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Eugene Hwang

Unexpected heterogeneity in CNS-PNET patients treated as a single entity

Eugene Hwang

“We found that some patients diagnosed with standard tools underwent much more treatment than necessary or intended,” said Eugene Hwang, M.D.

Eugene I. Hwang, M.D., a neuro-oncologist in the Center for Cancer and Blood Disorders, and other researchers at Children’s National Health System, Seattle Children’s Hospital and Research Institute, the Fred Hutchinson Cancer Research Center and the Hopp-Children’s Cancer Center at the NCT Heidelberg recently published the results of a clinical trial focusing on children with histologically diagnosed supratentorial primitive neuroectodermal tumors (CNS-PNET) and pineblastomas (PBLs).

The clinical trial, published online October 17, 2018 in the Journal of Clinical Oncology, included children and adolescents aged 3-22 with these brain cancers who were randomly assigned to receive carboplatin during radiation and/or isotretinoin after the standard intensive therapy (high-dose craniospinal radiation and months of inpatient chemotherapy).  Importantly, because each patient was treated prospectively according to the clinical trial design, the conclusions related to tumor biology were felt to be less affected by varied treatment plans.

“This trial really highlighted the importance of new molecular testing methods in accurately diagnosing some of the brain cancers included in the trial. We found that some patients diagnosed with standard tools underwent much more treatment than necessary or intended.” says Dr. Hwang. “Kids who aren’t receiving the right form of cancer treatment may not get better despite months and months of intensive treatment.”

During this clinical trial, 85 participants with institutionally-diagnosed CNS-PNETs/PBLs were enrolled. Out of the 60 patients with sufficient tissue, 31 were non-pineal in location, 22 of which represented tumors that did not fit in the diagnoses intended for trial inclusion.

The researchers discovered that patient outcomes across each molecularly-diagnosed tumor type were strikingly different. Patients with molecularly-confirmed supratentorial embryonal tumors/PBLs exhibited a five-year event free survival (EFS) and an overall survival rate of 62 percent and 78.5 percent, respectively. However, patients with molecularly-classified high-grade gliomas (HGGs) had a five-year EFS of 5.6 percent and OS of 12 percent, showing no benefit even with the chemotherapy and craniospinal radiation.

Researchers determined that for patients with CNS-PNETs/PBLs, prognosis is considerably better than previously assumed when molecularly-confirmed HGG are removed. Dr. Hwang and co-authors concluded that molecular diagnosis can greatly aid standard pathological diagnostic tools, preventing unnecessary intensive therapy for some patients while enabling more rational treatment for others.

“The findings from our clinical trial have highlighted the immense challenges of histology-based diagnosis for some types of pediatric brain tumors, and the enormous importance this has for children with brain cancer,” Dr. Hwang says. “We hope that ultimately our study will pave the way for molecular profiling to become a standard component of initial diagnosis.”

Yanxin Pei awarded St. Baldrick’s Foundation Research Grant for Childhood Cancer

Yanxin Pei, Ph.D., assistant professor at the Children’s Research Institute, was a recipient of a $100,000 grant that is being named the “Benicio Martinez Fund for Pediatric Cancer Research Grant” from the St. Baldrick’s Foundation, the largest private funder of childhood cancer research grants in the United States.

Dr. Pei studies medulloblastoma – one of the most common malignant brain tumors in children – and has identified a subpopulation of tumor cells that contribute to metastasis after radiotherapy. Her lab is now determining whether targeting these cells can eliminate or prevent the spread of medulloblastoma, thereby improving the outcome of patients with this disease.

In their latest round of funding, the St. Baldrick’s Foundation awarded 76 grants totaling more than $19.1 million to support physician-scientists studying innovative treatment options in the pediatric cancer space. The grants from St. Baldrick’s deliver on its commitment to support the most promising childhood cancer research and work to provide the best solutions possible for kids. The next St. Baldrick’s grant cycle will be announced in fall 2018.

“At St. Baldrick’s, we focus on funding research that has the best potential of giving kids the healthy childhoods they deserve,” said Kathleen Ruddy, CEO of the St. Baldrick’s Foundation.  “I’m proud to say that we have now funded more than a quarter billion dollars since 2005 to support lifesaving childhood cancer research.

Research and Education Week awardees embody the diverse power of innovation

cnmc-research-education-week

“Diversity powers innovation” was brought to life at Children’s National April 16 to 20, 2018, during the eighth annual Research and Education Week. Children’s faculty were honored as President’s Award winners and for exhibiting outstanding mentorship, while more than 360 scientific poster presentations were displayed throughout the Main Atrium.

Two clinical researchers received Mentorship Awards for excellence in fostering the development of junior faculty. Lauren Kenworthy, Ph.D received the award for Translational Science and Murray M. Pollack, M.D., M.B.A., was recognized in the Clinical Science category as part of Children’s National Health System’s Research and Education Week 2018.

Dr. Kenworthy has devoted her career to improving the lives of people on the autism spectrum and was cited by former mentees as an inspirational and tireless counselor. Her mentorship led to promising new lines of research investigating methods for engaging culturally diverse families in autism studies, as well as the impact of dual language exposure on cognition in autism.

Meanwhile, Dr. Pollack was honored for his enduring focus on motivating early-career professionals to investigate outcomes in pediatric critical care, emergency medicine and neonatology. Dr. Pollack is one of the founders of the Collaborative Pediatric Critical Care Research Network. He developed PRISM 1 and 2, which has revolutionized pediatric intensive care by providing a methodology to predict mortality and outcome using standardly collected clinical data. Mentees credit Dr. Pollack with helping them develop critical thinking skills and encouraging them to address creativity and focus in their research agenda.

In addition to the Mentorship and President’s Awards, 34 other Children’s National faculty, residents, interns and research staff were among the winners of Poster Presentation awards. The event is a celebration of the commitment to improving pediatric health in the form of education, research, scholarship and innovation that occurs every day at Children’s National.

Children’s Research Institute (CRI) served as host for the week’s events to showcase the breadth of research and education programs occurring within the entire health system, along with the rich demographic and cultural origins of the teams that make up Children’s National. The lineup of events included scientific poster presentations, as well as a full slate of guest lectures, educational workshops and panel discussions.

“It’s critical that we provide pathways for young people of all backgrounds to pursue careers in science and medicine,” says Vittorio Gallo, Ph.D., Children’s chief research officer and CRI’s scientific director. “In an accelerated global research and health care environment, internationalization of innovation requires an understanding of cultural diversity and inclusion of different mindsets and broader spectrums of perspectives and expertise from a wide range of networks,” Gallo adds.

“Here at Children’s National we want our current and future clinician-researchers to reflect the patients we serve, which is why our emphasis this year was on harnessing diversity and inclusion as tools to power innovation,” says Mark L. Batshaw, M.D., physician-in-chief and chief academic officer of Children’s National.

“Research and Education Week 2018 presented a perfect opportunity to celebrate the work of our diverse research, education and care teams, who have come together to find innovative solutions by working with local, national and international partners. This event highlights the ingenuity and inspiration that our researchers contribute to our mission of healing children,” Dr. Batshaw concludes.

Awards for the best posters were distributed according to the following categories:

  • Basic and translational science
  • Quality and performance improvement
  • Clinical research
  • Community-based research and
  • Education, training and program development.

Each winner illustrated promising advances in the development of new therapies, diagnostics and medical devices.

Diversity powers innovation: Denice Cora-Bramble, M.D., MBA
Diversity powers innovation: Vittorio Gallo, Ph.D.
Diversity powers innovation: Mark L. Batshaw, M.D.