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8-Humans: US biotech company plans EPO gene replacement trials

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TITLE:  Biotech Company Plans Trials on Gene Therapy for Anemia
SOURCE: The New York Times, by Andrew Pollack
DATE:   Nov 1, 2002

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Biotech Company Plans Trials on Gene Therapy for Anemia

A biotechnology company hopes to begin clinical trials next year on what it 
says will be a cheaper and more convenient way to provide the anemia drug 
erythropoietin, posing a potential challenge to Amgen and Johnson & Johnson.

The company, Ariad Pharmaceuticals, of Cambridge, Mass., said it planned to 
use gene therapy to provide patients with the gene responsible for 
producing erythropoietin, or EPO. Instead of constantly receiving 
injections of EPO, patients would be able to make their own.

EPO, a kidney-made protein that stimulates the body to produce oxygen-
carrying red blood cells, is the best-selling product developed by the 
biotechnology industry. Combined sales by Amgen and its licensee, Johnson & 
Johnson, exceed $5 billion a year, which has made it a tempting target for 
potential competitors.

Currently the protein is given either by intravenous infusion or by 
injection, with up to three injections a week. For patients undergoing 
kidney dialysis, a major market, the cost is at least $7,000 to $9,000 a 

At the meeting of the American Society of Nephrology in Philadelphia this 
weekend, Ariad and its collaborators at the University of Pennsylvania will 
report that 22 monkeys injected one time with the EPO gene were able to 
produce EPO for four years in some cases. Ariad uses a virus to carry the 
genes into muscles.

"All the moving parts now work really well long term, and the next stage is 
clinical and commercial," Ariad's chief executive, Harvey J. Berger, said.

With Ariad's system, the implanted genes do not switch on to make EPO until 
the patient takes a pill. That would allow patients to control how much EPO 
is produced and prevent harmful overproduction. Ariad said the pill would 
need to be taken once or twice a month.

Dr. F. John Gennari, a nephrologist at the University of Vermont, called 
the work intriguing because dialysis centers are always under pressure to 
cut the costs of EPO therapy. Dr. Steven J. Scheinman, chief of nephrology 
at SUNY Upstate Medical University in Syracuse, agreed, saying, "It might 
be nice to take a pill rather than get these injections."

Still, the clinical trials will take years and many challenges remain.

Gene therapy has a history of failure, even when scientists allow the 
implanted genes to be active all the time. Trying to switch genes on and 
off is even more difficult, and such switches tend to weaken over time. 
"You just better be sure your switch works," said John Monahan, chief 
executive of Avigen, another gene therapy company.

The therapy also carries safety risks, as highlighted by the 1999 death of 
teenager Jesse Gelsinger after he received gene therapy at the University 
of Pennsylvania, the same institution working with Ariad.

Questions might also be raised about the gene switch pill, a modified 
version of the drug rapamycin, which suppresses the immune system. Ariad 
maintains that its modified version is only minimally immunosuppressive. 
But dialysis patients who receive the gene therapy and then receive a 
kidney transplant would not be able to use rapamycin to fight organ 

Amgen's patents are another potential obstacle. Amgen has already sued to 
stop one company, Transkaryotic Therapies, from selling EPO produced by 
another means. That case is on appeal.

Dr. Berger said Ariad's system infringes only Amgen's patent on the EPO 
gene, which expires in 2004 - well before Ariad's treatment would reach the 

But a spokesman for Amgen disagreed, saying, "It would be certain that one 
or more other patents that expire much later" would be infringed.


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