Clinical Research


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.


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 lipids improved the rodents' heart and lung function.




An investigational drug known as APT102 significantly reduced damage to heart muscle from a heart attack and minimized the risk of bleeding during follow-up treatments, according to an animal study based on a decade of work by APT Therapeutics, scientists at Washington University School of Medicine in St. Louis and colleagues at Cornell and Harvard.

Standard heart attack treatment often causes heart tissue damage. Once the blood clot that causes a heart attack is removed from an artery, molecules from dead and dying cells mix with blood rushing back through the artery. One of these molecules, adenosine triphosphate (ATP), is inflammatory; another, adenosine diphosphate (ADP), causes more clotting.

Amyotrophic lateral sclerosis (Lou Gehrig's disease) is in the popular media because celebrities are dumping buckets of ice on their heads to raise awareness. Researchers probably wish they would donate money to research rather than raising awareness and hoping someone else donates money rather than dumping water on their heads too, but all medical outreach is good medical outreach.

Researchers at Mayo Clinic and The Scripps Research Institute in Florida have done something a little more practical; they developed a new therapeutic strategy to combat the most common genetic risk factor for ALS and frontotemporal dementia


Generex Biotechnology Corporation has announced publication of a follow-up study from a Phase I clinical trial of the immunotherapeutic agent AE37 in patients with prostate cancer. The study demonstrates an association between a specific immune response generated by AE37 and improved overall survival.  

A prior study showed that AE37-immunized patients had better overall and disease-free survival as a group than would be expected from their disease status and the current study shows that patients with the strongest immunological response did the best. In particular, both the presence of AE37-induced T cells in peripheral blood as well as a robust delayed type hypersensitivity (DTH) response elicited by AE37 correlated significantly with overall survival.

A recent study conducted by Mayo Clinic researchers recommends laparoscopic cholecystectomies (surgical removal of the gallbladder) for pediatric patients suffering from gallstones and other gallbladder diseases. 

A cholecystectomy is a surgical procedure performed to remove the gallbladder, a pear-shaped organ located below the liver on the upper right side of the abdomen. The gallbladder is responsible for collecting and storing bile, which is a fluid secreted by the liver.

During a laparoscopic cholecystectomy, four incisions are made in the abdomen. Then, a small video camera and other special tools are used to remove the gallbladder.


Rheumatoid arthritis is a condition that causes painful inflammation of several joints in the body - the joint capsule becomes swollen, and the disease can also destroy cartilage and bone as it progresses. It affects 0.5% to 1% of the world's population and doctors have used various drugs to slow or stop the progression of the disease.

Eidgenössische Technische Hochschule Zürich (ETH Zurich)  researchers have developed a therapy that takes the treatment of rheumatoid arthritis in mice to a new level: after receiving the medication the animals have been fully cured.


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.