A Pathway to Hope

Well before Margie Hempling McGlynn mopped floors and shoveled snow at her father’s South Buffalo pharmacy, tragedy struck her family—not once, but twice. One of seven children, McGlynn was four when her 6-year-old sister, Judy, was diagnosed with homocystinuria, or HCU, a rare metabolic genetic disease that took her life in 1972 at age 14. That same year, another sister, Susie, 9, succumbed to the same condition.

McGlynn’s father, Ed Hempling, was at a loss for how to help his daughters; HCU had been discovered just a year prior to their diagnosis, and little was known about how to treat it.

“I was lucky I was spared, says McGlynn, MBA ’83, BS ’82, recalling the family getting tested after Judy’s diagnosis. She later learned she wasn’t a carrier.

Both of McGlynn’s parents were genetic carriers of HCU, the most common type of which affects one in 200,000 people globally and is often diagnosed at birth. HCU is marked by elevated levels of an amino acid, homocysteine, which can cause severe cognitive and physiological effects on a child’s growing body. In the past, it often led to early impairment and death, but today it can be better managed through aggressive dietary restrictions and supplements.

McGlynn was determined not to let HCU affect another family member. She had also become fascinated with medicine while working with her father. Both factors motivated her to enroll in the undergraduate program in the UB School of Pharmacy and Pharmaceutical Sciences, adding a concurrent MBA in marketing from the School of Management (dual MBA/PharmD graduate program). In 1981, she was accepted to a national summer internship program at Merck with the intent to one day take over her father’s pharmacy business. But as she rotated through various departments at the pharmaceutical giant, she quickly realized that it was the ideal place to maximize her dual degrees.

“At the time, Merck had so many innovative products, like a Hepatitis B vaccine, Zocor for high cholesterol, etc.,” she says. She stayed for 26 years, eventually leading Merck’s global marketing and U.S. hospital and specialty business.

In 2009, McGlynn, by then president of Merck’s $8 billion global vaccine and anti-infectives business, took early retirement and established the Hempling Foundation for Homocystinuria Research, which supports pre-clinical HCU therapies around the world. She also spent four years as CEO of the International AIDS Vaccine Initiative (IAVI), where she traveled extensively to accelerate HIV vaccine research in developing countries.

After leaving IAVI in 2015 to focus on HCU, McGlynn co-founded HCU Network America, a nonprofit advocacy and support network for patients and their families. She has served on the boards of a few biotech companies, including one developing an HCU treatment she helped to fund, and recently joined the board of vaccine maker Novavax.

In 2020, McGlynn and UB announced a major gift to create the Margaret Hempling McGlynn Chair in Clinical Pharmacy, what she calls “a compelling, integrated clinical care program that addresses the interdisciplinary aspect of rare metabolic diseases.” The clinical pharmacy faculty member appointed to this newly endowed role will help educate and mentor health science students on clinical care for children with genetic and metabolic disorders, and collaborate with the UB genetics division and John R. Oishei Children’s Hospital pediatric metabolic clinic to support its patient care, education and research missions.

Metabolic diseases can be difficult to manage, requiring a coordinated medical effort across many health fields including genetics, psychiatry and dietetics, McGlynn points out. “All disciplines should be well-trained, not only on the disease but to really understand patients and what they live through,” she says. “Kids with HCU can’t eat the cake at birthday parties, and travel can be hard because they have to take refrigerated nutritional formula several times a day.”

McGlynn, who has a grown son and daughter and lives with her husband in Flourtown, Pa., believes her professional and personal story put her on a unique path to solving HCU’s mysteries. Her advocacy group is working to improve newborn screening, among other issues, and the enzyme replacement therapy her foundation helped fund is only a few years away from reaching the market.

As McGlynn’s remarkable journey continues, it enables her to support pharmacy at her alma mater. And as life often does, it sparks memories of pain, and promise.

Story by Lauren Newkirk Maynard

Photograph by Heavy Content Studios