Project Alive Releases Letter to the Hunter Syndrome Community

November 9, 2023

Project Alive, a Hunter syndrome research and advocacy organization, released the following letter to the Hunter syndrome community addressing their new shift in focus and updated mission statement.

To our community,
It is with sincere dedication to our community and our mission that we share our decision to move our funding efforts away from the original gene therapy trial for which we started raising money years ago. Many of you will remember the hope we all felt back in 2012 when a few determined and courageous parents began a collaboration with Nationwide Children's Hospital to fund a gene therapy trial we believed could bring curative medicine to our kids. No matter when you joined our community, you will no doubt share in the grief we feel that we have not been able to bring this cure to fruition--but we believe that you will also rejoice with us in how far our community has come since then.
A rare disease diagnosis can be heart-shattering, since up to 90% of rare diseases have no treatment at all. Back in 2012, the only approved treatment for Hunter syndrome was a welcome but wholly inadequate weekly treatment for our kids. This somewhat bleak landscape is what led parents to band together to form Project Alive to try to change the Hunter Syndrome diagnosis conversation from, “I’m sorry, but there is not much we can do but delay the inevitable,” to “I’m so glad we were able to reach a diagnosis, because now we can provide your child with a cure!”
It was a lofty goal. It was a worthwhile goal. And our entire community is indebted to these pioneering parents whose dreams helped lay the groundwork for all that has come since: more kids are being diagnosed earlier, as Hunter Syndrome has been added to the recommended uniform screening panel (RUSP); there are multiple new blood-brain-barrier crossing drugs in development; many of our boys are in clinical trials--and yet, until ALL of our kids have access to curative medicine, it will never be sufficient and we will keep pushing forward with our mission!
Our organization has been met with many challenges in our hope to fund the development of a gene therapy drug. After the trial stalled at Nationwide Children's Hospital, we had hopes that moving the trial to UNC, Chapel Hill would help to move the trial forward. But this has since proved to be cost prohibitive without an industry partner. It is unfortunate that the company we were counting on to help us has folded, because funding a gene therapy trial solely as a small nonprofit organization has become nearly impossible. We have been fighting hard behind the scenes to push this forward and to share more information with our community, but legally binding confidentiality agreements have hindered our ability to share much publicly. In addition, much of the conversation about the trial shifted outside of our control and outside of our inclusion in the agreements.
We are not giving up on gene therapy or a cure. If an industry partner or large donor expresses an interest in the trial, we will connect them with the team at UNC, Chapel Hill, who currently hold the IND for the drug. As a nonprofit dedicated to helping kids with Hunter syndrome, we simply cannot hold onto money for a trial that we are unable to fully fund--especially because there are wonderful ways we want to put our funds to use to benefit boys and men with Hunter syndrome.
With these factors in mind, along with lengthy consultation with members of our community, donors, researchers, and physicians, Project Alive is moving forward to fund other much-needed projects. We will continue to be true to our mission to fund a cure and find better treatments for our boys. We have already started planning how we use these funds, and we welcome your input.
As you know, we have not sat idle as we pursued gene therapy. We have been working alongside other rare organizations to help boys with Hunter syndrome receive an earlier diagnosis and have access to clinical trials. We have helped strengthen trial design by encouraging pharmaceutical companies to consult with parents and patients. We have developed a scientific advisory board and established a grant program to help foster research for Hunter syndrome. We have expanded our community education programs and provided support to families in all stages of the Hunter journey through webinars, workshops, and virtual meetups. We have established access to a free IEP advocate for our Hunter families. We have brought our community together to support each other and provide vital education via Project Alive’s annual Hunter Syndrome Community Conference, as well as the Hunter Syndrome Family Fair--and much more.
We are all part of an amazing community. Each of you placed a great deal of faith in Project Alive, our other great fundraising partners, and in our board and leadership. Although we are mourning this change of course, we truly believe that it has been through the efforts of our community that there are now numerous new drugs in development for our boys. This disease is relentless and progressive, and we have to recognize the changing landscape and adapt accordingly. Our mission was and is to find a cure for Hunter syndrome, and we are excited about the many ways we can put our funds to work towards that mission.
After much discussion with all our stakeholders (families, researchers, physicians, foundations, and industry partners), Project Alive has decided to expand our mission to encompass all the work we will do to support the Hunter syndrome community and the current and future research that will expand treatment options for Hunter syndrome. Our new mission statement is as follows:
Project Alive exists to find a cure for Hunter syndrome through research and advocacy, and provide education and resources to the Hunter syndrome community.
Our boys are counting on us to keep fighting, although this might look different than we had imagined when we started this fight. And that is exactly what we will do with your help.
Please reach out to any of our board members with any questions or feedback. We are all in this together.
Sincerely,
Project Alive Board of Directors and Staff