Accelerating clinical trials for stem cell transplantation

IMPACT is a partnership between Anthony Nolan, Leuka, NHS Blood and Transplant and the University of Birmingham (UK), which aims to deliver the UK’s first large-scale clinical trials program. The ambitious, 4-year pilot could transform our ability to deliver prospective clinical trials across all fields of stem cell transplantation and vitally improve outcomes for transplant patients. In this exclusive blog piece David Marks, Medical Director of IMPACT,  provides his thoughts on this exciting new period in stem cell transplant research.

The current status of stem cell transplants in the UK

Stem cell transplants give a second chance of life to 4,400 people a year in the UK [1], but sadly, fewer than half of patients will reach the milestone of 5-year survival. Much-needed improvements in patient outcomes can only be achieved through pioneering research. Clinical trials are crucial in forming the evidence base for new approaches to treatment, but not enough trials are taking place.

Allogeneic transplants, one of the main types of blood cell transplants carried out in the UK, are some of the biggest procedures the NHS performs. Most patients spend 4–5 weeks in hospital; take at least 6 months off work or education and each transplant costs approximately £100,000. The majority of patients return to work, school or higher education and live full lives however, some develop complications preventing a return to their pre-transplant lives.

Transplant is an effective therapy but we need to get better: to cure more people and to reduce post-transplant complications.

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In hematology the way we improve outcomes is by doing clinical trials. The UK probably has the most established and best clinical trials system in blood cancers worldwide. However, to this point, the UK has never focused on prospective transplant trials. Our clinical results are world class but we have under-achieved in clinical transplant research especially compared to the US, France and Italy.

In 1999 the Clinical Trials Committee of the British Society of Blood and Marrow Transplantation was formed. The result was a relatively disjointed approach: retrospective and very few prospective studies were conducted with limited infrastructure and limited collaboration. Since then the use of transplants has steadily increased but the amount of money we have to spend on this therapy has limits: we need to use it optimally.

The IMPACT partnership to accelerate clinical trials promises to help meet this need, and address the relative scarcity of clinical trial opportunities for stem cell transplant patients. For many years, transplant patients have struggled to access cutting-edge treatment – only one in twenty have participated in a trial, compared to one in five cancer patients – and clinicians know that many more patients would like the opportunity.

IMPACT will roll out 12 trials over the next 3 years. We plan to reduce the time it takes to complete a trial by a third, so new treatments reach patients much more quickly.

How will IMPACT speed up the clinical trial process?

IMPACT will speed up the clinical trial process in three ways. The first is through our dedicated research nurses, based at our partner transplant centers, which will recruit patients and monitor their progress during the trial. Historically, the difficulty of recruiting sufficient numbers of patients has been one of the major barriers to effective trials. By providing the platform for transplant centers to work collaboratively, IMPACT will facilitate access to a much larger patient population than would otherwise be possible at an individual transplant center.

The second way IMPACT will accelerate clinical trials is by providing much-needed infrastructure. At the heart of IMPACT is an operational ‘hub’, at the University of Birmingham (UK), which provides administrative support and regulatory expertise to help clinical researchers design, deliver and evaluate trials. The hub will speed up vital, but time-consuming, tasks such as developing trial proposals, writing the trial protocol, seeking and obtaining regulatory and ethical approval and monitoring trials, to allow transplant centers to focus on patient recruitment and delivering trials.

The third way IMPACT will accelerate trials is by facilitating data-sharing among transplant centers. The IMPACT hub will collect data on trial outcomes and provide statistical analysis to help in the evaluation of trials. This will ensure rapid publication of results and see treatments get from bench to bedside more quickly.

Our initial goals are simple but difficult to achieve. The three annual trials will largely be randomized trials comparing an existing, standard therapy with a new therapy that we hope will be better. These are the most complicated and expensive trials to deliver but they are the ones that change clinical practice and persuade commissioners that their money is well spent.

A change of culture is fundamental to IMPACT. A key to this is working together, not always putting our own institutions first and making compromises so that trials can happen, even if we are used to doing things differently in our own transplant centers.

The future of stem cell transplant research

If we succeed there will be a new breed of younger transplant investigators who will be working in a cooperative network. They will be chief investigator of their own trials; it will develop their careers and hopefully encourage them to perform other research that improves patients’ with a chance of a cure.

This is the beginning of an exciting period in stem cell transplant research. Over the next 4 years, IMPACT will facilitate up to 12 clinical trials, involving hundreds of patients, that will shed light on how new and existing treatments can deliver better outcomes for patients – and, ultimately, save and improve the lives of more individual’s having a stem cell transplant.

Author profile:

markDavid Marks, Medical Director of IMPACT, a partnership between Anthony Nolan, Leuka, NHS Blood and Transplant and the University of Birmingham. Marks has an extensive history in clinical trial delivery; he is director and consultant at Bristol transplant centre and holds an Honorary Professorship at the University of Bristol (UK). He was President of the British Society of Blood and Marrow Transplantation between 2002 and 2009 and established the Society’s Clinical Trials Committee in 1999.

Reference: 4,398 transplants carried out in the UK in 2016, source: (Accessed 8 November 2017)