US Tax Deductible EIN: 26-0256621
- (562) 212-1071
- 2409 E. Luke Avenue Phoenix AZ 85016
US Tax Deductible EIN: 26-0256621
- (562) 212-1071
- 2409 E. Luke Avenue Phoenix AZ 85016
The Cure Tay-Sachs Foundation is dedicated to funding the on-going research needed to find treatments and a cure for Tay-Sachs Disease.
Once the cure for Tay-Sachs has been found - and we will find it! - we will dedicate our efforts to promoting carrier-testing and Tay-Sachs awareness initiatives.
To begin the journey into Tay-Sachs Awareness, click here.
GRANT ANNOUNCEMENT!!!!
The CTSF is very pleased to announce our latest grant in support of future clinical trials for Tay-Sachs and Sandhoff disease. This is your donation money at work!!
The CTSF proudly awards the labs of Queens University in Ontario, Canada $154,660 USD. This money will support and pay for the already initiated work to best understand the methodology and volume of Vector (medicine) to administer to children Intrathecally. This work is part of the complicated journey leading to the safe administration of gene therapy to children.
We will keep you posted on the progress on this work right here.
Donations to the CTSF are fully tax deductible and can be made in any amount by credit card at: www.curetay-sachs.org
Thank you for your continuing interest in FINDING THE CURE!!!!!
Here's a message from Axovant Sciences in NY:
Dear Tay-Sachs and Sandhoff patient communities,
At Axovant, our mission is simple: to develop innovative gene therapies that have the potential to transform the lives of patients. We are writing to provide an update on AXO-AAV-GM2, our investigational gene therapy program for GM2 gangliosidosis.
Over the past year, we have worked tirelessly in partnership with our collaborators to lay the foundation for the start of the first-ever clinical trial for infantile and juvenile Tay-Sachs and Sandhoff disease. In October 2019, our partners at the University of Massachusetts Medical School presented positive data on AXO-AAV-GM2 which demonstrated evidence of potential clinical stabilization in children with GM2 gangliosidosis in an expanded access trial. AXO-AAV-GM2 was observed to be well-tolerated in two expanded access patients with no serious adverse events or clinically relevant laboratory abnormalities related to therapy. Following these encouraging results, at the end of 2019, Axovant submitted an Investigational New Drug Application (IND), to allow for initiation of a company-sponsored clinical trial in patients with GM2 gangliosidosis.
The IND application must include comprehensive information in three broad areas:
The standard timing for FDA review of an IND is 30 days. If there are no additional questions or objections, the trial sponsor is free to start the trial. In this instance, the FDA had additional questions after the 30- day mark on aspects related to manufacturing and the delivery device. Given these questions and subsequent responses go beyond 30 days, the FDA must refer to the extension as a "clinical hold". Pending clearance by FDA, Axovant expects to initiate the AXO-AAV-GM2 trial in 2020.
We want to thank you for your dedicated support over the last year, and we are committed to providing timely, clear, and transparent communications so you can make informed decisions and stay abreast of current developments. We know that you are counting on us to accelerate the development of investigational therapies as quickly as possible. There is no greater priority for the employees at Axovant Gene Therapies, and we will continue to work with urgency to initiate the clinical trial.
Sincerely,
Gavin Corcoran, MD
Chief R&D Officer
Parag V. Meswani, PharmD
SVP, Commercial Strategy & Operations
With the holidays right around the corner, we wanted to take a moment to thank our many donors and friends who helped to make 2019 a great year for the CTSF. It was a great year because we edged ever closer to real treatments for kids and adults with Tay-Sachs and Sandhoff. While true open clinical trials are never fast enough for anyone, we did see both Axovant and IntraBio make significant progress during the year. We also continue to fund promising research elsewhere. And while this is all good news, the bad news is that we lost far too many innocent young lives during the year. We will love them into eternity, and their inspiration will be with us forever.
The year saw fun fundraising ideas, lots of creativity and simple hard work by countless supporters and friends of the CTSF. As of this writing, we are just $29K shy of being a $5 million dollar Foundation! Who would have guessed what a bunch of angry parents could have achieved?!!!
So, we stay the course of funding novel therapies in search of The Cure as we enter 2020. On behalf of myself and the entire Board of Directors, we thank you for following our work and our mission, and we send blessings to you and your loved ones during the Holiday Season and always.
You can always write to me at rick.karl@curetay-sachs.org with any questions or ideas!
