US Tax Deductible EIN: 26-0256621
- (562) 212-1071
- 2409 E. Luke Avenue Phoenix AZ 85016
Since our creation in June 2007, the CTSF has raised $4,171,670 from thousands of kind and generous donors. We have issued research grants to 9 researchers/laboratories around the world, and made impressive progress towards our ultimate goal... The Cure!
IMPORTANT READ: Finally, perhaps, some HOPE from a "new" treatment:
I've been authorized by Mallory Taylor, CEO of IntraBio in the UK, to pass on to you some information on a drug that may have some potential, in some cases within our community, to slow the build-up of GM2. Commonly known as Tanganil, this over the counter (in France) and inexpensive drug has shown great promise and is worthy of further study. These studies are planned as noted below. Neither I nor the CTSF re...commend or advocate for this drug at this point, as it is not FDA approved for use in the US.
I pass on this information because doing so is consistent with our mission statement of communicating on-going research and discovery related to the treatment of Tay-Sachs. While this drug is NOT a cure, I believe any news of such significant importance should be shared with our community and our donors.
IntraBio Clinical Programs
Tay-Sachs Disease with IB1000
IntraBio Inc is pleased to announce significant progress in the treatment of rare neurodegenerative diseases with IB1000, a well-tolerated, modified amino acid that is administered orally. Most notably, IB1000 is showing promising effects in pre-clinical and Phase 1 observational clinical studies in patients with late-onset Tay-Sachs (TS) disease. These findings are also supported by results from in vitro cellular studies and studies in a Sandhoff animal model. In addition, patients with Niemann-Pick Type C (NPC) disease, and various subtypes of Cerebellar Ataxias (CA) have been treated with IB1000 and observed symptomatic benefit. A multi-national, randomized, controlled Phase 2/3 clinical trial of IB1000 in 108 CA patients was recently completed, with the final results expected in 2018.
IntraBio is now planning multi-national randomized, controlled pivotal clinical trials to evaluate the safety and efficacy of IB1000 as a therapeutic intervention in patients with TS, NPC and specific CA diseases. IntraBio hopes to commence enrollment in one or more of these pivotal trials in the EU in early 2018 to be followed by recruitment in North America. In these trials, the safety and efficacy of IB1000 will be determined for each indication using validated clinical and quality of life assessment scales. IntraBio will provide full details of clinical site locations and the clinical trial design including inclusion criteria as soon as they are available.
IntraBio is a global leader developing novel therapies and treatments for "orphan" and neurodegenerative diseases, including lysosomal storage disorders, which have high unmet medical needs. IntraBio has been granted Orphan Medicinal Product Designation by the European Medicines Agency (EMA) for two drug compounds, including IB1000 for the treatment of Niemann-Pick Types A, B & C diseases.
Chairman of IntraBio Inc
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.
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.
I picked a bad month to go on vacation and cause my monthly update to be late! We had an outstanding fundraising month at the CTSF, with most of the heavy lifting done by Dave and Nicki Luce and their annual Princess Riley Golf outing. I love the good folks of Coldwater Michigan and their surrounding communities!! In total, 165 kind and generous donors kicked in $32,033.13 to fund the advanced research that our kids need. Think about that.... 165!!! And I even closed the month a couple days early because I was headed out of hot Phoenix to somewhere cooler!! That is 6 a day, every day! How do we do that? Well, it's fundraisers, it's awareness (sometimes a check shows up in my mailbox and I have no idea who it's from or why), sometimes it's a nice corporate match. We get checks from Facebook's Network for Good, we get checks for #HikeforGrayson.... I love that family!!! Felicia had an idea, and hiked with it!!! I know there is more to come from some local Sacramento businesses. Who will be next to go viral with an idea?
My favorite day was when we got separate checks in my mailbox from The National Christian Foundation and Temple Avodat Shalom.... proving that indeed we can actually all get along!
We sponsored a football team, because we take care of our own. Go Justin and Josh!
I met with the CEO of IntraBio and his team in London regarding the drug Tanganil. We were the first group to get their official announcement of what they are doing and what their plans are. More importantly, I now have a much better understanding of the potential of the active ingredient in Tanganil, and what we as a group can and will do to get it approved in the US for clinical trials. You just would not believe how DUMB the reasons are that the drug is not sold here. (more on that later)
And, we have quickly funded a US based group to study the toxicity, or lack thereof, of Tanganil in Sandhoff Mice. I like quick. It's nice to have the money in the bank to be nimble. More on that later too.
It was a big month at the post office for me. It was a record mail out month for our Iceberg award ($1000+), and for Tee/Golf/Kids shirts. I sent out 460 of them!!! I believe that awareness and discussion is our greatest asset, and it's so cool to see pictures of folks wearing CTSF shirts in their day to day life.... Even if doing so caused long lines behind me at the post office. I got a lot of frowns from those behind me in line. If you read this far, I still have some more... although some sizes are gone.
Royce Stringer is designing our first ever Holiday appeal card and letter! We have thousands of addresses, thousands of e-mails, and thousands of friends on FB and Twitter. I'm excited for that.
This month, Samantha Rabinowitz is donating a portion of her Bat Mitzvah proceeds to us, with her folks matching that. Her cousin in Heaven, Emma, is so proud. There is a big flashing button on our website if you want to drain her parents a bit more! Ha. Also, don't forget that it is Liz and Becky Warren's 31st birthday this month.
What a month! A great fundraiser, a ton of awareness, the funding of a study on Tanganil for Sandhoff, a meeting with the CEO and his team related to a "new drug", 165 donations, and I even squeezed in a vacation.
Thank you for helping me ^Krystie^. I miss you more and more each day.
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.
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