Merging Laboratory Knowledge into Model Systems Streamlines New Treatment Protocols
By Dr. Gabi Hanna
COVID-19 has impacted our lives and industries in ways we never could have imagined. While the pandemic’s affect has been negative for the most part, it has inspired industries that are needed to help fight the virus. Among those is the pharmaceutical industry and in particular, the development of new drugs and treatments. The U.S. pharmaceutical industry has often been agonizingly slow to bring treatments to market, hampered by handoffs between academic and commercialization teams, and cumbersome processes for regulatory approval. The average time it takes a new treatment to get through clinical trials and achieve marketing approval is estimated to be more than 10 years by most industry experts. While proving new drugs safe and effective certainly should be done with care, the need to find ways to accomplish this more quickly is also clear, particularly to patients waiting for life saving treatments.
As a physician, I have had patients desperately seeking treatments for diseases without them for many years and sought to improve the situation by embarking on a career in translational medicine. As much as we want to bring new drugs to market to address decades old diseases that are killing hundreds of thousands of people, COVID-19 threw the entire need for quick drug development into hyperdrive, which has placed many other diseases on the backburner. However, it doesn’t have to be this way.
Aware of the snail-speed process of clinical trials, the pharmaceutical industry found itself under intense pressure to develop, test and mass produce a vaccine to treat COVID-19. The good news is, the research industry – both commercial and academic – was already experiencing a paradigm shift driven by translational research. Translational research employs cutting-edge technologies that enable innovative solutions in an effort to speed up the development process, while improving patient care more quickly and without sacrificing safety and efficacy. It is a relatively new term, and is defined as the area of research that targets improvements to health resulting from connecting novel discoveries in the biological sciences to human disease. Translational medicine aims to tie new knowledge and scientific findings from the laboratory to clinical practice in a direct and effective way that is meant to accelerate the path from the science to the product, pill or treatment that can make a difference to the patient.
The power and technology of available methods in private and academic research include whole-genome and targeted sequencing, high-resolution microscopy, mass spectroscopy, cell-based assays, and much more. These cutting edge medical-based technologies are just beginning to have impact on clinical experimentation, in large part because the venues for collaborative, interdisciplinary, cross-institutional interactions that sustain intellectual exploration of translational challenges have been scarce, largely guarded, and siloed.
Opening up the use and adapting powerful technologies aimed at actionable clinical data requires systematic collaboration and effort. Scientists, physicians, clinicians, along with teams of dedicated professionals who understand real-world constraints on the availability and quality of clinical samples, turnaround time for clinical experiments, and the ways in which inter-patient variability may impact experimental robustness, are just as important to the process as the new technological devices used in a lab.
Following my medical training, I joined Duke Clinical Translation Unit, where as director I was able to learn firsthand about the benefits and innovative research coming out the academia that could be directly applied to patient care. To further explore the role that translational medicine represents, I created and launched the first translational research project that explores treatment for acute pancreatitis.
The compound that is being developed to fill this critical, unmet clinical need for the treatment of acute pancreatitis, to mitigate the toxicity and organ failure associated with the disease that causes lengthy hospitalizations, organ failure and death, can help save both lives and resources. To meet the collaborative requirement of translational research, our patients, investigators and leaders on the project are students of translational medicine, as we all work towards advancing knowledge of this rare disease to bring this treatment to market.
This therapy for pancreatitis has been developed based on the knowledge of practicing physicians treating patients who have recognized that acute pancreatitis leads to the release of pancreatic digestive enzymes. These enzymes wreak havoc in the body, and in severe cases, proteases and lipases are activated in a cascading effect, causing massive necrosis and organ failure. There is no available treatment, but based on this knowledge and observations from the clinic, the team plans to conduct clinical trials on the lipase inhibitor identified as a potential treatment by translational medicine researchers familiar with the science, the patient needs and the disease.
This compound is a shot injected directly into the pancreas; while we may seek to one day adapt it into an IV or oral medication that is more convenient and marketable, right now our priority is getting the treatment to patients once we have the safety and efficacy data needed. We hope the new approach will make a meaningful impact on these patients’ lives right away. This is one example of the way translational medicine can be applied to real world medical situations, and we have plans to explore and develop additional treatments for other serious conditions that have no cure today.
For all its life saving innovations, the U.S. pharmaceutical industry has plenty of room for growth and innovation, particularly in the processes that bring drugs to market. Translational research and medicine have the ability to accelerate research safely and effectively in order to bring new treatments to market more quickly.
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