Novel CAR-T Cell Therapy Development against Metastatic Rhabdomyosarcoma in a Patient

Release Date: 18-Nov-2020

Hundreds of clinical research activities have indicated that prognosis for the patients suffering from metastatic rhabdomyosarcoma is very poor. But it has been witnessed that a patient suffering from metastatic rhabdomyosarcoma was able to receive complete remission of more than 20 months when treated with investigational chimeric antigen receptor T-cell therapy plus a lymphocyte-depleting chemotherapy regimen.


A group of researchers have identified a novel CAR T-cell regimen that acts and performs its function similar to a vaccine, leading to the activation and recruitment of the immune cells against the cancer cells. The trial for the same was deigned to target the protein molecule that is present on the surface of the sarcoma cells i.e. HER2 protein molecule. The strategy adopted by the researchers was to involve multiple CAR T-cell infusions preceded by chemotherapy. The primary aim of using chemotherapy in the trial was to develop a space for therapeutic CAR T cells that were introduced into the patients. The whole treatment plan included three cycles of chemotherapy and CAR T-cell infusions (induction) followed by subsequent CAR T-cell infusions without chemotherapy (consolidation).


After repeating the treatment with CAR T cells plus lymphodepleting chemotherapy, researchers were able to receive complete response in the enrolled patients. Also, the patients received CAR T-cell infusions with the addition of pembrolizumab for the overall boosting of the anti-cancer activity. The one specific patient enrolled in the clinical trial was able to observe complete remission for almost 2 years after stopping the infusions of T cells. The one specific patient that was able to observe complete response had high expression level of HER2 protein on the tumor cells, which further led to the evidence that it recruited the immune cells to attack the cancer cells. However, this led to the complete remission found in the patient. As per the researchers, it is estimated that such findings will be able to develop a complete novel hope among the researchers for targeting a therapy that is capable of introducing complete response in the patients.

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