Title: Precision Cellular Therapeutics
Funder: Department of Health and Social Care

Abstract: We aim to develop new kinds of cell therapies for blood disorders and blood cancer, and improved systems for following up patients receiving treatment to better support their care. One of the commonest cell therapies is a stem cell transplant, where healthy cells from a donor are used to permanently replace damaged blood and immune cells in the patient. Transplants are used to cure patients with inherited blood disorders or blood cancers. Transplants work in blood cancer patients because some of the donor immune cells attack and eliminate the cancer. We call this effect graft-versus-leukaemia. Unfortunately, donor cells may also overreact and attack healthy parts of a patient s body. This is called graft-versus-host disease and can cause serious health problems. If we could find a way of increasing graft-versus-leukaemia but avoiding graft-versus-host disease, this would increase transplant safety and cure more patients. Although the likelihood of identifying a stem cell donor has increased recently, many patients, especially those from an ethnic minority, still have no suitable donor. An important approach to address this unacceptable inequality is to use umbilical cord blood cells. However, cord blood units often lack enough stem cells for a safe transplant. If we could find a way to increase stem cell numbers in cord blood, we could use them in far more patients. Another reason why patients may fail to benefit from transplant and cellular therapies is that individuals from some communities may not get the same access to treatment, or respond less well once they do. At the same time, the side effects of some of the new cell therapies can be unpredictable and lead to long hospital admissions. When at home, it can then be difficult for patients to interact with their doctors and let them know how they are getting on. If we could better follow patients right from the start of their illness and throughout their treatment, we could ensure that more patients get access to cell therapies and deliver them with maximum safety. Our research plans to: 1. Find better ways of getting stem cell and immune cell grafts to eliminate blood cancer without causing graft-versus-host disease 2. Make immune cells that are more effective 3. Use novel methods to expand stem cells in the lab so we can treat more patients with cord blood 4. Safely modify stem cells so they work better and can be used to make other blood cells 5. Better ensure all patients in all communities are getting the same access to transplants and new cell therapies 6. Use new mobile technologies for patients so that they and their doctors can monitor symptoms and side effects of cell therapy, and better support their care out-of-hospital.

Lay abstract: We aim to develop new kinds of cell therapies for blood disorders and blood cancer, and improved systems for following up patients receiving treatment to better support their care. One of the commonest cell therapies is a stem cell transplant, where healthy cells from a donor are used to permanently replace damaged blood and immune cells in the patient. Transplants are used to cure patients with inherited blood disorders or blood cancers. Transplants work in blood cancer patients because some of the donor immune cells attack and eliminate the cancer. We call this effect graft-versus-leukaemia. Unfortunately, donor cells may also overreact and attack healthy parts of a patient s body. This is called graft-versus-host disease and can cause serious health problems. If we could find a way of increasing graft-versus-leukaemia but avoiding graft-versus-host disease, this would increase transplant safety and cure more patients. Although the likelihood of identifying a stem cell donor has increased recently, many patients, especially those from an ethnic minority, still have no suitable donor. An important approach to address this unacceptable inequality is to use umbilical cord blood cells. However, cord blood units often lack enough stem cells for a safe transplant. If we could find a way to increase stem cell numbers in cord blood, we could use them in far more patients. Another reason why patients may fail to benefit from transplant and cellular therapies is that individuals from some communities may not get the same access to treatment, or respond less well once they do. At the same time, the side effects of some of the new cell therapies can be unpredictable and lead to long hospital admissions. When at home, it can then be difficult for patients to interact with their doctors and let them know how they are getting on. If we could better follow patients right from the start of their illness and throughout their treatment, we could ensure that more patients get access to cell therapies and deliver them with maximum safety. Our research plans to: 1. Find better ways of getting stem cell and immune cell grafts to eliminate blood cancer without causing graft-versus-host disease 2. Make immune cells that are more effective 3. Use novel methods to expand stem cells in the lab so we can treat more patients with cord blood 4. Safely modify stem cells so they work better and can be used to make other blood cells 5. Better ensure all patients in all communities are getting the same access to transplants and new cell therapies 6. Use new mobile technologies for patients so that they and their doctors can monitor symptoms and side effects of cell therapy, and better support their care out-of-hospital.

Research activity codes: 5.9 RESOURCES AND INFRASTRUCTURE (TREATMENT DEVELOPMENT);7.4 RESOURCES AND INFRASTRUCTURE (DISEASE MANAGEMENT);8.5 RESOURCES AND INFRASTRUCTURE (HEALTH SERVICES)
Health categories: CANCER AND NEOPLASMS