Experimental CAR-T Therapy Puts 19-Year-Old With Rare Vasculitis Into Remission

A 19-year-old from the Lower Galilee spent nearly a year in worsening health before doctors at Rambam Medical Center identified a rare autoimmune disease, ANCA vasculitis, and treated him with experimental CAR-T therapy. His symptoms began in high school, with mouth sores, swelling, nosebleeds, coughing up blood, severe headaches, infections and sleepless nights. Over months, doctors first suspected a rare malignant disease, then advanced lymphoma, before the real diagnosis emerged.

According to Dr. Yonatan Botbol, head of pediatric rheumatology at Rambam, the illness had already severely damaged the young man’s lungs and later caused myocarditis and reduced heart function. He was treated with steroids, then tried a biologic drug and later chemotherapy after an allergic reaction and worsening bleeding. Doctors then considered cyclophosphamide, but because of his age and the risks, they instead proposed CAR-T, a treatment usually used for blood cancers.

The patient became the first person in Israel and the third in the world to receive CAR-T for ANCA vasculitis. The treatment was part of a clinical study in adult care at Rambam under Prof. Yolanda Braun. Dr. Ofrat Biar-Katz said the therapy works as a kind of reset for the immune system by removing the B cells that produce the harmful antibodies driving the disease. After collection and genetic modification of his T cells, he received lymphodepleting chemotherapy and then the infusion, with close monitoring for cytokine release syndrome and neurological side effects.

The response was rapid. Within about a week, he stopped taking the drugs he had depended on, and the bleeding, bloody cough, mouth sores and other symptoms disappeared. “I feel better in every way,” he said, adding that his face returned to normal and he can sleep again. Botbol said he is now in complete response and on no treatment, though his white blood cell count remains low and long-term durability is unknown. The patient, who completed his Bagrut exams despite repeated hospital stays, says he may take a year off and later study medicine, possibly rheumatology.

Rambam is also expanding home monitoring for CAR-T patients. Under a new model, selected patients can complete part of the post-treatment observation at home if they live within about an hour of the hospital, meet strict medical criteria and have a family member present. The hospital has already completed its first home-based CAR-T follow-up for a hematology patient, with two more expected.