#STEM CELL TREATMENT FOR ALS TRIAL#
To test the safety and efficacy of this treatment, a multi-center, placebo-controlled, randomized, double-blind Phase 3 clinical trial was conducted. The factors produced by the cells have the potential to slow down or stabilize disease progression. After collecting them, the cells are grown under patented conditions to make the MSC produce neurotrophic factors (NF), then these cells are delivered by intrathecal and/or intramuscular administration. NurOwn technology uses a subtype of the patients’ bone marrow stem cells known as mesenchymal stem cells (MSC). There is no cure for this disease, with most patients dying of respiratory failure within 2-5 years of onset.
Riluzole, and Edaravone, are the only two FDA-approved drugs to manage ALS symptoms, but only Riluzole has shown to extend survival time. Around 90% of patients are diagnosed with sporadic ALS, and only an estimate of 10% of the patients have a hereditary form of the disease. The exact cause of the disease reminds unknown.
Every year, approximately 30,000 patients die of ALS.
It is characterized by progressive damage to the nervous system that causes muscle control loss, including muscles necessary for breathing. NurOwn failed to significantly improve the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R).ĪLS or Lou Gehrig’s is the most common disease that affects the motor neuron system. announced the results of their Phase 3 clinical trial testing the efficacy of their stem cell treatment, NurOwn (MSC-NTF cells), in Amyotrophic lateral sclerosis (ALS) patients. Randomized, double-blinded, and sham-controlled studies would be valuable, but ethical implications should be considered.On November 17 th, BrainStorm Cell Therapeutics Inc. However, the determination of whether stem cells offer a viable treatment strategy for ALS rests on well-designed and appropriately powered future clinical trials. Most current clinical trials are assessing safety as their primary objective. Determination of an optimal window of treatment will also aid endeavors to identify promising treatment options. Several stem cell sources, cell doses, and methods of delivery are presently being evaluated in early phase clinical trials. While stem cells are unable to directly replace diseased motor neurons, transplanted stem cells secrete neurotrophic factors and differentiate into supportive cells, such as astrocytes and microglia, generating a neuroprotective milieu that can slow degeneration of motor neurons.Įncouraging results in preclinical animal models advocated human clinical trials. The premise of stem cell therapy for ALS is based on improving the diseased microenvironment. Stem cell therapy is considered an attractive approach for ALS that addresses the complex disease pathogenesis through multiple potential mechanisms. There is no cure for ALS despite numerous clinical trials current therapies are palliative and only extend survival a few months. Several trials have used more invasive procedures, and ethical concerns of sham procedures on patients in the control arm and on their safety should be considered.
#STEM CELL TREATMENT FOR ALS WINDOWS#
It will be critical to ensure that powered, well-controlled trials are conducted, that optimal treatment windows are identified, and that the ideal cell type, cell dose, and delivery site and method are determined. Several early-stage clinical trials have been launched to assess the potential of stem cells for ALS treatment.Īreas covered: This review covers the key advances from early phase clinical trials of stem cell therapy for ALS and identifies promising avenues and key challenges.Įxpert opinion: Clinical trials in humans are still in the nascent stages of development. Stem cells may be a viable solution to sustain and nurture diseased motor neurons.
Therapeutics to slow, stop, and reverse ALS are needed. No currently available treatment either stops or reverses this disease. Introduction: Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease of cortical, brainstem, and spinal motor neurons it causes progressive muscle weakness and atrophy, respiratory failure, and death.
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