THE UK’s leading LCH and HLH specialists turned out in force to meet patients and parents at The H R Trust’s recent Road Show.
Drs Peppy Brock, Vasanta Nanduri, Johann Visser, Kevin Windebank and Professor Matthew Collin headed to London’s Great Ormond Street to talk about LCH and HLH to about 32 patients and parents.
The clinicians were joined by H R Trust Trustees President Paul Kontoyannis, Vice-President Professor Peter Beverley and Treasurer Howard Ingleby. In his address to the audience, Mr Kontoyannis said: “We must work together to find what causes these terrible diseases. I want you know that you are not alone. Every child with LCH or HLH is our child and part of our family.
“We are very small, but we have a big agenda – not only to help the patients but to also solve these diseases. We are working on it and we hope to do it in our lifetime.”
He added that in addition to funding vital research programmes, there was also a need to fund a replacement for Professor Tony Chu, a specialist in Adult LCH, who is due to retire in three years time. The H R Trust also wanted to be able to employ a nurse to advise and support patients and their families.
“We have a responsibility to help, there are many needs besides research,” he concluded.
Professor Matthew Collin, one of the UK’s two specialists in Adult LCH who is based at the Freeman Hospital, Newcastle upon Tyne, advised that treatment of Adult LCH was behind that of paediatric LCH. After explaining how LCH affected adults, he added that an increasing proportion of adults had been diagnosed as children but due to the lack of specialist doctors, they faced a long journey in securing treatment.
However, he said: “After two years of research, it is an exciting time in terms of our knowledge about dendritic cells – a new horizon is dawning is this field.” He added that a current research programme was now looking at where dendritic cells were coming from.
Dr Kevin Windebank, Senior Lecturer in Child Health at Newcastle University as well as an Honorary Consultant in Paediatric Oncology at Newcastle Hospitals NHS, said there were 50 cases of paediatric LCH in the UK and Ireland each year.
Dr Johann Visser, Consultant Paediatric Oncologist at Leicester Children’s Hospital, explained about the new LCH4 Clinical Trial which is due to take place. For the first time, paediatric patients with LCH both in the UK and internationally, will be enrolled and monitored over a 10 year period, irrespective of whether they need treatment or not. He added that at least 600 patients would be needed for the trial to be a success and research was being developed to store tissue samples.
Dr Vasanta Nanduri, Consultant Paediatrician at Watford General Hospital and member of The Histiocyte Executive Board and co-chairperson of the Histiocyte Society Epidemiology/late effects study group, gave an informative talk about the possible late effects of LCH.
H R Trust Vice-President Professor Peter Beverley explained about the two research projects that the Trust has funded since 2005. The first project, which cost £150,000, was led by Professor Hans Acha-Orbea and looked at identifying genes that are switched on when normal dendritic cells, which are closely related to Langerhans’ Cells, become tumours. The study took place at the University of Lausanne, Switzerland.
The second three-year project, currently being funded at a cost of £220,954, is focussed on the Development of Gene Therapy for Familial Haemophagocytic Lymphohistiocytosis (HLH) due to perforin deficiency. Scientists Professors Bobby Gaspar and Adrian Thrasher together with Dr Marlene Carmo are working on replacing Perforin, the commonest gene to cause inherited HLH at UCL.
Professor Beverley announced that the next project to be funded by the Trust would be aimed at LCH and more details would be announced in January 2011. The project is due to start in Spring 2011.
He added: “When we fund research, we make sure that we are going to fund good research carried out by good people. I want you to know that we are making progress.”
Feedback from the Road Show has been excellent. Parent Jane Wynn said: “The Road Show was interesting and relevant. It was good to hear from different aspects in tackling the disease.”
Parent Gavin Weeden added: “All of the Road Show was useful but in particular late effects, research and clinical trials.”