Author Interviews, Cancer Research, J&J-Janssen, Leukemia / 17.05.2018

MedicalResearch.com Interview with: [caption id="attachment_41804" align="alignleft" width="132"]Andrzej Jakubowiak, MD, PhD Professor of Medicine Director, Myeloma Program University of Chicago Dr. Jakubowiak[/caption] Andrzej Jakubowiak, MD, PhD Professor of Medicine Director, Myeloma Program University of Chicago MedicalResearch.com: What is the background for this announcement? Would you briefly explain what is meant by multiple myeloma?   Response: DARZALEX (daratumumab) in combination with VELCADE (bortezomib), melphalan and prednisone - VMP - received U.S. FDA approval for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant (ASCT). With this most recent approval, DARZALEX is now the first monoclonal antibody approved for newly diagnosed patients with this disease. Multiple myeloma is an incurable blood cancer that occurs when malignant plasma cells grow uncontrollably in the bone marrow. Despite the introduction of new medicines over the last decade, which has led to significant improvements in outcomes for patients with multiple myeloma, multiple myeloma remains an incurable disease. In 2018, it is estimated that 30,700 people will be diagnosed and 12,770 will die from the disease in the United States.
Author Interviews, Epilepsy, Neurological Disorders, NYU/NYMC, Pharmaceutical Companies / 17.05.2018

MedicalResearch.com Interview with: https://www.gwpharm.com/epilepsy-patients-caregivers/patientsAnup Patel, M.D. Section Chief of Neurology Interim Division Chief of Neurology Nationwide Children’s Hospital MedicalResearch.com: What is the background for this study? Response: The study evaluated kids and adults with an epilepsy syndrome (Lennox Gastaut Syndrome – LGS) that is often difficult to treat and does not respond well to current medical treatment.  The study was a double blind randomized control trial evaluating how well a plant based, liquid solution, cannabidiol (CBD) product made by Greenwich Biosciences called Epidiolex helped to treat drop seizures (the most common seizure type in LGS) and how safe it was compared to placebo.  Two doses (10 mg/kg/day and 20 mg/kg/day) were evaluated compared to placebo.
Addiction, Author Interviews, JAMA, Opiods, Pharmaceutical Companies / 14.05.2018

MedicalResearch.com Interview with: “Big Lunch Extras Reading” by Big Lunch Extras is licensed under CC BY 2.0Scott E. Hadland, MD, MPH, MS Assistant Professor of Pediatrics | Boston University School of Medicine Boston Medical Center Director of Urban Health & Advocacy Track | Boston Combined Residency Program Boston, MA 02118 MedicalResearch.com: What is the background for this study? What are the main findings? Response: Numerous pharmaceutical companies have received media attention for their role in promoting opioid prescribing through speaker programs and other marketing plans in which large-value payments are given to a small number of doctors to promote opioids. In our study, we sought to tell the other side of the story. We wanted to identify whether low-value marketing, including industry-sponsored meals, which are commonplace in the US, were associated with increased opioid prescribing. We found that 1 in 14 doctors received opioid marketing from pharmaceutical companies in 2014, and those that received marketing prescribed 9% more opioids the following year. With each additional meal a doctor received, he or she prescribed more and more opioids the following year. Our sample included 43% of the active physician workforce in the US, suggesting how widespread and far-reaching this effect might be.
Author Interviews, Critical Care - Intensive Care - ICUs, Infections, Merck / 10.05.2018

MedicalResearch.com Interview with: [caption id="attachment_41611" align="alignleft" width="167"]Dr. Elizabeth Rhee MD Director, Infectious Disease Clinical Research at Merck Dr. Rhee[/caption] Dr. Elizabeth Rhee MD Director, Infectious Disease Clinical Research Merck MedicalResearch.com: What is the background for this study? What are the main findings? Response: High-risk patients, such as the critically ill, with suspected bacterial infections require prompt treatment with appropriate empiric therapy to improve survival. Given the high prevalence of multidrug-resistant (MDR) Pseudomonas aeruginosa in the ICU setting, new safe and broadly effective treatment options are needed for critically ill patients requiring antipseudomonal agents. Ceftolozane/tazobactam (C/T) is an antipseudomonal cephalosporin/beta-lactamase inhibitor combination with broad in vitro activity against Gram-negative pathogens, including MDR P. aeruginosa and many extended-spectrum beta-lactamase (ESBL) producers. It is FDA approved for complicated intra-abdominal and urinary tract infections in adults at 1.5g (1g/0.5g) q8h. C/T is currently being studied at 3g (2g/1g) q8h, for the treatment of ventilated nosocomial pneumonia, in the ASPECT-NP Phase 3 trial. This Phase 1 pharmacokinetic (PK) study investigated the penetration of a 3g dose of C/T in the epithelial lining fluid (ELF) of ventilated patients with proven or suspected pneumonia. This is the dose and patient population being evaluated in ASPECT-NP. ELF lines the alveoli, and investigators took samples in a group of 26 patients to see what amount of C/T was in the lung and what was circulating in the plasma during the dosing intervals. In mechanically ventilated critically ill patients, the 3g dose of C/T achieved ≥50% lung penetration (relative to free plasma) and sustained levels in ELF above the target concentrations for the entire dosing interval. These findings support the 3g dose that is included in the ASPECT-NP Phase 3 trial. 
Author Interviews, Critical Care - Intensive Care - ICUs, Infections, Merck / 10.05.2018

