Rare diseases affect children and adolescents in eight out of ten cases. Their access to medical progress therefore depends greatly on dedicated pediatricians who embrace the challenge of balancing bedside care and laboratory work with passion. To prepare aspiring physician-researchers for this demanding task—and give them the freedom to pursue top-level scientific work—the research network Alliance4Rare, initiated by the Eva Luise and Horst Köhler Foundation, also relies on structured Clinician Scientist Programs (CS4RARE). These programs provide participants with protected research periods during which they can focus on scientific projects on rare diseases without clinical duties.
Dear Dr. Lechner, you have been selected for the Alliance4Rare Clinician Scientist Program. What motivated you to take this special path between science and clinical practice?
From early on, I was fascinated by the fundamental disease mechanisms in medicine. Rare diseases are especially intriguing: many are based on clearly defined genetic causes, whose pathophysiology can be specifically studied. This understanding opens up the possibility to develop new therapeutic approaches, directly improving care.
At the same time, I am passionate about clinical work in pediatrics, a broad specialty with wonderful young patients in which the frequency of disease patterns—unlike adult medicine—varies greatly depending on the age group. Many rare diseases appear during childhood, so you encounter them more often in pediatrics. This means that, despite the breadth of the field, you always need to be attentive to details so as not to miss rare conditions.
University medicine plays a central role that goes beyond standard guideline-based care: it creates opportunities to identify new conditions, understand complex connections, and develop innovative therapies. This close connection between basic research and clinical work was the decisive reason for me to pursue the path of a clinician scientist.
As an “Anchor Scientist” in Berlin, you have a special role. Can you briefly explain what that entails?
As an Anchor Scientist, I am tasked with being a fixed link between my site and the Alliance4Rare network. This means I anchor the program’s structures locally while also promoting supraregional collaboration. This includes connecting suitable experts with each other, highlighting synergies between different projects, and jointly developing tools or methods that benefit all involved. Even though we research different rare diseases, we can benefit from a shared infrastructure and close exchange. This collaboration is vital to making progress in a field where individual diseases often affect only a few people.
What do you research, and what do you hope to achieve in the field of rare diseases?
My research focuses on rare disorders of hypothalamic satiety regulation. It involves genetic defects that cause children to develop abnormal hunger and satiety cues, which can lead to early and severe obesity. My goal is to decipher previously misunderstood disease mechanisms and thus lay the groundwork for targeted therapies.
For some of these diseases, pharmaceutical treatments are now available, enabling children to lead almost normal lives. However, many conditions remain poorly understood—this is where I want to make a difference and eventually help more affected families.
What opportunities does Alliance4Rare’s support provide in your daily work that you wouldn’t otherwise have?
After medical school, I worked in basic research before starting clinical training. Alliance4Rare’s support allows me to combine both: I can continue my pediatric specialist training while pursuing my scientific projects.
The special aspect of the Clinician Scientist Program is the protected time for both fields. Only when both clinic and research are given sufficient space can the vital bridge “from patient to laboratory and back to the patient” be built. This close link creates creative momentum that ultimately benefits affected children and their families.
Dr. Lara Lechner grew up near Frankfurt am Main and completed her medical studies at Charité in Berlin. For her MD/PhD, she investigated epigenetic influences on central hunger and satiety regulation in large human cohorts, using functional characterization via stem cell models. After graduating from medical school in 2022, she deepened her basic research knowledge as a postdoc at Charité and at Columbia University in New York, focusing on hypothalamic stem cell models, single-cell analyses, and advanced molecular genetics. Currently, Dr. Lara Lechner is a pediatric resident and clinician scientist at Charité, with scientific focus on rare, monogenic forms of childhood obesity, such as those altering hypothalamic satiety regulation.
