By making a gene in the liver work harder, a completely new type of drug has shown promise for treating advanced liver cancer.
The drug works by ramping up the activity of a gene we all have in our DNA that stops cancers growing and helps the liver function normally. The treatment uses a carefully designed piece of genetic material – called a small activating RNA – to artificially boost this gene.
Several RNA treatments for cancer are under development, but this is the first to show some benefit for patients in trials. People with advanced liver cancer rarely survive for more than two years, and no reliable treatments are currently available.
Advertisement
“The best response is with a patient who’s had almost a 75 per cent reduction in the size of her cancer, maintained now for a year and a half,” says Debashis Sarker of King’s College London. In another four patients, tumours have stopped growing. In two of these cases, this stall in growth has so far lasted for nearly a full 12 months, says Sarker, who presented from the trial at the annual meeting of the American Society of Clinical Oncology in Chicago today.
The 28 people enrolled in the trial are the first anywhere in the world to receive a small activating RNA. The gene it targets is active mainly in the liver, and works by switching on whole networks of other genes that together combat cancer by restricting the growth and multiplication of liver cells.
The gene also triggers immune cells to become more active, which may also help control the growth of cancer.
Double benefit
The treatment may also boost general liver function, although it is not clear yet whether this is the case. If it does, this could beneficial for people with advanced liver cancer who also have cirrhosis. “It means we could treat two life-threatening conditions at once,” says Sarker.
“For some years, scientists have been looking at whether switching off faulty genes using targeted RNA molecules could treat cancer,” says Justine Alford of the charity Cancer Research UK. “So it’s fascinating that for the first time, researchers are now trialling the opposite approach—using RNA to activate genes with beneficial anti-tumour effects in cells.”
“These are early but encouraging results, so we don’t yet know if this novel approach will be an effective treatment for liver cancer,” says Alford. “But with poor survival, liver cancer remains a significant burden on global health and we need research like this to find new ways to tackle it.”
