FDA Approves Futuristic New Cancer Therapy, To Cost $475,000, Sending Biotechs Surging

The FDA on Wednesday opened a new era in cancer treatment, when it approved a landmark, futuristic new gene therapy-based approach to treat childhood leukemia, one which has produced unprecedented results in patients with the deadly cancer. Even the FDA called the approval "historic."

"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.

The CAR-T cell treatment, developed by Novartis and the University of Pennsylvania, is the first type of gene therapy to hit the U.S. market, and one in a powerful but expensive wave of custom-made "living drugs" being tested against blood cancers and other tumors. The therapy is made by harvesting patients’ white blood cells and rewiring them to home in on tumors. Novartis’s product is the first CAR-T therapy to come before the FDA, leading a pack of novel treatments that promise to change the standard of care for certain aggressive blood cancers.

"This is a brand new way of treating cancer," said Dr. Stephan Grupp of Children's Hospital of Philadelphia quoted by the AP, who treated the first child with CAR-T cell therapy — a girl who'd been near death but now is cancer-free for five years and counting. "That's enormously exciting."

This first use of CAR-T therapy is aimed at patients ill with a common pediatric cancer — acute lymphoblastic leukemia, or ALL — that strikes more than 3,000 children and young adults in the U.S. each year. While most survive, about 15 percent relapse despite today's best treatments, and their prognosis is bleak.

The therapy will be marketed as Kymriah.

The price tag: $475,000 for a course of treatment. While the amount sounds staggering to many patients, it was far less than many analysts had expected. Still, David Mitchell, president of advocacy group Patients For Affordable Drugs, met with Novartis yesterday to talk about "how to arrive at a fair price for its new CAR-T drug," but said the meeting was "disappointing."

While the treatment’s approval had been seen as a foregone conclusion for months according to Stat News, its potential price has been the subject of speculation and debate. On Wednesday, Novartis revealed that it would charge $475,000 for a course of treatment, a price Bruno Strigini, the company’s head of oncology, said would allow (a few very wealthy) patients to access Kymriah while providing Novartis a return on its investment. The cost is well below Wall Street analyst expectations, which reached as high as $750,000 for a dose. And it’s cheaper than the roughly $700,000 price tag that U.K. regulators said would be fair considering Kymriah’s benefits.

In an attempt to make the drug more affordable, Novartis said it was working with Medicare on a system in which the government would only pay for CAR-T treatment if patients respond within a month.

Meanwhile, trial results of the drug have shown unprecedented success: in a clinical trial, a single dose of Kymriah left 83% of participants cancer-free after three months, results oncologists have hailed as a major advance for patients with few other options. The most frequent side effect was an inflammatory storm called cytokine release syndrome, a reaction to CAR-T that can prove fatal in some patients but is commonly controlled with immunosuppressant drugs.

“I think this is most exciting thing I’ve seen in my lifetime,” said Dr. Tim Cripe, an oncologist with Nationwide Children’s Hospital, at an FDA meeting on Kymriah in July.

Being truly revolutionary, the therapy has the price tag to justify it: the reason for the sky-high price of every treatment, unlike well-understood pills and commonly injected biotech drugs, CAR-T presents a radical new paradigm for doctors, regulators, and payers. CAR-T treatment uses gene therapy techniques not to fix disease-causing genes but to turbocharge T cells that cancer too often can evade. Researchers filter those cells from a patient's blood, reprogram them to harbor a "chimeric antigen receptor" that zeroes in on cancer, and grow hundreds of millions of copies. Returned to the patient, the revved-up cells can continue multiplying to fight disease for months or years.

It's a completely different way to harness the immune system than popular immunotherapy drugs called "checkpoint inhibitors" that treat a variety of cancers by helping the body's natural T cells better spot tumors, according to the AP. CAR-T cell therapy gives patients stronger T cells to do that job. For some patients, the new CAR-T therapy might replace bone marrow transplants that cost more than half a million dollars, noted Grupp, who led the Novartis study.

"I don't want to be an apologist for high drug prices in the U.S.," Grupp stressed. But if it's the last treatment they need, "that's a really significant one-time investment in their wellness, especially in kids who have a whole lifetime ahead of them."

