 Daniel Kerner, 7, and dad Marcus Kerner after a physical therapy session at Doernbecher Children's Hospital. (Photo by Ross William Hamilton) |
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Posted on 06/25/2007 9:05:49 AM PDT by Incorrigible
Stem Cell Treatment Battles Deadly Destroyer Of Brains
By ANDY DWORKIN
Daniel Kerner, 7, and dad Marcus Kerner after a physical therapy session at Doernbecher Children's Hospital. (Photo by Ross William Hamilton) |
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PORTLAND, Ore. — Doctors are halfway through a dramatic medical experiment that injects stem cells inside the skulls of children dying from the brain-destroying illness Batten disease.Since November, three children have been treated at Oregon Health & Science University with stem cells purified from fetuses. In animal tests, the cells made enzymes that Batten disease victims lack. Scientists hope for the same in people.
The trial's sponsor, California-based StemCells Inc., is limiting information on the results so far. But an independent safety committee reviewed the cases and let the trial continue.
"The fact that they've looked at the (data) does not mean that there have not been any serious adverse events,'' said Rodney Young, chief financial officer of StemCells Inc. Passing the review does mean no problems have been serious enough to stall the trial, which is mainly meant to judge the treatment's safety. Full information on safety and possible hints about the treatment's effectiveness won't be available until after the trial ends in late 2008 or 2009.
Dr. Nathan Selden, the neurosurgeon at Oregon Health & Science University's Doernbecher Children's Hospital who performed the operations, said doctors are thrilled to be "halfway through the trial, and extremely pleased that the monitoring board has validated the ongoing safety of the trial.'' Selden and Young declined to discuss the three patients treated so far.
The father of 7-year-old Daniel Kerner, the first child treated, said none of the three children has had serious problems related to the treatment.
Daniel "is getting better and stronger,'' said Marcus Kerner, an assistant U.S. district attorney in Santa Ana, Calif. "We know the other kids. They are the cutest kids you could possibly imagine, and they are doing wonderfully.''
"At this point, the way things are going, it looks like all the kids are going to make it,'' he said.
The stem cell study is the model of high-stakes science. Study subjects are the most vulnerable imaginable: Children with an incurable, deadly disease that leaves them unable to fully understand their treatment. In Batten disease, fatty substances build up in the brain, killing nerve cells and sapping a person's ability to see, speak, think and move. Affected children generally develop seizures and die by their teens.
The treatment is risky: StemCells purifies cells from the brains of aborted fetuses donated for research. The cells are injected through eight holes in the skull in a long surgery, whose risks include bleeding in the brain and infection from drugs used to control the immune system post-transplant.
Experimenters are moving into the trial's second phase, which will treat three different children with a higher dose of stem cells. They declined to say when the next surgery is scheduled but hope to finish all three this year.
Kerner gladly discusses what he calls "a miracle that's continuing to unfold.''
Before surgery, Daniel had lost much of his ability to speak and move. Soon after, his parents noticed language improvements. He said "Dad'' for the first time in two years. The other night, Kerner said, after watching the movie "Brother Bear,'' Daniel said, "I'm a bear.''
Daniel has gained enough leg strength to stand for more than a minute, using his hands to steady himself. "His legs are so strong that he was able to break the straps off the foot pedals'' on his wheelchair, Kerner said.
The changes have not all been dramatically good. "Before the surgery he was seizure-free,'' Kerner said. "Now, depending on how we adjust his medicines, he has minor issues with seizures. But nothing major.'' A weeklong follow-up visit at Doernbecher Children's Hospital last month showed "no problem at all associated with the stem cells,'' Kerner said.
Between doctor visits, Daniel leads a busy life. For his seventh birthday in January he went biskiing, schussing the slopes with guides in a sort of wheelchair on skis. He finished second grade with his special education class this month.
"It is like he's growing along with the cells,'' Kerner said. "We know there's a long road ahead, that you don't wave a magic wand over a killer disease and make it evaporate. What you do is what these kids are doing ... . These kids are fighting as hard as you can possibly believe.''
(Andy Dworkin is a staff writer for The Oregonian of Portland, Ore. He can be contacted at andydworkin(at)news.oregonian.com.)
Not for commercial use. For educational and discussion purposes only.
