Drug for rare lung cancer receives FDA approval

Normally, tumor cells evade T cells by expressing a checkpoint protein known as PD-L1 (left). Atezolizumab (Tecentriq) binds to PD-L1 and blocks it from binding to another checkpoint protein, PD-1 (right). This helps T cells regain their ability to kill tumor cells. Credit: Adapted from Onco Targets Ther 2017. doi: 10.2147/OTT.S90459. CC BY 3.0.

CANCER DIGEST – Jan. 7, 2023 – The US FDA has approved a new treatment for a rare type of lung cancer following a clinical trial led by the National Cancer Institute.

The drug atezolizumab (Tecentriq®) was approved for treatment of advanced alveolar soft part sarcoma (ASPS) or cancer of the tiny specialized air sacs of the lungs that allow for rapid gas exchange. Only about 80 people a year, mostly children and adolescents, are diagnosed with ASPS according to the NCI.

The trial is the largest study on ASPS, and the first conducted in the NCI-funded Experimental Therapeutics Clinical Trials Network that has resulted in a drug approval. The network connects sarcoma specialists at academic medical centers from across North America for the purpose of conducting clinical trials.

In the trial that resulted in FDA approval, 49 patients, ages two and up, with ASPS that had spread to other parts of the body were given infusions of the drug atezolizumab every 21 days. About one-third of those treated responded to the drug, meaning their cancer showed signs of shrinking, while most patients achieved stable disease, meaning the drug halted the growth and spread of the cancer. After two years of treatment patients were given the option of continuing treatment, or taking a break from treatment while under close monitoring. None of the patients who opted to take the treatment break saw their cancers progress.

Side effects of the drug occurred in 41 percent of the patients and included: anemia, diarrhea, rash, dizziness, hyperglycemia, and pain in the extremities, however, none of them patients stopped the study because of side effects.

“This approval represents a victory for rare diseases, which are understudied in clinical trials,” said principal investigator Dr. Alice Chen in a press release. “For this approval to go through in a rare disease, and to be able to make an impact on these young people’s lives, is very significant.”

The researchers are now conducting additional trials with atezolizumab for patients with ASPS, including using it in combination with other therapies.

Source: National Cancer Institute press release