Clinical Research

Achalasia is a rare disease, affecting 1 in 100,000 people, characterized by a loss of nerve cells in the esophageal wall and manifested as chest pain during eating, weight loss, and regurgitation of food.

When we swallow, a sphincter in the lower esophagus opens, allowing food to enter the stomach. Nerve cells in the esophageal wall control the opening and closing of this sphincter, but in people with achalasia, these nerve cells gradually disappear. Without these cells, the esophageal sphincter fails to relax, causing food to accumulate in the esophagus. This results in the swallowing problems, regurgitation, vomiting, nighttime coughing, chest pain and weight loss. 


New York, NY - July 24, 2014 -- Keryx Biopharmaceuticals, Inc. has published results from the long-term, randomized, active control Phase 3 study of Zerenex (ferric citrate), their investigational oral ferric iron-based phosphate binder for the treatment of hyperphosphatemia in patients with end-stage renal disease (ESRD) on dialysis. The PERFECTED study (PhosphatE binding and iRon delivery with FErric CiTrate in EsrD) was published online today in the Journal of the American Society of Nephrology (JASN).


A study has documented the safety benefits of aortic stent grafts inserted during minimally invasive surgery to repair abdominal aortic aneurysms; weaknesses in the body's largest artery that can rupture, causing potentially lethal internal bleeding. 

The study shows that patients who received the minimally invasive aortic repair procedure had a 42 percent reduction in preventable post-operative complications and a 72 percent reduction in mortality, compared with those who had undergone open repair surgery.


Each year, roughly 250,000 people in the United States require hospital care for
Clostridium difficile (C. difficile) infection and at least 14,000 people die from it, according to the U.S. Centers for Disease Control and Prevention (CDC). As a result, the CDC identified C. difficile as an urgent public health threat in its 2013 report on antibiotic resistance.

The National Institute of Allergy and Infectious Diseases (NIAID) has launched an early-stage clinical trial of CRS3123, an investigational oral antibiotic intended to treat C. difficile infection.  CRS3123 (previously known as REP3123) is a narrow-spectrum agent that inhibits C. difficile growth while sparing normal intestinal bacteria.


Doctors at the University of Arizona Cancer Center at St. Joseph's Hospital and Medical Center in Phoenix report  in Lancet Oncology that a new treatment for ovarian cancer can improve response rates (increase the rate of tumor shrinkage) and prolong the time until cancers recur.  

Trebananib (formally known as AMG 386; Amgen) is a first-in-class peptide-Fc fusion protein (or peptibody) that targets angiogenesis (the growth of new blood vessels into cancerous tumors) by inhibiting the binding of both angiopoietin 1 and 2 to the Tie2 receptor.

This is a different mechanism of action than other agents that also effect angiogenesis by inhibiting vascular endothelial growth factor (VEGF) such as bevacizumab (Avastin; Genentech).  


Bellerophon Therapeutics has completed enrollment of its 80-patient Phase 2 clinical trial of INOpulse for the treatment of pulmonary arterial hypertension (PAH). PAH is a life-threatening, progressive disorder characterized by abnormal constriction of the arteries of the lung, leading to increased blood pressure in the lungs and abnormal strain on the heart's right ventricle, eventually leading to heart failure.  
Amgen announced that it will collaborate with the National Cancer Institute and other public and private sector partners on the Lung Master Protocol (Lung-MAP), a clinical trial program that will use biomarker-driven research and genomic profiling to match squamous cell lung cancer patients to investigational treatments based on their individual cancer profiles.

In humans, no therapy for  amyotrophic lateral sclerosis (ALS), or Lou Gehrig's disease,  has ever been discovered that could extend lifespan more than a few additional months.

ALS was first identified as a progressive and fatal neurodegenerative disease in the late 1800s but only gained international recognition in 1939 when it was diagnosed in American baseball legend Lou Gehrig. It's known to be caused by motor neurons in the spinal cord deteriorating and dying, and has been traced to mutations in copper, zinc superoxide dismutase, or SOD1. Ordinarily, superoxide dismutase is an antioxidant whose proper function is essential to life.


Individuals who are hospitalized for the skin conditions of Stevens-Johnson syndrome and toxic epidermal necrolysis appear to have a high risk of recurrence, according to a new study.

Stevens-Johnson syndrome (SJS) and toxic epidermal necrolysis (TEN) are life-threatening conditions that develop primarily as responses to drugs, and result in extensive epidermal detachment (upper layers of the skin detach from the lower layers). Recurrence has been reported in isolated cases, and the overall risk of recurrence has been unknown, according to background information in the article.

Wine was once okay for pregnant women in moderation, then all alcohol was bad, then wine was good again because of the miracle product du jour,  resveratrol , but now it may be bad again.

A new research says that when taken during pregnancy, resveratrol supplements led to developmental abnormalities in the fetal pancreas. Resveratrol has been touted for its human health benefits for years and is readily available over the counter.