Professor O’Dwyer says: “We want to provide the greatest access possible, as novel treatments are making a difference to patients. For example, in multiple myeloma, a cancer of the bone marrow, there has been a stepwise increase in survival, over the last 15 years, as a result of the incremental improvements with each new drug developed. In the 1990s the median survival for the condition was three years. Today, 50 per cent of patients can expect to live five to seven years, and a small proportion of those with a better risk profile can actually be cured.”

Clinical trials in Ireland

The problem is, it may take years before a new drug is approved and the government agrees to pay for it. Frequently, the only way for patients to gain early access is via a clinical trial.

In this regard, an exciting area of blood cancer research is immunotherapy, which uses components of the immune system such as antibodies or cells to fight the cancer. Later this year BCNI will be launching, among others, a Phase I clinical trial to test a new antibody for multiple myeloma treatment. This antibody, which was approved last November by the US Food and Drug Administration but is not yet available in Europe, binds to the surface of myeloma cancer cells, inducing their death. It will be tested in combination with a standard initial chemotherapy regimen for myeloma, which may play an important role in reactivating the immune system.

“This trial is unique to Ireland,” explains Professor O’Dwyer. “It will be the first to combine this antibody with a backbone regimen containing cyclophosphamide for initial treatment. And we believe that, because of the immunogenic effects of the chemotherapy regimen, the combination has the potential to greatly enhance the antibody’s activity.”

Another promising research area focuses on small molecules similar to carbohydrates, called glycomimetics. Professor O’Dwyer is currently leading a clinical trial using one of these compounds as a novel therapy for acute myeloid leukaemia – a cancer of the blood and bone marrow and the most common type of acute leukaemia in adults. 

Patients benefit

 “It’s great to be able to give patients the opportunity to try novel treatments that could potentially help control their disease, especially when standard treatment options have been exhausted. Increasingly, with an improved understanding of blood cancer biology and targeted treatments, patients are deriving real benefit. In addition, we are better able to establish the optimal dose of new drugs, reducing the risk of side effects,” says Professor O’Dwyer.

Phase I clinical trials were once considered as a last resort, he adds. Now, they are increasingly viewed as a real option for many patients. “Ultimately, participation in clinical trials means patients can access promising, potentially-life saving treatments faster, for their own benefit, and that of future patients.”