Ghent University Hospital invests in expansion of GMP unit with viral vector production

(01-10-2021) The expansion of the lab should accelerate research into new therapies for incurable diseases.

cleanroomCancer, epilepsy, hereditary blindness, ... Today, gene and cell therapy offer more and more solutions for common, but life-threatening diseases. In order to be able to effectively apply the current research to patients, Ghent University Hospital is taking further steps in expanding a GMP unit (Good Manufacturing Practice Unit). This is a controlled environment where the complex gene and cell products can be produced in extremely sterile conditions. Ghent University Hospital is therefore the first hospital in Belgium to have such technical capabilities.

Facilitate research

'Researchers can contact us to test and evaluate their concept in a clinical study,' says Professor Linos Vandekerckhove. 'By facilitating their research into new, potentially groundbreaking applications of cell and gene therapy, we lay the foundation for making these therapies accessible to a wide group of patients,' adds Professor Bart Vandekerckhove. 'The unique thing about our GMP unit is that, in addition to research into cell therapy, we now also have the opportunity to support researchers in the field of gene therapy.'

Cell and Gene Therapy Breakthrough

CAR-T therapy is perhaps the best known form of cell therapy. Here, body cells are removed from the patient's body, genetically modified in a GMP unit and then re-administered to the patient. Due to the changes, the own immune cells suddenly respond to a disease, such as cancer. With this therapy, a type of immunotherapy, you actually use your own body cells as medicine. Until now, for example, CAR-T therapy could combat different types of leukemia in patients who could no longer be helped with 'classic' cancer treatments, such as chemotherapy.

Cell therapy has therefore become an integral part of medicine today. In particular, the approval of the use of CAR-T therapy by the US Food & Drug Administration or FDA in 2018 marked a milestone in the further development of cell therapy. ‘There was already a lot of academic research into the use of cell therapy for medical conditions, but at that time the interest of the pharmaceutical industry also grew exponentially. A new type of treatment was suddenly approved. For example, there will be a major commercial GMP for CAR T cells in Zwijnaarde, which is very good news for European patients and the ecosystem. More demand from the industry also stimulates academic researchers to focus on this,' says Tim Desmet, business developer at IOF (Industrial Research Fund) at Ghent University for cell and gene therapy. He supervises research groups and platforms, such as GATE (Ghent Advanced Therapies and Tissue Engineering), in internal development processes and collaborations with companies. From the IOF, he screens research results that may interest companies in further developing innovative applications of these therapies.

Today gene therapy also offers solutions for mainly hereditary disorders. Scientists find out which part of the DNA is responsible for the lack or malfunction of a protein. Patients are then injected with healthy and/or modified DNA instead of medicines or surgery. In order to get this material into the cell, it must be "packaged" in the right way.

The transporters of genetic material

Viruses are extremely suitable transporters for doing just that: getting genetic material into a cell. Researchers use innocuous viruses – so-called viral vectors – to introduce the genetic code into a cell (gene therapy) or to genetically modify cells in the lab (cell therapy), such as the CAR-T cells. There are several types of viral vectors, with the primary focus of the GMP unit being to obtain accreditation for lentiviral vector production for clinical research. ‘As a clinical GMP unit (Good Manufacturing Practice Unit), we focus on translating research into its effective application in humans. We produce products, be it living cells or DNA material, which we modify and then inject back into the patient,' says Professor Linos Vandekerckhove.

‘As a clinical GMP unit (Good Manufacturing Practice Unit), we focus on translating research into its effective application in humans. We produce products, be it living cells or DNA material, which we modify and then inject back into the patient.' Professor Linos Vandekerckhove

Affordable research

'We can already do a lot in the GMP unit today. For example, we are allowed to produce mRNA,' says professor Bart Vandekerckhove. 'But we noticed that researchers were reaching limits for their research, because we were not allowed to produce viral vectors until now. These viral vectors are scarce today and therefore very expensive. By now producing lentiviral vectors ourselves in the clinical GMP, we ensure that we can bring new gene and cell therapies to the patient faster and cheaper.'

'We can already do a lot in the GMP unit today. For example, we are allowed to produce mRNA,' says professor Bart Vandekerckhove. 'But we noticed that researchers were reaching limits for their research, because we were not allowed to produce viral vectors until now. By now producing viral vectors ourselves in the clinical GMP, we ensure that we can bring new gene and cell therapies to the patient faster and cheaper.'

'If you outsource that, you need a very large budget, for which it is very difficult to get financing,' says Professor Linos Vandekerckhove. 'Make no mistake, therapy is expensive. We need a high-tech lab, specialized personnel, and so on. An additional advantage is also that the cells or viral vectors do not have to be sent over great distances, which is a great cost and additional complexity that can now be avoided. This project will ensure that we save a significant amount of time and that we already save on transport costs. That already makes a huge difference,' Professor Catherine Vanderstraeten adds. 'It is for this reason that the Fund for Innovation and Clinical Research UZ Gent is awarding this important investment. In this way we hope to find more solutions in the coming years for patients for whom there is often no adequate solution.'

More information