The University of Birmingham has partnered with Leuka, NHS Blood and Transplant and Anthony Nolan to deliver the UK’s first large-scale clinical trials programme, to save and improve the lives of children and adults who receive stem cell transplants.
The ambitious four-year pilot, called IMPACT, will reduce the time it takes to deliver a clinical trial in stem cell transplantation by a third. The £4 million programme will establish up to 12 clinical trials involving approximately 1,500 patients – a marked increase on current numbers. In 2014 just one in 20 transplant patients were involved in a trial, compared to one in five cancer patients, due to relatively small patient numbers, lack of infrastructure and a complex regulatory environment.
Advances in stem cell transplantation have resulted in almost every patient now having a suitable donor but, despite this, more than 50% of all adult transplant patients still die within five years of their transplant. This clearly demonstrates an urgent need to improve patient outcomes through cutting-edge research.
IMPACT Medical Director, Professor David Marks, believes the programme will inform clinical practice and thereby benefit each of the 4,400 people a year who receive stem cell transplants in the UK.
He says: “New, and improved, treatments for stem cell transplant patients can only become possible if they are evaluated and approved as part of a clinical trial. We have been unable to advance treatments at the same rate of other developed countries but, by making it easier to set up trials and recruit participants, IMPACT will ensure that medical advances reach patients more quickly and help fill the gaps in our current understanding.”
Ten of the 32 UK hospitals which facilitate stem cell transplants will receive ring-fenced funding to employ a dedicated research nurse. A further 12 hospitals will use existing resource to participate in the pilot. The need for more investment for a programme of clinical trials focused on stem cell transplantation was first highlighted in the 2010 report from the UK Stem Cell Strategic Forum, an expert group set up by the Minister of State for Public Health. The Life Sciences Industrial Strategy, published in August, also called for an increase in the number of clinical trials by 50% over the next five years.
The first clinical trials, to be launched later this year, have been welcomed by patients. Deborah Harkins, from North Worcestershire, was diagnosed with acute myeloid leukaemia in January 2016. The 49-year old was treated with chemotherapy and had a stem cell transplant in June 2016.
Deborah, who is currently participating in a clinical trial for high-risk leukaemia patients, believes the UK could do better. She says: “I believe we owe it to donors, those who are giving people a second chance of life, to determine which factors make a stem cell transplant successful – at the moment we’re letting them down.
“I’ve never met a patient who wouldn’t consider being part of a clinical trial. The issue is that there aren’t any trials for patients to be part of. This is why IMPACT is so important. It will increase the number of patients on trials, and centres can share research staff and resources.”
An additional benefit of IMPACT is the ability – through the collection and analysis of diagnostic materials and samples during treatment – to drive fundamental scientific research in areas such as predictive biomarkers, genomic mechanisms of resistance to therapy and drug discovery. This will create opportunities for scientists to deliver basic research that will increase understanding of why stem cell transplants fail for 50% of patients.
By providing infrastructure to accelerate the establishment of clinical trials in the area of stem cell transplantation, IMPACT will increase patient access to potentially life-saving treatments while contributing to the government’s ambition to make the UK a leading destination for life sciences innovation.