Did You Know

Research Research New Possible Cause for Scientists Unexplained Miscarriages Discover New among Caucasians) in which mothers Immune Disease generate antibodies that attack and destroy in their fetuses and newborns. In severe cases, FNIT may lead to bleeding in the brains of the fetuses and newborns and cause neurological impairment or even death. Seventy-five percent to 95 percent of FNIT cases are caused by maternal antibodies to one specific antigen, HPA-1. However, these researchers discovered that another Researchers at the Laboratory of antigen, HPA-2, causes a type of FNIT Allergic Diseases at the U.S. National never described before that can lead Institute of Allergy and Infectious to miscarriages in more than 83 per - Diseases (NIAID) have identified a cent of mice. They also discovered new immune disease, which they that the HPA-2 antibodies sometimes have named PLCG2-associated anti - not only destroy platelets, but acti - body deficiency and immune dysreg - vate them and cause massive clotting ulation (PLAID). Symptoms of the in the placentas. Because only six to disorder include immune deficiency, Researchers at St. Michael’s Hospital eight reported live human births in inflammatory skin disorders and in Toronto, Ont., Canada, have identi - the world with FNIT caused by HPA-2 cold-induced hives (cold urticaria). fied a potential new cause for unex - have been reported, this research The team identified the genetic plained miscarriages in mice. In addi - suggests that the reason these cases mutation that causes PLAID in 27 tion, they have identified two possible are so rare is that most of the affected members of three unrelated families treatments to prevent these miscar - fetuses died through miscarriages, who suffered from cold urticaria. riages, which have broader implica - before doctors examined them. “This is one of the few examples in tions for the development of new drugs In mice, these miscarriages can be which the allergy symptom directed to treat heart attacks and strokes. prevented using at least two thera - us to a genetic syndrome,” says The researchers found that the pies. One is the transfusion of study leader Dr. Joshua Milner. “In same kind of blood clotting in coro - immune globulin (IVIG). The other is trying to understand the link nary arteries or blood vessels in the the transfusion of an antibody known between this group of conditions — brain that causes heart attacks and as anti-FcRn, which blocks the attack - autoimmunity, chronic infections strokes also happens in the placenta. ing maternal antibodies from crossing and cold urticaria — we not only The massive clotting can destroy the the placenta. identified a disease-causing muta - placenta, block blood flow to the The research was reported on in tion, but uncovered a unique and fetus and cause miscarriages. This the November issue of the Journal of fascinating genetic mechanism at condition is known as fetal and Clinical Investigation . It is hoped that the crux of allergy, immune defense neonatal immune the findings will be important in the and self-tolerance.” The research (FNIT), a bleeding disorder that development of safer anti-thrombotic was published online in the January affects between one in 800 and one drugs, which are under development 11 edition of the New England in 1,500 live births (most commonly by several companies. Journal of Medicine .

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Vaccines Vaccine May Halt Autoimmune Disease

A synthetic vaccine based on nan - have produced serious side effects. otechnology may halt autoimmune Rather than target the MMPs directly, diseases such as Crohn’s and rheuma - researchers at the Weizmann Institute toid arthritis. The vaccine works by in Rehovot, Israel, created tiny metal - tricking the immune system into lic vaccine molecules that fool the pro ducing antibodies that target an immune system into manufacturing enzyme that causes autoimmune its own MMP-suppressing antibodies. diseases. When tested on mice with a rodent With autoimmune diseases, some version of Crohn’s, the vaccine signif - members of the enzyme family, icantly reduced their symptoms. espe cially the enzyme matrix metal lo - Untreated mice suffered severe dam - proteinases (MMP), get out of control. age to their colons, while those injected MMPs are normally held in check with the vaccine experienced only nat urally by inhibitor molecules called “limited” inflammation. TIMPs, and previous attempts to More research is needed before for humans. The research was published mimic TIMPs with artificial drugs experts can be sure the therapy is safe in the journal Nature Medicine .

