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.

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


2021 End of the year update for our families, benefactors and supporters:


This has been a milestone year for Canavan disease. We have two separate gene therapy trials in progress, another in the pipeline and two other teams working on preclinical testing. It's taken me twenty four years of working every day to get to this point. I could never have predicted so long ago that we'd be where we are today. This amazing progress would not have been possible without the help of thousands of people along the way. Help has come from our community, politicians both locally and on Capitol Hill, the FDA, NIH, NINDS, my own family, and other affected families who joined our mission and helped raise money to fund new projects.

Over the past twenty four years the Canavan and ultra rare disease space has grown, changed and evolved. I have learned so much, so many things I never anticipated I'd need to learn. From hosting fundraisers and family networking events, creating and running a charitable organization, becoming a grass roots lobbyist and political activist, to working with every stakeholder in the Canavan community from small start-up biotechs to huge multinational pharmaceutical companies. I was able to create and launch the world's first patient registry for Canavan disease and this in turn helped attract many new people to work on developing therapies to help our precious children. Most of this was done while caring for Maxie and my other child full-time, largely on my own. Sometimes it feels impossible to go on with this mission since Maxie's unexpected passing on April 18th, 2020, but he knew that his life and journey were an inspiration to so many people and I continue this work holding his love in my heart as motivation to push forward.

As things have evolved over the past few years my work has been focused more on patient advocacy and representing the voice of our community to people who are interested in helping our children. It has become commonplace for companies to work with advocacy organizations, so we are continually adapting our mission to best suit the direction of research and drug development for Canavan disease. And my role in this organization has evolved, grown and changed along with the Canavan space.

With so much success behind us it would be easy to slow down, or take a break from this emotionally exhausting work, but we are still years, possibly decades away from the cure. We have been able to make quality of life improvements for patients living with Canavan disease using a combination of therapies. Some children will age out of eligibility for gene therapy which has shown some potential to help slow progression of the disease and improve the quality of life for patients. Watching children age out of being candidates for gene therapy is absolutely heartbreaking for me. I wholeheartedly believe there is sufficient data to show that gene therapy can help some patients living with Canavan disease, but with with that being said it is time to move beyond gene therapy and aggressively search for teams working on other novel approaches. After twenty seven years and several different technologies, gene therapy is still not delivering the results that look like it alone could be the cure. I fully support gene therapy because it seems to help, but looking at the clinical results in patients treated anywhere from several years to eight months ago the outcome as of now is extremely underwhelming. I went through two separate gene therapy trials with my own son and he showed some improvement, and I am thrilled for anyone fortunate enough to be able to get this treatment for their children. Unfortunately it's not even close to being a cure, and after twenty seven years the research doesn't appear to have made much meaningful progress as it pertains to improvements in treated patients. Gene therapy does appear to help and cures can take several decades to develop, but they can also involve a combined approach. If gene therapy alone will not move us towards a cure, and personally I don't believe it is enough, we need to look at other options. Putting all your eggs in one basket is never the best approach. So while we have at least five or six teams that I know of working on clinical trials, or moving towards the clinic, they are all using different types of gene therapy. You can only reinvent a broken wheel so many times before saying "maybe this is not the miracle we hoped for, or perhaps we need to look beyond gene therapy." Sometimes we get so close and personally attached to a project after investing decades of our life and millions of dollars it can become our sole focus because we believe in it so much, and we want it to work so badly that we blindly direct a majority of our resources to it. It's hard to move past something we want and believe in so much, but those emotions can make it hard to see the bigger picture. And at that point we need to take a giant step back and start looking at other options.

We are possibly missing a huge part of the Canavan puzzle. I have spoken with other teams and no one can say for sure whether another gene could be mutated, or contributing to disease progression. After identifying the ASPA mutation on chromosome 17 we have focused solely on that one gene. There are types of cancer that have several genes involved. It is time to take that step back and study the pathology of Canavan disease. We need to look more closely at this disease outside the scope of testing different gene therapy models on mice and children. We need to find the missing pieces of this puzzle. We have already cured hundreds of mice with gene therapy, but not one single child.

My hope is that one of the ongoing trials will still surprise me and deliver a more meaningful result than anticipated, but I will not gamble the lives of our children on that hope. The next phase of my mission will focus on directing funds to teams working on studying the pathology of Canavan disease and finding methods to treat it beyond replacing a single gene. I will continue to support every team and stakeholder working on gene therapy, even new ones, but it's time to expand our efforts to find the cure in whatever form it may take. I have also been actively involved in helping to develop a newborn screen for Canavan disease over the past few years. With more treatment options becoming available it is more critical than ever before to diagnose patients at birth and hopefully get them enrolled in clinical trials earlier to maximize the treatment I will update as more information becomes available about the newborn test.

