The Irish doctors who led the clinical trials for the development of a drug to treat a rare bone cancer have welcomed the announcement that treatment will be available for the first time.
Myelofibrosis, or MF as it is known, is a bone marrow disorder, which severely debilitates patients with the disease and can now be treated with a new drug called ruxolitinib.
The new drug, made by Novartis, has received European approval.
Some patients with MF can experience a grossly enlarged spleen, fatigue, shortness of breath, appetite loss and shortened survival.
Irish expert on the condition, Frank Giles, Professor of Cancer Therapeutics at NUI Galway and Trinity College Dublin, welcomed the announcement on the new treatment.
Dr Giles said: "Myelofibrosis is a rare and potentially devastating cancer with a poor prognosis and limited treatment options for many of those who suffer from it. While therapies, including blood transfusions and radiotherapy, have been used to manage some of the individual symptoms of MF, we have had no prior approved MF treatment option that directly treats the underlying disease.
"This is literally the first and only specific therapy for MF, a particularly important unique development that will begin to address the unmet needs of Irish patients with MF."
Dr Eibhlin Conneally, Consultant Haematologist at St James’ Hospital, who has extensive experience in clinical trials and clinically manages many of these patients said the new treatment offers a much-needed option to patients with MF.
Dr Conneally said: "It involves a move away from ‘non-specific cell-killing drugs’ towards safer, more targeted drugs that are really directed at the fundamental drivers of cancer. It can improve some of the more serious and debilitating symptoms of the disease and means that daily living improves which makes a real difference.
"Some patients can become more active, their pain is reduced, they get back their appetite and their sleep patterns improve."
However, despite having it’s EU license, the drug has yet to go through the HSE’s reimbursement process before it can be made available to all those who need it.
Dr Conneally said: "This treatment development is of huge importance to Irish patients so it is vital that it be made available to the Irish patients who need it as soon as possible. Irish patients have a right to this treatment that can improve their quality of life and give them a previously unavailable treatment option."
MF may result in serious complications including weakness, fatigue, shortness of breath on exertion, weight loss, night sweats, paleness, unexpected bruising/easy bleeding, increased likelihood of getting an infection, an enlarged liver (in two-thirds of patients) and bone pain.
The life-expectancy of someone diagnosed with MF is considerably reduced. People over the age of 50 are most likely to have this disease, with men and women equally at risk.
In some patients, MF can also develop into a particularly aggressive form of leukemia which is often difficult to treat and can be rapidly fatal.