In 2018 we set out with the goal to spark hope, ignite action, and inspire others to join us in the fight to create a world without ALS. And it was thanks to our wonderful community that we were able to achieve that goal. Throughout the year we provided patient services, hosted community events, and helped fund life-changing research to find new treatments for this devastating disease.
Three days before Christmas 2009, Turner Corbett’s family got the devastating news that his mom, Jennifer, had been diagnosed with ALS. Turner was just 12 years old.
Wanting to make the most of a difficult situation, in 2015, Turner created a benefit event that would raise money for ALS research. After running through some ideas with his friend Alex Doswell, they decided a dodgeball tournament would be the most fun and family friendly.
Based on information as of 5/5/2017
What is Radicava?
Radicava™ (edaravone) is a prescription medicine approved by the U.S. Food and Drug Administration (FDA) to treat people with amyotrophic lateral sclerosis (ALS).1
In clinical trials, some people given Radicava showed significantly less decline in physical function compared to placebo as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R), a validated rating instrument for monitoring the progression of disability in patients with ALS.1,2
Radicava is First Approved Treatment for ALS in Decades
The Food and Drug Administration’s (FDA) announced today that it has approved Radicava (edaravone), the first new treatment approved specifically for ALS in 22 years. The FDA approved Radicava less than a year after Mitsubishi Tanabe Pharma Corporation submitted a New Drug Application. The only other approved treatment specifically for ALS, riluzole, was approved in 1995.
“We thank the FDA and MT Pharma for working together to expedite the approval of the first new ALS-specific treatment in decades,” said Barbara Newhouse, president and CEO of The ALS Association. “We hope today’s announcement signals the beginning of a new chapter in the fight against this terrible disease. There are several drugs to treat ALS currently in clinical trials and we are hopeful that people living with ALS have even more therapies available to them sooner rather than later.”
ALS is a devastating disease with no cure.
But researchers are working to change that. The increased awareness and donations provided by events like the ALS Ice Bucket Challenge—and by people like you—are making a real difference in the pace of discoveries, bringing us ever closer to the end of ALS.
Here are just a few examples of recent advancements in ALS research and technology:
The ALS Association funds the world’s largest ALS research program, working with more than 150 labs around the globe. The ALS Association supports a wide breadth of specific fields of study that are critical to advancing ALS research.
A significant portion of the research funded by the ALS Association is done through the vast network of clinical research and clinical trials. The ALS Association funds a nation-wide network of Certified Treatment Centers of Excellence, which must meet the ALS Association’s clinical care and treatment standards based on the American Academy of Neurology Practice Parameters. In order to achieve this certification, the clinic must participate in ALS-related research and successfully complete a comprehensive site review.
$1 Million Grant from Ice Bucket Challenge Helped Spur Discovery
Today, researchers from Project MinE announced that they have identified a new gene NEK1 that ranks among the most common genes that contribute to ALS. The study revealed an association between mutations in the gene and ALS. The discovery of NEK1 gives scientists an exciting new target for drug development.
Approximately 10 percent of ALS cases are familial, meaning genes are inherited from a family member. The other 90 percent of ALS cases are sporadic, or without a family history. The NEK1 gene is present in approximately 3 percent of all ALS cases.
Thanks to nationwide advocacy efforts, Congress is taking action on several priorities for the ALS Community.
The Senate Appropriations Committee passed the fiscal year 2017 Health and Human Services spending bill and included $10 million to continue funding to the National ALS Registry. The National ALS Registry is a congressionally directed registry for people in the U.S. with ALS. It is a program to collect, manage, and analyze data about people with living with ALS, which helps doctors and scientists learn more as they work toward a cure. It is the only population-based registry in the U.S.
Pharmaceutical company Mitsubishi Tanabe Pharma Corporation submitted a formal proposal to the United States Food and Drug Administration for approval of a drug to treat ALS. The drug, Edaravone, gets rid of toxic waste that is a normal by-product of cell function. It is thought that this waste is not as effectively removed in ALS patients, causing damage to motor neurons, which are the cells that die in ALS patients. Edaravone would protect these neurons by helping rid the cells of this waste.
The Edaravone new drug application is supported by clinical research with ALS patients in Japan, and in 2015, Edaravone was approved as a treatment for ALS in Japan and South Korea. After being submitted to the FDA for consideration, it could either be automatically approved for use in the U.S., or it could require a Phase III study conducted in the U.S.
The Neurological Clinical Research Institute has recently made significant strides in working toward diagnosing ALS earlier and measuring the progression of the disease. The NCRI imaging team conducted its first successful PET imaging scan of a person living with ALS to measure inflammation in the brain, which is a promising first step in the TRACK ALS imaging biomarker study.
Dr. Nazem Atassi and his team at Massachusetts General Hospital developed a tracer that binds to support cells of the nervous system in the brain. The tracer serves as a marker of inflammation and tracks changes in the cells of the nervous system. The PET imaging scans measure inflammation in the central nervous system, where cells move to the already damaged area of the brain and worsen damage by further harming neurons.
