Is There a Better Way?
Only about half of lung transplant recipients survive five years after the procedure, according to the National Institutes of Health.
Bill Williams thinks that's not good enough. And while he's not a surgeon or even a medical doctor, his work could help drastically improve life expectancy.
A transplant patient must take immunosuppressant medications for the rest of his life so that his body does not reject the new organ. Unfortunately, that leaves him susceptible to any number of deadly infections and diseases.
Williams, chair of the pharmaceutics division in the College of Pharmacy, is working on a new method to administer the immunosuppressant drug for lung transplant patients so that it goes straight to the lungs without traveling through and damaging the rest of the body.
Currently patients take the drug as a capsule or tablet. Williams and his research partner, Dr. Jay Peters, a physician at the UT Health Science Center in San Antonio and an adjunct professor in the College of Pharmacy, decided to rethink how that medicine could be delivered.
We asked Professor Williams:
What's one thing the public should know about developing new drugs?
You have to have a healthy NIH (National Institutes of Health) and healthy funding, and a healthy pharmaceutical industry. That's what drives innovation. With a healthy government and a healthy industry working together, patient populations of these really small but significant illnesses don't get forgotten.
Think about a patient who gets cancer after lung transplant surgery. It's a small patient population, but that has a disproportionate cost to that patient, that patient's family and the health care system.
They developed new technologies that convert the drug particles into aggregates of nanoparticles so the medicine can be inhaled directly into the lungs by either a nebulizer or dry-powder inhaler.
"Our theory is that if we get it right to the site where the immune suppression is needed, then we would have fewer systemic side effects," Williams says.
The drug in this new delivery form tested successfully in healthy human volunteers. Williams and Peters have been working on this particular drug for about five years, from idea to testing to reformulating to testing again, building their intellectual property along the way. Each stage of the drug development process takes about 18 months. Next comes licensing out to a pharmaceutical company for full-blown clinical trials and submitting to the regulatory process.
If everything proceeds perfectly from now (a big if), lung transplant patients could be using the medicine within four years which, for patients getting transplants today, could be just in time.
Engineers' Breakthrough Could Revolutionize Treatment
A physical form of proteins developed by UT researchers could drastically improve treatments for cancer and other diseases, as well as overcome some of the largest challenges in therapeutics: delivering drugs to patients safely, easily and more effectively.
The protein formulation strategy, developed by faculty and students in the Cockrell School of Engineering's Department of Chemical Engineering, is unprecedented and offers a new and universal approach to drug delivery one that could revolutionize treatment of cancer, arthritis and infectious disease.
"We believe this discovery of a new highly concentrated form of proteins clusters of individual protein molecules is a disruptive innovation that could transform how we fight diseases," says Keith P. Johnston, a chemical engineering professor and member of the National Academy Engineering.
Typically, protein biopharmaceuticals are administered intravenously at dilute concentrations in a hospital or clinic. Scientists and engineers have long tried to produce safe drugs at higher concentrations, so that a patient could self-inject the drugs at home, similar to an insulin shot. But doing so has been stymied by the fact that proteins, in high-concentration formulations, form aggregates that could be dangerous to patients and gels that cannot be injected.
The Cockrell School research team has introduced a new physical form of proteins, whereby proteins are packed into highly concentrated, nanometer-sized clusters that can pass through a needle into a patient to treat disease. The novel composition avoids the pitfalls of previous attempts because drug proteins are clustered so densely that they don't unfold or form dangerous aggregates.
Since the research began in 2004, three patent applications have been filed through the university's Office of Technology Commercialization.
"This general physical concept for forming highly concentrated, yet stable, protein dispersions is a major new direction in protein science," Johnston says.
More than Just "Blockbuster" Drugs
Janet Walkow is the executive director and chief technology officer of the Drug Dynamics Institute (DDI) in the College of Pharmacy, which brings together scientists and investigators to work on novel solutions for disease and health care issues. She is passionate about the need for scientists and the general public to understand how medical therapies are developed.
Watch a clip from the Longhorn Network "Game Changers" episode about Walkow (below), where she discusses the reality of the cost of bringing a new drug to market and how universities like UT can help complete the work that big pharmaceutical companies ignore.
This story is part of our yearlong series "In Pursuit of Health," covering medical news and research happening across the university.
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