Biogen to Present Promising Results of Phase 1/2 Trial of Antisense Targeting SOD1

Next week at the American Academy of Neurology Meeting (AAN) in Philadelphia, Biogen will present promising results of the phase 1/2 study of its newly named investigational therapy tofersen (previously BIIB067), which is now enrolling in a phase 3 trial. Tofersen is an antisense oligonucleotide (ASO), a type of designer DNA drug, targeting SOD1.

The ALS Association is proud to be the first funder of antisense technology.  Our support   dates back to 2004, when antisense was just an idea in Dr. Don Cleveland’s lab at University of California San Diego (UCSD). This new potential treatment targets the second most common cause of inherited ALS, mutations in the SOD1 gene.

Continue reading Biogen to Present Promising Results of Phase 1/2 Trial of Antisense Targeting SOD1

A Look Back at Over $16 Million in Research Grants Awarded During 2018

Investments are Accelerating Innovation and Progress Toward a Cure

It’s ALS Awareness Month, and The ALS Association remains steadfastly committed to funding the most hopeful research around the world that could lead to effective treatments and a cure for ALS.

Since the ALS Ice Bucket Challenge soaked the world almost five years ago, we have tripled our annual research budget.

More than $108 million has been committed toward our mission, including over $89 million that has been committed to worldwide research collaborations, since 2014. In 2018 alone, we awarded more than $16 million ($16,079,742) in funding to support 47 new research projects in nine countries*.

To see a complete list of the research we have funded since the ALS Ice Bucket Challenge, click here. For a list of all the grants we fund, click here.

Continue reading A Look Back at Over $16 Million in Research Grants Awarded During 2018

Collaboration and Hope in Glasgow: A Look Back at the Annual ALS/MND Meetings and Research Symposium

We had the pleasure of joining ALS scientists, clinicians, health care professionals, industry and advocacy partners, and, most importantly, people with ALS and their families at the annual events hosted by the International Alliance of ALS/MND Associations and the MND Association last month. It’s always a great opportunity to come together and learn from each other.

Here are some highlights from the three events – the Annual Alliance Meeting, the Allied Professionals Forum, and the International Symposium on ALS/MND – which were held in Glasgow, Scotland (UK), December 4-9.

Continue reading Collaboration and Hope in Glasgow: A Look Back at the Annual ALS/MND Meetings and Research Symposium

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

How The ALS Association Speeds Innovation by Globally Funding ALS Research

Innovation plays a key role in The ALS Association’s fight to develop treatments and a cure for ALS and to empower people living with the disease to live their lives to the fullest. Throughout June and July, we have celebrated some of the key innovations helping us change the nature of ALS forever.

Research funded by The ALS Association helped develop the first mouse model that specifically expresses poly(GR), a type of dipeptide repeat protein associated with C9orf72, which uncovered a new ALS disease pathway. Dr. Leonard Petrucelli’s group at the Mayo Clinic in Jacksonville, Florida, recently published the research in Nature Medicine.

Continue reading How The ALS Association Speeds Innovation by Globally Funding ALS Research