A multidisciplinary approach to drug discovery and development speeds the overall drug process and fuels the treatments of the future.
Developing new pharmaceuticals is a long and costly process. The path between the discovery of a drug candidate and a drug hitting the market takes years of research and clinical testing that costs millions, if not billions, of dollars with many pitfalls and problems along the way.
Translational research aims to speed promising drug discoveries from the lab to clinical trials. Speeding the process not only brings products to the market faster and more efficiently, but it also significantly cuts costs. Translational research brings together scientists from different areas of expertise to approach the problem from multiple angles and through different methods.
The Ohio State Univ. Wexner Medical Center brings together computer science, information science, biomedical informatics (BMI) and information technology (IT) tools and methods to support and enable high impact research. This group comprises 31 academic experts working to identify critical factors for developing and implementing information technology tools for medical uses.
“The presence of a formal, academic BMI unit is important to ensure IT and informatics are optimally used to support clinical and translational research,” says Philip Payne, chair of the Department of Biomedical Informatics at The Ohio State Univ. College of Medicine. “Furthermore, we found it is equally important to recognize the differences and synergies between IT and informatics leaders and to ensure that informatics leadership is properly empowered to advance the clinical and translational research agenda.”
Combining these different fields allows for the “rapid translation of discoveries from the lab into treatments that directly benefit patients,” Payne concludes.
In late 2011, the U.S. National Institutes of Health (NIH) founded the National Center for Advancing Translational Sciences (NCATS) in an attempt to speed the medical discovery process through multidisciplinary research. Already, the center has yield impressive achievements that include a technology that allows a paralyzed individual to use a robotic arm through brainwaves and a microfluidic test that uses human tissue to detect possible toxicities of new drugs earlier in the course of development.
When developing new drugs, the private sector mostly focuses on medical conditions that affect a majority of patients as this allows them to recoup the millions spent in development quickly. However, a university or government program does not operate for profit, enabling them the freedom to focus on rare and neglected diseases. A rare disease is considered, by the NIH, to be a disease that affects less than 200,000 people. Devastating conditions such as Huntington's disease and cystic fibrosis are overlooked because pharmaceutical companies cannot recover the cost of producing the treatments when only a relative few would benefit from them. The case is the same for neglected diseases, those that affect people in poverty around the world. While the World Health Organization estimates that a sixth of the world suffers from conditions such as hookworm, there is little profit in developing drugs to address these problems.
Because of cost and distribution, translational research is frequently performed as a joint effort between academic institutions and biopharmaceutical companies. These ventures combine manpower and resources while sharing the cost. This also gives researchers an outlet for their completed pharmaceuticals. Companies can work with non-profit organizations, partially funding the project, in return for marketable end products.
One such example is a collaboration between the Karolinska Institutet, located in Stockholm, Sweden, and AstraZeneca, who are working together to open a translational research center focused on discovering biomarkers for diseases.
"This project is a great and important step for medical science,” says Jan Andersson, professor of infectious diseases at the Karolinska Institutet. “It's good for the patients, for the growth of the healthcare sector and for the future of the pharmaceutical industry."
There are also collaborations in the works to perform research on neglected diseases. The Univ. of Cape Town (UCT) in South Africa and Novartis International AG are working together to advance medicines that treat some African diseases, starting with tuberculosis (TB) and malaria. Their goal is to develop a clinical study site in Cape Town to conduct proof-of-concept studies of new compounds developed at the university’s Drug Discovery and Development Center (H3-D). Novartis is providing H3-D with chemical starting points for new medicines against TB.
“UCT is committed to providing a meeting point for Western and African expertise to collaborate on solving problems such as Africa’s substantial burden of disease,” says Max Price, the Vice Chancellor of the university. “This partnership with a pharmaceutical giant of Novartis’ caliber has the potential to benefit the entire continent.”
Translation research collaborations can also take place between small companies and larger businesses. Edison Pharmaceuticals, Inc., Mountain View, Calif., was formed in 2005 by patient families, physicians, scientists and foundations to find treatments for inherited mitochondrial disease, a rare, debilitating childhood disease that often results in death.
Edison Pharmaceuticals has entered a licensing agreement with the Japan-based Dainippon Sumitomo Pharma Co. (DSP) for developing and commercializing drugs for pediatric orphan inherited mitochondrial and adult central nervous system diseases. Edison Pharmaceuticals receives $35 million upfront and $15 million for R&D support. In return for the cash injection, DSP will have development and commercialization rights to the drugs in Japan.
Translational research allows scientists to work together and create new treatments and drugs in ways that would not be possible with traditional R&D. Taking a multidisciplinary approach to drug discovery and development allows more effective solutions to penetrate the marker faster. It also enables researchers, in both the non-profit and private sector, to focus on less profitable pharmaceuticals to help those with diseases that may be overlooked by mainstream R&D.