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Sanfilippo gene therapy trial in Australia

The Sanfilippo Children’s Foundation, a research group for a fatal genetic childhood disease, is thrilled to announce the biopharmaceutical company Abeona Therapeutics has been granted regulatory approval to conduct a clinical trial of its gene therapy drug for Sanfilippo Syndrome in Australia.

The trial, which will take place at Adelaide Women’s and Children’s Hospital in 2017, is the first Australian clinical trial of a potential treatment for Sanfilippo. Recruitment of Australian children for the trial is currently underway.

It is thanks to the work of the Sanfilippo Children’s Foundation and donors, including the Dick Smith Foods Foundation, that this clinical trial will now take place on Australian soil.

This is an inspiring story of how everyday Australians, patient groups, researchers and clinicians can work together to accelerate an emerging treatment and overcome traditional challenges posed by clinical drug development.

Megan Donnell, Founder and Executive Director of the Sanfilippo Children’s Foundation – and mother to Isla (8) and Jude (6), two Sydney siblings who tragically both have the catastrophic condition –  says that for Australian families with children battling this devastating neurodegenerative disease, clinical trial participation offers a rare opportunity to access an experimental therapy.

“Children whose parents were once told by doctors that there was no treatment or cure for Sanfilippo and that gene therapy was a generation away. Now there is a small glimmer of hope that Australian children will potentially face a different future than the one they were handed upon diagnosis.”

“We like to refer to the work of our Foundation as ‘people-powered medicine’, as it’s thanks to the incredible generosity of our supporters and donors, such as the Dick Smith Foods Foundation, that we have been able to reach this incredible milestone,” Ms Donnell said.

The Foundation won $50,000 from the Dick Smith Foods Foundation two years ago. The Dick Smith funds were a particular boost to the Foundation’s quest to raise $2.5 million by the end of 2015.

This clinical trial is the first of many projects currently being funded by the Sanfilippo Children’s Foundation. The Foundation recently funded an Australian PhD student in the neglected field of Sanfilippo research, and last year announced funding for a further five research projects, one of which could provide the missing piece of the puzzle.

The Sanfilippo Children’s Foundation still has significant to do if it is to realise its vision: to fund medical research so a cure can be found in time for children battling Sanfilippo today and those born with it tomorrow.


To learn more or make a donation, please visit: www.sanfilippo.org.au

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