Hematology, Oncology, Neuro-Oncology & Stem Cell Transplantation Updates: Fall/Winter 2021

Lurie Children’s Leads Novel Vaccine Trial for DIPG, DMG

Dr. Ashley Plant-Fox from Lurie Children’s is leading a phase I/II clinical trial for a novel off-the-shelf heat shock protein vaccine, known as rHSC-DIPGVax, for the treatment of diffuse intrinsic pontine glioma (DIPG) and diffuse midline glioma (DMG). Patients with newly diagnosed DIPG or DMG will be able to enroll after receiving radiation therapy and receive this novel vaccine rHSC-DIPGVax in addition to checkpoint blockade (anti PD1 and anti CTLA4 antibodies). Patients may receive treatment up to 1 year. Lurie Children’s will be the lead site with enrolling sites including Dana Farber Cancer Institute, Children’s Hospital Orange County, and Alberta Children’s. Read more

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Research into Nuclear Organization in Rhabdomyosarcoma to Refine Precision Medicine

Kyle MacQuarrie, MD, PhD, from Lurie Children’s recently received two grants – from the Northwestern University Clinical and Translational Sciences Institute and from Hyundai Hope on Wheels – to support his research into nuclear organization in rhabdomyosarcoma. Studying where genes are positioned spatially in the nucleus, and the differences between nuclear structure of tumor cells and normal skeletal muscle, may lead to improved molecular diagnosis and more precise prognosis for patients with rhabdomyosarcoma. It may also pave the way to discovery of a target for treatment.

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Dr. Liem on Panel for ASH 2021 Stem Cell Transplantation in Sickle Cell Disease Guidelines

Dr. Robert Liem from Lurie Children’s served on the panel formed by the American Society of Hematology (ASH) to develop evidence-based guidelines that are intended to support patients, clinicians and health professionals in their decisions about hematopoietic stem cell transplantation (HSCT) for sickle cell disease. The key recommendations in the recently published guidelines include considering HSCT for those with neurologic injury or recurrent acute chest syndrome at an early age and to improve nonmyeloablative regimens. Read more

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Advancing Genetic Research of Pediatric Brain Tumors

Angela Waanders, MD, MPH, Director of Precision Medicine in Oncology at Lurie Children’s, is the Executive Co-Chair of the Children’s Brain Tumor Network (CBTN), a multi-institutional collaboration that recently received support from the National Cancer Institute through the X01 Sequencing & Genotyping Resource Access program. It will allow CBTN to procure molecular characterization for thousands of brain tumor samples, providing an unprecedented level of insight into devastating cancers and paving the way for future therapeutic interventions. This characterization process will prioritize the most aggressive tumor types, supporting research for those cancers for which there is a lack of existing data.

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Speaking Out Against Racism

Lurie Children’s faculty A. Kyle Mack, MD, and Rachel S. Bercovitz, MD, with fellow Hannah Lust, MD, published an Op-Ed in the Scientific American titled Some Medical Examiners Say Sickle Cell Trait Causes Sudden Death -- They're Wrong. The authors maintain that the genetic factor that contributed most to the deaths of 47 Black men in police custody was the color of their skin, not the contents of their red blood cells.

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Innovative Oncodermatology Program for Cancer and Stem Cell Transplant Patients

The Lurie Children's Oncodermatology Program, led by Karina Vivar, MD, provides comprehensive skin care for patients who are undergoing or have undergone cancer therapy and hematopoietic stem cell transplantation. The skin, hair, and nails are uniquely affected by chemotherapy, radiation, stem cell transplantation, and newer targeted and immune checkpoint therapies. The clinic provides specialized assessment and treatment of these cutaneous side effects, as well as skin infections and growths which can occur in cancer patients, cancer survivors and transplant patients. The program also includes dermatological procedures and surgeries and has access to innovative and collaborative research. By running alongside clinics for survivorship, graft versus host disease, and neuro-oncology, the program faculty collaborate with other specialists to provide timely and convenient care for the whole patient.

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First Multidisciplinary Clinic for Opsoclonus Myoclonus Ataxia Syndrome

In January, Lurie Children’s is launching the first multidisciplinary clinic in the country for patients with opsoclonus myoclonus ataxia syndrome (OMAS), which can occur with or without the presence of neuroblastic tumors. OMAS leads to developmental regressions and delays that can have long-standing impacts. These patients require ongoing assessment and monitoring after completion of their treatment. Led by Elizabeth Sokol, MD, the clinic will include oncology, physical therapy and occupational therapy, with plans to include speech therapy as needed. It will provide patients with longitudinal evaluations of their development and help identify and optimize the services these children receive.

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Lurie Children’s Leads New Regional Thalassemia Collaborative

Sherif Badawy, MD, from Lurie Children’s is leading the development of a regional collaborative to improve access to care and services for patients with thalassemia across the Midwest. The new collaborative will work with State and local agencies to identify all newborns in the entire Central region by incorporating strategies for screening and follow-up of thalassemia into systems that are currently tailored to sickle hemoglobinopathies. It also will focus on coordination of relevant thalassemia education and training, as well as patient care coordination, for hematologists, primary care providers and other subspecialists through telementoring, telemedicine or other strategies. In addition, the collaborative will utilize electronically administered patient reported outcome measures to better understand the magnitude of the burden of disease that thalassemia patients experience. This project is funded by a grant from the U.S. Department of Health and Human Services, Health Resources and Services Administration (HRSA), Maternal and Child Health Bureau, Division of Services for Children with Special Health Needs.