More UCLH patients getting early access to new treatments via research

In the current financial year, UCLH has recruited the first participant in the world to three clinical trials – compared with two across the whole of the last financial year. They are:

• recruiting the first participant in the world to a CAR T cell therapy trial aiming to slow  - or even halt - the progression of multiple sclerosis
• recruiting the first global participant to a study looking at how safe and effective the medicine Xromi® is for babies and toddlers with sickle cell disease
• world-first recruitment to a study testing a medicine called nirogacestat to see how well it works for treating desmoid tumours, which are rare, non-spreading but sometimes harmful soft-tissue growths.

Last month UCLH recruited the first participant anywhere in the world outside the USA and Canada to the GB-PRIME trial to investigate whether a robotically implanted brain-computer interface can improve independence for people who are paralysed.

Treatments trialled at UCLH are also going on to be approved by regulators thereby paving the way for greater access for patients. This year, the first ever treatment for the rare neurodegenerative disease Friedreich’s ataxia – for which UCL and UCLH led the UK trial – was approved for use in the UK by the medicines regulator.

And earlier this year, Autolus Therapeutics, a UCL spinout company, received authorisation from the UK Medicines and Healthcare products Regulatory Agency for a next-generation CAR T-cell therapy developed to treat adults with an aggressive blood cancer. The authorisation follows trials carried out at UCLH.

As well as global firsts, UCLH has recruited the first participant in Europe on four occasions and the first UK participant on nine occasions. These figures to date represent an improvement on last year.

The figures underline UCLH’s ability to attract, set up, and begin commercial studies at speed - at a time when the life sciences sector has highlighted study set-up times as a crucial area for improvement across the UK.

This year’s European and UK ‘research firsts’ at UCLH include:

• recruiting the first European participant in a trial in Myotonic Dystrophy Type 1 (DM1), a genetic condition causing progressive muscle weakness
• being the first site in the UK to recruit to a Parkinson’s disease trial testing whether the drug ambroxol can improve motor and non-motor symptoms
• recruiting the first UK participant in a study of a new anti-cancer drug combination for advanced solid tumours such as ovarian and endometrial cancers.

Working with industry is a core part of UCLH and UCL’s research strategy. Commercially sponsored trials play a vital role in making the UK the best place in the world to do clinical research. These achievements are supported by the NIHR UCLH Biomedical Research Centre, the NIHR UCLH Clinical Research Facility, and the UCLH/UCL Joint Research Office.

Mihaela Sutu, Commercial Research Business Development Manager within the UCLH/UCL Research Directorate, said: “Thanks to our highly skilled scientists, clinicians, research delivery teams and world-class infrastructure, we’re on track to deliver more commercial research firsts than before - with 16 recruitment 'firsts' so far this financial year. It is an example of how we’re responding to the life sciences industry’s call to strengthen set-up and delivery of commercial trials. And the ultimate beneficiaries are the patients receiving life changing care.”

Find out more about commercial research at UCLH and UCL and use our dedicated email if you are a sponsor or CRO: uclh.commercial-research@nhs.net