From Patient to Provider: Impacting National Policy

Recently selected as a member of the National Advisory Council on Blood Stem Cell Transplantation, Brandon Nuechterlein, PA-C, hopes to enhance national policy for pediatric patients. As part of the advisory council, Nuechterlein will advise the U.S. Secretary of Health and Human Services on advancements in transplant research for kids and families facing a diagnosis he is all too familiar with — leukemia.

The role of the council is to provide expert, impartial analysis and recommendations on the latest scientific developments in blood stem cell transplantation and donation. They may also evaluate current research on adult stem cells and birthing tissues to develop new types of therapies for patients, with the intent of possibly including such therapies in consideration of a national level.

Nuechterlein’s unique experience as a physician assistant and a former patient will help inform his work on the council. In 1998, he was a 15-year-old living in Thailand, suffering from unexplained back and abdominal pain, along with intermittent fevers. After weeks of uncertainty, a series of tests revealed what his family had feared: he had leukemia — both acute lymphoblastic and acute myeloid, two of the most aggressive forms of the disease.

“In the 90s, cancer in Thailand was a death sentence,” Nuechterlein says. 

After a long and painful flight to the U.S., Nuechterlein was rushed to Children’s Hospital Colorado, where he underwent emergency surgery and started chemotherapy. Although he achieved remission, Nuechterlein relapsed just three months later. A bone marrow match was impossible to find due to his Thai and German heritage. Fortunately, Children’s Colorado had recently opened a clinical trial using expanded umbilical cord blood — an approach rarely used anywhere in the world at this time. Our experts located a single partially matched cord blood unit, cloned it to expand the number of cells and performed the transplant with total body irradiation. It worked.

Nuechterlein is 26 years post-transplant, with minimal long-term side effects. He now works at the very hospital that saved his life.

“After my experience at Children’s Colorado, I knew what an amazing organization it was," he says. “It really felt like coming home, and I never thought about working anywhere else.”

A career in blood stem cell transplantation

Nuechterlein has spent the past 16 years working at Children’s Colorado’s Center for Cancer and Blood Disorders. For a decade, with his own journey as a guide, he has helped run the Helping Oncology Patients Excel (HOPE) Cancer Survivorship Program and performed over 1,000 transplants.

The HOPE program assists pediatric cancer survivors with side effects from cancer treatment, cellular therapy and bone marrow transplants. Although such treatments are highly effective in treating cancer, side effects such as heart and lung complications, hormone disorders, secondary cancer, infertility, learning challenges, school difficulties and treatment-related fears and worries can appear after some time. Through the HOPE program, Nuechterlein works to minimize these late effects.

“Cancer and genetic diseases that we treat are terrifying because, for most families, they’ve had a perfectly healthy kid, and then suddenly, they must come here for treatment. And so having lived through it, I think it gives me an instant connection with the families, and it gives them some hope,” Nuechterlein says.

His dual identity as a former patient and current physician assistant shapes every part of his day-to-day responsibilities, especially his focus on reducing the toxicities of treatment and expanding access to care. From minimizing late-term effects to accelerating access to all pediatric patients, Nuechterlein’s work bridges clinical care with the reality of children’s lives.

Nuechterlein’s key goal is shaped around one question: “We’re curing more kids, but what happens after they’re cured?”

Nuechterlein has been on the front lines of several transformative advances in transplantation, such as haploidentical (half-matched) donor transplants using parents or siblings, new approaches to gene therapy for non-malignant conditions and efforts to reduce or eliminate chemotherapy through new immunotherapeutics, such as CAR-T cell therapy, and new monoclonal antibody treatments, such as blinatumomab.

These innovations have expanded transplant eligibility for children who previously would have been turned away — particularly those with genetic conditions or limited donor options due to factors such as ethnicity.

National appointment to impact blood stem cell transplantation policy

As one of only 15 experts across the country appointed to advise the U.S. Secretary of Health and Human Services, Nuechterlein’s unique experience can now impact hematopoietic stem cell transplant and immunotherapies on a national level.

One of Nuechterlein’s key advocacy efforts on the national advisory council is promoting cord blood transplantation. Unlike bone marrow donors, cord blood units are collected at birth, banked indefinitely and don’t require a living donor at transplant time. Cord blood is also immunologically flexible, allowing for more mismatches while maintaining successful outcomes, meaning that it can take 50% fewer donations of cord blood units to treat the same number of patients. This scale suggests how efficient cord blood units can be for the pediatric leukemia population.

“There’s only so many kids I can see and help in one day. But being on a council like this allows me to impact health for thousands and even tens of thousands of patients, which is a huge reward,” Nuechterlein says.

Beyond transplant access and new treatments, Nuechterlein is committed to advancing pediatric mental health support. Suicide is now the leading cause of death among pediatric patients, a risk exacerbated by cancer diagnoses or the loss of a sibling. Treatment can be isolating and traumatic — patients may miss school, be away from friends, lose hair or gain weight from steroids. He hopes the council will push for better mental health education and support for young patients facing these challenges.

Nuechterlein also plans to address systemic barriers to care, particularly state-by-state inconsistencies in Medicaid coverage, licensure and reimbursement timelines. When coverage delays continue for months, he has seen kids denied lifesaving treatment. Likewise, he is an advocate for reform in how clinical trials are designed and prioritized. Trials are often run in adults before children, creating decade-long delays in pediatric access to new therapies.

As he takes on his role in the council, Nuechterlein remains grounded in his mission to keep kids at the center of policy decisions, minimize late-term effects of treatment and ensure that no child is denied access to a transplant due to a technicality.

“We can get distracted by the amazing science and the groundbreaking milestones,” he says. “But we have to keep patients, ethics and access at the heart of everything we do.”