E-mail updates
Follow on Twitter
Subscribe to RSSDoctors at the Children's Hospital of Philadelphia used Xalkori to treat children with anaplastic lymphoma, a disease caused by gene mutation. NBC's Robert Bazell reports.
By Robert Bazell, NBC News correspondent
Tonight on "NBC Nightly News" we heard the story of Zach Witt, a vivacious 6-year-old who was close to death from a rare form of lymphoma. But he was brought back to health by a drug that has been on the market as a treatment for a form of lung cancer. It truly is a heartening tale.
The backstory to this achievement shows how progress is being made against certain cancers. In some ways it is very encouraging. But viewed in other ways, the progress is far slower than many would have predicted.
Before the late 1970s and early 1980s scientists had no idea what happened inside cells to make them cancerous. Then a series of discoveries revealed that the same genes that control cell growth and division as a fertilized egg becomes a human being can also cause cancer when the growth control genes become mutated.
Zach Witt's parents, John and Pam, describe Zach's battle against lymphoma and his remarkable recovery.
After these mutations were discovered the great and obvious hope was that there would be drugs to target them and stop the cancer from growing. There have been several targeted therapies, but far fewer than anyone expected. The gene mutation that drives Zach’s tumor, known as ALK, was discovered 25 years ago. But companies had little interest in developing a drug for that type of cancer –- anaplastic lymphoma -- because it affects only a few hundred children a year in the United States.
Eventually scientists discovered that ALK is also a driver of about 10 percent of lung cancers. Lung cancer is so prevalent that even 10 percent makes a substantial market. After extensive testing Pfizer won approval to market Xalkori to treat lung cancer with ALK – at a cost of about $100,000 a year for each patient. The drug works by binding with and inhibiting the action of the enzyme that is produced by the mutated gene.
And in a study out Wednesday, doctors at the Children’s Hospital of Philadelphia have shown that in eight children Xalkori can very effectively treat Zach’s type of lymphoma. In each child, evidence of cancer disappeared. The research will be presented at the annual meeting of the American Society of Clinical Oncology in June.
Scientists are decoding the genes of many cancers now, looking for situations like this where, because of a similar gene mutation, a drug already out there -- or combinations of them -- might help other cancers. But it is going slowly. The biology is not as simple as one gene mutation causes one cancer. In fact, there can be hundreds of gene mutations contributing to one type of cancer. Many scientists caution against expecting too many spectacular results.
As Dr. Yael Mosse, Zach’s doctor, put it, “Our goals have shifted. Now we feel that it is more impactful to make a big difference for a small group of patients rather than a small difference for a big group of patients.”
Leave a Comment:
Explain to me why a lifesaving drug's production is being entrusted to the holder of the patent. $100k for treatment per patient? Remind me how much it costs to produce these pills? How about adding in the R&D costs on top of the variable costs just to be fair. I bet it's still WAAAAAAAAAY below the $100K price tag...especially if Pfizer merely yanked the patents from a university and hired on their grad-students through the FDA vetting-phases.
I think that the world needs to get tough on how these drug patents are handled. The vast disparity in pricing between generics vs. brand is more than enough indication to see how a monopoly prices vs. a competitive market.
But of course, we don't want to remove the incentive for drug-makers to stop researching new drugs...but how about we stop with this vertical integration-nonsense and require that they not have the sole control over the production of the product they have sole ownership of the design.
I think it's high time that the makers be split from the manufacturing so that we can do away with these artificial shortages. The makers should be required to license the production by lump-sum agreements with multiple arms-length producers.
The more the patent-holder charges, the more their licensees are likely to produce. If there's not a whole lot of demand for the drug (too specialized), simply subsidize production among a small coalition of producers...but again, never once will any life-saving drug be produced by a single entity to hedge against price manipulation or shortages from problems in production runs.
You obviously have no idea of the time and money that goes into developing a new drug. You also do not seem to comprehend that for every drug that makes it to market there are many more that do not. The lost R&D money on the drugs that do not make it has to come from somewhere. The successful drugs not only need to recover the costs of their development, they need to fund all of the R&D that goes on at the drug company.
