Members of ALS Community Gather with Representatives from FDA and Industry to Inform FDA Draft Guidance on ALS

The ALS community recently presented its recommendations to the U.S. Food and Drug Administration (FDA) regarding the Amyotrophic Lateral Sclerosis: Developing Drugs for Treatment Guidance for Industry at a day-long event, called ALS Community Workshop: Therapy Development and Regulatory Pathways, which was held in Washington, D.C., on July 12. Over 90 people attended in person, with many more tuning in online.

Throughout the day, there were opportunities to make comments to the FDA and industry representatives, both in-person and online. The Association was proud to host the day to bring many ALS stakeholders together to provide targeted feedback and information to further inform the FDA’s Draft Guidance on ALS Drug Development.

The FDA’s Draft Guidance provides drug developers with recommendations for all aspects of clinical trials for ALS, from study design to risk-benefit considerations to patient selection. For many years, The ALS Association has worked closely with the FDA and members of the ALS community in the development of a comprehensive ALS Guidance to make clinical trials faster, shorter, and more responsive to special considerations inherent to people with ALS and their caregivers.

The Workshop was an opportunity to provide targeted feedback and information from the ALS community to further inform the FDA’s Draft Guidance on ALS Drug Development. After the Workshop, the Association composed a report to the FDA to address key points discussed at the Workshop and to make recommendations on how to improve the FDA’s final Guidance (listed below).

“The purpose today is to build a strong guidance together,” said Calaneet Balas, president and CEO of The ALS Association. “We will be taking the great ideas from this meeting and presenting them to the FDA for their consideration in developing the final Guidance document. We look forward to that document, and to seeing it used widely for making clinical trials faster, more effective, and more aligned with the needs of patients and caregivers. We believe that is the route to the fastest development of new treatments for ALS.”

Continue reading Members of ALS Community Gather with Representatives from FDA and Industry to Inform FDA Draft Guidance on ALS

Meet Dr. Don Cleveland: Prominent ALS Researcher Who Pioneered Antisense Technology

Our Every Drop Adds Up campaign builds on the idea that people coming together for a common goal can make the impossible happen, just as it did four summers ago when the ALS Ice Bucket Challenge soaked the world. Every person helped, research project funded, story shared, discovery made, piece of legislation passed, and auction bid placed – it all adds up!

By the beginning of 2019, three trials are slated to be underway to help develop antisense therapy for people with ALS, dividends on a bold investment The ALS Association made in 2004, when the technology was new. We sat down with Dr. Don Cleveland, a pioneer in the field, for the second part of our series highlighting antisense technology.

Dr. Cleveland, professor at the University of California, San Diego’s Ludwig Institute for Cancer Research, is a long-time funded ALS researcher. He has been working with The ALS Association since 2004 to develop DNA-based designer drugs to silence ALS genes, called antisense gene silencing or antisense technology.

In 1999, The ALS Association, along with the American Academy of Neurology, awarded Dr. Cleveland with the prestigious Sheila Essey Award for his significant contributions to research. He was recently awarded the $3 million prestigious 2018 Breakthrough Prize in Life Sciences for his numerous accomplishments in neurodegenerative disease research, including establishing antisense therapy in animal models of ALS.

He talked with us to discuss this important gene silencing technology that currently targets inherited ALS genes and could be potentially applied to non-inherited (sporadic) ALS in the future.

Continue reading Meet Dr. Don Cleveland: Prominent ALS Researcher Who Pioneered Antisense Technology

The ALS Association’s Early Investment in Antisense Technology Is Having a Real Impact

Our Every Drop Adds Up campaign builds on the idea that people coming together for a common goal can make the impossible happen, just as it did four summers ago when the ALS Ice Bucket Challenge soaked the world. Every person helped, research project funded, story shared, discovery made, piece of legislation passed, and auction bid placed – it all adds up!

When Dr. Timothy Miller and his colleagues from Washington University in St. Louis published preclinical data in The Journal of Clinical Investigation last month, showing how second-generation antisense drugs were effective in ALS mouse and rat models, it served as a vivid reminder that every research investment and discovery adds up.

Antisense technology, which The ALS Association has supported from the beginning, represents one of the most promising potential treatments for ALS. The goal is to prevent the production of the toxic proteins by preventing them from being created in the first place. Toxic proteins kill motor neurons, the cells that primarily die in ALS, leading to muscle weakness and, eventually, paralysis.

Doing so could slow or stop disease progression in people living with ALS. Specifically, an antisense drug selectively targets and binds to the mutated messenger RNA linked with ALS and prevents toxic proteins from being made.

