Like many stem cell researchers, my work is increasingly focused on developing ways to translate basic scientific discovery into treatments that help people suffering from disease or injury. The potential for stem cell therapies initially fascinated me as a developmental biologist, and over time I’ve learned the very real impact that our science can have on human health.
This translational focus has been key to my work over the last decade and has compelled my personal involvement with the development of a pediatric oncology hospital in the Netherlands, the Princess Maxima Center for Pediatric Oncology. What started about a decade ago as an initiative to bring pediatric oncology departments together into one facility has led to the development of one of the largest biomedical campuses in Europe, among the largest in the world, treating children and adolescents with all forms of cancer and its precursors, set to open in May in Utrecht.
I’m particularly proud of this initiative because it takes a collaborative approach to finding answers in the fight against cancer. The concept was forged through a coalition of patient groups which, working with physicians, sought to establish an integrated team of physicians and scientists that would work independently yet collaboratively to understand, treat, and hopefully cure pediatric cancers.
At the Princess Maxima Center, research and clinical care operate side-by-side, sharing information and understanding of issues across the broad spectrum of pediatric cancers. Research covers bioinformatics, molecular and cellular biology, epidemiology, trial research, research into late effects of therapy, psychosocial studies, and more, and we discuss our findings with physician colleagues in the clinic in real time, making decisions that can quickly inform treatment options.
Patients, their families, and their needs are at the core of this initiative. The clinic is organized around patients, not doctors, with separate departments for children with leukemia, brain tumors, and solid tumors, and a single research department that includes one layer of principal investigators. The hospital is owned by an oncologists’ organization, SKION, and a patient organization, VOKK.
The critical voice of patients in stem cell and other research is important and is one reason we highlight a patient perspective at the ISSCR Annual Meeting each year. At the 2018 Meeting in Melbourne, we will hear from patient advocate Daniel Feller, whose son Harry was born with a rare genetic disease called Usher Syndrome. The disease caused Harry to be born deaf, and has early onset Retinitis Pigmentosa, for which there is currently no cure.
Daniel immediately became actively involved in Harry’s treatment, and sought ways to address Usher’s Syndrome symptoms and causes. He founded Genetic Cures Australia (GCA) to support the growth of medical research within Australia, concentrating on emerging gene-based therapeutic approaches. GCA’s mission is to ensure that access to appropriate leading therapeutic trials is available to everyone in Australia living with an inherited disease. The immediate focus is on treatment for eye diseases.
Daniel and Harry will be speaking at the Public Symposium Monday, 18 June, at Federation Square in the Deakin Edge theatre. Daniel will also share his perspective in Plenary V, “Stem Cell Based Disease Modeling,” on Friday, 22 June.
My work with the new pediatric oncology center has brought me closer to patients and their families, and I see first-hand the experiences of patients like Harry and his father Daniel as they work with researchers in pursuing treatments and potential cures that can save lives. Pediatric cancer is rare, as is Usher’s Syndrome, making it even more important that governments and insurers hear from advocates urging development and testing of treatments.
Working together, patients, physicians, and researchers can advance the therapeutic potential of stem cell research and regenerative medicine and provide a ‘win-win’ for stakeholders working to find answers to intractable questions involving human health.Hans Clevers
ISSCR President 2017-2018