Muscular Dystrophy Treatment

Nov 16, 2006:

In promising new research, stem cells worked remarkably well at easing symptoms of muscular dystrophy in dogs, an experiment that experts call a significant step toward treating people.

“It’s a great breakthrough for all of us working on stem cells for muscular dystrophy,” said researcher Johnny Huard of the University of Pittsburgh , who wasn’t involved in the work.

Sharon Hesterlee, vice president of translational research at the Muscular Dystrophy Association, called the result one of the most exciting she has seen in her eight years with the organization. Her group helped pay for the work. She stressed that it isn’t yet clear whether such a treatment would work in people but said she had “cautious optimism” about it.

Two dogs that were severely disabled by the disease were able to walk faster and even jump after the treatments.

The journal Nature published the study online. The study used stem cells taken from the affected dogs or other dogs, rather than from embryos. For human use, the idea of using such “adult” stem cells from humans would avoid the controversial method of destroying human embryos to obtain stem cells.

The Nature paper focuses on Duchenne muscular dystrophy, a muscle-wasting genetic disorder that occurs in about 1 in every 3,500 male births. It is the most severe and most common childhood form of muscular dystrophy and the best known. In theory, the stem-cell treatment might also help other muscle dystrophies or even age-related muscle wasting, Dr. Hesterlee said.

Children with the disorder have trouble walking as early as preschool, and almost all of them lose their ability to walk between ages 7 and 12. Typically, they die in their 20s because of weak heart and lung muscles. There is no known cure.

The dog study was done by Giulio Cossu, director of the stem-cell institute at the San Raffaele Scientific Institute in Milan , Italy , with colleagues there and elsewhere. He said he hopes to start a small experiment in children in the next year or two.

Diabetes Treatment, Risk and Prevention

Diabetes is the single most important metabolic disease which can affect nearly every organ system in the body. It has been projected that 300 million individuals worldwide would be affected with diabetes by the year 2025. The reasons for this escalation are due to changes in lifestyle, people living longer than before and low birth weight could lead to diabetes during adulthood. Diabetes related complications are coronary artery disease, peripheral vascular disease, neuropathy, retinopathy, nephropathy, etc. People with diabetes are 25 times more likely to develop blindness, 17 times more likely to develop kidney disease, 30-40 times more likely to undergo amputation, two to four times more likely to develop myocardial infarction and twice as likely to suffer a stroke than non-diabetics.

Main key to handle Diabetes is to change your lifestyle. Diets, regular physical activity and weight reduction will help you to prevent diabetes.

Following sites will give you more information about Diabetes and how to handle it.

Developments to watch:

  1. One of the burdens diabetes patients face is the constant need to monitor their blood sugar by pricking their fingers. There will be a painless alternative in few years.  Oculir, San Diego, CA, USA based company is developing a monitor that reads blood sugar levels by inspecting the tiny blood vessels of the eye, without ever touching the eyeball! The monitor works by bouncing a harmless beam of infrared light off the white of the eye. Although invisible, the spectrum of light in the probe is just the right wavelength to interact with glucose molecules in blood flowing through tiny vessels in the thin membrane covering the eye. According to this company, the light reflected back is proportional to the amount of glucose in the blood. If everything goes well, commercial version of this device could go on sale in 2009.
  2. Medtronic, Minneapolis, US, created a system called Paradigm. This includes a monitor taped to a patient’s abdomen that continuously reads his or her blood glucose and transmits the data to a pump, which beeps or vibrates when blood sugar drops to a dangerous level. Patients adjust the pump, worn like a pager, to administer insulin into a port in the body. The system eliminates the need for repeated needle sticks to test blood and to inject insulin.  This is a good development in the battle of diabetes. FDA approved this system.
  3. For the first time, researchers are closing in on ways to prevent or at least limit  the devastating effects of type 1 diabetes. By using new treatments, including drugs normally  given to organ-transplant patients, doctors hope to stem the progress of the disease. If proven  effective, the new therapies could offer one of the first major advances in treatment since injectable insulin was first made available in 1922. In one clinical trial conducted by an international  network of researchers known as Type 1 Diabetes Trialnet, patients diagnosed with diabetes within past three months are given two immunosuppressant drugs: Mycophenolate mofetil (MMF) and Daclizumab  (DZB) from Roche AG. The hope is that the drugs will prevent diabetics’ immune systems from attacking  and killing insulin producing beta cells in the pancreas, just as they prevent a transplant patient from rejecting a new heart or kidney. For more information on this trial and similar trials visit   www.newonsetdiabetes.org and www.diabetestrialnet.org.
  4. Researchers for years have been looking for a substitute for insulin shots to control diabetes, and several leading pharmaceutical companies are in the process of developing their own inhalable alternative. Pfizer Inc. of New York, USA and Nektar Therapeutics, of San Carlos, Calif., USA jointly developed a powdered, inhalable insulin designed to replace shots for the treatment of diabetes. This powdered insulin comes with a specialized inhaler that can disperse the powder effectively inside the lungs. No more needles and pain. This product, known as Exubera, was approved in January 2006 by the U.S. Food and Drug Administration and by the European Commission. Exubera is currently available in Germany, Ireland, U.K and U.S.
  5. There also appears to be a link between gum infections and diabetes, as people with diabetes are more likely to have periodontal disease, according to U.S. Centers for Disease Control and Prevention. Researchers are now looking into whether there’s a two-way connection between the conditions to see if diabetes can be better controlled through treatment of gum disease.
  6. On October 17, 2006 FDA approved Merck & Co.’s drug Januvia to treat Type 2 diabetes. Januvia is the first in a new class of diabetes medicine known as DPP-4 inhibitors. The drug works by enhancing the body’s own ability to lower blood sugar, or glucose, when it is elevated. Swiss-based Novartis AG has a similar drug, Galvus, which is awaiting FDA approval. DPP-4 inhibitors work in a different manner than blood-glucose lowering drugs currently on the market, including sulfonylurea drugs, which stimulate the pancreas to release insulin, and metformin, which works on the liver to reduce blood sugar.
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