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Something to consider: it is now technically possible and within the grasp of hundreds of clinics worldwide to run gene therapy. The only reason they aren't yet
by reasonattlm 11y ago
Something to consider: it is now technically possible and within the grasp of hundreds of clinics worldwide to run gene therapy. The only reason they aren't yet doing this is that it takes some increment of time to get things lined up; funding, customers, propagation of knowledge, etc. But all the pieces are in place.
Myostatin or follistatin gene therapy is about as proven as these things get. It exists in heavily muscled natural mutants, including humans (look up brute whippets). Antibody myostatin blockade has just completed phase 2 clinical trials, adding muscle mass to 75 year olds. Chinese labs are turning out myostatin-free dogs. The BioViva CEO organized her own follistatin gene therapy earlier this year in an overseas lab. Five years from now it won't take starting a company level of networking to fly somewhere and do this.
But myostatin for muscle mass is just the best risk/benefit/knowledge picture out of hundreds of alterations that could in theory now be run up in humans, many of which are high risk in the sense that they've only been tried a few times in mice and not followed for a long time. For example adding additional lysosomal receptors to mice restores liver function in old mice to be the same as in young mice. Increased GDF-11 sends stem cells back to work and improves health in old mice.
And so on and so forth. The eager early adopter with a hundred thousand dollars to spend ten years from now could spend the time between now and then mining the research literature and organizing collaborators, making connections with labs overseas and setting up for multiple gene therapies down the line, many of which are associated with interventions shown to delay or turn back specific measures of aging in mice.
This isn't SENS, it isn't repair of underlying damage, but it is a step ahead of what is currently possible.
- jforman 11y agoThe greatest barrier here is the FDA. They don't let you perform medical interventions (even breakthroughs like this) without going through a rigorous process. Companies like Editas ($43M Series A) are going through those motions right now.
- mkempe 11y agoThe inevitable consequence will be either the abolition of the FDA; or a chasm between advanced medical therapy readily available in some places outside of the US and sclerotic, bureaucratic "healthcare" in the US. Long-term, where I live will depend on the above outcome.
- deleted 11y ago[deleted]
- jforman 11y agoCRISPR is far from being the first technology to be held up in the United States while being available elsewhere. There is very little chance the FDA will materially budge on their standards, and even less chance the FDA will be abolished. Most people have a high degree of trust in the FDA (they have a 65% favorability rating according to Pew). Note that the FDA does allow some compassionate use of experimental medications and technologies in extreme cases. I'm not familiar with Editas's regulatory strategy but it wouldn't be crazy for them to pursue that avenue for, say, a child cancer patient as an early step. But even they will be cautious, as any misstep (i.e., harm to a patient) will have major negative consequences for the technology as a whole.
- dekhn 11y agoI'm not denying running these experiments is becoming much more feasible for small institutions but you have a pretty naive understanding on how to turn these kinds of experiments into successful therapies. There is often a huge disconnect between what we can reliably and reproducibly show in a lab, and what can be turned into therapy. And that's not just as simple as mining the literature and running more trials.
- reasonattlm 11y agoOne of my points is that adoption is going to precede the current consensus definition of "successful therapy" by a decade, in much the same way as happened for stem cell therapies following the turn of the century.