Healthcare U.S. Sets New Goals for a Healthier Nation In October, the U.S. Department of of Americans who see a dentist regu - Health and Human Services (HHS) larly to around 49 percent, from a released a list of critical health prior - current rate of about 44 percent. For ities for the coming decade designed the first time, the goals include a to serve as a blueprint to help reach sec tion identifying social factors that the Healthy People 2020 objective help determine health. For instance, of improving the health of all a major goal is to increase the per - Americans. The goals are designed centage of students who graduate to help policymakers at the federal, from high school with a regular diploma state and community level make in four years from around 75 percent priorities for the coming decade. to 82 percent, a move made in recog - According to HHS Assistant nition of the fact that higher educatio n Secretary for Health Howard Koh, increasing the percentage of people is closely linked to better health. MD, MPH, the top priorities are with high blood pressure and diabetes Koh, who presented the list of expanding access to medical care and whose conditions are adequately pri orities at the annual meeting of increasing the number of Americans controlled with medication; increas - the American Public Health Association, with their own primary care provider. ing the percentage of young teens noted that over the previous decade, Other goals include increasing the who receive booster doses of the the average life expectancy of percentage of eligible Americans tetanus-diphtheria-acellular pertussis Americans has increased from 77 who are screened for colorectal vaccine from 47 percent to 80 percent years to 78 years. And, three out of cance r from the current 54 percent and increasing the vaccination rate four health objectives identified by to 70 percent and the percentage of with two doses of the varicella vaccine health officials to be met by 2010 were eligible women who have mammo - in this age group from 37 percent to either met or substantial progress grams from 70 percent to 77 percent; 90 percent; and increasing the number was made toward meeting them.

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Research People with Job’s Syndrome Have Impaired Immune Memory The NIAID team examined patients severe chicken pox or have difficulty with Job’s syndrome to better under - clearing the initial infection. This means stand how immune memory develops. that Job’s syndrome is one of the few Job’s syndrome is a condition caused by diseases that predispose patients to a mutation in a gene for the protein developing shingles, but it does not STAT3, which aids in the development affect their response to chicken pox. and specialization of specific types of Based on these findings, the immune memory T cells. The team researchers concluded that measuring observed that, when compared with circulating central memory T cells, or healthy people, patients with Job’s STAT3 function, could be a way to iden - A research team at the National syn drome lack a major population of tify someone who is at greater risk for Institute of Allergy and Infectious circulating memory T cells, which are developing shingles or could benefit Diseases (NIAID) has found that thought to be a source for long-term T from the shingles vaccine. In addition, people with Job’s syndrome (also cell memory. These low numbers of new therapeutics that boost the activity known as autosomal-dominant hyper- cen tral memory T cells are closely asso - of STAT3 also could help protect people immunoglobulin E syndrome) have a ciated with these patients’ increased from VZV reactivation. Further study lower number of immune memory susceptibility to varicella-zoster virus is needed, they said, to determine if cells, which makes them more suscep - (VZV) reactivation, causing them to have young, otherwise healthy people who tible to viral reactivation. The findings, a significantly higher chance of develop - experience episodes of shingles have which appear in the Nov. 23, 2011, ing shingles at a young age (less than 50 impaired memory T cells. In addition, issue of Immunity , provide a potential years old) and of experiencing repeated more research also is needed to better treatment strategy for people with Job’s episodes of shingles compared with understand what level of immune syndrome, as well as offer clues about healthy people. What’s unique about memory is needed to protect people how immune cells in healthy people this finding is that people with Job’s who have received the chicken pox control chronic viral infections. syndrome do not typically experience vaccine from developing shingles.

Medicines Grifols Offers Gamunex People and CoPay Card to CIDP Patients Places CSL Behring has received a up to $2,500 per patient per 12- 2012 EURORDIS (European month period, and it is open to CIDP Organization for Rare Diseases) patients who are not using any state Award for its pioneering work or federally funded healthcare pro - in developing and manufacturing gram such as Medicare, Medicaid, therapies used to treat rare and Medicare Advantage and Tricare; who serious medical conditions. In The Gamunex CoPay Card Program are not residing or receiving treatment 2011, the company received the by Grifols is a coupon program that in the state of Massachusetts; and who National Organization for Rare helps patients with chronic inflamma - are not getting treatments in hospitals Disorders Corporate Award for tory demyelinating polyneuropathy or hospital-associated outpatient new treatments for rare diseases (CIDP) cover the copay costs for clin ics. For additional information, go brought to market in the U.S. Gamunex-C. The program provides to gamunexcard.com .

