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so good, Leschly says—Bluebird expects to finish enrolling all 10 patients it wants to recruit at a single location in France over the next 12 months. Lots of gene therapy watchers will eagerly watch to see if the experience of the first patient can be reproduced in that larger group.
—The other key program at Bluebird is a treatment for childhood cerebral adrenoleukodystrophy, a genetic brain disorder. This program was written up in Science, and honored as one of the top 10 breakthroughs of 2009, after two boys had their condition stabilize following Bluebird’s gene therapy.
The first two patients are still doing well, Leschly says. Now Bluebird’s plan is to move aggressively ahead to confirm the result in a bigger clinical trial. The plan is to seek FDA clearance this year to initiate a company-sponsored, global trial, enroll patients in 2012, and talk with regulators about whether this trial will provide enough evidence to seek approval to begin marketing the therapy, Leschly says.
“We feel that based on the need, and based on the proof of concept that’s already been established in a Phase 1/2 trial, this new data can move us toward the accelerated approval process,” Leschly says. The goal will be to show the gene therapy approach is as effective as a bone marrow transplant for these patients, but without the complications (finding a genetic match, dealing with immune-system reactions) inherent with that process.
Bluebird is now working to make a better lentiviral vector, combined with a better manufacturing process, to take the treatment forward in clinical trials, Leschly says.
Naturally, I had to ask if changing the vector of this therapy means Bluebird will have to start over in clinical trials. That’s not the case, Leschly says, because the company isn’t changing the “heart” of the vector. The company is using some newer lentiviral manufacturing technology that should be more efficient at delivering the genes into cells, and make the treatment easier to manufacture, he says.
Wrestling with questions like this are a sign, Leschly says, of gene therapy starting to grow up. It’s time to move ahead more aggressively in larger clinical trials, and against multiple diseases, to see if gene therapy can finally get over the hump and create marketed products that help patients.
“There’s been a lot of scientific debate, and a few disasters,” Leschly says. “There’s been clinical data, but it hasn’t been particularly good. Now you’re seeing dramatic improvements in the safety of the vectors, dramatic improvement in the data in multiple diseases. That’s an exciting thing to hear.”
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