A clinical trial led by the National Cancer Institute (NCI), a division of the National Institutes of Health, has led to the first approval of a treatment for advanced alveolar soft tissue sarcoma (ASPS). ). The immunotherapy drug atezolizumab (Tecentriq) has recently been Approved by the US Food and Drug Administration (FDA) to treat adults and children 2 years of age and older with ASPS that has spread to other parts of the body or cannot be removed with surgery.
ASPS is an extremely rare cancer that affects most teenagers and young adults. Consent was based on data from a non-randomized phase 2 trial (NCT03141684) was supported by the NCI and led by Dr. Alice Chen, MD, of the Clinic of Developmental Therapeutics in the NCI Department of Cancer Diagnosis and Treatment (DCTD). Genentech, a member of the Roche Group and manufacturer atezolizumabprovided medicine with NCI through a collaborative research and development agreement. The results of the study are being prepared for publication.
“Forty percent of patients are treated at the NIH Clinical Center in Bethesda,” says James H. Doroshow, MD, director of DCTD. “Our ability to bring in patients from around the world is a key factor in our ability to conduct research.”
“This approval will make a huge impact on a rare disease that is particularly difficult to treat,” Dr. Chen noted.
This is the largest study of ASPS. This is also the first study conducted at Network of clinical trials of experimental therapy which led to a drug approval. Network has activated sarcoma specialists at academic medical center from across North America to enroll patients in the trial.
“This is an important milestone for investigators in the Trial Therapeutic Clinical Trials Network, as well as for the community,” said Elad Sharon, MD, DCTD, one of the study’s leaders. ASPS patients and research on rare cancers”.
This is also the first time atezolizumab has been approved for children. Dr. Chen notes that this was enabled by the involvement of the Pediatric Oncology Branch at NCI’s Cancer Research Center, which helps enroll children in the trial.
“This study is an important example of a partnership between pediatric and medical oncology, enabling children with very rare cancers to access effective new therapies,” said John W. Glod, MD , Ph.D., of the Pediatric Oncology Branch. “The entire research team is grateful to the patients who participated in this study and made this work.”
About 80 people in the United States are diagnosed with ASPS each year. The disease usually begins in the soft tissue that connects and surrounds organs and other tissues. Although the disease develops slowly, once it has spread it is often fatal and chemotherapy is ineffective. Approximately 50% of patients with metastatic disease are still alive at 5 years. New targeted treatments, including drugs called tyrosine kinase inhibitors, don’t work long-term. Recently, however, immunotherapeutic drugs have shown promise as possible therapies for ASPS.
Atezolizumab is an anti-PD-L1 immune checkpoint inhibitor that works by helping the immune system respond more strongly to cancer. The FDA has approved atezolizumab to treat patients with several types of cancer, including liver, melanoma, and lung cancer. cancer.
In 2020, the FDA approved the breakthrough therapy designation for atezolizumab for the treatment of patients with unresectable or metastatic ASPS. This indication means that atezolizumab, used to treat a serious condition, has met the FDA’s criteria for rapid drug development and assessment. Later that year, the FDA granted an orphan drug designation to atezolizumab for soft tissue sarcoma in general. This situation encourages companies to develop a drug for rare diseases.
The phase 2 trial enrolled 49 multiethnic patients 2 years of age and older with metastatic ASPS who received atezolizumab every 21 days. According to doctors, about a third of patients respond to the treatment by shrinking the tumor to some degree. Most other patients experienced stable disease.
After two years of treatment, patients have the opportunity to discontinue treatment and continue on treatment leave for up to two years with close monitoring. None of the patients on leave of treatment had disease progression during that time.
Serious adverse events occurred in 41% of patients receiving atezolizumab; These include anemia, diarrhea, rash, dizziness, hyperglycemia, and pain in the extremities. However, no patient left the study because of side effects.
“This approval represents a win over rare diseases that are being thoroughly studied in clinical trials,” said Dr. Chen. “It is significant that this approval is given to a rare disease that can impact the lives of these young people.”
Research groups are currently conducting additional trials with atezolizumab for patients with ASPS, including using the drug in combination with other therapies.
National Cancer Institute
quote: The clinical trial leading to atezolizumab approval for advanced alveolar soft tissue sarcoma (2022, December 28) retrieved December 28, 2022 from https://medicalxpress.com/news/2022-12 -clinical-trial-atezolizumab-advanced-alveolar.html
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