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Drug Stops Type 1 Diabetes in Its Tracks

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    Drug Stops Type 1 Diabetes in Its Tracks

    May 29, 2002 - -- In a dramatic finding that may someday improve treatment for all types of diabetes, a new high-tech drug is stopping type 1 diabetes in people newly diagnosed with the disease -- decreasing their need for insulin shots. Until now, no treatment has been able to stop the downhill progression of the disease.

    After taking the experimental drug for just two weeks, nine of 12 diabetics were able to decrease their need for insulin shots, and with very few side effects.

    "The most important implication of this research is that we can intervene in type I diabetes after onset and still have an impact," senior researcher Jeffrey A. Bluestone, PhD, director of the UCSF Diabetes Center, tells WebMD. "This opens up the door to a whole host of other approaches, not just this drug."

    Bluestone has filed a patent application for this new drug and has a commercial agreement regarding its use with Centocor and Johnson & Johnson Pharmaceuticals. Before it can be made available to the public, the drug will require many more years of study to confirm that it works as well as it seems and to be sure there are no hidden problems.

    In type 1 diabetes, the immune system, which normally protects against foreign invaders, destroys specialized pancreas cells, beta cells, which produce insulin. Over the years, high blood sugar from diabetes can lead to early heart attacks and stroke, as well as blindness and kidney failure.

    By targeting these specific immune cells, the new drug protects beta cells and preserves insulin production. The drug is an antibody that works against the destructive immune cells.

    This damaging process actually starts years before a person develops symptoms from high blood sugar -- as insulin levels slowly fall in the body. But this drug brings us one step closer to treating the disease before symptoms appear, if we can identify early on those who will later develop type 1 diabetes, Bluestone says.

    In the first human study, 12 people aged 7-27 with type 1 diabetes received daily injections of the new drug for two weeks. Patients began treatment within six weeks of being diagnosed with type 1 diabetes and reported only minor side effects.

    The study is featured in the May 30 issue of The New England Journal of Medicine.

    One year later, nine of the 12 patients were still producing the same amount of insulin. In fact, some were producing more insulin. This allowed them to decrease the amount of insulin shots they needed. In addition, blood sugar significantly improved.

    However, 10 of 12 similar patients who did not receive the drug had a dramatic drop in natural insulin production, and they required higher doses of insulin than the treated patients.

    No severe side effects were seen with the drug. The most common side effects were fever, rash, and anemia.

    In an editorial accompanying the study, Edwin A.M. Gale, MD, calls the findings "encouraging" but cautions that further studies are needed. He is professor of diabetic medicine at the University of Bristol, England.

    "The new treatment is very unlikely to avoid the need for insulin," Gale tells WebMD. "So why does it matter? Because it works in experimental animals even after the onset of diabetes, and practically nothing else does at this stage of the disease."

    "Treatment with [this] antibody preserved insulin production in patients with new onset type 1 diabetes," lead researcher Kevan C. Herold, MD, tells WebMD.

    "People with type 1 diabetes eventually lose their ability to make insulin entirely, and [those] who make some insulin have a much easier time controlling their disease than those who do not," he says. Herold is associate professor of clinical medicine at Columbia University College of Physicians & Surgeons.

    Although other drugs that suppress immune function have stopped diabetes in its early stages before, the tradeoff has been widespread shutdown of the immune system. This leaves people vulnerable to life-threatening infections and cancer. Herold says these drugs are too dangerous to use in children, especially since they would have to be taken forever.

    This new antibody is more of a silver bullet that specifically targets the immune cells causing diabetes without far-reaching effects on other body tissues. The researchers were able to accomplish this by combining certain parts of a human antibody with other parts of a mouse antibody.

    In the near future, a large study involving more than 80 patients will use multiple doses to enhance the effects of the drug, much as repeated vaccinations increase protection against viral diseases.

    An especially attractive feature of the new drug is that a single two-week treatment "seems able to re-educate the immune system permanently," Gale says. "If we get more conclusive evidence that it can produce lasting benefit after diagnosis, it might be feasible to use it before diagnosis."

    read read read...look at how diffrent they are in diabetes.
    no endless rat trials.


