CC Community News Digest (May 25-31)

Assorted news from the last week:

FDA issues its final guidance on the RACE Act and specifies when sponsors must conduct pediatric studies for new cancer drugs as well as when those requirements can be waived or deferred.

CAC2 Supporting Organization Day One Biopharmaceuticals, Inc. (Nasdaq: DAWN), a clinical-stage biopharmaceutical company dedicated to developing and commercializing targeted therapies for patients of all ages with genetically defined cancers, went public and announced the pricing of its initial public offering of 10,000,000 shares of its common stock.

Findings from an analysis of patients’ cognitive functioning after treatment of specific extracranial solid tumors and lymphomas commonly diagnosed in children showed a variety of cognition and functioning deficits.

Fennec Pharmaceuticals Inc., a specialty pharmaceutical company, today announced the resubmission of its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for PEDMARK™ for the prevention of ototoxicity induced by cisplatin chemotherapy in patients one month to < 18 years of age with localized, non-metastatic, solid tumors.

A new study exploits the characteristic epigenetic signatures of childhood tumors to detect, classify and monitor the disease. The scientists analyzed short fragments of tumor DNA that are circulating in the blood. These “liquid biopsy” analyses exploit the unique epigenetic landscape of bone tumors and do not depend on any genetic alterations, which are rare in childhood cancers.

SpringWorks Therapeutics is currently conducting a clinical trial to study an investigational MEK inhibitor called mirdametinib in patients with Neurofibromatosis Type 1-associated plexiform neurofibromas (NF1-PN).

Upcoming Webinars and Online Opportunities:

Join us for the 2021 CAC2 Annual Summit, a robust virtual meeting to unite CAC2 members and guests to advance our work for the childhood cancer community:  Thursday, June 24th noon – 5:00 pm ET and Friday, June 25th noon – 4:00 pm EDT.  Register here.  Read more about the Summit here.

ACCELERATE will host the 2nd episode of their webinar series Orphan Drug Regulations on June 29 at 11:00 EDT.  It will include topics such as

  • How does the orphan medicines regulation work in EU, US and Japan?
  • How did it impact the field of rare diseases? What are the issues now in Research & Development?
  • The oncology paradox: Why did the current adult-based approach not work for childhood cancers? How can we improve the situation to better target the ‘young patients’ needs?

Both in-person and virtual CureFest registration is now open!  Make your plans for September 25-26.

CAC2 member Mark Levine hosts a podcast called, “Help and Hope Happen Here” (available on Apple Podcasts, Spotify, and Google Podcasts).  Access recent podcasts with CAC2 Members (and visit Help and Hope Happen Here for interviews with other CAC2 members and thought leaders from around the community):

  • Kristen Connor (CURE Childhood Cancer Foundation)
  • Suzanne Gwynne (Ladybug House)
  • Laura Sobiech (Children’s Cancer Research Fund)

CAC2 member Janet Demeter (Jack’s Angels)  hosts Childhood Cancer Talk Radio.  Follow that link to access past episodes.  CAC2 Board Member Mariah Forster Olson (Neuroblastoma Children’s Cancer Society) is the featured guest this week.

Justine Kahn, MD, MS, a pediatric oncologist, and clinical investigator at Columbia University Herbert Irving Comprehensive Cancer Center, discusses the importance of diverse clinical trial participation within the oncology space.

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