Can a cancer therapy that transformed the treatment of blood malignancies also offer new possibilities for Alzheimer鈥檚 disease? More than three decades ago, Prof. Zelig Eshhar of the Weizmann Institute of Science, who passed away in the summer of 2025, laid the groundwork for a new type of cancer therapy based on genetically engineering a patient鈥檚 immune cells to target specific molecules in the body. Now, in a new study in the Proceedings of the National Academy of Sciences (PNAS), an international research team led jointly by scientists from the Weizmann Institute of Science and Washington University School of Medicine in St. Louis presents the first use of CAR-T in the treatment of Alzheimer鈥檚 disease. The therapy produced promising results in a mouse model of Alzheimer鈥檚, potentially paving the way for treating Alzheimer鈥檚 and other neurodegenerative disorders.
As the world鈥檚 population ages, neurodegenerative diseases 鈥� including Alzheimer鈥檚 鈥� are becoming an increasingly serious public-health challenge. The effectiveness of existing treatments, including those approved in recent years, has yet to be firmly established, and there is an urgent need for new therapeutic approaches. One of the central hallmarks of Alzheimer鈥檚 disease is the accumulation of amyloid-beta protein plaques in the brain, accompanied by signs of inflammation in brain tissue.
“”In future studies, we expect to demonstrate the use of engineered T cells also in enhancing recovery from severe brain damage and in promoting regeneration of brain tissue”
The research team headed by Prof. of Weizmann鈥檚 Systems Immunology Department and Prof. of WashU Medicine, and led by WashU Medicine postdoctoral fellow Dr. Pavle Boskovic, isolated T cells from the immune system of healthy mice and genetically engineered them to recognize and respond to amyloid proteins in the brain. The researchers then injected these engineered cells into mice whose brains already contained amyloid-beta plaques characteristic of Alzheimer鈥檚 disease. The injections led to a significant reduction in amyloid deposits, as well as a decrease in markers of brain-tissue inflammation.
鈥淲e report the first CAR-T cell approach for a neurodegenerative disease,鈥� said Kipnis, who earned his PhD from the Weizmann Institute. 鈥淚t represents an exciting step toward finding novel therapies for Alzheimer鈥檚 disease. Equally exciting is the prospect of adapting these versatile cells to deliver therapeutic agents for different neurodegenerative diseases beyond Alzheimer鈥檚, including amyotrophic lateral sclerosis (ALS) and Parkinson鈥檚 disease.鈥�
鈥淚n future studies, we expect to demonstrate the use of engineered T cells also in enhancing recovery from severe brain damage and in promoting regeneration of brain tissue,鈥� Amit reveals. 鈥淪uch future findings promise to bolster the notion that CAR-T technology can serve as a broad therapeutic platform for brain diseases 鈥� from cancerous tumors to stroke and chronic neurodegenerative diseases.鈥�
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The World Health Organization estimates that more than 55 million people (8.1 percent of women and 5.4 percent of men over age 65) are living with dementia. This number is estimated to rise to 78 million by 2030 and to 139 million by 2050.
Also taking part in these studies were Rotem Shalita and Maya Ben Yehuda, research students in the Amit lab.
Prof. Ido Amit鈥檚 research is supported by the Moross Integrated Cancer Center; the Elsie and Marvin Dekelboum Family Foundation; the Lotte and John Hecht Memorial Foundation; and Daniel Andreae.
Prof. Amit is the incumbent of the Eden and Steven Romick Professorial Chair.