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

Meet Nibha Mishra: 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.

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 fourth 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. Nibha Mishra from Massachusetts General Hospital and Harvard Medical School to learn about her unique research project aimed at understanding disease pathways associated with ALS fused in sarcoma (FUS) gene mutations.

Continue reading Meet Nibha Mishra: A Bright, Young ALS Researcher

Biohaven Initiates Expanded Access Program for ALS Drug BHV-0223

Biohaven Pharmaceuticals, Inc. initiated an expanded access program (EAP) of BHV-0223 drug, a sublingual, lower dose formulation of Rilutek® (riluzole). The drug uses a Zydis® orally dissolving tablet technology, which does not require swallowing or additional fluids. Riluzole is the first drug approved by the U.S. Food and Drug Administration to treat ALS. It prolongs life approximately three months.

“It is great for the ALS community that Biohaven is taking advantage of the FDA’s expanded access program,” said Calaneet Balas, president and CEO of The ALS Association. “We believe this is the first instance of a company offering expanded access for widespread use in ALS. Such programs enable people with ALS and their doctors to consider the use of investigational drugs.”

Continue reading Biohaven Initiates Expanded Access Program for ALS Drug BHV-0223

ALS Association-Funded Researchers Raise Their Voices to Discuss Latest Findings as Part of ALS Awareness Month

Researchers around the world working together for treatments and a cure for ALS are a main reason why we’re on the verge of changing the nature of the disease forever. As part of National ALS Awareness Month, we sat down with two ALS researchers at Johns Hopkins University in Baltimore who are funded by The ALS Association.

Dr. Ke Zhang, a postdoctoral fellow who was awarded a Milton Safenowitz Postdoctoral Fellowship in 2014 and another, Dr. Thomas Lloyd, associate professor of neurology and neuroscience, who we have supported for many years and currently fund.

ALS is a complex disorder with numerous molecular and cellular pathways involved in disease. All the underlying mechanisms of these pathways and how they could potentially connect and interact are still under intense investigation.

Together, with their colleagues, Drs. Zhang and Lloyd recently published an important paper in the prestigious journal Cell that links together two fundamental cellular pathways – nucleocytoplasmic transport and stress granule assembly – in ALS disease, moving them closer than ever to discovering new therapies for ALS.

Continue reading ALS Association-Funded Researchers Raise Their Voices to Discuss Latest Findings as Part of ALS Awareness Month

Update on Neuroscience in the Clinic: Antisense Oligonucleotide (ASO) Therapy

The 70th Annual American Academy of Neurology meeting, held in Los Angeles last week, provided an opportunity to check in on antisense therapies and the continued dividends from The ALS Association’s early investment in the technology.

Antisense oligonucleotide therapies are designed to prevent the production of disease proteins, saving the body of toxicity. Despite initial skepticism about the concept, The ALS Association took the lead in developing antisense therapies in 2004. As we learned in Los Angeles, that decision has contributed significantly to the progress made in the fight against ALS.

In December 2016, the Food and Drug Administration approved the first ever antisense drug, Spinraza. Spinraza targets spinal muscular atrophy (SMA), a common neuromuscular disease, which is the leading cause of genetic death in infants and toddlers. This is the first approved treatment for this disorder. This success provides hope for the future of antisense therapies targeting ALS.

Currently, there are more than 20 antisense drugs in preclinical stages or in clinical trials. This includes an ongoing trial to test antisense oligonucleotides that target SOD1, the second most common cause of ALS. That trial is currently in phase I/II. Also, work is being done to prepare the upcoming C9orf72 antisense clinical trial, slated to start in the near future (see more below). The C9orf72 mutation is the most common mutation associated with inherited ALS.

During the American Academy of Neurology Meeting in Los Angeles, experts from multiple neurodegenerative diseases came together to give an overview of ASO therapy past and present. Here is a short summary of the antisense technology presentations.

Continue reading Update on Neuroscience in the Clinic: Antisense Oligonucleotide (ASO) Therapy

The ALS Association Kicks Off ALS Awareness Month

We’re excited to kick off National ALS Awareness Month. The next 31 days will be dedicated to education about the disease and the work happening around the world to find a cure, and to advocating for people with ALS and their caregivers.

This year, we’re asking you to Raise Your Voice to elevate the fight against ALS.

As Simon Fitzmaurice, who lost his nine-year battle against ALS in 2017, wrote in his memoir, “It’s Not Dark Yet”: “Communication is key with ALS because the disease takes away your ability to speak. But it need not take your voice.”

Continue reading The ALS Association Kicks Off ALS Awareness Month

2018 AAN Conference Highlights ALS Environmental Risk Factors

At last week’s 70th Annual American Academy of Neurology (AAN) Meeting in Los Angeles, neuroscientists and neurologists from all over the world came together to present their important work and learn from each other. We are proud that many top ALS researchers we support attended and presented a wide range of research, on topics ranging from environmental risk factors to work leading up to clinical trials.

This is the first in a two-part series highlighting some of the exciting work presented at the AAN event. This first article discusses the research around environmental factors impacting ALS risk.

Continue reading 2018 AAN Conference Highlights ALS Environmental Risk Factors