Project Shui for Tay-Sachs

Caringbridge Journal Update

Commentary on New Experimental Neuronal Stem Cell Transplant 
November 17, 2010

*Video or Pictures of Tay-Sachs

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from the Caringbridge Online Journal

Wednesday, November 17, 2010 9:28 PM, EST

Something New in Peru
Pepsi Refresh
Mitzvah Bandz Bags
Another Parshas Vayeitzei

Hi All,
I've wanted to write for so long but my mom is always like, "We'll do it as soon as we finish this or that."  Today, she's like, "I can't do this or that until we write a Caringbridge update."

I'm going to start with the most pressing "issue".  It's not really an issue - it's more of an event, but almost like a crisis even.  Mom says it's another twist in the story of my life, another bitter sweet occurrence.

My new Tay-Sachs friend - who I haven't even met yet - had an experimental procedure in Peru this past month.  It's sort of created a frenzy and so many of us are just waiting and watching with bated breath.  Her dad put up a 50 second video of a physical therapy session done two weeks after the procedure and he received 700 emails and facebook messages that very day.

The video is on Keith Brown's facebook site and in his online journal on - the Progress After Peru page.  The video is of a 14 month old girl lying on her belly on an exercise ball. The therapist rolls her up and then down on her feet. (I've done that tons of times.) But when she gets to her feet, she stands, and with the therapist's hands on her hips and waist only, she lifts her head high, straightens up her back and looks around.  My mom cannot even write this without crying.  She's watched the video a miliion times and seeing my friend stand tall just chokes her up.  She doesn't even know how she feels.  It's just a feeling of overwhelm.

Why is that such a big deal? Because 2 weeks ago, before the procedure, she couldn't lift her head; it would just flop like mine does.  It's such a dramatic change in such a short time, it's impossible not to feel the miraculous nature of this treatment.

Well, my friend's name is Aspen Grace Brown and she's my neighbor in Alabama.  I don't think she knows she's my friend yet, but she is.  The procedure she received was an injection into her spinal canal in the lower lumbar region(near your buttocks).  The injection consisted of a cocktail of

               -Neuronal (Brain) Stem Cells
               -Brain Derived Neurotrophic Factor
               -Glial Derived Neurotrophic Factor
               -and Nerve Growth Factor

The cocktail gets into the spinal canal and circulates all around.  It eventually gets up to the ventricles in the brain and circulates all around up there, and then it gets into the brain itself.

The article that discusses it (Mom posted it on Project Shui on the "Something New in Peru" page) calls it a "Stem Cell Gene Enzyme Treatment" -- sounds like a combination of everything under the sun.

The stem cell injection into the spinal canal is different from the stem cell transplants that my other friends received. This injection does Not require any immunosuppression, and there is apparently no rejection.  The stem cells in this procedure are already differentiated into Neuron Stem Cells (a Neuron is a Brain cell); they go mingling with the other brain cells right away.  The cocktail also includes different Neurotrophic Factors (helps the cells grow) and nerve growth factor, which should enhance the effects of the transfused cells.

In a stem cell transplantation, the cord blood stem cells are transfused into the patient's blood, but only after the patient receives a ton of immunosuppressant medications, making them highly susceptible to infections.

In this new procedure, the patient is administered two of these injections a couple of days apart. Then they get on the plane and go home.  They do require follow up "booster" shots of the stuff, though.

Aspen was also given some oral agent that is supposed to increase levels of Hex A, the enzyme that Tay-Sachs kids are missing.  Mom is kind of skeptical about this medication.  It wasn't specified in the article, and up until now, enzyme replacement has not been realistic for various reasons (enzyme too large to pass through the tight blood-brain barrier...).  We'll have to see about that part. But if there is a Hex A pill out there somewhere, send some to me!  I don't mind if you have to smuggle it into the country.  Just stick it in a bottle of Miralax or something like that.

This procedure is done in Peru because it is not FDA approved.  The company/doctors that do it are StemCellRegenMed, Inc. Take a look at their site.  They're doing this stuff for many different diseases, including ALS, spinal cord injuries, diabetes, and even autism.  I don't know how it works for those illnesses, and as far as I was able to research, most patients are only a few months out from the initial procedure.  I think the cost is $30,000, but Mom's not sure - check the HelpForAspen web site.

Mom says she's on edge and is now flooded with emotions from every direction. 

Just a quick blurb about Pepsi Refresh.  Pepsi gives away tons of grant money every month for a number of different causes - see  Mom is gathering together the information, videos, pictures, etc. in order to apply for a grant for the Cure Tay-Sachs Foundation.  She's going for the December grants.  If a grant is not accepted, they apparently help you out with your application and web page so that you can resubmit the proposal the next month.  Mom's going for the gold - the $250K grant - at least this time around.  They also have $5K, $10K, and $50K grants.  Mom urges you take a look at the site.  Whether you want to raise money for Tay-Sachs, or for any other project, this is a great place to go.  There are applications in different categories. Animal stuff (rescues ...) are at the tops of most lists.  There are applications for various illnesses, for school programs, for community programs. Take a look.

When an application is accepted, it is posted on their site and people have to vote for your program.  This will be the big stumbling block b/c the Tay-Sachs community is so small.  We'll get to that later.  Anyway, when the time comes, one can vote for the same project once a day, every day (I think you can vote for more than one project each day).

We will definitely let you know how this unfolds.

Mom has packaged up some 1600 "Mitzvah Bandz Bags" that I'm giving to the Jewish Day School students in Atlanta. It's in celebration of my 8th Birthday, and each Bandz Bag has a pack of Silly Bandz in them.  I'm not soliciting donations for this project, just prayers.  Mom says that prayers from school-aged children are extra special.  Mom missed my birthday deadline.  She thinks we'll go with the start of Chanukah to give them out; that week coincides with the week I was diagnosed with Tay-Sachs 7 years ago, so it's kind of extra meaningful.

Anyway, we have an extra 80 packs of Christmas-themed Silly Bandz that we will give to my Tay-Sachs friends after we're done with all this.  Mom didn't think the Jewish Day Schools would appreciate that theme.

I have to go right now. Yoni is forcing Mommy off the computer.   I'll try to finish up this entry tomorrow.


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