Made from cells taken from a patient, which are then manipulated and administered back into said patient, autologous products have, perhaps, come of age this year through the US approvals of CAR T cell therapies Kymriah (tisagenlecleucel) and Yescarta (axicabtagene ciloleucel)
But developers are also looking at ‘off-the-shelf,’ or allogeneic, cell therapies which can be manufactured more in line with standard biotherapeutics, with consistent pharmaceutical release criteria, over time and from batch to batch.
This is the strategy of UK-based stem cell developer Reneuron, the firm said while presenting its second quarter results yesterday, as if successful the manufacturing of such therapies will be cheaper and easier than their autologous equivalent.
“It is much easier to have a good margin there because you can make big batches that can go to many patients, and you can have the product sitting on the shelf waiting for the patient as they walk in the door,” said CEO Olav Hellebø. “And that's what we do, everything we do is Allogeneic.”
The firm’s lead products are based on its CTX neural cell line and look to stimulate natural repair mechanisms in organs affected by diseases including stroke disability and critical limb ischaemia.
“The only theoretical disadvantage would be if there are any rejections or antibodies being formed and we've never seen that for our products they've been selected for that very reason and we also don't use any immune suppressants in our clinical trials.”
‘Bread and butter’ surgery
Hellebø added the administration of allogeneic products also has an advantage over that of autologous therapies, as while both are specialised the latter relies on training up and educating hospital staff and managing a timely supply chain.
“It's very much bread and butter surgery for a neurosurgeon,” he explained. “They use a stereotactic frame, they use MRI to pinpoint exactly where they want to deliver the cells. They drill a hole in through the skull and they deliver the cells that way.”
The process takes about two hours, he added: one-and-a-half to set up and 30 minutes to do the procedure.
Clinical batches of CTX cells are made from Reneuron's facility in Pencoed, Wales and distributed to trial sites across the UK.
The batches have a shelf life of around six months, Hellebø told stakeholders, but “that probably will double” as the firm continues developing its own cryopreservation technologies.