“Our hope for our granddaughter is to live a long and wonderful life,” Blanche Goldenberg says. This is why she and her husband, Steven, give to Children’s Hospital Colorado.
At age 2, the Goldenbergs’ granddaughter, Sylvia, was diagnosed with craniopharyngioma, a rare, slow-growing brain tumor. Sylvia had surgery at Children’s Colorado to remove the tumor, but her optic nerve had already been severely impacted. Pediatric neurosurgeon Todd Hankinson, MD, removed as much of the tumor as possible to prevent her vision from worsening.
Not only is craniopharyngioma hard to treat, it’s also extremely difficult to study. The tumors are comprised of both solid and cystic components. Because of how they grow, it’s nearly impossible to culture tumor cells and test how they respond to various treatments. In addition, it’s extremely rare – in the United States, only about 120 children are diagnosed each year. Because of this, it’s a little-researched disease with few treatment options.
But this is changing, thanks to generous donors.
After Sylvia’s operation, Steve and Blanche wanted to support craniopharyngioma research. Although based in Connecticut, the Goldenbergs recognized that some of the most groundbreaking research for the disease was happening at Children’s Colorado.
“Our hope is that our gifts will enable breakthroughs to benefit our granddaughter and many more children. The research will make a difference,” Blanche says.
Six years ago, Dr. Hankinson started a multi-institutional research consortium, Advancing Treatment for Pediatric Craniopharyngioma. Thanks to funding from the Goldenbergs and others, Dr. Hankinson was able to hire a full-time person on the Anschutz Medical Campus dedicated exclusively to craniopharyngioma research. Today, his team is making research advances that are giving kids like Sylvia new hope for a better quality of life.
“We’ve already made exciting discoveries about the biology of these tumors based solely on philanthropic support,” Dr. Hankinson says.
Dr. Hankinson and his team are now working to identify molecular targets that may be treatable within craniopharyngioma tumors. With that information, he hopes to identify existing drugs that might also work to treat these tumors, followed by clinical trials. If any of the drugs work — and several are showing promise — it would be the first-ever therapy to treat the underlying mechanisms of the disease.
“We could completely revolutionize therapies for these kids,” he says.
Until a cure is found, the team at Children’s Colorado will monitor and treat Sylvia’s tumor growth. Her visual impairment does not slow her down. She is learning to snowboard, and she lobbies at the Colorado State Capitol for health-related issues.
“It’s because of the amazing team at Children’s Colorado that Sylvia is a thriving little girl,” Blanche said. “Knowing that the research continues, we continue to have hope that her tumor will be eliminated.”