MedicalResearch.com Interview with: [caption id="attachment_41607" align="alignleft" width="124"]Becky Jayakumar, PharmD College of Pharmacy Assistant Professor of Pharmacy Practice Roseman University of Health Sciences Dr. Jayakumar[/caption] Becky Jayakumar, PharmD College of Pharmacy Assistant Professor of Pharmacy Practice Roseman University of Health Sciences MedicalResearch.com: What is the background for this study? What are the main findings? Response: Bacteremia (bloodstream infections) due to Gram-negative (GN) bacteria are a frequent cause of severe sepsis and pose serious therapeutic challenges due to multidrug-resistance (MDR). Ceftolozane/tazobactam (C/T) is a novel antipseudomonal cephalosporin combined with an established β-lactamase inhibitor. This retrospective, observational study evaluated the clinical outcomes of C/T real-world use in severely ill patients. Twenty-two patients with sepsis and/or bacteremia were included; 95% of whom had Pseudomonas aeruginosa that was resistant to almost all antibacterials with the exception of colistin. C/T successfully treated the majority of these complicated patients. In this real-world study, 77% of patients had a clinical response with C/T and 75% had a microbiological response. Clinical success rates were high and mortality rates were similar to other studies in this severely ill population.
Author Interviews, Critical Care - Intensive Care - ICUs, Infections, Merck / 10.05.2018

MedicalResearch.com Interview with: [caption id="attachment_41601" align="alignleft" width="200"]Amanda Paschke, MD, MSCE Senior principal scientist Infectious disease clinical research Merck Research Laboratories Dr.Amanda  Paschke[/caption] Amanda Paschke, MD, MSCE Senior principal scientist Infectious disease clinical research Merck Research Laboratories MedicalResearch.com: What is the background for this study? What are the main findings? Response: This study sought to evaluate a new beta-lactam/beta-lactamase inhibitor antibacterial combination, imipenem/relebactam (IMI/REL), compared with colistin plus imipenem for the treatment of infections caused by resistant Gram-negative bacteria. Patients enrolled in the trial had hospital-acquired or ventilator-associated bacterial pneumonia (HABP/VABP), complicated intra-abdominal infections (cIAI), or complicated urinary tract infections (cUTI) caused by pathogens that were non susceptible to imipenem, a carbapenem antibacterial. In this study, the primary outcome was a favorable overall response to treatment, which was comparable between the IMI/REL vs colistin + IMI arms. Colistin (often combined with a carbapenem) is currently among the standard of care treatment regimens for MDR infections.  A key secondary endpoint of the study was safety.  IMI/REL was well tolerated; among all treated patients, drug-related adverse events (AEs) occurred in 16.1% of IMI/REL and 31.3% of colistin + IMI patients with treatment-emergent nephrotoxicity observed in 10% (3/29 patients) and 56% (9/16 patients), respectively (p=0.002). Results of the trial support the use of imipenem-relebactam (IMI/REL) as an efficacious and well-tolerated treatment option for carbapenem-resistant infections. 
Alzheimer's - Dementia, Author Interviews, Merck, NEJM / 02.05.2018

MedicalResearch.com Interview with: Dr. Michael F. Egan MD Merck & Co. North Wales, PA 19454   MedicalResearch.com: What is the background for this study? What are the main findings? Response: A leading theory of Alzheimer's Disease is that it is caused by the buildup of amyloid plaques in the brain. Amyloid is composed of a sticky peptide called Abeta.  Abeta production can be blocked by Inhibiting an enzyme called BACE.  In animal models, BACE inhibtion prevent amyloid accumulation.  We aimed to see if a potent BACE inhibitor would slow clinical decline in Alzheimer's Disease. EPOCH was a Phase 2/3 randomized, placebo-controlled, parallel-group, double-blind study evaluating efficacy and safety of two oral doses of verubecestat an investigational BACE inhibitor, administered once-daily versus placebo in patients with mild-to-moderate AD currently using standard of care treatment. The primary efficacy outcomes of the study are the change from baseline in cognition (assessed using the Alzheimer's Disease Assessment Scale Cognitive Subscale, or ADAS-Cog),  as well as the change from baseline in function (assessed using the Alzheimer's Disease Cooperative Study – Activities of Daily Living, or ADCS-ADL)  after 78 weeks of treatment. Following the recommendation of the external Data Monitoring Committee (eDMC), which assessed overall benefit/risk during  the trial,  the study was stopped early, as there was “virtually no chance of finding a positive clinical effect.” Verubecestat did not reduce cognitive or functional decline in patients with mild-to moderate Alzheimer’s disease and was associated with treatment-related adverse events. 
Author Interviews, Cancer Research, Cost of Health Care, JAMA, Pharmaceutical Companies / 09.04.2018

