The Advanced Therapeutics & Experimental Medicine Cluster (Cluster 1) will integrate and support Theme-based Programmes in cell-, protein/peptide- and small molecule-therapeutics, harnessing critical study-enabling BRC infrastructures (CRF; Advanced Therapeutics Manufacturing (GMP) Unit; Clinical Trial Hub).
We have an extensive phase I pipeline and robust examples of phase II pull-through (e.g. adoptive regulatory T-cells, peptide immunotherapeutics, cytotopic/cytoprotective inhibitors). Cell-based therapeutics across Themes (Cardiovascular Disease, Infection & Immunity, Transplantation, Regenerative Medicine, Cutaneous Medicine and Oral Health) includes ground-breaking, first-in-human studies of regulatory and chimaeric-antigen-receptor gene-modified T-cells.
Innovation in protein-based therapeutics includes developing first-in-class melanoma-specific IgE monoclonals; new cytotopic strategies (coagulation inhibition); and cytokine modulation (low-dose interleukin-2 to expand endogenous/adoptive Tregs). Advanced peptide-based therapeutics are slated for a phase II evaluation in diabetes patients stratified by genotype (industrial partnership). Novel small molecule strategies include modulators of neuronal NOS for hypertension/heart failure (first-in-human) and tooth repair strategies centred on dentine signalling pathways.
Our research programmes cut across our BRCs nine research themes and integrate shared interests in drug modalities across all of our research clusters. The selection of projects listed below demonstrates the breadth of studies in which we are engaged:
- Cutaneous medicine programme on Cell Therapy in Rare Diseases in particular on Epidermolysis Bullosa
- Cutaneous medicine programme on therapeutic development of melanoma
- Immunity & infection programme on cancer, in particular on gamma-delta cells and CAR-T cells.
- Immunity & infection programme on diabetes, in particular on Type 1 diabetes
- Transplantation programme on cell therapies, in particular on liver and renal transplant.
- Oral Health Programme on tooth repair and regeneration and oral soft tissue restoration.
The advanced therapeutics and experimental medicine cluster has initiated significant biomedical and translational research studies focused on novel human therapeutics.
Our key successes include
- Type 1 diabetes first-in-man trial of a novel prototype peptide immunotherapy developed at Kings College London (KCL) for type 1 diabetes. With a next generation drug developed, a first-in-human study is currently under way and a Phase II study is being developed with a commercial partner.
- The first in human trial using naturally occurring regulatory T-cells as adoptive therapies to counter allograft rejection in renal transplantation has completed. A phase II study, in collaboration with Oxford University has been successfully awarded and is awaiting regulatory approval. A parallel regulatory T-cell therapy is also under development for the treatment of Crohns disease.
- A cell therapy trial for Epidermolysis Bullosa (EB) ran between June 2013 and 2015 where the first patient was dosed in a first-in-man study to evaluate the safety of allogeneic mesenchymal stem cells (MSCs) in children with EB. The results showed that treatment with MSCs improves both the appearances of the skin and the overall quality of life for children with EB and further similar studies focused on cell-based regeneration are ongoing.
- The cluster has also initiated a first in man clinical trial of T-Cell therapy for head and neck cancers, the 4th cohort of patients is now in recruitment
The cluster utilises a number of the services on offer across KHP. These include regulatory advice and support from the KHP Clinical Trials Office (CTO) and partner R&D departments at Guy’s and St Thomas’ NHS Foundation Trust and King’s College Hospital.
We also have access to clinical trial statistics, databases and management from King’s Clinical Trials Unit (KCTU), IP and licensing support and commercialisation advice from the King’s Commercialisation Institute
All of the cluster’s research investigators collaborate with their peers from other academic institutions in the UK and abroad as well as with commercial and non-profit organisations such as charities.
The cluster board is led by Professor Mark Peakman and brings together senior research clinicians from across Guy’s and St Thomas’ NHS Foundation Trust and King’s College London.
The cluster board meets monthly and comprises world class experts who are adept at translating research from the laboratory into clinical environments. It has oversight of the cluster’s portfolio acting as a forum to accurately and effectively support and monitor the activity within the cluster.