Neuroblastoma is one of the common solid tumors found in young children. The tumors often contain a number of MYCN copies—children with these neuroblastomas face a higher risk of poor prognosis.  The MYCN oncogene is sought to be a cause of a number of deadly solid tumors—including neuroblastoma. Recently, a research team uncovered a previously unknown and crucial link between polyamines and MYCN demonstrating that the polyamine pathway is entirely regulated by the MYCN oncogene.

Researchers have identified the gene responsible for the uptake of polyamines by neuroblastoma cells and have shown that this gene can be inhibited using a drug called AMXT-1501. By combining these two drugs, DFMO and AMXT-1501, with conventional chemotherapy, the research team was able to significantly increase survival in mice with established neuroblastoma tumours, as well as prevent tumour formation in mice who were tumour-free but genetically prone to developing the disease.

The newly discovered combination therapy is now being tested in the US in adult patients with a broad range of cancers, in Phase 1 clinical trials run by pharmaceutical company Aminex Therapeutics (see link below).

The objective of this study is to determine the safety and tolerability of oral AMXT 1501 dicaprate (AMXT1501) in combination with DFMO in patients with advanced solid tumors. Secondary objectives include characterization of plasma pharmacokinetics (PK) of AMXT 1501 as well as pharmacodynamic (PD) assessment of the impact of AMXT 1501 in combination with DFMO on polyamine uptake by circulating lymphocytes (blood cells).

To these aims, the study will evaluate the safety, PK and PD profiles of orally-administered AMXT 1501 and DFMO. Approximately, 52 patients will be enrolled to determine the maximum tolerated dose (MTD) and recommended phase 2 dose (RP2D) of AMXT 1501 and DFMO in combination. The MTD is defined as the highest dose level below at which dose escalation is stopped.

Study CRO:         Novella (IQVIA)

Study Sponsor:    Aminex Therapeutics

Source: ClinicalTrials

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