MedicalResearch.com Interview with: [caption id="attachment_41105" align="alignleft" width="133"]Aaron Mitchell, MD Division of Hematology/Oncology, Department of Medicine,  Lineberger Comprehensive Cancer Center The Cecil G. Sheps Center for Health Services Research The University of North Carolina at Chapel Hil Dr. Mitchell[/caption] Aaron Mitchell, MD Division of Hematology/Oncology, Department of Medicine, Lineberger Comprehensive Cancer Center The Cecil G. Sheps Center for Health Services Research The University of North Carolina at Chapel Hill MedicalResearch.com: What is the background for this study? What are the main findings? Response: Financial relationships between physicians and the pharmaceutical industry are very common. However, we are just beginning to figure out whether these relationships may lead to potentially concerning changes in physician behavior - whether physicians tend to prescribe more of the drugs made by a company that has given them money. We decided to ask whether oncologists who receive money from drugmakers are more likely to use the cancer drugs made by companies that have given them money in the past. In studying two specific groups of cancer drugs, one for kidney cancer and one for chronic myeloid leukemia (CML), we found that oncologists who had received payments such as meals, consulting fees, travel & lodging expenses from the manufacturer of one of these drugs tended to use that drug more. When looking at oncologists who received payments for research, we found increased prescribing among the kidney cancer drugs but not the CML drugs.
Allergan, Author Interviews, Bipolar Disorder, Depression, Mental Health Research / 05.04.2018

MedicalResearch.com Interview with: [caption id="attachment_41025" align="alignleft" width="133"]Dr. C. David Nicholson, PhD Chief R&D Officer  Allergan Dr. C. David Nicholson[/caption] Dr. C. David Nicholson, PhD Chief R&D Officer Allergan MedicalResearch.com: What is the background for this data milestone?  Response: Bipolar I depression refers to the depressive episodes of bipolar I disorder, the overarching brain and behavioral disorder. People with bipolar I disorder can have manic and depressive episodes, as well as mixed episodes that feature both manic and depressive symptoms at the same time. Bipolar I depression typically lasts at least two weeks, and can be difficult to differentiate from major depression during diagnosis. Once diagnosed, treating bipolar depression can be difficult given the few therapies available to manage these symptoms of bipolar I disorder. Additionally, patients with bipolar disorder may experience shifts from depression to mania or mania to depression as well as mixed states. More treatment options are needed so that physicians can find a therapy that will treat bipolar depression effectively, while also addressing the myriad of other symptoms that patients can experience. Cariprazine is already approved for the treatment of mania and mixed episodes. With this new data, we have the potential to also treat bipolar depression, effectively addressing the full spectrum of symptoms associated with bipolar I disorder with just one medication.
Addiction, Author Interviews, Opiods, Pharmaceutical Companies / 04.04.2018

MedicalResearch.com Interview with: http://usworldmeds.com/Mark Pirner, MD, PhD Senior Medical Director US WorldMeds   MedicalResearch.com: What is the background for this study? Would you briefly explain how lofexidine works? Response: LUCEMYRA (lofexidine) was studied in two phase 3 pivotal randomized, double-blind, placebo-controlled clinical studies, and a phase 3 open-label study. Clinical pharmacology studies included evaluation of drug-drug interaction studies that demonstrated lofexidine can be safely administered concomitantly with methadone, buprenorphine or naltrexone. LUCEMYRA is an alpha 2 adrenergic receptor agonist that reduces the surge of norepinephrine signaling in the brain which results from abrupt opioid withdrawal, and thereby reduces the severity of opioid withdrawal symptoms. 
Author Interviews, Boehringer Ingelheim, FDA, Pulmonary Disease, Rheumatology / 03.04.2018

MedicalResearch.com Interview with: [caption id="attachment_40947" align="alignleft" width="167"]Dr. Thomas Leonard, Ph.D. Executive director, Clinical Development and Medical Affairs, Specialty Care Boehringer Ingelheim Pharmaceuticals, Inc. Dr. Thomas Leonard[/caption] Dr. Thomas Leonard, Ph.D. Executive director, Clinical Development and Medical Affairs, Specialty Care Boehringer Ingelheim MedicalResearch.com: What is the background for this announcement? Would you briefly explain what is meant by systemic sclerosis? What are the disease symptoms and manifestations? Response: The FDA recently granted Fast Track designation to nintedanib for the treatment of systemic sclerosis with interstitial lung disease (SSc-ILD) – paving the way for Boehringer Ingelheim to take an important step in advancing this potential therapy for those affected by this disease. The designation was based on Boehringer Ingelheim’s Investigational New Drug application (IND) and the anticipated efficacy and safety data from SENSCIS™ (Safety and Efficacy of Nintedanib in Systemic SClerosIS), a double-blind, randomized, placebo-controlled global Phase III trial which is fully enrolled and includes more than 520 patients from 32 countries. The FDA’s Fast Track designation facilitates the development of new therapies that treat serious conditions and fulfill an unmet medical need in an effort to get treatments to those in need sooner, like those living with systemic sclerosis. Systemic sclerosis, also known as scleroderma, is a rare disease characterized by thickening and scarring of connective tissue of multiple organs in the body, typically affecting women between ages 25 and 55. Most people with the disease will develop some degree of lung scarring, or interstitial lung disease (ILD), which is the leading cause of death among people with systemic sclerosis. Nintedanib, currently marketed as Ofev®, is approved for treatment of a rare lung disease called idiopathic pulmonary fibrosis, or IPF, and has been shown to slow disease progression as measured by annual rate of decline in lung function. Because SSc-ILD and IPF share similarities in how the underlying lung scarring, or fibrosis, forms in people with the disease, Boehringer Ingelheim is evaluating the impact of nintedanib on SSc-ILD.
Author Interviews, Breast Cancer, Chemotherapy, JAMA, Novartis / 26.03.2018

MedicalResearch.com Interview with: [caption id="attachment_40799" align="alignleft" width="145"]Melanie E. Royce, MD, PhD Division of Hematology/Oncology University of New Mexico Comprehensive Cancer Center Albuquerque Dr. Royce[/caption] Melanie E. Royce, MD, PhD Division of Hematology/Oncology University of New Mexico Comprehensive Cancer Center Albuquerque MedicalResearch.com: What is the background for this study? What are the main findings? Response: BOLERO-4 is an open label, single-arm, Phase II study that evaluates the combination of everolimus plus letrozole as a first-line treatment for hormone receptor (HR)-positive/human epidermal growth factor receptor (HER2)-negative advanced breast cancer patients, as well as the use of everolimus plus exemestane beyond initial progression. Results of the BOLERO-4 trial published in JAMA Oncology showed that everolimus in combination with endocrine therapy is an effective first-line treatment option for postmenopausal women with HR+/HER2- advanced breast cancer. A total of 202 patients received everolimus in combination with letrozole as first-line treatment between March 7, 2013 and December 17, 2014. Median progression-free survival (PFS) in the first-line setting was 22.0 months (95% CI 18.1-25.1) with an overall response rate of 45% (95% CI 38.1-52.2) and clinical benefit rate of 74% (95% CI 67.7-80.1). A total of 152 (75%) discontinued treatment, primarily due to disease progression (51%) or adverse events (16%). Data from a smaller number of patients in BOLERO-4 also show limited efficacy with continued everolimus, combined with exemestane, following disease progression. Second-line treatment was ongoing in 16 (32%) patients, while 34 (68%) had discontinued. The most frequent reason for second-line treatment discontinuation was disease progression (56%). In the second-line setting, median PFS was 3.7 months (95% CI 1.9-7.4) with an overall response of 6% (95% CI 1.3-16.5) and clinical benefit rate of 28% (95% CI 16.2-42.5). Safety findings from BOLERO-4 are consistent with previous studies of Afinitor in advanced breast cancer. The most common (≥ 20% incidence) first-line all-grade adverse events were stomatitis (69%), weight loss (44%), nausea (37%) and anemia (35%). Most were ‘low grade’ in severity (grade 1 or 2) and generally well managed. Safety findings show the most common (≥ 10% incidence) second-line adverse events were stomatitis (20%) and weight loss (20%). Lower rates of stomatitis in second-line were noted. 
Abuse and Neglect, Author Interviews, Neurology, Pharmaceutical Companies / 23.03.2018

MedicalResearch.com Interview with: [caption id="attachment_40742" align="alignleft" width="200"]Joseph Horrigan MD, Pediatric neuropsychiatrist Dr. Horrigan[/caption] Dr. Joseph Horrigan MD Pediatric neuropsychiatrist Chief medical officer   MedicalResearch.com: What is the background for this study? Would you briefly explain what is meant by myotonic dystrophy? What are the manifestations of this disease? Response: This was the first pharmaceutical intervention study conducted in adolescents and adults with congenital and juvenile onset DM1. Myotonic dystrophy type 1 (DM1) is a disorder that impacts multiple body systems following a trinucleotide expansion repeat of the DMPK gene on chromosome 19. Children with Congenital DM1 present at birth with respiratory insufficiency, talipes equinovarus (clubfoot), feeding difficulties and hypotonia. There is a risk mortality rate in the first year of life. As children grow, they are at risk for intellectual impairment, autistic features, gastrointestinal symptoms, motor delay and a variety of muscle-based symptoms.
Author Interviews, Opiods, Pharmaceutical Companies / 23.03.2018

