Dr Sara Stoneham is a paediatric oncology consultant at University College London Hospitals. Here, she explains some of the barriers to researching rare tumours and what can be done to overcome them.
Research is the best way to improve outcomes for all children with cancer, but the rate of progress for children with rare cancers is much slower than for more common cancers. For some cancers, there might be fewer than 20 children diagnosed per year in the UK. Not only does this make it difficult to carry out research, but it also makes it difficult to find a funder willing to support a project that may only help a few patients.
The challenge
Rare cancers have less scientific consideration and less financial support to develop better, kinder treatments. For clinical trials to show that a new treatment is effective, it needs to be tested in many patients, to show that any improvement is not down to chance. This is much more difficult when a cancer is rare as there are so many fewer patients that not only have the disease, but also meet the
trial’s eligibility criteria.
Access to clinical trials is only part of the problem - progress is hampered at almost every stage of research for rare cancers. It can be slowed by drug companies that can be indifferent to developing new drugs for a rare cancer. There is also a shortage of storage facilities for leftover diagnostic samples for future research, meaning we learn less about how these rare cancers behave and what new treatments may be useful to try in the future.
How can we learn to speak the same language and design effective treatment options?
Current initiatives like biobanks, which store cancer tissue and cells left over from diagnostic tests, are an important way to support rare cancer research. By increasing the amount of cancer samples that a researcher can work with, they allow better quality research to be conducted. Biobanks can store samples indefinitely, and research has shown that frozen samples can still be useful over eight years after being stored. However, a more widespread change in the way research is conducted is needed to take treatments from laboratories to patients. Bringing international experts together is an important first step. By thinking differently and taking away barriers that exist between countries and clinical teams, we can re-examine what we think we know.
Sharing data to create an international ‘super-database’ can create large enough numbers to answer questions that no single country was able to answer before, leading to meaningful change. This may require joint international lobbying of national trial institutions and regulators to help overcome the differences in international law and national ethical frameworks, so we can ensure that all patients’ data can be kept confidential, but still put to best use.
If we have consent from patients across the world to use leftover diagnostic samples, combined with cancer genomic data, we can start to answer the questions that have been prioritised jointly by patients, families and researchers. By designing international trials together, we can identify barriers for each country and learn how to avoid these pitfalls. We can use novel statistical methods that allow us to answer important treatment questions quicker, but while still ensuring robust scientific methods are followed.
International collaborations are more likely to persuade research funders it is worthwhile to support rare cancer research. These collaborations can also increase access to rare cancer expertise by developing expert advisory panels that can make a difference for patients right now by offering specialist advice and most importantly, they can create a forum of like-minded experts, patients and families who share a common goal to improve rare cancer outcomes through meaningful, impactful research.
From Contact magazine issue 98 - Spring 2023
