Slashdot Mirror
FDA Approves First Cell-Based Therapy For Cancer (npr.org)
An anonymous reader quotes a report from NPR: The Food and Drug Administration on Wednesday announced what the agency calls a "historic action" -- the first approval of a cell-based gene therapy in the United States. The FDA approved Kymriah, which scientists refer to as a "living drug" because it involves using genetically modified immune cells from patients to attack their cancer. The drug was approved to treat children and young adults suffering from acute lymphoblastic leukemia, a cancer of blood and bone marrow that is the most common childhood cancer in the United States. About 3,100 patients who are 20 and younger are diagnosed with ALL each year. The treatment involves removing immune system cells known as T cells from each patient and genetically modifying the cells in the laboratory to attack and kill leukemia cells. The genetically modified cells are then infused back into patients. It's also known as CAR-T cell therapy.
The treatment, which is also called CTL109, produced remission within three months in 83 percent of 63 pediatric and young adult patients. The patients had failed to respond to standard treatments or had suffered relapses. Based on those results, an FDA advisory panel recommended the approval in July. The treatment does carry risks, however, including a dangerous overreaction by the immune system known as cytokine-release syndrome. As a result, the FDA is requiring strong warnings.
The treatment, which is also called CTL109, produced remission within three months in 83 percent of 63 pediatric and young adult patients. The patients had failed to respond to standard treatments or had suffered relapses. Based on those results, an FDA advisory panel recommended the approval in July. The treatment does carry risks, however, including a dangerous overreaction by the immune system known as cytokine-release syndrome. As a result, the FDA is requiring strong warnings.
Once it becomes FDA approved and a patent is awarded, one of the big pharma companies will come in with a blank check to the patent owner and will immediately proceed to bury this knowledge and it'll never be used again, all in favor of high-priced chemotherapy.
Let's put this in perspective. ALL already had treatments that put 98% of affected children into remission within a couple of months, with 8% of those eventually relapsing. So 90% are completely cured with existing therapies. There are other cancers where the numbers are an order of magnitude worse. I'm puzzled why the focus seems to be on diseases that medical science has already very nearly cured, rather than the ones that kill most of the people who get them.
Also, is this actually measurably better than existing treatments? If existing treatments fail to produce remission in 2% and allow a relapse in another 8%, you'd expect only about 20% of the patients to be in that 2% group that weren't helped by chemo. So a remission rate of only 83% is probably not statistically significantly different from what they would have gotten if they had used the current generation of chemo, and doesn't necessarily indicate any benefit for people who did not respond to chemo. So this could very well be a no-op, all at a tremendous cost that insurance won't cover (because it is experimental).
I'm not saying that the research isn't valuable, because it is, but in the zero-sum game of medical research, seeing the first approved cell-based cancer treatment be for a disease that is already all but cured just seems bafflingly backwards. I would have expected the first treatments to be for things like pancreatic cancer or mesothelioma or, if you want a childhood cancer with a high case fatality rate, perhaps neuroblastoma. Brain cancers kill significantly more kids than leukemia, despite being much less common.
Check out my sci-fi/humor trilogy at PatriotsBooks.
Make no mistake, NAZIS are in charge at the FDA.
i'd flip that coin
Hypereosinophilic Syndrome, or HES as it's commonly referred to is a cancer that affects roughly 1 in 18 million people. A bone/blood cancer that causes the marrow to create too many eosinophils. These eosinophils are toxic to the body in that when they die, they go 1 of 3 possible ways, each way killing cells surrounding them. Having been diagnosed with HES almost 8 years ago, after over 4 years of feeling like I'd slept in a bed of poison-ivy while having chicken-pox and a 3rd degree sunburn. At the time of diagnosis, my kidneys were failing, I had heart palpitations, my gall bladder was 4 times normal size from it trying to pull in as many eosinophils as it could, but with counts ranging from 23,000 to 54,000 per unit of blood (normal ranges are from 0 to 400, where 400 is a moderate to severe allergic reaction) there was no chance of it taking in enough to make a difference without destroying itself, and my optic nerves were being hampered with, showing up as ocular migraines, some occurring while operating a motor vehicle. To put it bluntly, I was months from death.
What saved me? A drug, designed for CML patients, and certain GISTs (gastro-intestinal sarcomas), called Gleevec or Imatinib. Within 2 weeks of starting the chemo, it had my numbers under control.
Now, if this treatment works for Leukemia, I'd imagine it could be used to similar effect with the poorly encoded genetic sequence inside my own bone-marrow.
I look forward to my next appointment with my oncologist.
and a patent is awarded,
Most of the individual steps that are involved rely on knowledge that has been published in scientific articles and spoken about in conference (I was there !) and thus constitutes "prior art".
The most expensive part (due to regulation, certification, approval, guarantee, etc.) is putting all the steps together into a process.
And that about the only thing that they can patent (because that one specifically might not have been published) the exact specific steps this process relies on.
Meaning that if such patent-trolling happens, it doesn't prevent any other company to putting together the necessary key steps (which again, are already available published knowledged, not patented) into a slightly different process (and do it enough slightly differently so that it's not covered by the above process) that can be used.
Lastly, TFS make it seem rather simple ("Hey just fix a gene in the white cell, inject it back and problem solved !") whereas in practice, it's extremely complicated and tedious steps.
e.g.: it takes a lot of cultures and trial-and-error until you get the "perfect antibody" that will work, that you can then CISPR-splice back into the patients' white sells.
It's not something that is quickly cobbled by a technician in a clinic's lab, it something that require a crew of biology/immunology university scientists and a whole bio-informatics division (= computers are used to detect potential best targets to accelerate the work of the lab team).
Meaning it's an expensive process.
Meaning that the company that decides to implement this on a large scale commercially has still lots of money to earn (they'll be basically selling the "service" of doing all this tedious steps per patient).
