Exploring Different Strategies to Fight Alzheimer’s

Emily BurkeAlzheimer's Disease, Drug Development, Drug Discovery, Drug Targets

TAKE THAT, ALZHEIMER’S Alzheimer’s pernicious amyloid-beta plaques and tau tangles, discussed last week, remain important targets for the biotech industry. In the past few years, however, companies have begun to search more broadly for new treatments. This Weekly looks at products in development that use different strategies to fight this heartbreaking illness. REVIVING THE BRAIN? Loss of neurons is Alzheimer’s … Read More

From Drug Development to Approval: A Recap

Emily BurkeClinical Trials, Drug Approvals, Drug Development, Drug Discovery, Drug Targets, FDA

From The Lab To The Patient In this issue of the Biotech Primer WEEKLY we will recap the past seven issues that highlight the journey a molecule takes from the lab to the patient. Beginning in the 1980’s, scientists took a new tack in developing drugs. They adopted an approach known as rational drug discovery. Using this methodology, researchers first … Read More

From Drug Development To Approval: Phase IV

Emily BurkeClinical Trials, Cocktail Fodder, Drug Approvals, Drug Development, FDA, Orphan Drugs

Pharma Finish Line: FDA Approval Last week, we focused on the final stage of clinical testing, Phase III trials, where drug developers assess the safety and efficacy of their drug in large patient groups. At the end of Phase III, drug developers face the moment of truth: does the study data support claims that the new drug is both safe … Read More

From Drug Development To Approval: Phase III

Emily BurkeAuthor Emily Burke PhD, Drug Approvals, Drug Development, FDA, The WEEKLY

Phase III Is No Guarantee Our last Biotech Primer WEEKLY explored the riskiest part of the human clinical trials pathway: Phase II. About 70% of drugs that enter Phase II never make it out. Most often, it’s because they fail to demonstrate effectiveness. Even making it to Phase III is no guarantee of success – about 40% of drugs fizzle … Read More

Drug Discovery 201

Emily BurkeDrug Development, Drug Discovery, Drug Targets

WE WANNA NEW DRUG “One that won’t make me sick/ One that won’t make me crash my car/ and make me feel three feet thick…” Huey Lewis is singing about love, but he voices very human concerns when it comes to the medicines that heal bodies and minds. Last time, the Weekly explored how researchers identify drug targets—the molecules in … Read More

Drug Discovery 101

Emily BurkeDrug Development, Drug Discovery, Drug Targets

On the Road to New Medicines For most of the 20th century, we discovered new drugs by trial and error. Scientists investigated countless unrelated compounds in animals to see which improved disease symptoms. For instance, in the 1950s and 60s, British scientists at Boots Laboratories tested hundreds of unrelated chemicals on guinea pigs searching for an alternative to aspirin for … Read More

The Science Behind Opioid Addiction

Emily BurkeBusiness of Biotech, Cocktail Fodder, Drug Development, Drug Targets, Mechanism of Action, The WEEKLY

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

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

Vaccines: Schooling The Herd

Emily BurkeBiologics, Biotech Basics, Clinical Trials, Drug Development, Mechanism of Action, The WEEKLY

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 Microbiome Magnified

Emily BurkeBiologics, Biotech Basics, Drug Development, Drug Targets

DECODING THE GUT-BRAIN AXIS There is no shortage of microbiome-focused startups in biotech right now. The link between the gut microbiome — the entire collection of microbes living in the gut — and diseases such as inflammatory bowel disease are well-established. New research has made it clear, however, that the gut microbiome also impacts neurological health, leading to the phrase “the gut-brain … Read More

The Science Of CRISPR/Cas9

Emily BurkeBiologics, Business of Biotech, Cancer, Clinical Trials, Deoxyribonucleic Acid (DNA), Drug Development, Drug Targets, Genetics, Genomics, Mechanism of Action, Ribonucleic Acid (RNA)

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

A Skin Cell With Stem Cell Diversity?

Emily BurkeBiologics, Clinical Trials, Drug Development, Drug Targets, Term of the Week, The WEEKLY

INDUCED PLURIPOTENT STEM CELLS SHOW PROMISE Imagine being able to reprogram one of your own skin cells to produce a functioning nerve cell or section of cardiac tissue. This may sound like science fiction — but the groundwork for this to become a reality is already in the works as researchers expand their ability to create and manipulate induced pluripotent stem … Read More

Epigenome: Writing, Reading & Erasing

Emily BurkeCancer, Clinical Trials, Deoxyribonucleic Acid (DNA), Drug Development, Drug Targets, Genomics, Mechanism of Action, Small Molecule Drugs

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

Emily BurkeBiologics, Biotech Basics, Cancer, Clinical Trials, Drug Development, Drug Targets, Mechanism of Action, Monoclonal Antibodies, Orphan Disease, Orphan Drugs

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

Viruses Blasting Cancer

Emily BurkeBiologics, Clinical Trials, Deoxyribonucleic Acid (DNA), Drug Development, Drug Targets, Mechanism of Action, Ribonucleic Acid (RNA)

Engineering Viruses To Attack Getting bacteria-eating viruses to combat antibiotic resistance isn’t the only way viruses are being hacked to defend team homo sapien. This week, we’ll turn our attention to another benevolent use of viruses: cancer-fighters known as oncolytic viruses. Oncolytic Virus Primer Oncolytic viruses are an immunotherapy — a type of therapy that harnesses the power of a patient’s immune system to combat a disease. … Read More