CRISPR in Personalized Healthcare

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How important will CRISPR or similar therapeutic approaches be in the development of personalized medicine? Also, will changes in FDA approach to regulation be needed in order to facilitate review and approval of products intended for personalized healthcare?

Personalized Medicine
CRISPR
FDA regulations
Rob Walsh
75 months ago

1 answer

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CRISPR technology will have an enormous impact in personalized medicine. This has already kicked off with ongoing Phase 1 somatic ex vivo clinical trials in patients with the single gene diseases b-thalassemia and Hunter syndrome https://www.voanews.com/a/second-man-undergoes-gene-editing/4242428.html. Stock markets are rallying in excitement with other trials (largely oncology) due to start this year http://www.gizmodo.co.uk/2018/01/the-first-us-human-crispr-trials-could-start-any-day-now/ ; https://www.popsci.com/china-crispr-immunotherapy-fda (in the US - already a lot going on in China). Of course the most controversial discussion concerns whether we should treat patients by targeting the germline and eradicate a suitable disease in present and all future generations. But there are a number of bioethical, political, regulatory, safety and scientific issues to deal with before this possibly could happen. We also need to be aware of biohackers who are taking it upon themselves to inject themselves with CRISPR produce purchased online. This is very dangerous both for their own personal safety and for the reputation of the fantastic promise that CRISPR medicine holds for the world.
With respect to its impact on the patient alive today, FDA regulation need not treat a new somatic CRISPR medicine differently from any other novel medical entity in terms of its safety, efficacy, specificity and merit compared with other appropriate treatments for a given disease. It's potential high price will likely be a matter of debate because it is a one-off cure (see Sovaldi example). If germline CRISPR treatment is ever allowed then this will set a new precedent because a treatment will affect all future generations, possibly creating the need for multi-generational clinical trials!

Mark Robertson
75 months ago

Have some input?