Descripción del proyecto
Una idea fantástica mejora la seguridad y la eficacia de las terapias en marcha
Hasta hace poco, el transporte de medicamentos delicados a lo largo de la cadena de suministro, desde que se produce hasta que llega al paciente, requería una técnica de refrigeración relativamente sencilla. A medida que las terapias han pasado de simples moléculas químicas a macromoléculas proteicas o células vivas, la cadena de frío ha tenido que adaptarse. La mayor parte de los compuestos biológicos deben criopreservarse y, tal como cualquier persona que haya congelado y descongelado un filete puede apreciar, no es fácil conseguir que las células se conserven en una forma óptima durante ese proceso. Las tecnologías disponibles en la actualidad utilizan disolventes orgánicos como crioprotectores, pero no son perfectos. El proyecto ICE_PACK, financiado con fondos europeos, está desarrollando crioprotectores poliméricos biomiméticos que estabilizarán las membranas y evitarán que las proteínas se degraden, lo cual reduce considerablemente el coste de la terapéutica y, al mismo tiempo, aumenta su seguridad, eficacia y accesibilidad.
Objetivo
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.
Ámbito científico
Programa(s)
Régimen de financiación
ERC-COG - Consolidator GrantInstitución de acogida
CV4 8UW COVENTRY
Reino Unido