Continue reading The ALS Association’s Early Investment in Antisense Technology Is Having a Real Impact

The ALS Association and Teva Grant Two Awards as part of Teva CNS Target Identification Crowdsourcing Challenge

The ALS Association and Teva Pharmaceutical Industries Ltd. (“Teva”) have announced the recipients of the TEVA CNS Target Identification Crowdsourcing Challenge awards for their outstanding proposals to identify novel ALS targets. The two awards will be granted to Dr. Philip Wong, along with Dr. Jonathan C. Grima and Dr. Jeffrey D. Rothstein, all from Johns Hopkins University School of Medicine in Baltimore, Maryland.

Continue reading The ALS Association and Teva Grant Two Awards as part of Teva CNS Target Identification Crowdsourcing Challenge

Clinical Trial: Orphazyme to Enroll 231 People with ALS in Phase III Arimoclomol Clinical Trial

The ALS Association Supported Research In Arimoclomol Development

Our Every Drop Adds Up campaign builds on the idea that people coming together for a common goal can make the impossible happen, just as it did four summers ago when the ALS Ice Bucket Challenge soaked the world. Every person helped, research project funded, story shared, discovery made, piece of legislation passed, and auction bid placed – it all adds up!

Biopharmaceutical company Orphazyme A/S launched a phase III clinical trial of arimoclomol, giving a dose to a person with ALS for the first time, the company announced recently. The randomized and placebo-controlled trial will enroll 231 people with ALS in North America and Europe to determine efficacy and safety of the oral drug.

Dr. Michael Benatar, executive director of the Kessenich Family ALS Center at the University of Miami and principal investigator of the study, presented data of the successful phase II clinical trial testing arimoclomol in patients carrying SOD-1 mutations at the International ALS MND Symposium in Dublin in December 2016.

The ALS Association, along with the U.S. Food and Drug Administration’s (FDA) Orphan Products Development Program, provided funding for the trial. The study was not powered to determine therapeutic effect; however, the data demonstrated safety and preliminary evidence for efficacy, which led to this new phase III trial. The open access data was later published in Neurology in January 2018.

“On behalf of the patients struck by this devastating disease, I am delighted to see this Phase III trial up and running. The ALS Association invested in the early clinical trials and are pleased to see this moving forward to a Phase III study and to seeing the potential of arimoclomol unfold.” (Dr. Lucie Bruijn, Chief Scientist, The ALS Association)

Continue reading Clinical Trial: Orphazyme to Enroll 231 People with ALS in Phase III Arimoclomol Clinical Trial

ALS Researcher Living With ALS Continues to Work Toward a Cure

Our Every Drop Adds Up campaign builds on the idea that people coming together for a common goal can make the impossible happen, just as it did four summers ago when the ALS Ice Bucket Challenge soaked the world. Every person helped, research project funded, story shared, discovery made, piece of legislation passed, and auction bid placed – it all adds up!

Dr. Rahul Desikan is incredible. He’s a prominent researcher of neurodegenerative diseases, including ALS, as well as a loyal husband, father, son, and friend. And on February 17, 2017, in a cruel twist of fate, he became a person with ALS.

Dr. Desikan entered the research and medical field at Boston University in Massachusetts, obtaining both his M.D. and Ph.D. He then completed his diagnostic radiology residency at University of California, San Diego (UCSD) before moving on to University of California, San Francisco (UCSF) for his neuroradiology fellowship.

In the fall of 2016, after years of hard work, he was offered an assistant professorship, along with his own research lab, at UCSF.

Continue reading ALS Researcher Living With ALS Continues to Work Toward a Cure

The ALS Association Partners with GNS Healthcare to Apply Artificial Intelligence to Accelerate Answer ALS Research

August marks a month-long opportunity to raise awareness and move closer to a world without ALS. Our Every Drop Adds Up campaign builds on the idea that people coming together for a common goal can make the impossible happen, just as it did four summers ago when the ALS Ice Bucket Challenge soaked the world. Every person helped, every research project funded, every discovery made, every piece of legislation passed, and every story shared – it all adds up!

We recently announced that we’re providing new funding to allow GNS Healthcare to use artificial intelligence (AI) to create a comprehensive disease model to advance research into ALS. GNS Healthcare will use its powerful machine learning platform, called REFS, in conjunction with the rich Answer ALS patient datasets, which are accessible to clinicians and scientists throughout the ALS research community. The project will be led by Dr. Iya Khalil, chief commercial officer and co-founder of GNS Healthcare.

Answer ALS, under the leadership of Dr. Jeffrey Rothstein, executive director for the Answer ALS Research Program, is collecting data from 1,000 people with ALS to build a comprehensive picture of the disease that includes clinical, genetic, molecular, and biochemical information that is openly shared with the global ALS research community. Together, this information will yield thousands of petabytes of new ALS-specific information that requires analysis. Continue reading The ALS Association Partners with GNS Healthcare to Apply Artificial Intelligence to Accelerate Answer ALS Research