Our Clinical Trials
Bringing our research to children with solid tumours.
” The opening of these trials represents hope for children diagnosed with these tumours in the future. Parents and patients have been waiting decades for new treatment options for childhood solid tumours. Finally, a new pioneering T-cell treatment option is in development, illustrating that change is happening and research is being taking forward to the clinic despite the challenges faced by scientists and medical professionals in the sector.”
In order to expedite the development of Next Generation T-cell therapy for children with solid tumours, central to the work of NexTGen are three clinical studies.
The clinical trials will translate approaches learnt through the research within NexTGen work packages into the clinical setting. These will be phase 1 studies, meaning the first time a new medicinal product has been tested in humans and so it investigates safe dose range, potential side effects and feasibility as a treatment.
One trial will be based in the United Kingdom at Great Ormond Street Hospital and University College London Hospital and the other two trials will be based in the United States at Children’s National Hospital, Washington DC and Dana-Farber Cancer Institute, Boston.
All the trials will use the patient’s own T-cells as a starting point. These immune cells will be engineered with a chimeric antigen receptor (CAR for short) which enables to T cells to seek out tumour cells that have B7-H3 on the outside. B7-H3 has been chosen as the target as it is widely expressed on several cancer types that are common in children and young people. The trial at Children’s National Hospital will administer this CAR in conjunction with T-cells natively specific for a second tumour marker known as PRAME. Additionally the T cells are are engineered to make them resistant to Transforming Growth Factor Beta, a substance produced within tumours that has a strong dampening effect on immune cells.
The trials in the UK and Washington DC will enrol patients with relapsed or refractory tumours in particular sarcomas including rhabdomyosarcoma,Ewings sarcoma and desmoplastic small round cell tumour. In these clinical trials, the CAR T-cells will be given to the patient into a vein.
At Dana-Farber Cancer Institute patients with brain tumours including atypical teratoid rhabdoid tumour (ATRT) and medullobastoma will be enrolled in the clinical trial. In this trial, the CAR T-cells will be administered into the fluid around the brain.
Please watch the videos below to learn more about CAR T-cells and our clinical trials.
Two clinical trials are now open for recruitment: in the UK at UCLH and GOSH; and in the US at Children’s National Hospital in Washington D.C.
UK and US Centres have the required referral information. Patients or their parents or carers who would want to consider taking part in this clinical study can be referred to our clinical trials team by their own oncology consultant.
US oncology centres will similarly be informed when the clinical trial at Dana-Farber Cancer Institute is open for recruitment.