MedicalResearch.com Interview with: [caption id="attachment_40738" align="alignleft" width="133"]Vishal Bala Senior Quantitative Data Analyst CareDash Vishal Bala[/caption] Vishal Bala Senior Quantitative Data Analyst CareDash MedicalResearch.com: What is the background for this study? What are the main findings? Response: Prior research into physicians and their relationships with the pharmaceutical industry has typically retained a narrow scope, focusing on how payments may be associated with prescription habits (sometimes limited to specific regions) for specific categories of drugs. For example, Modi et al. 2017 and Bandari et al. 2017 explored these connections in the context of some urologic drugs specifically. Research conducted by ProPublica in 2016 studied the connection between industry payments and physician prescriptions across some of the largest medical specialties, but was only able to look at “brand-name” vs. “generic” categories and were limited by overlapping timeframes for payments and prescriptions. CareDash took this analysis further by using Open Payments and Medicare Part D data to investigate the relationship between payments made by individual companies for specific drugs and the prescribing habits of the recipient physicians for those drugs. CareDash’s main findings are that healthcare providers who received payments for a drug from a pharmaceutical company are 5 times more likely to be high prescribers for that drug than those physicians who did not receive a payment. Physicians are 5.3 times more likely to prescribe a drug than their peers after they have received a payment for that drug from the manufacturer. When physicians already prescribe a drug significantly more often than their peers, they are 5.6 times more likely to later receive payment for that drug from the drug's manufacturer. Looking at the opioid drug class specifically, CareDash found that physicians receiving payment on behalf of an opioid were 14.5 times more likely to prescribe that opioid over alternatives.
Author Interviews, Flu - Influenza, Infections, Mental Health Research, Primary Care, Roche / 14.03.2018

MedicalResearch.com Interview with: [caption id="attachment_40568" align="alignleft" width="150"]James W. Antoon, MD, PhD, FAAP Assistant Professor of Clinical Pediatrics University of Illinois at Chicago Associate Medical Director, Pediatric Inpatient Unit Children's Hospital, University of Illinois Hospital & Health Sciences System Chicago, IL 60612  Dr. Antoon[/caption] James W. Antoon, MD, PhD, FAAP Assistant Professor of Clinical Pediatrics University of Illinois at Chicago Associate Medical Director, Pediatric Inpatient Unit Children's Hospital, University of Illinois Hospital & Health Sciences System Chicago, IL 60612  MedicalResearch.com: What is the background for this study? What are the main findings? Response: Oseltamivir, commonly known as Tamiflu, is the only commercially available medication FDA approved to treat the flu.  Since the 2009 H1N1 flu epidemic pediatric prescriptions for Tamiflu have soared.  In the United States, about 40% of Tamiflu prescriptions are given to children less than 16 years of age.  Following reports of abnormal behavior, such as hallucinations, self-injury and suicide attempts in adolescents on Tamiflu, the FDA placed a new warning about these neuropsychiatric symptoms on the drug label.  Whenever the FDA puts out label warning about a drug, doctors and the public take notice. Whether Tamiflu truly causes these side effects is unclear.  For this study we chose to focus on the most consequential of those reports: suicide. The potential link between a drug and suicide is a particularly difficult topic to study for a number of reasons. There are things that happen together or at the same time that can influence someone to attempt suicide and it is very difficult to know which thing is actually having an affect. In our study, other things that can influence suicide are socioeconomic status, mental health, trauma, abuse, among others.  Separating the effects of these confounders can be difficult. It is also possible that the disease itself, which in this case is the flu, causes the effect of suicide. Finally, and luckily, suicide is rare. Our database had 12 million children per year and over five year 21,000 attempted suicide. Of those, only 251 were taking Tamiflu. To get past these issues, we took advantage of a growing drug safety research collaboration between the Departments of Pediatrics and Pharmacy at our institution.  Previous studies have compared those on Tamiflu to those not on Tamiflu to see if there are more side effects in the Tamiflu group.  Our team utilized a novel study method called a case-crossover design. What’s different about this study is that we used each patient as their own comparison.  In other words, we compared each patient to themselves rather than a different group of people.  We essentially studied how patients behaved when the Tamiflu was in their system compared to other l periods where they were not on Tamiflu.  This allowed use to account for the personal differences noted above like mental health and socioeconomic status.   We also compared those children with flu who got Tamiflu and those with flu who did not get Tamiflu to see if the infection itself could be associated with increased suicide. After accounting for all these variables, we did not find any an association between Tamiflu exposure and suicide. Our findings suggest that Tamiflu does NOT increase the risk of suicide in children or teenagers.
Author Interviews, GSK, Herpes Viruses, Infections, Vaccine Studies / 10.03.2018

MedicalResearch.com Interview with: [caption id="attachment_40475" align="alignleft" width="200"]Herpes Zoster or Shingles of chest Wikipedia Herpes Zoster or Shingles of chest
Wikipedia[/caption] Anthony. L. Cunningham, MD The Westmead Institute for Medical Research Westmead, NSW University of Sydney, Sydney,   MedicalResearch.com: What is the background for this study? What are the main findings? Response: This study examines the reasons why the HZ subunit vaccine candidate (HZ/su vaccine) consisting of a single viral protein, varicella-zoster glycoprotein E, and and adjuvant (immunostimulant) combination AS01B is so effective as a vaccine to prevent shingles (>90%), especially in those over the age of 70 years and 80 years, as published in recent trials i.e. it combats the declining immunity in the aging which usually restricts vaccine efficacy to under 60% in these age groups. 
Author Interviews, Multiple Sclerosis, Ophthalmology, Pharmaceutical Companies / 08.03.2018

MedicalResearch.com Interview with: [caption id="attachment_40544" align="alignleft" width="159"]Sarah A. Morrow MD, MS, FRCPC Associate Professor of Neurology Department of Clinical Neurological Sciences University of Western Ontario (Western) Dr. Morrow[/caption] Sarah A. Morrow MD, MS, FRCPC Associate Professor of Neurology Department of Clinical Neurological Sciences University of Western Ontario (Western) MedicalResearch.com: What is the background for this study? Response: Acute demyelinating optic neuritis, which presents with loss of vision and painful eye movements, is common in multiple sclerosis (MS) occurring 50% of persons with MS. High dose (≥ 1g) corticosteroids administered through an IV became the standard of practice after the landmark Optic Neuritis Treatment Trial as IV administration. However, in that study the IV dose of corticosteroids was much higher (1 gram daily) than the oral dose (1 mg/kg). Thus, it is not clear if IV administration is still better if equivalent doses are used orally. Oral administration is much more convenient for patients and less expensive, and previous studies showed that it is preferred by patients. In this study, we asked the following question: are high dose (≥ 1000mg) IV corticosteroids superior to equivalent doses of oral corticosteroids for the acute treatment of optic neuritis? We randomly assigned fifty-five cases of acute optic neuritis to 1000mg IV methylprednisolone or 1250mg oral prednisone daily for three days and compared recovery of their vision over the next 6 months. 
Author Interviews, Dermatology, Eli Lilly, Sexual Health / 07.03.2018

MedicalResearch.com Interview with: [caption id="attachment_40411" align="alignleft" width="120"]Dr. Jennifer Cather MD Medical Director at Modern Dermatology and Modern Research Associate Dallas, Texas  Dr. Cather[/caption] Dr. Jennifer Cather MD Medical Director at Modern Dermatology and Modern Research Associate Dallas, Texas  MedicalResearch.com: What is the background for this study? What are the main findings?  Response: Genital psoriasis can be an uncomfortable and burdensome condition that many people living with moderate-to-severe plaque psoriasis experience. Due to the significant impact, Lilly conducted a 12-week Phase 3b clinical trial with patients with moderate-to-severe genital psoriasis treated with ixekizumab, which found that patients had a greater decrease in the impact of their condition on sexual activity compared to placebo as early as one week. Specifically, trial patients were randomized to receive ixekizumab (80 mg every two weeks, following a 160-mg starting dose) or placebo and researchers measured pre-specified patient-reported outcomes, including the Genital Psoriasis Sexual Impact Scale (GPSIS), which is composed of the Sexual Activity Avoidance (Avoidance) and Impact of Sexual Activity on Genital Psoriasis Symptoms (Impact) subscales. Patient-reported outcomes were also measured by the Sexual Frequency Questionnaire (SFQ) item 2, evaluating the impact of genital psoriasis on the frequency of sexual activity, and the Dermatology Life Quality Index (DLQI) item 9, evaluating the impact of skin symptoms on sexual difficulties. At 12 weeks, patients reported the following outcomes:
  • DLQI Item 9 0/1: 92.0 percent of patients treated with ixekizumab compared to 56.8 percent of patients treated with placebo reported no (0) or little (1) sexual difficulties caused by skin symptoms.
  • SFQ Item 2 0/1:  78.4 percent of patients treated with ixekizumab compared to 21.4 percent of patients treated with placebo (reported the frequency of sexual activity was either never (0) or rarely (1) limited by genital psoriasis.
  • GPSIS-Avoidance 1/2:  76.7 percent of patients treated with ixekizumab compared to 25.7 percent of patients treated with placebo reported never (1) or rarely (2) avoiding sexual activity due to genital psoriasis.
  • GPSIS-Impact 1/2:  85.7 percent of patients treated with ixekizumab compared to 52.9 percent of patients treated with placebo reported worsening of genital psoriasis symptoms during or after sexual activity was very low/none at all (1) or low (2). 
Author Interviews, Boehringer Ingelheim, Dermatology, Pulmonary Disease, Rheumatology / 05.03.2018

MedicalResearch.com Interview with: [caption id="attachment_40385" align="alignleft" width="200"]Donald Zoz, MD Senior Associate Director Clinical Development & Medical Affairs IPF/ILD Boehringer Ingelheim Pharmaceuticals, Inc. Dr. Zoz[/caption] Donald Zoz, MD Senior Associate Director Clinical Development & Medical Affairs IPF/ILD Boehringer Ingelheim Pharmaceuticals, Inc. MedicalResearch.com: What is the background for this platform? Would you briefly explain what is meant by scleroderma? How does it affect a person's skin and ability to function? Whom does this disease primarily affect? Response: “More Than Scleroderma™: The Inside Story” is Boehringer Ingelheim’s new global initiative highlighting real-life, inspirational stories of people living with the rare disease scleroderma. The new effort, created with support from the Scleroderma Foundation in the U.S., aims to raise awareness of the disease, dispel misperceptions and provide important resources to support and guide those on their journey with scleroderma. The initiative’s website http://www.morethanscleroderma.com/us/ features a powerful and inspiring collection of diverse photographs and video profiles of 10 people across the U.S. living with scleroderma and sharing their ‘inside story.’ Each tells their unique and moving experience with scleroderma through diagnosis to learning to live with the disease and manage it. Many less fortunate people in developing countries have to deal with this on a daily basis and only have the support of Orphan Drug Distributors and charities and I'm so thankful that people like this are available to support these people! Scleroderma, also known as systemic sclerosis, is a rare disease characterized by thickening and scarring of the skin, lungs and other organs. Scleroderma affects fewer than 200,000 people in the U.S. and typically affects women in the prime of their lives, between the ages of 25 and 55 taking a marked toll just as they are building their careers and bearing the responsibility of caring for their family. Nearly all people with scleroderma (more than 90%) will develop some skin symptoms including skin thickening, tightened skin around the joints, small red spots on the face and hands and hard lumps on pressure points and joints. Most people with the disease will also develop some degree of lung scarring, or interstitial lung disease (ILD). When the disease's signature thickening and scarring develops in vital organs, such as the lungs, there are potentially debilitating and life-threatening consequences.
Author Interviews, Autism, Pharmaceutical Companies, Roche, Vanderbilt / 16.02.2018

MedicalResearch.com Interview with: [caption id="attachment_40115" align="alignleft" width="123"]Dr Kevin Sanders, MD Principle Medical Director-Product Development Neuroscience Assistant Professor, Departments of Psychiatry and Pediatrics Vanderbilt University Dr. Sanders[/caption] Dr Kevin Sanders, MD Principle Medical Director-Product Development Neuroscience Assistant Professor, Departments of Psychiatry and Pediatrics Vanderbilt University  MedicalResearch.com: What is the background for this announcement?  Response: The FDA has granted Roche Breakthrough Therapy Designation for its investigational oral medicine balovaptan (previously known as RG7314), a vasopressin 1a (V1a) receptor antagonist for individuals with autism spectrum disorder (ASD). FDA Breakthrough Therapy Designation for balovaptan is primarily based on efficacy findings in the VANILLA (Vasopressin ANtagonist to Improve sociaL communication in Autism) study, a Phase II trial of balovaptan in adults with ASD. Trial results were first presented at the International Congress for Autism Research (IMFAR) in May 2017. Treatment effects were observed on the Vineland-II (secondary endpoint) and also demonstrated that balovaptan was safe and well tolerated by the subjects in the study. The Vineland-II is a scale that measures socialization, communication and daily living skills. This data was presented to the FDA and is part of the basis of the Breakthrough Designation. 
Author Interviews, Hematology, Novo Nordisk, Surgical Research / 15.02.2018

MedicalResearch.com Interview with: [caption id="attachment_40092" align="alignleft" width="200"]Stephanie Seremetis, M.D. Corporate Vice President and Chief Medical Officer Biopharmaceuticals at Novo Nordisk Dr. Stephanie Seremetis[/caption] Stephanie Seremetis, M.D. Corporate Vice President and Chief Medical Officer Biopharmaceuticals at Novo Nordisk MedicalResearch.com: What is the background for this announcement? Response: We’re proud and excited to make Rebinyn® (Coagulation Factor IX (Recombinant), GlycoPEGylated) available as a new extended half-life treatment for hemophilia B management. Rebinyn® is an injectable medicine used to treat and control bleeding in adults and children with hemophilia B. It can be used to treat bleeds when they occur and to manage bleeding during surgery. Rebinyn® is not used for routine prophylaxis or for immune tolerance induction in patients with hemophilia B. Hemophilia B is a serious, chronic, inherited bleeding disease that affects about 5,000 people in the U.S. People living with hemophilia B have low levels of clotting Factor IX protein in the blood, often resulting in prolonged or spontaneous bleeding, especially into the muscles, joints or internal organs. 
Author Interviews, Cancer Research, Novartis, Pancreatic / 05.02.2018

MedicalResearch.com Interview with: [caption id="attachment_39848" align="alignleft" width="133"]Dr- Lynn Matrisian Dr. Matrisian[/caption] Lynn Matrisian, PhD, MBA Chief science Officer Pancreatic Cancer Action Network MedicalResearch.com: Would you tell us a little about PNETs? How common is this type of pancreatic tumor? How does Lutathera differ from other treatments for this tumor?  Response: Pancreatic neuroendocrine tumors (PNETs) make up about 6 percent of all pancreatic cancer diagnoses. They are less common and slower growing than the more common type of pancreatic cancer, adenocarcinoma, and have a better prognosis. Lutathera® is a peptide receptor radionuclide therapy (PRRT) that was approved for the treatment of gastroenteropancreatic neuroendocrine tumors (GEP-NETs), including PNETs, that express somatostatin receptors. The drug is a somatostatin analog that is conjugated to a radionuclide (177Lu) to selectively deliver radiotherapy to the cancer cells. Other treatment options for PNETs include surgery (partial or complete removal of the tumor), chemotherapy (typically in combination) or radiation therapy (conventional as well as PRRT). Patients may also receive targeted therapies. Sutent® blocks platelet-derived growth factor receptors (PDGFRs) α and β, stem-cell factor receptor (c-kit) and vascular endothelial growth factor receptor (VEGFR)-2 and VEGFR-3, leading to inhibition of cell growth and angiogenesis. Afinitor® behaves as a rapamycin analog, blocking the mammalian target of rapamycin (mTOR) signaling pathway. Prior to Lutathera’s approval, there were two non-PRRT somatostatin analogs approved for PNET patients. These drugs were initially intended to mitigate some of the symptoms of the disease, but they were also found to slow the cancer cells’ growth. The approved somatostatin analogs are lanreotide and octreotide. 
Abbvie, Author Interviews, Hepatitis - Liver Disease, NEJM, Pharmaceutical Companies / 29.01.2018

