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posted by martyb on Saturday October 21 2017, @12:09AM   Printer-friendly
from the a-third-chance-at-life dept.

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


Original Submission

Related Stories

FDA Approves a Gene Therapy for the First Time 12 comments

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.


Original Submission

FDA Committee Endorses Gene Therapy for a Form of Childhood Blindness 13 comments

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


Original Submission

2017: Gene Therapy's Milestone Year 4 comments

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


Original Submission

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  • (Score: 3, Insightful) by frojack on Saturday October 21 2017, @12:27AM (22 children)

    by frojack (1554) on Saturday October 21 2017, @12:27AM (#585491) Journal

    So what are the down side risks of this drug that warrant subjecting patients to the torture of chemo, TWICE, with the disease progressing all the time?

    escarta has a Boxed Warning in its product label regarding the risks of cytokine release syndrome [wikipedia.org] (CRS) and neurologic toxicities. A Risk Evaluation and Mitigation Strategy (REMS) has been approved by the FDA for Yescarta. The REMS program will inform and educate healthcare professionals about the risks associated with Yescarta therapy. Training and certification on the REMS program will be an integral part of the final authorization for centers offering Yescarta. Additional information about the REMS program can be found at www.yescartarems.com. Please see below for Important Safety Information.

    Sounds like the largest risks are about the same as the chemo.

    --
    No, you are mistaken. I've always had this sig.
    • (Score: 2) by frojack on Saturday October 21 2017, @12:29AM (17 children)

      by frojack (1554) on Saturday October 21 2017, @12:29AM (#585492) Journal

      And then there's this bit:

      The list price of Yescarta in the United States is $373,000.

      Good luck with that Joe Sixpack.

      --
      No, you are mistaken. I've always had this sig.
      • (Score: 2) by Arik on Saturday October 21 2017, @12:58AM

        by Arik (4543) on Saturday October 21 2017, @12:58AM (#585511) Journal
        I think you answered your own question. The cost itself can be considered part of the package of effects. How much do two courses of chemo cost?

        --
        If laughter is the best medicine, who are the best doctors?
      • (Score: 1, Redundant) by The Mighty Buzzard on Saturday October 21 2017, @01:41AM (14 children)

        by The Mighty Buzzard (18) Subscriber Badge <themightybuzzard@proton.me> on Saturday October 21 2017, @01:41AM (#585518) Homepage Journal

        Yeah, the price is what gives me pause as well. I'm undecided on if insurance companies should simply not cover it.

        Think about it. Insurance companies are essentially a bunch of people pooling their money to turn peak healthcare costs into flat healthcare costs, with a fairly small percentage of overhead for management of the funds. They are decidedly not infinite piles of money. Paying out more for one person than they will ever be able to pay back in means you're lowering the quality of healthcare for everyone for the sake of one person who is unquestionably not paying their share.

        Before anyone starts in with "you heartless bastard", STFU. I, like every mentally healthy person reading this, care more about myself and my family than about some random stranger. Their health will always be my priority, not yours. This is exactly as it should be.

        --
        My rights don't end where your fear begins.
        • (Score: 1) by khallow on Saturday October 21 2017, @01:47AM (6 children)

          by khallow (3766) Subscriber Badge on Saturday October 21 2017, @01:47AM (#585521) Journal

          Paying out more for one person than they will ever be able to pay back in means you're lowering the quality of healthcare for everyone for the sake of one person who is unquestionably not paying their share.

          The problems comes when almost everyone is doing that. As long as average costs remain below average insurance premiums, the thing will be stable.

          • (Score: 3, Insightful) by The Mighty Buzzard on Saturday October 21 2017, @02:00AM (5 children)

            by The Mighty Buzzard (18) Subscriber Badge <themightybuzzard@proton.me> on Saturday October 21 2017, @02:00AM (#585526) Homepage Journal

            Indeed. I'm just not sure that's a good way to do things at all. You're essentially laying a monthly bet down that you will get expensively sick. Most people won't, therefore it does not make financial sense for anyone without a history of expensive diseases or disorders in their family to support the current system. It doesn't even do the most good for the most people like our more socialist-leaning types would prefer. It penalizes most people for the sake of a small minority.

