The top physician to many of the UK's CGD patients, Professor David Goldblatt has clinched millions of pounds in government research funding for a new biomedical research centre, and will be its first Director.
Launched today (22 January, 2008) the GOSH/ICH Biomedical Research Centre has been awarded £7 million a year (for five years) to research a range of diseases, which may include CGD.
Professor Goldblatt, Director of Clinical Research and Development for Great Ormond Street, told the CGD Research Trust it is too early to say exactly how the new centre could benefit research into CGD, but he will keep the CGD community informed. Another big name in CGD, Professor Adrian Thrasher cited his team's work on gene therapy for CGD (much of it funded by donations to the CGD Research Trust) as an example of important cutting edge research, in a lecture at the launch.
With guests from the Department of Health present, the hosts didn't dwell on whether the new grant was genuinely new money or recycled generosity. Only one jaded observer grumbled to this reporter: "it is the Government giving back some of the money they took back before", having allegedly failed to honour promises made under a previous funding round.
Nevertheless, the mood was celebratory. Re-hooking a big fish that somebody had swiped out of your keep net and thrown back in the pool would be no less an achievement, and GOSH Chief Executive Dr Jane Collins gave heartfelt thanks to Professor Goldblatt "for masterminding the success of that bid” and to Emma Pendleton, head of the hospital’s Research and Development Office.
In his opening words about treating children, Dean of the Institute of Child Health Professor Andrew Copp spoke of a new focus on "the adults they become" and stressed "the need to build capacity particularly in academic paediatrics. We need to train the leaders of the future - and engage increasingly with the Public." Gaining Foundation Trust status, which GOSH is determined to secure, would involve "the membership of lay people, which will help". Strength in experimental medicine, disease models and 'first in human' studies would all be part of the new centre's "push for translatable research".
GOSH’s chief executive went on to say: "The whole model for these biomedical research centres is very much built on the NIH [National Institutes of Health] model in America." She said the new centre was a really important development, and the children’s hospital was "fortunate in having such an effective research partner" in ICH. Dr Collins drove home the "…moral responsibility to find better ways of diagnosing and treating. We need to think perhaps a little bit more about the whole child, public health and growing up as adults." Funding at GOSH was increasingly earmarked for specific positive outcomes such as these whereas it used to come in bigger blocks, with fewer conditions attached.
Professor Goldblatt revisited the intriguing history of Great Ormond Street, noting how its fundamental compassionate values had not changed since the 18th century, when a great many children of desperately poor parents "were left lying around the streets of London", prompting the start of a Foundling Hospital in 1740. Another milestone was 1852 when Charles West founded the Hospital for Sick Children, Great Ormond Street at a house in Powis Place.
He recalled the surprising fact that while bombs fell on London in 1943, a committee was far-sighted enough to insist on the need for a formal academic school of child health, which led to the formation of the Institute of Child Health in 1945, and in 1966 the completion of the building that houses it. He finished with a sprint through the recent, (some might say slippery) history of UK research funding, outlined the structure of the new biomedical research centre and gave a preview of its building programme.
Another great catch for the day was to have the keynote address given by Dr John Gallin, Director of the US National Institutes of Health Clinical Centre in Bethesda, Maryland. He is the blue whale of clinical research, commanding a budget of $350 million a year and reeled off eye-popping statistics about what is achieved by doctors, scientists and patients attached to the centre. He finished by calling in effect for a new world order in clinical research, with far greater cooperation between governments and scientists around the world, who could share mega-databases of vital health information. Professor Goldblatt joked, “We will try and respond to your global vision – especially if you can send some of the dollars”.
Two hours of lectures followed on the theme From Science to Practice: Experimental Medicine at GOSH/ICH from some of the brightest stars in the business, who are the theme leaders of the new centre: Philip Beales (Professor of Medical and Molecular Genetics) who specialises in the area of congenital birth defects, Adrian Thrasher (Professor of Paediatric Immunology) and Francesco Muntoni (Professor of Paediatric Neurology) who leads the Dubowitz Neuromuscular Centre and conducts research into Muscular Dystrophy.
Recently arrived from Italy, Prof Muntoni began with a tongue in cheek apology “in case I have used the wrong logos on my powerpoint!” and one could understand, seeing the number of institutional brands and sub-brands (all state entities) jostling for position on the invitation to this prestigious event (The NHS, the NHS’s NIHR, UCL, GOSH, UCL Institute of Child Health, the new NIHR Biomedical Research Centre itself, and its American counterpart the US National Institute of Health Clinical Centre). This is the world where the CGD Research Trust must also compete for funding and profile, and our distinctive new branding will prove to be a great asset.
It was cheering to hear from Professor Thrasher that the child who recently got leukemia after gene therapy was currently doing well “on regular chemotherapy…the leukemia is now in remission”. He was also convinced that despite some opinions to the contrary, there is “no evidence for potent oncogenicity”.
Dr Gallin said, “I have to take my hat off to those of you who are moving gene therapy and cell therapy along. That takes a lot of courage.”
Dr Gallin also has a special interest in chronic granulomatous disorder, so the community is in the unique position of having CGD experts heading both UK and American top clinical institutes simultaneously – which is unlikely ever to be repeated. Ways must be found to take greater advantage of it.
Overall the event was a reminder of the tremendous effort and intelligence being applied to help find solutions to illnesses like CGD, and those in the patient community will want to do everything possible to help bring in the funding required to finish the job.
Report by James Robertson
IMPORTANT NOTE :
The information contained on this website is intended only as a guideline, not as a substitute for medical advice. Always consult your doctor if you or your child has any CGD symptoms or concerns.
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