watson-supercomputer

IBM’s Watson supercomputer discovers 5 new genes linked to ALS

You may have heard that IBM’s supercomputer, Watson, competed on Jeopardy! and is now being used to solve everything from business problems to diagnosing cancer. Today, we were thrilled to hear the latest Watson news: Working with a team at the Barrow Neurological Institute in Phoenix, headed by Dr. Robert Bowser, Watson helped researchers discover 5 new ALS genes. The Barrow researchers are excited about Watson’s future potential for further neuroscience work, and so are we!

Watch IBM’s new video below, and read more about the announcement here.

Our friend Ted

ted-haradaOur friend Ted Harada passed away this week.

Many of you may have known him. Ted was first diagnosed with ALS in August 2010 and immediately became a tireless advocate, volunteer, and voice of the ALS community.

He had served on The ALS Association Board of Trustees and on the board of directors for the Georgia Chapter, devoting precious hours of his life to steering our Association in the right direction.

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Singing for a Cure

Deborah Silver, an accomplished vocalist and performer, has been active in the fight against ALS since 2009, when her sister was diagnosed with the disease.

My sister, Marjie Block, was diagnosed with ALS in 2009. I was by her side visiting doctor after doctor hoping to get any diagnosis other than ALS. We even prayed for Lyme Disease….anything but ALS! Ultimately, this unfortunate diagnosis stayed the same and Marjie’s courageous battle began.

As our family rallied behind her, we became determined to win this seemingly unwinnable ordeal and have committed ourselves to fighting alongside all ALS patients. My children have launched several projects to raise funds for a cure including my daughter’s website, hopeheARTbyMadison.com, so far raising over $100,000. Having always felt there is a reason for everything, perhaps this disease ended up in our family because we simply will not rest until ALS is eradicated. This is why I am always trying to come up with ideas of how to raise even more funds for a cure.

Throughout this process, I have met some of the most impressive and special people in the world who also happen to be battling ALS. My heart goes out to everyone and their families struggling with this horrendous disease.

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FDA Accepts New Drug Application for Potential ALS Treatment Edaravone

Following up on our June 2016 blog post about edaravone, an intravenous drug therapy produced by Mitsubishi Tanabe Pharma Corporation, locally based in Jersey City, N.J., with a head office in Osaka, Japan, there has been recent news as to the status of the drug in the U.S.

At the end of August 2016, the U.S. Food and Drug Administration (FDA) accepted a New Drug Application (NDA) for edaravone, which has been used to treat stroke victims in Japan for the last twenty years. In a phase II clinical trial of edaravone, in a sub-group of people with ALS in Japan, researchers found a statistically significant improvement in the ALS Function Rating Scale-Revise. Edaravone works by getting rid of toxic by-products generated in cells, which is thought to protect against oxidative stress.

An “NDA” includes a comprehensive overview of a drug, including results and data analysis for the entire clinical development program and earlier preclinical testing, as well as the proposed labeling and manufacturing plans of the new drug.

This is extremely positive news for the ALS community, as acceptance of the NDA is the final stage of the development and approval process. Moreover, this is the first time that the FDA has ever accepted an NDA without first requiring an Investigational New Drug Application (IND), something that would have delayed the delivery of edaravone by many years. We appreciate the FDA for taking this step to help speed the approval of a potential ALS therapy. If approved, edaravone would be commercialized under the brand name Radicava™

While the company requested “priority review” for edaravone, which would have meant a six-month review time, the FDA accepted the application with a standard ten-month review. This means that a decision on the application is expected by June, 2017 based on timelines set by the Prescription Drug User Fee Act. This does not mean that the review will take the full ten months or preclude the FDA from acting sooner. We have been in contact with our colleagues at the FDA to reiterate the urgent need to move forward as quickly as possible.

The ALS Association joins the ALS community in urging a timely review of edaravone so that, if approved, it becomes available to people living with ALS as soon as possible. Every day makes a difference to people and their families fighting this disease. This truly is an exciting development for the ALS community, as it’s only the third potential ALS drug in history to advance this far in the drug development process (Riluzole and Myotrophin were the first two).