Identification of Combinations For Pediatric Rhabdomyosarcomas

Dieter Zopf
Published: Wednesday, Oct 24, 2018



Dieter Zopf, Principal Scientist at Bayer AG, Research and Development, Pharmaceuticals, Oncology, discusses a study analyzing effective combinations with regorafenib (Stivarga) for the treatment of pediatric rhabdomyosarcomas.

Regorafenib was tested with drugs that were recommended by a consortium of European experts, Zopf says. Results of the preclinical study suggest that more work should be done with regorafenib in combination with irinotecan and vincristine, according to findings presented at the 2018 ESMO Congress. There was a heterogenous pattern of response in the preclinical models ranging from complete remission (CR) to insensitivity, notes Zopf. CRs were observed in monotherapy with irinotecan, vincristine, and paclitaxel.

Zopf adds that although treatment interruptions were required due to toxicity issues, no treatment-related deaths occurred. This was significant because only 1 mouse was used for each drug. A stepwise dosing strategy was utilized, starting with half the standard dose, then three-quarters the dose, then the standard.


Dieter Zopf, Principal Scientist at Bayer AG, Research and Development, Pharmaceuticals, Oncology, discusses a study analyzing effective combinations with regorafenib (Stivarga) for the treatment of pediatric rhabdomyosarcomas.

Regorafenib was tested with drugs that were recommended by a consortium of European experts, Zopf says. Results of the preclinical study suggest that more work should be done with regorafenib in combination with irinotecan and vincristine, according to findings presented at the 2018 ESMO Congress. There was a heterogenous pattern of response in the preclinical models ranging from complete remission (CR) to insensitivity, notes Zopf. CRs were observed in monotherapy with irinotecan, vincristine, and paclitaxel.

Zopf adds that although treatment interruptions were required due to toxicity issues, no treatment-related deaths occurred. This was significant because only 1 mouse was used for each drug. A stepwise dosing strategy was utilized, starting with half the standard dose, then three-quarters the dose, then the standard.

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