AI’s Role in Rare Disease Drug Repurposing With: Dr. David Fajgenbaum

Dr. David Fajgenbaum, Co-founder of Every Cure, explains how AI is accelerating the search for treatments for rare diseases through drug repurposing. By scanning all existing drugs across all diseases, AI dramatically increases the chances of finding matches for conditions that otherwise have no effective therapies. While any individual rare disease may have low odds of an existing treatment, the cumulative approach across all conditions reveals many untapped opportunities. Fajgenbaum emphasizes the moral obligation to connect patients with available therapies—especially when they already exist in local pharmacies—rather than forcing them to wait years for new drug development.

About the Guest

Dr. David Fajgenbaum is Co-founder of Every Cure and a physician-scientist focused on accelerating drug repurposing for rare and common diseases. Learn more at: https://en.wikipedia.org/wiki/David_Fajgenbaum

Notable Quote

"If a treatment for your disease exists at the local pharmacy, we have a moral obligation to find it."

Key Takeaways

  • AI can scan all drugs and diseases to identify repurposing opportunities.
  • Not all rare diseases have existing drug matches, but many do.
  • Finding available treatments can prevent unnecessary suffering.

Transcript Summary

How is AI transforming rare disease treatment?

AI helps identify potential repurposing opportunities by analyzing all existing drugs across all diseases. While any single rare disease may have low odds of an existing match, looking across all diseases increases the likelihood dramatically.

Why focus on repurposing?

If a treatment exists and is sitting in a local pharmacy, patients should have access to it immediately—waiting years for new drug development is unacceptable.

What’s the broader impact?

By systematically scanning for drug-disease matches, AI can uncover life-saving treatments faster and more cost-effectively.

About the Series

AI and Healthcare—with Mika Newton and Dr. Sanjay Juneja is an engaging interview series featuring world-renowned leaders shaping the intersection of artificial intelligence and medicine.

Dr. Sanjay Juneja, a hematologist and medical oncologist widely recognized as “TheOncDoc,” is a trailblazer in healthcare innovation and a rising authority on the transformative role of AI in medicine.

Mika Newton is an expert in healthcare data management, with a focus on data completeness and universality. Mika is on the editorial board of AI in Precision Oncology and is no stranger to bringing transformative technologies to market and fostering innovation.

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