[paula_w]

I attended the Udall Centers Meeting in Pittsburgh last week. There was a NINDS meeting in the morning and then a two and 1/2 day meeting for the researchers from each center to report their work.

Needless to say it was fascinating but troublesome for all the reasons I've stated many times before. They are so interested in genetics now.....and how they interact. Cracking this code completely may be a mission impossible. They are doing fantastic work...but there are many genes........

The second most talked about subject was finding a better PD model. Old rats are hard to get. The MPTP model doesn't progress. Lab rats go for $ 8 -10,000 so remember that the next time someone asks a trial participant to pay for ANYTHING.

This was a surprise. i discovered the second day that the man sitting next to us was Heiko Braak. He spoke, but I must confess a lot of it was over my head.....

Ron, I asked one question on the last day after working up the nerve. it was about the blood brain barrier. Braak wasn't in the room. They all reacted much like Todd Scherer at FOX. Saying that not much had been done about it but they didn't discredit it. Then afterward, a member of Ole Isacson's team from Harvard came over and said in a nuclear stain test they did watch leakage into the substantia nigra, but that it isn't looked upon that favorably as a theory - he didn't know why.

I have the book with all the abstracts and will try to plug them in as subjects arise.

Then I was off to Joan Snyder's Shake Rattle and Roll. Had a lovely time - great bands! Joan knows her music. I met Robert [rd42] and his wife.......a lovely couple and Robert is way too young to have this illness....we must help his generation, it makes me sick to see such a good young guy like him have this damn illness.

Needed to add that the third issue to crop up very frequently was alpha synuclein.

more later,
paula

[rd42]

It makes me sick too

It was great to put a face to the name and great to meet you Paula.

Smoke 'em if ya got 'em...

Robert

[lou_lou]

dear paula,
so what happened to the great stem cell promise? gone with the wind.
because, it must be illegal to have a cure for people.
if you think that's alot of cash for rats, then ask
Greg G. at the U of Kentucky -how much old lady rhesus monkeys go for -

in the $100,000 category -? maybe higher now...
luv
tena
nothing new under the sun...

The Dangerous Promise of Gene Therapy
By Sophia M. Kolehmainen
Gene therapy has not yet proven successful in curing disease.
In the mid-1980's scientists began to extol the promises of gene-therapy. Conceptually (and if you consider the world only at the sub-microscopic level), gene therapy is a logical, straightforward solution to genetic disease: if a gene seems to be causing a disease, then to cure the disease scientists must remove the "bad" gene, and substitute or add a "good" gene. The reality is much more complex. Though more than three hundred gene therapy protocols, involving more than four thousand patients, have been approved for human trials in the United States, gene therapy has yet to fulfill its promise of curing any genetic disease.
Jesse died because of a gene therapy experiment.




Jesse: gene therapy gone wrong

On September 17th, 1999, eighteen-year-old Jesse Gelsinger died as a result of his voluntary participation in a gene-therapy experiment, becoming the first known human victim of this technology. Jesse's experience illuminates important elements in gene therapy that should make government agencies, scientists, and the public take the need to regulate and oversee this technology very seriously.

Jesse had a rare genetic disease, known as ornithine transcarbamylase (OTC) deficiency, which affected his ability to rid his body of ammonia, a usual, but toxic, breakdown product of protein. Half of children with OTC die in their first month of life, and half die before their fifth birthday. Jesse had a mild form of the disease because some of his enzymes were functioning normally. He was therefore able to control the disease with diet and drugs, though he needed to take 32 pills a day.
Ironically, Jesse would not have benefited from the experiment.



The therapy was intended to help babies with a rare genetic disease.







The experimental protocol for which Jesse volunteered had no chance of providing him, or any of the other volunteers, with any benefit.
It was designed only to test the safety of a treatment that would be used on babies with the fatal form of OTC.
The scientists who designed the protocol at the University of Pennsylvania's Institute for Gene Therapy, Dr. James Wilson and Dr. Mark Batshaw, believed that OTC could be surmounted with gene therapy.
They hoped to infuse babies who had OTC with genes that would help them produce the missing enzymes.
In order to get these genes into the patient's cells, Dr. Wilson developed a weakened cold virus (known as adenovirus) which was designed to enter the cells as any virus would, but, instead of delivering disease, it was supposed to deliver the corrective OTC gene.
Wilson and Batshaw hoped that the infusion of adenovirus and corrective genes could be used to reduce infant fatalities by controlling the high levels of ammonia in babies with OTC immediately after birth.




Doctors did not foresee serious side effects or fatality.
Wilson and Batshaw worked together to develop the OTC protocol and, in 1995, they submitted it to the National Institute of Health's (NIH) Recombinant DNA Advisory Committee (RAC) and the Food and Drug Administration (FDA) for review and approval, as is required for all human experiments involving gene therapy.

