Pills, Peptides, & Proteins

Emily BurkeBiologics, Biotech Basics, Drug Discovery, Small Molecule Drugs

Promising Peptide Therapies The front runners in the game of drug delivery include small molecule and large molecule drugs, but there is another class that lands right in between: peptides. Several companies, including Rhythm Pharmaceuticals (Boston, MA), Kalos Therapeutics (San Diego, CA), Aileron Therapeutics (Cambridge, MA), and Bicycle Therapeutics (Cambridge, MA) have emerged as prominent players in the peptide arena. Let’s review the differences between the drug classes and … Read More

The Multiple Myeloma Landscape

Emily BurkeBiologics, Cancer, Clinical Trials, Drug Development, Drug Targets, Easily Confused, Genetics, Mechanism of Action, Monoclonal Antibodies, Small Molecule Drugs

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

Emily BurkeBiologics, Biotech Basics, Cancer, Clinical Trials, Drug Development, Drug Targets, Mechanism of Action, Small Molecule Drugs, The WEEKLY

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

Stopping A Big Problem: Blood Clots

Emily BurkeCocktail Fodder, Drug Approvals, Drug Targets, Mechanism of Action, Small Molecule Drugs

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

The ABCs of DNA & RNA

Emily BurkeBiologics, Biotech Basics, Cancer, Cardiovascular Disease, Clinical Trials, Deoxyribonucleic Acid (DNA), Drug Development, Drug Targets, Mechanism of Action, Monoclonal Antibodies, Ribonucleic Acid (RNA), Small Molecule Drugs, The WEEKLY

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

Hypoxia-Inducible Factor (HIF) Compounds & Drug Discovery

Emily BurkeCancer, Clinical Trials, Cocktail Fodder, Deoxyribonucleic Acid (DNA), Drug Development, Drug Targets, Mechanism of Action, Small Molecule Drugs, Term of the Week, The WEEKLY

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

The Mechanics Of Melanoma

Emily BurkeBiologics, Biotech Basics, Cancer, Clinical Trials, Drug Development, Drug Targets, Mechanism of Action, Monoclonal Antibodies, Small Molecule Drugs, The WEEKLY

Spectrum Of Therapies Melanoma accounts for less than 1% of skin cancer cases yet causes the vast majority of skin cancer deaths. If detected early enough, melanoma is almost always curable. If not, its ability to metastasize makes it difficult to treat. Melanoma is more common in young adults than many other types of cancer, with 25% of new cases occurring in people under age … Read More

Therapeutic Antibody Primer

Emily BurkeBiologics, Biotech Basics, Cancer, Clinical Trials, Drug Development, Drug Targets, Easily Confused, Mechanism of Action, Monoclonal Antibodies, Small Molecule Drugs

Basics & Innovations Monoclonal antibody (mAb) therapeutics burst onto the healthcare scene 20 years ago, and today they remain one of the most versatile and effective therapeutics available. The tried and true mAbs are still in high demand, and we suspect this first wave of derivative products clamoring their way through the pipeline will be equally as successful. In this WEEKLY, we’ll … Read More

Eye Of The Cytokine Storm

Emily BurkeBiologics, Biotech Basics, Cancer, Clinical Trials, Drug Development, Drug Targets, Mechanism of Action, Small Molecule Drugs

Understanding CAR-T Safety Chimeric antigen receptor therapy (CAR-T) is our top technology pick of 2016, blazing trails in drug development with its innovative approach of using engineered immune cells to fight cancer. Despite its success in clinical trials, safety concerns remain. Serious adverse events, including death, have occurred as a result of “cytokine storms” during CAR-T treatments. We reviewed the basics of CAR-T in last week’s … Read More

Unwiring The Biology Of Fibrosis

Emily BurkeAntisense, Biologics, Clinical Trials, Drug Development, Drug Targets, Mechanism of Action, Monoclonal Antibodies, Small Molecule Drugs, Term of the Week, The WEEKLY

Fibrotic Disease Innovations Fibrotic diseases — organ and tissue disorders that occur as a result of the buildup of excessive scar tissue — are sounding the alarm in the biotech sector. A silent epidemic creeping up on the Western world involves non-alcoholic steatohepatitis (NASH), or liver fibrosis associated with obesity and type 2 diabetes. Left untreated, NASH can lead to liver failure. Currently, … Read More

The Science Behind The Opioid Epidemic

Emily BurkeBiotech Basics, Clinical Trials, Cocktail Fodder, Drug Development, Drug Targets, Mechanism of Action, Small Molecule Drugs

A Big Pain The opioid addiction crisis gained attention at the highest levels of U.S. policy circles this past year. Presidential candidates that disagreed on nearly everything else vowed to make fighting the epidemic a priority if elected. In July, the U.S. Senate overwhelmingly approved a bill to strengthen prevention, treatment, and recovery efforts. And no wonder – according to … Read More

Killing Cancer At Its Stem Cell Origin

Emily BurkeBiologics, Cancer, Clinical Trials, Drug Development, Drug Targets, Mechanism of Action, Monoclonal Antibodies, Small Molecule Drugs

Killing Cancer When it comes to killing cancer, eradicating every single cell is the goal. But did you know there are different kinds of cancer cells? One specific type, called cancer stem cells (CSC), is catching the eye of drug developers. Discovered more than a decade ago, CSCs are hypothesized to be the drivers of cancer growth and metastasis. Let’s … Read More

Antibiotic Resistance

Emily BurkeBiotech Basics, Clinical Trials, Deoxyribonucleic Acid (DNA), Drug Approvals, Drug Development, Drug Targets, Mechanism of Action, Small Molecule Drugs, The WEEKLY

THE STATE OF ANTIBACTERIALS 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. If one bacterium … Read More

Without A Treg To Stand On

Emily BurkeBiologics, Cancer, Clinical Trials, Drug Development, Drug Targets, Mechanism of Action, Monoclonal Antibodies, Small Molecule Drugs, The WEEKLY

Regulatory T-Cells Make A Pathway After our focus on T-cell activated immunotherapies revving up the immune response, we turn to the other side of the coin—a subset of helper T-cells known as regulatory T-cells. Regulatory T-cells (Tregs) suppress the immune response.  The exact mechanism by which this suppression occurs is not entirely understood, but some of the effect is due … Read More

On A Tumor’s Turf

Emily BurkeBiologics, Cancer, Clinical Trials, Drug Development, Drug Targets, Mechanism of Action, Small Molecule Drugs, The WEEKLY

TACKLING THE SPACE AROUND SOLID TUMORS Covering the science behind T-cell-based immunotherapies has been the name of the game for our past couple of issues. CAR-T and TCR therapies show significant promise in early phase blood cancer clinical trials, but what about solid tumors? Previously mentioned Juno Therapeutics’ (Seattle, WA) Armored CAR technology has declared war on tumors, and as you will read … Read More

Battling CLL: Venclexta

Emily BurkeCancer, Drug Approvals, FDA, Genetics, Mechanism of Action, Monoclonal Antibodies, Small Molecule Drugs, The WEEKLY

 Pushing the Self-Destruct Button Breakthrough drug Venclexta charged onto the marketplace three months early to battle chronic lymphocytic leukemia. AbbVie’s (North Chicago, IL) and Roche’s (Basel, Switzerland) new therapy gained a quick approval after 80% of patients in the 106-person clinical trial responded to the small molecule inhibitor. Chronic lymphocytic leukemia (CLL) is the most common type of leukemia in adults, with approximately … Read More