Raises $180,000 for PKU research

July 17, 2018

For Ph.D. candidate Daelyn Richards, it was not a typical scientific talk. She was in a ballroom. Audience members were dressed in suits and dresses. A 60 Minutes correspondent emceed the event.

Richards was speaking at a benefit gala for the National PKU Alliance, invited by that organization in May to describe her lab's efforts to develop better treatments for PKU or phenylketonuria. PKU is a rare metabolic disorder currently treated with onerous and expensive dietary management.  

"The starting bid was $25,000, which is almost more than I make in a year," said Richards. "So the pressure was on."

But Richards, who competes in marathons and Ironman triathlons, enjoys a challenge. In seven minutes, she passionately described the promising work led by her mentor, Cary Harding, M.D., professor of molecular and medical genetics, OHSU School of Medicine.

For example, the U.S. Food and Drug Administration recently approved a first-of-its-kind enzyme therapy for PKU, a treatment developed in part through a clinical trial led by Dr. Harding, who specializes in metabolic diseases.

Richards' focus in the Harding Lab is to develop other novel treatments such as a gene therapy strategy that uses CRISPR technology in an animal model to edit mutated genes and deliver a cure. It was this work that she presented at the gala.

"My project has had great success in correcting PKU model mice with liver-directed, adeno-associated virus (AAV) gene therapy with permanent effects," she explained.

Her main message to the audience that evening: It's working, and there's hope. At the conclusion of her presentation, she brought the audience to its feet with tears and cheers. She cried herself. Families with children living with PKU came up and hugged her afterward. Her talk raised $180,000 towards PKU research.

"It was an emotional and unforgettable evening," she said.

Richards first became hooked on the promises of gene therapy after taking a gene therapy class as an undergraduate at Portland State University. She graduated with honors from PSU and joined OHSU's Program in Molecular and Cellular Biology, where she found a happy home in the Harding Lab.

Since then, Richards' research has been garnering accolades. She gave invited talks at this year's American Society for Gene and Cell Therapy Annual Meeting and the National PKU Alliance's Annual Conference. She received 2018-2019 and 2016-2017 Tartar Trust Fellowships to support her research. And at this year's Research Week, she won the Fast Pitch competition's best graduate student award for her presentation "No regerts (sic): Permanent gene therapy for PKU."

"Daelyn is very energetic and positive," said Dr. Harding. "She has a knack for explaining her complicated research to lay people in a clear, concise and entertaining way. Her creativity is a tremendous asset to her science."

Richards' interest in PKU doesn't end at designing and running scientific experiments. She lights up when she describes how she volunteers each summer at PKU Family Camp where local PKU families come together for workshops and fellowship.

Among her camp responsibilities is to give updates on PKU research. "Honestly, my favorite part of the science I do is sharing it with the community it aims to treat," she said.

She also spends time understanding the experience of PKU. "It's so impactful to hear about the struggles these patients go through every day just to get by," said Richards. "I hear from the kids about how hard it is to be different and how they struggle with their diet. I hear from adults, some of whom have to choose between paying rent and paying for their diet. But if they go off their diet, they suffer severe neurological consequences."

She added, "Science can be cold, but when you bring people into it, it motivates me for the long, lonely times in the lab. I just keep thinking about these families and kids and how we need to help them."

She's even gotten involved in public policy, helping PKU patients engage Congress on advocacy issues. "This year, I will be heading an advocacy session to help campers write their representatives regarding the proposed Medical Nutrition Equity Act, which would require insurance companies to help cover the cost of the medical diet for PKU and allied disorders," she said.

When she's not volunteering with PKU families, she mentors middle and high school students from underrepresented backgrounds through the On Track OHSU! outreach program, volunteers with the Chemo Pal Mentor Program to help children during chemotherapy visits and teaches STEM classes at Portland's Saturday Academy.

As she works to complete her Ph.D., Richards is looking ahead to life after graduation. Though she plans to stay in the field of genetics and PKU, she's decided not to pursue a bench science career in an academic lab. She's considering options in industry, she says, but her primary goal will keep her in school. She's applying to medical school in order to merge her interests in people and research into a career as a physician-scientist.

"I feel a responsibility to use these skills and training to directly benefit people," she said. "It sounds corny but it's true. Why else are we all here?"

Pictured: Ph.D. candidate Daelyn Richards (top, bottom), with 60 Minutes correspondent Scott Pelley at the May "Lifting the Limits" PKU benefit gala