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Blood Health

Getting new drugs to patients faster through clinical trials

Michael O’Dwyer

Professor of Haematology, NUI Galway and Director, Blood Cancer Network Ireland

Professor Michael O’Dwyer is director of Blood Cancer Network Ireland (BCNI). The Network aims to facilitate patient access to innovative blood cancer therapies through early stage, or Phase I, clinical trials.

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. This is 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% of patients can expect to live 5-7 years.”

Clinical trials in Ireland

However new drugs may take years before approval. Government must also agree 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. This therapy uses components of the immune system such as antibodies or cells to fight the cancer. BCNI will be launching, 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. This 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 therapy for acute myeloid leukaemia. Myeloid leukaemia is a cancer of the blood and bone marrow – 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.

A last resort used to be phase 1 clinical trials. Patients see these as a real option now. 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.”

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