As for shortage, drug companies do not create artificial shortages. Many drugs are made in runs once a year. The amount of the drug that is anticipated to be needed is run off and the production line is switched over to make something else. There are very few drugs that have high enough demand that they are being manufactured year round. As such, if demand exceeds projections shortages can develop. It takes time to start up production of many drugs so shortages can persist for some time until a new production batch can be made. Many drugs are not simply mixing together some different chemicals and putting them in a tablet or capsule. There are processes to manufacture the drug that take time to initialize and run to completion. Please take some time to learn about how drugs are made before you comment. You would be surprised hat goes into making some drugs. And for the record, I have no connection what-so-ever to and drug company.
i don't see how our thinking will make it acceptable to tell someone who needs a cure that they can't have it due to money. in india, for example, cost to the patient and society, or the larger health care system which absorbs the costs, is a big part of a patient's treatment plan and so it's tied to the person's expected outcome. this raises ethical problems.
as for bringing drugs to market, it's expensive for sure. however, we can't forget that a lot of the basic science and research used by drug compnies in the development of new therapies comes from public- funded uiversities and research centers.
$100,000 for a course of treatment for cancer is not uncommon. in fact, it's actually rather modest. if it's an accepted therapy, insurance is pretty much required to pick up the cost.
JS well said. Seriously thinks new and innovative drugs are grown from trees. FDA approval or disapproval coupled with the vast scientific research costs/ tests as well as future complications of said drugs play well into the minds of drug manufacturers. History has shown pushing a helpful drug into the market too soon could result in lawsuit after lawsuit when it's shown that these promising drugs damage heart valves, or cause some horrible side effect that render the drugs/ patients odds, of survival vs. complications/ interactions, useless. Hate to say it but breakthrough drugs and their helpfulness are an enormously complicated endeavour.
So you obviously buy into the same line of reasoning and misinformation regarding
big oil. Big oil, big Pharma, and the medical industry in general are very similar
in that you bleed all blood you can from that turnip, before you discard it --
all in the name of ineffective,same rathole research and results. You charge
all you can, for as long as you can - until either market pressures or legislation
change the scenario. Big Pharma profits come at the expense of your money
or your life. This makes "blood diamonds" look absolutely pure in comparison.
Why is it that the same medications in Europe, Japan, Africa, China, Mexico, etc.
- are often 1/4 or less than priced in the U.S. -- because big Pharma prices to
what the world markets will bare. Life is cheap in Africa, therefore not a great
deal is going to be spent on expensive drugs. Big Pharma would rather make 5% than 0% - so therefore priced accordingly. Besides the U.S. market will move that 5% to 5000%. Europe, Japan, etc. have pricing controls via single payer insurance. Big Pharma is another example of over rampant capitalism at its worse - just like Wall St. investment bankers. It is obvious that only tie you have to big pharma is that you have been completely brainwashed somewhere along the line - that is if you truly believe what you indicate you do. Of course there is the possibility that you are a big Pharma troll.
I recently watched a documentary about a Dr. Brusynski its brusynskimovie.Tells alot about the fda and the drug companies as partners
Sorry Burzynskimovie
Seriously?? Seriously?? You are an idiot. Hey, let's pretend we know how much money a company spends to find a cure for cancer and dictate to them what their pricing should be. You have made us all dumber by reading your acked out post.
You've got it all figured out!!! Maybe you should run for President and get the country out of debt.
It just blows my mind to read how some people actually think.
Wow, such vitriol. Sorry to mess in your cereal this morning, everyone...LOL
Anyway...I'm up for alternatives...but am I the only one that sees a problem with life-saving drugs/treatments that would literally bankrupt most people to be able to receive, yet cost far far less to provide compared to how they're priced?
I'm well aware of the costs for developing and vetting of pharmaceuticals as they go through the myriad of trials in order to meet FDA approval...funny how amazingly profitable these companies manage to remain regardless of that...the way some posters on here behave, you'd almost think these were non-profit companies doing philanthropic work!
(I personally think that if the FDA were adequately staffed and had sufficient oversight, the vetting processes could probably be shortened and cost everyone A LOT less money.)
BTW, to the naysayers about price-manipulation, let's review a few notable examples
NY Times - 2004
Reuters - 2011
WSJ - 2012
CBS News - 2012
Considering the pricing disparity between the US and Canada or Mexico for the exact same pharmaceuticals, what more proof does one need to see that these companies are not operating competitively and are able to actively discriminate between customers and try to maintain a captive market (read: US citizens) by lobbying the US government.
Now that we got the sources out of the way, let's talk about the supply and price issues.
I suggested above requiring multiple producers for these products; figuring that by increasing supply and reducing/removing the ability for the patent-holder to also dictate the market-price, it would increase competition and drive down overall prices. And just as importantly, the patent holder receives royalties on the licensing, so they profit from both their own production runs as well as royalties.
Overall, the problem lies in the fact that we have a highly inelastic market-demand for numerous types of drugs/treatments, and an issue of monopolization which further encourages the raising of prices. A completely unfettered free-market leaves many without the ability to afford medication, and a totally regulated market removes the incentive for developing new drugs/treatments...it's not an easy fix.
