In a study published in PNAS, T cells were engineered to recognize amyloid proteins in the brain; the injections reduced the extent of deposits and markers of brain inflammation—offering a new direction for degenerative brain diseases
Could a groundbreaking development that revolutionized blood cancer medicine also be good news for Alzheimer's treatment? More than three decades ago, Prof. Selig Ashchar of the Weizmann Institute of Science, who passed away a few months ago, laid the foundation for a new type of cancer treatment – genetically engineering the patient's immune system cells so that they act against a specific target in their body. Now, in a study published today in the scientific journal (PNAS) An international research team led by scientists from the Weizmann Institute of Science and Washington University in St. Louis presents the first use of a method developed by Prof. Ashhar, called CAR-T, to treat Alzheimer's. The findings in a mouse model of Alzheimer's showed promising results and may pave the way for future treatments for Alzheimer's and other degenerative brain diseases.
The world's population is aging, and degenerative brain diseases, especially Alzheimer's, are a growing health problem. The effectiveness of existing treatments, including recently approved new treatments, is still not sufficiently proven, and there is an urgent need for new treatments. One of the main hallmarks of Alzheimer's is the accumulation of amyloid-beta protein deposits in the brain, as well as markers of inflammation in the brain tissue. The research team led by Prof. Ido colleague From the Department of Systemic Immunology at the Weizmann Institute and Prof. Jonathan Kipnis From Washington University School of Medicine in St. Louis, led by postdoctoral fellow Dr. Pavel Boskowitz, they extracted immune system T cells from a healthy mouse and genetically engineered them to recognize and respond to amyloid proteins in the brain. The researchers injected the engineered cells into mice whose brains already had amyloid-beta deposits characteristic of Alzheimer's. The injections led to a significant reduction in the deposits as well as a decrease in markers of inflammation in the brain tissue.
"In future studies, we expect to demonstrate the use of engineered immune cells in rehabilitation from acute brain injuries and in promoting neural repair and regeneration."
"The study represents the first demonstration of feasibility for treating degenerative brain disease using the CAR-T method," says Prof. Kipnis, a Weizmann Institute graduate who currently heads a research group in the US. "This is an exciting step on the path to developing new treatments for Alzheimer's and other diseases, including ALS and Parkinson's."
"In future studies, we expect to demonstrate the use of engineered immune cells in rehabilitation from acute brain injuries and in promoting neural repair and regeneration," reveals Prof. Amit. "These future findings are expected to strengthen the concept that CAR-T technology can serve as a broad therapeutic platform for brain diseases – from cancer, to stroke, to chronic degenerative diseases."
Research students Rotem Shalita and Maya Ben-Yehuda from Prof. Amit's lab also participated in the studies.
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