            --
            My rights don't end where your fear begins.
            • (Score: 1) by khallow on Saturday October 21 2017, @02:29AM

              by khallow (3766) Subscriber Badge on Saturday October 21 2017, @02:29AM (#585535) Journal
              The thing is large health care expenses that you can't fully cover are more destructive than paying a relatively small amount every year for decades. Health insurance really is about providing cash flow for extreme health problems with the idea that everyone is at risk of these large costs.
            • (Score: 0) by Anonymous Coward on Saturday October 21 2017, @04:03AM (2 children)

              by Anonymous Coward on Saturday October 21 2017, @04:03AM (#585555)
              Most everyone is going to come down with some kind of expensive disease as they age, and the older you get, the higher the chance of that happening. The number of people who will eventually get seriously ill or injured or will otherwise require expensive medical treatment is by no means a small minority!
              • (Score: 0) by Anonymous Coward on Saturday October 21 2017, @04:59AM

                by Anonymous Coward on Saturday October 21 2017, @04:59AM (#585565)

                even being warehoused in a nursing home (whether you are relatively healthy or not) is not cheap. you effectively get drained of your financial assets before Medicaid takes over paying for it.

              • (Score: 2) by The Mighty Buzzard on Saturday October 21 2017, @10:32AM

                by The Mighty Buzzard (18) Subscriber Badge <themightybuzzard@proton.me> on Saturday October 21 2017, @10:32AM (#585615) Homepage Journal

                See, there's where we fundamentally differ. I do not want to live past an age where I can take care of myself and under no circumstances do I want to be a huge financial burden to my family. I can't even mentally put myself in the position of anyone who does think like that even for the sake of understanding their argument.

                --
                My rights don't end where your fear begins.
            • (Score: 4, Interesting) by fritsd on Saturday October 21 2017, @09:51AM

              by fritsd (4586) on Saturday October 21 2017, @09:51AM (#585612) Journal

              That is an interesting way to put it, but essentially I can see nothing wrong with what you say. Maybe it's just a difference in viewpoint.

              So why does the USA have a large army, again? That also costs every taxpayer money, for unclear benefits:

              Indeed. I'm just not sure that's a good way to do things at all. You're essentially laying a monthly bet down that you will get foreign bandits on the border, and terrorists. Most people won't, therefore it does not make financial sense for anyone without a history of expensive gang warfare or terrorist attacks on their family to support the current system. It doesn't even do the most good for our proud nation like our more jingoistic types would prefer. It penalizes most people for the safety of a small minority.

              FTFY.

              That's *an* argument for pacifism; I didn't claim that it was a *good* argument :-)

        • (Score: 0) by Anonymous Coward on Saturday October 21 2017, @04:55AM (1 child)

          by Anonymous Coward on Saturday October 21 2017, @04:55AM (#585561)

          could you pay for a heart attack without insurance? no. How long could you afford kidney dialysis without your (or Medicaid) insurance?
          could you even afford to pay out of pocket to bring a new child into the world? pray there are no complications (NICU)...
          shit, pay out of pocket to get a non-complicated broken arm fixed.

          we ALL pay into a system that pays out way more for a few unlucky stiffs, one way or another. Yet we eventually all end up in the same terminal condition anyways. ultimately, how fucked up is THAT system?

          • (Score: 2) by The Mighty Buzzard on Saturday October 21 2017, @10:39AM

            by The Mighty Buzzard (18) Subscriber Badge <themightybuzzard@proton.me> on Saturday October 21 2017, @10:39AM (#585616) Homepage Journal

            Exactly. We're all going to die anyway and we have a choice of either leaving our assets to our our families or to the insurance companies, hospitals, and big pharma. It's really not a choice I need even a moment to consider.

            --
            My rights don't end where your fear begins.
        • (Score: 1, Interesting) by Anonymous Coward on Saturday October 21 2017, @05:10AM (1 child)

          by Anonymous Coward on Saturday October 21 2017, @05:10AM (#585568)

          so... whats better then..