Care to propose your own suggestions?
$100000 a year per treatment ,I guess the poor people are screwed.
Not only that but soon the drug company(s) making the drug will manufacture a fake "drug shortage" to jack up the price even higher. Pharmaceutical companies only care about making perverse profits not people, and as such have no right to perverse profits. People are worth far more than profits.
Nothing new there... In the future, you want to visit Mars? You'd best start saving you're millions today.
When you are fighting for your life...cost doesn't matter.
Godspeed to the researchers...and for those needing treatment, just hold on a little longer! Costs will eventually come down...
There is no point in having a cure to a disease if the people dying from the disease cant afford it.
I remember back in my country when some poor couple try to ask for treatment for their 6 months old boy. The doctor asks if they have any money and then they said "we'll pay you back the rest later, this is all we have". The doctor then replies "No money, no treatment, your son can drops dead for all I care".
Sound familiar?
cost doesn't matter is our thinking...but it's not reality. as the gent above say, in some places no money, no medicine.
try getting into the md anderson cancer center...with no money. it will never happen.
Dee,
I'm very aware of the MD Anderson Center and being turned away.... I was working full-time for a hospital no less. Great insurance and all. Anyway, diagnosed with cancer and lived relatively close to MD. I figured I'd get a 2nd opinion there and talk to them about treatment. Well, they took my insurance info and then invited me to come. They let me know that I needed to bring a check for 50K, yes, $50,000. Turns out my insurance wasn't so good and that was to cover any "out of pocket" expenses. Thanks, but no thanks, I'm taking my chances with the "cheap" oncologists... shame really.
"When you are fighting for your life... cost doesn't matter."... Typical 1% logic.
Though in essence this is true, but in application it takes your money to determine their services and in many scenarios, your money will never be enough for their services. To give up your way of life, sell your home.. Your vehicles, any land, 401 early withdrawal and clean out your bank account with the chance of being cured is not good wisdom when you discover that your now cured but but live on the streets.
Saul...
Have you been diagnosed with stage 3 lymphoma? My wife has...trust me, you'll do anything...pay anything...and live on the streets, if you have to!
BTW Saul...I am NOT a 1%er...very far from it, pal!
Can you imagine how crowded the world will be when cures are found for everything?
Which is why we should be colonizing other planets.
dubya wanted to go to mars...he should have.
Morlack,
Colonizing other planets will do little to solve any population issue on Earth. It just isn't going to be cost effective to ship the "excess" people to another planet safely -- the transport costs will be too high (both in financial terms and "environmental footprint" terms). The most we would ever do is send breeding pairs or human genetic material to create "test tube" babies on other planets. These won't offload a significant number of humans from this tiny, insignificant, and (in terms of the universe) completely irrelevant orb of rock we call home.
The only way to deal with the inability of Earth to sustainably support an unlimited number of humans is to reduce fertility of Earth dwellers -- hopefully voluntarily.
The only reason I can think of for humans to colonize other planets is to prepare for the day when Earth is unable to sustain human life. For example, when the Sun gets so hot that temperatures on Earth are too high for humans to survive. However, since humans are pretty bad at evolving, something else will probably get us long before then.
I say that humans are bad at evolving for two reasons. Most importantly, we take too long to reach fertility and there are too few of us -- so there are relatively few genetic mutations being tested every million years compared to, say, ants or roaches. Secondarily, but fixable, humans go to great lengths to keep undesirable mutations in the gene pool, and actually let them breed, rather then letting individuals with such mutations die.
As the daughter of a pancreatic cancer victim ( and yes I said victim) I would love to see a cure for all cancers. While we have some person saying that in order to control the population we need to allow those that are ill to die as an expedient way to keep the population down, I say ... go pound sand!!!
I watched my mother die a HORRIBLE death, and when things got to the end, you prayed for her to be out of the pain. HOW DARE YOU!!!! I won't say what I am really thinking, but I'm pretty sure you can guess.
As for the children in this story, I am so grateful for them and their parents. They get a chance at life and that is good for them.
The only benefit from colonizing another planet is for corporation who sponsor it to get rich. Watch Avatar? That is how big corporation is doing with all the mines throughout the world.
i know you really just didnt say something that stupid. lets make sure that you and your loved ones dont ever have to worry about that.... keep your illnesses and diseases and be happy with those you are family with... we all want the "cure" especially for our children... i hope your not a parent, i feel sorry for that child. i pray he/she never gets sick.
