Immunotherapy for amyloidosis

点击翻译按钮获取中文摘要

For 30 years the MRC has supported my work on a rare disease known as amyloidosis. We have discovered much of what is known about this condition and about a normal protein in the blood, known as serum amyloid P component (SAP), which contributes to the disease. Our MRC funded research on SAP led us to invent a new diagnostic test which has delivered significant improvements in treatment and survival of amyloidosis patients. We went on to establish the UK NHS National Amyloidosis Centre, the world?s leading centre for diagnosis and management of amyloidosis. The survival of our patients is not bettered anywhere else. However amyloidosis, which is responsible for the death of one per thousand of the population, is still an almost universally fatal disease. Alzheimer?s disease and maturity onset diabetes, both of which are associated with amyloid, inflict prolonged suffering on patients and carers, at great cost to society. We have now invented new medicines that specifically target SAP and amyloid, and produce clinical benefit in animal models which closely resemble human disease. Our most advanced drug, that removes SAP from the blood, helpfully slows disease progression in amyloidosis patients but this is not sufficient. We have now developed another novel treatment, using antibodies to SAP, which completely removes the amyloid deposits that cause disease. Our achievements have so impressed GlaxoSmithKline, the second largest pharmaceutical company in the world, that it has agreed to collaborate with us to develop these medications. They have insisted that our university research team shares the early risk in the long, difficult and extremely costly process of drug development. They are therefore deliberately not funding our component of the programme. The present proposal seeks the funding necessary for our contribution, while GSK conduct their own in house development work. This represents extraordinarily good value for the MRC and for the patients who will ultimately benefit from potentially life saving new treatments. Enabled by the laboratory research proposed here, the GSK collaboration will bring our key inventions into clinical trials as soon as possible and, within the 3 year period of the grant, the basic laboratory work, for which we now seek MRC support, will translate towards benefits for sick people.