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2005 November 11 - University student awarded fellowship to help save the lives of children with liv

A student at the Royal Free & University College Medical School, London has been awarded a three year CLDF PhD fellowship by a national children’s charity, which could help save the lives of children with liver disease.

Ilan Shulman, 22, of Hendon, North London, has been awarded the prestigious CLDF PhD fellowship, worth over £70,000, from Children’s Liver Disease Foundation, the only organisation in the UK fighting childhood liver disease.  The project will enable research into gene therapy for the metabolic condition, Ornithine transcarbamylase (OTC), and has the potential to save lives and reduce the need for liver transplantation in the future.

OTC deficiency is a serious metabolic disorder caused by a missing enzyme in the liver cells affecting one in 40,000 – 80,000 births.  In affected babies the OTC gene in the liver is abnormal and does not make enough of the OTC enzyme, so the liver is not able to clear toxic substances from the blood.  Nitrogen accumulates and is converted into ammonia rather than urea.  When ammonia reaches the brain through the blood it may cause irreversible brain damage and/or death.  Some cases of OTC may be treatable by transplant but the usual outcome is death in infancy or childhood.

The cause of OTC has been identified as a single gene defect.  This project will study a particular means of replacing the defective OTC gene - gene therapy - and aims to research and develop effective and safe means for delivery of the normal gene for the metabolic disease OTC as gene therapy.

Ilan Shulman will work under the supervision of Professor Humphrey Hodgson DM FRCP, FMedSci, Professor of Medicine at the Centre of Hepatology, Royal Free & University College Medical School.  Successful completion of the project will entitle Ilan to submit the work for a Doctor of Philosophy.

Children’s Liver Disease Foundation, a member of the Association of Medical Research Charities, has awarded two PhD fellowships in 2005 for projects which support its aims.  The PhD fellowship applications are reviewed by the CLDF medical advisory committee which comprises of leading researchers and clinicians.

Ilan Shulman says, “I am delighted that the trustees have awarded us a CLDF PhD fellowship.  This vital research has the potential to prevent the tragic symptoms of OTC deficiency, avoid the need for liver transplantation and develop an understanding of gene delivery therapy”.

Catherine Arkley, Chief Executive of CLDF says, “One of our prime objectives is to promote research into the causes of childhood liver disease. Few people are aware that every day at least two children in the UK will be diagnosed with liver disease, that’s a great incidence that childhood leukaemia.  This project could offer vital hope for families of children with liver disease in the future, and the research could also have far reaching benefits”.

CLDF has donated over £4m to pioneering research projects since its inception in 1980, leading to vital advances in understanding and treating paediatric liver disease.

For further information on the work of the Foundation, please visit www.childliverdisease.org or call 0121 212 3839.

 For further information on this story please contact Rachel Markham,

Children’s Liver Disease Foundation on 0121 212 6012, e-mail: communications@childliverdisease.org

Note to editors:

The Children’s Liver Disease Foundation is a unique national charity that fights childhood liver diseases through funding pioneering research and educating healthcare professionals and the general public. Formed in 1980, it provides professional, emotional support to families affected and is the only organisation of its kind in the UK.

CLDF PhD Student Fellowships:

In 2005 CLDF awarded two prestigious CLDF PhD student fellowships to encourage the best young science graduates to embark on a search career in paediatric hepatology research.  Fellowships are awarded to university departments annually by open competition for projects that are relevant to the aims of CLDF.  University departments are invited to submit applications which may include a named student or alternatively students may be recruited by the host department after the award of the fellowship with the approval of CLDF.  Students must submit a brief annual report via their supervisor and progression onto the next year of the fellowship is dependent upon the report being satisfactory.