Professor Laurent Servais' Inaugural Lecture - watch the film
Kellogg College was pleased to host the Inaugural Lecture of Kellogg Fellow and Professor of Paediatric Neuromuscular Diseases, Professor Laurent Servais.
Accelerating Clinical Development in Rare Diseases – How can we be more efficient?
When it comes to translating exciting pre-clinical discoveries in the field of rare and devastating diseases in the brains and muscles of children into a disease-modifying therapy in humans, the question “How can we be more efficient?” is a key concern for any physician involved in the process.
Too often, early findings in humans cannot be confirmed in large trials, leading to major disappointment in the field and a waste of time, money, human resources, and most importantly, of hope.
In this lecture Prof Servais explored how we can improve efficiency now.
Innovative technologies are enabling us to accelerate clinical trial development, by demonstrating the presence or absence of an objective improvement on motor function in a limited period of time on a limited number of patients. New clinical trial design and statistics are allowing us to compare patients with their own evolution, rather than with another patient. And new born screening is empowering us to treat patients before irreversible damage, and to maximize the therapeutic effect.
Combining these different approaches has the potential to accelerate successful clinical developments, to avoid progressing drugs that have no objective efficacy into large trials, and even more significantly in the case of new born screening, to offer babies affected with severe but treatable conditions the best chance of having a life free of disability.
Watch the film