          Say there is just one big insurance company.

          6000 patients/year die from NHL. say this treatment is 100% effective. thats $240 million.

          Compare with Harvoni (hepatitis vaccine), at ~$120k. its only ~70% effective, but lets say its also 100% effective...
          so same cost treats about 20000 patients...

          so the insurance company has to make some choices.

          source: work for health insurance co. NO insurance co can pay for everyone in its coverage who can/should get Harvoni.

          thats just one treatment.

          • (Score: 0) by Anonymous Coward on Saturday October 21 2017, @05:14AM

            by Anonymous Coward on Saturday October 21 2017, @05:14AM (#585570)

            sorry... $2.4 billion.

        • (Score: 2) by Grishnakh on Saturday October 21 2017, @02:51PM

          by Grishnakh (2831) on Saturday October 21 2017, @02:51PM (#585668)

          This is stupid. Payouts for unexpected and very expensive things are the whole reason we have "insurance". WTF is the point of having insurance if it's just going to pay for stuff you can pay for on your own?

          You probably have a $1M policy for your auto insurance: if you cause a wreck, it'll pay out up to $1M in damages, medical payments, etc. for the other parties. There's no way a lot of drivers would ever be able to pay that back. Are you saying that car insurance policies like that shouldn't exist? That's stupid. This is the whole reason insurance was invented; most people never cause accidents with $1M in damages, so it doesn't matter if you'll never pay that back personally; the insured pool covers that risk collectively. But the big difference is that auto insurance doesn't cover your regular maintenance: oil changes, etc., nor does it cover non-accident repairs (your timing belt broke and now you need to replace your cylinder head). For some odd reason, health insurance is expected to cover this stuff to a certain extent, albeit with "copays" or "coinsurance", but the the idea here is that the insurance company can supposedly negotiate better rates than you can, though this frequently isn't really true, as you'll find if you ask a doctor's office for their "cash price"--all that insurance paperwork is a PITA and costs a lot in staff salaries.

        • (Score: 1, Insightful) by Anonymous Coward on Saturday October 21 2017, @04:53PM (1 child)

          by Anonymous Coward on Saturday October 21 2017, @04:53PM (#585697)

          Yeah, the price is what gives me pause as well

          Presumably, the price of these therapies will come down as technology advances and possibly when patents expire.

          This type of treatment is still in the very early stages. There is a large push to transition these CAR T-cell therapies into a one-size-fits-all or, at least, multiple broadly accepted histocompatibility-types. If any of these are successful, then the prices will come down considerably.

      • (Score: 3, Informative) by Anonymous Coward on Saturday October 21 2017, @12:14PM

        by Anonymous Coward on Saturday October 21 2017, @12:14PM (#585642)

        A friend of mine actually works on a program similar to this, though it's for another form of cancer.

        The $373,000 price tag is because the treatment is custom-tailored to each individual patient's immune system. They have to extract white blood cells (which are part of your immune system) and cancer cells from the patient, and then design a virus that will attack the white blood cells and instead of killing them, change them so that they view the cancer as an invading bacteria instead of ignoring it. Right now the process is mostly manual, so you're involving a bunch of PhD biochemists and a few man-months (or person-months) of work per patient using expensive lab equipment.

        But if the concept keeps working reliably - and my friend said that for the type of cancer he works on, it's over 80% effective - they'll start figuring out how to automate more of the process. It could be a $300 over-the-counter kit from a pharmacist in twenty years.

    • (Score: 1, Interesting) by Anonymous Coward on Saturday October 21 2017, @02:10AM

      by Anonymous Coward on Saturday October 21 2017, @02:10AM (#585531)

      A very good friend died of non-Hodgkin's lymphoma a couple of years ago. When diagnosed it was fairly well along, he was a tough guy (still doing heavy duty Judo in his mid-70s) and just felt like he was getting tired too quickly. Then got the bad news.

      After a tour of a chemo infusion facility (patients in agony), checking the odds of success (add a few months to his life) and also watching the cash-register numbers spin (metaphorically) in the oncologist's eyes, he refused chemo. I believe that Medicare (+ add'l insurance?) would have covered chemo, don't know the price, but likely well over $100000 (100K). In his eyes it was hardly worth it for the destruction of any remaining quality of life he had left.

