This however, is the real deal. It's quite remarkable and there's likely more stories like this for other diseases on the way.

I went to a gene therapy session this fall at the American Society of Human Genetics conference in Boston and was blown away by some of the success people are having. I quipped to colleagues that I felt like I was in a science fiction movie. The most remarkable one was where they used an approach similar to the one here to cure a fatal metabolic disorder (relaying this from memory, so some of my recollection may be off). Kids with the disorder have a busted enzyme that causes slow degeneration of neurons. They don't live past 6 or 7 if I recall. The team showed how modifying a certain kind of stem cell found in the body normally to have the correct copy of the enzyme cured several patients. The corrected cells naturally move to the brain where they differentiate into glial cells and produce the correct copy of the enzyme. It turns out that because the neurons in the brain are starved for this enzyme, they express receptors that allow them to take it up from the environment. So the repaired glial cells supply enzyme to the entire brain (i.e. it's not necessary to modify every neuron in the brain to have a correct copy of the enzyme). They can completely cure kids with this approach. All of their muscular and neurological tests are 100% normal.

They had videos of these kids running around and playing just as if nothing was wrong. In one case, a younger brother lived but his older sister (who was too old when the therapy came out) had died. It was hard not to get choked up looking at their smiling, happy faces as they ran around, thinking that if this therapy hadn't existed, they would be in a nearly vegetative state.

Gene therapy had a rough start with the early setbacks, but I'm getting the sense that the tide is rapidly turing.

The example that keeps coming to mind is HPV - it can cause cervical cancer etc. And, since there is an immunization for HPV, the disease is preventable, and to the extent it is the "only" cause of certain cancers, would also prevent those cancers.

So is HPV the "only" virus which causes cancer? Seems unlikely. I just don't know how much research is focused on bacterial or viral origins. Maybe it is not glamorous or mysterious enough to justify funding.

The 2005 nobel in physiology went to Barry Marshal for discovering that stomach ulcers, long a mystery, were caused by Helicobacter pylori bacteria. And in fairness to history, he wasn't even the first person to discover it, just the most recent person in western society.

http://westhunt.wordpress.com/2012/02/22/low-hanging-fruit/

The bottom line is that they are likely all correct. There is a staggeringly large amount of biology that we just don't know anything about. The complexity of it all is almost fractal. Just when we think we've got our heads around something, we peel another layer off the onion to reveal an entire landscape of which we were previously unaware. The best example of this is the recent findings in epigenetics. Before Darwin, Lamarck argued that acquired characteristics could be passed on to offspring. This was widely discredited. I learned about how it was patently false in high school. Now, we are finding that DNA methylation patterns (which work sort of like commenting out blocks of your DNA code) are modified and passed on to offspring. In some studies, if a mother is stressed, her offspring have stress response genes turned on by default at birth. In another, if there is starvation, the animal has starvation survival genes enabled at birth. It's quite remarkable and it means that in addition to our DNA which is fairly static, we have this parallel inheritance pattern that can be shaped and molded by the environment and passed to our offspring. The old "nature vs nurture" debate gets a whole lot more confusing (and might even be the wrong question entirely) with that in play.

It seems more likely that cancer can also be a disease of aging and degeneration. As we get older, things break. Cellular division is liable to be one of those things. There are identified mutations that dramatically predispose people to cancer, because one of the genes controlling cellular division is already broken (the "Knudson hypothesis").

Some researchers do prefer to use the viral cancers as a model for studying the others, because they luckily have a singularly identifiable cause, and therefore seem more likely to point to the causative mutations behind cancer as a general process. This is the premise for a paper that I helped author: http://www.nature.com/nature/journal/v487/n7408/full/nature1...

Nitpick: in the same vein, not all gastric ulcers are caused by H. pylori. Another common cause is chronic NSAID (e.g. ibuprofen) use.

https://www.khanacademy.org/science/healthcare-and-medicine/...

tl;dr

For whatever reason, Breast Cancer prefers to metastasize bone (which is a phenotype). Something as simple as giving cured Breast Cancer victims a calcium supplement can significantly reduce remission. Phenotypes >= Genotypes?

Scientific article cited in comment section:

http://www.nejm.org/doi/full/10.1056/NEJMoa0806285

"Finally, that one of the simplest of diseases managed to utterly confound us for so long, at the cost of millions of lives, even after we had stumbled across an unequivocal cure. It makes you wonder how many incurable ailments of the modern world - depression, autism, hypertension, obesity - will turn out to have equally simple solutions, once we are able to see them in the correct light. What will we be slapping our foreheads about sixty years from now, wondering how we missed something so obvious?"

