The ALS Association

ALS Ice Bucket Challenge Progress


The ALS Association:
Brian Frederick
(202) 464-8612

Elizabeth DeLuca
Sr. Director, Corporate Brand & Communications
(267) 468-4329


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

Washington, D.C. (August 16, 2018) — The ALS Association and Teva Pharmaceutical Industries Ltd. (“Teva”) announced today the recipients of the Teva CNS Target Identification Crowdsourcing Challenge awards for their outstanding proposals on novel ALS targets. The two awards will be granted to Philip C. Wong, Ph.D., and to Jonathan C. Grima, Ph.D., and Jeffrey D. Rothstein, M.D., Ph.D., all from Johns Hopkins University School of Medicine in Baltimore, Maryland.

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disorder that leads to death of neurons in the brain and spinal cord. There are only two drugs approved in the United States for treating ALS with limited effects, and new therapies that substantially prolong lifespan are urgently needed. An essential step toward developing new treatment avenues is to unravel the biological processes that go awry in this disorder and identify novel molecular targets for drug intervention.

To address the severe, unmet need for new therapies for disorders of the central nervous system, The ALS Association, the Huntington’s Disease Society of America (HDSA), and Teva launched The Teva CNS Target Identification Crowdsourcing Challenge to seek novel targets with therapeutic potential for treating neurodegenerative disease, pain, and migraine. The Challenge was launched in collaboration with InnoCentive, a global pioneer in innovation and crowdsourcing.

Two teams were selected by Teva and The ALS Association to each receive $10,000 for proposals of ALS targets that had been recently implicated in disease pathophysiology, and which were viewed as particularly interesting for exploration as therapeutic targets:

  • Philip C. Wong, Ph.D., of Johns Hopkins University School of Medicine: TDP-43 dependent splicing repression as a mechanism-based therapeutic target for ALS.
    • TDP-43 is an RNA binding protein that is found in neuronal cytoplasmic aggregates in more than 95 percent of ALS cases and is mutated in approximately 4 percent of familial ALS patients. Dr. Wong’s group has identified a role for TDP-43 in repressing splicing of cryptic exons – genomic regions flanked by splice sites, which are not normally incorporated into the protein coding mRNA (Ling et al., 2015). Loss of function of TDP-43 causes these cryptic exons to be included in the mRNA and disrupt expression of many proteins. Restoring this function of TDP-43 ameliorated the motor and survival defects in fly and mouse models that mimic the depletion of nuclear TDP-43 occurring in motor neurons of ALS patients.
  • Jonathan C. Grima, Ph.D., and Jeffrey D. Rothstein, M.D., Ph.D., of Johns Hopkins University School of Medicine: Targeting the nuclear pore in neurodegeneration.
    • Grima and Rothstein have previously proposed the Nuclear Pore Hypothesis of Neurodegeneration, and this has been the centerpiece of Grima’s graduate work at Johns Hopkins. This hypothesis explains that a defect in nuclear pore complex (NPC) and nucleocytoplasmic transport (NCT) function may in fact underlie the majority of neurodegenerative diseases, and what may distinguish one disease from another are the unique components of the NPC and NCT machinery that are affected. Initial studies (Zhang et al., 2015) identified components of the NPC as genetics modifiers of neurodegeneration in the most common genetic cause of ALS. Additional work from Grima, Rothstein, and others has demonstrated for the first time that defects in this pathway may extend beyond ALS to include other neurodegenerative diseases, such as Huntington’s disease (Grima et al., 2017) and Alzheimer’s disease (Eftekharzadeh et al., 2018). This proposal focused on targeting several of these key NPC proteins.

“This is a particularly exciting time in drug development for ALS, with many novel therapeutic approaches under investigation,” commented Lucie Bruijn, Ph.D., M.B.A., chief scientist for The ALS Association. “The ALS Association is pleased to recognize the two recipients of the Crowdsourcing Challenge, together with Teva, and look forward to the further development of these promising therapeutic targets.”

“We were enthusiastic about the level of interest in the program and number of submissions received,” said Steffen Nock, Ph.D., senior vice president, Specialty Research & Development, at Teva Global R&D. “We found several of the proposals to be particularly interesting for further investigation as therapeutic targets for ALS, and are glad to partner with The ALS Association in granting these prize awards.”

About The ALS Association
The ALS Association is the only national nonprofit 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 ALS 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

About Teva
Teva Pharmaceutical Industries Ltd. (NYSE and TASE: TEVA) is a global leader in generic medicines, with innovative treatments in select areas, including CNS, pain and respiratory. We deliver high-quality generic products and medicines in nearly every therapeutic area to address unmet patient needs. We have an established presence in generics, specialty, OTC and API, building on more than a century-old legacy, with a fully integrated R&D function, strong operational base and global infrastructure and scale. We strive to act in a socially and environmentally responsible way. Headquartered in Israel, with production and research facilities around the globe, we employ 45,000 professionals, committed to improving the lives of millions of patients. Learn more at

About InnoCentive
InnoCentive is the global pioneer in crowdsourced innovation, enabling organizations to solve their pressing problems through the power of the crowd. InnoCentive’s diverse global network of problem Solvers, proven Challenge Driven Innovation methodology, and purpose-built platform combine to fundamentally transform the economics of innovation and R&D through rapid solution delivery and the development of sustainable open innovation programs. Since 2001, leading commercial, government, and nonprofit organizations such as AstraZeneca, Ford, Thomson Reuters, NASA, Lumina Foundation, and Enel have been partnering with InnoCentive to solve problems and innovate faster, more cost effectively, and with less risk than ever before. For more information, visit

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