As we pass the six month milestone for the AAV Project, we wanted to provide the AHC community with a brief update and some clarity about next steps. Since June 2018, Cure AHC, AHC Foundation and Hope for Annabel have been collaborating on a gene therapy effort using Adeno Associated Virus (AAV) as a delivery system for functioning ATP1A3 to compensate for the mutated ATP1A3 associated with AHC. Our collaboration has involved pooling our financial resources and leveraging connections with scientists around the world to work together.
To eventually and hopefully get to a clinical trial with AHC patients, the AAV Project will have several phases with multiple steps in each phase. We are in just the first phase of the AAV Project where we aim to develop a viral vector to deliver extra ATP1A3 in mice to assess whether AHC symptoms improve after treatment.
In Phase One, the
three foundations, along with contributions from AHC organizations in UK
and Ireland, have already funded over $225,000 to complete the
1) Partnered with Dr. Steve Gray at UT-Southwestern who will help us lead the AAV Project – completed October 2018
2) Designed and produced four viral vectors to deliver functioning ATP1A3 – completed November 2018.
3) Injected non-diseased mice using the four viral vectors to deliver functioning ATP1A3 – completed November 2018
4) Assess which vector did the best job in delivering extra ATP1A3 to targeted areas of the mouse brain – started December 2018 and will be done March 2019
The next step will cost approximately $500,000 and is scheduled to start April 1, 2019 at the Jackson Labs. We will be selecting the best performing vector to deliver extra ATP1A3 to mice with AHC, and then perform a variety of behavior experiments to see whether the AHC symptoms show improvement. If the AAV treatment shows significant improvement in the mice with AHC, we would be on our way to the next phase of testing and closer to a clinical trial.
As a result of fundraising efforts, Cure AHC, AHC Foundation and Hope for Annabel have around $250,000-300,000 currently available to fund this next step of the AAV Project. While we are hopeful that continued fundraising will result in filling the gap in the coming weeks, we need every family to keep contributing to this effort.
Families and organizations wanting to support the AAV Project should feel comfortable directing fundraising efforts to one, two or all three of the foundations: Cure AHC, AHC Foundation or Hope for Annabel.
Here is the Cure AHC Fundraising site for the AAV Project, where you can either donate directly or start your own fundraising page easily! bit.ly/CureAHCAAV19. We have MANY ideas to help you quickly raise funds with minimal effort, and bigger ideas for those who want to do more. WE WILL SUCCEED TOGETHER! 🙂 Thank you in advance for helping to keep us “soaring on the wings of HOPE”!