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Research Research Positive Results for Phase II DNA Sequence Clinical Trial for ITP Patients Causes Most Symphogen has presented final of patients responded at day seven. Severe Lupus Phase II data demonstrating that its The median time to respond was 59 recombinant polyclonal antibody drug hours, and the median duration of candidate, rozrolimupab, exhibited a response was 14 days. The most com - favorable safety profile and induced a mon adverse events, mostly mild to rapid increase in blood platelets in moderate, were headache (18 percent), patients with immune thrombocytopenia pyrexia (13 percent), chills (8 percent) purpura (ITP). The Phase II study was and fatigue (8 percent). Four serious an open-label, multicenter clinical trial adverse events considered related to Researchers at the Catholic evaluating the efficacy, safety and tol - the drug were reported: decreased University of Sacred Heart in Rome erability of rozrolimupab (SYM001) in hemoglobin, extravascular hemolysis/ found that a genetic accelerator adult RhD positive, nonsplenectomized dizziness and two cases of transient known as HS1.2 is responsible for the ITP patients. Results demonstrated that rise in D-dimer values with out clinical most severe cases of lupus. HS1.2 at 300 ug/kg, eight of 13 (62 percent) symptoms. leads to enhanced activation of the “transcription factor NF-KB” (a mole - cule that reads the genes to make Research them work), which in turn dramatical - IDF Seeks Women Participants ly increases the aggressiveness of the For New PIDD Survey inflammatory processes underlying the disease. The researchers likened of PIDD on family planning, concep - HS1.2 to the accelerator of a car tion, fertility and pregnancy. because it boosts the pathological Study results will be used for immune response typical of the dis - research purposes, and to better inform ease by enhancing the production of physicians, patients and the parents antibodies that attack the patient’s of patients impacted by PIDD. It is body instead of defending it. They hoped that the data obtained from found that 30 percent of lupus this survey will help improve patient patients have the HS1.2 enhancer in health outcomes and better inform their DNA. The discovery could lead to patients and parents of those affected more targeted and effective thera - by PIDD on the management of these pies against lupus. The results of diseases before, during and after a their study were published in the pregnancy. Annals of Rheumatic Diseases . The survey is intended for women over the age of 18 who have a PIDD, The Immune Deficiency Foundation or the mothers of patients with a Did You Know? (IDF) is seeking female participants PIDD. It is a web-based survey, and an In May, The Texas Department for a survey focusing on women’s invitation to participate will be sent of State Health Services added reproductive/pregnancy issues as to interested participants by email. If severe combined immunodeficiency they relate to primary immunodefi - an invitation is not received, the IDF (SCID) to the that ciency disease (PIDD). The survey is may not have an email to reach the all newborns in Texas are screened made possible by an unrestricted participant, so participants are asked for at birth. educational grant to study the impact to contact IDF at (800) 296-4433.

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Resource New Guide Explains the Medicaid Appeals Process

A new guide from the Kaiser to navigate. The guide describes Family Foundation’s Commission on Medicaid’s appeals system, including Medicaid and the Uninsured provides the fair-hearing process and the a comprehensive look at the appeals appeals process required for process for the Medicaid program, Medicaid managed care organiza - which differs significantly from those tions. As coverage expands under available through the Medicare health reform and efforts proceed to pro gram and private health insurance. integrate services for dual eligibles The Medicaid appeals process pro - who are enrolled in both Medicare vides redress for individual applicants and Medicaid, protections through and beneficiaries seeking eligibility the appeals process will be increas - for the program or coverage of pre - ingly important. The guide is avail - scribed services, but the process is able online at www.kff.org/medicaid/ multilayered and can be complex 8287.cfm .

Clinical Trials Update Baxter has begun a Phase I U.S. and Canada, for the treatment lupus . SBI-087 is made to hit a tar - prospective, open-label study that of mild to moderate Alzheimer’s get called CD20, which is currently will assess the safety, tolerability disease. This second Phase III trial is blocked by Genentech and Biogen and pharmacokinetics of its lead identical in design to the first Phase Idec’s rituximab (Rituxan) for investigational candidate, BAX 855, III trial, and it will assess the safety patients with a different form of a longer-acting (PEGylated) form of and effectiveness of Gammagard autoimmune disease, rheumatoid a full-length recombinant factor VIII Liquid as a potential treatment for arthritis. The Trubion drug, which (rFVIII) protein, in previously treated signs and symptoms associated is also being tested for rheumatoid patients aged 12 years or older with with Alzheimer’s disease. arthritis, is being developed in severe hemophilia A . When used partnership with Madison, N.J.- for prophylaxis, Baxter’s Advate Neovacs has announced the full based Wyeth. requires patients to infuse every results for its TNF-K-003 clinical two to three days to reduce the trial with TNF-Kinoid in rheuma - In March, CSL Behring enrolled occurrence of bleeding episodes. toid arthritis . According to the its first patient in the PATH study, This Phase I trial is the first step in company, the results confirm the an international clinical trial assessing whether BAX 855 can be very good safety profile of the TNF- designed to evaluate the efficacy, infused less frequently. Kinoid. No patient withdrew from safety and tolerability of two the study because of an adverse dif ferent doses of subcutaneous Baxter has begun a second event, and no Kinoid-related seri - immunoglobulin (SCIG), compared Phase III clinical trial of its ous adverse events were reported. with placebo, in maintenance Gammagard Liquid 10% Immune treatment of chronic inflammatory Globulin Infusion (Human), which Trubion Pharmaceuticals has demyelinating polyneuropathy is marketed as Kiovig outside the started a clinical trial of a drug for (CIDP) .

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