• Mary Leonard, MD, MSCE
 

 

Translating Discoveries into Cures—Faster

Jennifer Yuan
Tuesday, May 23, 2017

Mary Leonard’s own children always know when she’s working in the hospital. They get unexpected text messages from her, reminding them to look both ways before they cross the street. It’s usually on the days when she’s walked through the pediatric intensive care unit and been acutely reminded of how devastating childhood injury and illness can be.

“My kids are 22 and 24,” Leonard says. “But my role as a pediatrician has always been deeply influenced by my role as a parent.”

Last July, Leonard became the first woman to serve as the Adalyn Jay Physician-in-Chief at Lucile Packard Children’s Hospital Stanford and the Arline and Pete Harman Professor and Chair of Pediatrics at Stanford University School of Medicine. She is now leading child and maternal health research into a truly exciting era.

It’s fitting, given that Lucile Packard Children’s Hospital was also the creation of a visionary woman. In 1987, Lucile Salter Packard and her husband, David Packard, made the founding gift to build our hospital. “My dream,” Mrs. Packard said then, “is that 50 years from now, the childhood diseases that cause so much anguish will have disappeared.” 

In the 30 years since, we have made tremendous progress toward fulfilling that dream. Children are surviving once deadly leukemias. Babies born with congenital heart defects and cystic fibrosis are living into adulthood. And diseases such as polio and tuberculosis, which were once the daily business of pediatricians, have largely become history.

Leonard saw this progress up close in her work as a pediatric nephrologist. Many years ago, she took care of a little boy—the same age as her own son—who had been born with a very rare genetic kidney disease. The boy struggled constantly against life-threatening episodes of high blood pressure, kidney failure, anemia, heart failure, and other complications. 

“I admitted him to the hospital 14 times,” Leonard says. “What’s remarkable is that today, the gene that causes his disease has been identified, and a cure is available. Given how devastating this disease used to be, it seems almost miraculous that we now have a cure.” 

She is determined to do the same for the many children who still suffer enormous challenges. Today, we can still do almost nothing for children with certain deadly brain tumors. Prematurity remains the number one cause of death in children under age 5 worldwide. And many mystery diseases remain incurable. 

In her own research in bone health, Leonard has seen young adults who survived previously incurable diseases, but who now have osteoporosis that normally would only occur in elderly adults. “Now that I am physician-in-chief, the breadth of death and suffering across our patients is much more on my mind,” Leonard says. “It motivates me every day to support our passionate physician-scientists and clinicians, who are working to develop and deliver new cures as quickly as possible.” 

Leonard is in the right place at the right time. Today, for the first time ever, the science has advanced to a point where we are within reach of fulfilling Lucile’s dream, in very tangible ways that were unimaginable just a decade ago. New techniques like stem cell and gene therapy are moving forward with unprecedented momentum, bringing the potential to cure hundreds of diseases affecting millions of children worldwide. 

“We can now imagine a future,” says Leonard, “when we can predict and prevent devastating birth defects and childhood illnesses before they occur. And when illnesses strike, we will not just treat them, but cure them definitively—keeping children and mothers healthy not just for today, but for their entire life.” 

It’s a worthy goal, to be sure, but what will it take to achieve it? All the scientific discovery in the world is of no use if there’s no way to trans¬late those insights from the lab into real-life treatments and cures for patients. That’s where Leonard, and your support, comes in. 

Building the Engine for Discovery 

At the Children’s Hospital of Philadelphia, where she spent 25 years before arriving at Stanford in 2014, Leonard directed the Office of Clinical and Translational Research, which provided the resources, environment, operations, and training to support and promote the clinical and translational research of more than 300 principal investigators. Here at Stanford University, where Leonard also serves as director of the Child Health Research Institute, she aims to build similar economies of scale. Stanford’s faculty and schools are already known for winning Nobel Prizes and topping best-of lists in medicine, biosciences, and engineering. The next step is to marshal all that scientific genius to solve the health problems of expectant mothers and children. 

Leonard’s focus is on building the infrastructure and capacity needed to translate research into cures. It may not be the headline-making stuff of science-fiction-come-true, but she is diligently working behind the scenes to mentor and equip a generation of disease-fighting researchers. 

You know that feeling when you’re driving a great car? You get in, and everything just works. It gets you from Point A to Point B so seamlessly that you don’t even have to think about what’s happening under the hood. 

That’s what Leonard aims to build at Stanford for child health and maternal research: a better engine for discovery that hums like a well-oiled machine, functioning in a systematic and efficient way. “What most excites me right now,” she says, “is seeing the spark of excitement for research in our junior trainees. I want to do everything I can to support them to reach their full potential.” Her goal is to allow scientists to focus on the research rather than having to piece together a new engine every time. 

That stuff under the hood, which will accelerate the discovery of new cures, includes support for recruiting clinical trials participants who fit strict criteria for each protocol; budgeting and reporting to meet the requirements of funders and regulatory agencies; biostatistics and data management; oversight to enhance safety for patients; and training for the next generation of qualified investigators. 

It’s a massive, admittedly unglamorous, and absolutely essential foundation for research that will not only shape the future of pediatrics, but potentially transform the lives of millions of children. 

Success is not inevitable, however. Government funding for medical research has been declining for decades, and it does not cover the costs of the basic infrastructure needed for studies to be completed. In the United States, philanthropy is now the single most important source of funding for fundamental, early-stage research and clinical trials. Without it, research will sputter along, and few if any true cures will ever reach the children who need them. 

“We are on the cusp of major breakthroughs in many life-threatening diseases in children,” she says. “Lack of funds slows down the engines that move these discoveries into clinical care. 

“But with philanthropic support,” Leonard adds, “we can get there—faster.”

This article first appeared in the Spring 2017 issue of Lucile Packard Children's News.

Learn more about Mary Leonard's research:
Bad for the bone: The toll of childhood chronic disease at supportLPCH.org/bone

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