Innovative Alternatives to Human Factor VIII

Despite massive expenditure of effort and money in fractionation of human blood plasma, it is clear that the end‐product factor VIII is deficient in quantity and its quality seriously compromised by infectious agents both known and unknown. These problems may not be soluble by ever greater blood collection drives and ever more stringent process controls. The average fractionation puts only 20 per cent of donated factor into the bottle of concentrate. Are radical new methods of purification needed? Meanwhile, the vast majority of our potential resources of factor VIII and IX, free of all known human pathogens are thrown away. In the United Kingdom last year, over 600,000 million units of sheep, pig and cow factor VIII were washed down the abbatoir gutters or consumed as black pudding. Already, some haemophiliacs with inhibitors are successfully treated with highly purified porcine factor VIII. Could new purification methods and chemical modifications make this natural abundance of animal factor VIII available to treat all haemophiliacs? Genetically engineered factor VIII made by cultured cells, pure and in unlimited quantities is a theoretical possibility which stands as a ‐ perhaps the ‐ supreme challenge to biotechnology today. Undaunted, several groups have taken up this quest. Huge obstacles bar the way but like Everest it attracts our highest endeavours because it is there. Finally, what thoughts should we entertain of gene therapy? “Ah, could we not seize this sorry scheme of things entire and mould it closer to the heart's desire?” There have been false dawns but recent progress on gene insertion and control raise more than a glimmer of fresh hope.