All Over Albany

Hannah's Hope Fund

Your three year old daughter is diagnosed with a rare disease.

There's no support group. There's no foundation. There are no clinical trials -- just well meaning doctors with advice about how to make her more comfortable.

If you took that advice, who could fault you?

But Matt Sames and his wife Lori couldn't let go without a fight.

The Rexford couple has managed to bring together an international community of scientists and doctors, and raised over $2.2 million to help save their daughter and others like her.

Now the Hannah's Hope Fund is in fourth place for a $250,000 Pepsi Refresh grant that will help support an upcoming clinical trial.

You may not have heard of Giant Axonal Neuropathy, but if you live in the Capital Region, you probably have heard of the Hannah's Hope Fund.

Matt and Lori Sames started the fund about three years ago, shortly after 3-year-old Hannah was diagnosed with this neurodegenerative disease.

"It's called an 'orphan disease,' because not many people have it -- only about 200,000," Matt says. "But there are about 6,000 of these rare 'orphan diseases,' and put together they affect more people than cancer and AIDS combined."

Hannah first showed symptoms of GAN when she was three. After taking her to a series of doctors, they found a neurologist who recognized the condition and showed them a photo of a little boy in a medical book. "He had fair skin and curly hair, just like Hannah," Mike remembers. "It turns out these are traits of kids with GAN."

They took Hannah for tests, and the disease was confirmed. Doctors said that Hannah would be wheelchair bound by time she was 12 or 13 and would eventually lose the ability to speak, swallow and breathe on her own.

"For the first three days I think I just stayed in bed. Lori got on the computer and started looking around for answers. Finally we decided that if anyone was going to save Hanna,
we had to do it ourselves. So I took over the marketing, we formed a foundation, got a 501c3, we hosted international symposiums, flew scientists in from around the world,
let them debate the disease in Boston, where they determined that gene therapy would be the way to go."

"We found a young scientist at UNC-Chapel Hill who wanted to spearhead this mission. We built a team of scientists from around the world -- France, Canada, in the US at Chapel Hill and Columbia University."

Sames says getting attention for GAN was a little easier than for some other diseases, mostly because doctors already know what causes it -- a genetic mutation that disrupts the production or regulation of a protein used by the nervous system. So all of the money they raised goes toward finding a cure.

Most of the funds they've raised have come from the Capital Region. Matt says people have been very generous.

"In my last career, I worked in television in the Capital Region, so that helped a bit in gaining attention. Channel 6 did a feature on Hannah and a little old lady in Niskayuna saw it. She heard about the Doris Buffet foundation and she took out a paper and pen and hand wrote a letter to Doris Buffett [Warren's sister]. Next thing you know we get a phone call that we got a $500,000 matching grant. In six months we raised $620,000. People have run fund raisers on their own and just brought us the checks, kids have foregone birthday presents and told people to just give them money for Hannah's Hope. The Capital Region has been very good to us."

Hannah's Hope Fund is the only foundation for GAN, so Sames says they get a call every five or six weeks from a family who needs help. "We got a call at 5 am on Sunday from a kid in New Zealand who wants to be part of the clinical trial."

Right now the first clinical trial is scheduled for the fall of 2011. Hannah and at least nine other kids will be go to North Carolina for the trial. That will run about $1.3 million, so the Refresh grant would help a lot.

"Three weeks ago we were 450... then we moved up to 250... and as of last night we were number 4."

The deadline for voting is the end of the month -- you can vote through midnight August 31st.