It remains unclear how lucrative a business opportunity Kymriah presents. There are about 3,100 new cases of ALL each year, but roughly 70 percent can be pushed into remission by standard therapy. That could leave just a few hundred patients who might be eligible for Novartis’s therapy, casting doubt on whether the company can get an outsize return on what will be a substantial manufacturing investment. Bloomberg estimates that the gene therapy is expected to generate $111 million in 2018 revenue, reaching blockbuster status, and $1 billion in sales, by 2024

However, as Stat News points out, the potential of CAR-T - a hot research area across several drug companies - goes far beyond Wednesday’s approval. Novartis is developing Kymriah for use in lymphoma, and its pipeline includes other CAR-T therapies targeting an array of blood cancers. Kite Pharma, recently acquired by Gilead Sciences, is awaiting FDA approval for a lymphoma therapy and is, like Novartis, developing a bevy of cell therapies it hopes can treat tumors liquid and solid. Juno Therapeutics, which slipped into a third place after its lead CAR-T ran into safety problems, has a similar focus.

News of the CAR-T's approval sent the Nasdaq Biotech index surging to its biggest gain since June 21, up as much as 2.3%. Two-thirds of the 160 stocks in the NBI are higher, with six names making new 52-week highs: PRTK, SGMO, BIIB, EXEL, FOLD, MYGN, while the IBB, the iShares Nasdaq Biotechnology ETF, is within a few dollars of 18-month highs reached in late July.

Comments

adanata Xibalba Wed, 08/30/2017 - 17:11 Permalink

 These unspeakable bastards..... there are MANY effective methods for preventing, treating and curing cancer and the genocidal "cancer industry" uses virtually NONE of them. They have "failed" because that was their intention; to protect their "medical" monopoly and their vulturous methods of killing you while stripping you of your wealth, all the while suppressing effective treatments and cures; crushing anyone for communicating healthy, natural alternatives [combined modalities; ketogenic diet, near infrared sauna, Rick's Oil, B-17/Laetrile, hydrogen pyroxide, cesium salts and many more]. There's a special place in hell for these sub-human scum bags.If you're diagnosed with cancer, I suggest you realize you and you alone will need to do the research that can save you. No one aligned with the medical industry will do it; they'd only lose money and that is not their goal. Look for REAL statistics; i.e. "early diagnosis" simply means they can start making more money from you earlier, mammograms are harmful and, again, early detection won't help you if you are going to accept slash/burn "medicine".  Ditto most other $$$$$$$ tests. I believe, as the globalists have poisoned our food/GMO, processed, anti-nutrient and water/fluoride and chlorine et al, while telling us we can meet all our dietary needs if we eat what they call a "balanced diet" and we don't need nutritional supplements etc, mandating poison "vaccines" and spreading known carcinogens into every household, cancer is slated to be a primary method for the "soft kill" of humanity on the trans-human road to world population reduction.

In reply to by Xibalba

kochevnik adanata Wed, 08/30/2017 - 19:39 Permalink

If you look at moat USA soil, it is mostly silica with little organic matter. There was study at Ellis island, which found skulls of USA children change shape from their parents. I suspect malnutrition. That is why unimporation and more domestic production will make Russians more healthy

Cancer treatment is best by prevention. For example, sugar is fertilizer for cancer cells. They do not like cyanide, released locally by the body from apricot pits

In reply to by adanata

Utopia Planitia adanata Wed, 08/30/2017 - 21:09 Permalink

If you want to treat YOUR cancer by adjusting your diet and TV-watching habits nobody is going to prevent you from doing so.  Most people prefer to use a treatment that at least gives them a chance at life.I do not know if you have any post-secondary education.  I have several advanced degrees in science, and have done medical R&D.  The human body is a remarkably complex system.  Once something like cancer strikes you have very few options that are going to give you a chance.  One of the complexities is that each different type of cancer requires a different approach for treatment.I will not suck up additional space debating with you further.  My main point is:  You make your choice and let others make theirs.  Or are you the proverbial dictatorial progressive who wants to make all decisions for everybody?

