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Donors Fuel Bold Ideas that Transform Care

September 8, 2020

Dr. Christopher Ng had a big idea about how to transform care for children with rare blood diseases. Today, his discoveries are transforming the way we treat blood disorders in kids.

The best and brightest

Five years ago, Dr. Christopher Ng, a pediatric hematologist at Children’s Hospital Colorado, walked into his mentor’s office with a stack of papers in hand. New research had just been published about bleeding disorders, and Dr. Ng had a big idea about how to build on the findings to transform care for children with rare blood diseases.

Dr. Christopher Ng, pediatric hematologist at Children’s Hospital Colorado

“I told him, ‘We have to do this,’” says Dr. Ng. “I believed that this idea was the next logical step to advance the field.”

There was just one problem: Securing adequate funding to do the research. 

For physician-scientists who are just launching their research, it can be extremely difficult to secure the resources needed to pursue outside-the-box ideas. That’s where donors come in.

“Philanthropy allows you to take risks in research; to pursue those game-changing ideas,” says Dr. Ng. “It enables researchers to say, ‘Look, I’m going to try something crazy.  And it might not work, but if it does, it’s going to be transformative.’”

“Philanthropy allows you to take risks in research; to pursue those game-changing ideas,” says Dr. Ng. “It enables researchers to say, ‘Look, I’m going to try something crazy. And it might not work, but if it does, it’s going to be transformative.’”

Armed with a bold hypothesis, Dr. Ng was able to secure a small grant from a foundation, followed by a bigger grant from the Tanabe-Bobrow Family Young Investigator Award Endowed Fund, which supports early- and mid-career researchers at Children’s Colorado.  

That initial funding gave Dr. Ng the time, resources and technical support he needed to pursue his research. Today, his discoveries are transforming the way we treat blood disorders in kids.

A breakthrough discovery

Dr. Ng’s research focuses on the biology of what makes blood clot. He describes clotting as a volume knob: If it’s turned too high or too low because of a medical condition, then it can be deadly.

“Everyone’s body is on a spectrum between bleeding or clotting at any given time,” Dr. Ng explains. “I’m trying to figure out how we can turn that volume knob up or down, and how we can do that in a very targeted way to provide better health for patients.”

But Dr. Ng and other researchers faced a challenge in their search for answers: it was nearly impossible to isolate and study a patient’s endothelial cells, which live inside the blood vessels and play a critical role in clotting. “You can’t take a tissue sample out of someone’s blood vessels, so those cells were really hard to access,” explains Dr. Ng.

Using seed funding from donors, Dr. Ng. spent several years adapting a technique that allows doctors to collect endothelial cells from blood samples and study them on a patient-specific basis. Today, not only can Dr. Ng’s lab collect and study patient-specific endothelial cells, they are using that data to get a very personalized picture of what makes each case unique – and how treatments can be better tailored to individual patients.

Dr. Ng says that could mean a sea change in the standard of care for patients, both here in Colorado and around the world.  

Belle
Belle, hematology patient, with her mom

“Under current protocols, we tend to categorize patients globally. In other words, if you have a disease, you fall into this box and almost everyone in the box gets similar care,” explains Dr. Ng. “I think my research will allow for a richer understanding of individualized patients and their risk factors. That could have significant implications for how we manage these chronic diseases.”

Dr. Ng gives the example of bleeding disorder patients who can require IV infusions multiple times per week starting as early as 1 year of age.

“That’s a heavy burden of care, and it can be a huge drain on quality of life,” he says. “But if we can understand the details of how and why each patient bleeds, then maybe we can better balance the benefits and burdens of the care we recommend.

The next big thing

Since sharing his initial findings, Dr. Ng secured a five-year, $1.25 million grant from the National Institutes of Health (NIH) to continue building on his laboratory’s success. Now he is studying ways to gather even richer patient data – analyzing everything from genetics, to cell activity to protein levels in the blood – to help caregivers to make better, more personalized treatment recommendations. His findings could have implications for a wide range of conditions beyond blood disorders, including adult diseases such as strokes and heart attacks.

“We’re working on the next big thing, and we’re looking ahead at how we can apply this to a multitude of disease categories,” says Dr. Ng.

It’s a perfect example of the exponential progress that philanthropy makes possible.

Dr. Ng is one of many researchers at Children’s Colorado who have turned smaller grants from donors and foundations into multi-million dollar investments from the NIH. In fact, physician-scientists at Children’s Colorado who were awarded philanthropic grants have, in turn, received federal funding dollars averaging five times more than the initial philanthropic investment.  

Together, these up-and-coming researchers are pioneering new treatments and cures to transform pediatric medicine on a global scale – and Dr. Ng says it’s only possible because of generous donors.

“Scientific discovery typically happens in small increments, but sometimes you have to make those big leaps forward,” says Dr. Ng. “And I think that’s what philanthropy enables us to do – pursue big, bold ideas. Not all of the ideas are going to be home runs, but if you don’t at least try, you’ll never see radical change.”

Supporting big ideas

Our physician-scientists continually have new ideas for ways to improve child health. But researchers need data to test new hypotheses. And without the start-up funds to do proof-of-concept research, it’s almost impossible to secure bigger grants for studies that will have a transformative impact on children’s health. Support our team of young investigators in their bold search for new treatments and cures.

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