News

Canavan Disease In The News!

Ilyce and Max Randell have represented our battle to save the children battling Canavan disease on national shows including: Dateline NBC, Leeza, Montel, NBC, FOX, ABC, WGN CBS News and Live with Regis and Kelly. We have won the 'Make A Difference Day' Award as well as the National Diet Rite Cola Zero Boundaries Award for our dedication to the children battling Canavan disease.

Our mission to cure Canavan disease has also been featured in the Philadelphia Inquirer, Washington Post, the Chicago Tribune, Pioneer Press, CJN, Daily Herald, JUF, as well as newspapers worldwide.

Now 21, Max continues to make appearances helping to raise awareness of Canavan disease and the desperate need for funding to support life saving medical research.

Exciting Medical News from Ilyce:

It is my honor and pleasure to share exciting news from Aspa Therapeutics about their gene therapy research. The official announcement is included below, but first I would like to explain why as the parent of an affected child I am so excited about this announcement. Three different delivery methods were tested and it was discovered that more new genes were expressed in the appropriate places through IV delivery. My son has had seven holes drilled into his skull during two previous gene therapy surgeries, but Aspa has discovered that their delivery system is more effective with just an IV! I'm grateful that this company has the vast resources necessary to fully investigate every possible approach to delivering the corrected gene to our children in the safest, most effective and least invasive way possible. As a mom who has been through two separate gene therapy trials that both involved brain surgery this is the best news I've heard about gene therapy for Canavan disease in a very long time!

Announcement from Aspa Therapeutics:
"Abstract presented at ESGCT about Aspa’s Canavan Disease gene therapy, on October 22, 2019
Aspa Therapeutics is pleased to share the news that a poster was presented on October 22, 2019 at the European Society of Gene and Cell Therapy meeting, describing an important development in gene therapy for Canavan disease.
The poster, written by David Scott and colleagues from the BridgeBio Gene Therapy laboratory, describes a study conducted in non-human primates comparing three different methods of delivering the gene therapy that Aspa Therapeutics is developing for patients with Canavan disease.

The researchers compared IV, (intravenous delivery, in which the treatment is administered through a small needle placed into a vein), IT (intrathecal, an injection into the cerebrospinal fluid in the space surrounding the spinal cord), and ICV (intracerebroventricular injection, where the treatment is infused through the skull into one of the large collections of cerebrospinal fluid that sits within the middle of the brain). The researchers then measured how well the therapy was able to reach the parts of the brain, spinal cord, and other tissues most relevant to Canavan disease.
 
While all 3 methods showed some ability to reach the targeted areas, IV delivery was optimal in reaching all brain regions, especially the deep brain structures that appear to be most relevant to Canavan disease.The researchers therefore recommended that that IV infusion be utilized to administer the Aspa investigational gene therapy to patients with Canavan disease.

IV infusion is one of the most common ways to administer treatments, and is the same mode of delivery used in the clinical trial that Avexis / Novartis recently conducted, leading to approval of their gene therapy for children with Spinal Muscular Atrophy Type 1 (SMA-1)."

They are on schedule for a treatment trial in 2020. Vector production and working with the FDA have already been underway for awhile, and families will not be asked to pay for this gene therapy! So much amazing news for our community. 

The next step is to gather more information from families and patients for the natural history study required by the FDA. As the parent of an affected child I'm thrilled to participate in this study because it will bring the treatment out of the lab and to our kids more quickly.
Aspa has been working with the FDA throughout this process to ensure that all required information will be supplied when the program is submitted for approval. The goal is to avoid unnecessary delays by submitting a near perfect application to the FDA on the first try!
Aspa will not be charging families to participate in the treatment trial or natural history study and they are even covering many of the costs associated with participation. To learn more follow the link below.
https://treatcanavan.com/


Canavan Research Illinois is a research partner with:


Local High School Wrestler Inspired by his Brother - WGN Television:
The Randell brothers' dual stories of determination:

https://wgntv.com/2019/07/07/local-hs-wrestler-inspired-by-his-brother/

Family of Teen Wrestler Fights Hard to Beat Odds - Patch News:
https://patch.com/illinois/buffalogrove/family-teen-wrestler-fights-hard-beat-odds?fbclid=IwAR0TnkFvgJQMcsg2TtBfdaqbUyESYvNGzz6VQnwDyqxiElQWoWgvByQGKhg

 

October is 'Canavan Disease Awareness Month'


Ilyce Randell also writes and contributes stories for publication to The Mighty.
As the parent of an affected child she uses her experience to help other families affected by Canavan disease.

Canavan Research Illinois Logo

Save the Date

The 22nd Annual Canavan Charity Ball will
be held on Saturday, October 17th, 2020 at the
Chicago Marriot in Schaumburg, Illinois.
Click here for more information.

 

 

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 possiblity.

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