The treatment has shown huge promise in helping patients treat cancer<\/a>, although its effectiveness has mostly been limited to treating blood cancers, not solid tumors<\/a>.<\/p>\n But an inspirational real-life success story involving a three-year-old patient shows scientists are making big progress with CAR T cell therapy against solid tumors too \u2013 and with potentially life-saving results.<\/p>\n In a report published in The New England Journal of Medicine<\/a>, researchers document a case of complete regression of hepatoblastoma<\/a>, a form of malignant liver cancer<\/a>, in a 3-year-old boy, after CAR T cell treatment.<\/p>\n “This case demonstrates that a durable complete response in a chemotherapy-resistant solid tumor can be achieved entirely in the outpatient setting without systemic toxicity,” says<\/a> first author and pediatric oncologist David Steffin from the Baylor College of Medicine in Houston, Texas.<\/p>\n CT images showing the tumor shrinking after the treatment. (Steffin et al., NEJM, 2026)<\/p>\n When the boy was first diagnosed, he had a mass in the left lobe of his liver that measured 11.2 cm by 9.6 cm by 7.1 cm (4.4 \u00d7 3.8 \u00d7 2.8 inches).<\/p>\n The cancer had metastasized to his lungs, with indications that it had also spread to his bones.<\/p>\n The boy received three bouts of chemotherapy and had his primary liver tumor surgically removed, along with two operations to remove the cancer that had spread to his lungs.<\/p>\n Despite the interventions, the cancer rapidly returned, with a new tumor appearing in his lungs.<\/p>\n After the rapid recurrence, the boy was enrolled in the CARE study<\/a>, the first human trial of an experimental form of CAR T cell therapy.<\/p>\n The boy received two doses of the experimental GPC3-CAR treatment, made from his own T cells, but engineered to recognize glypican-3<\/a> (GPC3), a protein that is highly expressed in liver cancers.<\/p>\n The researchers also engineered the therapy to include genes that would express the immune proteins interleukin\u201315<\/a> and interleukin\u201321<\/a>, to boost the CAR T cells’ longevity and tumor-killing ability, based on previous evidence from preclinical models<\/a>.<\/p>\n The boy received two infusions of the GPC3-CAR treatment. After the first infusion, the patient showed a partial response, indicated by CT scans and falling levels of a protein called alpha-fetoprotein in his blood, which is indicative of tumor activity.<\/p>\n Eight weeks later he received a second dose of the therapy, and follow-up scans showed no remaining signs of the disease, apart from some residual scarring.<\/p>\n After 12 months, the boy was still disease-free \u2013 an incredible result and turnaround for the patient and his family, given the severity of his illness before his participation in the trial.<\/p>\n “This study provides evidence that these novel CAR T cells may be a safe and effective modality for hepatoblastoma and highlights the need for further assessment in patients with GPC3+ solid tumors,” says<\/a> co-author and pediatric oncologist Andras Heczey from Seattle Children’s Hospital and the University of Washington.<\/p>\n Related: Experimental Drug Helped Cancer Patients Live 40% Longer in Clinical Trial<\/a><\/p>\n As the researchers note, liver cancer is the third leading cause of cancer-related death globally<\/a>, so making progress in this branch of immunotherapy treatments is hugely promising \u2013 especially since this experimental CAR T cell therapy produced a remission in a patient with a solid tumor.<\/p>\n Of course, it’s worth pointing out that this is just a single report of one patient in an early-phase trial, and we’ll need to see how other patients respond to the GPC3-CAR treatment before researchers know how effective and safe it is.<\/p>\n Continued testing is being carried out in the CARE study (at Baylor College of Medicine) and in a similar clinical trial<\/a> called IMPACT<\/a>, being run through Seattle Children’s Hospital.<\/p>\n The hope is that, with time, we’ll see more positive outcomes like this. For now, we’re off to a very promising start.<\/p>\n The findings are reported in The New England Journal of Medicine<\/a>.<\/p>\n![\"A](\"https:\/\/www.newsbeep.com\/us\/wp-content\/uploads\/2026\/09\/https:\/\/media.zenfs.com\/en\/sciencealert_160\/718d870e715ff3146c296373757b2371.jpg\"\/)
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