This would be the first successful use of fetal stem cells I've heard of if indeed this study completes without adverse affects. Wonder if there is a similar therapy for Batten disease using alternate stem cells.
Bump
However, it may have only been a matter of time before this obsticle was resolved, so now the real focus, as it always must be, is whether it is ok to sacrifice the weak for the benefit of the strong. This debate has parallels to Falun Gong members in China being made into involuntary organ donors for wealthy Asian and Western patients.
How long before the tumors appear?
Trying to put a “face” on Embryonic Stem Cell use. It will probably work for that goal.
I want to see it in a peer reviewed journal. How did they expand the cell line? This sounds very much like a corporate leak to engender enthusiasm. Sorry for being a curmudgeon.
There probably is, but you and I know that this demonstration isn't about using the best therapy to cure the disease.
Why does the title say “embryonic”, when the article repeatedly discusses stem cells from fetuses (which are “adult” stem cells).
Aren’t fetal stem cells different than embryonic stem cells...?
This is a "bait and switch" with the stem cell names. Fetal stem cells aren't embryonic stem cells. They are younger adult stem cells.
Sooo, one child is killed and her/his body parts are removed to give to another child.....
(Even so, Lord Jesus, come quickly...)
StemCells, Inc. is focused on the discovery and development of stem cell therapeutics to treat damage to or degeneration of major organ systems such as the Central Nervous System, Liver and Pancreas.We are a world leader in the discovery and development of human neural stem cell technology using cells derived from adult (i.e. non-embryonic) brain tissue.
We use a proprietary process to isolate, purify and expand rare candidate stem cells found in adult human tissue.
Sorry, my error for thinking they were synonyms.
You are correct, these cells are from aborted fetuses which is different from embryonic stem cells grow in a lab from embryos.
Perhaps admin mod can replace “Embryonic” in the title with “Fetal”.
Obviously, private embryonic research is not illegal, though the morality is in question. Creating embryos specifically for this research seems very unethical and morally wrong. However, if an unwanted embryo must be destroyed, then I would prefer that its loss not be in vain.
I certainly don't blame you. I'm always switching related terms. My whole family does that.
I grew up in a family where the television was called "the machine." ;-)
How do stem cells battle the destroyer of brains? (public schools)
I wonder if it is legal to transplant body parts of executed criminals.
Frankenstemcells
which is why it’s not enough just to not use federal funds. It must be banned completely.
The brain is blood-free, so rejection is not a problem. But the last time they tried injecting foreign stem cells into brains, the results were pretty terrible. They have no idea of what the long-term effects will be, and they are likely to be terrible for many.
Neural Tissue Transplants
http://www.caregivers.com/Category_Pages/document_display.asp?Id=12125&;
In the 1980s, neurosurgeons in Sweden and China transplanted tiny bits of aborted fetal brain tissue into the brains of patients with PD. The idea was that if these cells “took” the PD patient would be able to produce more dopamine. However, success of this procedure has been slow in coming, hampered by ethical and moral issues; the controversy is over the use of human fetal tissue for the implants. A Federal ban, lifted in 1993, will probably resume under the new US administration. If this happens, funds for this research may no longer be available from government agencies.
Results released this year from the first randomized, controlled clinical study of fetal implants for PD patients show that the surgery only helped a small number of patients under age 60. This was a study led by Dr. Curt Freed from the University of Colorado in Denver and Dr. Stanley Fahn of the Columbia-Presbyterian Medical Center in New York. Their study included 40 patients with advanced PD present for an average of 14 years. After one year the treated patients under age 60(nine in total) showed some improvement especially in movement. However, no patient over age 60 or the placebo group showed improvement.
The most shocking finding was that in 15 percent of patients, the cells grew too well resulting in too much dopamine produced in the transplant patient’s brain. As a side effect, these patients developed uncontrollable writhing and jerking movements, known as dyskinesias with a severity rarely seen. The scientists say they have no way to remove or deactivate these transplanted cells so this nightmarish side effect may be permanent.
Although some scientists are eager to proceed, the results of this study, plus the dispute over ethical issues surrounding it may mean that the grafting of human fetal tissue into the human brain may no longer be considered a safe, viable option for treating PD.
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