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.
On May 14- 16 more than 600 advocates took part in a very successful National ALS Advocacy Conference here in Washington, DC. Of the 600 advocates, 130 were people living with ALS. On Sunday and Monday, national experts and officials briefed participants on The ALS Association priorities and other “hot topics” that they might encounter in their congressional meetings and Chapter Executives prepared their state delegations for Hill meetings.
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.
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 Joanne Mattingly and Shannon Kennedy
My name is Joanne. I am 74 years old and was diagnosed with ALS September 2016.
My mother, Dorothy, had ALS and died in 1986.
As I navigated my illness, I reflected on my relationship with my mom when she was going through this. I remembered that I spent time with her, but we did not talk about her internal world. She was a kind and loving person but was not comfortable sharing her feelings.
I, being of another different generation, quickly found out that this illness encourages me to grow internally and share who I am with my family and friends. Their visits, calls and cards raise me up.