Cracking The Code to Rare Diseases with Human Tissue Research
November 26, 2012
As a contract research organization, our scientists get satisfaction from helping to create new medicines of other companies; we may not have our own compounds, but like all scientists, our team wants to make a difference. We follow avidly the progress of compounds we have worked on, to see if they make it through clinical trials and into pharmacies.
When the chance arose to work on the rare diseases initiative, we recognised it as an opportunity to apply our unique technology to programmes where we can feel that ownership and hopefully our team will participate in the development of revolutionary new medicines.
At Biopta, we recycle a precious resource- residual tissue from surgery, which is donated by patients, and use these tissues to better predict which medicines will make the most effective treatments for future patients. Despite its known value to medical research, approximately 90% of surgical tissue is simply incinerated, yet surveys show that over 95% of patients wish to donate such tissues to medical research. We believe that our tests are truly the missing link between high-throughput genomics and clinical trials in patients; after all, what could be closer to the patient than intact, functional human tissues?
So, how can these approaches benefit the development of rare diseases? Rare diseases are defined as those which affect less than 1 in 1500 people (although the definition varies from country to country). Clearly, there are additional challenges to human tissue research in the study of rare diseases, primarily, the sourcing of tissues from a very small patient population. But even here we believe that tests in human tissue can be valuable, by modelling responses to the test drug in tissues from those without the disease, mechanisms of action can be investigated and safety can be assessed. Moreover, it is possible to source tissues from patients rare diseases with the help of partners such as the National Disease Research Initiative and International Institute for the Advancement of Medicine; Biopta has a long history of working with these and other esteemed organisations which have a track in supporting ethically donated rare disease tissue specimens.
A news report today described young children in Wonchi, Ethiopia, being given table PCs from educationalists at Massachusetts Institute of Technology. The tablets were programmed in English and loaded with apps, children’s books, games and cartoons; however, English was a language the children had never heard, and they had no instructions other than how to charge the tablets from solar panels. Within two weeks, they were singing ABC songs and within five months, they had hacked the Android operating system.
Cracking the code to rare diseases may follow a similar path; scientists, after all, are simply extending their child-like curiosity into their careers. We sincerely hope Biopta can be part of the Rare Disease Science Challenge and help in the development of life-changing medicines.
Dr David Bunton, CEO, Biopta Ltd.
Dr. David Bunton is an experienced and respected human tissue pharmacologist and one of the co-founders of Biopta Ltd. Prior to forming Biopta, David was a Lecturer in Physiology and Pharmacology at Glasgow Caledonian University and remains a regular contributor to the field of human tissue research. David is a member of the British Pharmacological Society and Safety Pharmacology Society and is also active in promoting greater access to human tissues for medical research in the UK and USA
Sign up for updates straight to your inbox.