Proving the efficacy of cell therapy treatments to convince investors

‘What convinced investors, and what convinced our alliance partners were the clinical result we had in chronic lymphatic leukaemia, I think that’s important. I think with animal models, they are very helpful in a sense, in just the bare bones test system, but to prove efficacy, in the end is to run clinical trials.’ Jan Joseph Melenhorst, Director of Product Development & Correlative Sciences Laboratory from the University of Pennsylvania, discusses the necessary steps that must be taken for their cell therapy to become a viable treatment option.

Within his department at the university, Melenhorst examines the post infusion specimens of cells to better understand the potency and toxicity of the cells they infuse into patients. To enhance the potency of cells, additional gene editing tools can be included to modify the genome of the T-Cells and further enhance the expression of the targeting gene. Melenhorst speaks of his own experience with the CRISPR-Cas9 gene editing tool, but also draws upon the use of TALENs, stressing its importance to ‘the future of autologous and allogeneic cell therapies’. It is the autologous therapies Melenhorst believes are the current future of CAR-T therapy.

Whilst Melenhorst highlights recent breakthroughs, he is also conscious that:

‘We have not seen a product that has commercial viability at this point. It’s getting there, but I think automation of the process is really important. Allogeneic CAR-T cells are a good step in this direction, but in the end, in my mind, autologous products are the way to go. If you can streamline the process better, we can understand which cells actually drive the therapy – if we can put that in a process then the therapy will have a great future.’

Melenhorst is confident about the future that CAR-T cells can provide for the treatment of numerous cancers, but he is also very aware there is still a vast distance to overcome.

To overcome this distance, he believes in the power of ‘synergistic’ partnerships, such as the one his University of Pennsylvania has developed over the past four years with Novartis. The combined expertise and experience they have applied to their clinical trials have created, what Melenhorst describes as, a ‘well-oiled machine’. This in turn has allowed them to develop and trial new drugs faster and more efficiently.

Speaking further on the efficacy of their therapy, Melenhorst noted that the results ‘of our therapy were far better in patients than animals’ and that is was these results that ‘convinced [their] investors and alliance partners’. He remains confident in the necessity of clinical trials to improve any negative public opinion that currently exists about the financial burden of CAR-T cell therapies.

Watch the full interview with Jan Joseph Melenhorst, filmed at Cell Therapy Manufacturing & Gene Therapy Congress,  above or here.

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