- Rick and the CTSF Board
Tay-Sachs disease is caused by the absence or insufficient level of a vital enzyme called Hexosaminidase A (Hex-A). Without Hex-A, a fatty substance or lipid called GM2 ganglioside accumulates abnormally in cells, especially in the nerve cells of the brain. This ongoing accumulation, also called substrate, causes progressive damage to the cells. In Classic Infantile the destructive process begins in the fetus early in pregnancy, although the disease is not clinically apparent (symptoms do not start) until the child is several months old. By the time a child with Tay-Sachs disease is three or four-years old, the nervous system is so badly affected that life itself cannot be supported. Even with the best of care, all children with classic Tay-Sachs disease die early in childhood, usually by the age of 5, although some do live longer.
Many of you have asked how to set up a FB Fundraiser.... here ya go. You can create fundraisers for charitable organizations and personal causes on Facebook for Birthdays, Anniversary's, or any occasion!
1. Click Fundraisers in the left menu of your News Feed
3. Select Nonprofit/Charity (Cure Tay-Sachs Foundation)
4. Select a charitable organization, choose a cover photo and fill in the fundraiser details
5. Click Create
Cure Tay-Sachs Foundation (CTSF) and NTSAD have collaborated with TREND Community to collect information from families and individuals about their experiences using Tanganil, available without a prescription from Europe and indicated for the treatment of positional vertigo (dizziness). It has not been proven to be effective or approved for use to treat symptoms of GM1 and/or GM2 Tay-Sachs and Sandhoff.
Click the link below to download the TREND Community PDF to read more about this initiative, how it can help and how you can sign up to be a part of this unique way to participate in research.
Here is the link. The instructions to sign up are right at the bottom.
http://news.trend.community/home/gm1-gm2-tanganil-health-initiative
Rick Karl, CTSF
Email: Rick.Karl@curetay-sachs.org
Nikko was born on May 14 2015 and he is our firstborn son. Nikko since day one I always knew as his mother that he was angelic and filled a room with so much love with just his presence. What I didn't know was Nikko would be
Words cannot express what a privilege it was to be Brennan's parents. The Lord chose us to care for this precious child and now he is home. Brennan passed on March 4, 2016, in the comfort of his own home. It was fairly sudden and our hearts are broken. Brennan's parents, Holly and Royce Stringer, reside in Prescott, Arizona.
Alyssa Tyner was born on a pretty morning on June 19, 2014. We were so happy and blessed to finally have our family complete. Alyssa was the little sister to Leilani, who felt proud to be the big loving sister.
Krystie Anna Karl-Steiger was born on January 26, 2006 on a warm sunny winter day in California. Our precious little girl had a full head of hair, and gulped down her first bottle in record time.
Ryan Anthony Babo was born February 19, 2002. Like all Tay-Sachs children, he was absolutely beautiful. I remember lying in bed next to him when he was just a week old, watching him sleep, wondering what I had ever done in my life to deserve him.
Molly Grace was born on September 26, 2003. She was born 2 months early and weighed only 4lbs 0oz. Although she was so small she was very healthy.
Elise Catherine ten Berge was born on February 5, 2003. She was our first child and as all new parents do, we thought about what Elise's future would be like: her first word, her first step, and her first day of school.
Dakota Jean Bihn was born on May 30, 1999. She was a beautiful baby, weighing in at exactly 8 pounds. Everything about her seemed perfect. During Dakota's first year, she developed as a normal baby should. She sat up, rolled over, ate, and walked, all when she should.
Conner, a precious miracle and an amazing creature sent to Carl, Brenden and myself one cold, snowy January day. From the second he entered our life, I knew in my heart he was so incredibly special. His first smile, his first cry, even his first breath, he was--and remains--beautiful.
My name is Alexis Nichole Markowich or just "Lexi". I was born on March 9, 2001 to Craig and Nichole Markowich and I also have a big sister Brianne. When I was four, on August 4, 2005, we got the devastating news that I had Juvenile Onset Tay Sachs Disease (JOTSD).
Isabel was born in Buenos Aires on February of 2009. Until the age of two Isabel was a healthy little girl. She had the life that any other child could have: school, little friends at park, holidays and trips to Spain to visit her family. And always a big smile on her face...
Our daughter Lexi was born on March 30, 2011. She was born in Junction City, Kansas right outside of Fort Riley. I am in the army so.....
Our son Nathan Harney is almost 17 months old. He is a wonderful, happy baby who has beautiful blue eyes, a huge personality, and is the light of our lives. He is a gentle soul that we have been blessed with......
My sisters and I had what seemed to be a normal up bringing in Southwestern, Ohio to a set of parents who were regular too.....
On March 16, 2007, Isaiah came into the world at 9:41 PM weighing 8 lbs. 6 oz. he was greeted by his daddy, Grandma Lisa, and Great Aunt Chaundra.