      They gave him 3-4 months, he lasted 15 months and got to see his first grandchild be born and healthy.

      He was always a bit of a doper and lived in a state where he could get medical marijuana. As the disease progressed and he slowly wasted away, he had to use more, but was essentially pain free until the very last stages, perhaps the last a couple of weeks.

      During all this, he had the satisfaction of giving away nearly all his possessions while he was alive (including house), leaving very little of his estate to be dealt with.

      Not sure if he could have scraped together the $300K for this new treatment, but if the odds were as good as it sounds, then I think a bunch of his friends would have chipped in (assuming Medicare, etc wouldn't cover it).

    • (Score: 2) by deimtee on Saturday October 21 2017, @08:18PM (2 children)

      by deimtee (3272) on Saturday October 21 2017, @08:18PM (#585766) Journal

      So what are the down side risks of this drug that warrant subjecting patients to the torture of chemo, TWICE, with the disease progressing all the time?

      NHL has a weird survival curve to chemo treatment until you work out that there are actually two diseases called NHL.
      Approx 50% of patients get one type, and CHOP-R chemo cures it.
      The rest get the other type, and chemo does nothing at all except make the patient sicker.
      There is no diagnostic test to tell the two types apart except administering chemo and seeing if it cures you.

      --
      If you cough while drinking cheap red wine it really cleans out your sinuses.
      • (Score: 0) by Anonymous Coward on Sunday October 22 2017, @12:24AM (1 child)

        by Anonymous Coward on Sunday October 22 2017, @12:24AM (#585830)

        Is it as simple as two types? I read up on this (a friend died from it) several years ago and my memory is that there were many varieties.

        • (Score: 2) by deimtee on Sunday October 22 2017, @02:26AM

          by deimtee (3272) on Sunday October 22 2017, @02:26AM (#585847) Journal

          Two types is probably an oversimplification, two groups would be closer, but the the effect is almost the same. Chemo either cures or does nothing, it doesn't seem to have a range of delaying responses like chemo does with most cancers.
          I too did a lot of reading on it a few years back when a close relative got it. One of the papers pointed out that if you separated patients into two groups, they both had standard survival patterns, it was just that one was extremely lethal, and the other easily treated. The 50% survival figure is an artifact of the fact that they can't tell them apart pre-treatment. It's actually a mix of 0% and 100%

          I think that is why they are insisting on two rounds of chemo before this new treatment. It will cut their patient load in half by removing everyone chemo can help.
          Also, if there is an underlying difference then tuning the treatment for the version that chemo doesn't cure would actually inprove the overall survival, and be the ethical thing to do.

          --
          If you cough while drinking cheap red wine it really cleans out your sinuses.
  • (Score: 2) by jmorris on Saturday October 21 2017, @07:17AM (1 child)

    by jmorris (4844) on Saturday October 21 2017, @07:17AM (#585592)

    I see a lot of attention is going to the price tag. Run the numbers folks, when we are talking about a disease that only a few thousand people are candidates for treating and a cutting edge treatment like this costs a crapload to develop and you only get a decade to recover the expense, blamo, insanely expensive. And since this thing has to be created for each patient, the per dose costs are going to be high even after the patents expire. As we get more of these sort of treatments and discover ways to reuse some of the same building blocks for multiple diseases the cost will eventually drop.

    • (Score: 0) by Anonymous Coward on Monday October 23 2017, @01:37PM

      by Anonymous Coward on Monday October 23 2017, @01:37PM (#586317)

      I wrote this up-thread: a friend of mine works on a nearly identical program, but I'm pretty sure it's for another form of cancer. The cost is that they have to custom-tailor the cure to each patient. So it's not a single drug or set of drugs, you're effectively getting a medicine with substantial alterations just for you and it takes a team of researchers using expensive equipment a few months to figure out what those alterations are.

      The goal is to invest more and more into this kind of thing until the whole process can be automated. Then the prices will drop.

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