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First-in-class cancer treatment Vidaza launched

Published on 03/03/09 at 09:00pm

Vidaza, an innovative new treatment for a number of hard-to-treat blood cancers, has been launched in the UK by Celgene.

The drug is a new treatment for higher-risk myelodysplastic syndromes (MDS), a group of blood cancers which on average prove fatal within six to twelve months.

Fewer than 2,000 new cases of MDS are diagnosed in the UK each year, and until now no drug has been approved to treat the disease.

The only curative option for the disease is chemotherapy followed by stem cell transplantation, but patients aged over 60 are not eligible for this treatment because of its intensity. This means many are only able to receive supportive care, such as blood transfusions, to manage the severe symptoms of the disease.

Targeting 'genetic errors'

Vidaza is first of a new class of innovative drugs known as 'epigenetic therapies' which target the genetic errors in the faulty cells that cause the cancer.

Celgene and doctors who have helped the company develop the drug have hailed it as a major step forward in prolonging and improving the lives of patients with the incurable cancer.

Ghulam Mufti is professor of haemato-oncology and clinical director for haematological medicine at King's College Hospital, and one of the first haematology experts to study Vidaza in clinical trials in the UK.

He says 'unprecedented' clinical trial results show it significantly extends survival for patients with higher-risk MDS, and also reduces the burden of blood transfusions to patients and healthcare professionals.

Mufti said: "Vidaza is a breakthrough therapy that offers patients in this under-served population the chance to live cancer-free for longer than previous care had allowed."

Data published in The Lancet Oncology shows Vidaza extended survival in patients with higher-risk MDS but also reduced the burden of hospital stays, with a 25% reduction per year compared to conventional care regimens.

In contrast to the existing standard of care, Vidaza allows many higher-risk MDS patients to live blood transfusion-free during their treatment, and suffer fewer incidences of anaemia and infections.

Patient group plea

Despite the potential for improved treatment, a patient group says it is not optimistic that local NHS organisations will fund the treatment.

David R Hall, chairman of the MDS UK Patient Support Group, said "procedural inertia, ignorance of the degree of patient suffering and misplaced costing assumptions" make it extremely unlikely the drug would be funded on the NHS before a NICE appraisal.

There is currently no NICE appraisal planned for the drug, which Hall said could mean "a frustrating, unnecessarily long and painful wait for medication".

He concluded by saying he hoped the drug's introduction would attract an "appropriately urgent and sympathetic response" from local NHS decision makers.

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