Cancer may be similar, though I suspect it's a syndrome i.e. not a single cause and cure; but many causes, and therefore many treatments.

I knew someone a while ago (who recovered) who had a secondary brain tumor. His doctors basically admitted -- they had no idea what effect the cocktail of drugs he was taking would do. The drugs were complex in themselves, let alone in (probably unique) combination.

I think one very accessible innovation in medicine is genetics and drugs. Even just (relatively) simple genetic-drug effect matching could yield some big improvements.

It's a bit like whack-a-mole. Unfortunately, it takes time and money to find the best treatment paths.

Fortunately, more progress has made in the fight against melanoma in the last five years than the previous twenty. As a 2.5 year melanoma survivor (Stage IV in Summer 2012), I'm appreciative of this fact each and every day.

http://en.wikipedia.org/wiki/Cancer_immunology

Your body is constantly surveilling for cancer cells and eliminating them; in doing so, it selects for cells that can evade the immune system. By the time you have large tumors, the cells are already quite good and evading the immune system, at which point your adaptive immunity system can't help you. These scientists are trying to do first-order engineering on the immune cells to make them more effective.

We've seen this before in science, with antibiotics. A few major, early wins followed by the long silence of failure, due to second-order adaptation by cells.

But the key difference is that cancer does not pass from person to person, unlike bacteria. All cancer cells die before or with the patient, they don't stick around to "remember" how to defeat the hack.

If we do get some major, early wins, they are likely to be very reproducible, indefinitely.

[0] http://en.wikipedia.org/wiki/Devil_facial_tumour_disease

And we know that you can intentionally force the formation of tumors / cancer through introduction. We do this in mice as a regular course of study now.

I suspect that over the coming decades, we're going to find cancer is a far more communicable disease than previously thought.

That seems like a giant conflation to me.

[Edit: should be noted however that the vaccine is for the virus, not from the cancer that can arise from its infection.]

When you treat a person with a cancer drug, the cancer tumor population evolves (selection and natural variation), when you treat a population with an antibiotic, the bacterial population evolves. That was the point of my analogy.

With infectious disease, if there are ten possible mutations to make the pathogen resistant to treatment, each with a 1% chance of happening within the course of the disease in one patient, then pretty soon all mutations would be present in most of the whole pathogen population on Earth, because of natural selection.

With non-infectious diseases such as cancer, in the same scenario, there would be about a 10% chance of the cancer cells developing one or more of the mutations in each patient, period. A tragedy for those 10% of patients, but a cure for the other 90%.

Please read the entire book "Molecular Biology of Cancer". I'm just explaining small parts of it.

My question would be, why wouldn't cancer continue to evolve with our immune systems? We're not abolishing the process that leads to cancer, or making cancer impossible. This therapy kills it after it comes into existence. What would prevent a modified immune system from being passed on, and what would prevent an evolution on the kind of cancer that said immune system is susceptible to? That is, why wouldn't the next generation of cancer become that much more challenging to fight, requiring constantly evolving the therapy?

Not asking to be pro or con, but genuinely curious about this.

The fact that modifying your immune system doesn't automatically modify the part of your DNA that encodes how to create an immune system. Children don't inherit your experiences, they just inherit your DNA. (For the same reason, cutting your leg off doesn't cause your offspring to have a missing leg.)

1) Acquired antibodies are passed from mother to child.

2) Epigenetics

1) Ignores obvious intention of the author

2) "several"

Saying cancer is just a failure mode of biology isn't given cancer enough credit. It's more like cancer is a side effect of complex organisms, which is nearly impossible to eliminate, without also eliminating a number of highly advantageous features of cells.

I would hardly call antibiotics a "long silence of failure" - they are still pretty goddamn effective, even if they aren't quite as effective as they once were.

I'm not denying we got some easy wins with penicillin back in the day, or that people can't be treated with antibiotics.

But I've been in the medical industry long enough to maintain a very high level of skepticism when it comes to cancer treatment.

While anti-retroviral therapy for HIV/AID does not cure the disease, it has transformed a diagnosis that would otherwise have been a death sentence into a manageable illness.

If a "first order" treatment for cancer only extends lives by a few years or even decades, how is that not progress?

Because she matched a gene, she's able to take a medicine called Tarceva. In essence, this medicine makes her lung cancer a chronic illness -- it's present, but doesn't spread or metastasize. It's a similar strategy to the one now employed with people who are HIV positive.

Mom is still kicking, so this stuff is working.

http://www.penncancer.org/tcelltherapy/

If I had a promising new drug, I'd want someone with years of experience in getting a drug approved helping me out and the billions in capital to get it done wouldn't hurt either (which of course is available because Novartis sold a number of profitable drugs).

