MIGHTY

• Research type

  Research Study

• Full title

  Multi-modular Chimeric Antigen Receptor T cells targeting B7-H3 in Children, Teenage & Young adult sarcoma

• IRAS ID

  1009001

• Contact name

  Karin Straathof

• Contact email

  k.straathof@ucl.ac.uk

• Sponsor organisation

  University College London

• ISRCTN Number

  ISRCTN75533638

• Research summary

  Rhabdomyosarcoma (RMS), Ewing sarcoma (ES) and desmoplastic small round cell tumour (DSRCT) are cancers known as sarcomas. They develop in bones or surrounding soft tissue such as muscle. For some patients chemotherapy, radiotherapy and surgery can control and sometimes cure their disease. However, there are patients whose disease returns or does not respond to treatment and their outcome is often poor.

  Chimeric antigen receptor (CAR) T cells are blood cells genetically engineered to recognise and kill tumour cells. Lasting tumour clearance has been achieved in leukaemia and CAR T products have been approved for use as part of standard treatment for some blood cancers. Early clinical trial data indicate the CAR T cells may also work in non-blood cancers. Currently there are no CAR T cells approved for sarcoma.
  We have developed CAR T cells that are reprogrammed to recognise a specific target (B7H3) on the sarcoma cells. MIGHTY aims to test whether giving these CAR T cells to patients with RMS, ES or DSRCT is safe and what dose to use.

  T cells are collected from patient’s blood to make the CAR T cells. The CAR gene is put into the T cells so they find and attack the sarcoma.
  Patients have 2 chemotherapy drugs to make space for the CAR T cells. The CAR T cells are then given into a vein. Patients are monitored in hospital for at least 2 weeks. MRI/CT scans are used to look at the effect on the tumour.

  MIGHTY has been developed by clinicians, scientists and patient advocates brought together by the NextGen Cancer Grand Challenge Initiative. It is one of 3 studies in the UK and US assessing safety of CAR T cells in solid tumours in children, teenagers and young adults.

• REC name

  London - West London & GTAC Research Ethics Committee

• REC reference

  24/LO/0701

• Date of REC Opinion

  6 Dec 2024

• REC opinion

  Further Information Favourable Opinion