New AIP treatment in the works?!?

22 05 2013

I got an interesting email yesterday. It’s really technical, but here’s the exact text, straight from the American Porphyria Foundation:

Alnylams Pharmaceutical presented data on their new AIP treatment at the recent International Porphyria Congress in Lucerne Switzerland.

In pre-clinical models of the human disease, they demonstrated RNAi therapeutics targeting ALAS-1 can completely block the abnormal production of toxic intermediates of the heme biosynthesis pathway that cause the symptoms and disease pathology of AIP.

They expect to identify a final development candidate by late 2013 and advance ALN-AS1 into the clinic in 2014. ALN-AS1 is a subcutaneous RNAi therapeutic targeting aminolevulinate synthase-1 (ALAS-1) for the treatment of acute intermittent porphyria (AIP), an ultra-rare genetic disease. ALN-AS1 has the potential to be a therapy for the treatment of acute porphyria attacks, as well as a prophylactic approach for the prevention of recurrent attacks.

To view the presentations from Alnylam and Dr. Robert Desnick’s Mount Sinai team, see below: Note that Protect the Future doctor, Dr. Makiko Yasuda, made the following presentation on the Mount Sinai research.

http://www.alnylam.com/capella/wp-content/uploads/2013/01/Desnick-AS1-PorphyriaCongress-May2013.pdf

Alnylams presentation:

http://www.alnylam.com/capella/wp-content/uploads/2013/01/ALNY-Porphyria-AS1-Pres-May17-2013.pdf

Visit the Alnylam site and read details of this exciting news:

http://www.alnylam.com

Now, what I think this means, is that a new treatment, that happens to be some sort of subcutaneous device (?) can treat an attack more effectively than hemetin and glucose, and when used as a preventative, can provide two weeks or more of protection against triggers. I come to this conclusion after reading through the PowerPoint presentations and, while I’m no doctor, I’ve been around a lot of them lately. And I think I’ve picked up some of the foreign language they speak, known as medicalese. It’s a difficult, awkward, overly-verbose and nonsensical language, often scribbled illegibly. If you have more experience with this highly coded and secretive language, and better understand what the hell those PowerPoints are saying, please do share.
But, if I’m correct, and I think I am (wink wink) then maybe this means an end to weekly infusions in the future? Maybe it means every few weeks, I can just go get a little injection or something? That would be… Life-changing. Huge.
I’ll keep you updated on any more news about the study as I get it.

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One response

7 05 2014
prkralex

ALN-AAT is one of Alnylam’s genetic medicine programs, which are RNAi therapeutics directed toward genetically defined targets for the treatment of diseases with high unmet medical need. AAT deficiency-associated liver disease is caused by accumulation of mutant AAT protein (Z-allele or Z-AAT) in liver tissue with subsequent liver injury, fibrosis, cirrhosis, and, in some cases, hepatocellular carcinoma.

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