A trial of gene therapy for cancer in the liver that can be removed with surgery (GDEPT)

Cancer type:

Liver cancer

Status:

Results

Phase:

Phase 1

This trial was looking at a type of gene therapy. It was for primary liver cancer or bowel cancer that had spread to the liver. The people taking part were going to have surgery to remove the cancer from their liver.

With this type of gene therapy, a specially treated virus is injected directly into the cancer. It carries a gene that can turn a harmless drug called CB1954 into a very active anti cancer drug. The harmless drug travels through the bloodstream. When it reaches the virus infected cancer cells, it is activated by the gene in the virus and kills the cancer.

This trial was trying to find out how good the virus was at getting the genes into the cancer cells in the liver, and to find out about any possible side effects. The aim of this trial was to find the highest dose of the virus that could be given safely. The people who took part in this trial did not have the anti cancer drug.

Summary of results

The trial team found it was possible to give the treated virus in this way and they worked out the highest dose they could give safely.

The trial recruited 18 people. The first few people had a low dose of the virus. As they didn’t have any bad side effects, the next few had a slightly higher dose. And so on, until the highest safe dose was found. This is called a dose escalation study.

Nobody taking part had any serious side effects from the treatment. But some people had mild pain at the injection site and 4 people had a high temperature a few hours after having the injection.

The people in this trial did not have the anti cancer drug. They all had surgery to remove the cancer from their liver a few days after having the virus injection. The researchers were able to look at samples of the tissue removed during surgery to learn more about how the genes were working.

Having found a safe dose that get can get into cancer in the liver without causing bad side effects, the trial team hoped to proceed to the next stage of the research. This would mean people having CB1954 a couple of days after the treated virus.

We have based this summary on information from the team who ran the trial. The information they sent us has been reviewed by independent specialists (peer reviewed Open a glossary item ) and published in a medical journal. The figures we quote above were provided by the trial team. We have not analysed the data ourselves.