Dr Paul Huang is a group leader at the Institute of Cancer Research in London, whose research is focused on drug resistance in sarcomas with a view of moving towards a personalised approach to treatment.
Here, he shares how research suits his personality and explains why science is a two-way conversation.
What is your project about and how will it help patients?
Ewing sarcoma can be challenging to treat, particularly in a relapse setting or when patients present local disease with metastases.
There are a few clinical trials that address this, which have current standard-of-care chemotherapy as the backbone. We want to try and find out if we can improve patient outcomes, delay relapse, or even prevent relapse by adding multi-tyrosine kinase inhibitors (mTKIs) to treatment plans.
Can you tell us more about your area of research?
The two main trials that we are focusing on is the INTER-EWING-1 trial, led by Professor Bernadette Brennan at Manchester Children's Hospital, and the rEECur trial, led by Dr Martin McCabe at the Christie. They are studying different mTKIs, regorafenib or levatinib, in combination with backbone chemotherapy.
We know from experience with these drugs that some patients will respond to treatment, and some won't. My project is trying to devise a diagnostic tool to tell us in advance which patients are likely to respond, so that we can target the drugs to these patients.
For those that are unlikely to respond, we spare them from the side effects of the medication and potentially look at alternative treatments for those that are unresponsive.
In practice, this would mean that when a patient presents with Ewing sarcoma, a doctor would deliver the diagnostic test to check if a patient is likely to respond. Treatment would then be tailored to specific patients.
How will funding from the Bone Cancer Research Trust help you achieve this?
It is difficult to get funding for research into rare diseases. Without this funding, the project would not be able to commence. This is because it is not in the interest of pharmaceutical companies to develop a diagnostic test for a very small patient population.
If we were looking at a common disease, like breast cancer or lung cancer, you can imagine a situation where a company may see some commercial value in a larger patient population and be willing to invest in the research. This is because they will recoup the enormous investment that is needed to research, develop, and take a new medicine into clinical practice.
In this case, academic research in partnership with charities is essential to drive forward improvements in patient care for rare diseases such as bone cancer.
Is collaboration between charities important for rare cancers?
Ninety per cent of what we do is academic and can only be achieved through partnership with charities
It is very unusual that big companies are interested in rare cancers from a business perspective, as the investment on return is very low due to the volume of patients. In some ways, rare cancers are relegated to second class when considering new drug and biomarker development.
Because of this, it is absolutely essential to have partnerships between charities that have a very clear mission to support and help these patients.
What impact do you hope that your research will have, and in what timeframe?
Because we are dealing with rare patients, the clinical trials are five to ten year studies to recruit enough patients. We will not be able to gather any results until after the trials have ended.
The impact that we are hoping to have focuses on targeting the right drugs to the right patients, especially with younger patients such as those with Ewing sarcoma. High dose chemotherapy, in particular, is known to cause late effects and additional long-term effects.
Being able to identify the patients that we shouldn't be giving these drugs to is just as important as knowing the ones that we should be treating. Our aim is to tailor and personalise treatments, as opposed to continuing with the 'one size fits all' approach.
What are the biggest issues facing bone cancer research?
As we know, there are challenges with engaging companies about new treatments for rare cancer patients. It is important that researchers, charities, and patient advocacy groups lobby together and have a strong voice.
It is impossible to get something into patients without clinical involvement. It takes a lot of money to run a phase III trial and to have drugs approved for the clinic by the Food and Drug Administration (FDA), the European Medicines Agency (EMA) and the Medicines Products Regulatory Agency (MHRA).
Ultimately we will need a commercial partner and I think that by having a strong, united voice we can show why it is important to fund research into bone cancer alongside the more common diseases.
How did you come to specialise in this field?
From a young age I have always been interested in biology, and my dream was either to be a biologist or a biology teacher. I ended up being a biologist because it suits my personality.
All of us are impacted by cancer in some shape or form. The more common diseases such as breast or prostate cancer have at least two to three decades of research ahead of bone cancer. There is still so much that is unknown about sarcomas and bone cancer... I would say that we know less than ten per cent in comparison to the common cancers.
As a scientist, it stimulates me in trying to understand the biology of sarcomas and why drugs don't work in certain patients. I want to find out how to best develop combination drugs to achieve longer responses in patients.
Alongside the goal of developing something that will help people, I think as a scientist it is the innate curiosity. Trying to address key biological questions in a disease space where we don't fully understand what's going on that keeps me going.
Coming from an adult cancer background, what is different about studying a disease that mostly impacts young people?
There is a risk being in the laboratory setting that you only focus on the disease itself, and not on everything that comes afterwards, such as long-term and late effects.
There are unique challenges that come with treating younger patients. In the clinical practice, they are somewhat like the lost tribe. For certain diseases they are treated with regimens that were optimised either in adult clinical trials or in paediatric clinical trials.
Understanding the challenges that young adult patients face and what is unique about their biology that makes them different is something you gain appreciation for when you work in this area of research.
Do you have a message for our supporters who raise funds for research?
It is important for fundraisers to know that we are learning from you as patients as you are learning from us.
I believe that science is a two-way conversation. It is not just about us finding the latest discoveries that will help patients, it is equally important that we learn from patient experiences as well.
Please know that we are learning, we are listening, and we understand the challenges that patients and their families face on a day-to-day basis. We are working hard to try and address those challenges, but we couldn't do it without you. Thank you so much for helping us to help you.