CAC2 Childhood Cancer Community News Digest (December 20-January 2)

Assorted news from the last two weeks:

The FDA has granted rare pediatric disease designation to IMX-110 for the treatment of rhabdomyosarcoma, a life-threatening cancer found in children. This designation makes IMX-110 eligible for a fast track designation and a priority review designation in the future, according to a press release by Immix Biopharma, Inc.

FDA-approved TRK inhibitors entrectinib and larotrectinib have demonstrated safety and efficacy in treating a variety of solid tumors in pediatric and adult cancer patients.

The FDA granted orphan drug designation to silmitasertib for treatment of medulloblastoma.

A molecular building block of many animal proteins, the amino acid valine, plays a key role in cancerous growth seen in T cell acute lymphoblastic leukemia, a new study shows.

Upcoming Webinars and Online Opportunities:

VIVA Foundation for Children with Cancer in collaboration with the St. Jude Children’s Research Hospital will host a new webinar series that will focus on childhood cancer survivorship care to educate health care professionals about medical and psychosocial risks associated with childhood cancer and its therapy.  The first episode airs on Friday, January 7 at 3:00ET and will cover Late Effects and Care Planning.  Registration link:

View the December CAC2 All-Member webinar with Nickhill Bhakta, MD, MPH. who discussed why the academic approach of thinking about one childhood cancer with one treatment exposure with one chronic condition is not a realistic way of solving long-term disease burden experienced by survivors.

Please register to attend the 10th ACCELERATE Paediatric Oncology Conference, February 10-11 2022 (EU afternoons, US mornings).  The annual ACCELERATE Conference aims to strengthen international cooperation between academia, parent groups, biopharma companies, regulatory bodies and HTA bodies to improve and speed up the global development of new paediatric oncology drugs.