New Hope for MS

Feb. 22, 2013

By Medical Discovery News

Pictures from the 2012 presidential campaign depict Ann Romney, wife of Republican candidate Mitt Romney, as a woman with bright eyes, a luminous smile, and a dancer’s posture. But beneath the polished business suit of a potential first lady another battle raged.

Ann Romney was diagnosed with multiple sclerosis (MS) many years ago and experienced a flare up of her symptoms that forced her to curtail her campaign efforts. There is no known cause or cure for MS, although medications are available to slow the progression of the disease. However, researchers at the National Institutes of Health discovered the drug daclizumab appears to tone down the autoimmune response in MS patients, providing hope for those like Ann Romney who are trying to overcome the obstacles of living with MS.

MS is a type of autoimmune disorder, meaning cells in the body’s own immune system that are supposed to provide protection from invading infections instead attack the body’s own healthy tissues. In the case of MS, the immune system attacks the myelin sheath that covers nerve cells. Myelin is crucial in the conduction of electrical impulses to and from the brain. The loss of myelin, called demyelination, causes hardened scars in areas of the nerves and brain affected. The name multiple sclerosis actually means “many scars.” MS is the most common disease of the central nervous system in young adults, affecting 400,000 Americans. 

In the NIH study, researchers identified a unique type of immune cell called lymphoid tissue inducer (LTi) cells, which promote the development of lymph nodes and similar tissues in a fetus. While it is unclear what LTi cells do in adults, they appear to play a role in the immunity in the gastrointestinal tract. This study implicates these cells may contribute to MS, although they have not previously been linked to any autoimmune disorder.

MS patients in the study receiving daclizumab had reduced levels of LTi cells and reduced signs of inflammation in the cerebrospinal fluid, which surrounds the brain and spinal column, when compared to a control group that didn’t receive the drug. This drug is an engineered antibody that interferes with the signals produced by a molecule called interleukin 2 (IL-2) that promotes inflammation. Antibodies are specialized proteins made by the immune system that target and bind to antigens, in this case the IL-2 protein, to eliminate or block their actions.

By blocking IL-2 action, it seems like daclizumab reduces inflammation and the damage that happens in MS. More studies will have to confirm the role of LTi cells in MS before the development of drugs to selectively target LTi cells can begin in earnest. But one day, such drugs may become part of the treatment for MS and hopefully slow the progression of this disease more effectively. 

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Lifesaving Venom

By Medical Discovery News

Sept. 1, 2012

Reversing Cerebral Palsy

People have evolved a fear of snakes out of necessity. One bite from a venomous snake can prove fatal, so it’s ironic that a number of remarkable drugs are derived not only from poisonous snakes, but other deadly creatures as well. Researchers increasingly prize this group of animals, believing their venom holds the promise of effective treatments for major disorders ranging from high blood pressure to heart disease to cancer.

Depending on the type of poisonous snake, a bite can cause a drop in platelets and lead to uncontrollable bleeding. Or it can, like the venom of the Brazilian pit viper, incapacitate victims by causing their blood pressure to plummet. But in smaller doses these same characteristics can reverse disease.

One group of drugs, called ACE inhibitors, brings down blood pressure by decreasing chemicals that tighten blood vessels so that blood flows more smoothly. Another drug, called tirofiban, comes from the saw-scaled viper, which has venom that thins the blood and causes victims to bleed out. As a drug, it’s an anticoagulant used to dissolve blood clots in people with a minor or impending heart attack.

Another venom with similar mechanisms comes from the Malayan pit viper, except it also possesses a protein that may dissolve clots for as long as six hours after stroke symptoms start. An international study is targeting this venom’s potential to expand the three-hour window required for current drugs to be effective. Allowing people more time to get to a hospital for evaluation and treatment makes a significant difference on their prognosis.

Other deadly creatures also have lifesaving potential. Scientists are studying the fatal Deathstalker scorpion native to North Africa and the Middle East. Yet, its venom contains Chlorotoxin, which just happens to attach to cancer cells by binding strongly to a cancer-specific protein called matrix metalloproteinase-2. By fluorescently labeling Chlorotoxin, surgeons can easily identify cancer tissues and remove them. Scientists also figured out that by radioactively labeling Chlorotoxin, the toxin targets tumor cells and the radiation kills them.

Another example is Cobratoxin, which Western pharmacists began experimenting with as early as the 1930s on patients with diseases such as multiple sclerosis (MS) and asthma. More recently, a modified form of Cobratoxin has been shown to block the development of induced MS in 90 percent of lab rats. Cobratoxin seems to stimulate a molecule called interleukin 27, which slows an overactive immune response that scientists believe may be causing the disease. A related toxin molecule called Cobrotoxin has been shown in studies to impede the spread of HIV by blocking the receptors the virus uses to infect cells.

While the field of venom study has grown, researchers are concerned that negative environmental impacts on some of the threatened venomous species will limit their work. Ultimately that reduces the number of potential life saving or life changing drugs that are possible.

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