I thank each and every person who has stood by me for so long and I look forward to paving the way as I direct funding into new areas of research and explore anything and everything that shows promise to help our children. And I will never forget the older patients. Canavan is an aging population with many kids already well into adulthood and those patients are just as important to me as newly diagnosed babies. My son passed away six months before his twenty third birthday and even with all my work his last treatment was at three years of age. I feel in many ways like I let him down. This is a very personal mission for me. And Maxie's memory and love give me the strength, inspiration and courage to move forward and make my way through new and uncharted territory as I continue to search in every direction for the cure. We accept grant requests all year long and have an ongoing RFP (request for proposals) from any team working on Canavan disease, whether it's studying the disease or gathering pre-clinical data. We are always looking for projects that need funding or seed money. Please feel free to share this information, or let me know if you hear of any research in need of funding. We do our best to search for projects, but it's not always possible to locate every team or lab in need of funding. I am always happy to reach out to them personally and see if they need funding to move forward more quickly, or if they're willing and able to apply their work on a related disease to Canavan. A personal request and offer of funding is how many teams begin working on new diseases. It never hurts to make that plea on behalf of our beautiful children.

To learn more about Canavan disease and our mission, or to make a donation to help us search for the cure please contact me at ilyce@canavanresearch.org or visit www.CureCanavan.org.

With peace and gratitude,

Ilyce Randell
Director of Patient Advocacy and Cofounder
Canavan Research Illinois/Canavan Disease Research
#CureCanavan

Announcement from Ilyce Randell:

I am happy to share this exciting update regarding the gene therapy trial from Aspa Therapeutics/BridgeBio!

"Dear Canavan Community Members,
Today we are excited to announce the initiation of our first clinical trial for BBP-812, Aspa’s investigational gene therapy for Canavan disease.

The Phase 1/2 clinical trial, called CANaspire, will evaluate the safety and efficacy of a one-time, intravenous (IV) dose of BBP-812 in children with Canavan disease.

Participant screening will begin immediately at our first clinical trial site in the United States, Massachusetts General Hospital in Boston, Massachusetts, and at first will be limited to US residents. We are working as fast as we can to enable recruitment from outside the United States and are also working to add additional clinical sites in the US and abroad.
This milestone is a result of years of groundbreaking research and development by Dr. Guangping Gao at the University of Massachusetts Medical School, who has made the fight against Canavan disease a central focus of his scientific career. Aspa’s program has included extensive collaboration with Dr. Gao and his team including Dr. Dominic Gessler, along with other leading experts in gene therapy and Canavan disease. The combined efforts contributed to the completion of robust preclinical safety and efficiacy studies in animals, including mice and monkeys. Most importantly, this milestone reflects the ongoing support and insights all of you in the Canavan community have generously shared with us through our collaboration with patient organizations and families.
We want to highlight a few important details about the study:

BBP-812 will be administered as a one-time intravenous (IV) infusion. This is a non-invasive procedure.
CANaspire will enroll children with a confirmed diagnosis with Canavan disease who are 30 months of age or younger on the expected date the investigational gene therapy would be given
We anticipate enrolling approximately 18 participants.
We plan to begin screening participants who live outside the US as soon as possible, and will alert the community when this occurs.
There is no cost to participants or families for participating in the study.
Aspa will pay all travel costs to and from treatment sites for patients and their families.

We recognize that parents and caregivers of older children are also very interested in this treatment. Once we are able to see data in some of our initial patients and have a chance to review that data, we will work together with regulators and study investigators to create opportunities for older Canavan patients to receive BBP-812 as well.

We understand the urgency for all patients, and are proud that BBP-812 has been granted Fast Track designation by the FDA.

If you are interested in learning more about CANaspire, please visit clinicaltrials.gov to find the CANaspire listing. There you will find additional details on the inclusion and exclusion criteria You can also contact the Canavan Disease Study Team at +1.833.764.2267 or +1.617.861.4617 or email CANaspire@aspatx.com.

Investigators at the clinical sites will be responsible for participant screening and selection. Members of the Aspa team are not involved in the selection of individual trial participants.

As with any investigational therapy, there are risks that your child’s physicians and the study investigators will discuss with you. The initiation of CANaspire marks an important step forward in our mission to develop a potential treatment that will improve the lives of as many children with Canavan disease as possible.
As we move forward in this journey, we will share relevant information and program updates whenever we are able. Visit treatcanavan.com to stay connected and to learn about gene therapy.

On behalf of the Aspa Therapeutics Team,

Eric M David MD JD
CEO"

Thank you to everyone who has supported the mission of Canavan Research Illinois/Canavan Disease Research. Whether you are just joining our mission or have been with us from the beginning almost twenty four years ago, this progress would not be possible without our community of donors, sponsors and benefactors!

 

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.

 

 

2021 Spring Update:

Dear friends, family, and supporters,

As most of you probably know we've had to cancel all in person events for 2020 and 2021 due to the global pandemic, but we've been extremely busy working from home advocating for our community, raising funds, developing new projects and working towards increasing awareness about Canavan disease throughout the world.

