Clinical Research

Frogs, dogs, whales, snails can all do it, but humans and primates can't. Regrow nerves after an injury, that is — while many animals have this ability, humans don't. But new research from the Salk Institute suggests that a small molecule may be able to convince damaged nerves to grow and effectively rewire circuits. Such a feat could eventually lead to therapies for the thousands of Americans with severe spinal cord injuries and paralysis.

"This research implies that we might be able to mimic neuronal repair processes that occur naturally in lower animals, which would be very exciting," says the study's senior author and Salk professor Kuo-Fen Lee. The results were published in PLOS Biology.


XBiotech has begun its European Phase III study using its novel cancer drug Xilonix™ for the treatment of colorectal cancer.

Xilonix is an antiobody works to block a number of processes that tumors use to grow and spread, such as potentially inhibiting the formation of tumor blood supply and new metastasis. Blocking tumor-related inflammation may also inhibit or reverse wasting and other illness associated with the malignancy. 

The double-blinded placebo controlled study is evaluating the use of the monoclonal antibody therapy designed to block chronic inflammation associated with malignant tumor growth. The treatment is reportedly aimed at reversing disease symptoms associated with disease progression and survival.
Results  presented at the 2014 World Transplant Congress evaluated the safety and efficacy of CSL Behring's C1 Inhibitor (C1-INH) concentrate in preventing antibody-mediated rejection following kidney transplants in highly sensitized patients.

C1-INH is a human protein and an important inhibitor of the complement system. Antibody-mediated rejection is a major cause of kidney transplant failure and a prime barrier to improving long-term outcomes for transplant patients. The types of antibody-mediated rejection vary in acuity and severity. Approximately 10 to 15 percent of kidney recipients experience rejection within one year after transplantation.

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.