But human Factor VIII is a very large andcomplex molecule that is hard for nonhumancells to produce. Although theanimal cell lines do a good job of expressingfactor, it’s hard for nonhuman cells toproperly fold the factor into its final threedimensionalshape. Plus, they may not doa good job of attaching other moleculesto the factor’s surface, as human cells do.As a result, the improperly finished proteinsmay have increased immunogenicityand, for several reasons, may not work asefficiently.By using human cells to express and finishfactor proteins, the cells may deliver improvedbenefits, like more factor production,lower immunogenicity and greaterfactor efficiency. We’ll have to wait for theclinical trial results to verify this thinking.Custom-Designed Clotting FactorsNot all Factor VIII or IX molecules areidentical. They differ slightly among differentraces. This may be why AfricanAmericans have a significantly higher rateof inhibitors: commercial factor may differslightly from their own factor, makingthe product more immunogenic to them.Bioengineering of the factor molecule mayallow for more custom-designed factor forspecific populations, reducing the incidenceof inhibitors.Recombinant Porcine Factor VIIIInspiration Biopharmaceuticals is currentlyin Phase III clinical trial for its OBI-1, a newrecombinant porcine (pig) Factor VIIIto treat acquired hemophilia A (an autoimmunedisease) and hemophilia A withinhibitors. Porcine Factor VIII is differentenough from human Factor VIII that itusually escapes detection by the immunesystem (so inhibitors aren’t alerted), butsimilar enough that it functions to producea clot. For those with acquired hemophilia Aand hemophilia A with inhibitors, this productcould target their specific factor needs.BiosimilarsBiosimilar factor products will offer patientsless expensive “generic” versionsof name brand products after those patents,patent extensions and periodsof-exclusivityhave expired. Almost allmajor U.S. and international manufacturersof factor products have one ormore biosimilar products in Phase II or IIIclinical trials.These biosimilar products will definitelybe less expensive than the branded counterparts,but perhaps not by as much asmany of us would like. Unlike chemicalgeneric drugs (such as ibuprofen) thatmay sell for a fraction of the cost of thebrand-name drug, biosimilars are expectedto sell at only 10-to-30 percentless than current factor concentrates.That’s because biosimilars, unlike genericforms of chemical compounds that areeasily reproduced, can’t be made identicalto their original forms. The proteins inevery brand of factor are slightly different.Plus, there are dozens of variables in themanufacturing process that can alter theproteins, making them more immunogenicor less effective. That’s very important forhemophilia patients, since a more immunogenicproduct means it’s more likely toproduce an immune response and causeinhibitors to form.Because of these requirements, manufacturersof biosimilars must invest extensivelyin research, development, facilityconstruction and full clinical trials in orderto bring a biosimilar factor to market. Butthe good news is the process has begun.Biosimilars are in the works as a newoption for hemophilia patients.Innovating a Healthier FutureJust as the Renaissance ushered in a newera of rapid scientific exploration and innovationfollowing the Middle Ages, thehemophilia Renaissance is bringing a waveof innovations to the hemophilia community.Because of this swell in new productsand treatments, hemophilia patientscan look forward to a brighter future and ahigher quality of life.About the AuthorPaul Clement writes extensively for the hemophiliacommunity and is a frequent contributingeditor for the Parent Empowerment Newsletterfrom LA Kelley Communications. Mr. Clementsis a high school science teacher and father ofa son with severe hemophilia A. He is known forhis ability to translate complex topics into moreeasily understood language for parents whomust master the complexities of their child’shemophilia.34 InsideOut
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