Clinical Research

Investigators have announced discovery of a new molecule, the first of its kind, which allows for the multiplication of stem cells in a unit of cord blood. Umbilical cord blood contains adult stem cells used for transplants aimed at curing a number of blood-related diseases, including leukemia, myeloma and lymphoma.

For many patients this therapy comprises a treatment of last resort. 

Merck is discontinuing the clinical development program of its investigational MUC1 antigen-specific cancer immunotherapy tecemotide (L-BLP25) as a monotherapy in Stage III non-small cell lung cancer (NSCLC).

Tecemotide is an investigational MUC1 antigen-specific cancer immunotherapy that is designed to stimulate the body's immune system to identify and target cells expressing the cell-surface glycoprotein MUC1. MUC1 is expressed in many cancers, including NSCLC, and has multiple roles in tumor growth and survival. Tecemotide was being investigated in the Phase III START2, START and INSPIRE trials for the treatment of unresectable, locally advanced Stage III NSCLC.
The U.S. Food and Drug Administration has approved Keytruda (pembrolizumab), a new immunotherapy drug to treat advanced melanoma after it was tested on more than 600 patients who had melanoma that had spread throughout their bodies.

Because so many of the patients in the early testing showed significant long-lasting responses, the study was continued and the FDA granted the drug “breakthrough therapy” status, allowing it to be fast-tracked for approval. The largest Phase 1 study in the history of oncology, the research was conducted at UCLA and 11 other sites in the U.S., Europe and Australia.
In an oral presentation at the 2014 European Society of Cardiology (ESC) Congress in Barcelona, Dr. Jan Johansson, senior vice president of medical affairs at Resverlogix, disclosed that patients with cardiovascular disease (CVD) arising from atherosclerosis when given RVX-208 had a 55% (p=0.02) relative risk reduction (RRR) in Major Adverse Cardiovascular Events (MACE).
Positive results from its final preclinical toxicology study of SYN-004 have led Synthetic Biologics to get ready for clinical trials of the anti-infective, second-generation product candidate for Clostridium difficile (C. difficile).

Synthetic Biologics is in the final stages of preparing its SYN-004 IND application for submission to the FDA, with the expectation of initiating Phase Ia and Ib clinical trials in the fourth quarter of 2014, and a Phase II efficacy study in the first half of 2015.
In 2014, there will be an estimated 22,240 new cases of ovarian cancer in the United States and over 40,000 new cases in the European Union. Ovarian cancer is one of the most lethal gynecologic cancers. Women with recurrent or persistent ovarian cancer recur within 6-12 months of completion with a platinum-containing regimen and there remains a high unmet need for improved treatment options.
Enrollment in a randomized Phase II clinical trial of Pracinostat in combination with azacitidine in patients with previously untreated intermediate-2 or high-risk myelodysplastic syndrome (MDS) has been completed. The multi-center, placebo-controlled, double-blind study enrolled a total of 108 patients with a one-to-one randomization.

The Company plans to unblind the study approximately six months after the last patient was enrolled and report topline data in Q1 2015.

A newly developed antifungal named isavuconazole is as effective as voriconazole against invasive mold disease in cancer patients with less adverse effects, according to phase 3 clinical data.



A simple solution to a persistent problem. Credit: Ashok A. Kumar

By Ashok A. Kumar, Harvard University

Every year, 300,000 children are born with sickle-cell disease, primarily in Africa and India.

It is a genetic disorder that causes some blood cells to become abnormally shaped. The result is that those who suffer from it have a shorter lifespan.

 Reperfusion injury prevention isn't possible just yet. The administration of an experimental agent known as TRO40303 to patients who have had a heart attack, with the hope of preventing tissue damage when impaired blood flow is corrected (reperfusion), was disappointingly ineffective, according to results of a European study of patients with acute ST-elevation myocardial infarction (STEMI) presented today at ESC Congress 2014 and published in the European Heart Journal.