£2.1 million cash boost for child health research at UCL

UCL has received funding from Great Ormond Street Hospital Children’s Charity and children's charity Sparks for a number of projects to research some of the most difficult and hard to treat childhood diseases.

This year, GOSH is also partnering with rare disease charities Krabbe UK and Dravet Syndrome UK, with each charity providing funding for two projects at The UCL Great Ormond Street Institute of Child Health (ICH). 

This National Call is the largest annual charitable call dedicated to funding research into child health conditions. The funding will support 12 pioneering projects based at eight institutions across the UK from Sheffield to London, with the aim of  improving diagnosis and develop more effective and kinder treatments for children who desperately need them.

This injection of funds in to paediatric research will provide a huge boost to an area of research that is severely underfunded, receiving only five per cent of public and charitable research funding in the UK each year.

Dr Sara Benedetti (ICH) has been awarded £96,086 by GOSH Charity, Sparks and Krabbe UK to work on new strategies to maximise the potential of a stem cell treatment that could stop the toxic build-up of a molecule in the brains of children with Krabbe disease, a neurological disease that affects the brain and spinal cord, reducing neurological symptoms and improving life expectancy.

Dr Benedetti said, “Krabbe disease is a devastating neurological disease that affects the brain and spinal cord. In the most common infantile form, children are typically diagnosed at around 6 months old and rarely live beyond their second birthday. The funding will enable me to further my work to reduce neurological symptoms and improve life expectancy.”

Dr Rajvinder Karda (ICH) has been awarded £75,725, which has been jointly-funded by Dravet Syndrome UK, with GOSH Charity and Sparks providing the other half of the award. Dravet Syndrome is a rare, inherited type of epilepsy and is incredibly hard to treat. The majority of children have a mistake in the same gene causing their disease, which means fixing the genetic mistake could offer hope to many families. This team are trying to do just that by finding a way to correct the mistake using a technique known as gene therapy.

Other recipients of the funding are Dr Jasper De Boer (ICH) for research into the advancement of treatment for an aggressive childhood brain tumour (DIPG), Dr Owen Williams for research into the advancement of the treatment of leukaemia and Professor Dimiti Kullmann (UCL Queen Square Institute of Neurology)for research into the advancement of treatment into epilepsy.

Kiki Syrad, Director of Grants and Impact at Great Ormond Street Hospital Children’s Charity said, “We were delighted to receive a large number of high-quality applications from the UK pediatric research community on a range of diseases. For many children, research is their only hope. We look forward to seeing how Professor Kullmann’s project progresses, and the call re-opening later in 2019.” 

This release was first published 3 April 2019 by UCL.