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The U.S. Food and Drug Administration recently approved a gene therapy for the first time, to treat a form of leukemia. Now an FDA panel has endorsed a gene therapy for an inherited form of blindness. The FDA usually follows the recommendations of its advisory committees:
Gene therapy, which has had a roller-coaster history of high hopes and devastating disappointments, took an important step forward Thursday. A Food and Drug Administration advisory committee endorsed the first gene therapy for an inherited disorder — a rare condition that causes a progressive form of blindness that usually starts in childhood. The recommendation came in a unanimous 16-0 vote after a daylong hearing that included emotional testimonials by doctors, parents of children blinded by the disease and from children and young adults helped by the treatment.
"Before surgery, my vision was dark. It was like sunglasses over my eyes while looking through a little tunnel," 18-year-old Misty Lovelace of Kentucky, told the committee. "I can honestly say my biggest dream came true when I got my sight. I would never give it up for anything. It was truly a miracle." Several young people described being able to ride bicycles, play baseball, see their parents' faces, read, write and venture out of their homes alone at night for the first time. "I've been able to see things that I've never seen before, like stars, fireworks, and even the moon," Christian Guardino, 17, of Long Island, N.Y., told the committee. "I will forever be grateful for receiving gene therapy."
The FDA isn't obligated to follow the recommendations of its advisory committees, but the agency usually does. If the treatment is approved, one concern is cost. Some analysts have speculated it could cost hundreds of thousands of dollars to treat each eye, meaning the cost for each patient could approach $1 million. Spark Therapeutics of Philadelphia, which developed the treatment, hasn't said how much the company would charge. But the company has said it would help patients get access to the treatment.
Despite the likely steep price tag, the panel's endorsement was welcomed by scientists working in the field. "It's one of the most exciting things for our field in recent memory," says Paul Yang, an assistant professor of ophthalmology at the Oregon Health and Science University who wasn't involved in developing or testing the treatment. "This would be the first approved treatment of any sort for this condition and the first approved gene therapy treatment for the eye, in general," Yang says. "So, on multiple fronts, it's a first and ushers in a new era of gene therapy."
Also at MIT.
Previously: Gene Therapy Cure for Sickle-Cell Disease
Gene Therapy to Kill Cancer Moves a Step Closer to Market
(Score: 0) by Anonymous Coward on Friday October 13 2017, @03:40PM (8 children)
http://www.thelancet.com/journals/lancet/article/PIIS0140-6736(17)31868-8/fulltext [thelancet.com]
So the "testers" were aware of the treatment group and may have influenced performance on the maze. Also, they clearly are concerned that (besides vision) the test measures learning ability, since they attempt to "reduce learning effect". I wonder what else it may measure. Balance? Agility?
This is the type of stuff the FDA should be concerned with, not emotions. Also, who is to say if these people won't get eye cancer in a couple years (this study only looked 1 year later)? It may be worth the risk, I'm just saying that is the kind of thing the FDA should be concerned about.
(Score: 2) by Joe on Friday October 13 2017, @04:36PM (7 children)
You assume that they aren't?
As far as I know, AAV-based gene therapy has not been found to cause cancer. The vectors rarely integrate into the genome (and insertion is random, unlike retroviruses) and are not immunogenic enough to produce an inflammation-induced cancer. Obviously, follow-ups will be done on trial participants as well as post-trial surveillance once this hits the market and approval will be revoked or amended in response to new findings.
- Joe
(Score: 0) by Anonymous Coward on Friday October 13 2017, @05:10PM (6 children)
Why are these emotional testimonials included at all? Also in the appendix you can see half the control group improved as well. I have no idea how well that test corresponds to "seeing the moon", etc, but that indicates this is a problem that gets better/worse.
(Score: 0) by Anonymous Coward on Friday October 13 2017, @06:22PM (5 children)
The FDA is a government agency that is supposed to represent the will of the people. Voters, patient advocacy groups, and other lobbiest organizations have pushed for the inclusion of these testimonials and case studies.
This can go very wrong and biases the decision-making process, but the FDA isn't a scientific organization. There's even a strong push to eliminate efficacy requirements and rely solely on post-market observational data.
https://soylentnews.org/article.pl?sid=16/09/21/0255259 [soylentnews.org]
(Score: 0) by Anonymous Coward on Saturday October 14 2017, @02:49AM (4 children)
(Score: 0) by Anonymous Coward on Saturday October 14 2017, @05:34AM (3 children)
People that support the repeal of efficacy requirements typically do so because they have the misguided assumptions that government beurocracy is the main thing limiting medical progress and that the market will do a better job.
(Score: 0) by Anonymous Coward on Saturday October 14 2017, @09:54PM (2 children)
Original AC here.
As noted, the FDA panel didn't have any problem with concluding efficacy despite that the researchers failed to use basic tools like blinding the people collecting data. They also didn't seem to be concerned that there are other equally as good explanations for the results. Regardless of whether any alternative is better/worse, you can not rely on the approval of such an agency to indicate efficacy.
(Score: 0) by Anonymous Coward on Sunday October 15 2017, @02:06PM (1 child)
Is your point that the efficacy requirements are useless because they are not stringent enough, so we should:
A. Get rid of them entirely because if it can't reach a high enough threshold of confidence, then any data is useless.
Or
B. Increase the stringency, length, and sample size until we are certain that the medicine is safe and effective.
Well, "A" ignores Bayesian probability and "B" paralyses the decision making process by demanding perfect information. In the real world, people have to make decisions with imperfect information and with confidence levels well below those of nuclear physicists (and even they overestimated their confidence in the mass of a proton).
(Score: 0) by Anonymous Coward on Sunday October 15 2017, @06:23PM
My point is "you can not rely on the approval of such an agency to indicate efficacy". People should act accordingly, each in their own ways. Also, "perfect is the the enemy of good" is pretty funny in this case. We are looking at an approval based on a study run by people who didn't think to blind the researchers collecting the data.
Besides the direct issue with that, I can only imagine what else was going on that may skew the results.