Hepatocyte Microbeads for ALF

• Research type

  Research Study

• Full title

  Hepatocytes co-Encapsulated with mesenchymal stromal cells in alginate microbeads for the treatment of acute Liver failure in Paediatric patients (HELP)

• IRAS ID

  199623

• Contact name

  Anil Dhawan

• Contact email

  anil.dhawan@kcl.ac.uk

• Sponsor organisation

  King's College Hospital NHS Foundation Trust

• Eudract number

  2019-000316-29

• Clinicaltrials.gov Identifier

  NCT05491135

• Duration of Study in the UK

  12 years, 7 months, 1 days

• Research summary

  Acute Liver Failure in children is associated with high mortality without liver transplantation. In addition, donor organ shortage makes it difficult to provide this treatment to every potential patient. Liver transplantation is life-saving but it carries the risk of major surgery and complications from lifelong antirejection drugs to suppress the immune system. If bridged across the immediate crisis following acute liver failure, the immense regenerative potential of the liver means that the patient’s own liver may ‘re-grow’. This period is very time sensitive. Unfortunately, if the vital synthetic and detoxification function of the liver is not provided, the patient will often die before the liver can re-grow.
  Transplantation of liver cells (hepatocytes) can provide this ‘bridge’ with considerable advantages over whole organ transplantation. Firstly, hepatocytes are derived from donor livers which are otherwise unsuitable for transplantation. Secondly, unlike whole organs, they can be frozen and stored, thus act as an ‘off the shelf’ treatment. Thirdly, the technique of hepatocyte transplantation within microbeads coated with alginate (a gel originating from seaweed) and infused into the abdominal cavity is much less invasive than liver transplantation. Finally, the alginate protects the cells against the body’s immune system, avoiding the need for immunosuppressive drugs and the associated major risks. We have demonstrated the feasibility of intraperitoneal transplantation of hepatocytes alone in alginate microbeads in 8 children with ALF.
  Our previous work has shown that putting liver cells together with support cells called mesenchymal stromal cells (MSCs), can significantly improve the ability of hepatocytes to survive and function within the alginate microbead. This is a first in human clinical trial to assess whether HMB002 will be a feasible and effective form of cellular therapy for acute liver failure in children, as a bridge treatment to liver transplant or lead to regeneration of the native liver

• REC name

  London - West London & GTAC Research Ethics Committee

• REC reference

  22/LO/0292

• Date of REC Opinion

  21 Jun 2022

• REC opinion

  Further Information Favourable Opinion