Scientists develop novel treatment for Hairy Cell Leukaemia using drug administered to combat skin cancer

Published on January 16, 2014 at 4:17 AM · No Comments

A team of scientists from the University of Leicester has demonstrated a novel treatment for Hairy Cell Leukaemia (HCL), a rare type of blood cancer, using a drug administered to combat skin cancer.

The research, which is published today (Thursday 16 January) in the New England Journal of Medicine, indicates Vemurafenib, a BRAF inhibitor that has been approved as a treatment for advanced melanomas, is also successful in treating leukaemia. The study shows the treatment, which can be taken orally, cleared the malignant cells from the patient's blood and led to a complete clinical recovery in a number of days.

The study was led by the University of Leicester and involved treatment of a patient at the Leicester Royal Infirmary.

Dr Salvador Macip, from the University of Leicester's Department of Biochemistry, explained: "A genetic study of the patient's blood cells allowed us to identify a mutation in the BRAF gene that is commonly found in skin cancers. This knowledge enabled us to combat the cancer cells with Vemurafenib, which has had proven success as a BRAF inhibitor in melanomas, and showed similar success for this patient who had exhausted all other treatment options, which is fantastic. 

"What was most surprising was that the drug did not work in the way we expected it to. Whilst it successfully blocked BRAF and killed the cancerous cells, there was no ability to block the downstream cascade of signals. Therefore more research is required to better understand how this drug works to ensure we are able to use it in the best possible way.

"This is one of the first clinical examples of this treatment for HCL and we are the first researchers to do a biochemical study of the samples and discover that the drug does not do what it's supposed to be doing."

This approach to targeting cancer is an example of precision medicine with clinicians and research scientists working side-by-side to ensure the best treatment, tailored to the individual patient, was provided.

Professor of Haemato-Oncology at the University of Leicester, Professor Martin Dyer, who is Honorary Consultant Physician, Department of Haematology at Leicester's Hospitals, said: "Precision medicine in which clinicians and basic scientists collaborate to deliver novel and rapid personalised therapies to cancer patients like this is essential.

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