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
Related Stories
Dr. Lowe, from In the Pipeline, writes about an apparent cure for sickle-cell disease and the challenges of expanding expensive cures to developing countries:
News came recently of an apparent cure, via gene therapy, of sickle-cell disease in a young patient (whose condition was refractory to hydroxyurea and the other standards of care). Blood-cell diseases are naturally one of the main proving grounds for things like this, since their stem cell populations are in easily localizable tissues and the techniques for doing a hard reset/retransplantation on them are (in some cases) well worked out.
This is an important result, but all such approaches face a possible disconnect as they move forward. As it stands, such gene therapy is a rather expensive and labor-intensive process. Patients are carefully identified and handled one at a time, and there are a limited number of medical centers in the entire world that can operate at this level. The problem is, none of them are particularly close to the great majority of people who actually have sickle cell disease.
[...] Is there any hope that gene therapy and cell replacement could get to the point that you could carry it out at a useful rate in some of the places where it would be needed the most? That's going to to hard, but this article at Technology Review by Antonio Regalado shows some progress:
In October, (Jennifer) Adair demonstrated a new technology she thinks could democratize access to gene therapy. Tweaking a cell-processing device sold by German instrument maker Miltenyi, she mostly automated the process of preparing blood cells with a gene therapy for HIV that her center is also testing. Cells dripped in one end came out the other 30 hours later with little oversight needed. She even added wheels. Adair calls the mobile lab "gene therapy in a box."
[...] The many companies that are working on such therapies seem to be paying attention to this sort of work, because it's not only a possible path to getting clinical trials run (and eventually patients treated) in the regions where most such patients are to be found. Companies are going to be selling such things first to people in the wealthier developed countries, but that's only the beginning of the story (as it has been with antiretroviral drugs).
http://blogs.sciencemag.org/pipeline/archives/2017/03/08/gene-therapy-needs-machines
https://www.technologyreview.com/s/603762/this-lab-in-a-box-could-make-gene-therapy-affordable/
http://www.nejm.org/doi/full/10.1056/NEJMoa1609677
https://en.wikipedia.org/wiki/Sickle-cell_disease
https://en.wikipedia.org/wiki/Gene_therapy
Arthur T Knackerbracket has found the following story:
A new era of treating disease has moved a step closer to reality in the United States.
A Food and Drug Administration panel gave a thumbs-up Wednesday to a gene therapy that involves genetically engineering a patient's T-cells to fight a particular type of leukemia, The New York Times reports.
If the FDA agrees with the panel's recommendation and moves to approve the treatment for commercial use, it would be the first such gene-altering treatment to make it to market.
[...] Once the stuff of science fiction, altering human genes has been creeping into reality of late. Also on Wednesday, researchers at Harvard announced they'd managed to encode video files into the genetic material of living cells, demonstrating the viability of a "molecular recorder" that could lead to more disease treatments in the future.
-- submitted from IRC
https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm574058.htm
The U.S. Food and Drug Administration issued a historic action today making the first gene therapy available in the United States, ushering in a new approach to the treatment of cancer and other serious and life-threatening diseases.
The FDA approved Kymriah (tisagenlecleucel) for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL).
"We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer," said FDA Commissioner Scott Gottlieb, M.D. "New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses. At the FDA, we're committed to helping expedite the development and review of groundbreaking treatments that have the potential to be life-saving."
Kymriah, a cell-based gene therapy, is approved in the United States for the treatment of patients up to 25 years of age with B-cell precursor ALL that is refractory or in second or later relapse.
Kymriah is a genetically-modified autologous T-cell immunotherapy. Each dose of Kymriah is a customized treatment created using an individual patient's own T-cells, a type of white blood cell known as a lymphocyte. The patient's T-cells are collected and sent to a manufacturing center where they are genetically modified to include a new gene that contains a specific protein (a chimeric antigen receptor or CAR) that directs the T-cells to target and kill leukemia cells that have a specific antigen (CD19) on the surface. Once the cells are modified, they are infused back into the patient to kill the cancer cells.
Also at NPR, CNN, BBC, and FierceBiotech.
Novartis press release.
The U.S. Food and Drug Administration (FDA) has approved a gene therapy for non-Hodgkin's lymphoma (blood cancer):
The Food and Drug Administration on Wednesday approved the second in a radically new class of treatments that genetically reboot a patient's own immune cells to kill cancer.
The new therapy, Yescarta, made by Kite Pharma, was approved for adults with aggressive forms of a blood cancer, non-Hodgkin's lymphoma, who have undergone two regimens of chemotherapy that failed.
The treatment, considered a form of gene therapy, transforms the patient's cells into what researchers call a "living drug" that attacks cancer cells. It is part of the rapidly growing field of immunotherapy, which uses drugs or genetic tinkering to turbocharge the immune system to fight disease. In some cases the treatments have led to long remissions.