In reply to by adanata

adanata Utopia Planitia Thu, 08/31/2017 - 00:19 Permalink

It was 1966 when the first medical doctor I worked with told me point blank, "They'll never let anyone cure cancer" because of the money to be made. This was in response to the AMA (created; in addition to patent "medicine" Big Pharma, by the world's most fervent eugenicist; David Rockefeller) crowing about shutting down a clinic where cancer was being cured. I was so naive at the time, I didn't believe him. But forty years of medical research leaves NO DOUBT whatsoever regarding my conclusions.. You have either been indoctrinated by the medical industry or are part of it. What can cancer not tolerate? Where can it not survive? High heat; hyperthermia; which they do effectively in Germany and elsewhere (but you can do at home in a sauna). The absence of sugar/carbohydrate also a proven treatment/cure and known since Otto Warburg won two Nobel Prizes proving it. Cancer cannot survive in an alkaline environment which is why cesium salts were used to cure cancer before the creation of deadly Big Pharma and the Rockefeller controlled medical "industry". Hypoxia; low cellular oxygen is an absolute constituent of cancer; you cannot develop cancer unless you're hypoxic. Hydrogen peroxide (pharmaceutical grade) baths can oxygenate the cells of you body... and that's just the tip of the iceberg. You are ignorant regarding this subject matter. Btw ... if you have several "advanced degrees" in science et al, you need to sue the schools and get your money back. I'm sure you'll need it for your medical care..

In reply to by Utopia Planitia

caconhma ET (not verified) Wed, 08/30/2017 - 15:11 Permalink

Promises, promises. Remember President Nixon's war on cancer 50 years ago? It is still going on with Big Pharma and the WallStreet making a fortune and cancer patients live just few months longer with terrible side-effects.One of my relatives died from a pancreatic cancer before being treated. At his funeral, my cousin who is MD and a professor and specialist in cancer treatment said: He was very lucky dying without being treated!

In reply to by ET (not verified)

subversion caconhma Wed, 08/30/2017 - 15:16 Permalink

Lost a loved one as well to the system. Their treatment is what really kills people destroying the bodies ability to fight and then they blame the cancer when they die.While people like Burzynski are harassed and ignored by an industry that collects 100s of billions for "research" why would they ever want a cure. Many people are doing their own research and are finding out ways to solve this outside the medical community because that "community" really does not care about the health and life of people.

In reply to by caconhma

NoDecaf Bay Area Guy Wed, 08/30/2017 - 16:15 Permalink

We live in a non-binary self identifying world now. If they want to say billion but meant million then whats the problem? What are you racist or something? Get lost with your fake news and fake 'rithmatic. Stop trying to culturally appropriate math to fit your political agenda, we all can see what you're up to.

In reply to by Bay Area Guy

PrezTrump caconhma Wed, 08/30/2017 - 16:16 Permalink

"In an attempt to make the drug more affordable, Novartis said it was working with Medicare on a system in which the government would only pay for CAR-T treatment if patients respond within a month."THIS is the problem. That now they go to medicare to get EVERYONE to have to pay for it therefore causing soaring insurance premiums.  LYNCH THESE MOFOS.

In reply to by caconhma

RRA_223 caconhma Wed, 08/30/2017 - 16:21 Permalink

As a cancer survivor - twice - I'd say that Big Pharma probably does make a ton of money off cancer; but the alternative is that when they don't, there's no innovation or "investment" in the next potential cure.  30 years ago, the identical me would have been dead already and my two children would have never been born.   So your anecdote misses the majority of cancer patients who actually have options (and chances) - unlike that of pancreatic cancers, which tend to have atypically high mortality rates over very short time frames.  Might as well say "Big Auto" is making a killing over all those airbags and regulations the Federal Gov imposed for safety, and thus caused vehicle prices to increase dramatically.  

In reply to by caconhma

Creepy_Azz_Crackaah (not verified) RRA_223 Wed, 08/30/2017 - 16:36 Permalink

Well said.

In addition, if Big Pharma were making YUGE(!!!), ungodly, profits, simply invest in them and make a killing. Almost all are publicly traded. (Tip: They are not at the top of the profit makers by industry.)

Cell phones cost thousands of dollars and were the size of attache cases when they first came out. Give technology some time, and competition... prices will dramatically come down. Another Big Pharma will have a competing cancer cure soon. Or, we could beat the hell out of Big Pharma, as many want to do, taxing and regulating them until they can't afford research and innovation. Game over.

In reply to by RRA_223

Creepy_Azz_Crackaah (not verified) Deathrips Thu, 08/31/2017 - 12:30 Permalink

An analysis by Electronics Australia found that a typical 'Rife device' consisted of a nine-volt battery, wiring, a switch, a timer and two short lengths of copper tubing, which delivered an "almost undetectable" current unlikely to penetrate the skin. Several marketers of other 'Rife devices' have been convicted for health fraud, and in some cases cancer patients who used these devices as a replacement for medical therapy have died.

