AI Validation

Cures Within Reach addresses the fundamental problem of getting treatment options to patients faster. Our AI Validation efforts are designed to activate the emerging potential of AI technologies to identify new disease-drug repurposing combinations, validate the clinical relevance and accelerate the timeline to get these into the hands of patients in need. While there may come a day when clinical trials may be conducted entirely in silico, we are not there yet. Even reaching that vision requires amassing real-world evidence that AI-generated drug-disease matches are safe and clinically relevant – which requires clinical trials in real humans.

Realizing the potential of AI models’ hypotheses of approved drug – disease matches requires addressing three critical challenges:

1. Validation Gap: How do we know if an AI tool’s predictions are clinically valid and for which specific patient populations? While AI holds great promise for identifying new treatment options, we must validate these AI-predicted drug-disease matches in human clinical trials.
2. Knowledge Bridge: There is insufficient cross-talk between physician scientists and AI model developers to fully realize and generate more repurposing opportunities. The expertise needed to develop AI models rarely overlaps with clinical expertise.
3. Implementation Bottleneck: Even when AI identifies promising drug-disease matches, getting these combinations tested in clinical trials is a bottleneck. Without funding and infrastructure to support these trials, potentially life-changing treatments remain theoretical.

Our AI Validation efforts a) directly addresses these challenges by creating the infrastructure to move promising AI-identified repurposing opportunities from virtual to reality, and b) are designed to address these challenges while helping to feed more clinically validated data back into the AI models themselves.

We serve as a critical bridge in the translational infrastructure supporting the development, validation and implementation of AI drug repurposing tools. We help transform promising in silico predictions into in vivo clinical validation, breaking down silos between AI developers and clinical researchers to accelerate patient access to treatments.

On February 25, 2026, we announced the 6 AI Validation trials selected – read more details here. These 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
• Improving River Blindness Outcomes in Cameroon at University of Buea
• Repurposing a Lipid Lowering Drug to Address Lung Cancer at University of Illinois at Chicago
• Reducing Pediatric Schistosomiasis Infections with a Malaria Drug in Mali at University of Science Technology and Engineering of Bamako
• Treating an Ultra-Rare Pediatric Disease (CLIFAHDD) with an Anti-Nausea Drug at University of Texas Health Science Center at San Antonio

Funding Opportunity RFPs for AI Validation:

• Clinical Trials to Validate AI-Driven Drug Repurposing 2026: Coming soon.
• Clinical Repurposing Research to Impact Veterans/Military 2026: accepting budgets of up to $75,000 to a U.S.-based institution for a repurposing clinical trial in any disease area impacting veterans or active military, such as mental health, PTSD and brain injuries. LOI SUBMISSIONS DUE MAY 29, 2026. 
• Repurposing Research for Chicago 2026: accepting budgets of up to $125,000 for clinical repurposing trials from Chicago area research institutions in one or both of the tracks listed below. LOI SUBMISSIONS DUE MAY 8, 2026. 
  • Track 1 – Investigator Groups: clinical repurposing trials in any unsolved disease led by an Early Stage Investigator or an underrepresented researcher.
  • Track 2 – Patient Groups: clinical repurposing trials impacting specific patient groups, such as health disparities, pediatrics, rare diseases and/or veterans issues (including mental health, PTSD and brain injuries).

• Clinical Trials to Validate AI-Driven Drug Repurposing 2025: now accepting submissions of investigator-initiated, proof of concept, Phase I or Phase IIA clinical trials to validate artificial intelligence (AI)-driven repurposing opportunities in any unsolved disease, where the therapies supported by AI models are already approved. Eligible clinical repurposing trials must include AI-generated data as part of the preclinical support for the trial. Budget: up to $100,000, including a 10% institutional match, with additional funds available for community engagement. FUNDING DECISIONS MADE
  • Any unsolved disease is eligible, although there is a preference for rare diseases.
  • Both on- and off-patent drugs and biologics are eligible, although there is a preference for off-patent.
  • Data from any AI model is eligible, although there is a preference for openly accessible models and for models developed by nonprofits/government.

As we grow our efforts in AI Validation, we thank our lead funding partner:

This project is funded in part by: