National Institute of Diabetes and Digestive and Kidney Diseases

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National Institute of Diabetes and Digestive and Kidney Diseases

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News Article | May 3, 2017
Site: www.medicalnewstoday.com

The study - by researchers from the University of California-San Diego (UCSD) and colleagues from Human Longevity, Inc. in San Diego and the J. Craig Venter Institute in La Jolla, both in California - is published in the journal Cell Metabolism. Nonalcoholic fatty liver disease (NAFLD) is a condition characterized by a buildup of fat in the liver. According to the National Institute of Diabetes and Digestive and Kidney Diseases, it is "one of the most common causes of liver disease in the U.S." NAFLD is a different condition to alcoholic liver disease, in which the fat buildup is due to heavy alcohol use. In the new study - which involved 135 participants and establishes "proof of concept" - the researchers found that the stool-based test was able to predict advanced NAFLD with an accuracy of between 88 and 94 percent. First author Rohit Loomba, a professor of medicine and director of the NAFLD Research Center at UCSD, says that determining who has or is at risk for NAFLD is a "critical unmet medical need." Although there are dozens of new drugs in the pipeline, if it were possible to better diagnose the disease, then patients could be better selected for trials and "ultimately [we] will be better equipped to prevent and treat it," Prof. Loomba adds. There are two forms of NAFLD: simple fatty liver and nonalcoholic steatohepatitis (NASH). Simple fatty liver is a form of NAFLD in which there is fat in the liver but without inflammation or cell damage. This form does not usually lead to liver damage or complications. NASH is type of NAFLD where, in addition to fat buildup, the liver also shows signs of inflammation and liver cell damage. The inflammation can lead to scarring or fibrosis, and then to more severe cirrhosis, which alters the liver's fundamental biology. NASH can also progress to liver cancer. Nobody knows exactly what causes NAFLD, or why some of the people affected have simple fatty liver while others have NASH. Estimates suggest that around 20 percent of people with NAFLD have NASH. In the U.S., between 30 and 40 percent of adults are thought to have NAFLD, and approximately 3 to 12 percent have NASH. Being obese - and having conditions related to obesity, such as type 2 diabetes - raises the risk of developing NAFLD. Prof. Loomba and colleagues note that NAFLD is thought to affect up to 50 percent of obese people. In their study report, the researchers note how studies have shown that the makeup of a person's gut microbiome - the trillions of microbes that live in the gut together with their genetic material - may affect their risk for obesity. This set them wondering if there might also be a link between obesity-related liver disease and the gut microbiome. If this is true, then it may be possible to analyze the makeup of the gut microbiome from a person's stool sample and link that to their NAFLD status. To test this theory, the team first examined 86 patients with NAFLD diagnosed by biopsy - including 72 with mild or moderate disease and 14 with advanced disease. They sequenced the genes from the participants' stool samples - analyzing the presence, location, and relative abundance of various microbe species. This process identified 37 species of bacteria that differentiated advanced NAFLD from the mild or moderate stage with 93.6 percent accuracy. The researchers then validated the finding in a second group of 16 patients with advanced NAFLD and 33 healthy volunteers who acted as controls. This time, they found that by testing the relative abundance of nine species of bacteria - seven of which were in the 37 identified previously - they could differentiate the NAFLD patients from the controls with 88 percent accuracy. The researchers are keen to point out that so far, the test has only been trialed on a small number of patients in a highly specialized setting. Even if further studies validate it, a stool sample test for NAFLD is unlikely to be available for clinical use for at least 5 years. Learn how a protein discovery may offer a new treatment target for NAFLD.


Jacksonville, FL- Only 21 percent of adolescents with type 1 diabetes maintain the recommended A1C levels, According to the National Institute of Diabetes and Digestive and Kidney Diseases, often related to psychological and behavioral impediments. Researchers from Nemours Children's Health System have been awarded $1.8 million from the National Institutes of Health to develop and test a new Transdisciplinary Care Model, where an advanced practice nurse, psychologist, and dietitian will work together with the patient either in-person or through virtual, telemedicine visits to improve family management of diabetes. The principal investigator of the three-year project is Tim Wysocki, PhD, Co-Director of the Center for Health Care Delivery Science at Nemours in Jacksonville, FL. Endocrinologists Dr. Anthony Gannon from the Nemours/Alfred I. duPont Hospital for Children in Wilmington, DE, and Dr. Matthew Benson from Nemours Children's Hospital in Orlando, FL, will recruit patient participants for the trial. "The shortage of endocrinologists has been emphasized by the increase of diabetes, specifically type 1, among our youth," said Dr. Wysocki. "With the supply of new endocrinologists being outstripped, it won't be possible for diabetes patients to be seen by endocrinologists at every visit." The study, titled Transdisciplinary Versus Usual Care for Type 1 Diabetes in Adolescence, will focus on addressing adolescent struggles with management of the disease. The model will allow for healthcare force multiplication, while also addressing psychosocial barriers to self-care more effectively. There will be 150 patients participating in the trial at Nemours practices in the Delaware Valley and Florida. The Randomized Controlled Trial will compare effects of Usual Care with those of Trans-Disciplinary Care delivered in face-to-face clinic visits or via Telehealth on glycemic control, treatment adherence, healthcare use, T1D-related distress, quality of life, and treatment satisfaction. At the conclusion of the trial, substantial information will be available that could justify and inform a definitive future test of this model. It could also determine if Telehealth or face-to-face delivery of Trans-Disciplinary Care would be better justified for evaluation in a future trial. "Our team professionals managing this trial in Delaware and Florida were carefully selected due to their exceptional experience on this subject," said Wysocki. "We are looking forward to getting started." The study will be done in collaboration with Co-Investigators, Drs. Jessica Pierce and Matthew Benson (Nemours Children's Hospital in Orlando, FL), Drs. Jennifer Shroff Pendley, Julia Price and Anthony Gannon (Nemours/Alfred I. duPont Hospital for Children in Wilmington, DE), and Biostatistician, Dr. Andre Williams (Nemours Children's Specialty Care in Jacksonville, FL).


News Article | May 23, 2017
Site: www.npr.org

This story was updated on May 24 to clarify include new information on proposed cuts to Medicaid. The proposed budget unveiled Tuesday by the Trump administration doubles down on major cuts to biomedical research; programs to fight infectious disease outbreaks; health care for the poor, elderly and disabled; and prevention of HIV/AIDS. It restates the goals of the "skinny budget" the administration released in March, which was widely condemned by scientists and public health advocates. Mick Mulvaney, director of the Office of Management and Budget, said Monday that the goal is to cut back on public assistance and instead put people back to work. "We are going to measure compassion and success by the number of people we help get off of those programs and get back in charge of their own lives." No one thinks the president's budget will pass as proposed, since Congress has budget and spending authority. But it does provide a baseline from which negotiations may begin. "The president is right to take a close look at spending," says Sen. Chuck Grassley, R-Iowa. But "Congress has the power of the purse strings. I've never seen a president's budget proposal not revised substantially." Here's a rundown of the budget's medical research and health care proposals. Medicaid: The budget proposes cutting Medicaid and CHIP, the Children's Health Insurance Program, by $616 billion over 10 years, with almost half the savings occurring in the last two years. But it appears that the $616 billion number doesn't include the roughly $800 billion that would be cut from Medicaid funding by the House-passed version of the American Health Care Act. Mulvaney told the House Budget Committee Wednesday that there is some overlap in the two numbers, but the exact amount of the overlap remains a mystery. The joint federal-state programs provide health care and support services for 75 million low-income, elderly and disabled people, about half of whom are children. In 2015, federal and state spending on Medicaid was about $545 billion. The budget mirrors the changes in Medicaid included in the health care overhaul bill passed by the House earlier this month. Rather than the federal government matching state spending based on beneficiaries' health care needs, it would give states a fixed amount of money per enrollee or, alternatively, offer states a fixed block grant. That would cut the program's growth over time and reduce services because health care costs grow faster than the broad economy. Medicaid benefits for the elderly and disabled: Medicaid pays for services — including personal care, shopping or cooking for the elderly, and occupational therapy and work support for the disabled — that allow people to continue to live on their own. Under the law, those services are considered optional. But Medicaid is required to pay for nursing home and institutional care. "We'll see a return to more people with disabilities and more older adults not having access to services that allow them to remain at home," says Barbara Beckert, director of the Milwaukee office of Disability Rights Wisconsin. "Instead, we may see people forced into institutions, forced into nursing homes." Refugee benefits: The proposed budget makes the argument that the U.S. should reduce the number of refugees it brings into this country because those fleeing persecution in their home countries often end up using public assistance, including 50 percent who were on Medicaid in 2015. "The larger the number the United States admits for domestic resettlement, the fewer people the United States is able to help overall," the budget document says. National Institutes of Health: The NIH, which funds research into medical treatments and basic science, would see cuts of almost $6 billion, to about $26 billion. That would include a $575 million cut to the National Heart, Lung and Blood Institute and $838 million cut to the National Institute of Allergy and Infectious Diseases, which is involved in a wide range of diseases including AIDS and Zika. The National Institute of Diabetes and Digestive and Kidney Diseases would be cut by $355 million. The proposed cuts drew immediate and harsh criticism. The cuts would "cripple our nation's scientific efforts, undermining our economic growth, public health and national security," Mary Sue Coleman, president of the Association of American Universities, said in a statement. The cuts could "hobble our ability to provide tomorrow's cures and technologies." Centers for Disease Control and Prevention: The administration proposes trimming the CDC, which helps states and other countries fight infectious disease outbreaks, by $1.3 billion — 17 percent. That could include a $186 million cut in programs at the CDC's center on HIV/AIDS, hepatitis and other sexually transmitted diseases. The CDC's chronic disease prevention programs, such as those for diabetes, heart disease, stroke and obesity, would be cut by $222 million. The proposed cut to CDC "would be perilous for the health of the American people," says John Auerbach, president and CEO of the Trust for America's Health. "From Ebola to Zika to opioid misuse to diabetes to heart disease, the CDC is on the front lines keeping Americans healthy." Food and Drug Administration: A 31 percent proposed cut, from $2.7 billion to $1.89 billion, would be offset by $1.3 billion in proposed increased fees to be paid by drugmakers and device-makers. The budget shows a basic misunderstanding of how these agencies function, says Ryan Hohman, vice president of public affairs at the group Friends of Cancer Research. "To further suggest that private sector industry make up for such a significant cut to the FDA as proposed by the president shows a lack of knowledge for how user fees can be used and the scope of the FDA's pivotal role in assuring the safety of the daily lives of Americans." The budget doesn't explicitly address high drug costs, though Trump has frequently inveighed against drug prices, telling Congress in February that it should "work to bring down the artificially high price of drugs and bring them down immediately." Planned Parenthood: The family-planning organization has been the target of efforts to cut funding for years because it provides about one-third of the nation's abortions. This budget would be the first to bar a specific provider, according to Planned Parenthood. And it would bar the organization not only from Medicaid funding but also from any other Health and Human Services program, including the Title X family planning program, maternal and child health, STD testing and treatment, and Zika prevention. "From Day 1, President Trump has worked to keep his pro-life promises, including stopping taxpayers from being forced to fund abortion and abortion businesses," says Marjorie Dannenfelser, president of the anti-abortion group Susan B. Anthony List. "Taxpayers should not have to prop up Planned Parenthood's failing, abortion-centered business model." Planned Parenthood officials said Tuesday that many of their clients don't have other places to get health care. "We've already seen the results of these sorts of policies in Texas, so we know what would happen," says Kevin Griffis, vice president at Planned Parenthood Federation of America. "The heartbreaking truth is that if this budget were enacted, the results would be catastrophic for countless women and their families — cancers and diseases going undetected, higher maternal mortality and more unintended pregnancies."


News Article | May 29, 2017
Site: www.businesswire.com

DUBLIN--(BUSINESS WIRE)--Research and Markets has announced the addition of the "North America Dairy Alternatives Market (2016-2022)" report to their offering. There is a huge count of vegetarian population in the food industry, which is becoming one of the driving factors for the growth of dairy alternative market. According to the Vegetarianism in America study published recently, around 3.2 percent of U.S. adults, or 7.3 million people, follow a vegetarian-based diet. On the other hand, around 542,000 people in Britain follow vegan diet. Globally, 12% of the total population has decided to avoid non-vegetarian food in their meal. Hence, increasing vegan population would ultimately grow the demand for dairy alternative product. In recent years, growing number of consumers have stopped using dairy products due to various reasons such as dairy allergy, intolerance, and simply because it is trending. According to National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) estimation, 30 to 50 million Americans are lactose intolerant. Many people also feel symptoms such as diarrhea, nausea, stomach cramps, gas etc. with the usage of dairy products. Doctors also suggest that consumers who are allergic to milk must include plant based products in their diet. The key players operating dairy alternate market are as follows: For more information about this report visit http://www.researchandmarkets.com/research/23nxmn/north_america


News Article | May 9, 2017
Site: globenewswire.com

Support Potential of ADX71441 to treat Irritable Bowel  Syndrome, Interstitial Cystitis and Painful Bladder Syndrome Geneva, Switzerland, 9 May 2017 - Addex Therapeutics (SIX: ADXN) announced today positive results from multiple preclinical studies of ADX71441, a positive allosteric modulator (PAM) of the gamma-aminobutyric acid subtype B (GABAB) receptor, in models of visceral hyperalgesia. The studies were led by Prof. Jyoti N. Sengupta at the Medical College of Wisconsin, with the support of a grant from the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), a division of the US National Institute of Health. The findings strongly support the potential of ADX71441 in treating hyperalgesia in colonic inflammation (colitis) and bladder inflammation (cystitis). Chronic visceral pain syndromes related to the gastrointestinal or urinary tract represent an important unmet medical need as they can significantly impact the patient's quality of life. "These data represent an important step forward in the understanding of the role played by GABAB receptors in visceral pain and the potential of ADX71441 in large unmet medical needs, such as irritable bowel syndrome, interstitial cystitis and painful bladder syndrome," said Sonia Poli, CSO of Addex. "We look forward to continuing our collaboration with Prof Sengupta's group, and further evaluating the possibility of conducting clinical trials for ADX71441 in these compelling indications." The effect of ADX71441 was studied in reliable, reproducible and quantifiable preclinical models of visceral pain, which included behavioral measurements and electrophysiology recordings(1). Visceral motor reflex after colorectal distension (CRD) and urinary bladder distension (UBD) was significantly decreased (p<0.05) following systemic administration of ADX71441 (5, 10 and 50 mg/kg ip). The visceral analgesic effect of ADX71441 did not affect the normal function of the bladder as assessed by cystometry. In electrophysiology experiments, ADX71441 demonstrated significant inhibition of the response of UBD responsive lumbo-sacral (LS) spinal dorsal horn neurons to graded bladder distension. The effect was observed in spinal intact rats, but not in cervical (C1-C2) spinal transected rats. It also produced a moderate decrease in the spontaneous firing of these neurons in spinal intact rats. ADX71441 had no effect on the mechano-transduction properties or the spontaneous firing of UBD-sensitive pelvic nerve afferent (PNA) fibers. The current behavioral experiments to assess pain and the electrophysiology results indicate that ADX71441 produces visceral analgesia in animal models of cystitis and colitis.  The results indicate that ADX71441 produces this analgesic effect primarily by acting at the supra-spinal sites without affecting bladder motility. "We thank the NIDDK for their support and Prof. Sengupta and his team for their dedication to this important research with ADX71441," commented Tim Dyer, CEO of Addex. "These compelling results are a further example of how we are leveraging our collaborations with leading academic institutions to better understand the potential of the promising candidates in our pipeline." About GABA B receptor The GABAB receptor is clinically & commercially validated. Generic GABAB receptor agonist, baclofen, is marketed for spasticity and some spinal cord injuries, and French health authorities have approved its use for the treatment of alcoholism. Baclofen is also used off label for a number of other indications including overactive bladder (OAB), but its utility is limited due to variety of side effects and rapid clearance, requiring frequent administration of higher doses. About ADX71441 ADX71441 is a potent selective positive allosteric modulator (PAM) which potentiates GABA responses at the GABAB receptor. ADX71441 is a novel, first-in-class, oral, small molecule that demonstrated excellent preclinical efficacy and tolerability in rodent models of pain, anxiety, OAB, alcohol use disorders, nicotine dependence and in a non-human primate model of cocaine use disorder. ADX71441 has also proven efficacy in a genetic model of Charcot-Marie-Tooth Type 1A disease (CMT1A). ADX71441 has a different molecular mechanism from the generic drug baclofen in that it is a positive allosteric modulator, rather than an orthosteric agonist at the GABAB receptor, with a longer half-life, suitable for once daily administration. ADX71441 only acts when the natural ligand (GABA) activates the receptor, therefore respecting the physiological cycle of activation. It has been proposed that PAMs produce less adverse effects and lead to less tolerance than direct agonists. Addex Therapeutics (www.addextherapeutics.com) is a biopharmaceutical company focused on the development of novel, orally available, small molecule allosteric modulators for neurological disorders. Allosteric modulators are an emerging class of small molecule drugs which have the potential to be more specific and confer significant therapeutic advantages over conventional "orthosteric" small molecule or biological drugs. Addex's allosteric modulator drug discovery platform targets receptors and other proteins that are recognized as essential for therapeutic intervention - the Addex pipeline was generated from this pioneering allosteric modulator drug discovery platform. Addex's lead drug candidate, dipraglurant (mGluR5 negative allosteric modulator or NAM) has successfully completed a Phase 2a POC in Parkinson's disease levodopa-induced dyskinesia (PD-LID), and is being prepared to enter registration trials for PD-LID with support from the Michael J. Fox Foundation for Parkinson's Research (MJFF). In parallel, dipraglurant's therapeutic use in dystonia is being investigated with support from the Dystonia Medical Research Foundation (DMRF). Addex's second clinical program, ADX71149 (mGluR2 positive allosteric modulator or PAM) is being developed in collaboration with Janssen Pharmaceuticals, Inc for epilepsy. In addition, ADX71441 (GABAB receptor PAM) has received regulatory approval to start Phase 1 and is being investigated for its therapeutic use in Charcot-Marie-Tooth Type 1A disease (CMT1A), cocaine and alcohol use disorder and nicotine dependence. Discovery programs include mGluR4PAM, mGluR7NAM, TrkBPAM and mGluR3NAM & PAM. Disclaimer / Forward-looking statements: This communication does not constitute an offer or invitation to subscribe for or purchase any securities of Addex Therapeutics Ltd. This publication may contain certain forward-looking statements concerning the Company and its business. Such statements involve certain risks, uncertainties and other factors which could cause the actual results, financial condition, performance or achievements of the Company to be materially different from those expressed or implied by such statements. Readers should therefore not place undue reliance on these statements, particularly not in connection with any contract or investment decision. The Company disclaims any obligation to update these forward-looking statements.


Pre-Clinical Study to Determine Effectiveness of the Company's Immunotronics™ Platform in Preventing and Reversing Inflammation in Non-Alcoholic Steatohepatitis (NASH) LOS ANGELES, CA--(Marketwired - May 11, 2017) - Endonovo Therapeutics, Inc. ( : ENDV) ("Endonovo" or the "Company"), a developer of non-invasive electroceuticals for the treatment of vascular diseases and inflammatory conditions in vital organs, today announced it is commencing a pre-clinical study at a contract research organization to assess the therapeutic potential of its Immunotronics™ platform in preventing and reversing inflammation in Non-Alcoholic Steatohepatitis (NASH). The study is the first of several currently planned studies targeting inflammatory pathologies of hepatic origin. "We are delighted kick starting our liver disease pipeline targeting large and unmet clinical needs, particularly in fatty liver disease," commented Endonovo CEO, Alan Collier. The Company's Immunotronics™ platform is a non-invasive electroceutical device harnessing magnetically induced electrical field pathways in cells and organs eliciting an anti-inflammatory response in tissues and organs. "NASH is an inflammatory disorder in the liver and we believe our technology represents a potentially novel and equally important, non-invasive and non-pharmaceutical treatment preventing and reversing inflammation in the liver which can cause fibrosis leading to cirrhosis and ultimately end-stage liver disease or liver cancer," stated Mr. Collier. "In a crowded space flooded with pharmaceutical-based treatments, our platform remains highly differentiated and we believe non-invasive electroceuticals targeting inflammation in vital organs are the future of medicine," concluded Mr. Collier. Nonalcoholic Steatohepatitis (NASH) is a condition causing inflammation and accumulation of fat and fibrous (scar) tissue in the liver. Although a similar condition can occur in people who abuse alcohol, NASH occurs in those who drink little to no alcohol. The exact cause of NASH is unknown. However, it's seen more frequently in people with certain medical conditions such as diabetes, obesity, and insulin resistance. This combination of disorders is often called the metabolic syndrome. Like Hepatitis C, NASH is considered a "silent" liver disease because most people with NASH generally feel well and are not aware that they have a liver problem. However, if left untreated, NASH can be severe and lead to cirrhosis, a condition where the liver is permanently damaged and can no longer function correctly. This "silent" liver disease is being driven by the increasing number of Americans with obesity and diabetes. NASH is estimated to affect 2 to 5 percent of Americans, according to the National Institute of Diabetes and Digestive and Kidney Diseases, and as much as a quarter of the American population has fat in their livers, a precursor condition to NASH called Non-Alcoholic Fatty Liver Disease (NAFLD). There are currently no approved treatments for NASH, aside from weight loss, increased physical activity and avoiding alcohol and unnecessary medications. NASH is projected to become the leading indication for liver transplant by 2020. The market for a NASH treatment is estimated to reach $35 to $40 billion by 2025, according to an analyst with Deutsche Bank. Endonovo Therapeutics, Inc. is a leading developer of bioelectronic-applications in cell therapies and non-invasive electroceuticals. Endonovo's Immunotronics™ platform is dedicated to treating patients with life-threatening inflammatory conditions in vital organs using proprietary non-invasive electroceutical devices. The Company's non-invasive platform is based on magnetically-induced electrical field pathways that target the disruption of inflammation and cell death. The Company's Cytotronics™ platform harnesses the bulk electrical properties of cells and tissues, namely magnetically-induced electrical field pathways to expand and enhance the therapeutic potential of cell therapies and produce next-generation biologics. This press release contains information that constitutes forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. All statements, trends, analysis, and other information contained in this press release including words such as "anticipate," "believe," "plan," "estimate," "expect," "intend," and other similar expressions of opinion, constitute forward-looking statements. Any such forward-looking statements involve risks and uncertainties that could cause actual results to differ materially from any future results described within the forward-looking statements. Risk factors that could contribute to such differences include those matters more fully disclosed in the Company's reports filed with the Securities and Exchange Commission. The forward-looking information provided herein represents the Company's estimates as of the date of the press release, and subsequent events and developments may cause the Company's estimates to change. The Company specifically disclaims any obligation to update the forward-looking information in the future. Therefore, this forward-looking information should not be relied upon as representing the Company's estimates of its future financial performance as of any date subsequent to the date of this press release. Investors: Sign Up for Email Alerts on Endonovo


News Article | May 9, 2017
Site: globenewswire.com

Support Potential of ADX71441 to treat Irritable Bowel  Syndrome, Interstitial Cystitis and Painful Bladder Syndrome Geneva, Switzerland, 9 May 2017 - Addex Therapeutics (SIX: ADXN) announced today positive results from multiple preclinical studies of ADX71441, a positive allosteric modulator (PAM) of the gamma-aminobutyric acid subtype B (GABAB) receptor, in models of visceral hyperalgesia. The studies were led by Prof. Jyoti N. Sengupta at the Medical College of Wisconsin, with the support of a grant from the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), a division of the US National Institute of Health. The findings strongly support the potential of ADX71441 in treating hyperalgesia in colonic inflammation (colitis) and bladder inflammation (cystitis). Chronic visceral pain syndromes related to the gastrointestinal or urinary tract represent an important unmet medical need as they can significantly impact the patient's quality of life. "These data represent an important step forward in the understanding of the role played by GABAB receptors in visceral pain and the potential of ADX71441 in large unmet medical needs, such as irritable bowel syndrome, interstitial cystitis and painful bladder syndrome," said Sonia Poli, CSO of Addex. "We look forward to continuing our collaboration with Prof Sengupta's group, and further evaluating the possibility of conducting clinical trials for ADX71441 in these compelling indications." The effect of ADX71441 was studied in reliable, reproducible and quantifiable preclinical models of visceral pain, which included behavioral measurements and electrophysiology recordings(1). Visceral motor reflex after colorectal distension (CRD) and urinary bladder distension (UBD) was significantly decreased (p<0.05) following systemic administration of ADX71441 (5, 10 and 50 mg/kg ip). The visceral analgesic effect of ADX71441 did not affect the normal function of the bladder as assessed by cystometry. In electrophysiology experiments, ADX71441 demonstrated significant inhibition of the response of UBD responsive lumbo-sacral (LS) spinal dorsal horn neurons to graded bladder distension. The effect was observed in spinal intact rats, but not in cervical (C1-C2) spinal transected rats. It also produced a moderate decrease in the spontaneous firing of these neurons in spinal intact rats. ADX71441 had no effect on the mechano-transduction properties or the spontaneous firing of UBD-sensitive pelvic nerve afferent (PNA) fibers. The current behavioral experiments to assess pain and the electrophysiology results indicate that ADX71441 produces visceral analgesia in animal models of cystitis and colitis.  The results indicate that ADX71441 produces this analgesic effect primarily by acting at the supra-spinal sites without affecting bladder motility. "We thank the NIDDK for their support and Prof. Sengupta and his team for their dedication to this important research with ADX71441," commented Tim Dyer, CEO of Addex. "These compelling results are a further example of how we are leveraging our collaborations with leading academic institutions to better understand the potential of the promising candidates in our pipeline." About GABA B receptor The GABAB receptor is clinically & commercially validated. Generic GABAB receptor agonist, baclofen, is marketed for spasticity and some spinal cord injuries, and French health authorities have approved its use for the treatment of alcoholism. Baclofen is also used off label for a number of other indications including overactive bladder (OAB), but its utility is limited due to variety of side effects and rapid clearance, requiring frequent administration of higher doses. About ADX71441 ADX71441 is a potent selective positive allosteric modulator (PAM) which potentiates GABA responses at the GABAB receptor. ADX71441 is a novel, first-in-class, oral, small molecule that demonstrated excellent preclinical efficacy and tolerability in rodent models of pain, anxiety, OAB, alcohol use disorders, nicotine dependence and in a non-human primate model of cocaine use disorder. ADX71441 has also proven efficacy in a genetic model of Charcot-Marie-Tooth Type 1A disease (CMT1A). ADX71441 has a different molecular mechanism from the generic drug baclofen in that it is a positive allosteric modulator, rather than an orthosteric agonist at the GABAB receptor, with a longer half-life, suitable for once daily administration. ADX71441 only acts when the natural ligand (GABA) activates the receptor, therefore respecting the physiological cycle of activation. It has been proposed that PAMs produce less adverse effects and lead to less tolerance than direct agonists. Addex Therapeutics (www.addextherapeutics.com) is a biopharmaceutical company focused on the development of novel, orally available, small molecule allosteric modulators for neurological disorders. Allosteric modulators are an emerging class of small molecule drugs which have the potential to be more specific and confer significant therapeutic advantages over conventional "orthosteric" small molecule or biological drugs. Addex's allosteric modulator drug discovery platform targets receptors and other proteins that are recognized as essential for therapeutic intervention - the Addex pipeline was generated from this pioneering allosteric modulator drug discovery platform. Addex's lead drug candidate, dipraglurant (mGluR5 negative allosteric modulator or NAM) has successfully completed a Phase 2a POC in Parkinson's disease levodopa-induced dyskinesia (PD-LID), and is being prepared to enter registration trials for PD-LID with support from the Michael J. Fox Foundation for Parkinson's Research (MJFF). In parallel, dipraglurant's therapeutic use in dystonia is being investigated with support from the Dystonia Medical Research Foundation (DMRF). Addex's second clinical program, ADX71149 (mGluR2 positive allosteric modulator or PAM) is being developed in collaboration with Janssen Pharmaceuticals, Inc for epilepsy. In addition, ADX71441 (GABAB receptor PAM) has received regulatory approval to start Phase 1 and is being investigated for its therapeutic use in Charcot-Marie-Tooth Type 1A disease (CMT1A), cocaine and alcohol use disorder and nicotine dependence. Discovery programs include mGluR4PAM, mGluR7NAM, TrkBPAM and mGluR3NAM & PAM. Disclaimer / Forward-looking statements: This communication does not constitute an offer or invitation to subscribe for or purchase any securities of Addex Therapeutics Ltd. This publication may contain certain forward-looking statements concerning the Company and its business. Such statements involve certain risks, uncertainties and other factors which could cause the actual results, financial condition, performance or achievements of the Company to be materially different from those expressed or implied by such statements. Readers should therefore not place undue reliance on these statements, particularly not in connection with any contract or investment decision. The Company disclaims any obligation to update these forward-looking statements.


