Opportunities to re-purpose existing approved drugs to potentially treat children with cystic kidney disease lie at the heart of Professor Johnson’s Action funded work at the University of Leeds.
What inspired you to investigate this particular area?
I’ve been working on a group of inherited developmental disorders called ‘ciliopathies’ for the past 15 years. These conditions invariably have cystic kidney disease as a feature, and are a major cause of childhood morbidity and mortality. About 5,000 patients in the UK need either dialysis or transplantation every year as a result of kidney failure.
However, there are few effective drug treatments that can prevent or slow cystic disease progression in patients. I’m excited about opportunities to ‘repurpose’ existing approved drugs as potential treatments of cystic kidney disease. Re-purposing of known drugs can be rapid, allowing patients to benefit more quickly from research findings.
What does Action funding for this study mean to you?
We really appreciate this funding from Action: it will make a huge difference to this project. It’s often difficult to get funding to bridge the gap between a promising, but preliminary research finding and more complete testing in further studies. Action saved the day and the funding is allowing a proper exploration of the most interesting clinical development compounds and approved drugs.
What does a typical day look like for you … or is every day different?
One of the real joys of the job is that every day is different and it’s never boring. I love talking to my colleagues about their research, and it’s often the quick corridor conversations that spark a train of thought and are the most effective. I always talk to my team every day about their research and we celebrate the successes, commiserate on the failures and then decide on the next steps. Often, things don’t go to plan, and we need to work out if we stick at it, or move on and try to answer the research question from a different angle. This can be a calculated risk and we don’t always get it right. In between, I fit in teaching, supervision of students, peer review of grant applications… and there are always loads of emails!
Can you tell us a bit about your team?
There are seven people in my group. They are all working on different aspects of either genetics or the biology of inherited conditions. They include post-docs, a research associate, PhD students and an MSc student. We’re using some exciting cutting-edge technology including next generation sequencing, super-resolution microscopy, CRISPR-Cas9 genome editing and cell-based screening.
Who’s your research hero, and why?
I greatly admire Nikolai Vavilov, a geneticist in Soviet Russia who paid for his research with his life: a terrifying example of the damage that political ideology can do to reason and scientific debate. In my own scientific career, I have been taught by an inspiring succession of teachers and mentors. I try to follow their examples in the supervision of my students and mentorship of colleagues.
As a charity, Action began in 1952 with our founder’s quest to find a cure for polio. What led you to a career in medical research?
I had a fascination with genes and genetics from an early age after reading Richard Dawkins The Selfish Gene and set myself a clear and somewhat precocious goal of eventually doing research in the subject.
Action’s loyal and lovable mascot Paddington Bear™ is very fond of marmalade sandwiches. What’s your favourite snack?
Peanut butter and jelly sandwiches: I picked this up from my time in the States.
Tell us something that will surprise us!
I’m bilingual in Russian. I’ve enjoyed reading Pushkin and Tolstoy in the original.
Find out more about Professor Johnson's three year study here.