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To: SnowShredder who wrote (13888)2/4/2000 5:00:00 AM
From: SnowShredder  Read Replies (2) | Respond to of 59879
 
ARIA could be in play tomorrow...I listened to Victor's talk @ Keystone, I was impressed...this system could be used to reglulate the secretion of any protein desired (but it is still pre-clinical), not just insulin...tried to get the Science article today online(Science is one of the elite journals to get published in) ...I struck out...gotta wait for the hard copy to arrive : (

Best of Luck,

Where'd He Go?

biz.yahoo.com (announced 2/3/00 in the morning)

biz.yahoo.com (announced 2/3/00 in the evening)

>>>>>>>>>>

Thursday February 3, 8:46 pm Eastern Time
New gene therapy approach makes cells pump protein
WASHINGTON, Feb 3 (Reuters) - A new approach to gene therapy turns the body's own cells into little protein factories and has worked to help diabetic mice make their own insulin, researchers said on Thursday.

A team at Ariad Pharmaceuticals Inc. (NasdaqNM:ARIA - news) said their method may make the still-experimental science of gene therapy work better and with more control.

They genetically engineered mouse cells to produce extra insulin, but the cells did not release the insulin until ``told' to do so by a drug given orally.

Writing in the journal Science, the researchers, who worked with scientists at New York's Memorial Sloan Kettering Cancer Center and the University of Geneva in Switzerland, said their method seemed well-suited to helping the body with proteins that need to be secreted in bursts -- such as insulin.

``Within fifteen minutes of administering Ariad's drug, insulin appeared in the bloodstream and rapidly rose to peak levels within less than two hours, leading to a correction of blood sugar,' the Cambridge, Massachusetts-based company said in a statement.

``The level of insulin then subsided over the next two hours and returned towards basal levels.'

That is just what insulin is supposed to do naturally.

Currently, people with diabetes must monitor their blood sugar levels, and often have to take insulin to coincide with meals to make sure blood sugar is controlled properly.

Diabetes, which affects 16 million Americans, is a big target of gene therapists.

But gene therapy is a tricky technology. It is hard to get cells to absorb the new genes, and sometimes they do not ``express,' or order the production of proteins, when they are supposed to.

And there has been recent controversy over the death of an 18-year-old man as a result of gene therapy. Congress, the Food and Drug Administration (FDA) and the National Institutes of Health (NIH) are investigating the case.

Ariad chairman and chief executive officer Dr. Harvey Berger thinks his company's method is safer because it will use an adeno-associated virus (AAV) to carry the new genes into a patient's body. The viruses do not cause disease in humans.

Ariad's technology, called Regulated Accumulation of Proteins for Immediate Delivery (RAPID), uses gene therapy to command cells to make and store the target proteins. They are inactive and stable until a drug is given to the patent -- in this case a mouse -- that breaks apart the protein clusters.

``It will make gene therapy safer because you can shut it off and more effective because you can fine-tune the effects of the gene therapy,' Berger said in a telephone interview.

He said the approach would essentially let muscle cells do the work of the pancreas, which normally produces insulin.

``A diabetic patient will get an injection of the vector into muscle and then the muscle cells will make the insulin, store the insulin and then release it in response to the pill,' he said.

``Muscle is a natural factory for protein production. All we are doing there is asking it to make a different protein.'

Berger said the approach might also be used for the control of pain, treatment of cancer or heart disease that involves controlling the growth of blood vessels, and perhaps to treat obesity.

For example, cells could be engineered to produce a protein such as leptin, which is linked with appetite.

``You could never do that with an injectable protein because there is no way an obese patient would inject themselves 3 or 4 times a day,' Berger said.

Berger said it would take at least two more years of animal testing before the approach could be tested in humans.