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Academic spotlight: Professor Amit Nathwani
Professor Amit Nathwani (UCL Cancer Institute) founded Spur Therapeutics to develop innovative gene therapies for inherited diseases, transforming patients' quality of life.
26 November 2024
We spoke to Professor Amit Nathwani about his remarkable journey, from his groundbreaking work in gene therapy to founding Spur Therapeutics, and his mission to improve the lives of patients with inherited diseases.
Tell us more about your background and connections to India
I was actually born in East Africa, in rural Uganda, to parents who both originated from India, in the state of Gujarat. We left for the UK as refugees when I was 12 and moved initially to an army barracks near Gatwick airport. I couldn’t stop looking at all these flights coming in and out and was quite excited - I had never even seen a traffic light. Later I had the great opportunity to go and study medicine in Aberdeen and my favourite subject there was therapeutics.
How did you become interested in haematology?
I was always interested in drug development and how drugs work. I finished university, did my medical training and then loved the idea of haematology, primarily because as a junior doctor, I had the experience of taking a sample from a patient with leukaemia, taking the sample to the lab, getting it stained, looking under the microscope and making the diagnosis, and then an hour later going back to the patient, discussing the diagnosis and starting treatment. There aren't many branches of medicine where you can do that. It’s a nice journey, and it got me hooked on haematology.
Tell us about your journey into gene therapy
When I was doing my PhD, what was really exciting to me was trying to understand the regulation of a gene that's important for starting coagulation blood clotting. It's called tissue factor because it's abnormally expressed in a variety of different pathological states, including infection. So we wanted to understand what turns it on and what turns it off. As part of trying to understand that we were busy electrocuting human umbilical endothelial cells harvested from cord blood, and then transfecting them to introduce the DNA and measure a bacterial protein that we had introduced into these cells. That was really fascinating and I wondered if I could do that for medical conditions. This could potentially be curative.
Having grown up in East Africa, I had seen first-hand the impact of infectious diseases and how many kids die at a young age because of them, and also the impact of vaccination. I felt that if we could develop a new set of therapies that could offer a 'one and done' opportunity for patients, that would be amazing.
Can you tell us more about your work with gene therapy for haemophilia?
My initial focus was trying to develop gene therapy for thalassaemia and sickle cell disease because these were poorly served conditions. Right in front of my eyes, under the microscope, I could see on a daily basis that the genetically modified cells were declining with time as they were dividing. So I went back to my hemostasis roots and started working on gene therapy for haemophilia.
Haemophilia B is is pretty rare, affecting one male out of probably 30-50,000 newborns. Haemophilia A is more common and it affects about one male out of maybe 10,000 newborns. Going back 60 years, there was no treatment for patients with haemophilia. Patients died at a young age or were admitted for long periods of time because of painful bleeds in their joints. I felt that gene therapy would really be a game changer for them, particularly because you don't need very much of this protein. Even if you had 5% of normal levels, that would still make a big difference. Now we have patients who are able to play football without having to take an injection before they go out and do that.
What support did you get at UCL to develop your research into a spinout?
We developed three programmes that we licensed to a spinout company called Freeline Therapeutics (now Spur Therapeutics). We worked with Saint Jude Children's Research Hospital in the USA on haemophilia B and next moved on to haemophilia A and developed an approach that was licensed by BioMarin Pharmaceuticals Inc, with UCL playing an important role through UCL Business (UCL’s commercialisation company.). When I joined UCL it was slowly but surely becoming a powerhouse in gene therapy. It has been a lovely place to work, with some phenomenal colleagues to collaborate and socialise with.
What does the future hold for your work?
We are now trying to take gene therapy into Uganda as well as India. The majority of haemophilia patients live in the developing world, so if we could execute on the outreach to these countries, it would be amazing. It's a big challenge but we hope that one day we can.
What advice would you give to young scientists hoping to follow in your footsteps?
My advice would be: continue dreaming, continue aspiring to do better and if you have an opportunity to pursue your goals, ideas and dreams, go for it. And don't stop dreaming, because it's only if you dare to dream that you will develop these drugs.
One of the drugs that we're taking into clinic for cancer is a drug that I'm probably most proud of, which is one that has a built-in ‘off’ switch. The data looks really good and we hope to treat the first patient with these self-regulating drugs next year. We’re really looking forward to it.