Discovery isn’t enough: How U-M’s College of Pharmacy turns science into treatments that reach patients

By Vicki Ellingrod
Dean, College of Pharmacy

That work has never been more urgent than it is right now.

Consider what it takes to bring a single drug to market. The process typically spans 10 to 15 years and costs billions of dollars. Nearly 90% of drug candidates fail during clinical testing, not because the underlying science is wrong but because we lack reliable ways to predict how a compound will behave in the human body at clinically relevant doses. Additionally, the so-called “valley of death” – the period between discovery and into clinical trials – claims many promising findings because funding runs out before they can leave the lab. For every discovery that doesn’t make it out of the lab, countless patients may not receive lifesaving treatment.

The College of Pharmacy has been and will continue to meet these challenges from multiple directions at once.

From “undruggable” to FDA-approved

For nearly two decades, researchers Jolanta Grembecka and Tomasz Cierpicki pursued a target that the pharmaceutical industry had largely written off. The protein menin contains a binding pocket that, in patients with certain genetic mutations, drives the aggressive proliferation of leukemic cells. Targeting protein-protein interactions like this one was considered undruggable.

Grembecka and Cierpicki started this work at the University of Virginia in 2007 and brought it to Michigan in 2009. They produced the human protein, created the biochemical assays and solved the crystal structure of menin, the molecular blueprint that made it possible to design small molecules capable of fitting into that binding pocket and disrupting the cancer-driving interaction.

The result, after years of medicinal chemistry optimization, was ziftomenib, the first and only once-daily oral therapy for patients with relapsed or refractory NPM1-mutant acute myeloid leukemia. Unlike traditional chemotherapy, which kills cells indiscriminately, ziftomenib causes cancerous cells to differentiate into normal white blood cells and eventually die.

The work was done in collaboration with the College of Pharmacy’s Pharmacokinetics and Mass Spectrometry Core, directed by Duxin Sun.

This is what the translational discovery process involves: persistent, methodical work sustained over nearly two decades supported by federal funding from the National Institutes of Health and grant funding from the Leukemia & Lymphoma Society and the American Cancer Society. When this research can make its way out of the lab, these discoveries account for potentially saving lives, and in this case, the lives of patients who had very few options.

A gathering and what followed

In July, CIRCLE co-hosted the Bemidji Area Substance Use and Pain Management Gathering with the university’s Opioid Research Institute, which provided substantial programmatic support and expert speakers. More than 100 people attended, including university faculty, students and tribal health leaders and clinicians from across the region.

Topics were shaped with input from tribal partners and included both clinical evidence and culturally responsive perspectives. Among 42 respondents to a post-event evaluation, 96%agreed the gathering met its learning objectives and 93%reported greater social support for their work. Participants identified concrete changes they planned to make, including refining screening practices, strengthening harm reduction strategies and improving team communication.

They also named real barriers: limited administrative resources and time constraints. CIRCLE is not offering itself as a fix for those pressures. But the relationships built at the gathering have since generated new collaboration and contributed to research proposals now in development — a downstream product of convening rather than the original objective.

A framework that changed how the world approves drugs

In 1995, College of Pharmacy researcher Gordon Amidon developed the Biopharmaceutics Classification System (BCS), a framework for classifying drugs based on how they dissolve and how they cross the intestinal wall. The BCS gave the U.S. Food and Drug Administration (FDA) and regulatory agencies worldwide a scientific basis for granting “biowaivers,” allowing certain drugs to skip costly and time-consuming bioequivalence studies in human subjects when their absorption profiles are well understood.

The impact has been enormous. BCS accelerated the availability of high-quality generic medications, reduced the need for unnecessary clinical trials and provided a rational framework that countries around the world adopted. For patients, particularly in low- and middle-income countries, it means faster access to affordable medicines. It is one of the most consequential regulatory tools in the history of pharmaceutical science, and it came from the College of Pharmacy.

Amidon’s work is now being applied to newly available biosimilars by Anna Schwendeman,who is a faculty member at the college and co-leads the Center for Complex Generics, funded by the FDA. Complex Generics differ from standard generic medications because they involve scientific, manufacturing or regulatory complexities that can be challenging to overcome while maintaining the same therapeutic effect. Examples include peptide medications, liposomal formulations, and metered dose inhalers. By serving as the conduit between pharmaceutical companies and the FDA, Schwendeman is helping to develop the regulatory framework by which new complex generic medications make it to the market faster, ultimately reducing the cost of prescription medications for patients.

