RNAi for PsA

Does anyone know about or is anyone familiar with any research going on regarding RNAi drugs? Seems to be the hot and very promising thing in the biopharma sector at the moment.

Hi MikeS!

Are these drugs being talked about in connection with inflammatory arthritis? Admittedly I’ve only just done about 10 seconds of googling, but no immediate connection presented itself!

How are you doing? (Just noticed you’re about to start Humira).

RNAi is indeed promising.The broader term is called Precision Medicine. It has revolutionize cancer treatment as well as several childhood genetic conditions. And the key word there is GENETIC. Without getting into the details what happens is a persons actual genetics can be changed assuming there is a genetic marker identified and a variant in the marker identified. A single treatment can cost over 1 million dollars. For some conditions several treatments a year in the mid 6 figures can control or eliminate disease entirely.

The term RNA interference (RNAi) was coined to describe a cellular mechanism that use the gene’s own DNA sequence of gene to turn it off, a process that researchers call silencing . In a wide variety of organisms, including animals, plants, and fungi, RNAi is triggered by double-stranded RNA (dsRNA).

There are a few very stringent requirements. The disease/condition must be genetic and the variant recognized and very specific. This is very limited. We are almost there with Duchenes MD. With say ASD (Autism/Spectrum Disorder) we know its virtually always genetic. BUT although HCD-8 is primary there are another 42 variants identified and an other 1200 or so suspect. We are a very long ways off.

You may hear about RNAi and PsA but its not our PSA but rather Prostate Specific Antigen - prostate cancer.

We have no known genetic markers for PsA (if we did there would be a test) Though we know it tends to run in families, that’s about all we know.

So while we are making progress on many fronts, The Snake Oil salesmen have already glommed onto RNAi and have developed a broad range of Snake Oils, alternative medicines,clinics, etc. that do nothing but offer false hope and the opportunity to separate desperate patients from their money (lots of it) not at all unlike the Stem Cell clinic which are FINALLY being shut down and the operators being prosecuted.

FWIW, Biologic medicines are very close in that they are all Human (or synthetic human) DNA that alter inflammatory response. Simply (or not so simply) put, Biologics contain RNA which is what makes them work. RNA is a single-stranded where as DNA is double stranded. … RNA nucleotides include sugar ribose, rather than the Deoxyribose that is part of DNA . Functionally, DNA maintains the protein-encoding information, whereas RNA uses the information to enable the cell to synthesize the particular protein.

In short Humira and other Biologics are essentially functioning the way RNAi is hoped to. We are getting better ones (5 in the last month in fact) but until we know exactly which Gene variants (if any) cause PsA we can improve biologics for some patients but The “cure” offered by RNAi is a long ways off. The best way to look at it (though not technically correct is the biologics are “shotgun” RNAi

TJ

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