Rachaeli entered this world with her eyes open wide; God delivered her to us shortly before the start of the Sabbath on Friday October 25, 2002. It was a perfect delivery, following a perfect pregnancy. Rachaeli was absolutely captivating.
Kaiden was born December 20, 2006. He was my beautiful blue eye, blond hair angel. He was the best baby any parents could ask for. He smiled constantly and barely cried. I would tell my Mother all the time that he was my "special little man".......
Elise was born December 1, 2004 in Santa Maria, California. She was an absolutely perfect baby with an easy pregnancy. We never imagined we could love someone as much as we love Elise. The next 6 months were the happiest months of our lives......
Travis Cole Snellgrove was born to Wendi and Travis on April 12th, 2004. He was born with no problems and was home the next day. He was a very good baby and we had no problems with him for the first month, then he started having trouble.....
Dylan James Manning (commonly referred to as "DJ") was born on Thanksgiving Day, November 22, 2001. Dylans arrival was a long awaited event for his parents, Brian and Sherri of Atglen, Pennsylvania. Although Dylan was anxious to arrive almost four weeks early, he was not quick to reach his milestones. Doctors naturally gave him a little extra time to reach things like crawling and rolling over due to his prematurity.
Amelia was born September 29, 2006 and was a sweet, easy-going baby right from the start. Her baby book filled up as it should have first smile at four weeks, rolled over at four months, babbled at five months.
Charles Lee Anderson was born on February 26, 2006 to Bryce and Amy Anderson. His sister Anna was 14 and brother Ben was 11. Charles Lee was so perfect.
Emma Rose was born on May 17, 2004. She was very eager to enter our world - arriving before my doctor could even get to the hospital! She was a beautiful, healthy, and sweet baby.
My name is Lacie Wivell and this is my story. I was born on April 8, 2004, to Rick and Lisa Wivell. I had two older sisters, Amanda and Rikki, and two older brothers, Levi and Colby. I also had another brother, Dakota, that went to be an angel in 2001.
Mallory Rayne Salazar is a ray of light that came to us one rainy Saturday afternoon. She was born on July 14, 2007 and was 7lbs. 8oz. of pure perfection. From the first time I laid eyes on her, I knew there was something "special" about her. Little did we know just how much she would teach us.
Dr. Walia's lab novel gene therapeutic treatment approaches and its proof-of principle have recently been published. The ongoing work in his lab includes:
The outcomes from our research will help us and others guide planning of a clinical trial for patients with GM2 gangliosidosis to find a treatment they have been long waiting for.
Gene therapy for Tay-Sachs and Sandhoff diseases has been redesigned to reduce the potency of the vectors, thereby increasing safety. Studies have been conducted with guidance from the FDA about what is needed for clinical trials to begin.
The new vector system clearly restores hexosaminidase activity after injection into the brains of mice, and storage levels are decreased. Treated mice have increased life spans and show no side effects of the treatment.
To thoroughly test safety, the new vector system has been tested in non-human primates (monkeys) for almost 1 year, with no evidence of toxicity on MRIs or behavioral tests. A few steps remain before the clinical trial can begin: monkey tissues need to be analyzed microscopically by pathologists, large-scale vector manufacturing must be completed, and regulatory documents must be prepared and filed. All of these activities can begin when funding is available.
Currently we are continuing our in vivo efficacy and safety studies. We have observed a significant decrease in GM2 aggregates in the brains of mice engrafted with a high percentage of human cells transduced with the HexA/HexB lentiviral vector.
We have also seen an extension of lifespan in these mice. Human hematopoietic stem cells transduced with the HexA/HexB lentiviral vector also matured into normal immune system cells when engrafted in a humanized mouse model. We are repeating some of these experiments so that we have enough mice to get statistically significant data.
Once these experiments are completed, we will be able to have a pre-IND meeting with the FDA.
Tay-Sachs book written as part of Gold Star Award for Girl Scouts by Katelyn Foy Tay-Sachs Disease What It Is All About.
Tay-Sachs Awareness Video - see Dakota and her friends Tay-Sachs Pretty Girl Portraits Video by Eric Fier.
Have you ever heard the expression "One in a million?" I am sure most everyone has.I'd like to try and explain what One In A Million means to me.
The Cure Tay-Sachs Foundation is very grateful you decided to donate and we thank you for your generosity. Your donation will be used to support research to find a cure for Tay-Sachs disease.
The Cure Tay-Sachs Foundation is exempt under Section 501(c)(3) of the Internal Revenue Code, making this gift tax deductible to the fullest extend permitted by law.
Please click the PayPal button to make a donation today! You can donate using a credit card - without a paypal account - by following the instruction on the paypal system (lower left). Or, if you prefer, please send your donation (payable to the Cure Tay-Sachs Foundation) to the address below.
