Neurofibromatosis type 1 (NF1) is an autosomal dominant disorder caused by mutations in the NF1 gene, significantly increasing the risk of developing peripheral nerve sheath tumors (PNSTs).

A recent study is the first to explore the effects of cold atmospheric plasma (CAP) on NF1-associated PNSTs, revealing that these tumors are highly sensitive to CAP exposure, which leads to significant cell death primarily through apoptosis.

The findings suggest that CAP could serve as a promising therapeutic option for NF1 patients, particularly those suffering from aggressive malignant peripheral nerve sheath tumors (MPNSTs).

This study also highlights the potential of CAP as a non-invasive method to enhance drug delivery across the blood-brain barrier (BBB), which typically restricts drug passage.

CAP generates reactive oxygen and nitrogen species (RONS) that can disrupt tight junctions of the BBB, potentially increasing drug permeability and improving treatment efficacy.

Previous methods to overcome BBB challenges, such as focused ultrasound and nanoparticles, have limitations, underscoring the need for innovative strategies like CAP.

Researchers are utilizing an organoid model to screen FDA-approved drugs, aiming to identify effective treatments that can slow tumor growth in NF1 patients.

In vitro assays conducted on human cells demonstrated that CAP treatment led to increased levels of cleaved caspase-3, indicating activation of the apoptotic pathway, alongside a decrease in cell proliferation.

The study concludes that CAP treatment in the oropharynx may aid in the recovery of intensive care patients without causing cell damage.

The research team successfully established organoids from several NF1 patients, demonstrating that these organoids reflect critical features of the original tumors.

Further research is necessary to optimize CAP treatment parameters and assess the long-term effects of immune cell activation induced by CAP treatment.

In vivo studies using an NF1-associated MPNST xenograft mouse model showed that CAP treatment significantly inhibited tumor growth over time.