Rare cancer drug for young patients approved

pharmafile | January 3, 2023 | News story | Research and Development  

A new treatment option for adult and paediatric patients with a rare soft tissue cancer has been approved. The FDA has approved new immunotherapy drug atezolizumab (Tecentriq) for patients with advanced alveolar soft part sarcoma (ASPS) that has either spread to other parts of the body or cannot be removed by surgery for another reason.

ASPS initially begins in the soft tissue connecting and surrounding the organs and other tissues, spreading slowly, but often becoming fatal once this spreading begins. Chemotherapies are not currently effective against this cancer and current targeted treatments do not appear to have a lasting impact.

Approximately 80 people are diagnosed with ASPS in the US each year, with only 50% of patients with metastatic disease alive after five years. This cancer predominantly affects young adults and adolescents.

The approval follows a non-randomised phase 2 trial led by the National Cancer Institute (NCI), part of the US National Institutes of Health (NIH).

During the trial a third of patients responded to the treatment and showed signs of their tumours decreasing in size, and a majority of the remaining patients showed stable disease levels. Approximately 41% of patients experienced serious side effects such as anaemia, diarrhoea, rashes, dizziness, hyperglycaemia and pain in the extremities, but none of the patients left the trial due to the side effects.

Dr Alice Chen, from the Developmental Therapeutics Clinic in the NCI’s Division of Cancer Treatment and Diagnosis (DCTD) commented: “This approval will make a huge impact in terms of a rare disease that has been particularly challenging to treat.”

Chen continued: “This approval represents a victory for rare diseases, which are understudied in clinical trials. 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.”


Betsy Goodfellow

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