News Article | May 9, 2017
Site: www.eurekalert.org

Many contributors to patient satisfaction, such as sociodemographics and psychological factors, are beyond the physician's control BUFFALO, N.Y. -- Patient satisfaction is playing an increasingly important role in evaluating the quality of health care and reimbursing physicians for it. Exactly what drives that satisfaction has been difficult to determine. A new University at Buffalo study of 483 patients with irritable bowel syndrome (IBS) revealed that many factors that contribute to patient satisfaction are beyond the doctor's control. The results of the study were presented in Chicago today (May 9) at Digestive Disease Week during the Clinical Practice Distinguished Abstract Plenary. The study's title is "(Can't Get No) Patient Satisfaction: The Predictive Power of Demographic, GI and Psychological Factors in IBS Patients." "Ideally, patient satisfaction should be strictly based on how care is delivered," said Jeffrey Lackner, PsyD, professor in the Department of Medicine in the Jacobs School of Medicine and Biomedical Sciences at UB and senior author on the study. "But patient satisfaction is a subjective construct that is influenced by factors beyond quality of care." Lackner directs UB's Behavioral Medicine Clinic where he and his colleagues treat patients with a variety of painful disorders, including IBS. He also is a researcher with the Clinical and Translational Science Institute at UB, funded by a National Institutes of Health Clinical and Translational Science Award. In the UB study, 16 percent of participants said they were "very satisfied" with prior care for their digestive problems, while those who rated their experience as either below average or average to good was the same, at 42 percent each. Participants were asked to rate their experience on a scale of 0 to 10 with 0 being the worst health care possible and 10 the best health care possible. Surprisingly, the researchers found that patient satisfaction for these IBS patients was unrelated to either the severity or duration of their IBS symptoms or the impact that IBS had on their lives. Lackner noted that while patient satisfaction has proven difficult to characterize, it has far-reaching implications for many aspects of the doctor-patient relationship including patient loyalty, adherence to treatment, readmission rates and the potential for malpractice. In addition, it is becoming an increasingly important criterion for determining how care will be reimbursed. "Patient satisfaction is a significant metric that impacts reimbursement as health care emphasizes the value of care not the volume of care," Lackner explained. The goals of the study were to assess how patient factors, gastrointestinal symptoms and conditions and other physical and psychological factors impact patient satisfaction among IBS patients. Because IBS is a difficult and complex disorder that is associated with high rates of coexisting illnesses, Lackner said that gastroenterologists may be at a disadvantage in reimbursement schemes that focus on patient satisfaction ratings that can be influenced by nondigestive health factors. "We began this study because we don't really know what drives patient satisfaction for functional gastrointestinal disorders like IBS," he said. In the first part of their study, the UB researchers found that the more diagnostic tests (such as colonoscopies) that patients underwent, the more satisfied they were, but that finding seemed to be driven by whether or not a patient saw a gastroenterologist. "When we introduced into the model whether or not the patient had seen a gastroenterologist, the power of tests to predict patient satisfaction went away," Lackner explained. "This leads us to believe that for this population, the gastroenterologist provides reassurance that the patient doesn't have a life-threatening disease. So the reassurance they get from gastroenterologists is what predicts patient satisfaction, not the number of tests they undergo." Eighty percent of participants were female and the average age was 41. Participants were being evaluated for a large, multicenter National Institutes of Health-funded trial Lackner is leading that will evaluate the efficacy of non-drug treatments for IBS patients. "The bottom line is, there are a lot of factors that influence patient satisfaction and it turns out that many of them are not part of the delivery of care," said Lackner. UB co-authors on the study are Chris Sova; Rebecca Firth; Anne Marie Carosella, PhD; Gregory Gudleski, PhD; Leonard Katz, MD; Susan Krasner, PhD; Brian Quigley, PhD; Michael Sitrin, MD; Chris Radziwon, PhD. Darren Brenner, MD, of Northwestern University, Laurie Keefer, PhD, of Mt. Sinai and Jim Jaccard, PhD, of New York University, were also co-authors. The research was funded by the National Institute of Diabetes and Digestive and Kidney Diseases of the NIH. Founded in 1846, the Jacobs School of Medicine and Biomedical Sciences at the University at Buffalo is beginning a new chapter in its history with the largest medical education building under construction in the nation. The eight-story, 628,000-square-foot facility is scheduled to open in 2017. The new location puts superior medical education, clinical care and pioneering research in close proximity, anchoring Buffalo's evolving comprehensive academic health center in a vibrant downtown setting. These new facilities will better enable the school to advance health and wellness across the life span for the people of New York and the world through research, clinical care and the education of tomorrow's leaders in health care and biomedical sciences. The school's faculty and residents provide care for the community's diverse populations through strong clinical partnerships and the school's practice plan, UBMD Physicians' Group.


