ESSEY COMMITMENT TO CURES AWARD
“Why do neurons die?” These four words, asked in a dorm room at Brown University, would be an inspiration to launch a seven-year journey to develop novel therapeutics for amyotrophic lateral sclerosis and other neurodegenerative diseases.
This incredible story sounds like a Hollywood movie: Two undergrads strike up a friendship on a tennis court and discover a shared interest in neurons, and, more specifically, how they die. They decide to pursue their hunch about a novel approach that could slow neuronal death, and take the world of science by storm. But this story is true.
After methodically poring over an endless supply of scientific literature, Joshua Cohen (a biomedical engineering major) and Justin Klee (a neuroscience major) decided the best way to prevent degradation in these diseases was to focus on preserving neurons. They hypothesized that the combination of two therapies might have a positive effect on ALS.
Joshua received his BS in biomedical engineering from Brown University and gained research experience at Brown University and at the National Institute of Standards in Technology. Justin also researched at Brown University in neural systems and neurophysiology and Alzheimer’s disease at Harvard Medical School, before receiving his Sc.B in Neuroscience from Brown University. In 2013, the two friends founded Amylyx Pharmaceuticals, dedicated to the development of therapeutics for the treatment of neurodegenerative disorders.
The company’s focus was on a compound called AMX0035, which includes two small molecules which act synergistically to prevent nerve cell death by blocking stress signals within the mitochondria (which provide cells with energy), and the endoplasmic reticulum (which is involved in making proteins). The company incorporates unconventional approaches through strong partnerships with industry leaders, scientists, doctors, and organizations.
Despite the challenges of launching an ALS clinical trial, they designed their study, named CENTAUR, with input from patients, the Northeast ALS (NEALS) Consortium, the Neurological Clinical Research Institute at Massachusetts General Hospital, and the Barrow Neurological Institute. The trial was partly funded by Ice Bucket Challenge donations. In December 2019, the results from the CENTAUR Phase 2 trial showed AMX0035 significantly slowed disease progression in patients with ALS compared to placebo. The company is urgently working for the early approval of AMX0035, as a promising oral therapy.
In June 2021, Amylyx filed a New Drug Submission (NDS) for AMX0035 for the treatment of ALS with Health Canada. On November 2, 2021, Amylyx announced it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for AMX0035 for the treatment of ALS. On November 4, 2021, the company announced the launch of their Phase 3 Study, named PHOENIX, which will be conducted at approximately 65 sites in Europe and the U.S., including two California ALS Treatment Centers of Excellence. The trial will enroll approximately 600 participants with clinically definite or clinically probable ALS within 24 months from symptom onset which expands on the inclusion criteria used in the previous trial.
The Golden West Chapter Chapter is proud to recognize Amylyx with the 2021 Commitment to Cures Award for their efforts to champion drug discovery and development in the relentless pursuit of treatments and cures for people with ALS and related diseases.
Watch the full interview with the Essey Commitment to Cures Award recipient, Amylyx. Accepting the award on behalf of Amylyx are Co-CEO's and Co-Founders, Josh Cohen and Justin Klee.
DEAN AND KATHLEEN RASMUSSEN ADVOCATE OF THE YEAR AWARD
California State Assemblymember
Dr. Joaquin Arambula
Dr. Joaquin Arambula was born in Delano, in Kern County, California- the heart of the farmworker movement. He grew up in Fresno, where he attended public schools. He studied at Bowdoin College in Maine and then attended medical school at the University of Minnesota. His parents, Juan and Amy Arambula, as a former Assembly member and a health care advocate, instilled in their children a commitment to hard work, purpose, and public service.
Following graduation, Dr. Arambula chose to return to Fresno to serve his community as a physician. He saw the emotional and financial hardships of people struggling with illnesses that could have been prevented with access to quality health care. His experiences as an emergency room doctor in Selma in Fresno County forged his determination to improve health care for all people, especially the most vulnerable. Dr. Arambula eventually became the Medical Director of California Emergency Physicians at Adventist-Selma Hospital.
In 2016, Dr. Arambula was elected in April 2016 in a special election to represent California’s 31st Assembly District in the Central Valley. He is the first Latino physician elected to the California State Assembly, and proudly represents a district that includes 41% of the city of Fresno, as well as all the rural cities and communities in Fresno County.
Since his election, he has been a strong advocate in addressing the dire shortage of doctors and other health care professionals in the Central Valley. Driven by the belief that health care is a fundamental human right, he has authored legislation that improves health and mental health services, which also includes measures that ensure transparency regarding management of these services and their costs. He also has worked diligently for improvements in other areas, including public safety, job development, and support for veterans and service members.
In line with his commitment to access to health care, Arambula played an integral role in helping the Golden West Chapter to secure multi-year California State public funding to benefit the Chapter’s Wraparound Model of Care.
