25 January 2022
A woman in her 30s from the Midlands is the first patient globally to be recruited to a new trial, thanks to doctors at the Royal Free London.
The patient is part of a study called PHYOX7, run by Dicerna, aimed at evaluating the safety of a drug for people with a rare inherited genetic condition where oxalate is generated by the liver.
Oxalate is a substance found in many foods, but if there is too much it leads to oxalate crystals, causing stones in the kidneys and urinary tract and,in rare cases, kidney failure. People with primary hyperoxaluria don’t create enough normal enzyme in their liver to prevent the oxalate accumulating.
Dr Shabbir Moochhala (pictured left), investigator for the study and consultant nephrologist at the Royal Free Hospital, said: “The Metabolic Kidney Stone Service at the Royal Free Hospital is being recognised as a national specialist centre for rare kidney disease and a leading provider of clinical trials, which is why we were chosen to run the trial and the patient was referred to us from their local hospital. The patient will receive an injection once a month and the hope is that the drug will block the way the disease works and stabilise her condition.
“One of the things we are also doing is trying to encourage testing in other family members and other patients who might be at risk. This is a genetic disease and the more we can get stone-forming patients tested the better chance we have of trying to delay and disrupt the progress of the disease. This is a really important aspect of the work of the rare disease clinical research network which we are a part of.”
In addition the Barnet and Chase Farm Hospitals rheumatology research team has accepted the first patient in the UK on to the Novartis ONWARDS study.
This five year study will assess how the use of a protein has the potential to rebuild cartilage and therefore potentially slow or reverse the damage of osteoarthritis of the knee, which affects millions of people worldwide, and for which there is currently no therapy that can reverse the damage in the joints. The study is looking to recruit 550 patients globally.
Dr Hasan Tahir (pictured right), consultant rheumatologist at Barnet Hospital, said: “This is an exciting study to be part of and could potentially prove an important milestone in our quest to find improvements for osteoarthritis, which is a painful and all too common condition and can make it difficult to carry out everyday activities. I hope we can recruit more patients to the trial and work to improve the lives of people with this debilitating condition.”
Farhan Naim, director of research and development at the Royal Free London, said: “The speed at which we are recruiting to trials is great news for our patients. We are rightly proud of our reputation for being a research led trust and are committed to making ground breaking discoveries that could help patients worldwide.”