THE SCIENCE BEHIND OPIOIDS Concerns over the opioid epidemic continue to grow, with deaths from narcotic overdoses the leading cause of death in people under 50 last year. Nearly half of those deaths were attributable to prescription opioids. The directors of both the Center for Disease Control (Atlanta, GA) and the Food and Drug Administration (Silver Spring, MD) have called … Read More
Circadian Rhythm & Disease
AND THE BEAT GOES ON Earlier this week, the 2017 Nobel Prize in Physiology or Medicine was awarded to three American scientists (Jeffrey Hall and Michael Rosbash, of Brandeis University, and Michael Young, of Rockefeller University) for their work in deciphering the molecular basis of circadian rhythm – the 24-hour cycle that governs the inner workings of all life on … Read More
The Multiple Myeloma Landscape
BLOOD CANCER: MULTIPLE MYELOMA Plasma cells are the antibody-producing cells of our immune system which happen to play a critical role in our defense against infections. In multiple myeloma, plasma cells begin to grow and divide in an uncontrolled manner, forming a cancerous mass known as a plasmacytoma. Marrow — which produces plasma — no longer functions in our defense, it simply takes … Read More
Eye Of The Cytokine Storm
THE FLASH OF THE FIRST CAR-T Last week’s much anticipated FDA approval of the first chimeric antigen receptor T-cell (CAR-T) therapy for acute lymphocytic leukemia hails as the first gene therapy on the US market. Classified as a “cell-based gene therapy,” Novartis’ (Basel, Switzerland) Kymriah works by removing patients’ T-cells, using a viral vector to introduce a gene that will allow the … Read More
Vaccines: Schooling The Herd
VACCINATION NATION Back to school means shopping for new school supplies, adjusting to a new schedule, and making sure all required vaccinations are up to date. Every state requires school-age children to be vaccinated against certain infectious diseases including tetanus, hepatitis B, measles, mumps, rubella, polio, pertussis (whooping cough), and chicken pox. Vaccination policies are highly effective at eliminating many … Read More
The Science Of CRISPR/Cas9
CRISPR/CAS9 UPDATE As CRISPR/Cas9 adds new indications to its resume, legal battles over its IP continue to be waged in the US and Europe. On the clinical front, CRISPR/Cas9 entered its first human trial at Sichuan University (Chengdu, China) last fall for metastatic lung cancer, and is widely expected to do so in the U.S. by the end of the year. This … Read More
Stopping A Big Problem: Blood Clots
MEDS FOR THINNER BLOOD CAN EQUAL FEWER CLOTS, BUT HOW? The FDA’s recent approval of Portola Pharmaceuticals’ (South San Francisco, CA) new blood thinner drug Bevyxxa paved the way for the prevention of blood clots in patients hospitalized for conditions such as heart failure, stroke, and pulmonary disease. The medical term for blood clot is venous thromboembolism (VTE), but if we take it apart: “venous” means relating … Read More
Breast Cancer Subtypes
FOUR MOLECULAR VARIANTS EXPLAINED Hearing your doctor utter the words HER2-positive, HR-positive, triple-negative, or BRCA mutation can be devastating — even for the most resilient person. Simply put, breast cancer is a complex disease. A diagnosis can be derived from any combination of the factors listed above — or, none at all. The National Cancer Institute (Bethesda, MD) has outlined four molecular subtypes of the disease. … Read More
Epigenome: Writing, Reading & Erasing
FOUNDATIONS OF EPIGENETICS Genetic mutations — changes in the order of the A, C, G, and T nucleotide bases that make up a gene — have been the primary focus of cancer researchers over the last several decades. By sussing out mutations involved in regulating cell growth and division, scientists better understand the molecular range of different cancers and consequently develop … Read More
Nanobodies: These Are Not Your Mother’s mAbs
The Drug Kingpins Monoclonal antibodies (mAbs) are the undisputed drug kingpins. In 2013, the mAb market raked in $75 billion in combined sales, covering a whole range of indications from cancer and infectious disease, to autoimmune disorders, and even high cholesterol. Despite the success, mAbs have one chink in their armor: they cannot enter cells due to their large size, hampering their range … Read More
Phage — More Than Just A Phase
Old Way Of Fighting Bacteria Renewed One of the greatest public health challenges of the 21st century is antibiotic resistance, which occurs when a few bacteria in a given population develop a genetic mutation that enables them to survive — even in the presence of antibiotics. How do bacteria become drug resistant? Suppose a particular antibiotic inhibits an enzyme required for bacterial replication. … Read More
RNA Therapeutics March Onward
Taking Steps With Antisense With their high specificity and relative low manufacturing cost, RNA therapeutics may be tomorrow’s biotech sweetheart. In fact, chances are good that previously “undruggable” targets that cannot be accessed by small or large molecule drugs, are now within reach. However, the main roadblock continues to be delivery—getting the RNA drug where it needs to be, in high enough concentrations, to … Read More
Putting The NA in DNA
Nucleic Acid Therapeutics Small molecule, peptide, and biologic drugs aren’t the only players in the game of drug development. A fourth class of therapeutics differs from all three of these: nucleic acid-based drugs. These drugs are rising in prominence due to their potential to specifically target a wide range of diseases, including various types of cancer, autoimmune, and infectious diseases. Companies … Read More
The Science Of CRISPR/CAS9
CRISPR/CAS9 UPDATE CRISPR/Cas9 can’t seem to stay out of the news — from first in human to patent disputes, we here at the WEEKLY want to update you on this hot technology. A group of scientists from the State Key Laboratory of Proteomics (Beijing, China) and the National Center for Protein Sciences (Beijing, China) recently reported the first ever edit using CRISPR/Cas9 in healthy human … Read More
The PARP Race Is On
PARP1 INHIBITOR LINEUP PARP1 inhibitors are making a strong statement! Tesaro’s (Waltham, MA) just-approved Zejula has garnered predictions of blockbuster status. AstraZeneca’s (Cambridge, UK) Lynparza was the first PARP1 inhibitor to make it to market back in 2014, and their recent clinical trial results showed significant survival benefit in ovarian cancer. Clovis Oncology (Boulder, CO) achieved the second FDA approval of a PARP1 inhibitor with Rubraca in … Read More
The Intrigue Of HIF
The Versatility Of HIFs Quite a few headlines touting a term called “HIF compound” or “hypoxia-inducible factor compound” have intrigued us here at WEEKLY headquarters. GlaxoSmithKline (London, England), Akebia Therapeutics (Cambridge, MA) and more have HIF-inducing drugs in the pipeline which may prove to be attractive alternatives to Amgen’s (Thousand Oaks, CA) injectable Epogen currently on the market. In earlier stages of research, HIFs are being studied for their connection to tumor … Read More