerful combined stem cell and gene therapy approach to treat patients with amyotrophic lateral sclerosis (ALS or Lou Gehrig’s Disease). ALS is a devastating disease for which there is no treatment or cure. Progression from early muscle twitches to complete paralysis and death usually happens within 4 years. Every […]
CNS10-NPC-GDNF delivered into the motor cortex for the treatment of ALS
ALS is a neurodegenerative disease that results in the death of nerve cells in the brain and spinal cord, causing the muscles in the body to gradually weaken, leading to loss of limb function, difficulty breathing, paralysis, and eventually death. There are medications that can slow down the progression of ALS, but unfortunately there is […]
Progenitor Cells Secreting GDNF for the Treatment of ALS
ALS is a devastating neurodegenerative disease with no cure that specifically affect a patient’s motor neurons in the brain. A team at Cedars-Sinai is transplanting millions of genetically engineered stem cells into patients with ALS. When transplanted into the patient spinal cord, these cells become astrocytes, the support cells that keep nerve cells functioning. Due to […]
A Phase 3, Randomized, Placebo-controlled Multicenter Study to Evaluate Efficacy & Safety of Repeated Administrations of NurOwn® in Patients with ALS
BrainStorm Therapeutics is using mesenchymal stem cells that are taken from the patient’s own bone marrow. These stem cells are then modified in the lab to boost their production of neurotrophic factors, which are known to help support and protect neurons, the cells destroyed by ALS. The modified cells are then returned to the patients […]