Project Alive Gene Therapy

$1,103,500 of $2.5M goal

Raised by 889 people in 9 months
Created May 31, 2017
All funds raised benefit the mission of Project Alive, a 501(c)(3) not-for-profit, which is to support a cure for Hunter Syndrome / MPS II. Donations are tax deductible (Tax ID: 46-4617970).


Hunter Syndrome (MPS II) is a rare genetic disease that affects less than 500 boys in the United States. These boys are missing an enzyme used to break down cellular waste. When that waste builds up, it leads to progressive damage throughout their little bodies. Some never develop speech, but some boys learn like a typical child then begin losing skills and what they’ve learned at a young age. They eventually lose the ability to walk, talk and eat. Most do not live to see their teen years.

Gene therapy is a revolutionary approach to treating genetic diseases. It involves a one-time delivery of a normal copy of the defective gene which ideally causes the body to naturally produce the missing enzyme. It is the most promising ‘cure’ that these boys desperately need.


In 2013, after it had been performed on other forms of MPS with positive results, a group of Hunter Syndrome families initiated a partnership with researchers at Nationwide Children’s Hospital in Columbus, Ohio to begin gene therapy research in Hunter Syndrome / MPS II. Now, the researchers are in the final stages of developing the first gene therapy clinical trial for boys with Hunter Syndrome. The only obstacle is the substantial funding required for such a trial. If the necessary funds are raised, the trial could begin as early as late 2017 or early 2018.


In the world of medicine, this treatment is considered an ‘orphan drug’ because of the small patient population it seeks to treat. In the US, an orphan drug is a treatment for a disease that affects less than 200,000 people. Drug companies fund much of the research for more common diseases because in developing a potential blockbuster drug for a large patient population, they also are more likely to make a profit. Few companies are willing to invest in research for such a small population – only an estimated 500 boys with Hunter Syndrome live in the United States. Without drug companies pursuing a cure for Hunter Syndrome, families tackled that lofty goal and have finally reached the point where a potential cure is ready and waiting with one obstacle: funding it.

Documentary Series

Episode 1. 'To Choose A Path'

Episode 2. 'Noah'

Episode 3. 'Finn'

Episode 4. 'Just Like Me'
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Your continued support means the world to us, and it means a chance at LIFE for our boys! You’ve helped us cross the $1.1 million mark as of today, pushing us that much closer to the goal of giving kids with Hunter Syndrome the chance to grow up and #endhuntersyndrome!

Share the boys’ stories, share this update, and ask friends to partner with you in the saving kids!  Let’s raise the remaining $1.4 million by July to start this life-saving clinical trial by the end of 2018!
Follow @projectalive on social media to follow our #rarediseasefacts this month.
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YOU have blown 2017 out of the park! You've given over $1 MILLION toward a cure for Hunter Syndrome so far. Today, on a day of New Year's celebration worldwide, let's celebrate some of the major milestones you propelled Project Alive to hit in 2017:

1. You gave over $100,000 on Giving Tuesday towards a cure for Hunter Syndrome.

2. You gave over $1 million in 2017 toward our $2.5 million goal of funding the gene therapy clinical trial.

3. Project Alive signed the agreement to begin manufacturing the gene vector needed for the trial (a $1.4 million obligation).

4. The FDA approved the investigational new drug application (IND) required for the trial to begin once vector production is complete.

And as if that wasn't enough, in the waning hours of 2017, Project Alive was featured on the TV show "Inside the NBA"! Case Hogan, who also appeared on "The Doctors" this fall and was surprised with videos and gifts from his favorite NBA stars and legends, got to meet Charles Barkley, Shaquille O'Neal, Kenny Smith, and Ernie Johnson. We love their support of Project Alive!

CHECK OUT and SHARE this amazing video!
Over $1 MILLION in 2017! Wow!
Project Alive on "Inside the NBA"
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Hi all!

You've been a part of some incredible progress toward our goal - we just passed $750,000 and reached the first milestone payment for gene vector production! We're taking a moment of thanks, but then pivoting back to raising the remaining $650,000 needed to fund the vector production. Keep up the great work!

We're also excited about all of the press Project Alive has been getting lately. You saw the videos from The Doctors TV show in our last update, but have you seen these?

An article on Today.com: Mother races against time to fund research trial for son's fatal disease --> https://www.today.com/parents/hunter-syndrome-mom-races-find-cure-fatal-disease-t117429

A feature on People.com: Family of 10-Year-Old Boy with Deadly Disease Races Against Time to Fund Trial for Other Kids: 'We're Not Giving Up' --> http://people.com/human-interest/melissa-hogan-hunters-syndrome-clinical-trial/

A powerful story on Charlotte TV: Charlotte Boy Living With Rare Genetic Disease --> http://spectrumlocalnews.com/nc/charlotte/news/2017/11/11/charlotte-boy-living-with-rare-genetic-disease

Thank you for sharing these updates on your social networks and doing so much to help fund this trial!
From the People.com article
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If you missed the recent segment on the TV show "The Doctors" about Hunter Syndrome and one of our sweet families, the Hogans, you can watch it here in two parts!

Be sure to share these videos with your tribe on Facebook, Twitter, Instagram and more - we need your help in building even more momentum from this first national TV appearance for our Hunter Syndrome community!

We have passed $600,000 and continue to build toward our goal. Can you get five for $5? Share this update to get 5 people to give at least $5 on Monday, October 9? #5for5
Part 1 of The Doctors' segment
Part 2 of The Doctors' segment
Case gives big hugs on The Doctors
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$1,103,500 of $2.5M goal

Raised by 889 people in 9 months
Created May 31, 2017
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