Poised for Progress
Harnessing organoid technology to personalize care
At 5 years old, Quinn is a happy bundle of energy. As he waits for his check-up at our Division of Gastroenterology, Hepatology and Nutrition, he jumps back and forth between sitting on his mom Emily’s lap and bouncing in the chair next to her. His body is small, but his smile is big enough to light up the room.
“He just learned to ride his bike without training wheels,” Emily says, beaming with pride. “He loves to play with his siblings and take golf lessons—he’s just a typical kid.”
Watching him run around the playground, you’d never know that most of Quinn’s digestive system doesn’t function, or that he has to be hooked up to tubes for 13 hours every night to get the nutrition he needs to live.
Quinn was born with Hirschsprung disease, a digestive disorder where nerve cells are missing from a segment of the bowel. Without these cells, food can’t pass through the intestines.
Unfortunately, Quinn has the most severe form. And while multiple surgeries and feeding tubes help him live, there’s no cure for Hirschsprung.
“Quinn’s come a long way,” Emily says. “We’re making the best of what we have, but we hope and pray for future medical advancements. Cincinnati Children’s is doing great things, and I have faith in their research.”
Pioneering Innovation Inspires New Hope
Research innovation is just one of the reasons Cincinnati Children’s is consistently ranked among the top three hospitals in the nation.
Our scientists are an essential component of our care team, committed to discovering the most effective treatments so that children feel healthy and have the best quality of life. That’s how we measure success.
One of our latest achievements is the ability to generate miniature human organs—known as organoids—from a patient’s very own cells. This technology helps us better understand disease, test new medications and personalize care.
A recent breakthrough could revolutionize how we treat chronic digestive disorders like Crohn’s disease, colon cancer, irritable bowel syndrome and Hirschsprung disease—and it could be a life-changing development for kids like Quinn.
Our scientists are the first to succeed in using organoid technology to grow a human intestine with a functioning nervous system.
“Disorders affecting the GI tract are common,” says James Wells, PhD, chief scientific officer of our Center for Stem Cell and Organoid Medicine (CuSTOM). “In the past, we couldn’t study these diseases in humans. But with organoids, we’ve discovered the causes of diseases, like Hirschsprung, and are moving toward generating healthy tissue for transplantation-based therapies.”
This is big news. Now that we have a way to study organoids grown from a patient’s own cells, we can find new answers about a child’s specific condition, develop novel treatments and, eventually, uncover new cures.
For Quinn and his family, this discovery means hope.
Searching for a Better Outcome
From the time he was born, Quinn has relied on medical interventions not only to give him the nutrients he needs to thrive, but also to help him digest them.
It’s been a long journey of tube feedings and medical appointments. When he was just 6 days old, our surgical team gave Quinn an ostomy—a procedure that allows bodily waste to pass through an opening in his abdomen. A month later, he had a second surgery to insert a central line, which delivers a formula of essential nutrients through a vein in his chest.
It was an overwhelming process for Emily and Quinn’s dad, Kelly. “We feared the worst for Quinn when he was born,” Kelly recalls. “But his care team assured us he would get the nutrition he needed.”
With the help of our gastrointestinal team, he continued to gain weight and meet developmental milestones, and tube feeding became less frequent. Now in preschool, Quinn can go 11 hours tube free, every day. This gives him the freedom to do the things he loves. At night he receives nutritional formula through a central line, but that isn’t without its risks.
“Cincinnati Children’s saved his life, but these interventions have side effects, especially on the organs,” Emily explains as Quinn cuddles up in her lap. “His gallbladder isn’t functioning, and he’s also at risk for liver complications.”
That’s why our researchers won’t rest until they can improve outcomes for Quinn and kids like him all over the world.
CuSTOMizing Cures
Our scientists are on the forefront of organoid innovation. We’ve successfully bioengineered the small intestine, stomach, mini livers, and now, the colon.
“Mini organs can identify the unique characteristics of a disease in a single person,” says Michael Helmrath, MD, the clinical translation director for CuSTOM. “This is truly personalized medicine.”
Our goal is to generate genetically matched, healthy tissue that can be used to replace diseased or damaged organ tissue. Because it will be from the patient’s own cells, the risk of rejection is nonexistent.
Emily is optimistic that this vital work will lead to safer, more effective treatments—allowing her son to live his life to the fullest. And with the help of philanthropic partners, we’ll be able to get there faster.
Donors Spark New Possibilities
Philanthropy plays a key role in helping us advance bold research, and it was a critical component in our work with organoids. Generous donors, like the Farmer Family Foundation, are helping us take this life-changing work to the next level.
The Farmer Foundation helped us launch CuSTOM with a pilot grant in 2016. Recently, they pledged an additional $5 million to advance the pioneering research the center is doing.
The gift is unique not only in its generosity, but because it’s a challenge grant. This means the foundation is inviting others—in our community and beyond—to support this innovative research.
“The work being done at Cincinnati Children’s has the potential to completely transform care for children needing organ transplants,” says Amy Joseph, trustee for the Farmer Family Foundation. “We want to encourage others to get involved in speeding up this discovery—that’s why we made our gift a challenge grant. Working together, we can benefit kids sooner.”
This regenerative technology can potentially save lives—not only for children, but adults as well. Building on the accomplishments we’ve made so far, the Farmer Family Foundation’s gift enables us to scale this work, improving outcomes sooner for those suffering with Parkinson’s disease, infectious diseases, adult cancers and more.
Keeping Hope Alive
Emily has faith in the work we’re doing, and she does whatever she can to help. Along with caring for Quinn and teaching at his preschool, she also works with an advocacy group that offers support and education to families seen in our Intestinal Care Center.
“I really think Cincinnati Children’s is going to change the world with these organoids,” Emily says. “I have to keep hope alive for my son’s future.”
For more information on how you can support CuSTOM, please contact James Cleetus at 513-636-1166 or james.cleetus@cchmc.org.
