ALS News Today has published daily coverage of ALS-related advocacy events, clinical trials, and research throughout the year. As 2015 finally winds down, ALS News Today has compiled the top 10 ALS news stories of 2015, ranked according to the number of views each news story received.
Researchers have found the mechanism behind the degeneration of upper motor neurons in the brain, which current evidence has strongly linked to disease progression in patients suffering from amyotrophic lateral sclerosis (ALS). The findings, which were published in the journal Cerebral Cortex under the title “Corticospinal Motor Neurons Are Susceptible to Increased ER Stress and Display Profound Degeneration in the Absence of UCHL1 Function,” could lead researchers to better understand the cause of the disease, as well as develop novel therapies for treating it.
#9 — “Q Therapeutics Announces FDA Clearance of Its Stem Cell Therapy Q-Cells for Human Trials in ALS”
Q Therapeutics, Inc. announced its Q-Cells product has received Investigational New Drug (IND) clearance by the U.S. Food and Drug Administration (FDA) to start human Phase 1/2a clinical trials to evaluate the safety and tolerability of Q-Cells in ALS patients. While the experimental therapy is only in its beginning stages of clinical development, the ALS community is following the progression of this novel stem cell treatment carefully.
A study led by researchers at The Center of Free Radical and Biomedical Research at Universidad de la Republica in Uruguay revealed that antioxidant therapies like MitoQare may offer a promising treatment strategy in ALS. The study was published in the journal Free Radical Biology and Medicine and is entitled “Neuroprotective effects of the mitochondria-targeted antioxidant MitoQ in a model of inherited amyotrophic lateral sclerosis.” Remarkably, the animals under MitoQ treatment exhibited an improved muscular strength and a delay in the decline of the mitochondrial function in both their spinal cord and quadricep muscles. Markers of oxidative stress and pathological disease signs were noticeably reduced in MitoQ-treated mice. The team also found that MitoQ treatment significantly prolonged the life span of ALS mouse models.
Genervon Biopharmaceuticals announced that one if its lead products, GM604, was able to regulate the levels of a protein called TDP-43 and slow disease progression in ALS patients. In a recent trial conducted by Genervon, several groups of ALS patients were treated with GM604 and the results demonstrate that TDP-43 levels decreased by 63% in advanced patients treated for 12 weeks and increased by 6% in the placebo group. Furthermore, it was found that administering GM604 for 12 weeks significantly decreased the concentration of TDP-43 when compared to placebo.
The U.S. Food and Drug Administration (FDA) issued a statement calling on pharmaceutical firm Genervon to release all data pertaining to a recent small study involving the company’s ALS drug candidate GM604. In its statement, the FDA affirmed that it recognizes the critical unmet medical need for new, effective treatments for amyotrophic lateral sclerosis (ALS), and its commitment to work with drug companies and the ALS community to facilitate development and approval of drugs designed to treat this devastating disease.
Kadimastem, an Israeli biotechnology company focused on the industrial development and commercialization of human pluripotent stem cell (hPSC)-based therapeutic solutions for neurodegenerative diseases, announced its plans to begin human trials using its stem-cell technology to treat the ALS in the second half of 2016.
A report in September indicating that Genervon’s GM604 may slow Disease Progression in ALS Patients was propelled by earlier news in June that Genervon had filed a patent application based on data from the Phase 2A clinical trial (GALS001 study, NCT01854294) assessing the company’s product GM604 as a therapy for ALS.
Johns Hopkins University researchers presented their latest results revealing increased levels of endothelin 1 (ET-1) in astrocytes in the brain of ALS patients, and that increased levels of ET-B binding protein (the protein that binds to the ET-1 receptor) are associated with the disease.
#2 — “BrainStorm Reports Positive Phase 2a Clinical Trial Results for NurOwn in ALS Treatment at a Scientific Meeting”
BrainStorm Cell Therapeutics Inc. has presented promising results from a Phase 2a clinical trial of its product NurOwn for ALS in a research poster entitled “Autologous Transplantation of Mesenchymal Stem Cells Secreting Neurotrophic Factors (NurOwn™) in ALS: Results of a Phase 2 Clinical Trial.”
Genervon reported improvements in a 46-year-old-man with advanced ALS that was taking the Company’s drug candidate GM604. The patient in the study showed increased speech, swallowing and suction capabilities after a 12-week treatment. The drug was tested under “compassionate use,” one of the FDA’s strategies to allow new drugs to be used in life-threatening disease conditions.
Be sure to tune in to ALS News Today daily throughout 2016 and follow the latest news updates regarding breakthrough ALS therapy development and research.