      Before it can be made available to the public, the drug will require many more years of study to confirm that it works as well as it seems and to be sure there are no hidden problems.

      Other treatments are slow too. I'm sure they did rat trials at some point.

      "It is not easy to find happiness in ourselves, and it is not possible
      to find it elsewhere."
      --Agnes Repplier, writer and historian
      Emily, C-8 sensory incomplete mom to a 8 year old and a preschooler. TEN! years post.


        no emi. they are doing human trials at same time trying
        to fully understand and improve drug. while sci is witholding
        until they fully understand and have a perfect silver bullet.
        doing this at our expense...not theirs.


          New Drug Shows Promise In Common And Lethal Form Of Leukemia

          A new drug blocks the impact of a cancer-causing gene mutation found in a common and lethal form of leukemia, say researchers at the Johns Hopkins Kimmel Cancer Center. Their findings in animal and test tube models are featured the June 1, 2002, issue of Blood.
          Clinical trials to test the safety and effectiveness of the drug, CEP-701, in adult patients who have relapsed or stopped responding to standard therapy, and who have the mutations are now under way. CEP-701 appears to cancel out the effect of mutations of the FLT3 gene, first isolated by the Kimmel Cancer Center team in 1992 and shown to be a primary culprit in an aggressive, treatment-resistant form of acute myeloid leukemia (AML). Approximately 40 percent of AML patients have FLT3 mutations, and most of them will not be cured using current therapies, according to the Hopkins experts.

          "Right now, AML patients with FLT3 mutations have a dismal diagnosis with little hope of cure. We hope to change that with this new drug," says Donald Small, M.D., Ph.D., associate professor of oncology at the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins and study director. "Since it selectively targets the genetic error, CEP-701 turns it from a negative indicator to a positive one. This is what molecular medicine is all about, finding the cellular mistakes that work against us to cause cancer and turning them to our advantage to kill the cells," says Small.

          The investigators tested CEP-701 in mouse cell lines and human AML cells with FLT3 mutations and found the drug interfered with the signal of the altered gene and led to leukemia cell death. Ultimately, the investigators believe cures will be obtained by combining the new drug with chemotherapy. However, they must first test the drug's safety and effectiveness alone before they can combine it with other anti-leukemia drugs, Small said.

          CEP-701 is one of a new class of drugs called tyrosine kinase inhibitors, so-called because of their ability to block specific cell signaling proteins. "Mutant FLT3 uses its tyrosine kinase portion to signal leukemia cells to grow and also to prevent them from dying," explains Mark Levis, M.D., Ph.D., assistant professor of oncology at the Hopkins Kimmel Cancer Center and the paper's first author. "By inhibiting the gene's ability to communicate with cells, we can slow the growth and promote the death of AML cells. In essence, we render the gene powerless. It's as if it never existed," he says.

          The investigators also have developed a test to identify FLT3 mutations in AML patients. Adult patients who have been diagnosed with AML may contact Doug Smith, M.D. at (410) 614-5068 or Small at (410) 614-0994 to arrange for the test and to learn if they are candidates for the trial.

          AML, which is characterized by uncontrolled growth of the myeloid cells in the blood and bone marrow, strikes more than 10,000 adults and children each year in the U.S. It is the most common form of adult leukemia and the second most common type of childhood leukemia.

          In addition to Small and Levis, other participants in the research include Jeffrey Allebach, Kam-Fai Tse, Rui Zheng, Brenda R. Baldwin, B. Douglas Smith, Susan Jones-Bolin, Bruce Ruggeri, and Craig Dionne.

          The study was funded by the National Cancer Institute, Leukemia and Lymphoma Society, and the Children's Cancer Foundation.

          Partial funding for this study was provided by Cephalon, Inc., and Small is a paid consultant to the company. The terms of this arrangement are being managed by The Johns Hopkins University in accordance with its conflict of interest policies.