CAC2 Childhood Cancer Community News Digest (October 23-29)

CAC2 Supporting Member Day One Biopharmaceuticals today announced that the U.S. Food and Drug Administration (FDA) accepted its New Drug Application (NDA) for tovorafenib as a monotherapy in relapsed or progressive pediatric low-grade glioma (pLGG).

The frequency of cellular therapy trial activations enrolling child/AYA patients with cancer in the U.S. has increased over time. Most studies were phase 1 or 2, single institution-only, and not industry-supported. Future opportunities for cell therapy for pediatric cancer should include multi-institutional approaches.

An externally controlled analyses shows a relapse risk reduction in patients with high risk neuroblastoma treated with postimmunotherapy DFMO.

Study finds survivors of cutaneous melanoma, especially those diagnosed at a young age, are at an increased risk for secondary primary cancers.

ChatGPT shows promise in predicting cancer predisposition genes from clinical notes and could be a promising tool for pediatric oncologists.

The miraculous life-saving power of a bed: Pediatric cancer in Uganda.

The focus of the next stage of ALL therapy for AYA should not only involve novel treatment approaches but also standardization and optimization of supportive care measures, psychosocial support, adherence interventions, oncofertility treatment, and survivorship care.

NPR feature story:  The hospital ran out of her child’s cancer drug. Now she’s fighting to end shortages.

Upcoming Webinars, Online Opportunities, and Meetings:

Join the Ross K. MacNeill Foundation at their 2023 Orange Tie Gala for pediatric brain cancer research, November 4, 6:30-11:00pm CT.  Learn more and register.

Triage Cancer will offer a webinar on Cancer Caregivers on November 15 at 11:30 ET about the rights of caregivers at work, how to replace lost wages while acting as a caregiver, and practical tips to support caregivers.  Learn more and register.

Take Action:

A Draft Scientific Integrity Policy of the National Institutes of Health was released for public comment. The draft policy articulates the procedures and processes in place at NIH that help maintain rigorous scientific integrity practices. Additionally, the draft policy proposes several new functions to further enhance scientific integrity at NIH and throughout the biomedical research enterprise.  Comments will be accepted until November 9, 2023.

Phase 1/2 trial which aims to determine the safety and feasibility of anti-CD33 chimeric antigen receptor (CAR) expressing T cells (CD33CART) in children and adolescents/young adults (AYAs) with relapsed/refractory acute myeloid leukemia (AML). The trial will be done in two phases: Phase 1 will determine the maximum tolerated dose of CD33CART cells using a 3+3 trial design. Phase 2 is an expansion phase designed to evaluate the rate of response to CD33CART.

To help spread the word about the Childhood Cancer Data Initiative (CCDI), the National Cancer Institute has created a communications toolkit with a CCDI overview flyer, a one-pager with frequently asked questions about the CCDI Data Ecosystem, and sample social media posts and graphics.  Access the toolkit, and feel free to reference, copy, and use its content for inspiration.