Patients are being offered cutting-edge treatments, such as gene therapy, through a new Pharmacy Advanced Therapy Unit (PATU) at University Hospital Southampton (UHS).

The unit prepares doses of potentially life-changing drugs, most of which will be given to patients in the hospital’s specialist research facility, the NIHR Southampton Clinical Research Facility (CRF).

These products can be used to treat previously incurable cancers and genetic conditions.

Life-changing treatments

The unit enables research teams to deliver pioneering new treatments, known as Advanced Therapy Medicinal Products (ATMPs).

Gene therapies are used to treat or prevent disease by correcting the underlying genetic problem. These include a therapy for people with haemophilia B which was trialled in a Southampton-led trial and is now approved for treating patients in the USA.

In somatic cell therapy, cells taken from a patient or donor’s body have their DNA corrected, after which they are returned to the patient’s blood.

These treatments can transform the lives of patients with genetic conditions.

Clinical research studies

The new PATU means patients can now get early access to these treatments, by taking part in studies and trials at the facility. This new unit allows the facility to make these products on site. It means they can work with the pharmaceutical industry and developers to safely prepare and deliver these treatments.

The initial six figure sum required to establish PATU came from a pharmaceutical company, who needed these facilities to run their study, and UHS R&D, who recognised this unmet need. Since then, other companies have funded extra equipment to enhance the capabilities of the unit.

PATU was opened by Dr Helena Lee, Associate Director for Emerging Therapies at NIHR Southampton CRF. She was joined by Shingi Mutamba, Accountable Pharmacist for the unit.

Brand new capabilities

PATU has a segregated preparation area, with two mini-isolators - a contained unit that cleans the air within the area where the gene therapy is prepared. It also has a dedicated team, with two pharmacists, technicians and support workers. This enables the service to provide effective ATMPs of a high quality in a way that is safe for both staff and patients.

The results from trials and studies supported by PATU will inform how Southampton, and the rest of the country, will prepare and deliver these products as they are licensed in the UK.

Blood clotting disorder

John Curley, 52, was one of just over 50 participants who took part in a landmark gene therapy trial at UHS. Thanks to the therapy, he is now able to live a near normal life.

John was born with severe haemophilia B, a rare genetic disorder that prevents the blood from clotting, due to a lack of clotting factor IX protein. Even a small cut can be incredibly dangerous to someone living with the condition, as a wound will continue to bleed.

He used to regularly visit the hospital to have the clotting protein replaced through a drip. Later, as a teenager, advances in medicine meant he could self-administer it by injections at home.

‘Functionally cured’

In 2019, John took part in the HOPE-B study. To enable the study, one of the pharmaceutical companies who funded it gave PATU its first mini-isolator, providing a safe and sterile preparation area.

He received a single infusion of an experimental gene therapy. This gave him a new working copy of the gene that is faulty in haemophilia B, enabling his body to produce its own clotting factor IX protein.

He was one of the 96% of participants who, following the gene therapy, were able to stop their regular factor IX injections. It is what doctors would refer to as a ‘functional cure’.

Future generations

In John’s childhood, he was restricted in playing any physical sports such as rugby or football.

Today, John enjoys playing football, riding his motorbike and is able to go on holiday without having to worry about insurance or travelling with large volumes of medication.

The study was supported by the National Institute of Health and Care Research (NIHR). The results, published in the New England Journal of Medicine, offer the potential for a ‘functional cure’ for eligible people with the condition. This was John’s motivation for taking part.

He explains: “I have three sons but often think about what would’ve happened if I had a daughter. She would have inherited the abnormal factor IX gene and therefore been a carrier for the disease. If she’d have gone on to have a son, there would have been a chance that he would have haemophilia.

“I think I would have had major guilt for passing the gene on. It's amazing to think that worry could be eradicated soon and that something I was told was incurable can now be effectively managed with a single-dose treatment.”