2020 Entry Clinical Fellowship
Predicting individual treatment response in paediatric Multiple Sclerosis using observational real-world cohorts
Multiple Sclerosis (MS) is currently an incurable chronic disease in which the immune system damages tissue in the brain, spinal cord and optic nerve, collectively known as the central nervous system (CNS). Recurrent bouts of symptoms (called relapses) are caused by the formation of lesions in the CNS, in which a fatty tissue (called myelin) around nerve cells is lost. Over the years, people with MS develop progressive CNS problems, leading to permanent visual, physical and mental disabilities. Children can also develop MS; around 5% of patients presenting before their 18th birthday.
Children with MS often have a more aggressive disease than adults, with more relapses, and they reach the progressive disease at a younger age than those who present with symptoms in their adulthood. There are currently 14 licensed drugs that reduce the risk of MS relapses; to date doctors cannot predict which of these will work best for an individual child. The decision about which drug to give to a child is currently very complicated and is based on clinician/patient preference and on the MS centre practice.
My project aims to predict whether a child with MS will respond to a specific medication. This is a crucial step towards ‘personalised medicine’; this means that children with MS will be able to initiate the most appropriate treatment choices for them, based on their probability of responding favourably to that therapy.
This project will study children who are already on medications for MS and children who are starting a new medication, with a view to creating a registry of all MS manifestations that may influence individual treatment response; these include demographic factors, blood tests, imaging, genetics and clinical examinations. The majority of the tests and investigations reflect standard practice in MS, but the novelty is that we will merge them together to obtain information on the individual MS profile. In particular, I will initiate and contribute to the collection of real-world data for children starting a new medication.
Use of disease-modifying therapies in pediatric relapsing remitting multiple sclerosis in the UK
Neurology: Neuroimmunology and Neuroinflammation
Incidence of Paediatric Multiple Sclerosis and other relapsing demyelination conditions: 10-year follow-up surveillance of Paediatric Acquired Demyelinating Syndromes (ADS) in the UK
Developmental Medicine and Child Neurology
Paediatric Acquired Demyelinating Syndromes: A review of the epidemiology and risk factors
Journal of the International Child Neurology Association