A report in the journal JAMA Neurology revealed positive findings from the first Phase 1/2 clinical trial and the Phase 2 dose escalation study investigating BrainStorm’s NurOwn for the treatment of amyotrophic lateral sclerosis (ALS). NurOwn is a stem cell technology employing adult cells isolated from the disease-affected patient, developed…
Researchers at the National University of Singapore have suggested there is a fine balance between amyotrophic lateral sclerosis (ALS)-related protein TDP-43’s normal function and its potential to cause neurodegeneration. The degeneration seems to occur due to pathological factors that exaggerate the protein’s membrane association, resulting in the loss of its physiological functions as well as…
University of Bern researchers have discovered that the synaptic protein Homer-3 is linked to the death of Purkinje motor neurons in Spinocerebellar ataxia type 1 (SCA1), a motor neuron disease that, like amyotrophic lateral sclerosis (ALS), is characterized by extensive motor neuron loss. The study, titled “Impaired mTORC1-Dependent Expression of…
A new proof-of-concept study reports converting adult skin cells from ALS patients into muscle neurons without first transforming them into stem cells. The patient-derived muscle neurons were dysfunctional, but when treated with a small molecule, the team could restore neuronal function. The use of induced pluripotent stem cells (iPSCs)…
A research group — coordinated by Giuseppe Legname from The Laboratory of Prion Disease, SISSA, Trieste, Italy — has found a way to build artificial prions, assembling the proteins in a serial manner. The study was performed in collaboration with the Carlo Besta Neurological Institute in Milan and the results were published in the journal…
Researchers at the Salk Institute have discovered that a set of messenger RNAs, controlled by the microRNA miR-218, define a specific network of neuronal genes that are repressed to prevent neuromuscular damage and neurodegeneration. Loss of miR-218 was seen to lead to the onset of neurodegenerative diseases such as amyotrophic lateral…
The ALS Association has announced the Phi Delta Theta Fraternity is contributing to support the Milton Safenowitz Postdoctoral Fellow Program by awarding $100,000 over two years to Antonia Dominguez, Ph.D., a researcher focused on finding treatments for amyotrophic lateral sclerosis (ALS). The Milton Safenowitz Postdoctoral Fellowships were founded by the Safenowitz family…
Researchers at University Of North Carolina School Of Medicine are reporting the first evidence-based description of the neuronal protein clumps thought to be toxic to motor neurons and instigators of the neuronal degeneration seen in patients with amyotrophic lateral sclerosis (ALS). The laboratory discovery of the form of these unstable clumps, known as SOD1 proteins, and…
AB Science recently announced it has completed its targeted enrollment of 381 patients for an ongoing Phase 3 study evaluating masitinib in the treatment of amyotrophic lateral sclerosis (ALS). Masitinib is an orally administered tyrosine kinase inhibitor designed to target important immune system cells known as mast cells and macrophages. Its mechanism of action…
ISIS Pharmaceuticals, in collaboration with Biogen, has started a Phase 1/2 clinical study of ISIS-SOD1Rx (BIIB067), one of the drug candidates to come out of the strategic partnership between the two companies, in adult patients diagnosed with amyotrophic lateral sclerosis (ALS). ISIS-SOD1Rx’s mode of action relies on ISIS antisense technology that targets at…
