TWO IRISH universities are leading a new clinical study into treatment for a rare and very debilitating type of bone marrow cancer. The study into combined oral medications to treat advanced myelofibrosis will be conducted at NUI Galway (NUIG) and Trinity College Dublin.
The study is being run in conjunction with centres in Britain, France and Italy, and patients may be treated at either Galway University Hospital or St James’s Hospital in Dublin.
Myelofibrosis is a cancer which results in bone marrow failure, when fibrous tissue fills the normal spaces in which blood cells are formed.
The body responds by making cells in abnormal sites, including the liver and spleen, in an effort to maintain normal blood cell counts. The organs can become enlarged and painful, and in some patients the condition can lead to an aggressive form of acute leukaemia as well as bone marrow failure.
Prof Frank Giles, NUIG’s professor of cancer therapeutics, is leading the study with St James’s Hospital consultant haematologist Dr Eibhlin Conneally. It will involve using a combination of Ruxolitinib, manufactured by Novartis, and a separate pill that also targets the abnormal pathways that drive myelofibrosis.
Ruxolitinib has become the first and only product approved by the US Food and Drug Administration for treating the disease, and Prof Giles hopes it will be approved in Europe soon.
“This is a significant positive advance in treatment for these patients,” Prof Giles said yesterday. He explained that Ruxolitinib was specifically directed at an abnormally active enzyme or kinase that has been recently defined as a key driver of myelofibrosis.
“This kinase, called Jak-2, has emerged as a key target for therapy in myelofibrosis,” Dr Conneally said.
The treatment 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”, she has said.
Prof Giles was involved with the development of both of the drugs combined for the study. He said success in anti-cancer therapies was “increasingly driven by a continuous process which involves pre-clinical scientists unlocking the puzzles of what actually makes a cancer cell behave differently from its normal counterpart”.
