Today, CNN featured a profile on Larry Falivena, a person living with ALS since 2017. Larry is visiting all 30 Major League Baseball parks in one season to raise awareness of ALS as part of The ALS Association’s Challenge Me campaign. He began his "Iron Horse tour" on May 30.
Eighty years ago on July 4, Lou Gehrig gave one of the most famous speeches in American history. His speech marked his retirement from baseball because of his recent diagnosis of ALS. Gehrig was honored by many on the field that day, and his number 4 was retired, the first time a player had ever had his jersey retired. The New York Times called it “one of the most touching scenes ever witnessed on a ball field.”
I call ALS “the nice guys’ disease.” For, as indiscriminate as it appears, ALS certainly has a way of finding the best and most courageous people to attack. Watching people endure the wasting of their muscle control and seeing them defiant in the face of the sure and steady loss of communication and independence, makes me defiant too. People with ALS fight the disease with great courage. Seeing the fire and determination of my patients inspires me every day. Our team puts that inspiration to work in the clinic and in my lab, where we are on a promising path to finding effective therapies to slow ALS.
Furthering our understanding of disease and the creation of effective therapies won’t happen in a vacuum. My peers and colleagues in the medical research community know first-hand the necessity of global collaborations that bring diverse specialties into the process of understanding complex medical mysteries such as ALS.
Watching my mother struggle to interact with the world as her ALS progressed was extremely difficult for our family, and for me personally. Her struggle inspired me to find solutions that could help people like my mother better interact with computers and their environment, even after they have lost almost all of their ability to move. I had the motivation to advance technology in a way that would bring a new quality of life to people living with ALS under Steve Saling's motto that until medicine proves otherwise, technology is the cure.
In 2016, my colleagues and I published some remarkable results of a new drug called CuATSM in a mouse model of ALS. Moving CuATSM from mice to humans is a long and difficult road that involves the cooperation and scrutiny of many people. The checks and balances are critical to balance the risks with the benefits of any new therapy.
The ALS Association has always been committed to transparency, including providing complete and accurate financial information on our website. We continue to receive the highest ratings from charity watchdog groups tasked with reviewing our spending, fundraising, and management.
There was a lot of skepticism in the research community when we proposed development of designer DNA drugs as a way to treat ALS. This therapy is an approach that uses DNA-based designer drugs to "silence" genes that are known to cause a particular disease. The ALS Association was the first funding organization to invest in designer DNA technology research and development for therapy in neurodegenerative disease.
We launched the Challenge Me campaign last week. Challenge Me picks up where the ALS Ice Bucket Challenge left off. This time, we are challenging the world to do anything and everything they can to help end ALS.
The impact of an ALS diagnosis is profound – and not just for the person living with the disease. Daily activities, like making meals, doing laundry, mowing the lawn, and walking the dog, take a back seat when providing care for a family member with ALS.
Last week, Pete Frates and Pat Quinn unveiled a new campaign – Challenge Me -- to celebrate the fifth anniversary of the ALS Ice Bucket Challenge and reengage the millions around the world who took the Challenge. Pat and Pete brought the Challenge Me ethos to life with the Ice Bucket Challenge and they have continued to inspire the entire ALS community over the last five years.
Five years ago, we challenged our friends and family to dump buckets full of ice over their heads to raise awareness and funds for ALS. The rest is history. This year, we have a new message: Challenge Me.
Biogen has initiated a phase 3 clinical trial evaluating tofersen (previously called BIIB067), an antisense oligonucleotide (ASO), a type of antisense drug, targeting superoxide dismutase (SOD1), for the potential treatment of ALS. The trial is now enrolling and aims to enroll approximately 60 people with SOD1 ALS.
The ALS Association is pleased to announce a new scholarship program that will fund up to $5,000 per year for at least 30 students whose personal or family finances are compromised by the financial burden of ALS. The Jane Calmes ALS Scholarship Fund was established by Mark Calmes, vice chair of The ALS Association’s National Board of Trustees. The scholarship fund is named in honor of his late wife, Jane, who fought ALS for eight years and passed away in August 2017.
The ALS Association is proud to be a longtime supporter of the Airlie House ALS Clinical Trials Consensus Guidelines, which have been revised and published in Neurology, the most widely read and highly cited peer-reviewed neurology journal.
Our Milton Safenowitz Postdoctoral Fellowship Program continues to support young scientists and is the only program of its kind specifically funding early ALS postdoctoral fellows. The awards were founded in memory of Mr. Safenowitz by the Safenowitz family – through The ALS Association Greater New York Chapter. The program encourages young scientists to enter and, importantly, to remain in the ALS field.
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Next week at the American Academy of Neurology Meeting (AAN) in Philadelphia, Biogen will present promising results of the phase 1/2 study of its newly named investigational therapy tofersen (previously BIIB067), which is now enrolling in a phase 3 trial. Tofersen is an antisense oligonucleotide (ASO), a type of designer DNA drug, targeting SOD1.