Advancing Discovery: 2026 Research Impact Report

Breakthroughs in child health rely on stable funding that allow scientists to pursue bold ideas, develop new therapies and bring discoveries into clinical practice. Philanthropic support helps fill gaps in federal funding and accelerates promising research by both seasoned and early-career investigators.

For example, to date in 2026, donors have funded six Pilot Awards, providing investigators with financial support to move their innovative ideas forward. Philanthropy is also fueling two Research and Innovation Scholar Awards (RISAs), which help new investigators — faculty members in the first five years of their careers — apply their research training.

Catalytic investments like these allow researchers to collect the data needed to test their hypotheses, apply for larger grants and then translate their work from theory to lab testing, from clinical trial to FDA approval and, ultimately, to standard of care, helping to launch the careers of promising physician-scientists and advancing science through medical breakthroughs, new clinical innovations, more effective treatments and potential cures for childhood disease.

These philanthropy-funded awards also deliver a strong return on investment: nearly 60% of researchers who received RISAs between 2011 and 2025 went on to secure NIH funding, so, for every $1 invested in a RISA, they received $4.30 in NIH funding, a 4:1 return.

*According to data compiled by the Blue Ridge Institute for Medical Research.

At just three months old, Hannah was diagnosed by the surgical and research team with craniosynostosis, a condition in which the bones of a baby’s skull fuse too early, potentially affecting brain growth and development. Her parents were devastated, but deeply grateful to already be at one of the nation’s leading centers for craniofacial innovation. At the clinic, the family learned about two well-established surgical approaches, one highly invasive and one endoscopic. But the clinical and research team offered a third option: an emerging technique being studied and refined at Children’s Colorado: endoscopic surgery combined with spring-assisted skull expansion.

“They explained the research behind it, the data they were collecting and how this approach could potentially reduce trauma and recovery time,” Julie, Hannah’s mother said. “That sealed it for us.”

Because the procedure is time-sensitive, surgery was scheduled quickly. The results were remarkable: no blood loss, no transfusion and a smooth, rapid recovery. The spring, designed to gently guide Hannah’s skull into a healthier shape, was removed just two weeks later, far ahead of expectations. Hannah’s family also chose to participate in ongoing research, allowing her imaging, outcomes and clinical data to help advance the science behind craniosynostosis treatment.

Thanks in part to transformational philanthropy from the RMRA, Children’s Colorado is home to the leading Rett Clinic for clinical care and research in the region. Founded in 2010 as a collaboration between Children’s Colorado and RMRA, the Rett Clinic was designated a Center of Excellence by the International Rett Syndrome Foundation in 2017, recognizing that it provides coordinated, family-centered, multi-disciplinary care that improves health outcomes, advances Rett syndrome research, expands access to innovative therapies and serves as a trusted resource for the Rett community.

Much of this work is led by Tim Benke, MD, PhD, director of research at the Neurosciences Institute and The Ponzio Family Chair in Pediatric Neurology Research. Recent research includes a gene replacement trial for Rett syndrome, with remarkable results to date.

“One of our first patients with Rett syndrome to be treated with gene therapy recently reached a two-year milestone in the clinical trial,” Dr. Benke explained. “She’s a 9-year-old who hadn’t been able to sit up independently for more than five years. Her mother sent our team a video captured by a bedside camera at their home: while her mother stepped out of the room for a moment, the young woman simply sat up on her own. When her mother returned and asked what she was doing, the girl broke into laughter — a pre-teen delighting in surprising her mom.”

In early 2023, the FDA approved Vyjuvek, a topical form of gene therapy that strengthens the integrity of the epidermis and dermis. Later that year, the topical gel Oleogel-S10, known as Filsuvez, gained FDA approval.

The two treatments are complementary, according to Anna Bruckner, MD, who has spent her career treating EB patients. Ultimately, Dr. Brukner hopes her research will contribute to a disease-modifying treatment that’s not just a patch for the skin.

That progress, from decades with no real treatment options to genuine momentum toward better therapies and the possibility of a cure, recently reached a global audience. Matter of Time, a documentary following musician Eddie Vedder through two sold-out 2023 benefit concerts in Seattle for EB research, features Dr. Feinstein and one of his patients.

“This film brings EB to the world stage,” said Feinstein. “We’ve gone from having no treatments to truly believing that a breakthrough is within reach, thanks to the unyielding strength and hope of our EB community.”

A 2023 HHOW grant is funding Vanessa Ann Fabrizio, MD, MS, a Children’s Colorado bone marrow transplant and cell therapy physician, in her work to improve outcomes for patients treated with CAR-T cell therapy.

Chimeric antigen receptor (CAR) T cell therapy has transformed treatment for pre-B-cell acute lymphoblastic leukemia (pre-B-ALL), a fast-growing blood cancer that resists initial treatment. But despite an encouraging initial response, an estimated 40% to 50% of patients relapse after CAR-T cell therapy. Dr. Fabrizio and her team are working to make treatment last longer and work better by studying the health and type of a patient’s T cells at the moment they’re first collected, before lab engineering begins, to identify which cell types produce the most effective, long-lasting treatment, especially for patients with complex or varied cancers.

A Children’s Colorado patient recently became the 50th person to receive CAR-T cell therapy in the Phase 1 clinical trial Dr.  Fabrizio leads, which is open to pre-B-ALL patients who have relapsed or failed to respond to chemotherapy or bone marrow transplant. That milestone reflects the innovative work of Dr. Fabrizio, her team, and the broader collaboration between the University of Colorado and Children’s Colorado, and it’s just one example of how the research HHOW funds is helping advance care for patients and families.

Now, a newly established endowed chair reflecting Dr. Eilert’s vision is more than a tribute to his groundbreaking leadership; it is a testament to these ideals, and the collective commitment of physicians to the children and families we serve.

Dr. Eilert and his wife, Debra, committed the final funds needed to establish the Dr. Robert Eilert and Dr. Lori Karol Endowed Chair in Musculoskeletal Research. The Eilerts added Lori Karol, MD, to the chair’s name to honor the renowned pediatric orthopedic leader and former division chief, who had long advocated for the creation of an orthopedic research endowment and passed away in 2022.

“Research is what allows us to keep asking better questions and improve outcomes on behalf of our patients,” said David Howell, PhD, ATC, director of clinical research for the University of Colorado Anschutz Department of Orthopedics. “This chair doesn’t just honor Dr. Eilert’s and Dr. Karol’s legacies. It guarantees that the next generation of musculoskeletal researchers will have the support to pursue the kind of bold, patient-centered work that defined their careers. That’s a gift that will benefit children for decades to come.”