It's *definitely not* going to insta-kill the big pharma's golden-eggs-laying goose. Just replace one goose (chemotherapies manufacturing and selling) by another (service of adapting gene/cell therapies).
"Sufficiently advanced satire is indistinguishable from reality." - [Tips: 1DrYakQDKCQ6y52z6QbnkxHXAocMZJE61o ]
The thing is :
- these classes of methods are generic. With very few problems (it's not relying on killing the patient slower than killing the cancer. It's about specifically targetting the cancer) Eventually this method could be adapted to other cancers as well.
so the fact that they used it against leukemia isn't a major drawback for brain cancer.
(Unlike chemotherapies which rely a lot on the general charecteristics of the cancer, to find a way to poison it with a drug faster than the drug posions the rest of the patient. Different type of cancer = different type of characteristics. Poisons will therefore work differently)
- because leukemia is well studied and has already lots of studies done with other treatment, that gives a lot data point to compare against, and to combine with.
- leukemia happens to be a slightly lower hanging fruit here. (everything happens in the blood stream, which is where you'd be injecting the modified cells).
(but again, all fruits *on the same tree*. Not an entirely different tree like chemotherapies).
These are reasons to take it as a first target, before expanding to other cancers.
"Sufficiently advanced satire is indistinguishable from reality." - [Tips: 1DrYakQDKCQ6y52z6QbnkxHXAocMZJE61o ]
Cancer is more dangerous and painful than HIV. Hope they complete the therapy soon https://williamreview.com/7-ca... https://williamreview.com/7-ca...
http://williamreview.com/
For the love of Pete, why do this with ALL at first? ALL might be the most common childhood leukemia but it is the least common of all leukemia.
They should have gone after AML first, as that is the most common form of leukemia in the world, and it also the most agonizing and painful way to die of all of them.
'FDA approves new treatment for ALL cancer'
XML is like violence. If it doesn't solve the problem, use more.
studied. You have to remember - just 50 years ago, ALL was pretty much a death sentence and it tends to hit kids more than adults. 2nd - this treatment is used AFTER patients had failed to respond or relapsed. So this treatment this roughly an 85% success rate takes it from 90% to what, an almost 98% success rate (overall)? But the real question (and I haven't seen it answered by anyone yet) is whether or not it'll be effective for Philadelphia chromosome (9/22) patients. (I haven't had a chance to research that yet.) While ALL treatmentment are very good, for ALL Philadelphia patients the prognosis was still poor last I checked.
Philadelphia positive patients (9/22)
https://clinicaltrials.gov/ct2...
> genetically modified immune cells
I can hear the anti-GMO people's heads explode with this one.
Kite's $12b acquisition by Gilead on Monday may be a harbinger of anticipated success of these kinds of biotherapeutics.
Given the modularity of these CAR systems, I'd expect to see a cascade of more approvals as the therapeutic scaffold is perfected. It's not just giant pharma working on these systems either. Younger companies like Cell Design Labs, Chimera bio, Nkarta are taking very novel approaches to further refining the specificity and precision of these kinds of treatments.
The cool thing about the CAR system is that it's amenable to rational engineering from modular, biological components. The CAR approved here was the product of low-throughput painstaking labor, but many companies are trying to boost the rate of R&D.
You can dig into the structure of the approved CAR therapy here: https://serotiny.bio/notes/proteins/car19/
My father was diagnosed with stage 4 liver cancer that spread to 1 lymph node April 2016. he had 2 tumors one at 3 cm and the other at 5 cm. we were given no hope whatsoever. He was in such bad shape he couldn't get chemo or radiation. We had no choice then, other than to keep on taking him to the hospital, but all efforts to treat him in the hospital was to no avail, we Âalmost lose hope, until a friend of mine told me about a herbal doctor who cured his kidney disease, I was left with no choice than to contact the herbal doctor, because I was not ready to lose my Dad yet, so I contacted him and before the next day he sent me a mail, and I explained to him what My Dad was passing through, and he told me he could cure my Dad, but I had doubt in my mind because he asked me to send money to him to get the items to prepare the Cancer cure for my Dad, but I gave it a second thought and sent him the money, because nothing is worth more than human life, and as soon as he received it, he sent me a message and told me that he will prepare the cure in 2 days and send it to me on the third day, I was still thinking that I have fell victim of Scam, not knowing I was with the right doctor, so after 2 days he told me to send him my address, and behold I received a package on the third day from Courier delivery service, and I opened it and it was the cancer cure for my Dad, I followed his instructions on how to use the medication, and after 5 days of use I was shocked to see my Dad in a more healthy condition, and my Dad used the cure for two weeks, and the Herbal doctor told me to take my Dad to the hospital for check up, and I did, to my greatest surprise, the Doctor couldn't find any symptoms of cancer on my Dad, and this is how my Dad became hale and healthy again,Â
the herbal Doctor's name is Dr. Douglas, he is the best herbal doctor I have ever come across,
if there is anyone here that needs his help, kindly contact him on his email address via:
drikhidedouglashealinghome@outlook.com
He also has cure to various diseases such as:
HERPES
KIDNEY DISEASE
LIVER DISEASE
SYPHILIS
WEIGHT LOSS
BURNING OF FATS
ERECTILE DYSFUNCTION
PENIS ENLARGEMENT
BREAST ENLARGEMENTÂ
TREATMENT OF FIBROID OR OTHER PREGNANCY PROBLEMS
SEXUALLY TRANSMITTED DISEASES (STD)
I'm forever Indebted to this great doctor a life saverÂ
I wish every one here good luck in finding cure to there diseases or helping their loved ones get cure to certain diseases,Â
I know how it feels to have a sick person around you, it brings sadnessÂ