Clinical Research

An international team that developed a new gene therapy approach to treatment of Wiskott-Aldrich Syndrome, a fatal inherited form of immunodeficiency has reported results.

Wiskott-Aldrich Syndrome is a disorder that weakens the body's immune system. It is caused by a mutation in a gene that encodes the protein WASP. The most often used therapy is a bone marrow or stem cell transplant from a matching donor, often a sibling or relative. It can be curative for some patients, mostly those who have a strongly matching donor.

Veloxis Pharmaceuticals announced that LCP-Tacro successfully demonstrated non-inferiority compared to tacrolimus (Prograf®; Astellas Pharma) in its Phase III clinical trial, Study 3002.

The Phase III randomized, double-blind and double-dummy study in 543 de novo kidney transplant recipients, with Prograf as the comparator, met its primary efficacy and primary safety endpoints.

The study was conducted under a Special Protocol Agreement with the FDA and the results are considered pivotal for the planned U.S. regulatory filing expected to occur in the second half of 2013.
Resverlogix Corp. has announced that its Phase 2b ASSURE clinical trial evaluating RVX-208 in high-risk cardiovascular patients with low high-density lipoprotein (HDL) did not meet its primary endpoint of a -0.6% change in percent atheroma volume as determined by intravascular ultrasound (IVUS). The RVX-208 treated group had -0.4% plaque regression (p= 0.08).

Long-term cigarette smoking impacts morbidity and mortality, no question about that, but there may be a good reason to stop smoking in the weeks before surgery even if you don't intend to quit overall. 

In a review article, researchers from the University of California San Francisco and Yale University examined neurosurgical literature to characterize the impact of active smoking on neurosurgical outcomes. They found strong evidence for the association between smoking and perioperative complications throughout the surgical literature. 

When a liver from a deceased adult or adolescent donor is split into two separate portions for transplantation, with the smaller portion going to a young child and the larger to an adult, the child will benefit as much if they had received a whole organ from a donor close to their size, according to a paper in Liver Transplantation.      

As men get older they are more likely to suffer from andropausal syndrome (AS), also known as 'menopause' and by other complicated terms 'androgen deficiency in the aging male' or' late-onset hypogonadism'.

Men with andropausal syndrome have decreased levels of anabolic hormones, including testosterone, and it has been suggested that these hormone deficiencies are what cause the clinical symptoms.

Nearly 10,000 participants are in Istanbul at  the congress of the ERA-EDTA (European Renal Association - European Dialysis and Transplant Association) to share their knowledge and discuss the latest research findings. New pioneering studies have been presented: 


By SFP-iron administration, the ESA dose could be reduced by 35% while maintaining stable Hb levels. There were no SFP-related adverse effects and no cases of iron overload.


If you read THE WIRED WORLD IN 2013 Annual Trend Report you may have seen my article predicting we would make a big advancement on the road to growing one of the Big 5 organs from a person's own stem cells this year.  That will mean no more driver's license donor permission, no waiting lists and no immunosuppressive drugs.

A drug developed by Gilead Sciences and tested in an animal model at the Texas Biomedical Research Institute in San Antonio suppresses hepatitis B virus infection by stimulating the immune system and inducing loss of infected cells.  

Trigemina, a company focused on creating non-narcotic pain relief drugs, is enrolling patients in a Phase II clinical trial of inhaled oxytocin to treat chronic migraine. Trigemina's oxytocin product, known as T1-001 (no doubt to be renamed to something lyrical if it comes to market), has already shown promising results in preliminary studies, the company says.

This use makes perfect sense: Oxytocin is a general analgesic (pain reducer), and it also contributes to relaxation and healing.