Target ALS raises $250 million to accelerate ALS research

Funding will be applied toward research into biomarkers, ALS therapies

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by Steve Bryson, PhD |

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Target ALS has reached its capital campaign goal of $250 million to accelerate research and fuel scientific breakthroughs in amyotrophic lateral sclerosis (ALS).

The campaign was spearheaded by founder Dan Doctoroff, a former deputy New York City mayor who was diagnosed with ALS in late 2021. He was committed to raising the funds to increase investment in ALS by leveraging his deep relationships and fundraising talents.

“This is deeply personal for me given my family history; my dad and my uncle died from ALS, but my mission is to save the one in 400 people who will die of this disease,” Doctoroff said in a Target ALS press release. “Over the past 11 years, we have developed a unique collaborative model to advance ALS research, and the $250 million – and I’m not stopping until I can’t do it anymore – will advance discovery dramatically.”

Doctoroff and his team at Target ALS, a 501(c)(3) medical research foundation, set out to raise awareness and funds for ALS research and put together a comprehensive ALS research investment strategy with the help of specialists in ALS and other neurodegenerative diseases, along with those who’ve been affected by the disease. The strategy also incorporates the latest in scientific research and drug discovery.

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7 Pillars of research success

Over the next eight years, the $250 million will be invested across Target ALS’ Seven Pillars to fuel scientific breakthroughs and drive the development of biomarkers and ALS therapies.

In Pillar 1, called Diversification and Expansion of Talent, fresh perspectives and new technologies will be attracted to advance ALS research led by the next generation of researchers and clinicians. Through grant-making programs, Target ALS will support researchers at different stages of their careers to encourage long-term interest in ALS.

In Pillar 2, called Maintenance and Expansion of Scientific Core Facilities, ALS research  will be democratize by granting swift, unrestricted access to biosamples, datasets, and reagents for scientists worldwide. So far, more than 1,100 ALS projects have used these resources.

In Pillar 3, called Applying the Target ALS Model to Related Diseases, the focus will be placed on related diseases with shared genetic roots and underlying disease mechanisms, such as frontotemporal dementia and Alzheimer’s disease, to broaden the field of experts working on ALS and find new research paths.

In Pillar 4, called Discovery and Development of New Therapeutic Targets, collaborations will be fostered between academic scientists specializing in disease biology and industry partners with expertise in drug discovery. The goal is to expand the pipeline of potential therapies and speed their path toward clinical application.

In Pillar 5, called Discovery and Development of Toolkit of Biomarkers, new biomarkers will be found to aid in shortening the time to diagnosis, monitoring disease progression, and assessing treatment efficacy with accurate biomarkers, which will also improve the efficiency of clinical trials.

In Pillar 6, called Treatment of Ultra-Rare Forms of ALS, the focus will be on investigating ultra-rare ALS variants to uncover unique disease-related targets toward broader applications in ALS and other diseases.

In Pillar 7, called Prudent Growth of Target ALS Capacity, the aim will be to expand Target ALS’ team and leadership to strengthen its expertise across different fields and increase its capacity for further work supporting ALS research.

“The true achievement is not the fundraising itself, but what we will be able to accomplish with the resources entrusted to us,” said Manish Raisinghani, PhD, president and CEO of Target ALS. “We take this opportunity as a responsibility to help build a world where everyone with ALS lives, and we won’t stop until we realize this vision.”