Clinical Research

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…

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). The beneficial effect of RVX-208 on patients with diabetes mellitus was accentuated with a relative risk reduction in MACE that was lowered by 77% (p=0.01). These observed reductions in MACE may stem from the ability of RVX-…

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. C. difficile is classified by the CDC as an "urgent public health threat," given its high…

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. The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to the investigation of motolimod (VTX-2337) when administered in combination with pegylated liposomal doxorubicin (PLD) for…

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. The randomized Phase II study is designed to evaluate the safety and efficacy of Pracinostat compared to placebo when…

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. Invasive fungal infections are important causes of morbidity and death for patients with hematological malignancies. Many leukemia and lymphoma patients receive high-dose chemotherapy, sometimes followed by stem cell transplantation, compromising their immune systems. The genus Aspergillus comprises several hundred species of mold that are ubiquitous in the environment but pose little threat to people…

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. The disease can be managed if it is diagnosed early, which means it rarely kills in rich countries, such as the US. But in sub-Saharan Africa, where it is most prevalent, most children with the disease still die before their fifth birthday. The…

 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. Earlier studies that had generated high hopes for TRO40303. TRO40303 has been shown in animals and laboratory…

Oxidized lipids are known to play a key role in inflaming blood vessels and hardening arteries, which causes diseases like atherosclerosis. A new study at UCLA demonstrates that they may also contribute to pulmonary hypertension, a serious lung disease that narrows the small blood vessels in the lungs. Using a rodent model, the researchers showed that a peptide mimicking part of the main protein in high-density lipoprotein (HDL), the so-called "good" cholesterol, may help reduce the production of oxidized lipids in pulmonary hypertension. They also found that reducing the amount of oxidized…

Stiff Person Syndrome is a disease characterized by stiffness of the skeletal muscles, painful muscle spasms and, in severe cases, the disease can prevent movement and walking. It can be marked by a "tin soldier" gait. Autologous (from your own body) hematopoietic stem cell transplantation (auto-HSCT) has been used to successfully treat patients with autoimmune diseases such as multiple sclerosis and scleroderma, which are resistant to more conventional treatment. A regimen of high-dose chemotherapy and antilymphocyte antibodies rid the body of diseased immune cells (immunoablation)…