GD2 CAR-T Cell Therapy Clinical Trial
Introduction and Key Funding
In a groundbreaking clinical trial, 12 children in the UK will be the first to access CAR T-cell therapy for DIPG/DMG brain cancers. Following promising results from a similar study at Stanford University in the US, a £1.2 million grant has been awarded to a team of researchers at University College London (UCL) and Great Ormond Street Hospital (GOSH) to conduct this trial.
Principal Investigators
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Dr. Karin Straathof: Associate Professor at the UCL Cancer Institute and Honorary Consultant at GOSH
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Prof. Darren Hargrave: GOSH Charity Clinical Professor in Paediatric Neuro-Oncology and Honorary Consultant at GOSH
Research and Clinical Application
Dr. Karin Straathof and Prof. Darren Hargrave will employ the ‘GD2-CAR-T’ therapy, which has been previously used in UK trials for neuroblastoma, a children's cancer. The trial will be conducted at GOSH, with patients referred from across the UK from all treating centres.
Development of New Treatment for DMG
Researchers at UCL and GOSH aim to develop a new treatment for diffuse midline glioma (DMG), an aggressive type of brain tumour of the midline structures including diffuse intrinsic pontine glioma (DIPG). The treatment will use the patient’s own immune system to attack the cancer cells. DMG is a devastating cancer with some of the worst outcomes for children, and this clinical study will involve CAR T-cells, which are engineered to recognise and eradicate cancer cells.
How it works
GD2 CAR-T Cell Therapy uses the body's own immune system to fight cancer.
Previous Success with CAR T-Cell Therapy
CAR T-cell therapy has previously been used to successfully treat some childhood leukemias. Dr. Straathof’s team has also used this technology in a clinical study for neuroblastoma, a rare cancer that develops in nerve cells. They engineered T-cells to target a protein called GD2, found on neuroblastoma and DMG cells, marking a crucial step towards developing effective treatments.
Challenges of Treating DMG
Brain tumors are the most common cause of cancer-related death in children, with DMG being the most aggressive. DMG tumors develop in critical parts of the brain, making surgical removal impossible. Radiation therapy offers only temporary relief, often followed by more aggressive tumor growth. This trial at GOSH aims to develop effective CAR T-cell treatments for DMG and other high-risk brain tumors.
Trial Details and Eligibility
With required funding in place, the GD2 CAR-T cell therapy study has undergone all the rigorous reviews and approvals, and is now receiving patients. Recruitment will be open to:
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Patients with a brainstem or diffuse midline glioma proven by biopsy.
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Newly diagnosed patients who have or will undergo standard radiotherapy treatment and have not relapsed.
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Patients eligible to receive NHS treatment in the UK.
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Patients meeting all other study eligibility criteria as assessed by the patient’s local and the GOSH study team.
Due to the nature of this therapy and safety regulations, there will be limits on the number of patients treated simultaneously.
Role of Abbie’s Army in Funding
The charity Abbie’s Army has played a crucial role in funding this trial. Leveraging its position within the childhood cancer community, the charity has contributed a collaborative grant of £275,000 to the GOSH Children’s Charity. Additional funding has also been directed by the charity from other foundations in the sector.
Support from Other Parent-Led Charities
Our financial contribution to the trial included support of six other parent-led charities (in order of donation size) who wanted to help : Islastones Foundation, Bradley Lowery Foundation (including Kaleigh’s Trust and The Spider Ede Appeal - DIPG Awareness), Robert Andrew Munro Foundation, Eva’s Angels, Doing It For Daniel, and Edie’s Kindness Project.
Coordination of Funding
The coordination of funding between Abbie’s Army and the other primary funding groups has been facilitated by GOSH Children’s Charity.
Funding Details
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Phase 1 Clinical Trial of GD2 CAR-T Cells for 12 Patients: £1.2 million
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Abbie’s Army Grant Award: £275,000
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