Dear patients, instead of writing my own comments to prove how effective stem cell transplants can be, I prefer to let you see the information below and estimate whether it worths or not. Here are some facts:
Tucson, Arizona | Published: 11.28.2007
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A top scientist using stem cells from human embryos to cure disease and repair injuries will proceed with his work, he said in Tucson on Tuesday, despite a recent breakthrough showing the controversial embryos may no longer be needed.
That blockbuster breakthrough was announced last week by researchers in Wisconsin and Japan, who have discovered how to genetically program human skin cells to behave like embryonic stem cells.
That means the skin cells can develop into any cell in the human body in the brain, heart, liver, muscle or bones where they potentially can be used for lifesaving repairs or cures. Until now, only stem cells from human embryos each a potential human life could do that.
But scientists deeply involved in human embryonic stem-cell research are unlikely to scrap years of work, however controversial, to start over with the new skin-cell technology, said one noted for cutting-edge achievements in this field.
"I do think a great deal of this work could be done with the skin-cell-derived stem cells. But we'd have to start completely over, from scratch, and we are not going to slow down to do that, not at this point," said Hans Keirstead, a neurobiologist and stem-cell researcher at the University of California-Irvine.
Speaking at University Medical Center, Keirstead outlined his progress, which has taken off in the past three years after California sidestepped President Bush's ban on federal funding for research using human embryos.
In 2004, California voters overwhelmingly passed Proposition 71, allocating $3 billion in state funds to develop new embryonic stem-cell lines, attracting some of the best and the brightest researchers in the world to relocate there.
Since then, Keirstead and his team at the Reeve-Irvine Research Center (named for the late actor Christopher Reeve, who suffered a devastating spinal-cord injury) have used these new stem-cell lines to restore full mobility to rats paralyzed by spinal cord injuries.
To do that, he had to find a way to purify and differentiate human embryonic stem cells to develop into a type of nervous-system cell needed to restore spinal cell function. He is using similar techniques to transform embryonic cells into motor neurons to restore muscle function.
These breakthroughs have created a blueprint that can be used to create cells to replace those damaged by such human scourges as multiple sclerosis, Parkinson's disease, heart disease, stroke, ulcerative colitis, muscle atrophy in newborns, Lou Gehrig's disease and diabetes.
Embryonic stem cells taken from 5-day-old embryos discarded by fertility clinics "have the potential to address every single human disorder," Keirstead said. "They are the greatest single scientific advance in human history, with the potential to develop into any kind of cell in the human body."
Keirstead's work is expected to set the stage for the first human clinical trial in the world using embryonic stem cells, possibly as early as next year.
Only 40 years old, Keirstead is as passionate about defending the use of human embryos for this purpose as he is for the work itself.
"The ethical controversy starts with the fact that if the embryo were implanted in the uterus, it would develop into a human," he said. "But these embryos were destined to be destroyed.
"It is my personal feeling it's a very ethical decision to use this tissue to end human suffering, to better human life, than to destroy it.
"If you think this is a divine entity with tremendous spiritual value, I don't disagree with you. I value them no less."
Keirstead's unwillingness to abandon progress in embryonic stem-cell research, to begin again with non-controversial human skin cells, actually is shared by the skin-cell researchers at the University of Wisconsin.
They've warned that work with embryonic stem cells must continue, that their research remains in its early stages and that comparison studies must be done to ensure the genetically engineered skin cells do not behave in unexpected ways.
There also are concerns that the techniques used might disrupt human DNA and trigger cancer in patients questions that must be answered before they can be approved for human experiments.
He says that in his lab they focus on acute, sub-acute, and chronic. (Hans Keirstead, for those who haven’t met him, is kind of Robert Redford type. I’m just sayin’.) He’s describing an animal whose cord had been cut exactly 50%; within days it has lost a lot of tissue because of a “bad guy” molecule. A single injection — of a restorative molecule –that could be given by any nurse — gets rid of a lot of the secondary degeneration.
There’s a video taken from underneath a glass table top on which a rat is dragging itself along — a single dose of the molecule prevents a LOT of the secondary damage.
The technology to do this is in clinical testing –Medarex began clinical trials in 2006 for ulcerative colitis . . . the Keirstead approach is to find other (more profitable) diseases that studies can get funded for, then piggyback onto what’s learned and done in the process to bring the therapies to sci.
If Hans can get 1.5 million dollars, Medarex might provide him with $3 million worth of product to run a trial of his own
Subacutes– different beast completely from the acute. Wow, he puts up a picture of a human egg sitting in a fallopian with a tiny blue speck of sperm heading its way. Wow.
We’re going to talk about how to grow human embryonic stem cell lines just for clinical use. The job is to be able to do that with certainty that they’re pure, and then be able to grow them into whatever you wanted. Aborted fetuses are not a source for this for ethical, political, and practical reasons.
