A Chinese biotech firm has been granted permission to market the world's first commercial gene therapy medicine, New Scientist magazine reports. The country's medicines authority has approved Gendicine, a new cancer treatment, after promising results in a clinical trial. It will be launched in January by the firm SiBiono GeneTech, initially for the treatment of head and neck squamous cancers (a type of skin cancer).
The new therapy uses a known anti-cancer called p53, which makes a protein that triggers the self-destruction of cancer cells. In many tumours, p53 is faulty or inactive, so the cancer cells escape death and continue to multiply. Using an adenovirus to carry the therapeutic gene, SiBiono researchers tested the treatment on patients with nasopharyngeal cancer, which affects the upper part of the throat.
In the largest trial, 120 patients were given either radiotherapy alone, or Gendicine plus radiotherapy. Those given Gendicine received injections of the p53 gene directly into the tumour, once a week for eight weeks. In sixty-four per cent of the patients, the tumour regressed completely - three times the number treated using radiotherapy only. Around a third of the participants developed a fever, but there were no other side effects. The results will be published in China's national medical journal next month, according to Zhaohui Peng, head of SiBiono GeneTech, who also plans to submit an English version to an international journal.
Gene therapy pioneer French Anderson, of the University of California, who has been advising SiBiono, says their approach is simple compared to other gene therapies being developed. 'But sometimes simple is better' he said. If it proves successful, Gendicine will be launched in south-east Asia, before the firm seeks approval elsewhere.