The Visionaries: Sight for sore eyes

The eye has been a focus for several companies and academics to realise the potential of stem cell therapy. As the eye is immunoprivileged there is less chance of rejection or immune response. Also, end outcome is easier to access, and if required the cells can be directly observed at site of transplantation
The Institute of Opthalmology (IoO), UCL is at the forefront of most of developments with pioneering research, moving forward basic science into clinical trials.
Currently there are 2 clinical trials being undertaken in the UK in opthalmology using cell ular therapy: At Newcastle University using ex-vivo expanded limbal stem cells, and Advanced Cell Technology using Retinal pigmented epithelial (RPE) cells for Stargardt's disease at Moorfields Eye Hospital and Princess Alexandra Eye Pavillion, Edinburgh

Professor Francisco Figueiredo at Newcastle University is leading a Phase II clinical trial using Autologous limbal stem cells for unilateral limbal stem cell deficiency. The trial involves taking limbal stem cells from the patients healthy eye, which are then grown on an amniotic membrane expanded ex vivo and transplanted onto the eye. This can be used to treat patients who have had damage to their eye by acid/alkali that is very painful. Results from their first trial, showed significant decrease in pain in every patient and improvement in eye sight. The GMP manufacture for the current clinical trial is undertaken at the Newcastle Bioscience Cellular Therapy Facility. Look at this presentation for more details of the IMP manufacture.

House of Lords regenerative medicine report - what they recommend

The House of Lords Science and Technology committee have released their Regenerative Medicine report. This rather rigorous report was put together after a call for evidence last year, and has now been compiled to give an overview of the current landscape, in terms of current clinical trials and companies in Europe; translation in regards to the regulatory environment and manufacturing; aswell as commericialisation. 

Roslin Cells granted MIA (IMP) licence

Fantastic news from Scotland where Rosilin Cells Ltd. have announced they have been granted by the Medicines and Health Regulatory Authority (MHRA) a Manufacturer's Authorisation for Investigational Medicinal Products, MIA(IMP), for their GMP cellular therapy facility. In addition they have been awarded a 'Specials' licence that covers manufacture of ATMPs for specific patients. 

The company operates a seven clean room GMP manufacture facility tin partnership with the Scottish National Blood Transfusion Service (SNBTS) within the Scottish Centre for Regenerative Medicine (SCRM). Roslin Cells are focused on the development and manufacture of human embryonic stem cell (hESC) and induced pluripotent stem cell (iPS) lines, and have a collaboration with ACT.
Importantly this facility will now be available to companies and academic researchers under this licence

Roslin Cells press release

Cell therapy clinical trials: Industry

Since our last commentary on the Cell Therapy Catapult clinical trials database, they have released an update expanding to include 34 clinical trials currenty operating in the UK.  
This post will give more detail on the commercial companies sponsoring UK clinical trials in cell therapy. 

Azellon Cell therapeutics is a spin out company from University of Bristol building on the pioneering work from Professor Anthony Hollander. Professor Hollander was also part of the team involved in the first tissue engineered trachea transplant in Claudia Castillo, as the only stem cell scientist. Azellon have a Phase I/II clinical trial approved for treatment of knee meniscal repair using bone marrow derived mesenchymal stem  cells (MSCS) infused onto a biological scaffold.

Reneuron are a publicly limited company, who are the foremost stem cell company in the UK. Their PISCES (Pilot Investigation of Stem Cells in Stroke) is the 'world's first clinical trial of a neural stem cell therapy for disabled stroke patients'. While this is just a safety study, promising initial results application has been made for commencement of a phase II study later this year.

Cell therapy clinical trials: Part 1 - Academia

The Cell Therapy catapult recently published a database for the current UK clinical trial in cell therapy (See here).
It details 21 studies and it's quite striking that the majority of these are academia, with only ReNeuron, Cell Medica, ACT and Azellon Cell Therapeutics (Uni. of Bristol spin out) representing commercial companies and only Cell Medica at later stage clinical trials.

This post will concentrate on the great work that is being translated into clinical trials from academia

Cell Type & indications

The majority of these clinical trials are for autologous therapies.

Professor Anthony Mathur, Queen Mary University of London, is leading 3 clinical trials. REGENERATE trials for acute myocardial infarctionchronic heart failure and dilated cardiomyopathy use autologous bone marrow derived mononuclear cells (BM-MNCs) injected either intra-coronary or intramyocardial.
Prof. Mathur is also heading a European wide phase III trial using BM-MNCs for AMI in 3000 patients.  This trial will assess the effect to all cause mortality. With such a large recruitment, although results are not expected until 2017 it will be clear the importance of this therapy for this indication.

Stem Cells in rapidly evolving active multiple sclerosis (STREAMS), led by Dr Paolo Muraro. This use autologous bone marrow derived mesenchymal stem cells that have been cultured for 'up to 52 days'.

The TRANSEURO clinical trial is run by a European consortium to deliver fetal dopaminegeric mid-brain grafts to patient's with Parkinson's Disease.

Professor Nagy Habib, Imperial College London, is used bone marrow (and leukapheresis) derived expanded CD34+ adherent cell population for liver insufficiency, diabetes, and ischemic stroke. These trials have been ongoing since 2007, emphasising the trend of a much prolonged period for completion from academia led trials compared to commercial.

Great Ormond Street Hospital is conducting a phase I trial using gene therapy to produce 'safe' T cells to prevent GvHD . University College London also has a gene therapy T cell product for acute lymphoblastic leukaemia.

Birmingham and Edinburgh University have formed a collaboration conducting phase II trials using autologous CD133+ selected haematopoetic stem cells for liver cirrhosis

Prof. Francisco Figueiredo at Newcastle University is leading Phase II trials for limbal stem cell deficiency using autologous limbal stem cells from the untreated eye. Edinburgh University is targeting a very similar indication in corneal stem cell deficiency using an allogenic source of corneal cells manufactured on an amniotic membrane. The Institute of Opthamology, London, has a very similar product due to go into clinical trials at the end of this year

Cell Therapy UK - Intro

This blog will aim to keep track and updates on all cell therapy clinical trials and news, from academia and business in the UK. With the installation of the cell therapy catapult hopefully this will spark a new era that can really slingshot this new pillar of medicine into reality