5 ms·
I've commented on this around here before. You read lots of junk about cancer "cures" in the popular press. They are almost always in mice or something and I or
by JunkDNA 13y ago
I've commented on this around here before. You read lots of junk about cancer "cures" in the popular press. They are almost always in mice or something and I or someone else with a similar background always feels compelled to weigh in and remind folks that it's a long way from curing lab rats to curing people.
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.
- lmg643 13y agoI'm not an expert, just an interested layperson, but from what I read, I am starting to wonder whether cancer cures might be more basic than we realize, we're just not looking at the right things. 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/ http://westhunt.wordpress.com/2012/02/22/low-hanging-fruit/
- cLeEOGPw 13y agoPrevention of cancer is not a cure, and it would not prevent all types of cancer. Cancer can appear anywhere in the body without any significant "cause". Regular safe sun radiation can be enough to alter certain DNA part during regular cell divide and immune cells might fail to spot the cell and it can start reproducing. Unlike infectious diseases, cancer is the result of evolution within body, it is fundamental to every multicell organism.
- pak 13y agoViruses are only one potential cause of cancer, and there are several known tumorigenic viruses (HPV, adenovirus, EBV, polyomavirus, etc.). There are still researchers that believe that all remaining cancers are caused by yet undiscovered viruses (the "viral hypothesis"), but these researchers are becoming scarce. 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/nature11288.html 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.
- Double_Cast 13y agoYour link lost me at the first sentence. But the word "phenotype" reminded me of this 11 minute Khan Academy video. Maybe I'm just repeating the same thing. But I thought Sal's video was worth sharing and easily digestible. https://www.khanacademy.org/science/healthcare-and-medicine/healthcare-misc/v/systemic-thinking-about-cancer 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 http://www.nejm.org/doi/full/10.1056/NEJMoa0806285
- whitneyrblack 13y agomy classmate's mother makes $82/hr on the computer. She has been fired from work for ten months but last month her pay check was $12046 just working on the computer for a few hours. why not try this out=======> jobs25.ℭＯM
- jwilliams 13y agoI think one effect is that "cancer" is actually a very broad umbrella term. Different cancers have very different causes, characteristics and prognoses.
- JunkDNA 13y agoIndeed you are right. For much of the time people have thought that at least you could classify cancers by the tissues where they present themselves (e.g. colon cancer, breast cancer, prostate cancer, etc...). But more research is showing that this may actually not be so simple. At the molecular level, it's possible for two cancers from different tissues to be identical from a mechanistic standpoint. There is a lot of re-thinking going on in the oncology community about how cancers are classified and treated as a result.
- jwilliams 13y agoYeah. It then gets even more complex when a cancer spreads as another secondary cancer to a different part of the body. You suddenly get the specialists scratching their heads about who guides the treatment. 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.
- robbiep 13y agoThis is already beginning to happen- melanoma patients are screened for V600E mutations to determine if they can receive new treatments, as with breast cancer and tamoxifen or her petit although this happens with immunohistoxhemistry rather than sequencing. The V600E mutation also appears in some other cancers (bowel, from memory) so occasionally used there too
- foobar388383 13y ago