Fwd: [StemCells] Digest Number 91

From: Max More (max@maxmore.com)
Date: Sun Feb 18 2001 - 12:42:24 MST

Cases like this one, if known about more widely, would help the acceptance
of stem cell research...


>Date: 18 Feb 2001 10:19:41 -0000
>From: StemCells@yahoogroups.com
>To: StemCells@yahoogroups.com
>Subject: [StemCells] Digest Number 91
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>There is 1 message in this issue.
>Topics in this digest:
> 1. Therapeutic Cloning is a 5-Year-Old Girl's Only Hope
> From: DLewis0991@aol.com
>Message: 1
> Date: Sat, 17 Feb 2001 06:03:35 EST
> From: DLewis0991@aol.com
>Subject: Therapeutic Cloning is a 5-Year-Old Girl's Only Hope
>From: http://abcnews.go.com/sections/2020/DailyNews/2020
> Therapeutic Cloning is a
>5-Year-Old Girl's
>Only Hope
> By Thomas E. Goldstone
> Feb. 16 — When Kelly
>Rich turned 1, her
>parents Anne and Gordy started to notice she had not reached some of those
>important milestones that new moms and dads are waiting for. She was late to
>crawl, even later to walk, and never was able to speak.
> They grew concerned — even more so
>when, by the time
>she turned 18 months, she was still lagging far behind. So they brought her
>to the Cleveland Clinic where physicians were stumped, until they noticed
>that she was wringing her hands together — a possible indication of a
>little-known neurological disorder, Rett Syndrome.
> In 1997, it was confirmed that Kelly
>did in fact have
>Rett Syndrome, a multi-symptomatic disorder causing everything from scoliosis
>to seizures, from breathing problems to gastrointestinal trouble. A
>discriminating disorder, it affects only girls because it is caused by a
>mutation on the X chromosome.
> "At the time Kelly was diagnosed,
>there was no known
>cause and no known cure," says Gordy. "So we were given this diagnosis which
>seemed like a life sentence."
> 'Locked Box'
> Like Kelly, most girls begin showing
>symptoms between
>the ages of 6 months and 18 months, seeming to their parents to be in daily
>pain and constant mental distress. Maybe worst of all, they are completely
>unable to communicate; they cannot speak or sign, write or signal.
> "We knew that there was something
>there," says Gordy.
>"You could look in her eyes and see that something was going on there. She
>wanted to be with us." They could see that Kelly wanted to get out of this
>"locked box" that some twist of fate had put her in. And they were not going
>to give up until they set her free.
> They turned to the Internet. But there
>was not a lot
>to find. Rett syndrome had only been first diagnosed in this country in early
>1980s; until then, it was often misdiagnosed as either autism or retardation.
>Even today, many think thousands, if not hundred of thousands of girls
>continue to be misdiagnosed.
> One of the things they did learn on
>the Internet and
>from their doctors was that therapy was a way to help Kelly try to overcome
>the challenges of living with a brain affected by Rett syndrome. They do
>physical therapy to keep her muscle tone up and her bones strong.
>Occupational therapy helps her try to learn the motor skills she lacks, and
>music therapy seems to bring an otherwise unseen calm over her.
> The brain of a girl with Rett syndrome
>has very
>tightly packed neurons that are firing at double the rate of a normal brain
>and are firing constantly, never resting like they do in normal brains.
>Because the neurons in girls with Rett syndrome fire so fast and so
>ceaselessly, they essentially burnout before the girls are teenagers. After
>their neurons begin to burn out, most girls afflicted with Rett enter a
>period of gradual deterioration that leads to premature death.
> Hope for a Cure
> The Riches were determined to beat
>this disease
>before it beat them and their daughter, but they were told there was little
>treatment beyond therapy. Until, that is, they heard of an amazing discovery
>at Baylor University in Texas. Scientists there isolated the cause of Rett
>syndrome to a defect on the MECP2 gene.
> "We were extremely overjoyed that the
>gene had been
>found," says Gordy. "We were searching for the needle in the haystack. Once
>we found the needle, we can start to sew. We can fix it."
> Armed with the knowledge of exactly
>where the
>mutation lies, Dr. Robert Lanza of Advanced Cell Technology (ACT), thinks his
>company might be able to help Kelly. ACT is focused on what is called
>therapeutic cloning — taking one's DNA and using it to grow an embryonic
>clone in order to make what are essentially "spare parts" that can replaced
>damaged ones. Lanza says that diseases like diabetes or Alzheimer's might be
>easier than Rett syndrome to fix using these therapeutic cloning methods, but
>he holds out hope that ACT will someday be able to help Kelly and her fellow
>Rett patients.
> Banking on a Clone
> Simplified, the process — which is
>still in the
>research phase — might work something like this in Kelly's case: using
>sophisticated gene therapy techniques, Kelly's mutated DNA would be "fixed,"
>essentially removing the mutations that causes Rett. The "fixed" DNA would
>then be put into a de-nucleated donor egg, where the original DNA has been
>removed. This egg would then be coaxed into dividing as if it were an egg
>that had been fertilized by a sperm to then be the beginning of a new human
>life. What it is at this point is a embryonic clone of Kelly, but this time
>without Rett syndrome. The cells in this days-old clone are called stem cells
>and they have the miraculous potential to turn into anything. In the lab, the
>scientists at ACT would hope to be able to turn these stem cells into new
>neurons that could replace the defective neurons in Kelly's brain.
> It sounds like science fiction, but
>for their little
>girl's sake the Riches are banking, literally, that it becomes "science
>fact." Gordy sold a business and invested the money in ACT and the Rett
>Syndrome Research Foundation, which he started to fund researchers doing
>biomedical work on the disorder.
> He knows, however, that he is racing
>against the
>clock. "I think we have a 5 to 7 year window, so we are doing everything we
>possibly can to fund as much research, to get as many researchers involved so
>that in this window, hopefully some cure will be found," says Gordy.
> "When I look in her eyes," he says of
>his 5-year-old,
>"I see a person and I want to be able to communicate with her. I want her to
>say 'Goodnight Daddy.'"
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