Jesse was deemed eligible for the study and assigned to the final test subject group--the group that would receive the highest dose of adenovirus. At the time, the researchers believed that in the worst case, the trial might result in an inflamed liver. On September 13, 1999 Dr. Raper injected 30 milliliters of the adenovirus with the corrective OTC gene into Jesse's bloodstream. According to the physicians, Jesse's severe immune system reaction led to multiple-organ-system failure and he died on September 17th, 1999, four days after the gene-therapy injection.

Some test animals died before human trials.



Some reviewers were concerned but the FDA approved the trials.

The safety of gene therapy

Human gene therapy trials raise the questions:
http://www.actionbioscience.org/biot...lehmainen.html

Originally Posted by paula_w

I attended the Udall Centers Meeting in Pittsburgh last week. There was a NINDS meeting in the morning and then a two and 1/2 day meeting for the researchers from each center to report their work.

Needless to say it was fascinating but troublesome for all the reasons I've stated many times before. They are so interested in genetics now.....and how they interact. Cracking this code completely may be a mission impossible. They are doing fantastic work...but there are many genes........

The second most talked about subject was finding a better PD model. Old rats are hard to get. The MPTP model doesn't progress. Lab rats go for $ 8 -10,000 so remember that the next time someone asks a trial participant to pay for ANYTHING.

This was a surprise. i discovered the second day that the man sitting next to us was Heiko Braak. He spoke, but I must confess a lot of it was over my head.....

Ron, I asked one question on the last day after working up the nerve. it was about the blood brain barrier. Braak wasn't in the room. They all reacted much like Todd Scherer at FOX. Saying that not much had been done about it but they didn't discredit it. Then afterward, a member of Ole Isacson's team from Harvard came over and said in a nuclear stain test they did watch leakage into the substantia nigra, but that it isn't looked upon that favorably as a theory - he didn't know why.

I have the book with all the abstracts and will try to plug them in as subjects arise.

Then I was off to Joan Snyder's Shake Rattle and Roll. Had a lovely time - great bands! Joan knows her music. I met Robert [rd42] and his wife.......a lovely couple and Robert is way too young to have this illness....we must help his generation, it makes me sick to see such a good young guy like him have this damn illness.

Needed to add that the third issue to crop up very frequently was alpha synuclein.

more later,
paula

[lou_lou]

http://actionbioscience.org/


http://www.gene-watch.org/programs/p...insurance.html

[lou_lou]

National Children's Study adds 22 centers

WASHINGTON, Oct. 5 (UPI) --

The National Children's Study announced
Thursday that 22 new study centers had received contracts for its survey of 100,000 U.S. children.
The goal of the study is to follow 100,000 children from birth to the age of 21, examining the role of genetic and environmental factors in children's and adult health.

"Today's announcement represents a milestone for the National Children's Study," said Dr. Duane Alexander, director of the National Institute of Child Health and Human Development. "The addition of new study centers will move the study closer to its goal of recruiting more than 100,000 children representative of the entire population of American children."

The 22 centers are located in 20 states across the United States, 15 in the East and 11 in the West. They were selected for geographic and demographic diversity, for the number of children born and the number of babies born with low birth weights.

Copyright 2007 by UPI.

[paula_w]

Some good things are being done with adult cells. Dr. Craig Cady, for whom Joan Snyder raises money, has gotten them to behave like a neuron. That's where the funding is so they have more to report.

ESCR - the money has to be there at both ends - investment and return.

I talked to Dr. Gerhardt - He is working with a medium sized molecule and can't say anymore - it's so competitive. He spoke about his wife...so sad...here we spend all this time on GDNF while his wife dies of cancer.

He is supportive of a bill of rights.


paula

[indigogo]

Thanks for the great report, Paula!

Looking forward to seeing you and your brain on the road again next week in NYC - where you can fill it with even more information and knowledge ... and give it a break with some laughter, white wine or vodka tonics in the bar?

[Todd]

Paula,

You were sitting next to Dr. Braak? Man, that's one guy whose brain I'd like to pick. His theory matches my personal story to a tee! Thanks for the report!

[paula_w]

Yes Todd...I simply have to read more of him I keep hearing this theory,name, stages. I didn't recognize him though and may not have even supposed to've been sitting in that row now that I think about it..lol I'll post more of what I have from him.

Carey - it's a busy place is it not? Looking forward to the reunion. It's been awhile! I have enough laundry that if I start now......

And what could be more comforting, than to get a call from my gastroenterologist reminding me that on Tuesday, day before departure, I have a yearly check-up at 1:00 p.m.

paula