Novartis Farydak was approved by the FDA to treat multiple myeloma 2015. Now, researchers in Australia believe that the drug can be combined with a clinical early phase agent to deal with neuroblastoma, a type of nerve tissue cravings most commonly seen in young children .
Professor Murray Norris and his colleagues at the Children’s Cancer Institute Australia for Medical Research tested Farydak in combination with an investigational drug called CBL0137 in a mouse model of neuroblastoma. In mice who received the cocktail, “the tumors disappeared and never returned throughout the experiment, while the tumors continued to grow in mice that received either no treatment or just drug treatment,” said Norris in a statement. He presented the results at the 30th EORTC-NCI-AACR Symposium on molecular targets and cancer therapy in Dublin.
The two drugs offer two different mechanisms: One launches a direct attack on the cancer cells ability to repair their DNA, and the other induces robust immune response, explains Norris. The results are especially encouraging because the tubing was more effective than other chemotherapy combinations that the laboratory has tested in aggressive neuroblastoma, he added.
Farydak belongs to a family of medicines called histone deacetylase (HDAC) inhibitors. By blocking HDAC enzymes in multiple myeloma, the drug changes to genes that suppress the growth of cancer cells and lead to their death.
CBL0137, known as a curaxin, acts by binding to the Facilitat Chromatin Transcription (FACT) complex, which is often found in tumor cells but not in normal cells. It leads to double inhibition of NF-kB-one protein complex that plays a key role in the immune response and has been linked to cancer and heat shock factor 1 (HSF1). It also activates the tumor-suppressing p53 gene.
“Unlike conventional chemotherapeutic drugs that interact with DNA, CBL0137 is harmful to DNA and therefore relatively toxic,” says Norris.
Incuron, a joint venture Cleveland BioLabs curved from 2015, now owns CBL0137, currently in phase 1 studies in solid tumors and lymphoma. A phase 1 clinical study CBL0137 alone in children with neuroblastoma is now planned for 2019.
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Other researchers also explore new ways of treating neuroblastoma . Researchers at Nemours Children’s Hospital and University of Central Florida have suggested that the Zika virus’s ability to infect immature nerve cells can also be used to kill neuroblastoma. And New York-based Y-mAbs Therapeutics, recently publicized in a $ 96 million IPO, develops naxitamab as a first-hand treatment for neuroblastoma. The drug targets GD2, an antigen on the surface of cancer cells that has emerged as a promising therapeutic target.
As Norris sees it because the CBL0137-Farydak combination dose can activate an immune response, it can “significantly increase the effect of immunotherapy drugs that are otherwise ineffective for neuroblastoma.” The team follows that logic, tests the combination with immuno-oncological drugs in mice .