Gene therapy offers hope to head and neck cancer survivors with radiotherapy-induced dry mouth

The condition, caused by irreversible damage to salivary glands during radiotherapy, severely impairs speech, eating, and quality of life.

The AQUAx2 study is the first UK trial to test the gene therapy AAV2-hAQP1, which uses a viral vector to deliver the Aquaporin 1 (hAQP1) gene to damaged salivary glands. This gene helps the glands transport water into the mouth, offering a single-dose solution to restore moisture and reduce symptoms. Sponsored by MeiraGTx, the international study spans the UK, US, and Canada.

Led by Professor Stefano Fedele (Head of Clinical Research and Programme Director, MSc in Oral Medicine at UCL Eastman Dental Institute)  and supported by UCLH teams, the trial addresses an unmet need for effective treatments, as current options provide limited relief and come with side effects.

Professor Fedele said: "I am incredibly proud to be part of this research.  It tackles an unmet need and offers UCLH patients a new regenerative medicine intervention that we hope will revolutionise the treatment of this debilitating, and so far considered irreversible, condition. Setting up this study has been a team effort and it would have not been possible to deliver it without the help and hard work of multiple UCLH staff and committees, as well as the NIHR UCLH BRC, to whom I am very grateful."

Nathaniel Rackowe (pictured), a London-based artist, is the first patient to receive the therapy. He hopes it will not only help him but also improve post-cancer lives for others.

Further information

• Source: NIHR UCLH Biomedical Research Centre
• Professor Stefano Fedele academic profile
• Main image (NIHR UCLH BRC) Left to right: Christopher Banzuela - Research nurse, Nathaniel Rackowe – Trial participant, Prof Stefano Fedele - Professor of oral medicine, Rachel Knight - Senior clinical trials manager, Alpha Cham - Research nurse, Sara Titus - Research dental nurse