In reply to by Deathrips

drstrangelove73 caconhma Wed, 08/30/2017 - 16:34 Permalink

Medical Oncologist for 35 years-University of Texas and NIH.All due respect,you guys are way off base.Most of what I do these days was undreamed of when I began.Treatment and cure rates for many malignancies have improved dramatically ,though there are many malignancies,and pancreatic carcinoma is one of them,for which the therapy we have is not very effective for most patients.Its a terrible disease,and a bad trip for many with or without treatment.Most of the misery comes from treatment given to patients who are unlikely to benefit.The 2 most difficult encounters I've had today We're with patients who had metastatic cancer and were in such poor shape they are unlikely to benefit even with very effective therapy which doesn't exist for the diseases they have.Why were the interactions difficult? Because neither could believe they'd be better off not having treatment!!They could not accept it...

In reply to by caconhma

drendebe10 drstrangelove73 Wed, 08/30/2017 - 18:48 Permalink

Cost effectiveness anakysis would be helpful for what you all do, your limited successes not withstanding. @ what point is enough, enough with surgery, XRT & chemotherapy. Much of cancer patient pain suffering treated with large opioid doses might be better managed with nonsurgical treatments like nerve ablations but most oncologists are reluctant to use these types of quality of life treatments . Go figure

In reply to by drstrangelove73

pods WillyGroper Wed, 08/30/2017 - 15:45 Permalink

I thought about that post from Karl. This one is a different drug though. Not an anti-inflamatory, this one is really complicated therapy.I wish them the best of luck because this is the cutting edge of therapy, but the cost is crazy.  Not outrageous, but still high. The hepatitis therapy is outrageous because of the ROA versus this one.  There is a lot that has to go on with this therapy. And at least they are looking to help the immune system instead of poison the body with chemo.pods

In reply to by WillyGroper

pods Modern Cimmerian Wed, 08/30/2017 - 17:12 Permalink

A simple oral therapy that costs this price. This one involves drawing blood, spinning it down, separating the white cells, multiplying them and then doing their magic on them to make them kill cancer cells, then sterile filtering into a bag for administration.  (that is the simple version)What this is, in reality, is a tailored batch of drug for each person that receives it.  A small CTM (Phase I) batch can cost up to 100 grand for anything outside of oral delivery.  This therapy is basically making a gmp batch for every person that receives it. While trying to recoup the development costs.  It is a shit ton of money, but in reality, this might have a tough time turning a profit by the company. Especially if 75% of the people with the indication can be treated with conventional therapies.But this is cutting edge stuff and Novartis would like to be the brand leader in this area for future indications and medications.  pods

In reply to by Modern Cimmerian

Overfed pods Wed, 08/30/2017 - 19:08 Permalink

What most people fail to realize is that the R&D on a product like this is astronomical. I've known a few people who have died of cancer, and I can tell ya' that the "natural" treatments like calcium, coffee enemas, hash oil, and so on are total bunk.This is quite a breakthrough.

In reply to by pods

mkkby pods Thu, 08/31/2017 - 00:57 Permalink

Finally an intelligent comment.  If this really works, it will be applied to many other cancers/diseases.  In a few decades robots will do the work and reduce the cost significantly.Think about the first computers.  Huge expensive machines that were labor intensive to build.  Thirty years later they're mostly produced cheaply in automated factories.The technology/science is what's important here.  Engineering will bring down the costs, as will  competition once it goes off patent.But yeah, for the next 10 years you are dead unless you have $500k.  Just like in 1955 -- no way non millionaires could afford a computer.

In reply to by pods

Utopia Planitia Modern Cimmerian Wed, 08/30/2017 - 21:21 Permalink

Sounds like you do not value your life much.  That is your choice.  Some of us value life a great deal.How do you plan to do the R&D that is required at no cost?  How to you propose doing the tens of thousands of hours of research, in expensive labs, following piles of government regulations, etc. etc. etc. to create a new therapy?  If you do not wish to participate in any of this then don't.  But don't tell the rest of us we should just sit back and let a treatable disease take someone we love because YOU don't think it is worth it.

In reply to by Modern Cimmerian