At the World Medical Innovation Forum on May 19-21, faculty will deliver rapid fire presentations highlighting new discoveries and insights on ways gene and cell therapy will transform the future of health care. We asked Brigham speakers to give us a preview of what they’ll be presenting at this year’s forum. More information on the World Medical Innovation Forum can be found here.

Versatile Polymer-Based Nanocarriers for Targeted Therapy and Immunomodulation

“Nanomedicines, as originally conceived, were developed to increase the uptake of chemotherapy agents by tumors and to reduce their off-target toxicity. Now that therapeutic targeting of the immune system in cancer has proven efficacious, the question becomes: Can nanomedicines serve to effectively target and modulate the immune system? Nanomaterials’ ability to enhance the accumulation of immunomodulators in tumors and lymphoid organs, and to modulate the interactions with specific immune cells, points at their high potential in this field. Polymer-based nanomaterials can help to safely engage with the appropriate target cells at the right time and place, while delivering the drugs in a pattern that maximizes efficiency, while eliminating toxicity.

My lab has developed polymeric nanoparticles for in vivo gene therapy that enable targeting cancer and immune cells with unprecedented transfection efficiencies, in a range of solid tumors. We have been using these vehicles to identify, and study the mechanisms of action of, combination cancer immunotherapies, some of which we are looking to advance to the clinic for the treatment of solid tumors.”

-Natalie Artzi, PhD, Assistant Professor at the Department of Medicine, Division of Engineering in Medicine

Oncolytic Viruses: Turning Pathogens into Anticancer Agents

“Oncolytic viruses are genetically engineered viruses that infect and replicate selectively in tumor cells, while sparing normal cells. Several types are in clinical trials for various cancers and one is FDA-approved for melanoma. Each oncolytic virus is akin to a different drug and my NIH-funded laboratory at the Brigham has been engineering different types of oncolytic viruses. We are particularly excited about one where we have modified the virus, herpes simplex, by removing viral pathogenic genes (that harm normal cells) and substituting these with human genes that allow this modified virus even more selective killing of cancer cells with higher potency when compared to previous versions of oncolytic viruses. We are now testing this new advanced version in preclinical models with the intent of translating it into clinical trials for patients with malignant brain cancer.”

-Nino Chiocca, MD, PhD, Chair, Department of Neurosurgery

Unlocking the Secret Lives of Proteins in Health and Disease

“Genetic mutations cause many human diseases, most often driven by errors in the DNA that translate into errors in protein function. Proteins are ultimately the ‘business end’ of what goes wrong inside human cells and must therefore be the focus of our efforts to decipher the mechanisms that drive cells to malfunction, leading to devastating diseases.

Our lab is specifically interested in unlocking the ‘secret lives’ of proteins that are misshapen, and often toxic to cells, as a result of genetic mutations. Specifically, a major focus of the lab is the role of the TMED family of cargo receptors in protein quality control, trafficking, and secretion. We recently revealed that TMEDs handle several misfolded membrane and secretory protein cargoes including mucin 1, uromodulin and rhodopsin. This work has implications for more than 50 diseases collectively known as toxic proteinopathies affecting the kidney, the eye, the brain and beyond.”

-Anna Greka, MD, PhD, Nephrologist and Principal Investigator, Division of Renal Medicine, Department of Medicine

RNA Therapy for Brain Cancer

“In my laboratory, we study the role of non-coding RNAs in the regulation of cell biology, and particularly how their unique features can be exploited to engineer a novel and versatile gene therapy platform. Camouflaged as microRNA genes, our “RNA drugs” are recognized by the ubiquitous microRNA processing machinery, which cleaves them into multiple active pieces, each targeting desired cellular pathways. This strategy allows for an unparalleled high degree of multitargeting, ideal to treat complex, polyfactorial pathologies like cancer or neurodegenerative diseases.”

-Pierpaolo Peruzzi, MD, PhD, Neurosurgeon and Principle Investigator, Department of Neurosurgery

Repurposed Tumor Cells as Killers and Immunomodulators for Cancer Therapy

“Despite great leaps in the treatment of malignant neoplasms over the past decades, solid tumors remain the most challenging tumors to treat. We have created CRISPR/Cas9 edited and engineered bi-functional therapeutic tumor cells (ThTC) that simultaneously eliminate residual tumor cells and elicit active immunity which prevents tumor recurrence in primary and recurrent highly malignant brain tumors, glioblastomas (GBMs) as well as other primary and metastatic solid tumors. Given the complexity of delivering drugs across the blood brain barrier, and tumor resection/biopsy being part of standard of cure for GBM patients, our local ThTC treatment offers a ready availability for personalized cellular therapy for GBMs and serves as a potential cure for this devastating disease. We anticipate that our engineered tumor cell platform will have major contributions in finding a cure for GBM patients and is likely to define a new treatment paradigm for patients with other solid tumors.”

-Khalid Shah, MS, PhD, Vice Chair of Research, Department of Neurosurgery


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