The chief of pediatric hematology, oncology, and stem cell transplantation at Stanford Medicine Children's Health says new ideas in immunotherapy, stem cell transplants, gene therapy, and gene editing are helping care providers do things they've never done before.
Innovation in pediatric healthcare often focuses on the tiniest of factors affecting the tiniest of patients. And Tanja Gruber, MD, PhD, says research around the makeup and functions of the gene are enabling care providers to get a better idea of how to tackle cancer.
HealthLeaders recently sat down—virtually—with Gruber, chief of pediatric hematology, oncology, and stem cell transplantation at Stanford Medicine Children's Health to discuss innovative ideas like immunotherapy, stem cell transplants, gene therapy, and gene editing, and how they're helping to improve outcomes for millions of children.
Q: What new technologies or strategies are you now incorporating into care management?
Gruber: This is an exciting area of pediatric oncology. The community is very proactive about new science and technology within the clinical space, and that is opening doors to new care models and treatment plans for children and adolescents. Current areas of innovation specific to pediatrics are in immunotherapy, stem cell transplant, gene therapy, and gene editing.
For example, at Stanford Medicine Children’s Health, we have a large chimeric antigen receptor (CAR) T-cell therapy program, which boosts a child’s own immune system to target specific proteins on cancer cells and fight the disease. This has been used for types of leukemia and lymphoma, and there are currently clinical trials exploring this modality for other types of cancers and tumors.
Tanja Gruber, MD, PhD, chief of pediatric hematology, oncology, and stem cell transplantation, Stanford Medicine Children's Health. Photo courtesy Stanford Medicine Children's Health.
This is important because we believe in multi-modality through a combination of treatments, and multi-disciplinary via specialties across the care spectrum. In pediatrics (and pediatric cancer), it’s not enough to treat the disease itself. We must incorporate other healthcare professionals as a part of a long-term, holistic treatment plan – whether physical therapy or mental health. It’s also worth noting that artificial intelligence (AI) is an emerging topic in the pediatric community. The adult field has led the charge here; however, pediatrics is starting to get more involved.
Q: What are the challenges you deal with in using these new treatments?
Gruber: Location or proximity to specialty care facilities and children not being able to administer certain tests for themselves present a new set of challenges. Immunocompromised children also face a plethora of different challenges from their adult counterparts that require unique treatment plans. We saw this during the pandemic. The pediatric community was incredibly cautious around viruses, and relied on vaccinations to help prevent complications that can happen with infections and infectious diseases.
Another growing area of concern is that earlier detection and treating children with cancers earlier in life will likely result in more strain on the healthcare system as they venture into adolescence and adulthood. Survivors need to be monitored post-treatment, they need frequent follow ups, and they need to be watched closely as they grow. Add onto this the physical, mental, and emotional toll that cancer treatment places on the body at a time when children are growing and vulnerable, and you see the critical importance of holistic, multi-disciplinary care.
Q: Do you feel there is enough healthcare innovation directed toward pediatric care, or is it difficult to find the right technology and/or companies to address what you need?
Gruber: Our community is very proactive about pediatric healthcare innovation from a technology and strategies perspective. However, one area that we must continue to advocate for is drug development. We cannot be left out of that conversation. Currently, the industry is looking into mandates and requirements for certain portions of research and development spend to be targeted to pediatrics. This is – and will continue to be – a game changer.
We’ve also seen impressive responses from patients on clinical trials. The conversation is about education. Enrolling children in clinical trials takes on more complexity – how you communicate with families, how you coordinate with research, how you gather consent, how you administer care, and more. I’ve been really impressed with how the pediatric community has provided innovative care opportunities and world-class treatment through clinical trials.
Q: How have advances in stem cell transplants and gene therapy affected pediatric care?
Gruber: A lot of the work with gene therapy has been geared toward inherited disorders as opposed to cancer, but that is shifting. And it will be critically important in the long run to make sure pediatrics isn’t left out of this conversation.
With stem cell transplants, the positive results are clear. A lot of people might not be aware, but this care has been around for decades and is continuing to evolve. In fact, Stanford Children’s Pediatric Stem Cell Transplantation program was established in the 1980s, and we have transplanted more than 1,000 patients since that time.