"Colonizing other planets will do little to solve any population issue on Earth".. (whatslegal)
Your looking at it wrong, other planets will be for the wealthy.. That should clear out the lot of 'em including politicians THEN the rest of us left behind might be able to balance the budget and become a wonderful Nation again.. (well, I can dream).
Yes I have kids and no you don't need to feel sorry for them. They will be raised without blinders on.
So lets discuss the real problems we will have when there is limited death. Do you think staving to death will be more fun than dying of cancer? Do you think being killed in riots will be more fun than cancer? How about the wars that will be fought over resources? Hey if we can keep people from dying of cancer, Aids, heart disease, etc. that's just more people we can send to war to die.
Dying is not fun or glamorous it's part of life and it will happen to everyone. Learn to live with it. Oh and some of you are not the only ones who have lost family to cancer.
Improved education per capita followed by improved standards of living does wonders to reducing population growth.
Look at every country with such figures
The Economist - 2009
We don't need to go out to colonize other planets in order to satisfy some sort of never-ending population increase. We need to colonize other habitable planets in order to reduce the likelihood of humanity being wiped out in some mass-extinction event like the Earth has gone through every couple million years.
Plus, just the added benefit of drawing resources from space (which is going to be a necessity if we want to delve into deep space) as opposed to relying solely on the Earth for all of them would do wonders towards improving the Earth's environment and re-prioritizing land away from (non-farming) industrial use to instead be used for people or nature.
Google RUN FROM THE CURE. You can grow your own cure in the back yard.
worn-0ut 4024611 I have been doing just that. I used to hang out with my grandpa. He showed he what and how to used different plants/herbs to treat certain ailments. In his early years doctor rarely treated blacks. He wanted me to be prepared.
Your Grandpa was a very smart man. Stay strong my friend.
There are lots of herb or natural cure for lots of illness. The problem is the drug company don't want a cure, they get more profit with treatment than cure.
Even if there is a cure for cancer comes out tomorrow, it will be hide away or denounce as some whack method so the corporation can make more money from treatment.
cancer is miserable, for sure. i hope we can figure out, as a society, how all people in need can take advantage of the advances in treatment.
The advances in treatment only advance when there are millions in profit to be made from it. They even have the audacity to admit it. See my post #8.
Greedy thieves.
Its all about money period.
They don't give a @!$%# weather they cure any body.
Just make money if it fails then the greedy lawyers make money.
"The gene mutation that drives Zach’s tumor, known as ALK, was discovered 25 years ago. But companies had little interest in developing a drug for that type of cancer –- anaplastic lymphoma -- because it affects only a few hundred children a year in the United States."
Doesn't that one statement really tell it like it is? Only a few hundred childrens lives a yr in the US are not worthy of being saved because there is no money in it. So therefore, the only reason a few thousand are worthy of being saved is because there's millions of $'s of profit at stake. Certain elements of humanity becomes more and more dispicable every single day that passes. What a pathetic bunch we are, the human race.
In addition -- treating specific cancers is generally a failed long term strategy. Instead we
should be treating the conditions which allow cancers to exist - basically for most people - immune
systems which have not evolved at the same rate as damaged chromosomes - due to increased
exposure to myriads of environmental carcinogens. Any fundamental breakthroughs involving
improving the "smarts" of our immune system, or the de-cloaking of common cancers, will be
breakthroughs indeed, and will benefit almost all cancer patients. Unfortunately in relative terms
very few research dollars go this way.
At a $100,000.00 a year for treatments the insurance companies would let someone die before paying out that much. Medicine stands to make more treating cancer than finding a cure.
So, they wouldn't develop this drug because it only affected a few hundred patients a year and therefore it was not profitable to do so? Someone needs kicked in the head!!
Furthermore; yes, generics are cheaper. But veterinary medicine is even cheaper. For instance, I use an albuterol inhaler as a rescue inhaler- for asthma. I was paying about $35 per prescription with insurance. At the same time my wife was working as a technician at an animal hospital who told me that they were paying $3 a prescription for the same medication from a wholesale distributor. Now, you can argue about purity and safety all you want, (my argument isn't based on pills or other meds with a short expiration time or the potential for massive over/under dose issues) but this was the same exact drug in the same exact packaging (an inhaled drug in a metal canister and dispenser) that you would buy at your local Wally World Pharmacy.
Now, when it comes to saving lives and adding to the comfort of a patients life; how is any of this morally right?
I'm available for a small fee.
"The gene mutation that drives Zach’s tumor, known as ALK, was discovered 25 years ago. But companies had little interest in developing a drug for that type of cancer –- anaplastic lymphoma -- because it affects only a few hundred children a year in the United States."
