Historically, finding a new molecule that leads to a new drug discovery has been more difficult than finding the proverbial needle in a haystack.
However, advances in information technology, chemistry and medicine have substantially increased the probability of a successful outcome.
The Utah Science Technology and Research (USTAR) initiative and the University of Utah (U of U) have created a new core facility with a goal of developing pharmacological therapies. The facility will build upon the university’s established track record as a leader in translational research and drug development, coupled with USTAR's proven ability to commercialize technologies.
“The idea is to bridge the gap between small molecule chemistry and the potential biological targets that those molecules might hit as drugs,” said Cynthia Burrows, chair of the chemistry department. “We have great scientists on the medical school campus who understand disease and the underlying biology behind the disease. We have a group of chemists on campus who know how to make molecules and understand the properties of drugs. The core can facilitate collaboration between those groups. “
The USTAR Synthetic and Medicinal Chemistry Core has been established at the U of U and will span traditional departmental boundaries to facilitate the research of chemists, biologists, materials scientists, engineers and others to develop what are called lead compounds and identify new targets for treating disease , and new materials for biomedical and energy-related industries.
Leading the core are Paul Sebahar, senior medicinal chemist with more than 12 years of experience in the pharmaceutical and biotechnology industry, and Ryan Looper, associate professor and Henry Eyring fellow in the department of chemistry. Initially housed in the department of chemistry, the core will partner with the U of U’s Drug Screening Resource to stimulate Utah’s biotechnology industry.
“We are here to support those at the university who are interested in research at the early stages,” said Sebahar. “We want to bridge the gap between industry and academics, and hopefully take early discovery projects to a point where they can attract commercial partners.”
The USTAR Synthetic and Medicinal Chemistry Core was created with the vision to increase Utah’s research capacity and fuel the economy by developing new and repurposed pharmacologic therapies for diabetes, cardiovascular disease, neurological and neuropsychiatric disorders, cancer and infectious diseases.
“We realized that there is a need to support ongoing research at the University of Utah through the re-design of initial screening hits and preliminary small molecule discoveries to more pharmaceutically relevant lead compounds with commercialization potential,” said Looper. “The core is poised to translate early stage discoveries from world-class research programs within the U of U and other state institutions to clinical successes and commercially viable products and services.”
U of U organizations involved in the core are the Huntsman Cancer Institute and the Moran Eye Center, the departments of chemistry, medicinal chemistry, biochemistry, human genetics, pathology and oncology. The objective is to reach out to more disciplines including biomedical engineering and the newly formed Center for Cell and Genome Science.
A major objective of the core is partnering with local companies that have initiated biopharmaceutical projects, but lack the medicinal chemistry resources to advance their small molecule research. One example of an industry collaboration is with Curza Global, a U of U spin-out. The team is working to develop a new antimicrobial agent that will inhibit and disperse biofilm formation of relevant pathogenic bacteria. The resulting product could help fill a pressing need for new therapies to combat bacterial infections that are resistant to the current arsenal of antibiotics.
“There are a number of strengths on the health science campus that we can add to. The idea is to partner as much as possible with everyone,” said Burrows. “We’d like to see fundamental work in chemistry in terms of constructing new molecules and new understanding, in addition to identification of a chemistry and biology connection to disease.”
Tom Parks, vice president for research for the U of U, says there is a need for stronger drug research and development infrastructure in the state. The core will help the state’s life sciences industry by allowing Utah researchers and companies to take advantage of the market opportunities in the global pharmaceutical industry, with annual sales of nearly one trillion dollars.
“The partnership between the University of Utah and the USTAR program to fund a Synthetic and Medicinal Chemistry Core facility on our campus will provide a key resource for drug development programs at the university and for local companies,” says Parks.
According to Burrows, one benefit of the new core will be to increase the likelihood of securing federal research grants for biomedical research. The National Institute of Health (NIH) has an increasing emphasis on translational research, from the lab to the clinic, and this facility will accelerate drug discovery and development.
The core is already partnering with several research teams around campus on important projects. One project is with the Moran Eye Center to develop new compounds for glaucoma treatment. Another is with the department of pathology to develop compounds for immune-mediated diseases. There is also a partnership with the Huntsman Cancer Institute to identify classes of compounds with the ability to treat drug resistant cancer cells.
Looper said one of the ultimate goals is to accelerate commercialization in a shorter amount of time. It is his hope that new discoveries can reach the marketplace in as little as one to three years.
“Interest generated from the novelty of the compound or biological target, efficacy and safety dictate the timeline for commercialization of any project,” said Looper. “After securing data, this would hopefully be followed up by industrial partnerships or collaborations within the state of Utah to move compounds through the FDA or EPA regulatory process.”