First promising results with CAR-T and chemotherapy in children and adults with advanced sarcoma

CAR-T against HER2 in advanced Sarcomas

The International AACR conference was recently held in the US. The results of a Phase I study were presented in 10 patients with advanced sarcoma between the ages of 4 and 54. The study is open to recruitment and plans to recruit more and more sarcoma patients, including children. The treatment is given to patients whose tumor expresses on its surface a protein called “HER2”, which is common in about 40% of sarcoma patients, including the most common sarcoma type, Osteosarcoma.


One of the children who participated in the study, with Rhabdomyosarcoma that had metastasized into his bone marrow, was in a full remission that lasted 12 months. He received additional treatments and is now 17 months in full remission!

Another patient with Osteosarcoma, metastatic to the lungs, also achieved full remission that lasted 32 months! Three more patients achieved stable disease and among five others, the disease progressed.

In general, patients in this clinical trial experienced a low toxicity to the treatment. As expected, all patients had reductions in blood count, which improved after treatment, and none of them developed any infections due to decreased blood count.

The future

Today, treatment options for children and adults with recurrent or resistant sarcomas are very limited. These patients are offered general chemotherapy regimens whose chances of success are low and can be toxic. Therefore, the results of this study, which carry great hope for these patients and the very existence of this clinical trial, is a tremendous opportunity to benefit from innovative treatment that is not for nothing, one of the most advanced in the world.

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