GAN Warriors made the news!

The Ravalli Republic shared the GAN Warriors story! Below is the link to that article.
 The Parents of GAN are doing all they can to save their children. It's up to them to find the cure.
What an inspiration they are of love and courage for their children.
 Thank you Ravalli Republic for helping us spread the awareness of this deadly condition.
More awareness means a faster cure for these boys and time is not a friend of GAN.
 http://ravallirepublic.com/news/local/article_45a4ded4-a76e-11e1-a05f-0019bb2963f4.html

http://blogs.scientificamerican.com/guest-blog/2012/05/24/the-valley-of-death-looms-for-8-kids-with-a-rare-disease   

The link above is a very interesting article I wanted to share. I copied this section because it puts a lot in perspective for me. These children JUST want a chance to live, to walk and ride bikes. They JUST want to run and play with their friends, to grow up and have families of their own. I want this for them too and I know if you are taking the time to read this, then you want it for them also.  They are so close with this experiment and it can't come soon enough.
At this point every dollar counts, every prayer counts, every positive thought sent counts.
 Because you care, you are their hope!

"Never doubt that a small group of thoughtful, committed citizens can change the world. Indeed, it is the only thing that ever has." - Margaret Mead
Help move the 'Mountain of GAN' Please make a Tax Deductible gift today @
http://hannahshopefund.org

Here it the part of the article I copied:
 

 

Hannah’s Hope has funded researchers at Emory University, Columbia University, the University of North Carolina and elsewhere to carry out the steps preceding the gene transfer experiment. And results have been so promising that the Food and Drug Administration (FDA) gave a tentative go-ahead this April, pending further toxicity studies in animals. Those are underway now, but cost $646,000 – plunging Hannah’s Hope Fund to the breaking point.
Lori estimates other costs:
- $430,200 for a 2-year natural history study for 12 patients to track the course of this ultra-rare disease, so that the researchers can determine exactly what to check to assess success.
- $220,000 to manufacture the viruses and their human gene cargo
- $600,000 for the phase 1, 2-year trial for 8 children
- $450,000 for a small Phase 2 trial to assess efficacy
That’s a total of $2,346,200. Then, hopefully, a biotech or pharmaceutical company will step in for the required larger phase 3 trials that precede FDA approval.
It sounds like a lot. But for comparison’s sake, $2,346,200 equals:
- approximately 1/30th of Johnny Depp’s fee for his next film
- 1/10th of what Alex Rodriguez earns in a year
- 10 speeches for Bill Clinton
- 6 of Kate Middleton’s wedding dress
- 2 years worth of nanny-time for Brad and Angelina’s brood
- Slightly more than Kim Kardashian’s useless $2 million engagement ring
- .07% of the money spent on the Obama and Romney campaigns as of March 2012.
The government isn’t funding the GAN gene therapy trial. So what the tiny GAN community needs, desperately and now, is a Michael J. Fox, Christopher Reeve, or Julia Roberts.

http://blogs.scientificamerican.com/guest-blog/2012/05/24/the-valley-of-death-looms-for-8-kids-with-a-rare-disease/

GAN Warriors awareness night!

Our first "GAN Warriors awareness night" was a huge success. The turn out was wonderful and the help was amazing. Thank you, thank you to all of you who came and who helped make this night so much fun. It certainly was a night to remember.
The slide show of the GAN Warriors was shown and a talk was given about GAN and Hannah's Hope Fund.
We also had a cake making challenge and that was incredible. There were two categories to enter, one being TASTE and the other being LOOKS. The day of the event only 20 people had registered to enter the contest, but there ended up being 64 cakes that showed up!
 Prizes for the winners were a  Kindle Fire, Ipod Touch, MP3 Walkman, camera, greenhouse gift certificate,and Bob Wards gift cards. A HUGE thank you to the local businesses that donated these prizes and made this event so exciting. It wouldn't have been possible without you!  
 Woody( from Toy Story) was there to greet the children and give them a balloon. Over two hundred balloons were blown up and by the end of the night almost half were on the gym ceiling.

Levi was so excited to see Woody and even more so when Woody gave him one of his guns to keep.
Just his smiles over that, made this night worth it!

What is GAN?

What is GAN?

