A team of researchers from the Wyss Institute at Harvard University developed a promising new treatment for a severe genetic disorder, powered by artificial intelligence. The breakthrough utilizes an AI-driven drug discovery process with innovative disease modeling to identify a potential therapy for Rett syndrome, a disorder primarily affecting girls.

Researchers identified vorinostat, a medicine used to manage cutaneous T cell lymphoma, as a potential therapy.

AI Battles Rett Syndrome

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Rett syndrome affects approximately 1 in 10,000 girls globally. Mutations in the MeCP2 gene cause the disease and are historically viewed as a neurological disorder. However, it also impacts non-neurological systems, such as the digestive, musculoskeletal, and immune systems. This makes it difficult to develop a treatment.

The Wyss Institute team’s study was published in Communications Medicine.

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Researchers discovered that vorinostat showed promising disease-modifying abilities across neuronal and non-neuronal tissues. In preclinical models, it was “superior” to trofinetide, the only current approved treatment.

A key was the Wyss Institute’s computational algorithm, nemoCAD. The AI approach analyzes changes across gene networks across multiple organ systems to predict drug candidates. Traditionally, drug prediction targets a single molecule. Wyss Institute’s “target-agnostic” method rapidly and effectively identified potential therapies.

Researchers used genetically engineered Xenopus laevis tadpoles that replicated Rett syndrome features to model the disease. The team compared gene expression changes in the modified tadpoles to healthy ones. NemoCAD then predicted drugs to reverse the pathological changes. Finally, vorinostat emerged as a top candidate. According to the researchers, it significantly suppressed symptoms such as seizures, unusual movements, and gastrointestinal issues in the modified tadpole models.

“The identification and further development of vorinostat as the potentially first curative treatment for Rett syndrome would not have been possible without our unique AI-enabled computational approach to drug discovery, and its combination with an innovative disease model that broadly mimics the features of Rett syndrome,” said Donald Ingber, M.D., Ph.D., senior author and Wyss Founding Director.

Vorinostat is already FDA-approved for treating a blood disease. Unravel Biosciences, a Wyss-enabled startup, is now repurposing it for Rett Syndrome.