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 $4,237,025 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!
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.
Dear Mr Karl:
Thank you once again for expressing your continued interest in the investigational compound, Nacetyl-D, L-leucine, and its potential therapeutic applications for the treatment of Niemann-PickDisease type C, Tay-Sachs, ataxias and other types of lysosomal storage and neurodegenerative diseases.
In your recent inquiry, you asked for the status of the preparatory activities for the planned clinical trials. We are very pleased to inform you that IntraBio Inc and Pierre Fabre Laboratories have now concluded an agreement to enable IntraBio to gain full access to all documents related to N-acetyl- D,L-leucine and its enantiomer, owned by Pierre Fabre, to support the preparation of the required documents to seek regulatory approval in order for these clinical trials to be initiated in a timely manner.
Both companies are also engaged in ongoing discussions to explore the opportunity for Pierre Fabre Laboratories to provide further technical support for these clinical trials to be carried out by IntraBio as the study sponsor.
With your continued support, we very much look forward to ensuring that the clinical development will lead to new methods of treatment to benefit patients in these rare and life-threatening conditions where there is unquestionably a clear unmet medical need.
For IntraBio Inc
Mallory Factor, Chairman
For Pierre Fabre Laboratories
Silvia Gatti-MacArthur, Head of Central Nervous System Innovation Unit
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
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 didn't write an update for October, as I was pretty upset with the passing of Mackenzie Plick. Honestly, I just didn't feel like it. So, this update is for both October and November.
We had so many nice folks do fundraisers. Can you believe we had over 340 individual donations during just two months? It's really getting hard to track them all. That's as great problem for me. Of course, this total includes a big outpouring of support during "Giving Tuesday". For the two months, we brought in $50,295. On top of that, there is about $20,000 due to us from FB and the Network for Good, and also from a Go Fund Me campaign. $70,000 in two months is quite a good number. And I got to send out a bunch of Iceberg Awards, my favorite thing to do.
There is new news from FB. They have taken their 5% fee down to ZERO! Apparently, Mr. Zuckerberg is going to cover that for the company. Nice of the guy! So, if you donate thorough a FB fundraiser for us, we don't pay a dime. (What about you PayPal?). The only bummer is that I don't always get very good reporting from FB (ok, it sucks) on who donated, so I cannot thank you personally on behalf of the CTSF.
Donations came in for so many kids, I won't list them all here. We love you Mackenzie, and we now send our love to Amelie in the UK. RIP to you both. We also send our prayers to Nikko and her parents in Illinois. If you want see all the donations we have gotten for the year, and for all those who they were dedicated to, it's all on our website!
Our first annual Holiday Cards will go out on Monday to about 2500 folks. If you recently donated and you get one, please forgive me. Managing that many addresses and stuffing that many envelopes is a hell of a lot of work. Many thanks to The Anderson and Stringer families for all their help getting out a really cool card. It will also go out to 3000 folks via e-mail.
The researchers we are funding are doing great, and I'll have news on that in the coming months. Also, I just posted a letter from the "Tanganil People" about where they are headed with their variation of that drug. Damn, 2017 has been a good year for this miserable club of ours!! (Great people with a bad reason to know each other).
I only have one thing on my Christmas list this year. I am asking that everyone do ONE FUNdraiser for us in 2018. Make it part of your New Year's Resolution list... No weight loss is required!! It doesn't need to be big or elaborate or a bundle of work. But I can't do this alone. Like everything in life, success comes from who you know... And all of you know nice folks. If nothing else, and these can be big, just pop a fundraiser for a special occasion of yours onto FB for us. What could be easier... And you will really know which of those 1000 friends are really friends!
By the way, we have shirts now in every size. Cool new Orange ones too (love orange). And, next week, we will have hats and hoodies. Need some stuff for a fundraiser?..... just tell me. Make a meaningful donation based on what you are able to do, and I'll send you something cool. The local Post Office knows my first name!
So that's it. I wish you all the warmest of Holiday wishes, and time with your loved ones. Thanks for all your support this year. There is Hope for these kids now, and those not yet born. That hope did not come from the Government or some big celebrity, it came from you. God Bless you all.
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.
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