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Death halts CAR-T drug trials
Posted 7 September 2017
Two clinical trials investigating French biotech Cellectis' CAR-T candidate UCART123 have been put on hold after a patient died due to toxic reaction to the therapy.
The 78-year-old man experienced a severe cytokine release syndrome (CRS) reaction together with capillary lead syndrome eight days after he was dosed with the therapy. He died the day after.
The clinical trial, which was investigating the CAR-T as a treatment for blastic plasmacytoid dendritic cell neoplasm (BPDCN), was halted by the FDA along with a second trial in acute myeloid leukaemia.
Currently CRS is a common side-effect of CAR-T and though usually resolved with corticosteroids and Roche's Actemra (tocilizumab), Cellectis' patient had failed to improve after administration of the drugs.
Nor has this been the first fatality to occur with the new class of therapies as US-based Juno Therapeutics last year also had a clinical trial placed on hold after patients died from cerebral oedema.
Cellectis' stock fell heavily following the news after initially riding high on its CAR-T promise especially as last week saw the first-in-class approval of Novartis' CAR-T Kymriah (tisagenlecleucel) and Gilead's multi-billion dollar purchase of Kite's candidate axicabtagene ciloleucel.
Improvements on these initial CAR-T therapies - including addressing safety shortcomings - now remain high on the checklist as the space heats up.
What's next for CAR-T?
Takeda has just announced a partnership with Japanese biotech Noile-Immune in the hopes of scoring a CAR-T success with solid tumours, an as-yet unbroken market with most candidates in the near pipeline targeting blood cancer indications. Kymriah was approved for paediatric and adolescent acute lymphoblastic leukaemia.
"We recognise the enormous potential of next-generation CAR-T cell therapy technology to deliver transformative medicines in oncology," Head of Takeda's Oncology Unit Chris Arendt said.
Meanwhile scientists in the US are attempting to address the severe safety issues, including CRS, associated with the therapies by developing nanoparticles that could lessen the CAR-T cells' capacity for attacking healthy tissue while also extending their treatment life.
In research partially sponsored by Juno Therapeutics, researchers at the Fred Hutchinson Cancer Research Centre formulated the nanoparticles to deposit proteins into cells to affect their behaviour, hoping that by doing so the CAR-T cells can be stopped from attacking non-cancer tissue.
Last week, Roche's Actemra was approved in tandem with Novartis' Kymriah as a specific treatment for CRS induced by CAR-T therapy.
Yajun Ma
yajun.ma@lushmedia.com.au
Posted 7 September 2017
Two clinical trials investigating French biotech Cellectis' CAR-T candidate UCART123 have been put on hold after a patient died due to toxic reaction to the therapy.
The 78-year-old man experienced a severe cytokine release syndrome (CRS) reaction together with capillary lead syndrome eight days after he was dosed with the therapy. He died the day after.
The clinical trial, which was investigating the CAR-T as a treatment for blastic plasmacytoid dendritic cell neoplasm (BPDCN), was halted by the FDA along with a second trial in acute myeloid leukaemia.
Currently CRS is a common side-effect of CAR-T and though usually resolved with corticosteroids and Roche's Actemra (tocilizumab), Cellectis' patient had failed to improve after administration of the drugs.
Nor has this been the first fatality to occur with the new class of therapies as US-based Juno Therapeutics last year also had a clinical trial placed on hold after patients died from cerebral oedema.
Cellectis' stock fell heavily following the news after initially riding high on its CAR-T promise especially as last week saw the first-in-class approval of Novartis' CAR-T Kymriah (tisagenlecleucel) and Gilead's multi-billion dollar purchase of Kite's candidate axicabtagene ciloleucel.
Improvements on these initial CAR-T therapies - including addressing safety shortcomings - now remain high on the checklist as the space heats up.
What's next for CAR-T?
Takeda has just announced a partnership with Japanese biotech Noile-Immune in the hopes of scoring a CAR-T success with solid tumours, an as-yet unbroken market with most candidates in the near pipeline targeting blood cancer indications. Kymriah was approved for paediatric and adolescent acute lymphoblastic leukaemia.
"We recognise the enormous potential of next-generation CAR-T cell therapy technology to deliver transformative medicines in oncology," Head of Takeda's Oncology Unit Chris Arendt said.
Meanwhile scientists in the US are attempting to address the severe safety issues, including CRS, associated with the therapies by developing nanoparticles that could lessen the CAR-T cells' capacity for attacking healthy tissue while also extending their treatment life.
In research partially sponsored by Juno Therapeutics, researchers at the Fred Hutchinson Cancer Research Centre formulated the nanoparticles to deposit proteins into cells to affect their behaviour, hoping that by doing so the CAR-T cells can be stopped from attacking non-cancer tissue.
Last week, Roche's Actemra was approved in tandem with Novartis' Kymriah as a specific treatment for CRS induced by CAR-T therapy.
Yajun Ma
yajun.ma@lushmedia.com.au
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