The Hope of DFMO

DFMO, or difluoromethylornithine, is an ODC inhibitor which we’ve been funding research into since 2010.

After almost twenty years of dedicated work by Dr. Giselle Sholler and the Beat Childhood Cancer Research Consortium, hundreds of children diagnosed with neuroblastoma have experienced positive outcomes from DFMO, a well-tolerated, oral medication. In fact, data has confirmed that DFMO can reduce the risk of relapse by 50% when used as a maintenance therapy following remission from standard of care therapies.

We have made great strides to have this life-saving drug available to all children.

As a first step to ensuring DFMO is available to every child, everywhere, a New Drug Application was formally submitted to the FDA last year and remains under review.

The FDA moved the direction of the DFMO application to the next stage of approval through ODAC.

The FDA's Oncologic Drugs Advisory Committee (ODAC) review process took take place on October 4, 2023. This ODAC meeting presented a unique opportunity for families and their clinicians to provide perspectives in letter submissions that was available to ODAC members and FDA in advance of the meeting.

Parents, physicians, clinicians, and supporters were given this unique opportunity to show them WHY this drug is so important and HOW it has a substantial impact on the lives of children fighting cancer. Not only will this approval save LIVES of many with neuroblastoma, it will open the door for more effective and less toxic drugs for all types of cancers in children!

Children and their parents should never be told there is nothing more that can be done when there are true drugs available and the restriction of access is simply a FDA approval!

FDA'S ODAC Votes YES to DFMO for Pediatric Neuroblastomas

October 4, 2023 became a historic day. ODAC voted Yes!

The decision marks the first time the FDA has relied on a single externally controlled trial to support a potential approval in oncology. Due to the successful meeting, the approval process is back on track and we hope to have more information to share in the coming weeks as the process moves forward once again.

Targeted Oncology Article

OncLive Article

DFMO PSM Analysis

Thank YOU to all of our donors and supporters. Real progress is being made because of you!

It is with appreciation and pride that we continue to partner together to beat childhood cancer for every child, everywhere.

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