So the "few hundred children" don't matter, right?? They apparently are expendable. And $100K a year? Once again, something only for the rich. The poor need not apply. Bastards!
Read Melody Petersons Book about drug company corruption and greed titled Our Daily Meds.
Gee, WhatsLegal, you wouldn't happen to be German, would you??? Seems to me I've heard something before about "undesireables" being weeded out....
this is amazing! this is bittersweet. this is something that the government has been sitting on for YEARS! we all know it, then when they knew it was the best time to be the hero, they mysteriously give it to help this little boy! its fabulous that it happened! he is blessed. but the only hard part of understanding that i have is how is it we can pay someone MILLIONS to throw a football or act in a comedy sitcom, but we cant offer something to save someones lives for a fraction of the cost..... seriously? how can anyone who knew about this particular drug keep it under cover while year after year watched others die around them? for what? a litle government money, maybe some 10 minutes of fame? please... we all know what the government is all about. sooner or later they will be judged. i just hope i get a front seat to that judgement day, and i hope the jury of peers is gonna be all the souls that could have had their demise prevented just for a little while longer.....
to js in sd, I think yu are either a "wannabee" or yu have mucho dinero - invested in the drug companies.
Why those "poor" ole drug companies only make thousands of % profits on "MOSTTTTTT, ... of their drugs. FYI.
Several years ago a good friend of ours had cancer, he was given a test prescription and started getting better. Walking more and not sleeping as much; then the FDA pulled it off saying it was bad. So I would venture to say if it is too good they will pull this one also.
Was probably hemp oil - they wanted to see if the anecdotal claims were real or not. :-)
Search "run from the cure"
This is why there will never be a cancer cure.There's NO MONEY in it. Billions spent every year on so much research that cancer "research" is a business unto itself. And any drug produced will be priced to remburse the drug company first. The powers that be in Washington D.C. are so beholden to big pharma for campaign money that they will never change the rules, which is unforunate for anyone not in the 1%. And unfortunately, so many Americans are brainwashed to believe that these people have your best interests at heart.
At a cost of about $100,000 a year for each patient, who has that kind of money get real, greedy people, no morals. STUPID GREED.
Basic Research - testing theory
Lab Research - formulating compounds
Clinical trials - testing on lab animals
Phase one study - small number of human volunteers to determine efficacy and tolerable dosage
Phase two study - larger group of human volunteers to determine unwanted side effects
Phase three study - really large groups in final medical demonstration and data collection
FDA approval application
Crank up production and marketing if approved. Hope you have disclosed everything to avoid lawsuits.
New drug development is extremely risky and expensive. Few companies can afford the years and multi-million roll of the dice on a new drug that can be killed off at any of the major steps. To make it worse, the patent clock starts running on filing, usually long before a viable drug is developed. The investment must be recouped before the patent expires if management wants to stay employed.
You conspiracy and secret plant people need to re-line your propeller beanies with fresh tin foil.
Dr. Brusynski in Houston should sue the drug company, the FDA and Congress. That rail roaded him when he found this cure of cancer years ago. He took many of his patients that were gave no chance of living to Congress.Saving them and curing their cancer 100%. Now the drug company and FDA gets all the money and credit for what Dr. Brusynski found years ago? As the world turns in America with greed of the almighty dollar over the well being of us. Dr. Brusynski problem was he found this cure with no chemo. Imagine how much money the drug companies would lose.
I have been reading the prior posts (with a certain amount of disgust) about how the money should be spent on cancer treatments and how it should be spent on developing cures. Do any of you have cancer? How do you think this 6 year old feels about you know-nothings having his life in your hands and don't think his life is worth a measley $100,000? To let you all in a little secret, that $100,000 is the TOTAL cost of developing that drug per year. The cost to treat this little boy might be in the tens of thousands of dollars. How do I know? I have cancer, but mine is incurable. I have what is known as Multiple Myeloma. My cancer affects the blood plasma and bones of my body. There is no "magic pill" for me as there was for that little boy. When I was diagnosed I was given 5 years to live. I am in my 3rd year since diagnosis. I know what that little boy went through and I seriously doubt that anybody who has posted here has the guts to go through what he did. Ask your self one question....How much is a little boys life worth? I know what your answers will be. "HE'S NOT MY KID SO WHAT DO I CARE."
God bless you, pastrider. Sending prayers your way tonight.
$100,000 really isn't that bad folks, my kidney cancer chemo-therapy pill is $900.oo a day and it is not the most expensive treatment out there, and it is very similar to this drug.
I rejoice with this family!
Great that we are using drugs that are already out there to treat different types of cancers rather than trying to start from scratch. However, we also need to focus on making these drugs more affordable to patients.