MEBO chat with NIH ORDR office :details to follow

Details will follow soon of :
MEBO web conference with NIH ORDR dept team.
Tue 18th Sept

Info given after the chat :

Thank you for speaking with us earlier today. In follow-up to our conversation, here are links to some of the programs and resources we spoke about.

RDCRN (Rare Disease Clinical Research Network) Funding Opportunity Announcement - https://grants.nih.gov/grants/guide/rfa-files/RFA-TR-18-020.html

Toolkit for Patient Focused Therapy Development - https://rarediseases.info.nih.gov/toolkit

R13 Grants (Support for Conferences and Scientific Meetings) - https://grants.nih.gov/grants/funding/r13/index.htm

All of Us Research Program - https://allofus.nih.gov/

Registries Program (RaDaR): https://rarediseases.info.nih.gov/radar

Additional registry information outside of NIH:

NORD (National Organization Rare Disease) - https://rarediseases.org/iamrare-registry-program/
Sanford CoRDS (Coordination of Rare Diseases at Sanford) - http://www.sanfordresearch.org/specialprograms/cords/

Additional info given :

Thank you for the time to talk to us about TMAU yesterday.

In terms of drug development for TMAU, given the available knowledge of the TMA metabolism, it seems possible that small molecules could be developed to modulate TMA levels.

The attached PDF entitled TMA inhibitor is an example that I found.

You may have seen this, but It turns out that the same approach has implications for treating heart disease, so there is already drug development ongoing

https://newsroom.clevelandclinic.org/2018/08/06/potential-new-class-of-drugs-developed-at-cleveland-clinic-may-reduce-cardiovascular-risk-by-targeting-gut-microbes/

There may be other labs or companies working in the same area, so this is not intended to be a complete list.

Along the same lines, another possible therapeutic strategy that is worth considering would be breaking down or trapping TMA.

While gene therapy to the liver is theoretically possible, it seems unlikely at the moment given the serious risks of gene therapy, and since TMAU is not a life threatening disease.

An alternative that could be considered is using genetically modified microorganisms in the gut to do the same things.

These links discuss some examples of that strategy.

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4049937/
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4666514/

Here are two companies working in this area that I know of;
there may well be more

https://www.synlogictx.com/
https://aobiome.com/

As I mentioned yesterday, this information is not an endorsement of any company; just some ideas for you to consider and discuss with your scientific and medical advisors.

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