2003 LEMBERG MEDAL: JOHN HOPWOOD
John Hopwood completed his PhD on cartilage proteoglycans under Clem Robinson’s supervision at Monash University in 1972. As a Fulbright Scholar, he then moved with his wife and three children to Chicago. John took up a postdoctoral position under Albert Dorfman in the Department of Pediatrics, Wyler Children’s Hospital, University of Chicago. Al introduced him to metabolic disorders, particularly the world of lysosomal storage disorders (LSD), LSD patients and families. In 1974 Dr Hopwood became the Joseph P. Kennedy Jr. Fellow in the Neurosciences and continued research concerned with the synthesis, structure and degradation of mucopolysaccharides. Late in 1975 he moved to work with Ulf Lindahl at the University of Uppsala, Sweden, to identify heparin sequence that enabled anticoagulant activity. In 1976, John was recruited by Tony Pollard (Hospital Scientist in the Department of Chemical Pathology at the Children’s Hospital in Adelaide) to establish a national service for the diagnosis of LSD, particularly the mucopolysaccharidoses. LSD are a group of more than 45 individual genetic disorders that seriously affect children and adults. Currently, Professor Hopwood is Head, Biochemical Genetics Program, Chemical Pathology Services at the Women’s and Children’s Hospital and an affiliate Professor in the Department of Paediatrics, University of Adelaide.
In Adelaide, John established the Lysosomal Diseases Research Unit to focus research to achieve early diagnosis and effective therapy for all LSD patients. The unit pioneered the purification of many lysosomal enzymes, and the cloning and characterisation of their respective genes. The research outcomes have been applied worldwide to provide highly sophisticated diagnostic services for patients suspected as affected by one of these disorders. The unit produced and characterised recombinant human lysosomal enzymes from these genes and completed studies to demonstrate the efficacy of enzyme replacement therapy in a number of LSD animal models. This research encouraged biotechnology companies to develop effective therapies for these disorders. Evaluation of the clinical efficacy of these therapies is being trialled in the North America, Europe and Australia.
His recent research has aimed at developing newborn screening detection and effective therapy to prevent central nervous system pathology for LSD patients. Importantly, this research continues as a collaborative effort with many colleagues within the unit and worldwide. John has supervised 17 PhD students, published 282 papers in international journals and books, and holds more than 30 patents.
John has also been a major contributor to the establishment of Lysosomal Diseases Australia (LDA – an umbrella organization established to increase public, professional and institutional awareness of LSD), the Global Organisation for Lysosomal Disorders (GOLD – formed to globally unify professionals, patients and biotechnology companies to improve education about LSD and to achieve early diagnosis and effective therapies), and the Howard Florey Lysosomal Diseases Therapeutic Centre in Adelaide to progress the introduction of therapies into clinical practice.
Perhaps John’s most rewarding personal experience is the transfer of therapies from the research laboratory to clinical trials in Australian LSD patients, in the same hospital that has given him long-term support and encouragement to his research to achieve this end point.