20/20 Tonight (ABC 10 PM EST) is on Cloning, mainly Therapeutic Cloning. Robert Lanza from Advanced Cell Technology is interviewed advancedcell.com
The show also discusses 'briefly' research at Boston Children's Hospital and their work on creating human organs.
Plus, there is a strange group in Canada- almost a cult- Aurelians (sp?) who formed the company Clonade which is well funded with private donations; and they are determined to clone a human.
abcnews.go.com
Cloning as Cure
— 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.'" |
