Research. Drug developed in Cambridge approved for treatment of multiple sclerosis

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Jewel Byrd
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1 Research Drug developed in Cambridge approved for treatment of multiple sclerosis Approval concludes nearly 40-year epic journey from fundamental research to discovery of an effective treatment for active relapsing multiple sclerosis A transformational new treatment for multiple sclerosis (MS) - the result of over three decades of research in Cambridge - was approved today by the EU agency responsible for regulating new drugs. The European Medicines Agency (EMA) has approved the drug Alemtuzumab, to be known by the brand name Lemtrada and previously called Campath-1H (for Cambridge Pathology 1st Human ), for the treatment of MS. In recognition of the highly effective new treatment, the University of Cambridge has produced a video ( which explores the history of the drug, showing the many challenges as well as successes experienced during the course of this development. In 1975 scientists Cesar Milstein and George Kohler working at the MRC Laboratory of Molecular Biology (LMB) in Cambridge invented the technology for making large quantities of an antibody targeted at one This announcement marks the culmination of more than 20 years work, with many ups and downs in pursuing the idea that Campath-1H might help people with multiple sclerosis along the way. Professor Alastair Compston specificity, so-called monoclonal antibodies (work for which they were awarded the Nobel Prize for Physiology or Medicine in 1984). Building on this research, Herman Waldmann, Geoff Hale and Mike Clark from the University of Cambridge, with Greg Winter and Lutz Riechmann in LMB, produced the first monoclonal antibody for potential use as 1 of a 8medicine, Campath-1H (now known as Alemtuzumab), adopting the technique of humanising these antibodies 31/10/2013 in10:01 order to minimise risk of the drug being rejected by patients.

2 Drug Campath-1H developed in was Cambridge subsequently approved licensed for treatment for the of treatment mult... of chronic lymphocytic leukaemia. However, the 1980s Cambridge clinical scientists also began to explore its use in autoimmune diseases, which occur when the body s immune system mistakenly attacks healthy tissue. In 1991, Alastair Compston (Professor of Neurology and Head of the Department of Clinical Neurosciences) began to explore the use of Alemtuzumab as a treatment for the autoimmune disease MS. He and Dr Alasdair Coles, also from the University, led the subsequent research to develop Alemtuzumab in partnership with Genzyme, a Sanofi company, which now owns rights to the drug. Commenting on today s decision by the EMA, Professor Compston said: This announcement marks the culmination of more than 20 years work, with many ups and downs in pursuing the idea that Campath-1H might help people with multiple sclerosis along the way. We have learned much about the disease and, through the courage of patients who agreed to participate in this research, now have a highly effective and durable treatment for people with active MS if treated early in the course. Multiple sclerosis is an autoimmune disease in which the body s immune system mistakenly attacks nerve fibres and their protective insulation, the myelin sheath, in the brain and spinal cord. The resulting damage prevents the nerves from firing properly and ultimately leads to their destruction, resulting in physical and cognitive disabilities. Alemtuzumab reboots the immune system by first depleting a key class of immune cells, called lymphocytes. The system then repopulates, leading to a modified immune response that no longer regards myelin and nerves as foreign. But in so doing, roughly one third of MS patients develop another autoimmune disease after Alemtuzumab, mainly targeting the thyroid gland and more rarely other tissues especially blood platelets. Dr Coles research team is investigating how to identify people who are susceptible to this side-effect. Furthermore, they are testing whether this side-effect can be prevented, using an additional drug which boosts repopulation of the immune system, in a trial funded by the Moulton Charitable Trust and the Medical Research Council. Dr Coles, from the Department of Clinical Neurosciences, said: Alemtuzumab offers people with early multiple sclerosis the likelihood of many years free from worsening disability, at the cost of infrequent treatment courses and regular monitoring for treatable side-effects. We are very pleased and proud of this outcome, said Professor Herman Waldmann, who moved to Oxford University with his team in 1994 to work on the manufacture of Campath-1H, to understand its mode of action, and to apply the drug in a number of disease areas. In particular, we have great admiration for the neurology team in Cambridge with whom we have worked on this project for so many years. Their commitment and focus has been exemplary, and this has been a good example of basic and clinical science collaboration at its best. Multiple sclerosis affects almost 100,000 people in the UK, 400,000 in the US and several million worldwide. Symptoms of the disease can include loss of physical skills, sensation, vision, bladder control, and intellectual abilities. Although approved for use in EU, the drug has not yet been assessed by the National Institute for Health and Care Excellence (NICE) for the treatment of MS. ( This work is licensed under a Creative Commons Licence ( you use this content on your site please link back to this page.. If 2 of 8 31/10/ :01

3 3 comments 4 Leave a message... Best Community Share Scoopup Doggydogdo 19 days ago Surprise surprise. 20 years to produce a drug that will help early stagers, and still leave 30% + with residual thyroidal problems for which the patient will need to take further unproven drugs. As for 100,000 sufferers in the UK, try double that. I cannot believe how the medical profession kisses up its own arse on matters like these. Insteaf, why not try an excellent qualified homeopath, a 100% organic diet and I mean 100%, make your own food, use as many organic body and cleaning products as possible, and minimise modern living and you will have a great chance to manage MS for a long time drug free. 3 Reply Share REGGIE a month ago Just what i need!!! sign me up 1 Reply Share ccdemuth a month ago Congratulations. What spectacular news. 1 Reply Share ALSO ON UNIVERSITY OF CAMBRIDGE RESEARCH Farming the 'long-necked thing : moving from cows to camels 4 comments a month ago AvatarNaeem Khan Niazi Dear Thomas I belong to Pakistan.Country like Pakistan Have much potential of local breeds of camel and we should find any strategy to utilize the efficiency of camel Cambridge s criminal past revealed in centuries-old court records 2 comments a month ago WHAT'S THIS? AvatarAmina While working in the University Library for long hours on my thesis, I would go through Cambridge's history with criminal stains. Compiling it formally is an interesting initiative as Future internet aims to sever links with servers 1 comment a day ago Avatartroyth It seems like an involuntary content distributed network (CDN) for popular online content. It seems this would create a positive feedback system, where popular content would Reporting from Zimbabwe: a visit to Harare s biggest township 2 comments 3 months ago AvatarTom Kirk Thanks for sharing. 3 of 8 31/10/ :01

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5 Published 17 Sep 2013 Image Credit: Johnny Settle Share 120 reddit Related articles Campath: from innovation to impact New hopes for the nervous system: multiple sclerosis New treatment for multiple sclerosis: Cambridge University translates research at the bench into a drug at the bedside New MS drug proves effective where others have failed New treatments for pain and MS being developed in open innovation programme Subjects Multiple Sclerosis (MS) Drug Campath Alemtuzumab Treatment Innovation Spotlight On Innovation Spotlight On Neuroscience Antibody 5 of 8 31/10/ :01

6 People Alastair Compston Alasdair Coles Places School of Clinical Medicine Department of Clinical Neurosciences School of the Biological Sciences Department of Pathology Related organisations University of Oxford Further links Dr Alasdair Coles research lab Sign up to receive our weekly e-bulletin Subscribe Research Horizons 6 of 8 31/10/ :01

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SOL, ARGENTINA POMPE DISEASE. This is Genzyme

SOL, ARGENTINA POMPE DISEASE. This is Genzyme SOL, ARGENTINA POMPE DISEASE This is Genzyme MARIA, GAUCHER DISEASE TONY, FABRY DISEASE MICHELLE, MULTIPLE SCLEROSIS BRANT, THYROID CANCER JIMENA, MPS I GENZYME Genzyme has pioneered the development and