ALS Biotech Company Treeway Announces Promising Phase I Trial Results For Drug TW001

Treeway, a biotech company in the Netherlands with a mission to develop a cure for ALS, recently announced promising results of its phase I trial of its lead drug, TW001. TW001 is an oral formulation of edaravone. Radicava®, the most recently FDA approved drug to treat ALS, is an intravenous version of edaravone. The study showed that TW001 was well tolerated and stable, while improving bioavailability compared to Radicava.

“An oral formulation of Radicava will be pivotal in providing a more convenient means to take the medication in comparison to keeping to a drug infusion schedule,” stated Dr. Lucie Bruijn, chief scientist of The ALS Association.

Based on the promising phase 1 trial results, Treeway is preparing for an upcoming phase III study that will test a larger population of people living with ALS to understand both safety and whether the drug impacts disease progression. The company’s goal is to move as quickly as possible to bring TW001 to the ALS community.

Continue reading ALS Biotech Company Treeway Announces Promising Phase I Trial Results For Drug TW001

Guest Post: ‘ALS is What I Have, Not Who I Am’

By Alan Alderman

Note: In recognition of ALS Awareness Month, we’ve been sharing a lot of new stories about people living with the disease and their caregivers, the volunteers and health care providers who help make the lives of people with ALS better, the powerful fundraisers and the participants in Walk to Defeat ALS and Team Challenge ALS events, and the researchers fighting for a cure. This is the thirteenth profile in that series.

Bad things happen to good people every day. That’s just the nature of life. But it doesn’t mean we stop living and loving.

My ALS story officially started on a warm fall afternoon in September 2001. I had been having difficulties with speaking and swallowing for several months. After more than two months of seeing about a dozen different medical professionals, and having more tests than one can imagine, I sat in the small examination room of a local neurologist.

The doctor had just finished an EMG and asked if he could be excused for a minute. What was I to say? No?

He stepped out of the room. I sat alone on the examination table for what seemed like an eternity but was probably only a few minutes.

When he returned, he sat on the small rolling stool and rolled in front of me. He looked me in the eyes, placed his hands on my knees, and said, “Alan, I believe you have ALS.”

Continue reading Guest Post: ‘ALS is What I Have, Not Who I Am’

Margot Vaughan: ‘We Are Too Close to Quit’

In recognition of ALS Awareness Month, we’ve been sharing a lot of new stories about people living with the disease and their caregivers, the volunteers and health care providers who help make the lives of people with ALS better, the powerful fundraisers and the participants in Walk to Defeat ALS and Team Challenge ALS events, and the researchers fighting for a cure. This is the 12th profile in that series.

Margot Vaughan has been fighting ALS for more than two years. But she hasn’t been fighting alone.

“My family is totally devoted to me,” Margot said. “They keep me going and I’m lucky and grateful to have such an amazing family by my side.”

Margot and Thomas, an attorney, have been married nearly 31 years. They live in southwestern Connecticut, while their two daughters – Kirsten, 26, and Caroline, 23 – settled in the New York City area after finishing college.

“Being in New York, it’s easy to get home to Connecticut, which is great,” Kirsten said. “I can have dinner with my mom [on week nights] and Caroline and I go see her every weekend.”

Continue reading Margot Vaughan: ‘We Are Too Close to Quit’

NeuroBANK – The Patient-Centric Platform For Clinical Research – Wins 2018 Bio-IT World Award

Funded by The ALS Association, NeuroBANK™ is the patient-centric platform for clinical research for numerous Association programs developed by the Neurological Clinical Research Institute (NCRI) at Massachusetts General Hospital. The platform received the Best Practices Award for Personalized & Translational Medicine at the 2018 Bio-IT World Conference and Expo in Boston earlier this month.

By providing the infrastructure and core services for clinical patient data that are shared with the international ALS community, NeuroBANK helps accelerate efforts across the research pipeline to discover future treatments for ALS.

“I couldn’t be more proud that our NeuroBANK platform has been awarded this Best Practice Award,” Alex Sherman, director of the Center for Innovation and Bioinformatics at NCRI, said. “NeuroBANK is changing the way researchers think about data, share data, and collaborate. The ALS world is coming together, teaming up against the disease by pooling its resources, collaborations, and data, all centered around NeuroBANK.” Continue reading NeuroBANK – The Patient-Centric Platform For Clinical Research – Wins 2018 Bio-IT World Award

Mexiletine Reduces Muscle Cramping in People with ALS

A recent study led by Dr. Bjorn Oskarsson from the Mayo Clinic Jacksonville and supported by The ALS Association demonstrated that mexiletine, a drug approved by the U.S. Food and Drug Administration, reduced the frequency and severity of muscle cramps in people with ALS compared to a placebo.

This could lead to an improved therapeutic treatment of people with ALS. More than 90 percent of people with ALS experience muscle cramping and how clinicians currently treat cramps varies widely.

Continue reading Mexiletine Reduces Muscle Cramping in People with ALS

Meet Anthony Giampetruzzi: A Bright, Young ALS Researcher

In recognition of ALS Awareness Month – with a theme of “Raise Your Voice” – we’re sharing new stories nearly every day in May. They’re stories about people living with the disease and their caregivers, the volunteers and health care providers who help make the lives of people with ALS better, the generous fundraisers and the participants in Walk to Defeat ALS and Team Challenge ALS events, and the researchers fighting for a cure. They are all part of our ALS community, helping us fight toward a world without ALS. This is the eighth profile in that series.

 The ALS Association is happy to continue our tradition of supporting bright, young scientists in ALS research through our Milton Safenowitz Postdoctoral Fellowship Program. These awards encourage young scientists to enter and, importantly, to remain in the ALS field.

We are proud that 90 percent of our funded postdoctoral fellows go on to start their own ALS research labs and continue to mentor more young scientists, further adding innovative ideas to the field.

This year, we are supporting six new postdoctoral fellows out of a highly competitive applicant pool. This is the third in a series of six articles highlighting the dedication and unique contribution each fellow makes to ALS research, while getting to know the person behind the lab coat.

Today, we sit down with Dr. Anthony Giampetruzzi from University of Massachusetts Medical School to learn about his unique research project aimed at identifying new ALS therapeutic targets.

Continue reading Meet Anthony Giampetruzzi: A Bright, Young ALS Researcher

Passionate ALS Advocacy Fuels the Fight for a Cure

In recognition of ALS Awareness Month – with a theme of “Raise Your Voice” – we’re sharing new stories nearly every day in May. They’re stories about people living with the disease and their caregivers, the volunteers and health care providers who help make the lives of people with ALS better, the generous fundraisers and the participants in Walk to Defeat ALS and Team Challenge ALS events, and the researchers fighting for a cure. They are all part of our ALS community, helping us move toward a world without ALS. This story highlights our National ALS Advocacy Conference – which embodies the true spirit of Raise Your Voice.

We’d like to extend a huge thank you to the more than 570 ALS Advocates who participated in our 2018 National ALS Advocacy Conference in Washington, D.C., this week. Nearly every state in the country was represented and nearly half of the participants were attending for the first time.

Most importantly, more than 100 people living with ALS made the trip to Washington to tell their stories and ask their members of Congress to fund research and do away with the five-month wait for Social Security Disability Insurance.

“Attending the ALS Advocacy Conference was one of the most rewarding things I have ever done,” said Karen Dunn, a first-time participant from Pennsylvania who was attending on behalf of her friend Melissa Cordek, a 37-year-old mother diagnosed with ALS two years ago. “I encourage everyone to become an ALS Advocate.”

Continue reading Passionate ALS Advocacy Fuels the Fight for a Cure