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.
Today, the University of Massachusetts Medical School announced that it was advancing Tay-Sachs research to a Phase I/II clinical trial tentatively scheduled to begin in March/April 2019. This is an enormous advancement for CTSF supporters and represents the culmination of 11 years work for the principal researchers whom we have supported with over $2.2 million dollars in direct grants.
This work, headed by CTSF friends Miguel Esteves, Doug Martin, and Heather Edwards, has already begun the work of manufacturing the Gene Therapy "Vector", which is being made by Nationwide Children's Hospital in Ohio. The CTSF would like to acknowledge the generosity of the Blu Genes Foundation of Canada in allowing this 5-month production process to begin in the last two weeks. The FDA has provided strong guidance up to this point, and the path toward IND (Indication of New Drug) approval looks solid. This will allow the researchers to quickly move from the drug manufacture to the clinical phase of this work... The treatment of real kids.
Many of you have stuck with the CTSF since it's inception. We are grateful and know that you join with us in anticipation of a great outcome from these trials. And, while we have a head start on the costs of the trial itself, it looks like the total cost of the trial will approach $2 million. We still need to raise a big chunk of that.
Today we celebrate with our researchers, our fellow Foundations, UMASS, and all those that have donated to make trials in 2019 possible. Take a brief minute today to say a quick prayer that this effort works in a way that helps heal our kids, both those living today and those yet to be born. And allow yourself to celebrate a little bit too.
www.curetay-sachs.org - Rick
The CTSF brought in just shy of $70,000 in August.... a pretty good month. Many thanks to Dave and Nicki Luce, Nike, CTI, Inc., and Facebook Fundraising. This month looks a little sleepy in terms of events, etc. So, we need your help to make Sept a success. Reach out to friends and friends of friends... and keep those Facebook fundraisers going!!! Exciting news on the research front is right around the corner... yes, it really is!!! (and it will of course, be expensive!)
Bless you all
The CTSF would like to acknowledge the second of two very sizable gifts from the estate of Mary Titleman. Ms. Titleman wished these donations to be made in memory of her and her deceased husband's (Daniel Titleman) deceased children, Enid Deborah Titelman and Marcia Barbara Titelman. A special thanks to Greg Doren, Mary's Nephew, who has been managing his Aunt's estate.
God Bless all
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
"Holy Cow! Over 2000 people "Like" the CTSF Facebook page now, and our this website has been visited 6000 times in the last 12 months. I'm humbled! It's March 5th, and we have already brought in over $100,000 this year for research. With what we have planned for the rest of the year, you ain't seen nuttin yet! Of course, we plan to write checks for over $800,000 this year. Our funding will see the first Gene Therapy treatment this year, and two other clinical trials on affected children! There will be obstacles, but there has never been..... not even close... this much hope. ALL our hope has been from your help. So, if you have a rich crazy Uncle someplace, now is the time to send him our website address. www.curetay-sachs.org. Our work is never too late for our kids and those kids yet to be born. They deserve a full and happy life. That's why we do this"
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.
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.
