Clinical trial to investigate treatment of acute lymphoblastic leukaemia to open at GOSH

28 Jun 2016, 2:12 p.m.

Acute Lymphoblastic Leukemia

A Phase I clinical trial (CARPALL) has opened at Great Ormond Street Hospital (GOSH) to investigate the use of immunotherapy with genetically modified T cells for acute lymphoblastic leukemia (ALL). The trial is led by Professor Persis Amrolia, who is supported by the NIHR Great Ormond Street BRC.

Relapse is the primary cause of treatment failure for paediatric ALL with 25 children in the UK dying per year from untreatable leukaemia relapse. Emerging data shows unprecedented responses to immunotherapy, with genetically redirected T cells expressing second-generation chimeric antigen receptors (CARs) enabling them to recognise and kill ALL cells. However, it is unclear how sustained these remissions are, as most responding patients have been consolidated with stem cell transplant (SCT).

The trial will take place at GOSH, University College London Hospital (UCLH) and Manchester Children’s hospital. It aims to treat 15 children and young adults with high-risk relapsed ALL to investigate the durability of responses to CD19CAR T cells, and whether this strategy could remove the need for SCT. If successful, it could mean that the short term mortality and late toxicities of SCT could be avoided.

Bagri family philanthropy enables UCL study into rare childhood neurological condition treated at GOSH

A generous donation from the Bagri family will underpin a landmark study into gene-editing treatments for Dihydropteridine reductase deficiency (DHPR deficiency), an ultra-rare childhood neurological condition, treated at GOSH

Creating communities and lasting involvement

At GOSH, patient and public involvement and engagement (PPIE) builds lasting relationships and communities that shape research over time.

Junior researchers are crucial to the future of paediatric research

Junior researchers are central to the success and sustainability of paediatric research at GOSH and the University College London Institute of Child Health.

First UK baby receives pioneering surgery in the womb for rare bowel condition

A baby boy has become the first child in the UK to undergo pioneering surgery while still in the womb for gastroschisis.