The professor who came up with this science is going to get a cut of that, as will the university.

Yikes!

My wife was diagnosed in September with stage 1 breast cancer. She has triple negative breast cancer which is the most aggressive kind to get. She is on week 6 of a 16 week Chemotherapy regimen. Then she has 8 weeks of radiation.

Insurance has already spent over 100k on her lumpectomy and chemo drugs and doctor appointments since September.

Every week when we go to her oncologist office, the waiting room is always full with patients we have never seen before. More than half of them are new patients filling out their new patient paperwork. And they are getting younger and younger in age. We have seen teenagers in his office with breast cancer.

There is just too much money (doctors, surgery, drugs) to be made from treating this disease. What are all of these trained oncologist surgeons/doctors going to do if cancer gets cured? What are the drug companies that make these expensive chemo drugs going to do if cancer gets cured?

There is no way they are going to cure this horrible disease. There is no money to be made in the cure.

That said, I am very sorry to hear about your wife's condition and I hope that she comes out of it fine. That must be an extremely difficult situation and I'm not sure if I were in your situation I wouldn't have a similar opinion.

The "they" who treat cancer are not the same "they" who will cure it. It's like saying auto mechanic design cars to fail. Auto mechanics don't design cars at all, and have nothing to do with that.

Something to keep in mind is that cancer isn't just one disease, it's a huge cluster of diseases with similar presentations. I survived testicular cancer this year, which happens to be one of a group of cancers that is almost completely curable. Thirty years ago it was not the same story, with a 90% five year mortality. Now it's 95% five year survival. That said, two close family members passed away this year from other types of cancer, one kidney and another colorectal.

I guess my point is that each type of cancer potentially requires a different cure, and some are easier nuts to crack than others. Leukemia has a promising future as one of those curable cancers.

Look at the new HCV treatments. Previously, you'd have to take two drugs for most of your life. Three different companies have developed treatments that cure HCV in 90%+ of the patients with only 12 weeks of treatment.

Old news ?

http://m.vice.com/weediquette-show/stoned-kids

But anyway, a life is worth everything(?)

All that said, in this specific case, the therapy is enormously complex, time consuming, and expensive. It's not a simple chemical compound that can be manufactured in bulk. Furthermore, it has to be tailor-made in a lab under highly controlled conditions for each individual receiving the treatment. As a result, it is likely to be extremely costly when it finally makes it out of the experimental phase.

Can I ask a stupid q? How exactly inexpensive is it? Thinking in line of 3d printing, how possible could it be to get the chemicals needed, and the machines needed to mix them appropriately, to make a known drug? Ie, how possible would be to "download a pill"? The recipe, and you make it yourself at home?

Yes, you can make many drugs at home with the right setup and technical knowledge. No, robots don't actually help that much.

http://www.theverge.com/2013/12/6/5184168/fda-approves-new-1...

$84k for a 90% cure to Hep-C is peanuts when compared to the alternative of liver transplant and chronic hospitalizations.

It's sort of like people complaining that Lipitor costs $5K/yr. The fact it prevents a number of heart attacks, means that it's actually a cost saving treatment.

check out the healthcare sector margins (notice major manufacturers, vs. generics manufacturers):

http://biz.yahoo.com/p/5qpmd.html

You know those "greedy insurance companies" that the US is struggling to wrangle with complicated legislation and broken website markets? They only make 3% margin.

Of course, that's after paying out executive salaries, including bonuses for "cost cutting".

Healthcare is the most profitable industry, even more profitable than banks. Insane...

Every penny you have is peanuts compared to a slow painful death.

I'm a single-payer advocate. I too think market-based healthcare fails society. Within the context of it, though, this isn't as outrageous as the plain dollar amount seems.

- Many people don't spend every penny they have. They spend every penny they can get out of their health plan. That's not a "market failure" by any means agreed?

- If they must pay out of pocket, and they have no kids, then why not? If they do have kids, then that seems a bit selfish to me. Maybe the right thing to do is save the money and buy cheap painkillers.

Not every sad story is Oliver Twist. And the argument that single-payer health-care will be cheaper is a crap-shoot at best. The countries that do it well do many things well. I don't see dramatic savings from US single-payer education. Or from single-payer defense spending.

Compare that with the number of healthcare related bankruptcies in say, the UK.

Interestingly, it turns out that research commissioned by the Canadian government shows that 15% of people over the age of 55 who declare bankruptcy cite a medical problem as the primary reason. Medical bankruptcies can, as I've been saying for a while, be driven by something other than the lack of free government provided medical care.

http://www.theatlantic.com/business/archive/2009/09/bankrupt...

The statistic about Canada backs up the idea that isn't true.