New Program Ensures Safety and Quality in Novel Therapeutics and Precision Medicine

Using precision medicine to treat pediatric cancer

For some kids, cancer treatment doesn’t just look like pills, radiation and chemo. Instead, it’s removing immune T cells from their bodies, training those cells to target specific antigens on tumor cells and then infusing those cells back into the body. Treatments like these, which use genetics and modified cells, are being used more frequently in pediatrics, and across disciplines too. To undertake and deliver these advanced treatments to more children with a greater range of conditions the standard procedures and processes must be adapted.

“These are very complex new treatments that are coming online, and they're different than traditional drug trials and pharmaceuticals that are coming to market,” says David Brumbaugh, MD, Chief Medical Officer at Children’s Hospital Colorado. “All our mechanisms for onboarding new treatments, for our safety programs associated with delivering those new treatments and researching those treatments were really designed around traditional pharmacologic treatments like drugs. But it's different now because these are personalized.”

Now, a child’s care team has many more considerations to make.

“How do we make sure that we're handling cells or drugs correctly at all points? That we're appropriately transporting cells out to the lab that edits out the defective gene, and that the cells match that patient's cells that were sampled?” Dr. Brumbaugh explains. “All of these things put a lot of new challenges on our system. We realized that we really needed to create a new structure to support onboarding and safe delivery of these novel treatments that are very different than the drug treatments of yesterday.”

Building a cross-functional team 

To do this, Children’s Colorado created a cross-functional team to build a new program called ExACT, or Excellence in Advanced Complex Therapeutics. This hospital-wide initiative is led by team members from the hospital’s Precision Medicine Institute and Colorado Child Health Research Institute in partnership with experts in many different disciplines. Its goal is to standardize the process by which novel therapeutics are used in treating kids with all kinds of conditions and needs.

Coordination of complex treatments and systems

Coordinating delivery of these complex novel therapeutics is no small feat, according to Jenae Neiman, RN, MBA, VP of Research Operations and Meg Macy, MD, Medical Director of the Colorado Child Health Research Institute. They are among the leaders bringing ExACT to life and are devoted to leveraging the team’s communal learnings to safely and effectively manage the current and future use of novel therapies. 

“Our goal is to design systems that are highly reliable so that as volumes increase, we have systems and processes in place to meet the demands.”

- JENAE NEIMAN, RN, MBA

Focus on safety and quality

With these systems in place, the team can continue its focus on safety and quality — two factors that have long been at the heart of Children’s Colorado’s novel therapeutics work. But because the work must continue even as these systems are being built, Dr. Macy describes ExACT’s approach as “building the plane as we are flying it — maintaining the already functioning systems and processes while creating new ones where needed.” The team worked to identify the most critical processes first and has designated taskforces to get them up and running by early 2026.

That has resulted in three working groups: one on ordering products, such as skin grafts, cell therapies or n-of-1 drugs; another on onboarding new treatments, including preparing, intaking patients and standardizing rollout; and finally, one aimed at ensuring quality and compliance in delivery of these novel therapeutics. These groups are governed by an overarching, multidisciplinary steering committee that keeps an eye on the big picture and will help shape the future direction of this work. Among them is Scott Demarest, MD, Chief Precision Medicine Officer.

“There's a tidal wave coming of these types of treatments,” Dr. Demarest says. “These are new therapies for us organizationally, and we want to be the go-to place because we have built the infrastructure to deliver them efficiently, in cost-effective manners, sustainably, and above all else, safely for patients.”