Delivery of novel therapies and biomarkers

During BRC3 our researchers have successfully pulled novel therapies and biomarkers through into clinical trials:

  • Professor Mike Ehrenstein led the BEAT-Lupus trial which completed recruitment in 2020. The BEAT-Lupus trial administered belimumab after rituximab to patients as treatment for SLE. This was the first double blind randomised controlled trial of a biologic therapy to show a clinical benefit for systemic lupus erythematosus (SLE). The results will be submitted to NICE and are expected to change clinical practice.
  • METRIC is the largest prospective multicentre diagnostic accuracy study of MRI and ultrasound in Crohn’s disease to date led by Professor Stuart Taylor. The trial results have directly impacted on NHS practice, providing hard evidence that tests exposing patients to ionising radiation (with the risk of cancer induction) should be replaced by safer, cost-effective, and well tolerated alternatives. The work has directly influenced the recommendations of international (European Crohn’s and colitis organisation) and national (British Society of Gastroenterology) clinical practice guidelines, the former also including new guidance on training curriculums.
  • A Phase 1 clinical trial of a new X-CGD Gene Therapy supported by both UCLH and GOSH BRC’s showed promising results as an effective treatment for some patients. X-CGD is an inherited condition which causes repeated life-threatening infections for which currently the main alternative was a stem cell transplant from a matched donor which comes with a potentially life-threatening risk. This new treatment works using the patient’s own stem cells removing the risk of rejection.
  • The DESPIAD trial opened in 2018. DESPIAD is a trial for a new treatment of Alzheimer’s disease, miridesap. Alzheimer’s is caused by the presence of amyloid plaques in the brain. Serum amyloid P (SAP) is a normal protein that occurs in everyone, small amounts can travel via the bloodstream and reach other organs including the brain where it binds to the proteins in the amyloid plaques and stops them breaking down. Miridesap was developed by Professor Sir Mark Pepys from the UCL Wolfson Drug Discover Unit, the drug works by successfully removing SAP from the bloodstream and stops SAP from reaching the brain. Miridesap may also remove SAP already present in the brain. It is hoped that this drug may reduce brain damage caused by Alzheimer’s disease.
Pipette adding fluid to one of several test tubes