Blood cancers

Cheap and rapid ‘non-viral’ CAR T-cell therapy in Monash sights

Wednesday, 22 Jul 2020

Monash University researchers are aiming to overcome the high costs and lengthy manufacturing times associated with CAR-T cell therapy by eliminating the use of an inactive virus vector to genetically encode the T cells.

Using powerful nanoscale tools, scientists at the Monash Institute of Pharmaceutical Sciences (MIPS) believe they can overcome the most expensive and time-consuming step to CAR-T cell manufacturing, thus paving the way for accessible and affordable blood cancer treatment

Currently the CAR T-cell therapy manufacturing process takes several weeks because of the need to grow large batches of replication-defective viral vector, and the many technical steps involved mean this costs more than $600,000 per patient.

However the team at MIPS, led by Professor Nicolas Voelcker and Dr Roey Elnathan, have initiated the use of nanotechnologies to simplify the process by completely eliminating the use of viral vector.

“There is an urgent need for a scalable, low-cost, streamlined CAR-T cell manufacturing process that does not rely on viral transfection methods,” says Dr Elnathan.

“At MIPS we’re using nanotechnologies to enable targeted delivery of non-viral synthetic molecules into the interiors of primary immune cells which are notoriously difficult to transfect. We’ve already shown that this can be done through a scalable route without the viral component.”

The MIPS team has already demonstrated that silicon nanosyringes can directly transport bioactive molecules – DNA, RNA, proteins, and gene-editing tools – into primary immune cells, with their preliminary findings recently published in the journals: Advanced Materials and Small.

According to Professor Voelcker, this breakthrough ”is a platform for making a paradigm shift at the interface of nanotechnology and immuno-oncology, which will have huge benefits for patients around the globe.”

“The non-viral process would reduce the complexities and eliminate the safety issues associated with viral vectors, with the potential to change the business model of the therapy from boutique to widely affordable.”

“Furthermore, the nanotechnology has the potential to be conducted entirely within the hospital, offering huge benefits for patients and their healthcare team.”

MIPS is collaborating with a Japanese company ULVAC Inc to scale up fabrication of the silicon nanosyringes, which will be critical for rolling out the new technology.

The team is now benchmarking the efficiency and duration of CAR expression against viral delivery. Once this is achieved, they anticipate it will take several years to optimise potency and functionality in preparation for clinical-grade non-viral CAR-T therapy.

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