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Cell Therapy

Local or systemic injections of cells, fibroblasts, mesenchymal stromal cells (MSC’s) and other bone marrow derived cells, in an aim to ameliorate the condition. The aim is to eventually replace the missing protein with gene modified/gene edited cells which are patient derived therefore reducing rejection risks

Professor John McGrath, Leading EB Researcher and dermatologist. Guys and St Thomas’s, King’s College London, UK.  Clinical Trials to inject fibroblasts locally and establish effectiveness of the blood stream delivery of a specific subset of bone marrow cells (mesenchymal stromal cells) plus injections of gene modified fibroblasts

Professor Jakub Tolar, Stem Cell Institute, University of Minnesota, USA. Pioneered the use of Bone Marrow Transplantation and working on iPSC’s for RDEB and gene editing for RDEB using a variety of ‘molecular scissors’

Published Materials

British Journal of Dermatology

2013 EB TALEN Molecular Therapy

EB TALEN in Nature News 2 May 2013

EB stem cells

Startribune:”Informatics” helps doctors unlock medical mysteries in mounds of data

Local or systemic injections of cells, fibroblasts, mesenchymal stromal cells (MSC’s) and other bone marrow derived cells, in an aim to ameliorate the condition. The aim is to eventually replace the missing protein with gene modified/gene edited cells which are patient derived, therefore reducing rejection risks.

Fibroblast therapy

Professor John McGrath, Guys and St Thomas’s, King’s College London, UK. Clinical Trials to inject fibroblasts locally and establish effectiveness of the blood stream delivery of a specific subset of bone marrow cells (mesenchymal stromal cells or MSCs) plus injections of gene modified fibroblasts

Further information

 

Mesenchymal stromal cell therapy

EBSTEM

This was the first UK trial aiming to lead to a treatment for children with RDEB. It sought to establish if Mesenchymal Stromal Cells (MSCs) from unrelated donors who do not have EB can benefit children with RDEB in a safe efficaceous way. It led to reduced skin inflammation, blistering and better wound healing.

BBC news report

Journal of Investigative Dermatology article

 

ADSTEM

A follow-up study in adults understanding how allogeneic MSCs given intravenously can modify disease severity in RDEB, Results from EBSTEM suggested that individuals responded differently and there may be factors that make them good or poor responders. Studying the safety profile of MSCs in adults is important because they have greater scarring and there is an increased risk of skin cancer.

Update: this trial has now ended and sample analyses and monitoring have begun

 

LENTICOL F

Dr Waseem Qasim and Professor Adrian Thrasher, Professor John McGrath, Institute of Child Health, Guildford Street, London, UKA set of clinical trials. This treatment is effectively correcting a sufferer’s owns cells, to be given at local sites, and then working towards correcting cells for intravenous use, aiming to give a whole body treatment.

Clinical trial info

To follow Lenticol F:

Gene Therapy Lenticol M gene modifying patient mesenchymal stromal cells


Fibrocell
 – clinical trial info

Professor Jakub Tolar, University of Minnesota, USA. Pioneered the use of Bone Marrow Transplantation and working on iPSC’s for RDEB and gene editing for RDEB using a variety of ‘molecular scissors’