Ensuring vital access to DFMO for Children with Neuroblastoma

DFMO (difluoromethylornithine) is an experimental drug that has shown promise in treating neuroblastoma, a childhood cancer. It functions by inhibiting the enzyme ornithine decarboxylase, which plays a crucial role in cell growth. By blocking this enzyme, DFMO may help slow down or stop the proliferation of cancer cells.

Neuroblastoma Australia is an organization dedicated to improving outcomes for children with neuroblastoma. They support research initiatives, including studies involving DFMO, to explore its efficacy and potential as a treatment option. Their efforts focus on raising awareness, funding research, and providing resources for families affected by the disease.

Together, DFMO and Neuroblastoma Australia represent a hopeful intersection of innovative treatment research and dedicated advocacy, aiming to enhance survival rates and quality of life for children battling this challenging cancer.

“It was great to see the news that Norgine has opened an Extended Access Program for the groundbreaking neuroblastoma drug, DFMO. This decision follows Minister for Health Mark Butler’s creation of a landmark bridging arrangement to secure immediate access in July 2024 for the drug whilst it is going through standard regulatory approval.

This was a request Neuroblastoma Australia campaigned for over a number of months this year with support from many families and politicians. Since this announcement, numerous children prescribed DFMO in Australia have been able to access this vital drug for free, without having to undertake risky international travel and fundraise large amounts of money.

On behalf of the families we represent, Neuroblastoma Australia would like to thank everyone who campaigned hard to secure affordable, immediate and equitable access to this life-saving drug for children fighting high-risk neuroblastoma.

Along with Health Minister Mark Butler, we would like to thank Shadow Minister for Health Anne Ruston and the numerous current and former parliamentarians from Labor, the Coalition, the Greens and Independents who took up the cause of these brave children.

Neuroblastoma Australia hopes that the example of early access to DFMO shows the future potential for expedited access of internationally approved life-saving drugs here in Australia. This is particularly crucial for Australians fighting rare cancers and diseases where alternative treatment options are limited.”

Ensuring vital access to DFMO for Children with Neuroblastoma

For more information about neuroblastoma, click here.

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Ensuring vital access to DFMO for Children with Neuroblastoma