Case study: how clinical trial helped one cancer patient

Seven years ago, Frank O’Duffy noticed a rather innocuous-looking mole on the shin of his right leg. He was initially reluctant to take any action, but a suggestion from a friend to get it checked out prompted him to go for tests with a local surgeon in Nenagh.

The mole turned out to be malignant, and over the course of the next four years, he had repeated operations to remove lymph glands in his groin that were registering high levels of melanoma.

A CT scan in November 2013 revealed that the disease had spread to his liver; it would later go on to affect his lungs.

“I was told that the normal things like chemotherapy and surgery wouldn’t be of any benefit to me, but there was this drug that was being trialled and there was a window to get into it, and for that reason I was extremely fortunate,” he says.

The trial was what is known as a double-blind test in which patients were given either one of two cancer drugs produced by Bristol-Myers Squibb – nivolumab or ipilimumab – and a placebo. He could even have been given both medications, depending on the luck of the draw.

After some promising initial results in 2014 his condition began to deteriorate significantly early the following year, and he was taken off the trial – or “unblinded”, as it is known in the medical community – at which point he found he had been given ipilimumab.

Game of chance

The original trial may not have worked out for him, but his participation opened the door for O’Duffy to try another relatively new product from Merck called pembrolizumab which is proving more effective, and his liver is now practically cancer-free.

Accepting that participation in clinical trials is often a game of chance for those who do not stand to benefit from conventional treatment options anyway, the 65-year-old appreciates the opportunity he was given and would encourage others in a similar position to do the same.

“The thing that I would say to anyone else thinking of going on a trial is that you have the opportunity to get a drug that wouldn’t be available otherwise. Particularly in the cancer area there’s so much going on out there, so much development that there are great opportunities to get these drugs early on.”

No matter how sick the person, it can often be difficult convincing patients to try unlicensed pharmaceutical products because of the fear of being used as a mere “guinea pig”, according to Derick Mitchell, chief executive of the Irish Platform for Patients’ Organisations, Science and Industry (Ipposi).

“The attitude is still based on the guinea pig feeling. That’s the initial reaction,” Mitchell says

“The more educated and informed the public is about the area of trials, just about debunking some of the myths that are out there about trials, you actually improve the quality of the research that’s being conducted and you get a more supportive public response to the research that’s being conducted.”

Side effects

Existing regulations stipulate that those conducting clinical trials must clearly outline any potential adverse side effects of taking the relevant active ingredients. It is then up to the prospective participant to weigh up the possible benefits and pitfalls of their involvement.

Mitchell says t he has witnessed “very isolated incidents” of triallists complaining to his organisation about unexpected side effects arising from certain tests, which he says are then referred on to the Health Products Regulatory Authority for thorough examination.

While he is convinced that patient safety is regarded as being of paramount importance for clinical investigators, he does have reservations about how some trials are conducted.

“We would certainly want pharmaceutical companies and indeed clinical investigators to think about the patient input when they’re designing the trial,” he says.

“Very often when you’re looking at what the real clinic result is, the indicators that you chose at the beginning of the trial are really not that relevant to the patient in the trial. We’ve seen that happen time and again where the patient on the trial feels, ‘Well, this is not really looking at what I would like to see happening’.

“It’s something that is definitely coming through a lot more that pharmaceutical companies are really designing their trials according to patient-reported outcomes: what patients feel are relevant to their ongoing lives.”