Descrizione del progetto
Un’idea davvero fresca migliora la sicurezza e l’efficacia delle terapie in movimento
Fino a poco tempo fa, il trasporto di farmaci sensibili lungo la catena di distribuzione, dalla produzione al paziente, richiedeva una tecnologia di refrigerazione relativamente semplice. Quando le terapie sono passate da semplici molecole chimiche a macromolecole di proteine a cellule viventi, la catena del freddo ha dovuto adeguarsi. Molti prodotti biologici devono essere crioconservati e, come ben sa chiunque abbia congelato e scongelato una bistecca, mantenere le cellule in forma smagliante durante il processo non è un compito semplice. Le tecnologie attualmente disponibili utilizzano come crioprotettori solventi organici non perfetti. Il progetto ICE_PACK, finanziato dall’UE, sta sviluppando crioprotettori polimerici biomimetici che stabilizzeranno le membrane e proteggeranno le proteine dai danni, riducendo significativamente i costi dei trattamenti, rafforzandone al contempo la sicurezza, l’efficacia e l’accessibilità.
Obiettivo
Ten years ago small molecules were the drugs of choice for most diseases. This situation has dramatically changed such that in 2017, 4 in 5 of the best-selling drugs were proteins, delivering major healthcare benefits from cancer to infectious disease. Cell-based therapies are also emerging, with the promise of making the incurable, curable (especially in oncology). However, the use of these ‘biologics’ has accelerated beyond the currently available technologies to stabilize these therapeutics against the environmental stresses imposed by the pharmaceutical cold chain. 75 % of current biologics must be cryopreserved to ensure they reach the patient in a functional format, requiring the addition of cryoprotectants (organic solvents) which are toxic, do not enable full post-thaw recovery, are complex to remove, do not stabilize cell membranes efficiently, lead to batch-to-batch post-thaw variabilities in function and cause toxic plasticizers to leach from transport bags.
ICE_PACK will develop unprecedented macro-and supra-molecular biomaterials to transform the biologic cold chain with long term impact by making these therapies more effective, cheaper, safer and more widely available.
We will synthesize polymeric cryoprotectants, inspired by Nature’s macromolecular ice modulators, capable of inducing ice nucleation, stabilising cell membranes and protecting proteins from denaturation, and apply these to cryopreserve real therapeutics. An integrated approach will be taken whereby ice nucleation and growth control will be studied in parallel to investigating the biochemical and molecular biology impact of cold stresses. Major fundamental scientific advances will also emerge, linking macromolecular structure to ice nucleation and to biochemical pathways of cold tolerance and stress.
The long term outcomes of this ground breaking ERC project will be the transformation of cryopreservation strategies, suitable for 21st Century biologic medicines.
Campo scientifico
Programma(i)
Argomento(i)
Meccanismo di finanziamento
ERC-COG - Consolidator GrantIstituzione ospitante
CV4 8UW COVENTRY
Regno Unito