Experts said the results of the early-phase study were “exciting” and “promising” after patients who had exhausted all other treatment options showed an impressive response.
The drug, known as ONX-0801 in the phase 1 clinical trial, was tested in 15 women with advanced ovarian cancer as part of a wider trial run by the Institute of Cancer Research (ICR) and the Royal Marsden NHS Foundation Trust in London.
The aim was to test its safety, but the results were so good that researchers are keen to move the drug to the next stage of research as soon as possible.
In the trial, ONX-0801 significantly shrank tumours in seven of the 15 ovarian cancer patients.
In those patients whose tumours had the particular molecular target for the drug, the results were even more impressive, with seven out of 10 women responding.ONX-0801 is the first in a new class of drugs discovered at the ICR. It attacks ovarian cancer by mimicking folic acid to enter the cancer cells.
The drug then kills these cells by blocking a molecule called thymidylate synthase, thereby causing irreparable DNA damage.
Ovarian cancer cells have an abnormally large number of receptors for folic acid, called alpha folate receptors. This means these cancer cells respond particularly well to the treatment.Dr Udai Banerji, deputy director of the drug development unit at the ICR and the Royal Marsden, who led the study, said much more research was needed but the results were exciting.
He said: “As this is a completely new mechanism of action it should add upward of six months to patients’ lives with minimal side-effects in extremely late phase ovarian cancer.
“This is much more than anything that has been achieved in the last 10 years.”Because the new therapy is so specifically targeted at cancer cells, it leaves healthy cells alone.
This means it does not have the side-effects often seen with chemotherapy such as infections, diarrhoea, nerve damage and hair loss.
Experts have also created tests to detect the cells that will respond particularly well to the treatment, meaning doctors can identify those women who will benefit the most.Professor Paul Workman, chief executive of the ICR, said: “It’s really exciting to see such positive results in an early-stage trial.
“It looks a highly promising treatment with the potential to have huge benefits for women with ovarian cancer, and I’m very keen to see it progress to later-stage trials.”
The ICR, the Royal Marsden and healthcare company BTG are now looking for partners to fund next-stage clinical trials as soon as possible. The drug, which will be known as BTG945 going forward, will be highlighted at ASCO by the UK’s Department for International Trade, as one of the UK’s best biopharma assets available for licensing or partnering, the ICR s