Hi everyone,
I've had an idea for a few years that I'd like to start moving towards trying to implement. It's inspired by David Pearce's Hedonistic Imperative.
The idea is to sketch out a detailed road map for developing an in vivo gene therapy for mood improvement. It seems like the essential technology for this now exists, but I haven't seen any sign of anyone working practically on this specific problem. Presumably some day some big biotech company will take this on, but I'm curious whether some enthusiastic amateurs can make any meaningful progress. Things like:
- Fleshing out the technical theory of a therapy. I'm thinking AAV-based, but open to other ideas. What specific viral (or other) vector variant to use, what promoter, what transgene to try, how to deliver to the target brain area, potential side effects, etc. Eventually aiming towards a fully designed viral vector code, with delivery method.
- Thinking about testing. Assume we think we have achieved the previous step. Without the backing or budget of a major lab, how difficult is it to carry out animal tests? Could it be done somewhere in the world with sufficient scientific standards but lower cost and/or regulatory barriers?
- Ultimately human testing will be the end goal, this is well down the road and probably the most challenging aspect but still worth thinking about.
Rather than thinking in terms of attracting capital, licensing, commercialization etc, I want to focus on establishing a working therapy, though of course some capital would be required to get anything done.
I have some ideas about these things of course, but would like to get more brains involved if there is interest. For example, I lack the expertise to bridge the gap between designing a viral vector and having something physically ready to administer to an organism.
Alternatively if anyone knows anyone who's already working on a similar project I'd appreciate being pointed in the right direction. I'm already in discussion with someone from a project with similar aims but focusing more on germline therapy.
Please comment or PM me if interested or have questions!
I work in gene therapy and can tell you that it has huge risks, which means that it's mainly considered for diseases that have no other treatment and would otherwise be fatal. For example, the latest gene therapy trial for X-linked myotubular myopathy has had four children die, probably because of AAV toxicity. The reason gene therapy is being considered for XLMTM is because it involves loss of function of a single gene in a specific cell type, as well as meeting the criteria I said before. Mood disorders aren't good candidates for gene therapy.
Yes, I saw that case. It seems that they were using an extremely high viral dosage. In my understanding one of the reasons AAV is typically used is the low immunogenicity at normal dosages and overall good safety profile. Obviously no pioneering method is without risk.
Whether mood disorders qualify under your criteria is debatable - certainly for a large proportion of people no currently available treatment is effective. Fatality rates may be relatively low, but I suspect a fairly large cohort of people living with mood disorders would rather tolerate some risk than live out the rest of their lives with no hope of treatment.