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Blood disease drug targets genes

People suffering from a range of blood diseases could extend their lives by using a revolutionary new drug which targets genetic flaws in cells.

Trials of the drug Vidaza, otherwise known as azacitidine, found that it targets genetic faults in the blood cells of people with high-risk myelodysplastic syndrome, chronic myelomonocytic leukaemia or acute myeloid leukaemia.

It works by blocking a process called methylation, which shuts down genes essential for the development of blood cells.

A study published in the journal Lancet Oncology showed that the drug also helped the body produce healthy blood cells.

Dr Shabih Syed, scientific director of the charity Leukaemia Research, said: "Patients who received Vidaza in clinical trials not only had longer survival but also spent less time in hospital, had fewer serious infections and less need for transfusions.

"The availability of this drug will greatly improve the quality of life of patients and also ease some of the burden on NHS treatment facilities."

According to the trial involving 358 patients, 50.8% of patients treated with azacitidine survived after two years, compared with 26.2% of those on chemotherapy.

The drug's manufacturer said that average life expectancy for someone receiving conventional treatment was 15 months, compared with 24.5 months for someone using the new medicine.

Copyright © Press Association 2009

Leukaemia Research