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.

Right now children need YOUR help!

The FDA has 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 will take place on October 4, 2023. This ODAC meeting presents a unique opportunity for families and their clinicians to provide perspectives in letter submissions that will be made available to ODAC members and FDA in advance of the meeting.

Here is where we must 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 will 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!

Writing a simple letter is how we can stand up and fight for our children:

If you are willing to participate, here are some suggestions:

  • Be brief: we recommend you limit you letter to one page.

  • Make it ‘skimmable’: Put your main messages in the first sentence(s) of each paragraph.

    Advisors will have much to read, so make it easy for them to quickly get your viewpoint.

  • Focus: answer the following question – Based on your experience, why is it important for children to have access to DFMO?

Where to Submit Your Letter

Letter Submission to ODAC/FDA

Once you have written your letter, post it by copying it into the "comment" box on their website here: Under Comment Category, select Individual Consumer (or another category as appropriate to your profession).

Thank you, in advance, for sharing your perspective on DFMO. It is so important for all FDA advisors to hear from you.

The deadline for submission is September 20.

Should you have questions, please do not hesitate to reach out to:

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

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