Experimental treatment amazes

"A severely disabled girl who travelled to Mexico for experimental stem cell treatment has made incredible improvements within days of the surgery, her family says. Jaide Pearson, 8, has a new sparkle in her eye. Her family say she can now hold her head up and sit unaided in a car for the first time in her life. Jaide is a blind quadriplegic with epilepsy and cerebral palsy. Her Levin family raised $43,000 to take her to Fernando Ramirez's Tijuana clinic a month ago, hoping for a miracle cure. The treatment is banned in New Zealand and most other countries. Neurological specialists here warn it is still experimental and may result in "unwanted neurological functions".

But Jaide's family has been amazed by her improvement. "She used to sit with her head dangling on her chest in her wheelchair. She can now hold her head up, it is wonderful," Jaide's grandfather Norm Pearson said. With more strength in her torso the family no longer had to rely on a special car baby seat – she could sit normally on the back seat and her seizures were not as frequent or severe, her mother, Jayne Pearson said. "Her eyes are clearer and brighter, she is more vocal making vowel-type sounds. She seems less baby–like, I am just rapt with the improvements. I sneak into into her bedroom at night to spy on her and think, `what is she going to do next?' it is so cool."

More and more we are hearing about families fund raising to obtain this expensive treatment in Mexico to treat children with developmental disabilities, along with adults with various conditions from Motor Neurone Disease to Spinal Cord Injury and often there are claims of almost miraculous cures. With a steady flow of people and claims of hundreds of people being helped should have produced good quality case studies instead of anecdotal claims ranging from improvements to complete recovery, but these don't seem to be appearing. There are several reasons why even totally ineffective treatments seem to work, from ignoring the natural course of a disease or a disability, the placebo effect, cognitive biases such as confirmation bias which lead any change to be attributed to the treatment or crediting the improvement to what the person wants it to be, not what it really is. It's very hard to tell from the subjective descriptions whether there is any real improvement in condition and it's not apparent that any objective measurements of baseline condition were taken to identify whether or not there was any actual improvement that can be attributed to the stem cell treatment rather than any other factors.

At this stage, outside of some treatments such as bone marrow transplants which have been used for years and some promising findings for neurological disorders such as Parkinson's disease these treatments are experimental and are not recommended as a medical treatment. Stem cells have huge potential for treating a range of conditions but the first principle should be 'do no harm' and these treatments being offered in Mexico are sheer quackery and have the potential to damage a very promising area of science. They've bypassed regulation and the basics of testing the treatment and gone straight to delivering the treatment in humans. Willie Terpstra is one example of the caution that needs to be taken when hearing of these types of miracle cures. Almost immediately after treatment, there were claims of instant improvement which was unusual considering that even if the treatment was expected to improve the condition it would take not hours or days but weeks or even months for it to work. This apparent improvement was short lived, and she died some twenty months after receiving the treatment. If nothing else, the sheer cost of a failed treatment in the region of $40,000 to $100,000 should cause food for thought. What's worse, if anything, is that the people that have travelled or are going to travel to have the treatment seem to be under the illusion that even if it doesn't help, it won't do any harm. That doesn't appear to be the case and offering the treatment without any reasonable expectation of it helping and without an quantification of potential risks means it simply isn't possible for people investigating the treatment to make any kind of informed decision as to benefits and risks. It's not really going to help much if children undergoing these treatments develop tumours and other effects at a later stage.

A follow-up article by the Paediatrics Society of New Zealand states there is no proof of benefit.

"...Society president Rosemary Marks said the therapy was "highly experimental", with no controlled studies to back claims of dramatic improvements. Her advice to parents is "don't go there". A society information pamphlet on stem-cell treatment was being finalised for release on the kidshealth website. A draft had been sent to doctors.

It said the stem-cell operation carried risks of introducing viral diseases or bacterial infections, and even malignant tumours developing after the transplant. "Our advice is that stem-cell treatment for children with cerebral palsy is unproven and has potential hazards. Therefore, we do not recommend it," it said. The treatment for cerebral palsy involved injecting adult stem cells into the bloodstream or an area around the spinal cord. In theory, these would develop into nerve cells that would take over the function of damaged areas of the brain. "Most centres offering this treatment also follow the treatment with intensive physical-therapy programmes," the pamphlet said. "It is very difficult to know whether improvements are the result of the stem-cell treatment itself, or the intensive physical therapy, or are the result of the child growing and developing."...

