--- title: "Research and Therapeutics FY26" description: "Recent research and therapeutic advances at Harvard Medical School." canonical: "/research-and-therapeutics-fy26" published: 2026-09-29 --- # Research and Therapeutics FY26 ## Frame ### Research and Therapeutics ### at Harvard Medical School ## Frame #### Transforming Medicine Through Curiosity-Driven Research For more than 240 years, Harvard Medical School researchers have pursued curiosity-driven research and translated discoveries into advances that have transformed medicine. Today, faculty across HMS and its clinical affiliates are investigating conditions including Alzheimer’s disease, bipolar disorder, deafness, and sickle cell disease, and developing new tools that leverage artificial intelligence and other cutting-edge approaches to accelerate discovery and improve diagnosis, treatment, and patient care. **Advancing AI** ## Frame ##### Advancing Medical AI for Real-World Clinical Settings “Everyone in the medical AI community needs to work together to ensure that it is being developed and implemented in a responsible way.” \- Marinka Zitnik Marinka Zitnik, PhD, is associate professor in the Department of Biomedical Informatics at Harvard Medical School, associate faculty at the Kempner Institute for the Study of Natural and Artificial Intelligence at Harvard University, associate member at the Broad Institute of MIT and Harvard, and affiliated faculty at the Harvard Data Science Initiative, a University-wide initiative that advances data-driven research, education, and collaboration across disciplines to address complex societal challenges. Marinka Zitnik, PhD ##### Bridging the Gap Between AI Models and Clinical Reality Medical AI systems can perform well on standardized tests, yet behave differently in hospitals and doctors’ offices. Marinka Zitnik, PhD, and her colleagues are developing ways to evaluate these systems in real-world clinical settings, helping clinicians identify errors and limitations before AI tools are used in patient care. Their work aims to make medical AI more reliable, safe, and effective. **Read more** **Bipolar** ## Frame ###### Pioneering Insights into Bipolar Disorder Pathogenesis “You can grow thousands and thousands of brain organoids from any one of us. If the blood comes from a patient with a disorder, then every single cell in that organoid carries the genome, and genetic risk, of that patient.” \- Paola Arlotta Paola Arlotta, PhD Paola Arlotta, PhD, is the Golub Family Professor of Stem Cell and Regenerative Biology at Harvard University, principal faculty member at the Harvard Stem Cell Institute, institute member at the Broad Institute of MIT and Harvard, and associate member of the Stanley Center for Psychiatric Research at the Broad Institute. Her research focuses on the development and engineering of the cerebral cortex, with an emphasis on understanding how diverse brain cell types are generated, integrated into functional neural circuits, and implicated in human neurodevelopmental disease. ##### Human Brain Organoids: Avatars for Bipolar Disorder Research Paola Arlotta, PhD, and her colleagues have developed human brain organoids that allow researchers to study bipolar disorder and test potential treatments without exposing patients to experimental drugs. Created from human cells, these “avatars” contain millions of brain cells and can reflect the genetic risk of the person whose cells were used to create them. The organoids are the only experimental model of the brain available to scientists today and give researchers a way to observe how bipolar disorder affects human brain cells and to assess the potential benefits and risks of new therapies. This approach could help advance treatments that are more precisely tailored to patients. **Read More** **Alzheimer's** ## Frame-T ###### Unlocking Alzheimer's: The Lithium Connection “Our method of going from the laboratory to the clinical trial has been validated many times. In this case, we are lucky to be able to move forward rapidly because of enlightened philanthropy.” – Bruce Yankner Bruce A. Yankner, MD, PhD Bruce A. Yankner, MD, PhD, is professor of genetics and neurology at Harvard Medical School, director of the Harvard Neurodegeneration Training Program, and co-director of the Paul F. Glenn Center for Biological Mechanisms of Aging. His research focuses on the pathogenic mechanisms underlying Alzheimer’s disease, Down syndrome, and Parkinson’s disease. ##### Lithium's Role in Preventing and Reversing Alzheimer's Dr. Yankner and his team showed that lithium occurs naturally in the brain and has the potential to prevent or even reverse Alzheimer’s disease—a major leap in understanding a disease that affects more than 50 million people worldwide and that has been frustratingly difficult to treat. They showed that amyloid—a misfolded protein fiber that is one of the hallmarks of Alzheimer’s—binds to and neutralizes lithium and that lithium levels can vary among older adults. These findings suggest that reduced availability of lithium in the brain could play a role in Alzheimer’s disease and point to new questions about whether restoring or preserving lithium’s activity might help prevent or treat the disease. **Read More** **Therapies** ## Frame ###### Translating Groundbreaking Research into Life-Changing Therapies ###### Unraveling Hereditary Deafness: David Corey's Discoveries ###### CRISPR's Breakthrough: Gene Therapy for Sickle Cell Disease Decades of research by Stuart H. Orkin, MD ’72, the David G. Nathan Distinguished Professor of Pediatrics at HMS, and his collaborators helped establish the scientific foundation for the first CRISPR-based gene therapy for sickle cell disease. Dr. Orkin and fellow sickle cell researcher Swee Lay Thein, DSc, were recognized with the 2026 Breakthrough Prize in Life Sciences for their contributions to the field. ###### Foundational Advances in Cancer Immunotherapy David Corey, PhD, the Bertarelli Professor of Translational Medical Science at Harvard Medical School, and his colleagues in the Corey lab are developing strategies to treat hereditary deafness. This year, they identified a tiny loop on TMC1—a protein essential for hearing—that helps control the channel’s sensitivity to sound. Their findings shed light on how hearing evolved, help explain how mutations in TMC1 cause deafness, and could guide the development of future gene therapies. Pathbreaking work in cancer immunotherapy by married researchers Arlene Sharpe, MD ’82, PhD ’81, Kolokotrones University Professor and chair of immunology at HMS, and Gordon Freeman, PhD ’79, professor of medicine at Dana-Farber and HMS, helped lay the foundation for a new class of lifesaving cancer treatments. In December, the University of Zurich honored them with the inaugural Gretener-Thürlemann Prize, recognizing outstanding researchers in medicine, chemistry, and physics for foundational and pioneering research of societal benefit. **Read More** **Watch Video** **Read More** **Read More** **MMSc** ## Frame ###### Master of Medical Sciences inTherapeutic Sciences The Master of Medical Sciences in Therapeutic Sciences program provides world-class training in early drug discovery and prepares students like Valerie Cavanaugh, Class of 2027, for careers in pharmaceutical companies, biotech firms, academic labs, and research institutes. **Inline Asset** Watch:Opening New Opportunities Through the HMS Master's Program in Therapeutic Sciences ## Frame **Make a Gift →** Office of Alumni Affairs and Development 25 Shattuck Street Boston, MA 02115 giving@hms.harvard.edu **Digital Accessibility** **Privacy Policy**