The Food and Drug Administration (FDA) has approved the drug for use in treating advanced alveolar soft tissue sarcoma in patients whose tumor has spread to other organs or cannot be removed with surgery.
- Advanced alveolar soft tissue sarcoma is a rare tumor that usually occurs in adolescents and young adults.
- There are more than 50 types of soft tissue sarcomas.
Advanced alveolar soft tissue sarcoma (ASPS) is a rare cancer that begins in the soft tissues that connect and surround organs and tissues. The growth of the tumor is slow, but it is usually fatal once it has spread. Chemotherapy does not work against this cancer. As for new targeted treatments, in particular tyrosine kinase inhibitors, they have no lasting efficacy.
Approval of atezolizumab in patients aged 2 years and older
The immunotherapy drug atezolizumab (Tecentriq) was recently approved by the US Food and Drug Administration (FDA) for use in patients with PMDS that has spread to other parts of the body or which cannot be removed by surgery.
This authorization follows the positive results of the phase 2 of a clinical trial conducted by the National Cancer Institute (NCI) which is part of the US National Institutes of Health (NIH). Atezolizumab has been approved for people aged two years and older. “This approval will have a huge impact on a rare disease that has been particularly difficult to treat”said Dr. Alice Chen, of the Developmental Therapeutics Clinic in the NCI’s Division of Cancer Treatment and Diagnosis (DCTD).
Atezolizumab is already approved in the United States for patients with liver cancer, melanoma or lung cancer. This medicine promotes the immune system’s response to cancer.
Atezolizumab: tumor shrinkage in 1/3 of patients
During phase 2 of the clinical trial, 49 patients aged two years and older were recruited by the scientists. All the volunteers were affected by a PMDS which had spread. As part of the research, they received an infusion of atezolizumab every 21 days.
Result: the researchers found a reduction in the tumor in a third of the subjects. In the majority of cases, the disease stabilized in patients. A break from treatment was granted to some people who had received the treatment for two years. This cut lasted a maximum of two years with close monitoring. No progression of the disease was observed in these patients.
However, 41% of patients who received atezolizumab infusions developed serious side effects such as diarrhea, rash, dizziness, hyperglycaemia or anemia. But none of the participants wanted to stop the clinical trial because of the adverse effects caused by the treatment.
Additional clinical trials are currently underway with atezolizumab. The aim would be to combine it with other therapies in patients affected by SAPM. “This approval represents a victory for rare diseases, which are understudied in clinical trials”said Dr. Alice Chen.
