Innovation
Science and Humanity are always on the move fostering continuous innovation.
Science and Humanity are always on the move - growing, developing, expanding horizons. We know we can help more people by pushing boundaries, by making learning and discovery a part of our identity.
Innovation for us means finding new and better ways to help more people. This is especially crucial when it comes to rare and ultra-rare diseases and conditions, since, by definition, often so little is known about them, and every new understanding can be a key resource.
An example of this kind of innovative approach is a research project made in collaboration with Toscana Life Sciences Foundation and IRCCS Hospital San Raffaele in Milan, regarding the potential use of the “waste” material normally discarded after targeted proteins are removed from the total “proteome” (the entire set of thousands of proteins) found in plasma.
Plasma-derived medicines producers isolate desired proteins through fractionation, and the remaining plasma material is generally discarded. But what if this “waste” could be further processed to exploit the potentials of other proteins still within it?
The research article has been published in Communications Biology, within the Nature Portfolio.
Although the plasma proteome provides opportunities to develop novel protein replacement therapies, particularly for rare diseases, the high cost of plasma together with small patient populations impact negatively on the development of plasma-derived orphan drugs. Enabling therapeutics development from unused plasma fractionation intermediates would therefore constitute a substantial innovation.
Communications Biology
Nature
Andrea Caricasole
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The research went beyond speculation, demonstrating the feasibility of the idea by isolating a specific protein from unused plasma fractionation intermediates and showing in mice studies promising potential as a therapy for an adult-onset ultra-rare neurological disease.
These data demonstrate the feasibility of transforming industrial waste plasma fraction into a raw material for manufacturing of new candidate proteins for replacement therapies, optimizing plasma use and reducing waste generation.
Communications Biology
Nature