$1 Million
Hannah's Hope Fund for GAN April 15, 2013
In This Issue: Golf Tournament ValleyCats Vote Scientific Update   In the News
Heroes for Hope Golf Tournament, Celebration Dinner, 
International Food & Wine Pairing

Non-Golfers are invited too!  
Come and enjoy dinner, the wines of Italy, Spain & France and a silent auction.
And! We will be making an exciting announcement about our Heroes for Hope campaign!

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Click here for full details and registration information.

valleycats logoWe Won!  
We offer our deep gratitude to Greg Floyd of CBS 6, the ValleyCats and all of our amazing HHFans who diligently voted in the ValleyCats Bobblehead contest.  Here is the text from the ValleyCats web site:

The winner of the ValleyCats 2013 Media Bobblehead Bracket Buster Tournament is CBS 6 News Anchor Greg Floyd. Floyd is representing Hannah's Hope Fund, who will receive a $1,500 cash donation and will be a partial game sponsor on Monday, August 19 at "The Joe." Greg Floyd will be immortalized into a bobblehead as a giveaway to the first 1,500 fans through the the gates on August 19. Hannah will be incorporated into the design of the bobblehead.

Floyd defeated News Channel 13 Sports Director Rodger Wyland. His charity, the Thomas Patrick Morrison Foundation, will receive a $1,000 cash donation. All of the charities in the contest will be recognized on August 19.


GAN Gene

Scientific Update

We are fortunate that the NIH Clinical Investigator, Dr. Carsten Bonneman, has agreed to take on our GAN gene delivery clinical trial.  He will be reviewing the Natural History data and will clinically assess a few of the children with GAN before he begins the initial trial.
The final safety studies are underway (data will be in by mid-June). Then, we will incorporate the results into our Investigational New Drug (IND) application for the FDA.  We will go in front of the Recombinant DNA advisory council in June, and then we need to obtain Institutional Review Board (IRB) approval to begin a trial.  That is three regulatory hurdles to navigate so please keep sending your positive energy and prayers our way.
Dr. Bonneman indicated that the Phase 1 trial cannot start until he knows exactly how we will measure the efficacy of GAN gene therapy.  We need to be able to determine:
1.      Did GAN gene delivery stop progression?
2.      Did it slow progression?
3.      Did it have no impact?
4.      Did it make them sicker?
5.      Did it restore function to axons not dead yet (did they improve)? 
Emory University is performing analysis to predict if GAN patients are likely to launch an immune response. Lori and Dr. Gray (Chapel Hill) met with Doctors Kurtzberg and Markert at Duke, who are helping to solve the problem of immune response.  For those patients for whom immune response will be an issue, there are options available including, 
  • A bone marrow transplant to induce tolerance
  • A thymus transplant: the thymus is the primary immune system organ. A transplant would essentially provide a new immune system from a donor who has functional gigaxonin.  
  • Immunosuppression drugs    
Lori coordinated a meeting with the leaders in the field of gene therapy and enzyme replacement therapy to come together and solve this issue of immune tolerance for the entire field.  That meeting will be in Salk Lake on May 15th.  

In the News

polar plunge

The GAN Warriors Polar Plunge Raised Over $35,000! 

The GAN Warriors was formed in honor of Jason and Charee Spencer’s three young boys, 6-year-old Levi and his 4-year-old twin brothers, Aaron and Alan, who have all been diagnosed with GAN. The money the GAN Warriors raise goes to HHF.

There were 133 people willing to brave Montana's freezing temperatures to help!  WOW!

Click here for more pictures and news about the Polar Plunge and the GAN Warriors.

Hannah's Hope is Funding Scientific Research and the Results are Being Published

In February, a team of Belgium researchers was published in Human Gene Therapy.  Click here to read the abstract.

In January, an article was published in the Daily NorthWestern of NorthWestern University in Chicago about the GAN research being done by the Department of Cell and Molecular Biology.  Click here to read the article.

In December, an International team presented a poster on GAN at the American Society for Cell Biology.  Click here to read more.

Hannah's Hope Received Rare Genomics Institute Award

The Rare Genomics Institute awarded Hannah's Hope $25,000 to be used for Bioinformatics Consulting. This will help HHF with storing, retrieving, organizing and analyzing biological raw data which will be instrumental in our development processes.

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