Collaboration to Boost Rare Disease Clinical Trials

(Gerd Altmann, Pixabay)

12 May 2023. An organization seeking new uses for today’s drugs and a contract research company aim to increase the number of clinical trials for rare diseases testing already approved drugs. The collaboration joins Every Cure, a not-for-profit group in Philadelphia that promotes new uses for approved drugs on the market, with Worldwide Clinical Trials, a contract research organization or CRO in Research Triangle Park, North Carolina conducting clinical studies and analytical services.

Every Cure was formed in 2022 in a partnership with the Clinton Global Initiative to find new applications for drugs already approved by the Food and Drug Administration, particularly for rare diseases. The group says it’s developing an open-source system with large-scale databases and analytics based on artificial intelligence algorithms to predict the efficacy of some 3,000 approved drugs against an estimated 9,000 other diseases with as yet no approved treatment.

The organization cites the experience of its co-founder David Fajgenbaum, a physician-scientist diagnosed with the rare disorder Castelman’s disease while in medical school, but successfully treated with an inexpensive drug approved years earlier for preventing immune rejection by kidney transplant patients. The group says it seeks to break through antiquated industry practices, such as restrictive data silos that make it difficult to identify additional uses for current drugs, particularly those with generic versions at lower cost. Every Cure’s Roadmap project — short for Repurposing Of All Drugs, Mapping All Paths — identifies potential paths for repurposing current drugs to treat rare diseases.

Trials to test approved generics against rare diseases

Worldwide Clinical Trials conducts clinical studies at all phases under contract to sponsoring companies and organizations. The company says it can organize trials in a range of disease types, including rare diseases, as well as offer bio-analytical services and research from real-world data sources. Rare diseases are defined as disorders affecting fewer than 200,000 individuals in the U.S., or five in 10,000 people in Europe.

Under the new collaboration, Worldwide Clinical Trials is serving as the clinical study practice partner for Every Cure, but financial and intellectual property details of their agreement are not disclosed. Worldwide Clinical Trials is providing its clinical research expertise in rare diseases to organize trials of generic drugs already approved for other conditions identified by Every Cure’s analytics and algorithms. “The combination of Every Cure’s computational A.I. algorithm,” says Fajgenbaum in a Worldwide Clinical Trials statement, “with Worldwide’s clinical trial expertise will have a substantial impact on finding and advancing cures for years to come.”

“Every Cure’s method,” notes chief operating officer at Worldwide Clinical Trials Dave Bowser, “of mining medical records and real-world data to discover new uses for drugs that are already approved and available is commendable, and the Worldwide team is committed to helping scale these approaches to all drugs and all diseases.”

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