Acute myeloid leukaemia is the most common aggressive blood cancer and there are about 2,200 new cases in the UK each year. It can be hard to treat, especially in patients over 65 years of age and is often fatal within a year of diagnosis. Therefore, there is an urgent need for more effective therapies. The lack of suitable treatment has been identified as a major unmet need by the national blood cancer charity Bloodwise, which is co-funding the study.
The drug is in the earliest phase of clinical testing and does not have marketing authorisation or approval. The purpose of the clinical trial is to start to gain an understanding of the side effects of the drug and possibly identify early evidence of effectiveness in treating AML. Patients with AML and who are aged 18 and older are being sought for the study.
The drug is to be tested as part of a long-term collaborative research project between cancer researchers at Oxford University in the United Kingdom and Stanford University in the United States. The clinical study is also a collaboration between Oxford University Hospitals NHS Foundation Trust, which runs the Churchill, the University of Oxford and Stanford University.
Research between the University of Oxford and Stanford University found that leukaemic stem cells that propagate the disease protect themselves from being “eaten” by immune cells by expressing a “don’t eat me” signal, called CD47.
Researchers developed this new drug to block CD47, and it is hoped will enable the body’s own immune cells to eliminate Acute Myeloid Leukaemia cells.
This clinical research is funded in the UK by the NIHR Oxford Biomedical Research Centre, the Medical Research Council and Bloodwise and in the US by the California Institute for Regenerative Medicine and Ludwig Cancer Research.
Chief Investigator for the clinical trial, Consultant Physician and Professor of Haematology Paresh Vyas said: “This is an important study as it aims to provide much needed treatment for a large patient group where we have made limited impact in improving survival with good quality of life.
“It is also important as it is a radical new approach. It is an exciting and unique collaboration between two of the world’s most pre-eminent universities and is a great example of how universities and the NHS can develop drugs from concept to the clinic in partnership.”
George Freeman MP, Parliamentary Under Secretary of State for Life Sciences at the UK Government’s Department for Business, Innovation and Skills and the Department of Health, said: “This new drug could revolutionise our approach to tackling this form of leukaemia and potentially deliver a life-enhancing treatment so desperately needed by patients.
“Supporting the development of innovative medicines like this with trials in the NHS is why the Government invests £1 billion each year in the National Institute for Health Research, helping to confirm the UK’s position as a world-leader in medical research and accelerate access for NHS patients to new treatments.”
Dr Matt Kaiser, Head of Research at Bloodwise, said: “Drugs that harness the immune system have had considerable success in treating other types of blood cancer. This new type of treatment could be a very exciting prospect for patients with acute myeloid leukaemia, for whom alternatives to current treatments are desperately needed.”