RE: gene therapy studies
I just came across this. I was wondering if anyone knows if any gene therapy studies for Cystic Fibrosis are using this adeno-associated virus (or AAV).
They are reviewing 28 other studies using this virus, wondering if CF is one of them.
Woman's Death in Drug Study Probed
By CARLA K. JOHNSON, Associated Press Writer
6 hours ago
CHICAGO - A woman whose death in a gene therapy study shut it down and prompted a review of the safety of 28 other studies was experiencing multiple organ failure when she got to the hospital, a spokesman said.
Jolee Mohr, 36, died July 24, 22 days after receiving her second injection of an experimental drug made of genetically engineered viruses she hoped would help her arthritis.
Robb Mohr said he believes his wife thought the drug would help her, even though the research was to determine the drug's safety, rather than its effectiveness. The University of Chicago Medical Center, where Jolee Mohr died, is investigating the cause of death.
"By the time she got to us, she was in liver failure and kidney failure, she was on a ventilator and she was septic" or responding to severe infection, hospital spokesman John Easton told The Associated Press. The hospital will send tissue samples to multiple labs for testing.
Targeted Genetics Corp. of Seattle has halted the study, and more than 100 patients involved are being evaluated, said company spokeswoman Stacie Byars. The company believes it's too early to speculate on the woman's cause of death, Byars said.
Alan Milstein, a New Jersey attorney who is representing Robb Mohr in a possible civil lawsuit, said Jolee Mohr believed the experimental therapy would be in her best interests.
"She wasn't going to risk her life for science or medicine or the profits of some company," Milstein told The Associated Press on Saturday. "She had mild rheumatoid arthritis."
Milstein also represented 18-year-old Jesse Gelsinger, who died in 1999 in his fourth day of a gene therapy experiment at the University of Pennsylvania. Gelsinger had suffered from an inherited disorder that blocks the body from properly processing nitrogen. The Food and Drug Administration concluded that the gene therapy injection intended to try to cure him instead killed him.
Milstein said he's not sure who's to blame for Jolee Mohr's death, but "we certainly believe the death was connected to the research trial she was in."
The experimental drug uses a virus to try and block a substance that fuels the joint inflammation behind crippling forms of arthritis.
<b>Twenty-eight other gene therapy studies have been reported to the FDA that used, or are using, the same virus, called adeno-associated virus or AAV.</b>
The FDA has said that it was not aware of any serious side effects in any of the AAV studies but that as a precaution, officials are reviewing the ones still actively treating patients.
In addition, the National Institutes of Health's advisory committee on gene therapy will meet in September to discuss the potential scientific implications of Mohr's death.
I just came across this. I was wondering if anyone knows if any gene therapy studies for Cystic Fibrosis are using this adeno-associated virus (or AAV).
They are reviewing 28 other studies using this virus, wondering if CF is one of them.
Woman's Death in Drug Study Probed
By CARLA K. JOHNSON, Associated Press Writer
6 hours ago
CHICAGO - A woman whose death in a gene therapy study shut it down and prompted a review of the safety of 28 other studies was experiencing multiple organ failure when she got to the hospital, a spokesman said.
Jolee Mohr, 36, died July 24, 22 days after receiving her second injection of an experimental drug made of genetically engineered viruses she hoped would help her arthritis.
Robb Mohr said he believes his wife thought the drug would help her, even though the research was to determine the drug's safety, rather than its effectiveness. The University of Chicago Medical Center, where Jolee Mohr died, is investigating the cause of death.
"By the time she got to us, she was in liver failure and kidney failure, she was on a ventilator and she was septic" or responding to severe infection, hospital spokesman John Easton told The Associated Press. The hospital will send tissue samples to multiple labs for testing.
Targeted Genetics Corp. of Seattle has halted the study, and more than 100 patients involved are being evaluated, said company spokeswoman Stacie Byars. The company believes it's too early to speculate on the woman's cause of death, Byars said.
Alan Milstein, a New Jersey attorney who is representing Robb Mohr in a possible civil lawsuit, said Jolee Mohr believed the experimental therapy would be in her best interests.
"She wasn't going to risk her life for science or medicine or the profits of some company," Milstein told The Associated Press on Saturday. "She had mild rheumatoid arthritis."
Milstein also represented 18-year-old Jesse Gelsinger, who died in 1999 in his fourth day of a gene therapy experiment at the University of Pennsylvania. Gelsinger had suffered from an inherited disorder that blocks the body from properly processing nitrogen. The Food and Drug Administration concluded that the gene therapy injection intended to try to cure him instead killed him.
Milstein said he's not sure who's to blame for Jolee Mohr's death, but "we certainly believe the death was connected to the research trial she was in."
The experimental drug uses a virus to try and block a substance that fuels the joint inflammation behind crippling forms of arthritis.
<b>Twenty-eight other gene therapy studies have been reported to the FDA that used, or are using, the same virus, called adeno-associated virus or AAV.</b>
The FDA has said that it was not aware of any serious side effects in any of the AAV studies but that as a precaution, officials are reviewing the ones still actively treating patients.
In addition, the National Institutes of Health's advisory committee on gene therapy will meet in September to discuss the potential scientific implications of Mohr's death.