People in Ireland who are living with a rare bone cancer will, for the first time, be able to avail of a new treatment.

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 recently received European approval and means that the unmet needs of Irish MF patients can now be met.

Some patients with MF are impacted by multiple severe complications such as grossly enlarged spleen, fatigue, shortness of breath, appetite loss and shortened survival.

"Myelofibrosis is a rare and potentially devastating cancer with a poor prognosis and limited treatment options for many of those who suffer from it," said Frank Giles, who is an Irish expert on the condition and professor of Cancer Therapeutics at NUI Galway and Trinity College Dublin. 

"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, who is a consultant haematologist at St James' Hospital, has extensive experience in clinical trials and clinically manages many MF patients.

She said: "This new treatment offers a much-needed option to patients with MF and 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.

"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," said Dr Conneally.

MF is a rare, often unrecognised, disease that develops when bone marrow - the soft, fatty, substance inside bones that produces blood cells - is replaced by scar tissue. The abnormal stem cells produce more mature cells that grow quickly and take over the bone marrow, causing the formation of more scar tissue and chronic inflammation.

As a result, the bone marrow becomes less able to create normal blood cells and blood cell production may move to the spleen, causing enlargement, or to other areas of the body.

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 fifty 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.

Ruxolitinib is made by Novartis.