What if we had limitless sources of something like human heart tissue,not to grow new hearts, but to try out things that might
2 guys and a rat show up at the FDA and say, we’ve made a rat walk again . . . the FDA says, so what. Do it again, under our regulations.
OEG’s are the cells that make myelin, the conductor that makes transmission of information through the cord possible.
He cooked up a soup to make pure OEG’s . . . it’s cooking time is 42 days. They did this by simulating the media in which our own brains create these things. This is the only place in the world where a pure and inexhaustible source of any kind of cell has been developed.
Hans just radiates confidence.
Geron is going to submit their IND to the feds this summer and they plan to go to clinical trial right away. They went to the FDA and said they’d treated thoracic injuries in rats and the FDA said, well you can’t test humans with cervical injuries until you repeat the work with cervical injuries. Jeebus.
About safety . . . think about inserting a bolus of oeg’s into a cord . . . could be a problem. So they did a test of a very, very tiny, light little injury and did their transplant on that one. The injury was so small that it didn’t even cause deficit in function, and the insertion of oeg’s didn’t hurt anything.
Geron’s safety studies have done very well . . . except we only know this because the CEO of the company stood up in front of Reuters to say that he’s going after an IND (federal permission) to begin trials. (Wahoo! No wonder Hans is so smiley.)
The clinical trials for this will happen this year.
Is it possible to create an inexhaustible source of motor neurons out of human embryonic stem cells? He’s talking about repairing muscle groups one at a time . . . the thumb-mover, for example.
“We can grow one to 10 billion cells a week of these things. . . any one human is only going to take a few million.”
After 6 weeks, the cells display markers just like they should — meaning just as they would display if they were motor neurons that came into being in the usual way.
The true test, though, is to see if your motor neuron does anything with muscle. Myoblasts can be cultured from a biopsy . . . a hunk of your own muscle. We culture them together and omg I just saw a video of a hunk of myoblast throbbing under the influence of motor neurons grown from stem cells. “It doesn’t just look like a motor neuron, or smell like a neuron, or talk like a neuron . . . it is a motor neuron.”
How do you get those motor neurons to grow fast? They’ve created a substance that works. He doesn’t name it, just refers to it as an “attractant.”
Two months after transplantation . . . here’s a slide that shows –2 months after transplation — that the motor neurons are bundling themselves together and heading out to get to work. And–holy freakin’ smokes– he’s got rats that were 6 months post injury . . . he gives them the “attractant” shots into their non-functional limbs, leaving one limb untreated. The video shows the rats clearly able to use the treated limbs while the untreated one drags along.
The strategy to use this treatment starts with type 1 infantile SMA, followed by terminal ALS, followed by chronic sci. The reason to test other populations first is that the window is very, very quick (the patients are terminal); if he began with chronic sci, it would take a lot longer.
The list of challenges to clinical translation has 10 items on it, and the last one is the isolation of academics — we need to engage industry, the FDA, the government. Whose job is it?
We’re way thrilled here, people.
He’s saying that he’s learned a lot from the sci population about what it wants. Well, obviously complete reversal is the goal in the end. But should we ignore what could be accomplished along the way? They’re going to use their “attractant with motor neuron” therapy to target a single muscle . . . then move on to another one.
The oeg’s and motor neurons that will be part of the treatment do not come from our own bodies . . . which means that those who use those treatments will have to be on lifelong immunosuppression– which carries some issues and is undeniably a detriment. Which is why they’re working ways to use our own tissues to create the cells that can fix what’s wrong with us.
Yikes, I hope I captured the gist of that, because it was quite amazing and delivered at warp speed.
Spinal muscular atrophy (SMA), the number one genetic killer of children under the age of two, is an often fatal disease that destroys the nerves controlling voluntary muscle movement, which affects crawling, walking, head and neck control, and even swallowing.
WHO IS AFFECTED SMA is one of the most prevalent genetic disorders.
One in every 6,000 babies is born with SMA.
SMA can strike anyone of any age, race or gender.
One in every 40 people carries the gene that causes SMA. The child of two carriers has a one in four chance of developing SMA.
7.5 million Americans are carriers.
THE TYPES OF SMA SMA Patients are classified into four types based on milestones achieved at onset of SMA. Type I and II are the most prevalent.
Type I, or Werdnig-Hoffmann Disease, is the most severe form of SMA. Type I SMA strikes infants between birth and six months old. Children affected with Type I cannot sit without support.
Type II affects infants between seven and 18 months old. Type II patients may be able to sit unaided or even stand with support. They are at increased risk for complications from respiratory infections.
Type III, also known as Kugelberg-Welander Disease, is the least deadly form of childhood-onset SMA. It strikes children as early as the age of 18 months, but can surface as late as adolescence. Type III patients are able to walk, but weakness is prevalent. Most patients eventually need to use a wheelchair.
Type IV is the adult form of the disease. Symptoms tend to begin after age 35.
SMA does not affect sensation and intellectual activity in patients. It commonly is observed that patients with SMA are unusually bright and sociable.
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