With TxGNN, Kempner researchers introduce an AI ‘Dr. House’ to find treatments for rare disease

In the medical drama “House,” which ran for eight seasons beginning in 2004, the titular Dr. House routinely solves mysteries involving rare diseases, drawing on a wealth of experience and reasoning skills to come up with unprecedented treatments. Now, researchers affiliated with the Kempner Institute at Harvard University have come up with an AI “Dr. House”: TxGNN, a graph foundation model that can predict if a drug might be able to help treat a rare disease, even if it has never before been tested on that disease. 

This work, led by Marinka Zitnik, Kempner Institute associate faculty member and assistant professor of biomedical informatics at Harvard Medical School, has now been published in Nature Medicine.

“These diseases remain medical mysteries, and the lack of treatments is a major problem for those affected,” says Zitnik. “AI holds the potential to guide experimental studies in biological labs, helping to solve these mysteries.”