News Article | May 9, 2017
Site: globenewswire.com

Support Potential of ADX71441 to treat Irritable Bowel  Syndrome, Interstitial Cystitis and Painful Bladder Syndrome Geneva, Switzerland, 9 May 2017 - Addex Therapeutics (SIX: ADXN) announced today positive results from multiple preclinical studies of ADX71441, a positive allosteric modulator (PAM) of the gamma-aminobutyric acid subtype B (GABAB) receptor, in models of visceral hyperalgesia. The studies were led by Prof. Jyoti N. Sengupta at the Medical College of Wisconsin, with the support of a grant from the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), a division of the US National Institute of Health. The findings strongly support the potential of ADX71441 in treating hyperalgesia in colonic inflammation (colitis) and bladder inflammation (cystitis). Chronic visceral pain syndromes related to the gastrointestinal or urinary tract represent an important unmet medical need as they can significantly impact the patient's quality of life. "These data represent an important step forward in the understanding of the role played by GABAB receptors in visceral pain and the potential of ADX71441 in large unmet medical needs, such as irritable bowel syndrome, interstitial cystitis and painful bladder syndrome," said Sonia Poli, CSO of Addex. "We look forward to continuing our collaboration with Prof Sengupta's group, and further evaluating the possibility of conducting clinical trials for ADX71441 in these compelling indications." The effect of ADX71441 was studied in reliable, reproducible and quantifiable preclinical models of visceral pain, which included behavioral measurements and electrophysiology recordings(1). Visceral motor reflex after colorectal distension (CRD) and urinary bladder distension (UBD) was significantly decreased (p<0.05) following systemic administration of ADX71441 (5, 10 and 50 mg/kg ip). The visceral analgesic effect of ADX71441 did not affect the normal function of the bladder as assessed by cystometry. In electrophysiology experiments, ADX71441 demonstrated significant inhibition of the response of UBD responsive lumbo-sacral (LS) spinal dorsal horn neurons to graded bladder distension. The effect was observed in spinal intact rats, but not in cervical (C1-C2) spinal transected rats. It also produced a moderate decrease in the spontaneous firing of these neurons in spinal intact rats. ADX71441 had no effect on the mechano-transduction properties or the spontaneous firing of UBD-sensitive pelvic nerve afferent (PNA) fibers. The current behavioral experiments to assess pain and the electrophysiology results indicate that ADX71441 produces visceral analgesia in animal models of cystitis and colitis.  The results indicate that ADX71441 produces this analgesic effect primarily by acting at the supra-spinal sites without affecting bladder motility. "We thank the NIDDK for their support and Prof. Sengupta and his team for their dedication to this important research with ADX71441," commented Tim Dyer, CEO of Addex. "These compelling results are a further example of how we are leveraging our collaborations with leading academic institutions to better understand the potential of the promising candidates in our pipeline." About GABA B receptor The GABAB receptor is clinically & commercially validated. Generic GABAB receptor agonist, baclofen, is marketed for spasticity and some spinal cord injuries, and French health authorities have approved its use for the treatment of alcoholism. Baclofen is also used off label for a number of other indications including overactive bladder (OAB), but its utility is limited due to variety of side effects and rapid clearance, requiring frequent administration of higher doses. About ADX71441 ADX71441 is a potent selective positive allosteric modulator (PAM) which potentiates GABA responses at the GABAB receptor. ADX71441 is a novel, first-in-class, oral, small molecule that demonstrated excellent preclinical efficacy and tolerability in rodent models of pain, anxiety, OAB, alcohol use disorders, nicotine dependence and in a non-human primate model of cocaine use disorder. ADX71441 has also proven efficacy in a genetic model of Charcot-Marie-Tooth Type 1A disease (CMT1A). ADX71441 has a different molecular mechanism from the generic drug baclofen in that it is a positive allosteric modulator, rather than an orthosteric agonist at the GABAB receptor, with a longer half-life, suitable for once daily administration. ADX71441 only acts when the natural ligand (GABA) activates the receptor, therefore respecting the physiological cycle of activation. It has been proposed that PAMs produce less adverse effects and lead to less tolerance than direct agonists. Addex Therapeutics (www.addextherapeutics.com) is a biopharmaceutical company focused on the development of novel, orally available, small molecule allosteric modulators for neurological disorders. Allosteric modulators are an emerging class of small molecule drugs which have the potential to be more specific and confer significant therapeutic advantages over conventional "orthosteric" small molecule or biological drugs. Addex's allosteric modulator drug discovery platform targets receptors and other proteins that are recognized as essential for therapeutic intervention - the Addex pipeline was generated from this pioneering allosteric modulator drug discovery platform. Addex's lead drug candidate, dipraglurant (mGluR5 negative allosteric modulator or NAM) has successfully completed a Phase 2a POC in Parkinson's disease levodopa-induced dyskinesia (PD-LID), and is being prepared to enter registration trials for PD-LID with support from the Michael J. Fox Foundation for Parkinson's Research (MJFF). In parallel, dipraglurant's therapeutic use in dystonia is being investigated with support from the Dystonia Medical Research Foundation (DMRF). Addex's second clinical program, ADX71149 (mGluR2 positive allosteric modulator or PAM) is being developed in collaboration with Janssen Pharmaceuticals, Inc for epilepsy. In addition, ADX71441 (GABAB receptor PAM) has received regulatory approval to start Phase 1 and is being investigated for its therapeutic use in Charcot-Marie-Tooth Type 1A disease (CMT1A), cocaine and alcohol use disorder and nicotine dependence. Discovery programs include mGluR4PAM, mGluR7NAM, TrkBPAM and mGluR3NAM & PAM. Disclaimer / Forward-looking statements: This communication does not constitute an offer or invitation to subscribe for or purchase any securities of Addex Therapeutics Ltd. This publication may contain certain forward-looking statements concerning the Company and its business. Such statements involve certain risks, uncertainties and other factors which could cause the actual results, financial condition, performance or achievements of the Company to be materially different from those expressed or implied by such statements. Readers should therefore not place undue reliance on these statements, particularly not in connection with any contract or investment decision. The Company disclaims any obligation to update these forward-looking statements.


