Trustees Report 2014-15

TRUSTEES REPORT 2014~15

Over £34,000 was raised in the year, so a huge thank you to all of our fantastic supporters. This, together with funds raised in previous years, has enabled us to support the potentially ground breaking initiative detailed here:

Smiles sponsors GOSH/Institute of Child Health research project

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SMILES SPONSORS BREAKTHROUGH RESEARCH PROJECT

After six months of bureaucratic time wasting we finally dotted the ‘i’s and crossed the ‘t’s on the project to develop a bio-marker for monitoring the clinical outcome in children with spinal lipoma and we met with Dominic Thompson and Professor Copp on 14th October to finalise the details. We are the sole sponsors of this exciting, £58,000 initiative. Here, in layman’s terms, is what we are funding:

Smiles Study – Prof Andrew Copp (Professor of Developmental Neurobiology at the Institute of Child Health at UCL) and Mr Dominic Thompson (Neuro-Surgeon, Great Ormond Street Hospital)

‘Spinal lipoma’ describes a group of childhood conditions in which fatty tissue (lipoma) is present from before birth, stuck to the lower end of the spinal cord. This condition is estimated to occur once in every 4,000 pregnancies and can lead to progressive disability as children grow older. Weakness or pain and disorders of urination are commonly encountered because of gradual stretching of the spinal cord during growth. Surgical ‘untethering’ operations are performed regularly by Mr Dominic Thompson at Great Ormond Street Hospital.

Only a proportion of children with spinal lipoma deteriorate and need surgery. Others remain healthy and need no operation. Therefore, surgeons are faced with a dilemma: whether to operate on all children when they are young – and subject many to an unnecessary operation – or to operate later, only if symptoms appear. Delaying the operation in children who deteriorate might mean a worse outcome than if they were operated upon earlier in life.

What is needed is a simple blood or urine test that could be performed on very young children, when they are first diagnosed with spinal lipoma. The test would guide the doctor as to whether the child is at high risk of deterioration, and so needs early surgery, or is at low risk in which case surgery could be delayed and perhaps avoided altogether. In the Smiles Project, we will carry out research with the aim of developing such a test.

Our idea is that fat molecules may leave the spinal fluid and enter the bloodstream and urine of children with particularly severe spinal lipomas. These molecules will be detected using a sophisticated procedure called ‘mass spectrometry’ in which all the different fat molecules are separated out, to provide a ‘fingerprint’ for that particular patient. By comparing children who deteriorate with those who stay healthy, we hope to identify a fingerprint that can predict whether a particular child is likely to deteriorate or not. If successful, the Smiles Project could significantly improve how children with spinal lipoma are managed clinically in the future.

We will continue to report on the development of this project and future initiatives. We know that Grace would have been extremely proud of her charity’s success.

Peter Boxall, Trustee, 31st May 2015