Clinical Research

Clinical trials are obviously important.  The results influence what drugs are approved by regulatory bodies, which can have a billion-dollar impact on companies.

Clinical trials have always been sponsored by industry - there has never been a meaningful  government-controlled research effort toward drug creation - but they have increasingly come under fire, due to claims that an industry sponsor can influence results and how they are reported to present their company and products in a better light.


New cells develop in the heart but how these cardiac cells are born and how frequently they are generated remains unclear. New research from Brigham and Women's Hospital used a novel method to identify these new heart cells and describe their origins - a Multi-isotope Imaging Mass Spectrometry (MIMS) imaging system that demonstrates cell division in the adult mammalian heart. 



Cardio3 BioSciences (C3BS) announced it has received authorization from the Belgian Federal Agency for Medicines and Health Products (FAMHP) to begin its Congestive Heart failure Cardiopoietic Regenerative Therapy (CHART-1) European Phase III trial for C3BS-CQR-1 in Belgium. This represents a world premiere for a regenerative medicine product targeting heart failure to be tested in the context of a Phase III trial. C3BS-CQR-1 is an autologous stem cell therapy for heart failure. 


Autologous stem cells from bone marrow 3 or 7 days following a heart attack did not improve heart function six months later, according to a new clinical trial. 

The results of this TIME (Transplantation In Myocardial Infarction Evaluation) trial were presented by Jay Traverse, MD of the Minneapolis Heart Institute Foundation Tuesday, today at the 2012 Scientific Sessions of the American Heart Association in Los Angeles.

The results of this trial mirror a previous, related study (LateTIME) which found that autologous bone marrow stem cell therapy given 2-3 weeks after a heart attack did not improve cardiac recovery. Both TIME and LateTIME were carried out by the Cardiovascular Cell Therapy Research Network (CCTRN).

Medivir AB has announced plans for a phase II proof-of-concept study of an all-oral regimen for the treatment of hepatitis C containing of Medivir/Janssen's protease inhibitor simeprevir and Vertex's nucleotide analogue hepatitis C virus (HCV) polymerase inhibitor VX-135. Janssen will conduct a drug-drug interaction study with simeprevir and VX-135 to support the planned initiation of a phase II proof-of-concept study in early 2013, pending discussions with regulatory authorities. 


Keryx Biopharmaceuticals, Inc. has announced the initiation of a Phase 2 study of Zerenex (ferric citrate), an ferric iron-based phosphate binder drug candidate, in managing serum phosphorus and iron deficiency in anemic patients with Stage 3 to 5 non-dialysis dependent chronic kidney disease ("NDD-CKD").

In the United States alone, over one and a half million people suffering from Stages 3 to 5 NDD-CKD have iron deficiency anemia, however, there are currently no oral iron supplements with an FDA label in NDD-CKD. Also, there are currently no FDA approved phosphate binders in NDD-CKD.


Glybera is the first gene therapy approved by regulatory authorities in the Western world. niQure announced it has received approval from the European Commission for the gene therapy Glybera(R) (alipogene tiparvovec), a treatment for patients with lipoprotein lipase deficiency (LPLD, also called familial hyperchylomicronemia) suffering from recurring acute pancreatitis.

Patients with LPLD, a very rare, inherited disease, are unable to metabolize the fat particles carried in their blood, which leads to inflammation of the pancreas (pancreatitis), an extremely serious, painful, and potentially lethal condition. The approval makes Glybera the first gene therapy approved by regulatory authorities in the Western world.