An even more recent discovery that has made it out of the lab and into patient care occurred in 2025, when. Dan Hertz and colleagues both within the college and in other areas of the university succeeded in their quest with the FDA to get the label changed on capecitabine (Xeloda), a common therapy for cancer, because it can be toxic or fatal for patients who carry variants of a specific DPYD (dihydropyrimidine dehydrogenase) gene. As a direct result of this work, it is now recommended that all patients be tested for the variants before taking the drug. The impact? Patients’ lives are saved.

The constant drumbeat – we close the gap between scientific discovery and patient care.

A care model built for the people who need it most

The gap between knowledge and care is not always molecular. Sometimes it is a matter of who delivers the care and how often a patient is seen.

In 1999, the late Hae Mi Choe developed the Hypertension Pharmacist Program, embedding specially trained pharmacists in primary care clinics to provide close, consistent follow-up for patients with uncontrolled high blood pressure. The results were striking: 66% of participants had their blood pressure under control within three months, compared with 42% of non-participants. The program expanded from a single clinic to all 14 U-M Health primary care sites, then into retail pharmacies through a partnership with Meijer. Patients could receive clinical care in a more convenient setting with access to their electronic health records.

The Centers for Disease Control and Prevention (CDC) recognized the program as a national best practice. The CDC is now scaling our model to health systems in the southeastern United States that serve primarily Black patients, communities where persistent disparities in blood pressure control translate directly into higher rates of stroke and heart disease. Additionally, the college is partnering with the tribes of Michigan to implement this model, working to solve some of their most pressing health care challenges locally.

This impact shows what is possible when a pharmacist has a seat at the table and is adopted into care teams. It is a system of care designed to reach the people who need it most, proven to work in the real world and now being adopted at a national scale because we demonstrated that it could be done.

Changing the odds for the next generation of drugs

Earlier this year, U-M launched the Institute for AI-Driven Therapeutics Discovery. It is led by Duxin Sun, the same researcher at the college whose pharmacokinetic expertise helped bring ziftomenib from bench to bedside. AI-Tx addresses a question the entire pharmaceutical enterprise faces: if nearly 90% of drug candidates fail in clinical testing, and if the root causes of those failures are predictable, can we fundamentally change the odds?

The institute brings together researchers from the College of Pharmacy, the Medical School, the College of Engineering and the School of Information. The aim is to embed artificial intelligence across every stage of the discovery pipeline, selecting better targets, optimizing molecular structure and predicting how drugs will perform in patients at clinically relevant doses so that fewer promising compounds fail late, when the cost in money and time is greatest.

This is about giving scientists better tools to accelerate discovery. And it reflects something that has been true of the college’s approach to therapeutics for a long time: the most important innovations are often not only about the medications themselves but the systems, frameworks and methods that make better drugs possible and, most importantly, improve human health and save lives.

What the College of Pharmacy’s therapeutics legacy tells us

There is a thread that connects Prescott’s insistence to make pharmacy a science, Amidon’s framework that reshaped global drug regulation and Schwendmen’s work to reduce medication costs, Grembecka and Cierpicki’s two decades of work on a target the industry walked away from, Hertz’s insistence that precision pharmacotherapy can help save lives, Choe’s proof that pharmacists being a part of care teams can effectively manage chronic disease at scale and Sun’s institute applying computation to the drug development pipeline. In each case, someone at the U-M College of Pharmacy identified a gap between what science knew or could be and what patients could benefit from.

At the College of Pharmacy, we’re celebrating our sesquicentennial this year and have remained central to the university’s mission. Twenty-nine of our faculty were named to Stanford University and Elsevier’s list of the world’s top 2% of scientists. We produce the third-highest number of invention disclosures on campus, after the College of Engineering and the Medical School. Not bad for a college that is less than one-tenth their size. In fact, since 1985, the college has been issued 644 patents, which translates into more than one patent a month issued for the last 40 years. To top it off, we recently moved into a new 142,000-square-foot facility, the world’s tallest mass-timber research building, with 22 laboratories designed for the interdisciplinary work that will be a part of what allows us to lead at pharmacy’s edge for the next 150 years and more. The building’s use of mass timber is expected to reduce our embodied carbon footprint by 40%, reflecting our commitment to the future.

Our world needs therapeutics that are more personalized, accessible, developed faster and delivered more equitably. Unfortunately, chronic conditions are more common and health inequities demand the kind of systematic, evidence-based science that the College of Pharmacy has both built and advanced for a century and a half. We are the medications experts, the kind of experts healthcare needs as medications become more complex and sophisticated. We are proud to be leading at pharmacy edge to solve these challenges and improve the lives and health of Michiganders, the nation and our world.