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CAR-T therapies: All you wanted to know and were too afraid to ask

Immuno-oncology has become a popular field of research with UK companies at the forefront of the latest innovations
CAR-T therapies: All you wanted to know and were too afraid to ask
CAR-T therapies re-engineer the blood to recognise cancer cells that have been hiding in the body that haven’t been destroyed

A new cutting-edge cancer treatment has been given the green light by the UK authorities after a deal over pricing was reached with its developer Novartis AG (SWX:NOVN).

Kymriah will be available for children and young people with aggressive leukaemia and is one of a new generation of treatments that uses the body’s own immune system to tackle the killer disease.

It what’s called a CART-T therapy, which stands for chimeric antigen receptor T-cell. As the name suggests the technology involves T-cells, white blood cells that help the immune system fight disease and infection.

Re-engineering T-cells

CAR-T therapies re-engineer the blood to recognise cancer cells that have been hiding in the body that haven’t been destroyed.

In clinical trials, Kymriah has proved effective, even in patients with the advanced form of the disease, so its rollout here in the UK will be welcomed by young leukaemia sufferers. But because it is a complex, personalised treatment the Novartis innovation is also eye-wateringly expensive with a list price of £282,000 per year.

And it isn’t exactly a silver bullet. About 400 children are diagnosed with acute lymphoblastic leukaemia each year but nly 15 might be suitable for the CAR-T treatment.

Hot area of research

Nevertheless, this area of immuno-oncology has become a popular field of research with UK companies at the forefront of the latest innovations.

Leading the pack is Autolus Therapeutics, a spin-out from University College  London, which is now listed in the US with a market capitalisation in excess of US$1bn.

Backed in its formative stages by Arix Biosciences and Neil Woodford’s Patient Capital, it is running five CAR-T programmes covering six blood-borne cancers and solid tumour indications.

Smaller, with a market cap of US$162mln, but a potential pocket rocket is AIM-listed MaxCyte (LON:MXCT), which is gearing up to begin clinical trials of MCY-M11, a chimeric antigen receptor that targets solid tumours.

The phase I study will evaluate the safety of the potential treatment in people with relapsed, or difficult-to-treat ovarian cancer and peritoneal mesothelioma (a form of cancer that affects the abdomen). MaxCyte used its CARMA platform to develop the treatment.

Unsung hero

Finally, Oxford Biomedica (LON:OXB) has been slightly overlooked in all the hullaballoo about Kymriah.

Yet without its technology, the treatment wouldn’t exist. For OXB is the sole manufacturer of the lentiviral vector that encodes the CD19-directed chimeric antigen receptor in Kymriah.

For those who didn’t understand the foregoing, a lentiviral vector is a method used to insert, modify or delete human genes.

Effectively, it re-programmes the T-cells.

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