Researchers at Cedars-Sinai in Los Angeles, funded by The ALS Association with ALS Ice Bucket Challenge donations, are dedicated to finding unique avenues for treating ALS. Using animal models, they transplanted specially engineered neural cells into the motor cortex of the brain, the area responsible for muscle movement.
They found that the transplanted cells protected motor neurons (the cells that die in ALS), delayed disease progression, and extended survival in animal models.
The ALS Association is committed to helping improve clinical trial design, in order to increase trial efficiency that will more quickly lead to effective therapeutics.
We awarded Dr. David Ennist and colleagues at Origent Data Sciences, Inc. two grants to support research exploring how machine learning algorithms, a type of computational tool, can optimize clinical trial design. Dr. Ennist’s work, recently published in the journal Annals of Clinical Trial and Translational Neurology, looks closely at optimizing patient randomization into clinical trials.
The CENTAUR phase II clinical drug trial, sponsored by Amylyx Pharmaceuticals and funded by ALS Ice Bucket Challenge donations, began an open label extension, giving people with ALS who completed their trial period an opportunity to continue taking the drug, AMX0035.
We sat down with Dr. John Landers, professor of Neurology at the University of Massachusetts Medical School, to discuss the recent discovery of the newest ALS gene, KIF5A, made possible by The ALS Association funding through ALS Ice Bucket Challenge donations. Dr. Landers is the co-leader of the U.S. arm of Project MinE, which is the largest ALS whole genome sequencing effort of its kind. The discovery findings were published in the journal Neuron.
This massive collaborative effort between researchers from Project MinE, Genomic Translation for ALS Care, Answer ALS, the New York Genome Center (NYGC) ALS Consortium, the CReATe Consortium, the Target ALS Postmortem Tissue Core, the National Institutes of Health (NIH), and others supported or funded in some way by The ALS Association contributed to this gene discovery.
Researchers from collaborative initiatives funded by The ALS Association, with money raised through the ALS Ice Bucket Challenge, announced the discovery of a new ALS gene, KIF5A, which will help drive new discoveries and fuel the ALS treatment pipeline.
Researchers from organizations Project MinE, Genomic Translation for ALS Care, Answer ALS, the Target ALS Postmortem Tissue Core, the New York Genome Center (NYGC) ALS Consortium, the CReATe Consortium, the National Institutes of Health (NIH), and others came together to collaborate and share detailed genetic and clinical information needed to make this important discovery. The Ice Bucket Challenge enabled The ALS Association to support the above unique collaborative initiatives in precision medicine to accelerate the discovery of new treatments for ALS.
We recently sat down with Dr. Carlos Castañeda, assistant professor of biology and chemistry at Syracuse University. Thanks to funding from the ALS Ice Bucket Challenge, The ALS Association has funded Dr. Castañeda twice through our global research program, which supported this work.
Dr. Castañeda talked with us about the important findings in his recently published paper in Molecular Cell. His research explores cell degradation pathways with a focus on ubiquilin-2 (UBQLN2), a shuttle protein associated with ALS. Importantly, this work further links dysregulation of protein quality control pathways to ALS disease states.
Research funded with donations from the ALS Ice Bucket Challenge recently uncovered evidence that promoting an increase in a specific immune cell in the brain and spinal cord of a mouse with ALS was associated with increased motor function, pointing to a potential treatment in the future.
During ALS disease, motor neurons degenerate and eventually die, resulting in a loss of the connection to muscles, which in turn weaken and eventually paralyze. Several studies have shown that microglia, which are a type of immune cell in the brain and spinal cord, increase the severity of neurodegeneration in ALS. Others have shown that microglia can contribute to maintaining the health of neurons.
Drs. Virginia Lee, Krista Spiller, and colleagues from the Perelman School of Medicine at the University of Pennsylvania in Philadelphia set out to understand how microglia function in the brain and spinal cord to either protect against or contribute to neurodegeneration.
The ALS Association currently funds Dr. Spiller under our Investigator-Initiated Starter Grant program using donations from the ALS Ice Bucket Challenge. Their paper was published in the February 20 issue of Nature Neuroscience journal.
They found that microglia can play a protective role in the brain and spinal cord in a TDP-43 mouse model. This suggests that encouraging healthy microglia to proliferate in cells could translate into a potential ALS therapy in the future.