Issue 27 – Spring 2015

THIS PRIzE WAS VERY DIFFERENT, because my peers had nominated me for a lifetime achievement. I felt it was like a gift from the network of people who have accompanied short or long parts of my journey in paediatric endocrinology. Such a trip is always a long story, with both exciting and disappointing episodes, where the overall direction of the work appears as important or even more important than the pieces of work themselves. The day before I received the prize, I was quite anxious about whether my lecture would be interesting. I came across a colleague who knew I was the recipient. He rightly stressed that there were as many different profiles of Andrea Prader Prize lecturers as there were recipients and concluded, ‘Just be yourself!’. How does this prize push the boundaries of paediatric endocrinology? My co-workers and I twice received the Henning Andersen Prize, which we felt gave a real boost to our research. The Andrea Prader Prize comes at a time in life when you are thinking about the entire landscape more than about your own tree and its fruits (though, as a colleague of mine pointed out, old trees do not bear old fruits!). The role of those of us who are Andrea Prader Prize recipients is to shed new light from our past onto the future of others. However, I do not feel in a position to give lessons to anyone, and rather want to share the lessons I have learnt myself, while leaving it to others, particularly younger colleagues, to consider those while building their future. This message, which I attempted to give in my prize lecture, is that ‘questioning does not stop with finding’. Where has your clinical and research work taken you? Under the direction of my late mentor Paul Franchimont, I became interested in the mechanism of puberty as long ago as 1972, before graduating as an MD. It took me decades to become aware that, because puberty is crucial for species’ survival, redundancy is protectively characterising the mechanism. So, investigators will be ESPE award winners 2014 In issue 26 of the ESPE Newsletter (Winter 2014, pages 3–4), we reported the award winners from the 53rd Annual Meeting of ESPE in Dublin last September. Now we are delighted to talk to some of them in person, and to find out what their prize has meant to them and to their research. Andrea Prader Prize winner: Jean-Pierre Bourguignon kept busy for a few more decades (as I was) before they eventually come to the ultimate key in that neuroendocrine mechanism. A colleague of mine described me as a ‘monomaniac’ for my approach to research. This may have been the case, but is no longer, since we have embarked on studies of endocrine-disrupting chemicals (EDCs) and extended our interest from the hypothalamus to other brain areas. As a paediatric endocrinologist, I am fascinated by the concept of the developmental origin of health and disease, which has huge implications for EDC research and also for patient education, particularly in informing pregnant women and parents of young children about the precautionary principle. Where is paediatric endocrinology heading in the next 5–10 years? For the past 3 years, I have started to interact with stakeholders and policymakers in order to value the contribution of (paediatric) endocrinology in the EDC arena. This has been an opportunity to become aware of some gaps between health sciences and the rest of the society. Beyond our efforts to promote training of young colleagues, paediatric endocrinologists have a mission to educate those setting the financial resources for research and the regulations for society. This is particularly challenging in a context of increasing and unequally disclosed conflicts of interest or biases. Again, the first step is self questioning before calling others into question. Then follow Albert Einstein’s suggestion: ‘Learn from yesterday, live for today, hope for tomorrow. The important thing is to not stop questioning.’ Jean-Pierre Bourguignon, Liège, Belgium , WE ARE BASED AT the Jesse z and Sara Lea Shaffer Institute for Endocrinology and Diabetes, at Schneider Children’s Medical Center of Israel. As part of the ongoing fight to help children with endocrine problems including diabetes, the Institute invests much effort in both basic and clinical research. The underlying aspects of the molecular basis of endocrine diseases are studied in the Pediatric Endocrinology and Diabetes Research Laboratory adjacent to the hospital. The proximity of the laboratory to the clinical service, and the close teamwork between laboratory researchers and medical care providers, enable us to transfer the patient’s problem directly from the clinic to the laboratory, where innovative solutions are sought and vice versa. The clinical research unit comprises clinicians, nurses, dieticians, psychologist and social workers, as well as several clinical research co-ordinators. A vast variety of clinical research projects are under investigation at any given time including intervention studies in endocrine diseases and diabetes. Recently, we have been engaged in research in two main areas: investigating the relationship between nutrition and growth improving the metabolic control and quality of life of patients with diabetes. Nutrition and growth Our studies aim to find the factors that influence bone growth and are focused on changes within the growth plate. One of the outcomes is the use of knowledge we have gained to develop an enriched diet to be consumed by children in the evening before bedtime. This formula was studied in a double blind, randomised, placebo-controlled study with 200 subjects and, as a result, it is now being commercially produced and will be distributed internationally. It will be given to normal short and lean children who have no indication for medical treatment such as growth hormone. ESPE Young Investigator Award winner: Oliver Semler EArtificial pancreas Extensive research shows that tight glucose control is the primary factor in avoiding the devastating complications of diabetes. However, this is associated with an increased risk of a severe hypoglycaemic episode, which is one of the most feared and dangerous events for patients and their parents. So the ultimate challenge is to keep blood glucose within a normal range without severe hypoglycaemic events. Although new advanced technologies have recently been introduced, patients’ diabetes control and quality of life have not yet dramatically improved. With this in mind, we established the Diabetes Technology Center (DTC), focusing solely on new technologies in diabetes, and, in particular, towards achieving an artificial pancreas system. The staff of the DTC have developed the MD-Logic system, which links sensors with insulin pumps via novel computerised control algorithms that dictate insulin delivery in response to real-time glucose sensor data. The MD Logic system was tested in a number of studies, assessing its safety and efficacy. Our system was the first to be tested outside the hospital at a diabetes camp, but the greatest breakthrough was testing our system at a patient’s home during a regular daily routine. Our artificial pancreas technology has recently received the CE mark and was licensed for integration into insulin pumps made by Medtronic. Moshe Phillip, Petah Tikva, Israe FELT VERY GRATEFUL TO MY TEAM, which was honoured by the award I received. It was a compliment to our clinical work in taking care of patients with osteogenesis imperfecta in an interdisciplinary team. Our work includes genetic and basic science research to understand new molecular findings regarding causes of brittle bones, as well as clinical trials investigating new physiotherapy or pharmacological treatments. Based on the genetic results, we have been able to initiate a new approach to treatment with an antibody against osteoclasts. This might offer a future alternative to the current bisphosphonate treatment of children with osteogenesis imperfecta. Our next steps will be to describe the pathophysiological steps causing brittle bones in more detail, working together with international partners to improve the treatment, independence and quality of life of these patients. The cycle of translational research will always continue by characterising patients, investigating the differences between them and adapting therapies based on these new insights. Oliver Semler, Köln, Germany