Giant Axonal Neruopathy

Giant axonal neuropathy is an inherited condition involving dysfunction of a specific type of protein in nerve cells (neurons). The protein is essential for normal nerve function because it forms neurofilaments. Neurofilaments make up a structural framework that helps to define the shape and size of the neurons. This condition is characterized by abnormally large and dysfunctional axons, which are the specialized extensions of nerve cells that are required for the transmission of nerve impulses.

Giant axonal neuropathy generally appears in early childhood. It progresses slowly as neuronal injury becomes more severe. Signs of giant axonal neuropathy usually begin in the peripheral nervous system, which governs movement and sensation in the arms, legs, and other parts of the body. Most individuals with this disorder first have problems with walking. Later they may lose sensation, coordination, strength, and reflexes in their limbs. Hearing and visual problems may also occur. Extremely kinky hair (as compared to others in the family) is characteristic of giant axonal neuropathy.

How do people inherit giant axonal neuropathy?

This condition is inherited in an autosomal recessive pattern, which means both copies of the gene in each cell have mutations. The parents of an individual with an autosomal recessive condition each carry one copy of the mutated gene, but they typically do not show signs and symptoms of the condition.

Currently, there is no cure for GAN.  As the disorder progresses, patients become quadriplegics, dependent on a feeding tube and ventilator. These children who are diagnosed with the disease have a life expectancy no longer than their early twenties while some die much younger.

  

     

   The mission of Hannah’s Hope Fund is to raise funds for a treatment and cure of GAN.  Lori and Matt Sames co-founded HHF following the diagnosis of their youngest daughter, Hannah, in March of 2008.  At the time of Hannah’s diagnosis, Lori was only able to find one scientist in the world actively studying GAN, Dr. Pascale Bomont, in France.  Dr. Yanmin Yang, Stanford University, had studied GAN, but her NIH and MDA grants ran out in 2006.

In August of 2008, HHF brought 20 research scientists together in Boston for the first ever symposium on GAN.  The goals of this meeting was to discuss everything known about GAN, and prioritizes therapeutic approaches, as well as prioritize basic research studies needed to learn more about underlying disease mechanisms.  Gene therapy was prioritized as the #1 therapeutic approaches coming out of this meeting. 

   Dr. Jude Samulski, Director of the University of North Carolina (UNC) at Chapel Hill Gene Therapy Center, sent research associate, Dr. Steven Gray to the symposium to assess whether or not gene therapy was a viable approach for GAN.  HHF began funding Dr. Gray’s work on GAN Oct. 1, 2008. Due to the efforts of Dr. Gray, utilizing more than 25 years of experience obtained at the UNC Gene Therapy Center, there is now a gene therapy approach for GAN. 

On Jan. 26th, 2012, HHF and the UNC clinical team had a Pre Investigational New Drug (Pre IND) meeting with the FDA, and with their guidance, we now have a clear path forward to a GAN gene therapy clinical trial.  The GAN disease community will likely be the first disease community to receive a therapeutic gene to the spinal cord.  The goal is to stop disease progression. Gene therapy will not reverse cell death, so it’s critical these kids receive treatment ASAP.  Every clinical trial is a human experiment.  It will not be known if gene therapy will be an effective treatment for GAN until our clinical trial(s) end.

  

     Through grass-roots fundraising, Hannah’s Hope Fund has had to bear the burden of funding the gene therapy project and a FDA required Natural History Study to track the natural progression of the disease as well as show efficacy of the IND.  HHF, primarily a family and volunteer driven charity, is working tirelessly to move all the children suffering from GAN one step closer to receiving a treatment that will stop the progression of this disease and ultimately save their lives. We are deeply appreciative of our loyal supporters who have made all of this possible.

                                                                                                                                                                   

  Charee says Alan has been having head aches a lot lately, and wants to be held all the time. He's been dizzier too, and not walking well. The twins will be getting walkers soon. They will have a little walker parking lot in their entry. Please do what you can to share their story. More publicity might mean faster medicine.
Thank you for your prayers and your help in this sweet families behalf.
Together, we can make  difference!

Please take a moment to watch this touching video ! (Abridged version)

"If you would like to let the family know you care" you can! at GAN Warriors P.O. 1864 Hamilton MT 59840-1864 All Checks and money contributions should be made to "Hannahs Hope Fund" all contributions are tax deductible!