Lupus is a complex autoimmune disease that affects more than 1.4 million Americans. Its treatment in the United States alone costs about $7 billion per year. Despite this impact and cost, the Food and Drug Administration has approved only one drug for its treatment in the last 60 years.
“Double-Hoo” Amrie Grammer, the founder, chief operating officer and chief science officer at Charlottesville-based biotech company AMPEL Biosolutions, is working hard to make new inroads to the treatment of lupus and other autoimmune diseases.
Grammer earned University of Virginia bachelor’s (1989) and master’s degrees (1991) in chemistry and pharmacology, respectively, and holds a Ph.D. in immunology from the University of Texas Southwestern Medical Center.
Over email, we talked with Grammer about her Lupus research and her company’s collaborations with UVA.
Q. May is World Lupus Awareness Month. What are some of AMPEL’s breakthroughs in drug identification for treating this disease, and how does data science play into that?
A. Lupus is a highly complex disease, with more than 100 different genes contributing to susceptibility. Even in genetically identical twins, one can emerge with lupus while the other does not. Over the last six years, AMPEL has created the world’s largest lupus patient data set and with that has developed innovative tools to bring potential treatments to lupus patients.
Our research and development program is focused on using big data tools for improving health care. We began in 2013 by developing a multi-pronged approach to rank existing drugs in the marketplace to be repositioned for the safe, effective treatment of lupus. As a result, we can identify drugs that should be considered top priorities for treating lupus patients.
Last fall, AMPEL’s system for drug repositioning was confirmed by the success of the phase II trial of Stelara [an immunosuppressive drug] in lupus, meaning for the first time in 60 years, lupus patients have a new treatment option.
And this spring we announced a breakthrough in precision medicine for the treatment of lupus patients. Until now, rheumatologists have not had a tool that predicts when a lupus patient is about to experience a flare-up. AMPEL’s approach to personalized medicine identifies the groups of genes expressed in the blood of lupus patients in real time, assesses if the patient is flaring and provides decision support to physicians for top drugs that could be prescribed to precisely treat that patient.
In addition, our approach supports clinical trial enrollment for testing new drugs by ensuring that patients entered into a trial actually have the potential to respond to the drug tested, which is not always the case in clinical trials, and the reason so many trials fail.
AMPEL also has patented tools and machine-learning approaches for a clinical genomic test that can predict top drug options for a patient based on individual gene expression. Our work really demonstrates the importance and powerful role that data science is playing in drug discovery and personalized medicine.
Q. Why did you locate your business in Charlottesville?
A. I loved living in Charlottesville as a student. After years as a translational researcher at the National Institutes of Health Autoimmunity Branch, my goal was to figure out a way to return and use my scientific training to form a company.
Charlottesville has the vibrancy of collaboration and brims with excitement for new ideas. UVA recruits smart, creative students and teaches them out-of-the-box thinking – exactly what startup companies need. There is synergy between disciplines here. For example, I serve on the board of UVA’s Fralin Museum of Art, a museum that encourages exhibitions that merge art and science.
A few weeks ago, wearing my hat as a board member of the statewide Virginia Bio organization, I brought Virginia companies together with UVA students on AMPEL’s patio for a quarterly networking event. A striking draw to Charlottesville for me was UVA’s entrepreneurial and innovative environment that reaches outward to the community.
“UVA provided me with a solid foundation in science, writing, public speaking and strategic thinking. A prepared mind is crucial to seeing solutions where others see roadblocks.”
- Amrie Grammer
Q. How is AMPEL collaborating with UVA?
A. AMPEL has an active collaboration with the lab of Dr. Shu Man Fu, an immunologist and rheumatologist at UVA, to identify new drug targets in lupus. We’re also exploring collaborations with faculty in the biomedical engineering department and with the Data Science Institute. We host “meet and greet” seminar luncheons for UVA students at the beginning of the fall and spring semesters – some on Grounds and some at our Charlottesville office, and, during monthly seminars, feature UVA faculty. Our chief medical officer, Dr. Peter Lipsky, has guest-lectured in biomedical engineering professor Mete Civelek’s bioinformatics class.
AMPEL’s first employees were UVA grads, and we recruit a new class of interns every year from around the University, such as Darden, chemistry, biology, biomedical engineering, data science, cognitive science, public health and the M.D.-Ph.D. program.
AMPEL participated in [the Department of] Biomedical Engineering’s capstone program last year and are hosting data science capstone students this summer. Our interns have won international awards and been accepted to prestigious programs around the country to receive their Ph.D.s, J.D.s with patent law concentration and M.D.s with a science focus.
Triple-Hoo Adam Labonte, staff scientist at AMPEL, and Brian Kegerreis, Rodman Scholar and biomedical engineering graduate, published a groundbreaking paper last December that describes the genes that differentiate “active” from “inactive” lupus, potential drugs to change disease state as well as a machine-learning approach to bringing awareness to the cell types that are the biggest contributors to disease pathogenesis.
Haley Davis, a rising UVA third-year student, won first prize in “Physiology and Immunology” at the Sigma Xi’s “Big Data” Student Research conference in Silicon Valley last October for her work with AMPEL to prioritize potential drug targets for lupus. Madison Smithers, an AMPEL intern, Jefferson Scholar and rising UVA third-year, was short-listed for the 2018 international Nature Research Innovating Science Award that celebrates achievements of women in science. And Brian Ho, an AMPEL intern and recent Darden graduate, was selected for Danaher’s prestigious six-year Scientific Management Training Program.
Q. How did UVA prepare you for a career as a biotechnology entrepreneur?
A. UVA provided me with a solid foundation in science, writing, public speaking and strategic thinking. A prepared mind is crucial to seeing solutions where others see roadblocks. It is this mentality that penetrates the work that we do at AMPEL.
UVA provided me with rigorous training, faculty mentoring and exposure to collaboration that was a fabulous foundation for my academic career at the NIH. As I’m celebrating my 30th reunion this June with friends from my undergraduate years, I am grateful for the opportunities that my education at UVA provided and for the collaborative Charlottesville community that I am part of today.
Figuring out how to make a difference in the lupus field has been challenging. My thinking about the disease started at UVA during my master’s degree training with Dr. Ron Taylor, who is an expert on “complement,” a part of the immune system that is a readout for severity of lupus. And my interactions with UVA faculty, such as Dr. Jim Demas, as part of Alpha Chi Sigma, a professional chemistry fraternity, exposed me to scientific debate and collaboration.
UVA’s pharmacology curriculum further drove my thinking about how cells interact with each other and are signaled to regulate gene expression.
I can say that my preparation at UVA has helped in my company’s approach toward making a big difference in the treatment of lupus patients.