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Glioblastoma, the most common and aggressive form of brain cancer, is notoriously difficult to treat. Once arisen, the tumor rapidly invades healthy brain tissue, making removal by surgery nearly impossible and chemotherapy or radiation therapy success short-lived. Even immunotherapy drugs, increasingly relied upon when first lines of treatment fail, have proven ineffective, leaving glioblastoma patients with very few options. But this may change soon.
In 2018, the Foundation for the National Institutes of Health (FNIH) established the FNIH Trailblazer Prize for Clinician-Scientists to recognize “the outstanding contributions of early career clinician-scientists” whose research “translates basic scientific observations into new paradigm-shifting approaches for diagnosing, preventing, treating or curing disease.”
Chimeric antigen receptor (CAR) T therapy, in which a patient’s own immune T cells are genetically engineered to target and kill their tumor cells, have been the subject of intensive research efforts since the first patients were treated in 2011. Fueled by the promise of immune cells that can serve as a “living drug” against cancer, scientists are committed to making CAR T cells safe and effective for more patients. Their investment is warranted: after a decade in remission, those first patients to receive CAR T cells were declared “cured” of leukemia.
Adenoid cystic carcinoma (ACC) is a rare but aggressive cancer that usually develops in the salivary glands and is often diagnosed in younger adults. Because of its rarity, ACC has received relatively little attention from cancer researchers, and as a result, there are no approved therapies for the disease.
Craniopharyngiomas are a rare type of brain tumor that arise near the pituitary gland and are very difficult to treat, whether surgically or with radiation therapy, without inflicting vision loss, memory loss, or hormone disruption. Even in cases when the tumor is successfully removed, craniopharyngiomas are notorious for coming back.
If you asked a hundred people to rate a hundred movies, you would generate enough data to be able to make some predictions. Someone who enjoyed Notting Hill would likely enjoy Pretty Woman, for instance; the new Guardians of the Galaxy movie will likely be a hit with longtime Marvel fans. This is an example of a bipartite dataset, which measures interactions between two types of entries—in this case, movies and viewers—and can be used not only to predict unmeasured interactions but also to reveal the underlying rules governing a system.
At the annual meeting of the American Association for Cancer Research, held this spring in Orlando, former Damon Runyon Clinical Investigator John V. Heymach, MD, PhD, started with the bad news.
In the context of cancer, “drug addiction” has a different meaning—counterintuitively, it’s when cancer cells, not patients, depend on continuous treatment for survival. This can happen if, after the drug target is inhibited, some compensatory signaling pathway is turned on that serves a similar function in the cancer cell. When drug treatment stops, the cell goes into “withdrawal” and this alternative pathway becomes overactive, so much so that it leads to cell death.
Due to their critical role in so many cellular functions, proteins that span the cell membrane are the target of more than half of all FDA-approved drugs. Some of these transmembrane proteins are single-pass, meaning they cross the membrane only once, while others are more complex, multipass proteins, meaning they cross the membrane in at least two places. Drugs targeting the latter are primarily small molecule inhibitors, named for their size relative to antibodies and other large proteins.
A major challenge in treating brain cancer is delivering drugs across the blood-brain barrier (BBB), the dense network of cells and blood vessels that prevents toxins and pathogens from entering the brain. Unfortunately, the BBB also bars entry to therapeutic molecules, leaving highly toxic radiation or chemotherapy treatment as the only recourse for many patients with brain cancer.
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