My own son Maxie who passed away unexpectedly on April 18th, 2020 had received two separate gene therapy treatments. The first one was at eleven months old, and the second one was at three years old. Maxie was not cured by these treatments, but his overall health, motor function and quality of life was improved. He lived an extremely happy and productive life until he passed away at twenty two and a half years of age. One of my goals was to ensure that a third gene therapy trial would begin during his lifetime. Unfortunately I did not reach this milestone before he passed away, but I am thrilled to let our supporters know that the research you helped us pay for with your donations for over twenty years from 1998-2018 has finally led to the approval of a third gene therapy trial. The first patient is tentatively scheduled for surgery on April 8th. This is just ten days before the one year mark of Maxie's passing. I'm absolutely certain that he will be looking down and smiling, knowing that all of our hard work, dedication, sacrifices and countless hours spent growing this organization have finally led to more children receiving gene therapy. Although the families and handful of children who are scheduled to receive this treatment don't know our benefactors personally, it is only because of your tireless efforts, loyalty and generosity for over two decades that their treatments are being made possible. Without your long-term financial support the small lab doing this research would have closed its doors 15 years ago. Your belief in our mission, the commitment to keeping that lab open and operational, and all the funding CRI was able to direct there for over twenty years have finally paid off!

Gene therapy has had many ups and downs. It's fallen in and out of favor with the public and FDA. Through all this we never gave up. I saw firsthand how the treatments helped my own son. I made it my mission to do everything humanly possible to make this treatment available to more children. Enough people stuck by my side and believed in my mission and this organization enabling us to financially support a lab that at times had virtually no other source of funding. I know Maxie won't be here on earth to see this dream become a reality when the first child receives treatment. I am certain he will be looking down and know that he was a leader in this effort by participating in the early trials, and then by allowing the public into his life for over two decades. Maxie's journey served as an example and inspiration for people to keep moving forward even through unimaginable obstacles.

On behalf of Maxie and Canavan Research Illinois I am extending a personal "thank you" to every single person who has ever financially supported this organization in any way. Your donations have and will continue to help us offer hope and medical interventions to other families around the world who have children affected by Canavan disease. When this small group of children receives their gene therapy treatments we will all know in our hearts that decades of hard work has finally achieved one more milestone we set out to reach! This is not something that happened over weeks, months, or a few years. The research began over 24 years ago, and many of you have been with us from day one of this marathon of a journey.

There are also other teams working on gene therapy and different potential treatments for Canavan disease. We hope to see additional clinical trials in 2021, and in upcoming years. One of the major roles of Canavan Research Illinois is patient advocacy. As the landscape in ultra rare disease research has changed and evolved over the past 24 years, so have the programs and roles of CRI. We have formed several important partnerships with other charitable organizations, pharmaceutical and biotech companies and other stakeholders in the Canavan and rare disease space.

Another exciting and necessary project we've been directly involved with for over a year has been to assist in the development of a newborn screening test (NBS) for Canavan disease. With potential treatments on the horizon, early diagnosis is more important than ever before. Medical interventions are believed to be most effective if delivered before the onset of symptoms. So identifying affected babies at birth is critical, and we have expanded our mission to include the development of a newborn screening test for Canavan disease. This test will also include other related rare diseases. I will be sharing detailed information about the NBS, and the role of CRI in our fall newsletter when we have more information about how this process will work. One of our roles will be to expand our community reach and fundraising efforts to make sure that families who need this information are able to receive it before their children are born. Early detection will become increasingly important as new treatments and clinical trials become available. CRI has committed to adding this community outreach program and increased awareness campaign to our mission. As the Canavan space changes and evolves, so does our role and mission.

In 2016 Canavan Research Illinois launched the first international patient registry for Canavan disease. This is still the world's largest single database of Canavan patients and has been used by pharmaceutical companies, other advocacy organizations, venture capitalists and many other stakeholders interested in developing potential treatment options to help our children. We also continue to seek out new teams around the world who are in need of funding for novel research projects for Canavan disease.

Thank you all again for your tireless support, loyalty and belief in this organization and our mission. When the next child receives gene therapy after so many years without any new treatments you will all know that your continued financial support, and perseverance over the span of two decades made it possible. I could never have anticipated when I received Maxie's diagnosis on March 6th, 1998 that what began as an effort to save his life would have grown into this worldwide advocacy effort. I didn't plan or expect to still be doing this after Maxie passed away. But this fight is far from over and everything I do now is in honor of Maxie's amazing life, inspiring journey, and the unconditional love he shared with his family and the world. Nothing we've achieved together would have been possible without your support. We are still desperately in need of funds after canceling our largest fundraisers two years in a row. Your financial contributions will enable us to continue our work, develop new projects, and grow this organization and allow us to evolve with the ever changing Canavan space. I would never have been able to do anything without the support of tens of thousands of people around the world who have supported our mission.

Donations can be made at www.CureCanavan.org. We also have acknowledgement cards available for your donations. Please let us know where to send cards if donations are made in honor or in memory of a loved one. I invite anyone who is interested in donating in Maxie's memory to let us know it's in honor of "Maxie Forever".

This fight is far from over! These recent achievements are not a cue to rest, but to serve as inspiration to push forward more vigorously, and with a renewed conviction to our achieve and expand our mission!

With hope and gratitude,

Ilyce Randell
Director of Patient Advocacy
Canavan Research Illinois