ALS research that NYSCF has supported for 8 years is now progressing to clinical trials for patients with this devastating disease. NYSCF began supporting Dr. Kevin Eggan's laboratory in 2007 when he was unable to obtain funds to make ALS diseased cells in a dish. NYSCF has been supporting his laboratory since then and the continued success of this research has culminated with two related papers that were published today on a potential ALS treatment that will now go to clinical trials.
The two papers, published today in Cell Stem Cell and Cell Reports, outline a discovery in which an epilepsy medicine may be useful in the treatment of ALS or Lou Gehrig's disease. First author on both papers was Evangelos Kiskinis, PhD, a NYSCF - Druckenmiller Fellow alumnus, working in the laboratory of Dr. Eggan at the Harvard Stem Cell Institute.
While a great deal more research needs to be done before determining the drug's sucess, the researchers are currently designing an initial clinical trial to test the safety of the drug in ALS patients with clinicians at Massachusetts General Hospital.
NYSCF – Druckenmiller Fellow Dr. Wee-Wei Tee of New York University published a paper earlier this month in Cell on the key role a specific gene plays in priming the developmental genes of embryonic mouse cells. Gene priming is a nuanced and little understood process leading to gene activation and deactivation leading to pluripotentcy, or the ability for a cell to become any type of cell in the body.
NYSCF – Robertson Neuroscience Investigator Dr. Ed Boyden of MIT published his latest research in Nature Methods describing a new technique to study brain function by controlling two separate populations of neurons with different colors of light. Dr. Boyden is the creator of the reserach field of 'optogenetics' which uses wavelengths of light to study brain and neuron activity.
Dr. Scott Noggle, Director of the NYSCF Laboratory and NYSCF – Charles Evans Senior Research Fellow for Alzheimer’s Disease, presented the latest in Alzheimer’s research at the Alzheimer’s Disease Resource Center’s (ADRC) 10th Annual Alzheimer's Disease Education Conference & Expo. Dr. Noggle gave a talk on the ability of stem cells to model the disease ‘in a dish’ and the implications of this technology on Alzheimer’s research and potential future treatments and cures. The event was held on March 6th in Melville, NY.
NYSCF - Druckenmiller Fellow Dilek Colak was the lead author on a study uncovering a genetic trigger for the most common form of intellectual disability and autism. Published in Science, the Weill Cornell study describes the identification of a mechanism that shuts off a gene associated with Fragile X syndrome. Additionally, the scientists showed that a drug blocking this silencing mechanism could prevent the disease from developing, suggesting a potential therapeutic treatment for all related Fragile X disorders including but not limited to mental retardation and multisystem failure.
NYSCF Senior Research Fellow Dieter Egli presented his research on a technique to prevent the transmission of mitochondrial diseases to the Food and Drug Administration (FDA) Advisory Committee meeting on Cellular, Tissue and Gene Therapies. The meeting, held in Gaithersburg, Maryland, aimed to give recommendations to the FDA on whether to approve human clinical trials for techniques to prevent this group of devastating, and often fatal, diseases and other infertility treatments.
Dr. Egli's research and discovery that transfering the nucleus of an egg from a woman with mitochondrial disease into a donor egg allows her to have her own genetic children without the threat of mitochondrial disease was recognized as a top scientific breakthrough in 2012.
Dr. Andrew Sproul, NYSCF Staff Scientst, presented the NYSCF Alzheimer's disease research team's latest research at the Alzheimer's Disease Resource Center's "What You Need to Know About Alzheimer's" event in East Hampton.
Dr. Sproul shared the NYSCF Alzheimer's team's recent discoveries and explained NYSCF's participation in large scale Alzheimer's initiatives through new Alzheimer's disease research consortia.
The event was held at the Jewish Center of the Hamptons in East Hampton on January 12th.
Dr. Gaby Maimon, a NYSCF – Robertson Neuroscience Investigator at The Rockefeller University, has been named a recipient of the Presidential Early Career Award for Scientists and Engineers. This award is the highest honor bestowed by the United States Government on science and engineering professionals in the early stages of their independent research careers.
Dr. Maimon focuses his research on the neural behavior in the fruit fly with implications on human cognitive function and learning.