The first patient has been successfully recruited into an important new cancer study being co-ordinated by a team of experts at Cardiff University (Cardiff University, 2017). The trial could have profound ramifications for treatment of an uncommon form of cancer that affects the elderly, who cannot tolerate aggressive chemotherapy treatments.

MONOCLE is a Phase II trial to test the effectiveness of the drug tefinostat, and monitor for side effects in patients with chronic myelomonocytic leukaemia (CMML).

Chronic myelomonocytic leukaemia causes a build up of cells called monocytes in the bone marrow and blood. Tefinostat is a drug, taken orally, which only becomes active after it is inside monocytes. In lab studies, performed in the Cardiff Experimental Cancer Medicine Centre, tefinostat was effective against monocyte tumours, including chronic myelomonocytic leukaemia and some types of acute myeloid leukaemia. Chronic myelomonocytic leukaemia mainly affects older people and the average length of survival is eighteen months. There are currently very few treatment options for chronic myelomonocytic leukaemia, and there is a pressing need for new drug treatments which can target the abnormal monocytes without causing unacceptable side effects.

The Phase II trial will be led by Principal Investigator, Dr Steve Knapper, Clinical Reader in the Division of Cancer and Genetics at Cardiff University. Steve Knapper is co-lead of the Early Phase Clinical Trials work package in the Wales Cancer Research Centre. The trial will be managed by the largest team of academic clinical trials staff in Wales, the Centre for Trials Research at Cardiff University.

Steve Knapper said “We are really excited to be in a position to open MONOCLE which will, for the first time, allow us to assess the effects of tefinostat in patients with CMML – an often-neglected disease…The trial will be available at hospital sites throughout the United Kingdom. I am extremely grateful to our funders Bloodwise and the CRT Pioneer Fund for making this study possible.”

Blood and bone marrow samples from MONOCLE patients will be tested in the Cardiff laboratory to help better understand how tefinostat works and to identify which patients are most likely to benefit in future. MONOCLE aims to determine the tolerability and safety of the Tefinostat compound. There are currently no other chronic myelomonocytic leukaemia-specific studies being held in UK.

Dr Alasdair Rankin, Director of Research at Bloodwise, who are funding the trial along with Cardiff University, said “The outlook for CMML is currently extremely poor, with only around a 20% five-year relative survival rate and traditional chemotherapy-based treatments unsuitable for the majority of older patients…Early results for tefinostat have been promising and we’re delighted to be supporting the next stage in what we hope will bring a new urgently needed treatment option closer to patients.”