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New clinical trial for drug to treat rare form of leukaemia opens

The Bloodwise logo. Bloodwise appears in black text against a white background
Posted by
13 Feb 2017

The first patient has been recruited to a new clinical trial part-funded by Bloodwise.

The ‘MONOCLE’ study is a ‘phase 2’ study using the drug Tefinostat to treat chronic myelomonocytic leukaemia (CMML).

Around 450 people are diagnosed with this type of blood cancer in the UK each year, with long-term survival rates currently very poor. The vast majority of those affected are elderly and cannot tolerate aggressive chemotherapy treatments. Allogeneic stem cell transplant is a potentially curative treatment but this is only possible in a small minority of younger and healthier patients.

CMML is a disease caused by the over-production of white blood cells called monocytes. Taken orally, Tefinostat is a highly potent drug that selectively targets macrophages and monocytes – central cells of the immune system. Tefinostat has successfully completed a Phase I study with promising signs that it could treat CMML patients.

In addition to funding from Bloodwise, the trial has also received an investment of £2.8 million from the CRT Pioneer Fund to progress the development of Tefinostat for the treatment CMML.

The MONOCLE study will be led by Principal Investigator Dr Steve Knapper, Clinical Reader from the Institute of Cancer & Genetics at Cardiff University. It will be managed by the Centre for Trials Research at Cardiff University and aims to determine the tolerability and safety of the Tefinostat compound. The trial will initially recruit 19 patients with the potential to increase to 40 based on results. There are currently no other clinical trials taking place in the UK specifically looking at treating CMML.

Dr Steve Knapper, Clinical Reader from the Institute of Cancer & Genetics at Cardiff University commented: “We are very excited to be in a position to commence the MONOCLE study which will, for the first time, allow us to assess the effects of Tefinostat, a monocyte-directed therapeutic agent, in patients with this often-neglected haematological malignancy. Hospital sites across the United Kingdom will be open for recruiting patients into the trial. I am extremely grateful to our funders Bloodwise and the CRT Pioneer Fund for making this study possible.”

Dr Alasdair Rankin, Director of Research at Bloodwise, said: “The outlook for CMML patients is currently extremely poor, with an approximate 20% five-year relative survival rate. Traditional chemotherapy-based treatments are unsuitable for the majority of older patients. Early results for Tefinostat have been promising and we are delighted to be supporting the next stage in what we hope will bring a new and urgently needed treatment option to patients.”

Dr Robert James, Managing Partner, Sixth Element Capital, Manager of the CPF, commented: “We believe Tefinostat has great potential in treating Chronic Myelomonocytic Leukaemia and has the possibility for rapid market authorisation in a very well-defined patient population where there is a significant clinical need and little in development. The start of the Phase II trial is a significant milestone and our funding will help drive forward this potential new treatment to approval in the next few years. There are also other potential uses for the compound which we are exploring but commencing the MONOCLE study is a great step forward. MONOCLE has been driven by the work of Dr Knapper and we very much look forward to working with him and his colleagues to develop Tefinostat with the hope of bringing a successful treatment to CMML patients.”

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