ALS Fundraiser

Hope Gala Raises $1 Million

Team Chris Combs hosted their second annual Hope Gala to Defeat ALS on September 29th at the Umstead Hotel in Raleigh, NC. The event raised over $1 million for the second year in a row for Project ALS research, attracting almost 400 guests and featuring a live auction and raffle.

Chris Combs, husband, father of three, and former pitcher for the North Carolina State Wolfpack and Pittsburgh Pirates, was diagnosed with ALS in 2016. Since then, Chris, his wife Gena and their entire community have worked tirelessly to fundraise and collaborate on key Project ALS research initiatives. Thank you to the incredible Chris and Gena Combs.

Watch a beautiful video of the Combs family and the Hope Gala:

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Project ALS Gala photos

Kleiner Family Honored at Project ALS Gala

Carol Kleiner, wife, mother of three, rower, and resident of Blue Bell, PA, was honored at the nineteenth annual Project ALS Gala: Tomorrow is Tonight, on October 25th, 2017 at Cipriani Wall Street in Manhattan. The event was attended by over 400 guests and raised over $1 million for ALS research.

“Carol Kleiner, along with her husband Bob, and children Meredith, Jennifer, and Andrew, are an inspiration. Their work ethic, bravery and generosity have pushed Project ALS research to new heights. They have united their entire community in the fight against ALS. We felt their family was more than deserving of this honor” said Meredith Estess, Project ALS president.

Carol was diagnosed with ALS in September 2013 and has since raised over $2 million for Project ALS through multiple fundraising events, including Carol’s Crew, an initiative made up of Carol’s rowing friends, and A Date With a Plate, a cocktail party debuting intricately decorated table settings by top designers.

Carol and her family created the Kleiner Family Initiative at Project ALS which has funded an ALS pre-clinical core at Columbia University, the first dedicated effort among academic research centers to rapidly and thoroughly test promising drugs before they reach ALS human clinical trial. The core will ultimately provide doctors with more effective therapeutic options for people with ALS.

Co-directors of the pre-clinical core are Dr. Serge Przeborski, a leading neurologist and expert in ALS and Parkinson’s pathology, and Dr. Hynek Wichterle, a pioneer in stem cell biology and ALS. Currently, the pre-clinical core is testing three promising compounds: alsterpaullone, AR-12 (Arno Therapeutics), and AMX0035 (Amylyx). Drs. Przedbosrski and Wichterle and their team have assembled a predictive panel of laboratory tests to measure for such outcomes as optimal dose and delivery, brain penetrance, and, ultimately, efficacy.

“Project ALS has their thumbs on the pulse of current research. I have never seen an organization bring nascent ideas to clinical trial so quickly. It has not been hard for Carol and me to conclude that Project ALS is worthy of our commitment” said Bob Kleiner.

The Kleiners came to Project ALS through friends Cori Miller and David Jaffe. Jaffe is on the Board of Directors of Project ALS. A short documentary video of the Kleiner family was featured at the Project ALS Gala last week and Bob Kleiner spoke to the audience about Carol’s experience with ALS and Project ALS.

Watch a video of the Kleiner family:

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ALS disease under microscope

Study in Mice Reveals that Body’s Own Defense Against ALS Actually Drives Disease Progression at Later Stages.

Breaking news from Project ALS Autophagy Team: Study in mice reveals that the body’s own defense against ALS actually drives disease progression at later stages.

Findings underscore complexity of deadly neurological disease; lay foundation for therapies that could eventually prevent onset of ALS

Read press release

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Project ALS and Amylyx Enter Collaboration to Test AMX0035

New York, NY and Cambridge, MA (September 7, 2017) – Project ALS and Amylyx Pharmaceuticals today announced a collaboration to undertake pre-clinical studies with Amylyx’s oral compound AMX0035 to advance the understanding of the compound’s neurobiological effects. The studies to be conducted at the Project ALS Pre-Clinical Core at Columbia University’s Motor Neuron Center will complement the company’s recently initiated Phase 2 clinical program of AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS).

The Project ALS Pre-Clinical Core at the Columbia University’s Motor Neuron Center has established a unique integrated and standardized platform for the testing and validation of new therapeutic strategies in recognized experimental models of ALS and for biomarker discovery. The Core, developed in collaboration with Project ALS, will accelerate the translation of new promising therapies to patients by facilitating speedy testing of new therapeutic leads discovered by laboratories studying motor neuron biology, genetics and genomics.

“The collaboration will bring together a promising therapeutic candidate for a devastating disease with leading edge, fundamental neuroscientific research at Columbia,” said Valerie Estess, director of research for Project ALS. “The studies in this collaboration will provide greater insight into the neurobiological effects of AMX0035, and hopefully optimize its beneficial effects.”

The collaboration is an outgrowth of previous studies by The Project ALS Pre-Clinical Core at Columbia University of tauroursodeoxycholic acid (TUDCA), one of the components of AMX0035. “We look forward to evaluating AMX0035 in models of ALS. It is exciting that TUDCA has independently shown promise in both our labs and in studies conducted by the company,” said Drs. Hynek Wichterle and Serge Przedborski, co-directors of The Project ALS Pre-Clinical Core and tenure faculty in the Departments of Pathology and Neurology at Columbia University.

AMX0035 is a combination of two drugs, sodium phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA). Previous studies of PB and TUDCA as individual agents demonstrated efficacy in cellular and animal models of ALS.  AMX0035 has been shown to synergistically prevent nerve cell death and neurotoxic inflammation, hallmarks of ALS, in preclinical models. In addition, PB and TUDCA have been individually tested in ALS clinical trials and each demonstrated safety, tolerability, and preliminary signs of efficacy.  AMX0035 recently entered Phase 2 clinical development to evaluate its safety and efficacy in ALS. The trial, called CENTAUR, is a 24-week, randomized, double-blind, placebo-controlled study in 132 participants with ALS. More information on the CENTAUR trial can be found at www.clinicaltrials.gov, NCT03127514 and at www.Amylyx.com/Trials.

“Amylyx is very excited to partner with Project ALS and the researchers at Columbia’s Motor Neuron Center to advance these experiments. We hope these studies will provide valuable insights into both AMX0035 and ALS biology that will ultimately improve the lives of patients with ALS,” said Kent Leslie, Chief Scientific Officer of Amylyx.

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