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Cures for many rare diseases might already exist. Why aren’t we using them?

July 5, 2021, 1:00 PM UTC
Discontinued medicines should be repurposed to treat rare diseases like cystic fibrosis and muscular dystrophy.
Thana Prasongsin—Getty Images

Scientists may have already developed cures for cystic fibrosis, muscular dystrophy, and rare cancers—they just don’t realize it yet. 

Most people assume that experimental treatments either conclusively fail clinical trials or earn FDA approval. But there are a vast number of medicines whose fate lies somewhere in between. 

Some experimental medicines, for instance, show initial promise in clinical trials but are discontinued by drug companies for strategic or financial reasons. Once these drugs are abandoned, the research data is locked away, and the projects are largely forgotten. But with minor tweaks, some of those discarded drugs could be turned into game-changing therapies for currently untreatable rare diseases. 

It’s not uncommon for drugs developed for one condition to effectively treat a different illness. The antiviral drug amantadine, for instance, was originally developed for influenza, but was repurposed as a Parkinson’s therapy. Right now, the anti-inflammatory colchicine—long given to patients with gout—is being investigated as a COVID-19 treatment. 

Deciphering which discontinued medicines ought to be repurposed or repositioned isn’t easy, of course. Piecing together the relevant data can be challenging, since the original research teams have usually long since disbanded. Pharmaceutical firms are also generally reluctant to spend tens, or even hundreds, of millions of dollars investigating whether a discontinued medicine could treat rare diseases, since the market for these treatments is typically small and running trials on a low number of available rare diseases patients can be challenging.

But given the potential for these abandoned drugs to save lives, the obstacles are well worth overcoming. A number of companies and nonprofits are trying to do just that.

Take the biotech startup SpringWorks Therapeutics. The company spun off from Pfizer in 2017 for the specific purpose of developing compounds that were gathering dust on Pfizer’s shelves. SpringWorks was able to create a financial incentive for Pfizer to revisit these compounds, in part by granting the firm a 10% stake in the venture. And right now, SpringWorks is at work on an array of drugs for a number of different rare tumors. (SpringWorks sponsors some Children’s Tumor Foundation events.)

The learnings from the SpringWorks example sparked the BRIDGE Initiative, aimed at speeding up the development of treatments for children with tumors. A collaboration between CureSearch for Children’s Cancer, FasterCures, and my organization, Children’s Tumor Foundation, seeks to find incentives and pathways for pharmaceutical and biotech firms to release their discontinued drugs or take a risk on repurposing them. 

One incentive we are exploring is the opportunity for firms to raise their environmental, social, and governance (ESG) scores by offering us the opportunity to repurpose or recycle their discontinued but potentially valuable medicines for neurofibromatosis (NF) and pediatric cancer. ESG is a metric for gauging a firm’s contribution to improving our society and protecting our planet. And it’s something that many firms are eager to boost. 

The BRIDGE Initiative has also had real success finding investors who are willing to finance the development and commercialization of abandoned treatments. 

Another example is the National Institutes for Health’s Therapeutics for Rare and Neglected Diseases (TRND) Repurposing Screening Group. The program partners with researchers, nonprofits, and pharmaceutical companies to identify drugs with the potential to treat rare diseases, and shepherd those medicines into clinical trials. 

It’s up to biotech companies, investors, and nonprofits to collaborate and transform more discontinued medicines into rare disease treatments. Giving new life to these discarded drugs could save patients’ lives. 

Annette Bakker is president of the Children’s Tumor Foundation.

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