A TREATMENT FOR a rare bone cancer will be made available for Irish patients for the first time, it was announced today.
Myelofibrosis (MF), a bone marrow disorder which severely debilitates patients can now be treated with a new drug called ruxolitinib.
The new drug recently received European approval which is an important development as some patients with MF experience multiple severe complications such as grossly enlarged spleen, fatigue, shortness of breath, appetite loss and shortened survival.
Frank Giles, Professor of Cancer Therapeutics at NUI Galway and Trinity College Dublin today welcomed the announcement as he said the life-threatening cancer has 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,” he said.
“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.”
People over the age of fifty are most likely to have this rare disease, which affects about 0.75 out of 100,000 people in the EU annually, 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 .
The new treatment involves a move away from ‘non-specific cell-killing drugs’ towards more targeted drugs that are directed at the fundamental drivers of cancer.
Experts say the drug can improve some of the more serious and debilitating symptoms of the disease meaning an improvement in daily living. Some patients can become more active, their pain is reduced, they get back their appetite and their sleep patterns improve.
Despite having it’s EU license, Dr Eibhlin Conneally, Consultant Haematologist at St James’ Hospital said the drug has yet to go through the HSE’s reimbursement process, which could take some time, 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,” she said.
“Irish patients have a right to this treatment that can improve their quality of life and give them a previously unavailable treatment option.”