Assemblymember Arambula served as the chair for Assembly Budget Committee No. 1 on Health and Human Services. On April 30, 2018, Rusty Selix, a professional California mental health care advocate who had been recently diagnosed with ALS, testified for the Committee, along with lobbyist Fred Noteware and Fred Fisher, President & CEO of the Golden West Chapter. These dedicated ALS advocates urged state officials to support the Chapter’s evidence-based wraparound care model of care, which combines medical, community and home-based services and support, has been proven to help people with ALS live better and longer with the disease. It is also designed to accelerate the search for effective treatments and cures for ALS. Assemblymember Arambula quickly recognized the importance of the issue and the critical need for this state funding, and became an advocate for the request.
On June 27, 2018, California’s then Governor Brown signed budget bill SB 840, dedicated $9 million in appropriations, given over the course of three years, in support of California’s ALS community and positioned the Golden West Chapter to serve as the administrator for statewide distribution of funds annually. This was an important recognition of the diverse and ongoing needs of people living with ALS and their families, acknowledgment of the work of the Golden West Chapter, and an unprecedented commitment of state funding. In 2021, Governor Gavin Newsom and the California legislature committed to the renewal of these annual appropriations to the Golden West Chapter, to support vital programs and multidisciplinary ALS care across California. This time, the annual funding was extended to five years, for a total of $15 million, in continued funding for the care and support of the ALS community.
The Golden West Chapter salutes Dr. Joaquin Arambula for his many efforts as a healthcare advocate and for helping to accelerate the search for effective treatments and cures for ALS.
Watch the full interview with the Dean and Kathleen Rasmussen Advocate of the Year Award recipient, Dr. Joaquin Arambula
A Note from the Essey Family
Every year, our family participates in Champions for Cures and Care in memory of our mother, Sheila Essey, and for the second year, our father, Richard “Dick” Essey. Dick died in April of 2020 at the age of 97 of natural causes at his home in San Francisco. It was his wish that his family continue his strong support of the Golden West Chapter. We plan to fulfill that wish for years to come.
Dick was deeply invested in the Chapter’s success. In 1995, after watching his wife, Sheila, suffer through her debilitating struggle with ALS without adequate support services, he founded what was originally called the Greater Bay Area Chapter of The ALS Association as a way to help people with ALS and their caregivers. Now, 25 years later, his vision and legacy are honored through our family's continued support of the Golden West Chapter, the largest and most successful chapter in the country.
He also established the Sheila Essey Award for ALS Research, annually honoring the year’s most promising researchers with a cash prize of $50,000, in association with the Golden West Chapter of The ALS Association, the American Brain Foundation and the American Academy of Neurology.
We salute the honorees of tonight’s virtual event and are proud to continue to support the important mission of the Golden West Chapter.
About Dean & Kathleen Rasmussen
Dean Rasmussen is fondly referred to as the Golden West Chapter's "Godfather of Advocacy.” After losing his father to ALS, Dean put his grief into action through philanthropy and advocacy.
His vision and philanthropic support served to catalyze the creation of the Association’s federal public policy program and the Chapter’s program in California. He has spearheaded The Golden West Chapter’s Advocacy efforts in both Sacramento and Washington, D.C. and served as the catalyst for the creation of the Chapter's public policy program. Dean is an emeritus trustee of the National ALS Association; Founder and Emeritus Chair of The ALS Association Federal Advocacy Committee; and is an ongoing supporter of ALS advocacy efforts throughout California and Hawaii.
Kathleen O’Brien Rasmussen, served as an Advisory Trustee and, later, the Chair of the Board of Directors of The ALS Association Greater Los Angeles Chapter. She successfully co-chaired the merger of the Greater Bay Area and Los Angeles Chapters into the Golden West Chapter in 2010. She is an Emeritus Trustee of the Harvard Medical School Neuro Discovery Center. She recently returned to serve as a board member for the Golden West Chapter and is an active part of the Chapter's Marketing and Communications Committee.
Both Dean and Kathleen enjoy traveling, spending time with their family, especially their grandchildren. They are also passionately involved in the fight against neurodegenerative disease through their efforts with the Golden West Chapter and the Michael J. Fox Foundation for Parkinson’s Research.
Bryan Deierling, Chair
The ALS Association Golden West Chapter’s mission is to discover treatments and cures for ALS, and to serve, advocate for and empower people affected by ALS so they can live their lives to the fullest.
Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s Disease, is a fatal neurodegenerative illness that affects nerve cells in the brain and spinal cord. Everything we do advances the search for effective treatments and cures for ALS. The Chapter currently serves more than 1,800 people with ALS and their families in 31 counties in California and the state of Hawaii.
Proceeds from Champions for Cures and Care will provide critical funding for The ALS Association Golden West Chapter’s mission priorities in the areas of care services, public policy, and cutting edge ALS research—all of which are essential to achieving our vision: creating a world without ALS.
VP, Community Outreach
818-865-8067 ext. 225