ALS Ice Bucket Challenge Progress
  • Sign up
  • |
  • Login
  • |
  • Find local services
  • |
  • |

 

Download the PDF version here.

The Golden West Chapter of The ALS Association helped fuel a significant number of research innovations, advancements in drug development and clinical trials, and new collaborations designed to accelerate the pace of discovery. We are excited to share some important milestones and promising progress in the search for effective treatments and cures for ALS, made possible by your support.


 

In collaboration with the Barrow Neurological Institute and the Northeast ALS Consortium, The ALS Association launched a major $3 million initiative, investing in the first-ever ALS Platform Trial, at the Sean M. Healey & AMG Center for ALS at Mass General. This ground- breaking trial will test multiple therapies for efficacy at the same time, providing time and cost savings and increasing patient access.

Celebrated the extraordinary group of scientists who were honored with inaugural Healey Center International Prize for Innovation in ALS/MND. This remarkable research team who brought the first antisense oligonucleotide (ASO) therapy, tofersen, to clinical trial included Chapter Commitment to Cures honorees: Frank Bennett, PhD, Ionis Pharmaceuticals; Don Cleveland, PhD, UCSD; Richard Smith, MD, Center for Neurologic Study; and Toby Ferguson MD, PhD, Biogen.

Amylyx Pharmaceuticals, Inc, announced that the AMX0035 clinical trial, funded by The ALS Association, demonstrated statistically significant treatment benefit for people with ALS in the CENTAUR Phase 2 trial.

Biogen, our Chapter’s 2020 Commitment to Cures honoree, announced positive results for the drug tofersen* showing reduction of toxic levels of SOD1 protein and slowing disease progression in people with ALS. The Phase 3 trial to confirm its efficacy and safety is underway. *The ALS Association funded the drug’s underlying technology.

The Golden West Chapter joined a broad coalition of dedicated patient advocates, families facing ALS, Nobel Prize winners, and respected medical professionals and scientists in supporting the passage of Proposition 14 –  the Californians for Stem Cell Research, Treatments & Cures Initiative of 2020. The Initiative authorizes $5.5 billion in state general obligation bonds, with $1.5 billion dedicated to development of treatments for diseases of the brain and central nervous system.

 

The ALS Association released “Principles for Urgent, Patient-Centered ALS Clinical Trials,” a series of recommendations to clinical trial sponsors and regulatory bodies worldwide. The principles set out clear guidelines to advance therapeutic development that are efficient, impactful, and respectful to people with ALS.

The Golden West Chapter partnered with ClinWiki.org in the launch of a new online clinical trial platform, crowd-sourced in the style of Wikipedia and Yelp. The overall goal is improved access to information for people with ALS and their families, and to increase innovation for better and faster trials. A key collaborator was our steadfast community partner, the Martha Olson-Fernandez Foundation (MOFF), which funds ALS care initiatives on the Central Coast of California and issues grants for promising ALS research.

The ALS Association contributed $3.3 million to fund a centralized, cloud-based, genome- sequence database. The first of its kind, this novel process to discover new ALS genes will allow all researchers to share genomic data and conduct standardized analyses, leading to identification of new ALS drug targets.

The Golden West Chapter hosted the 10th annual California ALS Research Summit, bringing together world renowned researchers, investigators, clinicians, biotech companies, government representatives, and advocates in ALS and related fields.

The ALS Association collaborated with Target ALS to fund the TDP43 Biomarker Initiative. Mutated TPD43 is a toxic protein found in ALS. This project will lead to development of a biomarker test to reliably measure TDP43 in human biofluids, tremendously impacting ALS clinical research and drug development.