CAC2 Childhood Cancer Community News Digest (June 20-26)

Assorted News from Last Week:

Kazia Therapeutics Limited, an oncology-focused drug development company, is pleased to announce that the United States Food and Drug Administration (FDA) has awarded Orphan Drug Designation (ODD) to Kazia’s paxalisib for the treatment of atypical rhabdoid / teratoid tumors (AT/RT), a rare and highly-aggressive childhood brain cancer.

Bridge therapy ahead of autologous stem cell transplantation (ASCT) consolidation may benefit certain high-risk neuroblastoma patients with residual disease following induction regimens, according to a multicenter retrospective study.

Novartis announced the FDA has granted accelerated approval to the BRAF inhibitor dabrafenib (Tafinlar) combined with the MEK inhibitor trametinib (Mekinist) for the treatment of adults and children with unresectable or metastatic solid tumors with a BRAFV600E mutation, who have progressed following prior treatment and have no adequate alternative treatment options.

Upcoming Webinars, Online Opportunities, and Meetings:

Join advocates from around the country for the 2022 Rally for Medical Research! The Hill Day will take place on Wednesday, September 14, with advocates meeting their congressional representatives and staff. The evening before, on September 13, there will be a  reception to celebrate medical research with all partners and participants.

Children’s Cancer Cause is hosting a virtual externally-led Patient-Focused Drug Development (PFDD) meeting on Reducing Cardiac Late Effects in Pediatric Cancer Survivors, on September 15, 2022. The conference is an opportunity for survivors and caregivers to educate representatives of the FDA, academic researchers, and pharmaceutical companies about their personal experiences managing the impact of cardiac late effects on daily living and long-term health.  Registration link.

Take Action:

Children’s Cancer Cause has established an annual award, the Survivorship Champion’s Prize, to be presented to a group, program or institution making significant advances in programs and services to provide life-long health maintenance for survivors of pediatric cancers.  These awards recognize the importance of the unique challenges associated with the post-treatment services for survivors, especially as they transition to non-oncology adolescent and/or adult health care services.  This year, there are awards in two categories (click here for more information and to apply):

• Champion’s Prize Award: $10,000
• Recognition Awards for 1) Program Impact; 2) Collaboration; and 3) Scalability: $5,000 each

During June, on Childhood Cancer International social media wants to share stories, testimonials and activities regarding childhood cancer survivorship. If you have content that would like to share with us please send it to .