The potentially debilitating condition known as gastroparesis, which results when stomach muscle contractions function abnormally, causing the stomach's contents to empty too slowly, affects as many as five million Americans. Often the cause of the disease is unknown and the underlying bioelectrical activity that initiates and coordinates gastric contractions, known as slow waves, is not fully understood. However, a first-of-its-kind portable wireless device developed by an NYIT-led research team can monitor stomach motility to enable physicians to measure and ultimately better understand slow wave activity. Aydin Farajidavar, Ph.D., assistant professor of Electrical and Computer Engineering at New York Institute of Technology (NYIT) School of Engineering and Computing Sciences, today presented results captured from his study - the first portable wireless device developed and validated in clinical settings to document gastric contractions in patients suffering from gastroparesis. Farajidavar's work, "A Novel System and Methodology for Continuous Ambulatory Monitoring of Gastric Slow Waves," was selected as a Poster of Distinction for presentation during Digestive Disease Week 2017. Further, it was rated in the top 10 percent of all AGA (American Gastroenterological Association) abstracts selected for poster presentation at DDW, the world's largest gathering of physicians, researchers, and industry in the fields of gastroenterology, hepatology, endoscopy, and gastrointestinal surgery. "From an engineering perspective, we know that the wireless device works effectively; the system and methodology we developed enable physicians to document slow waves in patients with gastroparesis. The system can help us to better understand the effect of electrical stimulation on gastric contractions and to examine a variety of hypotheses about the gastric activity," Farajidavar said. This research project is part of an ongoing effort in NYIT School of Engineering and Computing Sciences' Integrated Medical Systems laboratory to develop devices to better diagnose gastrointestinal disorders and diseases. The team's developed system consists of a portable module that can wirelessly transmit data to a back-end receiver connected to a PC to display and store for off-line analysis. The device can also log data on a memory card for long-term monitoring. In addition to three NYIT graduate engineering students and a postdoctoral fellow, Farajidavar's research team includes Thomas L. Abell, M.D. and Abigail Stocker, M.D., world-class gastroenterologists from University of Louisville Medical Center, which is participating in the National Institute of Diabetes and Digestive and Kidney Diseases-sponsored Gastroparesis Clinical Research Consortium. Each patient in the study, registered at University of Louisville under the care of Drs. Abell and Stocker, received two temporary electrodes and leads via endoscopy prior to having a permanent stimulator. One of the leads connected to the gastric stimulator; the other was connected to the developed recording system. The gastric waves were recorded wirelessly for short periods of time (approximately 10 minutes) before and after turning on the stimulator. Then each patient received the developed portable module, set in data-logging mode. Patients returned approximately five days later; in most cases, signals were recorded and analyzed successfully in terms of frequency and amplitudes. The frequency, amplitude, and shape of the short waves varied between the patients, and for each patient, varied depending on fed- and fast-states. "It is significant that the monitoring of the gastric activity was captured over the course of several days with patients in the trial utilizing portable devices. Now that activity in the stomach can be measured objectively, this ultimately could revolutionize how some digestive diseases can be diagnosed and treated," Farajidavar said. "This result could not have happened without the close collaboration between the NYIT engineering team and the University of Louisville physician team, and the patients who volunteered to participate in this study. I am privileged to have such hard-working students and postdoctoral fellow in my lab at NYIT." New York Institute of Technology (NYIT) offers 90 degree programs, including undergraduate, graduate, and professional degrees, in more than 50 fields of study, including architecture and design; arts and sciences; education; engineering and computing sciences; health professions; management; and osteopathic medicine. A non-profit independent, private institution of higher education, NYIT has 10,000 students attending campuses on Long Island and Manhattan, online, and at its global campuses. NYIT is guided by its mission to provide career-oriented professional education, offer access to opportunity to all qualified students, and support applications-oriented research that benefits the larger world. To date, 100,000 graduates have received degrees from NYIT. For more information, visit nyit.edu.

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