The Challenge of Finding a Cure for ALS
Over the more than 150-year period since ALS was first described, little progress was made due to our lack of knowledge of the nervous system and of the disease itself. In the last two decades, a veritable explosion of promising basic science took place, including the discovery of new ALS genes and new candidate therapeutic targets.
Yet despite these discoveries, ALS remained an incurable disease.
Why? A series of mutually-reinforcing barriers were standing in the way, including:
Gaps in Funding: Few funding opportunities supported the translation of basic science ideas into validated drug targets and treatments.
Siloed Research: ALS researchers were operating in traditional silos, competing with each other rather than collaborating.
Lack of Scientific Resources : Researcher lacked access to the tools and resources necessary to advance promising ideas.
Limited Industry Investment: Given the limitations of translating ideas into the clinic, industry saw ALS as a high-risk area.
Bias in Funding Decisions: ALS research funding relied on a peer review process with inherent conflicts of interest.
Intellectual Property Restrictions: ALS scientists needed access to data and resources with no strings attached.
A New Way to Accelerate Progress
The world’s greatest scientific achievements are accomplished when people choose to work together.
These collaborations change lives and change the way we live them. In many cases, it has translated into groundbreaking medical research and lifesaving treatments.
As champions for the power of collaboration, we created our unique Innovation Ecosystem. Over the last ten years, our approach has dramatically accelerated research results leading to the development of over a dozen therapeutic approaches for ALS.
The world’s greatest scientific achievements are accomplished when people choose to work together.
The Innovation Ecosystem
These collaborations change lives and change the way we live them. In many cases, it has translated into groundbreaking medical research and lifesaving treatments.
Collaborative Funding
Target ALS fosters collaboration by exclusively funding multi-investigator research consortia that pursue novel approaches for ALS drug discovery and development.
Scientific Tools and Resources
ALS researchers worldwide have access to a range of critical tools ranging from biospecimens to genomic datasets to stem cells with no strings attached.
A Fairer Grant-Making Process
All ALS funding decisions are reviewed and ultimately decided by a conflict-free Independent Review Committee.
Engagement Between Academia and Industry
Target ALS offers the pharma/biotech industry access to its funding and resources to promote collaborative research projects across academia and industry and to seed ALS drug discovery programs in the industry.
No Intellectual Property Restrictions
Target ALS does not place any intellectual property (IP) restrictions on data generated by its funded research projects or scientific tools and resources, helping to facilitate the organization’s collaborative approach.
High-Impact Networking and Communication
Target ALS fosters ongoing networking and communication between scientists in academia and the pharmaceutical and biotech industries, among other stakeholders. This includes an annual summit in Cambridge, MA attended by 300+ scientists, foundations, venture capitalists, industry leaders, and donors.
Our Seven Pillar Strategy
We have developed a bold Seven Pillar Strategy to expand both the breadth and depth of our Innovation Ecosystem, fueling scientific breakthroughs to develop effective biomarkers and treatments for ALS.
Pillar 1: Diversification and Expansion of Talent
To pave the path for future breakthroughs, we encourage ideas on ALS research from the next generation of scientists. We provide support to researchers at different stages of their careers through our grant making programs to encourage long-term interest in ALS.
Pillar 2: Maintenance and Expansion of Scientific Core Facilities
Our eight Scientific Core Facilities democratize ALS research by providing expedited no-strings-attached access to biosamples, datasets, and reagents for scientists worldwide to carry out their research. Over 1,100 ALS projects have already benefited from use of these resources worldwide.
Pillar 3: Applying the Target ALS Model to Related Diseases
ALS and neurodegenerative diseases like Frontotemporal Dementia, Alzheimer’s Disease, and others share common genetic causes and underlying disease biology. Supporting research in related diseases expands the field of experts working on ALS and opens new avenues of study.
Pillar 4: Discovery and Development of New Therapeutic Targets
By fostering collaboration between academic scientists with deep understanding of disease biology and industry partners with drug discovery expertise, our approach both expands the pipeline of potential treatments and accelerates their trajectory towards the clinic.
Pillar 5: Discovery and Development of Toolkit of Biomarkers
Early diagnosis, disease tracking, and treatment evaluation all depend on one critical element: biomarkers. Biomarkers are characteristics of disease that can be accurately measured and reproduced. Reliable biomarkers will allow for more efficient clinical trials.
Pillar 6: Treatment of Ultra-Rare Forms of ALS
Target ALS accelerates research for all forms of ALS, including the ultra-rare genetic forms that only affect a few people worldwide. Cases of ultra-rare ALS offer specific disease-causing targets primed to test new technologies that could have broader applications in ALS and beyond.
Pillar 7: Prudent Growth of Target ALS Capacity
Target ALS is not simply supporting ALS research; we are driving it. We have welcomed new members to our staff and leadership, expanding the breadth of expertise across fields to scale up our efforts. We are building the team that will defeat ALS.
Join Us to Change the Prognosis for Patients
For the first time in the 150+ year history of this disease, we face a historic opportunity to radically change the prognosis for patients.
With your support, Target ALS can expand the Innovation Ecosystem, further advancing the development of new therapies that can be translated rapidly into the clinic and aid in the discovery of much-needed ALS Biomarkers.
Together, we can achieve historic breakthroughs and change the prognosis for patients.
