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Friday
Gene Therapy Trials Show Promise Against Parkinson's (click for more):
"The first studies of human gene therapy for Parkinson's disease have shown that the technique is safe and can reduce symptoms for patients, two groups of researchers have reported.
All of the 24 patients who received therapy in the two separate trials received some benefit and none had any significant side effects, researchers reported at neuroscience meetings Tuesday and last week.
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Gene therapy has a tarnished reputation because of problems encountered in trials involving other diseases, said Katie Hood, deputy chief executive officer of the Michael J. Fox Foundation for Parkinson's Research.
The Food and Drug Administration temporarily halted gene therapy trials in 1999 after an 18-year-old being treated for a mild genetic disorder died after a violent reaction to the procedure. Trials were halted in 2005 after three French children being treated for inherited immunodeficiency disease developed leukemia and one of them died.
'It's very encouraging that two companies were able to show benefits with no significant adverse effects,' Hood said. 'Safety is obviously the first hurdle.'
Experts and the researchers themselves, however, cautioned patients against investing too much hope in the findings because Parkinson's studies are notorious for showing placebo effects.
Only when the techniques are tested in controlled trials, now in the planning stages, will researchers be able to determine whether the benefits are real and lasting.
Parkinson's, which strikes as many as 100,000 Americans each year, is characterized by severe tremors and rigidity in the limbs, and loss of muscle control. It results from the death of brain cells that produce the neurotransmitter dopamine, which plays a key role in transmitting commands from the brain's muscle-control centers. The disorder's cause is unknown.
Both teams used the same gene therapy technology, inserting a desired gene into the common adeno-associated virus. AAV readily infects humans but has never been shown to cause disease..."
"The first studies of human gene therapy for Parkinson's disease have shown that the technique is safe and can reduce symptoms for patients, two groups of researchers have reported.
All of the 24 patients who received therapy in the two separate trials received some benefit and none had any significant side effects, researchers reported at neuroscience meetings Tuesday and last week.
ADVERTISEMENT
Click here to find out more!
Gene therapy has a tarnished reputation because of problems encountered in trials involving other diseases, said Katie Hood, deputy chief executive officer of the Michael J. Fox Foundation for Parkinson's Research.
The Food and Drug Administration temporarily halted gene therapy trials in 1999 after an 18-year-old being treated for a mild genetic disorder died after a violent reaction to the procedure. Trials were halted in 2005 after three French children being treated for inherited immunodeficiency disease developed leukemia and one of them died.
'It's very encouraging that two companies were able to show benefits with no significant adverse effects,' Hood said. 'Safety is obviously the first hurdle.'
Experts and the researchers themselves, however, cautioned patients against investing too much hope in the findings because Parkinson's studies are notorious for showing placebo effects.
Only when the techniques are tested in controlled trials, now in the planning stages, will researchers be able to determine whether the benefits are real and lasting.
Parkinson's, which strikes as many as 100,000 Americans each year, is characterized by severe tremors and rigidity in the limbs, and loss of muscle control. It results from the death of brain cells that produce the neurotransmitter dopamine, which plays a key role in transmitting commands from the brain's muscle-control centers. The disorder's cause is unknown.
Both teams used the same gene therapy technology, inserting a desired gene into the common adeno-associated virus. AAV readily infects humans but has never been shown to cause disease..."