A trial of gene therapy combined with chemotherapy to treat breast cancer nodules in the skin (BC2)

Cancer type:

Breast cancer

Status:

Results

Phase:

Phase 1

This trial looked at gene therapy injections and cyclophosphamide tablets to treat breast cancer nodules in the skin.

This trial tested a gene therapy called MetXia. MetXia works by introducing genes into cancer cells. It is a virus that has been altered so that it is no longer infectious or harmful in any way.

Genes are coded messages that tell cells how to behave. The virus is combined with a gene that tells liver cells to make an enzyme called cytochrome P450. As chemotherapy drugs like cyclophosphamide pass through the liver, the enzyme changes them into an active form that can kill cancer cells.

The researchers wanted to see if MetXia could carry this gene directly into cancer cells. The theory is that the enzyme could then be made inside the cancer cells. It would change cyclophosphamide into its more active form inside the cells. They would then be more likely to be killed by the chemotherapy, and there would be fewer side effects to the rest of the body.

The researchers wanted to be able to see the effect the treatment had on cancer. So this trial recruited people who had cancer nodules (lumps) on their skin.

The aims of this trial were to find out

The best dose of MetXia to use
How well MetXia carries the gene into cancer cells
How the combination of MetXia and cyclophosphamide affects cancer cells

Summary of results

The research team found that MetXia was a safe treatment, and that it did carry the gene into the cancer cells.

This phase 1 trial recruited 8 people with advanced cancer that had spread to the skin. Seven of them had breast cancer, and 1 had cancer of the ureter. Everyone taking part had 2 doses of MetXia, 24 hours apart. They had MetXia as injections into the nodules on their skin. They also took cyclophosphamide tablets for a total of 4 weeks.

The research team found that everyone taking part had some side effects, but they were usually mild. The most common side effects were

A drop in white blood cells
Feeling or being sick
Tiredness
Hair loss
Pain and redness at the injection site

The research team took biopsies 1 week and 3 weeks after treatment. They analysed the cells to see if they contained the gene. Out of the 8 people taking part, they found the gene in

7 of the samples taken 1 week after treatment
All 8 of the samples taken 3 weeks after treatment

They also looked at how well the cancer had responded to treatment 2 months after the injections. They found that

1 person’s cancer had got smaller
1 person’s cancer stayed the same size
6 people’s cancer continued to grow

During the trial the cancer did stop growing in 4 people, but for 3 of them, it started to grow again by the end of the trial.

The research team concluded that these results were encouraging. They found MetXia was safe to use, and that it was able to carry the gene into cancer cells.

We have based this summary on information from the team who ran the trial. As far as we are aware, the information they sent us has not been reviewed independently (peer reviewed Open a glossary item ) or published in a medical journal yet. The figures we quote above were provided by the trial team. We have not analysed the data ourselves.