"The results are pretty remarkable," said Dr. Frederick L. Locke, a specialist in blood cancers at the Moffitt Cancer Center in Tampa, and a leader of a study of the new treatment. "We're excited. We think there are many patients who may need this therapy."
He added, "These patients don't have other options."
About 3,500 people a year in the United States may be candidates for Yescarta. It is meant to be given once, infused into a vein, and must be manufactured individually for each patient. The cost will be $373,000.
Also at The Associated Press, CNN, and STAT News.
Previously: FDA Approves a Gene Therapy for the First Time
FDA Committee Endorses Gene Therapy for a Form of Childhood Blindness
The Food and Drug Administration (FDA) has approved a gene therapy to treat RPE65-mutation associated retinal dystrophy. But it will be expensive:
A first-of-its-kind gene therapy received approval from the Food and Drug Administration on Tuesday to treat a rare, inherited form of childhood blindness.
The FDA marketing clearance of Spark Therapeutics's Luxturna is historic for scientific and financial reasons. Luxturna is the first gene therapy approved in the U.S. that targets a disease caused by mutations in a specific gene.
A Spark spokesman said the company will not disclose the Luxturna price tag until early January. Wall Street analysts expect the gene therapy to command a $1 million price tag—another first, but not necessarily a welcome one. At a time when drug prices are coming under intense scrutiny, Spark will need to convince insurers, politicians, and pharma critics that the benefit to patients offered by Luxturna justifies its high cost.
Also at NPR.
Previously: FDA Approves a Gene Therapy for the First Time
FDA Committee Endorses Gene Therapy for a Form of Childhood Blindness
In a milestone year, gene therapy is finding a place in medicine
After decades of hope and high promise, this was the year scientists really showed they could doctor DNA to successfully treat diseases. Gene therapies to treat cancer and even pull off the biblical-sounding feat of helping the blind to see were approved by U.S. regulators, establishing gene manipulation as a new mode of medicine.
Almost 20 years ago, a teen's death in a gene experiment put a chill on what had been a field full of outsized expectations. Now, a series of jaw-dropping successes have renewed hopes that some one-time fixes of DNA, the chemical code that governs life, might turn out to be cures. "I am totally willing to use the 'C' word," said the National Institutes of Health's director, Dr. Francis Collins.
[...] The advent of gene editing — a more precise and long-lasting way to do gene therapy — may expand the number and types of diseases that can be treated. In November, California scientists tried editing a gene inside someone's body for the first time using a tool called zinc finger nucleases for a man with a metabolic disease. It's like a cut-and-paste operation to place a new gene in a specific spot. Tests of another editing tool called CRISPR to genetically alter human cells in the lab may start next year. "There are a few times in our lives when science astonishes us. This is one of those times," Dr. Matthew Porteus, a Stanford University gene editing expert, told a Senate panel discussing this technology last month.
Previously: Gene Therapy Cure for Sickle-Cell Disease
Gene Therapy to Kill Cancer Moves a Step Closer to Market
U.S. Human Embryo Editing Study Published
FDA Approves a Gene Therapy for the First Time
Gene Editing Without CRISPR -- Private Equity Raises $127 Million
FDA Committee Endorses Gene Therapy for a Form of Childhood Blindness
FDA Approves Gene Therapy for Non-Hodgkin's Lymphoma
Gene Therapy and Skin Grafting for Junctional Epidermolysis Bullosa
Gene Therapy for Spinal Muscular Atrophy Type 1
Biohackers Disregard FDA Warning on DIY Gene Therapy
CRISPR Used to Epigenetically Treat Diseases in Mice
Gene Therapy Showing Promise for Hemophilia B
Gene Therapy for Retinal Dystrophy Approved by the FDA
CRISPR Treatment for Some Inherited Forms of Lou Gehrig's Disease Tested in Mice
(Score: 2) by DannyB on Friday October 13 2017, @02:44PM (3 children)
Google could help by being a responsible corporate citizen.
Google would invest in treatments or cures if they would but recognize that blind children are deprived of advertisements for life.
(alternately wire VR headsets directly into the visual cortex)
The anti vax hysteria didn't stop, it just died down.
(Score: 0) by Anonymous Coward on Friday October 13 2017, @03:30PM (2 children)
Wow. This came out of left field. Not quite the article where I would have predicted a Google hate comment to have been inserted, particularly as the first comment.
Working through some issues today, maybe?
(Score: 2) by takyon on Friday October 13 2017, @04:20PM (1 child)
Could easily substitute "Facebook" and "blind Puerto Rican children" in that comment without changing it.
However Alphabet/Google does have not one but TWO medical branches: Calico [wikipedia.org] and Verily [wikipedia.org].
[SIG] 10/28/2017: Soylent Upgrade v14 [soylentnews.org]
(Score: 2) by DeathMonkey on Friday October 13 2017, @05:32PM
You could also substitute McDonald, Boeing or freaking Blackwater and they would all be equally relevant to the actual article: AKA not relevant at all!
(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.