The ALS Association Golden West Chapter to Honor Three Scientists
for their “Commitment to a Cure” in the fight against ALS/Lou Gehrig’s Disease

May 29, 2014- Los Angeles, California

2014 Commitment to a Cure Award to three scientists – C. Frank Bennett, PhD, Don W. Cleveland, PhD, and Richard Smith, MD

The ALS Association Golden West Chapter cordially invites you to an evening of elegance at its Hollywood Red Carpet Gala, an event dedicated to conquering ALS. A Night at the Esseys will be held on Thursday, June 5, 2014 at the historic Hollywood Roosevelt Hotel, home to the first Academy Awards ceremony, and will feature a wine & champagne reception, live orchestra, gourmet dinner, and live and silent auctions. 

The Essey Awards are the Golden West Chapter’s highest honor, named for one of the Greater Bay Area Chapter’s founding members, Richard Essey. These awards are presented annually to esteemed members of the ALS community for their outstanding efforts in research, philanthropy and public awareness. Among this year’s honorees, the Golden West Chapter will present the 2014 Commitment to a Cure Award to three scientists – C. Frank Bennett, PhD, Isis Pharmaceuticals, Inc; Don W. Cleveland, PhD, Ludwig Institute for Cancer Research, University of California, San Diego (UCSD); and Richard Smith, MD, Center for Neurologic Study – who have relentlessly pursued research which may lead to a treatment for people with ALS and related diseases.  The team has collaborated over the last decade on an innovative idea, which was an elegantly simple approach to dealing with a very complicated problem. Their therapy, called antisense oligonucleotide therapy (ASO), aims to block the expression of mutant genes which have been found to cause the inherited forms of ALS. This therapy involves administering a small molecule that has the potential to interrupt the cascade of events that leads to the loss of muscle function which characterizes ALS. If this therapeutic approach slows or stops the progression of ALS, this will be a medical milestone.

“Often unheralded, drug development is only possible because of the willingness of people with ALS and their families to participate,” said Richard Smith, MD, Director of the Center for Neurologic Study and Principal Investigator on The ALS Association’s sponsored ASO research study. “Ultimately, the history of every drug is not only in the facts of its discovery. It is in the personal stories of these patients, who are willing to face risks and discomfort, all without knowing whether or not they will personally benefit.”

“There’s never been a more exciting time for therapy development in ALS. We expect that gene silencing approaches that directly target the cause of disease will get to human clinical trial within the next two years,” said Don W. Cleveland, PhD, Ludwig Member and Chair of the Department of Cellular and Molecular Medicine at the University of California, San Diego (UCSD).

"This study is an excellent example of the versatility of antisense to target disease-associated proteins that cannot be approached with other technologies," said Frank Bennett, Ph.D., Vice President of Antisense Research for Isis Pharmaceuticals. "Drs. Smith and Cleveland's findings are encouraging and we hope to move these compounds forward in order to better understand their potential for treating this form of ALS and to broaden the application of antisense into numerous neurodegenerative disorders."

It is particularly noteworthy that this work on ASO therapy was conducted through the joint efforts of a physician, a university scientist, and a biotech company, all of whom have worked tirelessly towards the single goal of finding a cure for ALS, a goal that has so far been elusive and unattainable. Their collaborative efforts have been supported by The ALS Association’s TREAT ALS™ (Translational Research Advancing Therapies for ALS) program, which supports these kinds of innovative approaches often before academic and industry partnerships or other funding would be possible.  “As the growing number of ALS studies attest, many cases of the disease begin with the expression of a mutant gene,” said Lucie Bruijn, PhD, MBA, Chief Scientist of The ALS Association. “Blocking that expression, therefore, stands out as a promising definitive therapy, potentially stopping the complex cascade of events which lead to motor neuron death before it starts.”

“It is through our generous donors that these kinds of discoveries are made possible, and ASO therapy has emerged as a highly promising approach in combating ALS as well as other diseases,” Bruijn continued. “In fact, this approach may represent the most effective model for studying disease development, new drugs and treatments. After years of preclinical research, the first human studies were completed in 2012. These studies, reported in a prestigious medical journal, demonstrated the safety and practicality of antisense therapy. As a result of this recent success, a clinical trial is tentatively scheduled for 2015.”

“Today, there is much for people with ALS and their families to be hopeful for,” said Fred Fisher, President and CEO of The ALS Association Golden West Chapter. "There has been significant progress in the field of ALS research, and our great hope is that we will have the technology that will not only help us to better understand ALS but also find effective treatments to halt its relentless progression. The ALS Association Golden West Chapter is proud to recognize these researchers for their unwavering dedication and efforts that bring us closer to our vision of creating a world without ALS.”