October 2025

How AI-Driven Drug Repurposing is Helping Treat Complex Diseases  

Most medications are approved for a single purpose. For example, a drug is licensed to treat breast cancer. But what if that same drug could also help with multiple sclerosis, or another unrelated disease?


This approach is called drug repurposing, and it’s one of the most promising frontiers in medicine today.


One of its pioneers is Dr. David Fajgenbaum, a physician who turned his own battle with a rare disease into a groundbreaking medical discovery.

Dr. David Fajgenbaum’s Story: From Patient to Pioneer


At just 25 years old, Dr. David Fajgenbaum nearly died four times in three years from Castleman Disease, a rare disorder that causes the immune system to attack vital organs. On his fourth relapse, he was administered last rites.


Fortunately, chemotherapy did offer a temporary reprieve, but Dr. Fajgenbaum knew it was not a sustainable solution.


He began to search for an answer, using himself as the research subject. He scrutinized thousands of pages of his own medical records and collected and analyzed his blood samples for clues.


Dr. Fajgenbaum’s research pointed to an overactive immune pathway (mTOR). With his doctor’s support, he repurposed an existing drug, Sirolimus, to treat himself. Sirolimus was originally developed and approved as an immunosuppressant for organ transplant patients. Repurposing this drug saved his life.


In 2022, building on this breakthrough, Dr. Fajgenbaum founded Every Cure, a nonprofit dedicated to uncovering hidden uses for existing drugs.



Researchers at Every Cure use an AI platform called MATRIX to compare thousands of drugs against thousands of diseases, generating millions of possible treatment matches.

Why drug repurposing can be a lifeline for those with difficult-to-treat or rare conditions


Existing drugs have already gone through rigorous testing for safety so they can often be redirected to new conditions at a faster rate, as well as at a fraction of the cost, of traditional drug development.


This approach is especially critical for rare diseases, where patient populations are too small to attract large pharmaceutical investment.


Some of today’s best-known therapies were discovered this way:

·       Sildenafil (Viagra) — first tested for blood pressure, later used for erectile dysfunction and pulmonary hypertension.

·       Thalidomide — once a failed pregnancy drug, now used against multiple myeloma.

·       Metformin — a diabetes drug now being studied for cancer prevention and healthy aging.


There are over 18,000 known diseases and 4,000 approved drugs for more than 72 million possible drug–disease combinations. 


AI tools like MATRIX can scan through massive datasets, prioritize the most promising matches, and flag drugs that are likely to help in diseases they weren’t originally designed for in record time.

RCM Health Consultancy: Accessing New Thinking for Complex Cases


MATRIX is making unprecedented advances in drug repurposing and giving new hope to people living with conditions that were once thought of as untreatable.


At RCM Health, we are proud to use the MATRIX AI tool when exploring novel treatment options for our clients who have complex medical challenges.


Just as Dr. Fajgenbaum’s discovery opened a path forward for Castleman’s disease, we apply the same spirit of innovation to find possibilities beyond the obvious.



Read about Dr. Fajgenbaum’s inspiring journey in TIME and the AAMC.

To learn more about RCM Health services and how we assist Clients with complex issues:


647-350-5500


info@rcmhealth.ca


Raymond Rupert  

CEO  

RCM Health Consultancy Inc.

www.rcmhealth.ca