DevelopAKUre: A patient-led clinical trial

2014 Conference

Abstract: DevelopAKUre is one of the UK’s first patient-led clinical trials. We aim to test a possible treatment, a drug called nitisinone, in patients diagnosed with alkaptonuria (AKU). AKU is a rare, genetic disease. Due to a malfunctioning enzyme, patients are unable to break down protein correctly and instead produce a black pigment, called HGA. HGA stains their urine dark, turns their bones black, and makes cartilage brittle. This means patients experience very severe osteoarthritis early in life. Currently the best treatment for patients is through surgery to replace their broken joints; with most patients having many different surgeries replacing nearly all major joints in their body.

The AKU Society is a patient group dedicated to finding a treatment for the disease. We funded basic research in Liverpool, built an international consortium and in 2012 successfully gained a grant from the European Commission to run clinical trials in Europe. We believe this is the first time a UK patient group has led clinical research. Now that the trials are underway, we lead on identification of new patients, recruitment and support through the almost 6 years of studies.

This talk will teach the public about the importance of clinical research, that it can be led by patients and not just doctors or private companies. It will also look at the connections between patient groups and industry; one of our partners is a medium-sized pharmaceutical company and we believe the future of research must include more collaborations between patients and companies.