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Lilliana, Max, Natalya, and Tommy

Year End Update:

2019 has been one of our most successful years since we began our mission over two decades ago. Fundraising efforts and family involvement are at an all time high and gene therapy is moving forward after many years of waiting!

With all the hype and excitement about Aspa's new lab, I have understandably received a few questions related to funding, and whether or not we (CRI) will still need to raise money in light of the vast resources available to Aspa Therapeutics. The answer is yes. This is just one team, and one approach to treatment using one type of gene therapy. Canavan Research Illinois is still fully committed to helping all families and children affected by Canavan disease, and that means leaving no stone unturned in the quest for the cure.

With any first stage treatment trial, the main goal is to make sure the treatment is safe for humans and shows some potential to help patients improve. The best approach is to examine different things that can slow down the relentless progression of Canavan disease. We need to focus on ways to buy more time for the kids while we work on improving their quality of life. This can be done by examining treatments to potentially "fix" any part of the disease that we can. For example, even improvement of vision or general health makes a huge difference for these precious children. As of now, there is still no approved treatment or cure for Canavan, only symptom management. Anything we can do to help the children is extremely important.

No one knows right now if Aspa's or any gene therapy will result in the cure for Canavan, it's too early in the clinical research process. And I would caution parents to keep realistic expectations in mind when dealing with any experimental medicine.

Gene therapy for Canavan disease is not new, it's been used since 1997 and we still have a long way to go. My own son was treated twice, once at age eleven months and again at three years of age. He is now twenty two years old, and while it helped him it was not even close to being a cure.

During the past two decades, Canavan Research Illinois has provided millions of dollars in funding to explore many different medical interventions. In addition to gene therapy, we have provided funds for programs to study several medications as well as stem cell research, but nothing has made a substantial difference yet. It may be that the cure will ultimately involve a combined therapeutic approach, so we must keep raising money and looking at every possibility.

Enzyme replacement therapy is one neglected area, but we need much more funding to explore that option. The worst thing to do is begin a project then become burdened with delays and problems due to a lack of funding. When we begin funding any project we carefully consider all ethical issues, the budget, and proposed timeline for the preclinical research in order to maximize the impact of our resources.

So yes, we are still aggressively raising money so that we'll have enough to fund new projects that we find throughout the world. We have done some internal restructuring to offer more family services at our annual event. And we can optimize the reach of our funding by partnering with other organizations whenever we find a novel research program that needs seed money or an existing program that needs additional funding. We are currently working with other organizations to look for new projects. The research you see today began the same way we will fund new projects in the future. Success doesn't mean it's time to stop, it means that we're making progress and it's time to work harder!

Most of the research that you see today in the rare and ultra rare disease space headed for the clinic or already in clinical trials was originally funded by smaller family run organizations like Canavan Research Illinois, and our mission continues.

As excited as I am about everything with Aspa it doesn't allow me to stop working or stop raising money, but it does take a small burden off my shoulders and off affected families because we now have one fully funded lab on schedule or a gene therapy treatment trial in 2020. This is absolutely amazing news, but it doesn't mean we stop or even slow down our mission to discover the cure for Canavan because as of now we don't know where that will be.

Thank you for your continued support, we could not have been this successful for this long without our dedicated and loyal supporters!

Sincerely,

Ilyce Randell
Director of Patient Advocacy
Canavan Research Illinois

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