Dear Every Cure community,

Thank you for your continued support of our mission. In addition to sharing important updates this month, we’re excited to announce that you can now indicate the specific diseases you’re interested in receiving targeted updates about. You can sign up here, and we’ll be in touch if and when we have updates on a repurposed treatment for that disease.

In this month’s Repurposing Roundup, we’re pleased to spotlight another Every Cure-led publication and tell Nioti’s inspiring story of how a repurposed drug transformed her life.

Thank you again for being part of this work.

David Fajgenbaum, MD, MBA, MSc 

Co-Founder & CEO, Every Cure

A new Every Cure-led publication in Psychiatry Research Communications highlights botulinum toxin type A (BoNT/A), best known as Botox, as a promising repurposing opportunity for major depressive disorder (MDD), a condition that affects hundreds of millions of people worldwide. While many patients benefit from existing antidepressants, a substantial number do not experience adequate relief from available treatments or cannot tolerate them, underscoring the need for new therapeutic options.

Using our AI platform and disease-agnostic approach, we identified Botox as a promising repurposing opportunity for MDD. After extensive review by our team and independent Scientific Advisory Council, it was selected as an Every Cure Repurposing Program for advancement. As part of this program, we conducted a rigorous systematic review and meta-analysis of existing clinical trials to evaluate the strength of the current evidence supporting Botox for MDD

The analysis identified placebo-controlled studies looking at glabellar Botox injections for more than 500 patients with depression. Across these studies, Botox showed meaningful improvements in depression symptoms compared to those who received a placebo, and the treatment was well tolerated, with no serious side effects reported. Several other studies suggested potential benefit in patients who had not responded to other treatments.

This publication reflects Every Cure’s broader approach: using computational methods to identify overlooked treatment opportunities and rigorously evaluating them against the best available evidence to help bring effective therapies to patients faster. Looking ahead, we will continue to advance this program, including exploring what additional evidence may be needed to support clinical adoption.

Read the full paper: https://www.sciencedirect.com/science/article/pii/S2772598726000164 

After years of searching for answers across multiple countries, Nioti was diagnosed with Rosai-Dorfman Disease (RDD), a rare inflammatory condition that was little known to her doctors. She struggled with painful lesions across her body and other symptoms so severe that she was even unable to attend her son’s wedding.

The family tried numerous treatments and consulted multiple specialists, but nothing helped — until they connected with Dr. Luke Chen. Dr. Chen introduced them to lenalidomide/dexamethasone (len/dex), a repurposed drug that showed promise for RDD, and helped them secure access to it. 

The results were transformative. Nioti’s lesions began to heal, her strength returned, and today she has regained her independence — walking on her own, cooking, and spending time with her family. 

Nioti’s story highlights the promise of lenalidomide for RDD, an active program here at Every Cure. A recent study published in the American Journal of Hematology, which included contributions from Dr. Chen, Every Cure, and other experts, demonstrated strong clinical results for len/dex in RDD patients. We look forward to continued collaboration with Dr. Chen and other experts as we advance this program to help more patients. 

See Nioti’s full story here

Read more about lenalidomide for RDD: https://everycure.org/rdd/ 

Over the past month, Every Cure’s mission has been shared and recognized by leading organizations and media outlets — helping bring drug repurposing into the mainstream.

Click the photos below for the full stories.