Currently, we are identifying ways to evolve stem cell transplantation, including using new antibody-based methods instead of traditional chemotherapy or radiation to prepare patients’ bodies for the transplant. As a part of this, we’re working with our community via the Pediatric Transplantation & Cellular Therapy Consortium and the Blood & Marrow Transplant Clinical Trials Network to advance these innovations.
Q: What new technologies or strategies are on the horizon for pediatric healthcare? What are you looking forward to using?
Gruber: A topic that’s being discussed in the industry is early detection. Typically, pediatric and adolescent patients do not have access to earlier screenings, as childhood cancer is rare. That is changing as research shows genetic mutations that drive certain inherited disorders also increase the risk of childhood cancer.
Another example, which is still in its infancy, is identifying predisposition for pediatric patients. This opens up so much opportunity for care. This can be the result of individual testing, family testing, or other genetic warning signs. It encourages adolescents to take control of their healthcare and puts a focus on providers helping patients make a smooth transition into adult care.
We are having ongoing conversations in pediatrics about when to start screenings and how to educate patients and families. We are fortunate at Stanford Medicine Children’s Health as we have access to that transition with our colleagues from Stanford Health Care. As an industry, this isn’t common, so more work needs to be done.
Q: What has surprised you, good or bad, about how technology is incorporated into pediatric care?
Gruber: A wonderful surprise has been how well patients and families are responding to clinical trials – and how our population continues to advocate for pediatric innovation. Speaking to patients and families about how clinical trials and technology innovations can improve care and deliver better outcomes has been a positive experience.
Similarly, seeing the community rally around these initiatives to make sure all patients, regardless of location, are made aware of and have access to state-of-the-art care has been heartwarming. For me, the fact that the patient has not been lost in technology conversations shows the true desire of our pediatric provider community to do whatever it takes to improve outcomes for sick children.
Q: Has technology allowed you to collaborate more with other healthcare providers on a patient's care? If so, how has this improved care?
Gruber: Collaborate is the perfect word to describe how we’re able to improve care, together, with our network of healthcare providers. In California, pediatric patients have access to clinical trials that are funded through philanthropy. Collaboration allows us to extend the reach of our world-class care centers and provide greater access to care throughout rural areas or underrepresented communities.
The pediatric oncology community and Stanford Medicine Children’s Health are mindful about health equity, and there are efforts to leverage resource-rich hubs to help patients and families overcome travel, housing, treatment concerns, and more when it comes to getting cancer care to underrepresented minorities in outlying communities. With the help of philanthropic efforts and initiatives like the Ronald McDonald House, pediatric patients and families who otherwise would typically not be able to participate in clinical trials are supported. This would not be possible without collaboration.
Q: How do you see your role evolving?
Gruber: Patient-centered care, backed by research and innovation, remains at the heart of our roles in pediatrics. However, in recent years, shifts have taken place that put a greater emphasis on communication. Communication, both between researchers and practitioners, as well as between providers and families. With new technology comes a need for education. But technology for technology’s sake doesn’t help patients in the long run.
Encouraging communication between these groups is crucial to ensure research and innovation is targeted, providers feel technology can be applied in a way that makes sense for the patient, and patients are aware of the care options they have and feel comfortable with what lies ahead. You add in the push for multi-disciplinary treatment and the movement for health equity, and those communication lines become even more critical to advancing pediatric care.
“It’s not enough to treat the disease itself. We must incorporate other healthcare professionals as a part of a long-term, holistic treatment plan – whether physical therapy or mental health.”
— Tanja Gruber, MD, PhD, chief of pediatric hematology, oncology, and stem cell transplantation, Stanford Medicine Children's Health
Eric Wicklund is the associate content manager and senior editor for Innovation, Technology, Telehealth, Supply Chain and Pharma for HealthLeaders.
Through immunotherapy, stem cell transplants, gene therapy, and gene editing, pediatric care teams have the potential to affect the course of cancer and inherited disorders.
Earlier detection is key to affecting outcomes, but other areas of innovation include drug development and integrated care to improve the patient's whole health journey.
Pediatric innovation also involves collaborating with patients and their families, as well as with the community at large, to ensure everyone has access to clinical trials and care.