A dual-action drug offering hope to people with therapy-resistant cancer tumours could be available within five years.
The Royal College of Surgeons in Ireland and Almac Discovery, a biopharmaceutical company, are working together to explore the potential of the drug.
The research is a critical step on the journey to making the drug available to patients for whom all other forms of therapy have failed.
Prof Tracy Robson, who is leading the research team at RCSI, said it took 10 years to develop the drug with Almac Discovery, a research-driven oncology company based in Craigavon, Northern Ireland.
Clinical trials started in September 2015 and researchers recently established the maximum tolerated dose.
“We started off with a low dose of the drug and then incrementally increased it so we can establish the the best dose to use without toxicity. We have seen no toxicity at all, which is really good news,” she said.
The researchers will now move on to select ovarian cancer patients for whom all other treatments have failed. The drug, ALM201, has the potential to treat other types of cancer.
They will pre-select ovarian cancer patients who have a particular gene signature that will tell them whether their tumours have blood vessels and are most likely to respond to the drug.
Ovarian cancer ranks among the top 10 diagnosed and top five deadliest cancers in most countries — the drug has the potential to improve the 20% survival rate.
The drug was developed initially to cut off the blood supply, starving them of oxygen and nutrients. However, researchers also demonstrated that it can target cancer stem cells that are resistant to radiotherapy or chemotherapy.
Researchers will focus on how a novel protein, called FKBPL, occurring naturally in the body, targets cancer stem cells, so they can be more easily killed by chemotherapy and radiotherapy.
“Cancer stem cells are a major barrier to successful radiotherapy and chemotherapy and can result in failure of these treatments,” said Prof Robson.
“Our initial data demonstrates that ALM201 can transform these cells so they are no longer resistant to these therapies.”
Prof Robson said it could take another five years before it is approved for use by the regulatory authorities.
“If the drug shows really good efficacy, the authorisation process could be speeded up significantly because it then becomes unethical not to give the drug to patients,” she said.
Stephen Barr, the president and managing director of Almac Discovery, said ALM201 continued to surprise them as they uncovered the complexities of its effects. “The interaction of ALM201 with cancer stem cells, if proven with this research, opens up potential further treatment options for cancer patients that can be explored in the clinic,” he said.
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