At Anthony Nolan, we’re proud to participate in groundbreaking clinical trials with partners across the UK.
Clinical trials are a vital part of blood cancer research – they enable us to work towards minimising the risks and side effects of stem cell transplants, and improving quality of life for transplant recipients.
You can read more about recent and ongoing clinical trials that we actively support below:
Phase I/II Clinical Trial
What does this trial do?
It aims to investigate the safety of Adenovirus-specific T-cells given to high-risk paediatric patients after a stem cell transplant.
Dr Waseem Qasim (ICH GOSH)
The Human Adenovirus (ADV) causes respiratory and intestinal tract infections – similar to the common cold.
ADV is fairly common in the general population, and is well-tolerated in people with a healthy or normal immune system. But it can cause major problems, such as pneumonia and even death, in patients recovering from a stem cell transplant.
Cell Medica’s product is manufactured using a donor’s white blood or T-cells, which are responsible for attacking and fighting off infections. Scientists activate and expand these donor T-cells in the laboratory to boost their activity – specifically, against ADV.
The finished T-Cell therapy can then be infused into the patient if they acquire the ADV infection.
Pilot studies have shown the benefits of T-Cell therapy, and it’s hoped that it can restore the patient’s immunity and give them lasting protection against ADV. T-Cell therapy also causes less toxicity than standard anti-viral drugs, which don’t always succeed, and also stop working once the course is finished.
Anthony Nolan provided major support on this clinical trial. Our Donor Provision Coordinators (DPC) contacted over 40 UK and international donors to provide a blood sample, which was then used to manufacture the T-Cell therapy.
The investigators are now analysing the data, and aim to finalise the Clinical Study Report (CSR) in 2017.
Interventional non-randomised phase I/II trial of 1st generation CD19 Chimeric Antigen Receptor (CAR) T-cell Immunotherapy directed against B-Cell precursor acute lymphoblastic leukaemia
What does this trial do?
It aims to determine the feasibility and safety of the ‘CD19 CAR’ T-Cell in children with high-risk B-cell precursor acute lymphoblastic leukaemia.
Professor Persis Amrolia, BMT GOSH
B-Cell precursor acute lymphoblastic leukaemia (ALL) is a common cancer in children. Current drug therapy and stem cell transplantation can successfully manage or cure the disease for many patients. However, 20% of children will relapse after transplant, after which no further treatments are presently available.
Several research groups have shown that genetic modification of donor immune cells (T-cells) with an artificial chimeric antigen receptor (CAR) allows them to recognise and target cancer tumours in patients.
Once the modified CAR T-cell binds to its specific protein marker on the surface of a cancer cell, a signal is sent to the body’s immune system to kill that cancer cell. Researchers on the CD19TPALL study have developed the ‘CD19 CAR’ T-cell to specifically target paediatric B-Cell leukaemic tumour markers.
Children with ALL who had relapsed after transplant, or who had a high risk of relapse or graft failures, were invited to participate and receive up to two infusions of the gene therapy, two months post-transplant.
In a unique approach, during the second part of the study, the team investigated if vaccination could be used to improve the survival/expansion of the infused T-cells.
The recruitment phase of the study has ended; it showed that vaccination could improve the persistence of CAR T-cells, but efficacy was limited. The investigators have now refined this approach further and have initiated a new study with more potent ‘second generation’ CD19 CAR T-cells.
Anthony Nolan was glad to support this innovative gene therapy study. Our transplant coordinators ensured that donors requested by transplant centres for the trial were fully informed of the aims of the study, and what would happen to them.
Donors were required to provide 80ml of blood approximately 40 days pre-transplant; usually at their pre-transplant medical visit.
We worked closely with our stem cell collection centres to ensure that donors wishing to participate were counselled and consented, and that blood samples were transported to the manufacturer’s laboratory as quickly as possible.
We also liaised with trial coordinators at Cancer Research UK and participating transplant centres to communicate the status of each donor’s involvement, and resolve any logistical problems as they occurred.
Randomised Phase II Clinical Trial of Investigative Medicinal Product (CTIMP)
What does this trial do?
It aims to determine whether allodepleted donor T-cells are safe and can be used to improve patient immunity.
Professor Persis Amrolia (UCL/ ICH)
One of the major problems following a stem cell transplant is graft versus host disease (GVHD), where the donor’s immune cells attack the patient: this can cause rash, diarrhoea, liver problems and even death.
To reduce the risk of this complication, transplants from unrelated donors are often depleted of their T-cells. However, this results in delayed immune recovery and viral infections, which can cause serious complications and even be fatal.
In this clinical trial, stem cell donors and their recipients will each provide 500ml of blood, two weeks before the donation date.
These bloods will be mixed in the laboratory, and the bad donor T-cells that react strongly against the patient and are thought to be instrumental in causing GVHD are removed, leaving the good immune cells that fight against viruses. This process is called allodepletion.
The allodepleted T-cells will then be given to the patient in three increasing doses post-transplant, to help them fight infections and improve their immunity.
In this study, the investigators will compare the recovery of immunity and the incidence of GVHD in patients treated with allodepleted donor T-cells and control patients, to see if this approach can safely be used to improve immunity after a transplant.
At Anthony Nolan, we’re providing major ongoing logistical support to the researchers undertaking this very important trial. We ensure the donor is scheduled to provide the study sample on the appropriate date. We also provide the study coordinator with confirmation of donor consent, virology results and sample collection, to ensure speedy transport to the laboratory.
We’re providing donors with written information on the study aims and objectives, to help them decide whether they wish to provide the 500ml blood sample.
We also book all travel arrangements and additional clinic appointments for sample collection.
The study plans to recruit 24 patients; recruitment will end in July 2017, with final completion of patient follow-on and data analysis one year afterwards.
For more information about our ongoing research work, visit our Research Institute hub.