Moving Toward the Pharmaceutical Industry of the Future with Translational Medicine

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 over 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 treated 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.

Translational medicine 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.

Following my medical training and surgical residency, I chose to pursue a career in research where I could impact more than one patient at a time – even millions of patients -- by developing new treatments for diseases with none or few satisfactory treatment options. This path led me to the 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 applied to patient care.Most recently, I have established my own company, Lamassu Pharma, in order to further explore the role that translational medicine techniques can play in accelerating time to market, and the first treatment we’ll be exploring is one for acute pancreatitis.

My company, Lamassu Pharma is a leader in translational research and recently received Small Business Innovation Research (SBIR) grant funding from the National Institutes of Health (NIH). The funding will be used for further development of its lead therapeutic compound, RABI-767, a novel small molecule lipase inhibitor licensed from the Mayo Foundation for Medical Education and Research and being developed with translational medicine techniques.

The compound is being developed to fill a critical, unmet clinical need for a treatment for acute pancreatitis, to mitigate the toxicity and organ failure associated with the disease that causes lengthy hospitalizations, organ failure and death, saving both lives and resources.The patients, investigators and leaders on the project are students of translational medicine as we all work towards advancing knowledge of this rare disease and bringing 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, my 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 an injection made directly to 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. I chose to pursue a career in research to impact patient with disease that are today untreatable. It is my goal as an entrepreneur and innovator to work to decrease time to market, and to use new technology and processes, like translational medicine, to accelerate research and bring treatments to market more quickly.

Gabi Hanna, MD, CEO, Lamassu Pharma