Phase I trial of multilamellar mRNA lipid particles for recurrent pediatric high grade glioma
Due to the killing capacity of the immune system, immunotherapy targeting recurrent pediatric high grade glioma holds significant potential to meet the need for more effective and less toxic treatment options for patients with this disease. We have developed a new mRNA vaccine called RNA-nanoparticles. RNA-nanoparticles are small enough to communicate with the immune system redirecting it against a patient's specific cancer. These nanoparticles will be personalized against a patient's specific brain tumor and the technology is amenable to "off the shelf" manufacturing.
Project Goals
By employing RNA-nanoparticles encoding for tumor specific targets as an innovative and versatile platform, we can reprogram the intratumoral environment of pediatric high-grade glioma into an immune activated state. These particles have been active in mouse brain cancer and in pet dogs with glioma. Based on these data and promising safety data generated to date, we intend to conduct a phase I trial evaluating the safety and activity of this novel mRNA vaccine platform in children with recurrent high-grade gliomas.
Project Update 2024:
Cancer is not just simply uncontrolled cell division, but rather, a unique and personalized ecosystem that is naturally selected in an environment it actively subverts. Targeted therapy often fails to overcome this heterogenous environment leaving only the ‘nuclear approach’ (i.e. surgery, radiation and chemotherapy). However, there is hope- hope in what nature has provided to protect us from enemies both foreign and domestic. Our immune system is a lethal fighting force and actively eliminates most cancers before they arise. Unfortunately, when a cancer manifests, the immune system has failed; and worse, it actively shields, nourishes and protects cancer cells. Although new drugs have been developed to redirect the immune system against cancer, most immunotherapies fail and cannot overcome the regulatory tumor immune microenvironment. Thus, the starting point of successful cancer immunotherapy is the reprogramming of the tumor immune microenvironment. In this regard, we have created personalized mRNA cancer vaccines (over 10 filed patents) that simultaneously reprogram the tumor immune microenvironment while generating sustained tumor specific immunity. We have received FDA-IND approval to run first-in-human studies, and on the verge of treating children with recurrent high-grade gliomas
