Project 1

"Nanocarriers" for drug delivery to mast cells

Evan Scott is working to develop “nanocarriers” to successfully transport drugs directly to mast cells in aggressive systematic mastocytosis (ASM), a form of leukemia that currently has few treatment options.

“Through funding from the CSMI, we aim to develop novel strategies to address ASM by combining a versatile slow-releasing nanoparticle delivery system, developed by the Evan A. Scott Research Group, targeting a unique human mast cell receptor, with pre-clinical testing in the Bruce Bochner Lab involving human malignant mast cell lines and a novel mouse model,” said Scott.

Principal Investigator

Evan Scott

Project 2

Treatment of glioblastoma with spherical nucleic acids

Co-Principal Investigator Alexander Stegh, along with the Mirkin Research Group, is utilizing the CSMI grant to tackle glioblastoma by utilizing spherical nucleic acids (SNAs) to trigger an immune response against the most deadly form of brain tumor.

“Vaccines, drugs, and modified human cells that activate the immune system against tumor growth can improve the outcomes and prolong the lives of patients diagnosed with some type of cancers, but have failed to provide survival benefits for patients with glioblastoma,” said Stegh.

Spherical nucleic acids (SNAs), nanostructures consisting of ball-like structures with DNA and RNA arranged on the surface of a nanoparticle, can be digitally designed as precise, personalized treatments to shut off genes or cellular activity. Utilizing SNAs, developed by Mirkin, the research team will seek to activate the stimulator of interferon genes (STING) pathway, a major mechanism that regulates immune responses, against tumor tissue.

“We will test the hypothesis that SNA-mediated activation of the STING pathway alone and in combination with FDA-approved immunotherapies, such as checkpoint inhibitors, will effectively kill tumor cells in both cell and animal models,” said Stegh. “These studies will pave the way for testing STING-SNAs in early clinical trials for the treatment of glioblastoma patients.”

Principal Investigator

Alexander Stegh

Project 3

Other projects

Nathan Gianneschi received a CSMI grant to study a new first-in-class therapeutic for Huntington’s disease.

Samuel I. Stupp received funding to advance his research on the discovery of novel immunomodulatory peptide-amphiphile nanostructures for autoimmune diseases.

Principal Investigators

Nathan Gianneschi

Samuel Stupp