Dear Every Cure community,

It’s been another exciting month at Every Cure as we push forward our mission to save and improve lives by repurposing drugs!

We’re so pleased to highlight two important publications that build upon and reinforce our mission. In one, we worked with partners to evaluate a promising treatment for a rare disorder called Rosai-Dorfman-Destombes disease, which is one of our 10 active drug repurposing programs. In the other, we introduced a new way to measure which diseases have the greatest unmet need, helping us to focus on the diseases where treatments can make the biggest difference.

Thank you for your continued support. We could not do this work without you.

David Fajgenbaum, MD, MBA, MSc 

Co-Founder & President, Every Cure

A new study published in the American Journal of Hematology highlights a promising repurposing opportunity for Rosai-Dorfman-Destombes disease (RDD), a rare and potentially life-threatening inflammatory condition with limited treatment options. Led by collaborators at Dalhousie University, Every Cure, and other institutions, the study found that a combination of two existing drugs, lenalidomide and dexamethasone (“len/dex”), may be an effective and well-tolerated treatment for patients with RDD. Every Cure contributed to this work by using our computational pharmacophenomics platform and medical expertise to help validate this promising treatment approach.

Researchers evaluated 11 patients with RDD who received len/dex over an average of 18 months, many of whom had already tried multiple treatments without success. The results were encouraging: the treatment was generally well tolerated, and 10 of 11 patients experienced clinical or radiographic improvement, including those who had not responded to prior therapies. 

In parallel, Every Cure applied its computational pharmacophenomics approach to assess nearly 3,000 FDA-approved drugs for RDD, identifying both lenalidomide and dexamethasone among the top candidates. This alignment between real-world outcomes and computational predictions highlights the potential of len/dex for RDD and underscores the power of pharmacophenomics to uncover additional impactful repurposing opportunities. Len/dex for RDD is one of Every Cure’s 10 active drug repurposing programs that we’re currently advancing forward while we continue to search for additional repurposing opportunities to advance.

Photograph from: Int J Ped Otorhinolaryngology. 44(2), 1998, 174. 

More information: https://everycure.org/rdd/ 

Full publication: https://onlinelibrary.wiley.com/doi/10.1002/ajh.70309 

Every Cure takes an “all vs. all” approach to drug repurposing, evaluating all existing drugs across all diseases to identify the most promising opportunities. Guided by our North Star of patient impact, we aim to advance treatments for conditions where the need is greatest and the potential to reduce suffering is highest. However, until recently, there has not been a systematic way to assess and compare diseases to determine where unmet medical need is greatest. 

A new study by Every Cure, with findings published in PLOS Medicine, introduces a scalable approach to quantifying unmet need across all 22,000+ diseases. Using large language models, we developed a framework based on 11 criteria, including disease severity, available treatments, and quality of life. This enables consistent, evidence-based comparisons across diseases, rather than relying on intuition.

These insights are now integrated into Every Cure’s pipeline, helping prioritize opportunities with the greatest unmet need and potential impact. We believe no patient should suffer while a treatment hides in plain sight, and this approach helps ensure we’re looking for treatments for diseases with the greatest suffering first.

With over 2,500 views in less than two months, we hope this tool will continue to be used by other medical researchers and funders to address diseases with the greatest unmet medical need.

Full publication: https://journals.plos.org/plosmedicine/article?id=10.1371/journal.pmed.1004798

Over the past month, Every Cure’s mission has been shared across leading news media, podcasts, and scientific convenings — helping bring drug repurposing into the mainstream.

Click the photos below for the full stories – particularly the in-depth feature in Men’s Health.