....Christchurch School of Medicine researcher and paediatric oncologist Michael Sullivan wrote a review of cord-blood storage for Nature magazine last year. While the benefits of using cord blood to treat cancer had been proven, there was "no evidence" that using your own cord blood had benefits, he said...Sullivan said families were being exploited by a stem-cell industry that was "nothing more than alternative therapy masquerading as genuine medicine". While the possibilities for stem-cell treatments were huge, they were still some years away, he said.

I'm not sure whether the caution of the Paediatrics Society will be heeded, but hopefully some will take notice. It can take years for a treatment to be introduced for medical applications. For those who might potentially be helped, this must be an incredibly hard wait but jumping into using uncontrolled experimental treatments in countries that have lax regulations isn't the way to go.

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Blame it on George

I know it might sound slightly ridiculous, but how much of this kind of quakery do you think can be blamed on America?
The patently stupid ideas of the last administration have put real stem cell research 8 years behind where it should be, and opened the doors for the kind of therapies that can now be accessed only in developing countries.

Perhaps if Dubyah and his right wing religous cronies had not been so blinded by "pro life" objectors this poor child from Levin could have benefited from some real stem cell therapy with proven results.

I guess we will never know.

I am a parent and I know that if my child was sick I would want to do anything to help them, this would make me an easy target for con-artists, and bogus therapies. Though I hope I would be able to think rationally about what is best for my family.

I really feel sorry for the parents, and I truly hope that this treatment will provide some real benefit for them.

I can understand the

I can understand the desperation myself, but this is being used wrongly and in situations where people are unlikely to benefit and it's simply exploitation to give them false hope. I've heard of it being used for genetic conditions and even with all the will in the world stem cells cannot replace someone's defective DNA in all their cells. In other cases, such as the Cerebral Palsy children mentioned in the article, I'm fairly doubtful about this helping at all either as my quick scan of the research into the use of stem cells for strokes and spinal injuries only involved treatment at the time of acute injury which meant that the cells could potentially work on the injured area preventing damage. In these children, the injury happened many years ago and it's very unlikely IMO they will work on dead tissue.

From what I understand of the situation in the USA, it was that George W. stopped experimentation of new stem cell lines so scientists were limited to use of existing lines so it didn't grind to a complete halt. Internationally, other countries have differing policies so there were other experiments going on so even that development didn't prevent all research.

The difficulties that currently being faced in doing this research are immune compatibility (may have been solved), differentiating the cells into the appropriate tissues. purifying the cells so that undifferentiated cells aren't accidentally transplanted, preventing tumour growth in the transplanted cells and proper delivery of the cells so they perform a useful function and survive in the host (at the moment they would just be as likely to go to work on a cut on your finger as the brain). There's already one example of tumours developing which I linked to, which exactly what you'd expect given that science still has so much to learn about stem cells and how to control them so they do what we want them to do - http://www.wired.com/wiredscience/2009/02/stemcelltumor/

Emotions over-ride common sense

You are right of course, I know that this will probably never work, but like with so many other quack therapies the emotional side of a sick person will beat out the rational side.

I was kind of being facetious about W, I know he didn't stop the research altogether but it is a good example of how the religious right can influence politics in the US.

There was a great epiosode on Media 7 about stem cell research in the media, you can view it here.

http://sciblogs.co.nz/terms/2009/10/09/media-7-on-experimental-stem-cell...

I did read about the boy who developed tumours after stem cell therapy, it seems like it should happen more often if you are placing undifferentiated cells into your body and allowing them to replicate. I think the research has a long way to go before it can be used as a 'therapy'. Its use in growing human tissue for transplants seems to be much closer to fruition.

http://sciblogs.co.nz/fisheye-perspective/2009/10/11/bioengineered-perso...

I really love the sciblogs site, its great the we finally have something like this.

Absolutely, you do want to

Absolutely, you do want to hope that there is something out there. I don't blame any of the parents for thinking as they do, but I'd love to shut down those clinics just shoving stem cells in and hoping it will work. My fear is it's going to do a lot more harm than good in the long run.

Imagine if all of that money went into doing a proper clinical trial to assess whether there is any realistic hope for this being an real treatment for these children. Instead it's just a waste because they aren't being followed up at all.