First person with MS gets ‘off-the-shelf’ CAR T-cell therapy
August 11,2025
A person with multiple sclerosis (MS) has for the first time been treated with an “off-the-shelf” CAR T-cell therapy called azercabtagene zapreleucel (azer-cel), a donor-derived approach that’s never before been tested in the disease.
The experimental treatment was given at Nebraska Medicine’s Fred & Pamela Buffett Cancer Center as part of a U.S.-based Phase 1 clinical trial (NCT06680037) that’s enrolling up to 32 adults with progressive forms of MS. The study is testing if genetically engineered T-cells from healthy donors can safely and effectively target the rogue immune cells that drive inflammation and damage in MS.
“This is a completely novel concept in MS therapy,” Rana Zabad, MD, a neurologist and MS specialist at Nebraska Medicine, said in a university news story. Zabad is leading the trial alongside CAR T-cell therapy expert Matt Lunning.
CAR T-cell therapy is a type of immunotherapy wherein immune T-cells are genetically modified to carry a man-made receptor — called a chimeric antigen receptor, or CAR — on their surface, which enables them to target a specific protein. The cells are then expanded into the millions and infused into the patient to recognize and destroy the harmful cells. Traditional CAR T-cell therapies use a patient’s own T-cells, but the manufacturing process often takes several weeks and researchers aren’t always able to collect enough cells from a patient to create the treatment.
TG Therapeutics‘ azer-cel contains T-cells from healthy donors. Termed allogeneic, or donor-derived, CAR T-cell therapy, the approach allows for the production of an “off-the-shelf” treatment that could be used in numerous patients.
“We’ve never used allogeneic CAR T-cell therapy to target the immune cells responsible for driving inflammation and damage in the central nervous system. This trial is about pushing boundaries carefully and thoughtfully to explore what could be possible,” Zabad said.
CAR T-cell hittar nya svårbotade sjukdomar att behandla. Här handlar det om donerade T-celler från friska individer som modulerats och sen injicerats till en MS-patient,vilket gör det extra intressant.
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