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.
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:
Family of Teen Wrestler Fights Hard to Beat Odds - Patch News:
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.
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:
In 2019 and beyond Canavan Research Illinois will bring hope to more families. Over the past several years CRI has been able to reserve significant funding in order to advance research initiatives aimed at helping older patients; "older" refers to children past two years of age.
With good care (and even without treatment) most kids affected by Canavan disease are living into adolescence, and many well into their teens and young adulthood. There is virtually nothing available beyond symptom management to help "older" patients.
We have always known that we needed treatment options for older kids, but most funds we raised were used to advance gene therapy research. Now funding for gene therapy research is being taken care of by pharmaceutical companies, freeing up more funding for other projects.
The current potential gene therapy trial is only for 4 to 6 kids, and it's being funded by the parents of those children hoping to receive treatment before (and if) the product ever becomes available commercially. That technology, if approved, will be more geared towards babies, so unfortunately the older children won't benefit.
Our goal has always been to look at ways to help the older patients who are largely being cast aside. Even if they are too old to be cured, it is still necessary to try and reverse some damage, halt additional disease progression and improve their quality of life. The brain's ability to heal itself is amazing and not fully understood. We need to focus more on helping the older kids who have no treatment options. Significant funding is already set aside, but it is not enough. We still need additional funds to move forward and help the older patients.
Out of approximately 600 patients living with Canavan disease throughout the world only 4-6 children have any hope for treatment within the next year, and they are paying for it privately...let that sink in! Canavan Research Illinois is a beacon of hope for the other 99.17% of the children who have largely been abandoned and dismissed. As a patient's advocate, and mother of a 21 year old son who has Canavan disease I will not forget about the older kids, and I will make sure no one else does either!
Please help support CRI and our mission to help ALL children living with Canvan disease. It's absolutely critical to target this disease early, but in reality by the time a gene therapy clinical trial is funded and approved there are only a handful of kids under two available to participate. These kids are growing up and getting older and they make up a vast majority of patients. With so many patients living into their teens we absolutely need to help kids over the age of two!
Your financial support can help millions of people affected by related diseases As a single gene disorder Canavan serves as a great model for teams studying many more common brain diseases such as Alzheimer's, ALS, and Parkinson's. There is always hope, and we need to gain a better understanding of the brain's potential to heal itself. My own son is 21 years old and living with a degenerative brain disease, but I still have hope that he can be helped. My goal is to improve his quality of life and anyone else in a similar situation. It's never too late, and I will never stop fighting for Maxie and all these kids!
Director of Patient Advocacy at Canavan Research Illinois