Genentech, a member of the Roche group, has initiated a Phase I clinical study evaluating GDC-0134, an oral investigational medicine designed to block dual leucine zipper kinase (DLK), for the potential treatment of ALS. The trial is enrolling now in sites across the United States.
The ALS Association is proud to announce three large research organizations — Project MinE USA, Answer ALS and the New York Genome Center (NYGC) — will be working collaboratively toward their mission for treatments and a cure for ALS. These global collaborative projects, supported by The ALS Association through ALS Ice Bucket Challenge donations, will share genome sequencing information and research expertise to move efforts forward more rapidly and efficiently. Read more to learn about how global partnerships advance ALS research.
The June issue of Scientific American on newsstands this month features, “Unlocking the Mystery of ALS,” which details the significant advances of ALS research over the years. The authors, Drs. Leonard Petrucelli at the Mayo Clinic in Jacksonville and Aaron Gitler at the Stanford University School of Medicine, thoughtfully explained the complicated science behind ALS, while weaving a story of its breakthroughs and the steps needed to get to the ultimate goal – an end to ALS. They covered genetic discovery, starting with SOD1 in 1993 through today, which has sky rocketed in recent years, largely in part due to the donations dedicated to ALS research through the ALS Ice Bucket Challenge. Exciting therapeutic advances were explained, like a gene silencing technique, called antisense oligonucleotide (ASO) therapy, a way to track disease progression and improve diagnosis, called biomarkers, and targeting the support cells of the brain, called glia, and more were all described in a clear way.
Last week, President Trump submitted his budget request to Congress. Included in the proposal are changes to funding for Medicaid, cuts to Social Security Disability Insurance (SSDI), cuts to the National Institutes of Health (NIH) and the complete defunding of the National ALS Registry at the Centers for Disease Control (CDC). The ALS Association is very concerned that these proposals will negatively impact people affected by ALS and will slow down the progress we have made in research for treatments and a cure. We do not believe this is a partisan issue – Members of Congress on both sides of the aisle have expressed concerns about the cuts contained in the President’s budget.
Today, we sit down with Stephen Winthrop, our newly elected Chair of The ALS Association Board of Trustees to hear his thoughts on the fight against ALS and the role of The ALS Association in that fight. Stephen first joined the Association as a Trustee in 2015. He brings his expertise in fundraising, financial management, grant writing and information management services for non-profit organizations gained from a career in management consulting. He was diagnosed with ALS in November 2013 and immediately focused his energy on the fight against ALS. We are proud to be led by such a great leader and tireless ALS advocate.
The ALS Association is at the forefront of the global research effort to find treatments and a cure for ALS. We believe that innovation and collaboration will be the key to winning this important fight. Only by coming together with others around the world who are experts in their fields will we make significant progress. We know collaboration leads to progress.
We’d like to let you know about an exciting opportunity for innovation in ALS called the Springboard Challenge. The Springboard Challenge is a planning grant award sponsored by Partners HealthCare and the Wellcome Trust and which was announced at the World Neuroscience Innovation Forum in London on March 27.
By: Our Colleagues at ATSDR
One of the questions that people living with ALS often ask is – what can I do to help ALS research? People with ALS can do that, with a personal contribution to the National ALS Registry (Registry) – in the newly launched National ALS Biorepository (Biorepository).
The Agency for Toxic Substances and Disease Registry (ATSDR) launched the National ALS Biorepository as a part of the Registry in January 2017. This launch came after a pilot study that showed it was feasible to include a biorepository. The Biorepository is collecting biological samples from persons with ALS. It’s different from other biorepositories because it does not limit who can take part to a specific area, study, or clinic. This means that participants’ samples may help researchers everywhere work toward better understanding the causes of, and possible treatments for ALS.