illi News - Cures Within Reach Selected Three Clinical Trials in Rare/Ultra-Rare Diseases to Validate AI Predictions

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These clinical trials highlight opportunities that AI models offer the rare/ultra-rare community

CHICAGO - illiNews -- Cures Within Reach (CWR), a global nonprofit that identifies and funds clinical trials testing approved therapies for unsolved diseases, recently selected for funding six clinical trials to validate AI model drug-disease predictions, including three impacting rare and ultra-rare diseases. The selected trials offer the opportunity for scientists to unlock the power of AI validation to transform rare disease research by rapidly identifying repurposed treatment opportunities, cutting through years of traditional trial-and-error and delivering hope to patients who have long had none.

"CWR is committed to addressing the fundamental problem of getting treatment options to patients faster, and AI validation represents a pivotal step in turning powerful computational predictions into real treatments for patients," said Barbara Goodman, president and CEO of Cures Within Reach. "By supporting early-stage trials, we are bridging the gap in the development and implementation of AI tools, transforming in silico discoveries into in vivo clinical validation to accelerate access to therapies, especially for rare diseases where time and options are limited. This approach not only has the potential to rapidly create opportunities for rare disease patients in need, but also builds the clinical data needed to continuously strengthen AI models, unlocking a faster, more scalable future for how we discover and deliver treatments."

The three clinical trials starting later this year in rare/ultra-rare diseases funded by CWR that will validate AI models' predictions are:

Using a Diabetic Drug to Restore Awareness to Patients with Reduced Consciousness at Casa Colina Hospital
Repurposing a Seizure Treatment for DeSanto-Shinawi Syndrome at Mayo Clinic
Treating an Ultra-Rare Pediatric Disease (CLIFAHDD) with an Anti-Nausea Drug at University of Texas Health Science Center at San Antonio

With support from lead partner Biohub and additional support from Lyda Hill Philanthropies and the Searle Funds at The Chicago Community Trust, the six trials were selected for funding after a disciplined process in order to validate AI model predictions that accelerate drug development especially for rare diseases.

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"CLIFAHDD is a devastating, ultra-rare disease with no treatments and a high risk of early childhood mortality. Identifying existing and available drugs that have the potential to meaningfully help patients, including applying new AI models to help, is the fastest path to treatments for children and adults with CLIFAHDD," said Dr. Jeremy Tanner, a founder of Channeling Hope, parent to a daughter with CLIFAHDD, and neurologist and Assistant Professor at the University of Texas Health Science Center at San Antonio. "By repurposing a drug with a well-known safety profile, we're able to move directly into clinical testing for the first therapies in a field that typically moves far too slowly for affected children, and for ultra-rare diseases that most pharmaceutical companies are not interested in. Securing funding for this study is a crucial milestone which allows us to translate years of promising science into real-world impact. As a father of a four-year-old daughter with CLIFAHDD, this effort represents more than just research. It's our shared hope of helping our children live to their highest potential - to be able to walk, to be able to speak, and to be able to enjoy each day to the fullest."

"The Biomedical Data Translator ("Translator") is a knowledge graph-based software system designed to help researchers find connections across hundreds of publicly-available data sources from many different subfields of the biomedical sciences, enabling users to surface new research hypotheses and find the 'knowable unknowns' in the underlying data," said Tyler Beck, Program Director at the National Center for Advancing Translational Sciences (NCATS). "Translator was used by Mayo Clinic, a Cures Within Reach awardee, to identify drug repurposing opportunities in rare diseases that have the potential to make a difference in the lives of those who otherwise may never find a treatment, supporting NCATS' mission to bring more treatments to all people, more quickly."

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"Artificial intelligence for drug repurposing is giving us a powerful way to identify overlooked therapies by uncovering connections humans alone might miss, building on medicines with known safety profiles," said Nicole Small, CEO of Lyda Hill Philanthropies and LH Capital, Inc. "Funding these trials gives the opportunity to validate an entirely new model for how we discover treatments, rather than advancing a single therapy. We believe that philanthropy plays a unique role in this early risk reduction by creating opportunities where traditional funding often falls short. If successful, this approach has the potential to fundamentally reshape clinical research, making it faster, more efficient and more equitable for the millions of patients still waiting for answers."

CWR will open its next Request for Proposals to validate AI model drug-disease predictions in April 2026 and will select more AI validation trials by late 2026. CWR encourages anyone interested in clinically validating AI models and/or impacting rare diseases to reach out.

About Cures Within Reach
Cures Within Reach (CWR) is a US-based nonprofit leader testing already approved therapies for unsolved diseases by leveraging the speed, safety and cost-effectiveness of repurposing: driving more treatments to more patients more quickly. CWR provides seed funds for pivotal studies that, when successful, allow a catalytic effect of follow-on funding for trials that build clinical evidence for physicians to make decisions with their patients (called off-label use) or for regulatory approval. CWR's 2026 initiatives include validating AI drug-disease predictions and clinical trials impacting veterans, pediatrics and LMIC-based patients. CWR currently has a global portfolio of 50 funded trials at 40 institutions in 33 diseases in 12 countries. Visit cureswithinreach.org.