There is a hold-up at the interface between basic science and clinical science. It can take 14 years and up to $350 million to bring a drug to a patient. One of the main ways of cutting down this delay and expense is to identify and target specific patients, and find out who will actually benefit from a medication.
This means exploring genetic factors and understanding how they affect various people with different ailments. That's the message from Professor Des Fitzgerald of the Department of Clinical Pharmacology in the RCSI. There are, he explains, many medications which will work well on some patients, but not on others, and genetic factors are among the prime suspects for this.
This will affect all stages in the development of a medication, and especially clinical trials. If it can be known in advance which genetic factors are relevant, this can mean a big saving in time - and money - and help to get life-saving drugs to patients.
Most people have heard of the Human Genome Project, which has provided a map of the various building-blocks of life. This, though, is only a start in the work of finding out how various combinations of genes work together and interrelate.
This research is going ahead at astonishing speed and the RCSI has been determined to take full advantage of it. In July 1998, the RCSI launched a 50-50 joint venture with Genset, a company based at Evry in France. The venture, called SurGEN, is performing the first large-scale human genomics study in cardio-vascular diseases.
Speaking at its opening, Prof Fitzgerald, its clinical director, said: "In Ireland it's estimated that more than one in three deaths each year results from heart disease. To date, there is little information as to what genetically predisposes an individual towards this condition. The work of SurGEN will give a vital insight into this whole area."
He went on to explain that SurGEN brings together "the extensive recruitment capability and phenotyping expertise of the RCSI and Genset's cutting-edge genomics technology platform in the identification of genes associated with common diseases, such as cardiovascular disease.
"Previous clinical trials tended to look at the effect of a particular drug on a particular disease. The DNA samples and phenotype information from SurGEN are key resources to better understand the fundamental causes of the disease and the reasons why selective drugs work in certain instances."
The strategy is to get a large sample of volunteers, numbering tens of thousands and to build a database of samples of DNA and blood. This will give researchers the base to discover the various genes and create a better understanding of the molecular pathways implicated in cardiovascular diseases and their complications.
Under the terms of the agreement, the RCSI will have rights to use this bank for clinical research and to publish the results of such research. Genset will have exclusive access to this DNA bank to conduct its gene discovery and pharmaco-genomics programmes, and to patent and commercialise any discoveries made as a result of this research.
For researchers, the collaboration meant that they could get access to a kind of data nor normally available. As Pascal Brandys, then chairman and chief executive officer of Genset, said: "With this collaboration, a systematic analysis of genes associated with cardiovascular diseases, their complications and their responses to treatment is becoming possible. Largescale association studies based upon actual human clinical data was the needed breakthrough in this field, which has been mostly restricted so far to cellular and animal models."
Genset was founded in 1989 and, as of last August, it had 536 employees (328 in R & D). It is quoted on the NASDAQ. On August 1st last, Andre Pernet, a member of its board of directors, was appointed president and chief executive. Prior to joining Genset as a director, Pernet was corporate officer and vice-president, pharmaceutical products research and development of Abbott Laboratories heading an organisation of 2,700 scientists.
Just a year ago Genset acquired a majority holding in Pacific Oligos which was spun off from a specialised group of the Centre for Molecular and Cellular Biology (CMCB) at the University of Queensland in Brisbane. Since 1998 it has had a subsidiary in Singapore.
Other current corporate partners include Synthelabo, the pharmaceutical subsidiary of L'Oreal for prostate cancer gene discovery; Johnson & Johnson for schizophrenia; the Genetics Institute, a subsidiary of American Home Products, for the distribution of SignalTag, its secreted proteins gene library; and Abbott Laboratories for pharmacogenomics or the analysis of drug response genes.
Their website is www.genxy.com
