Here is another post from JD in regards to the Scarpa NR:
The results of the Scarpa study were extraordinary by any measure. Some of us are doing a lot of work to see if we can find studies to which we can compare this study. The trouble is that most studies that we find are with Elaprase, Shire's highly refined and potent version of the human enzyme, iduronate-2-sulfatase (I2S). Elaprase even has glycans in it that extend its half life, likely a significant factor in Elaprase's low dosing amounts of 0.5 mg/kg, one-twelfth of the dose used by Scarpa with MTfp-I2S.
The enzyme that Scarpa and biOasis used, which I understand to be natural I2S, makes it difficult for us to directly compare MTfp-I2S efficacy with any numbers we can find with Elaprase animal studies. I have scientific papers that show Elaprase dosing levels that are all over the place, 30, 40 mg/kg and even higher doses.
When we have one of the world's leading experts involved in this study, as we do with Scarpa, then we can rest assured that he (and biOasis) conducted the study in the way they did for good reasons. Scarpa and the scientists at all of the pharmaceutical companies that are interested in the results, all understand how our results relate to those of Shire's Elaprase.
The bottom line is this - biOasis is in the business of getting drugs into the brain in an efficacious manner. With Scarpa, biOasis has accomplished that, not in only a merely hopeful manner, but in a manner that has very clear commercial possibilities.
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