The ALS Association

ALS Ice Bucket Challenge Progress

The ALS Association to Present at BIO, World Biotechnology Convention

June 16, 2015

Washington, D.C. (June 16, 2015) — The ALS Association will once again participate in the annual BIO International Convention, which will this year be held June 15-18 in Philadelphia. More than 15,000 leaders from the biotech and pharmaceutical industries participate in the convention, providing The Association with key opportunities to identify and grow partnerships that advance the development of treatments for ALS.

ALS (amyotrophic lateral sclerosis) is a progressive neurodegenerative disease that affects neurons (nerve cells) in the brain and the spinal cord. Eventually, people with ALS lose the ability to initiate and control muscle movement, which often leads to total paralysis and death within two to five years of diagnosis. There is no cure and no life-prolonging treatments for the disease. 

During the convention, The Association will host an exhibit on the convention floor, deliver presentations to industry leaders, speak on panels during breakout sessions, and hold more than 20 meetings with individual companies to stimulate and strengthen industry partnerships and bring more companies into the field of ALS drug development.

Lucie Bruijn, Ph.D., M.B.A., Chief Scientist for The ALS Association, will present an overview of The Association’s research programs, its ability to fund development of promising new treatments, as well as its deep support for research on the causes of ALS. Dr. Bruijn will specifically highlight academic-industry partnerships and the drug development program. This program was key to the development of antisense technology for ALS, the first neurodegenerative disorder to benefit from this technology. It is now being applied to multiple neurodegenerative disorders and is in clinical trials for spinal muscular atrophy (SMA). This program has been greatly accelerated in 2015 by the unprecedented outpouring of financial support during the 2014 ALS Ice Bucket Challenge.

A key feature of The Association’s research program is the leveraging of expertise and ability through facilitating partnerships between academic researchers and biotechnology and pharmaceutical companies.

“Our approach is to catalyze treatment development through bringing together the best researchers with the companies that can accelerate the search for new therapies,” Dr. Bruijn said. “We will be continuing that work at the BIO meeting.”

In addition to Dr. Bruijn’ s presentation, Steve Gibson, the Chief Mission Strategy and Public Policy Officer for The ALS Association, will be a featured panelist during a breakout session focused on the role of patient advocates in shaping regulatory and science policy. Gibson will highlight the multiple ways people with ALS have been involved in affecting public policy, including The Association-sponsored “National ALS Advocacy Day,” during which people with ALS and family members meet with their elected representatives about the importance of ALS research, care, and funding for treatments.

Gibson will also share The Association’s work to advance patient-focused drug development and to ensure that patients are able to play an active role throughout the drug development process, including engaging both industry and the Food and Drug Administration (FDA).  As part of the panel discussion, he will present on The Association’s groundbreaking initiative to create a guidance document for ALS drug development that will be submitted to the FDA.

“This initiative will include the active participation of the entire ALS community,” said Gibson.  “Other ALS organizations are involved along with industry, clinicians and researchers, NIH and, most importantly, people with ALS and their families.”

The guidance will incorporate the ALS community’s views in areas such as trial design, biomarkers, surrogate endpoints, patient-reported outcomes, and benefit-risk, and will serve as a roadmap to help industry navigate the development process. “The goals are to make the drug development process, including clinical trials, more efficient, more predictable, faster, and more effective at assessing efficacy much earlier,” said Gibson.  “This will speed access, reduce costs, help ensure resources are most effectively utilized and incentivize industry to enter the ALS market and develop new treatments for ALS.”

In addition to The Association’s exhibit booth, Iron Horse Diagnostics, Inc., which has received funding from The Association, will host a booth featuring its development of a commercial biomarker for ALS diagnosis. The ALS Association recognizes the strategic importance of so-called biomarkers in diagnostics and the development of therapeutics in ALS and has provided key funding for advancing this diagnostic test toward the clinic. Further information on this effort can be found here:

More information on the BIO meeting is available here:

About The ALS Association
The ALS Association is the only national non-profit organization fighting Lou Gehrig’s Disease on every front.  By leading the way in global research, providing assistance for people with ALS through a nationwide network of chapters, coordinating multidisciplinary care through certified clinical care centers, and fostering government partnerships, The Association builds hope and enhances quality of life while aggressively searching for new treatments and a cure.  For more information about The ALS Association, visit our website at

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