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Current therapies for glioblastoma multiforme (GBM), the highest grade malignant brain tumor, are mostly ineffective, and better preclinical model systems are needed to increase the successful translation of drug discovery efforts into the clinic. Scientists at the National Cancer Institute (NCI) have developed and characterized an orthotopic genetically engineered mouse (GEM)-derived model of GBM that closely recapitulates various human GBM subtypes and is useful for preclinical evaluation of candidate therapeutics.

Ovarian cancer is one of the most common and lethal types of gynecological malignancies worldwide, accounting for approximately 295,000 new cases and 185,000 deaths annually. The high lethality rate is due to multiple reasons, including recurrence and the resistance of recurrent tumors to chemotherapy. Cell line models are crucial for preclinical cancer studies, to identify mechanisms of disease, to study drug resistance, and to screen for candidate therapeutics. 

Early detection of liver cancer, such as hepatocellular carcinoma (HCC), is key to improve cancer-related mortality. More than 800,000 people are diagnosed with this cancer each year throughout the world. Liver cancer is also a leading cause of cancer deaths worldwide, accounting for more than 700,000 deaths each year. Currently, millions of Americans and possibly billions in the world are considered at risk for developing liver cancer.

Nanoparticles such as lipid-based nanoparticles (LNPs) represent a relatively new era of targeted drug delivery systems wherein these biocompatible particles can carry the drug(s) of interest to a specific tumor site. The new generation of nanoparticles, known as stealth nanoparticles, are engineered to have a coating of polyethylene glycol polymer (PEG) or other glycolipids that enable them to evade the immune system and have a longer circulation lifespan as well as improved bioavailability to diseased tissue and reduced non-specific toxicity.
 

The thymus is an integral part of the adaptive immune system as it generates T cells. Its function diminishes rapidly as the body ages, leading to a compromise of the immune system in the elderly. Reconstitution of adaptive immunity through mass production of different T cell types is therefore a therapeutic need in immunocompromised populations. Furthermore, production of T cells with specific receptors targeting cancer cells is an important cancer immunotherapy approach.

Cancer cells can spread to various regions in the body in a process called metastasis which is associated with non-responsive to treatment and thus reduced survival. Identifying the markers of metastasis has been a major concern in the field of cancer diagnosis and therapy. Interestingly, research has shown that there is an increase in myeloid progenitors and myeloid cells at various stages of metastasis in an attempt by the immune system to  suppress cancer cells. This presents a promising technology for cancer immunotherapy.

Hematopoietic progenitor cells (HPC) are multi-potent hematopoietic lineage cells that can differentiate into any type of blood cell, including but not limited to erythrocytes, T cells, B cells, and natural killer cells. As such, they have high therapeutic potential in the fields of regenerative medicine and cancer immunotherapy, especially when generated from patient-derived induced pluripotent stem cells (iPSC). Currently, the most efficient protocol to produce HPCs is co-culturing human iPSCs (hiPSC) with mouse stromal cells as a two-dimensional (2D) monolayer.

Recombinant immunotoxins (RITs) constitute a promising solution to hematologic cancers (e.g., Multiple Myeloma [MM]). RITs are chimeric proteins composed of a targeting domain fused to a bacterial toxin. Upon binding to a cancer cell displaying the target antigen, RITs are internalized, metabolized and the released toxin kills the cell. While highly active and effective, current RITs have short half-lives, requiring them to be used in high concentrations for treatment. At such high concentrations, RITs may show nonspecific activity and kill healthy cells.

Research and development leading to the discovery of novel antibiotics has waned in recent years. At the same time, the emergence and spread of antimicrobial resistance has compounded the global danger to human health from bacterial infections. 

Many known chemotherapeutic drugs kill abnormal cells through a process called apoptosis. Bcl-2 proteins are negative regulators of apoptosis that control cell survival and death. Increased expression of anti-apoptotic Bcl-2 proteins commonly occurs in up to 30% of all cancers, providing cancer cells a pro-survival advantage to evade cell death, grow, and proliferate. Drugs targeting these specific anti-apoptotic proteins are potential anti-cancer therapeutics.