MedicalResearch.com Interview with: [caption id="attachment_39686" align="alignleft" width="247"]In the United States, hepatitis A, hepatitis B and hepatitis C are the most common types, but also included are hepatitis D and E. CDC/ E.H. Cook, Jr. In the United States, hepatitis A, hepatitis B and hepatitis C are the most common types, but also included are hepatitis D and E.
CDC/ E.H. Cook, Jr.[/caption] Stefan Zeuzem, M.D. Professor of Medicine Chief Internal Medicine Goethe University Hospital Frankfurt, Germany MedicalResearch.com: What is the background for this study? What are the main findings?  Response: Chronic hepatitis C virus (HCV) infection is a major global public health problem with more than 71 million people infected worldwide, and can result in significant morbidity and mortality, including liver cirrhosis, hepatocellular carcinoma, and death.1 This publication describes the efficacy and safety results from two Phase 3 clinical trials, ENDURANCE-1 and ENDURANCE-3, in patients with chronic HCV genotypes (GT) 1 or 3 infection who were treated with an all-oral, once-daily combination regimen of direct-acting antiviral agents (DAA) glecaprevir (GLE) at 300 mg and pibrentasvir (PIB) at 120 mg. The findings from ENDURANCE-1 trial show that the GLE/PIB combination regimen (G/P) given for 8 weeks to HCV GT1 chronically infected non-cirrhotic treatment-naïve or treatment-experienced (with sofosbuvir and/or interferon with ribavirin) patients was safe and well-tolerated, achieved high efficacy with a sustained virologic response at post-treatment week 12 (SVR12) rate >99% and was non-inferior to 12-week treatment with G/P. The trial also included subjects who were co-infected with human immunodeficiency virus (HIV), and all of these subjects achieved SVR12 while maintaining HIV suppression throughout the study. ENDURANCE-3 trial results show that the G/P regimen given for 8 weeks to HCV GT3 chronically infected non-cirrhotic treatment-naïve patients was safe and well-tolerated, achieved high efficacy in this historically difficult to cure GT with an SVR12 rate >94%, and was non-inferior to 12-week treatment with G/P, which in turn was non-inferior to the treatment with 12-week DAA regimen of sofosbivir and daclatasvir. 
Allergan, Author Interviews, JAMA, Ophthalmology / 05.01.2018

MedicalResearch.com Interview with: Steven Woloshin, MD MS [caption id="attachment_25281" align="alignleft" width="180"]Steven Woloshin, MD Professor of The Dartmouth Institute Professor of Medicine Professor of Community and Family Medicine Dr. Steven Woloshin[/caption] Professor of The Dartmouth Institute Professor of Medicine Professor of Community and Family Medicine The Center for Medicine in the Media Dartmouth Institute for Health Policy and Clinical Practice Lebanon, New Hampshire MedicalResearch.com: What is the background for this study? What are the main findings? Response: There has been a lot of debate about the legal maneuvers (ie, transferring patents to the Mohawk Indians) Allergan has employed to delay marketing of generic alternatives to Restasis (cyclosporine ophthalmic emulsion 0.05%).   But there is a more fundamental question that has received little attention:  Does Restasis work?  It is not approved in the European Union, Australia or New Zealand where registration applications were "withdrawn prior to approval due to insufficient evidence of efficacy" in 2001.   Although Canada approved Restasis, its national health technology assessment unit, unconvinced of meaningful benefit, recommended Canada not pay for it - according to our research, no Canadian provincial or federal drug plan currently does.   Nevertheless, Americans have spent $8.8 billion in total sales between 2009 and 2015 on Restasis, including over $2.9 billion in public monies through Medicare Part D.
Author Interviews, Infections, Pfizer, Vaccine Studies / 22.12.2017

MedicalResearch.com Interview with: [caption id="attachment_39062" align="alignleft" width="200"]Judith Absalon, M.D., M.P.H Senior Director, Vaccines Clinical Research  Pfizer Pharmaceuticals Dr. Judith Absalon[/caption] Judith Absalon, M.D., M.P.H Senior Director, Vaccines Clinical Research Pfizer Pharmaceuticals MedicalResearch.com: What is the background for these two studies? Response: Invasive serogroup B meningococcal disease (MenB) is uncommon, yet serious, is unpredictable and can strike at any age, including healthy teenagers and young adults, with potentially long-lasting and devastating consequences, including death. The data from these two Phase 3 studies, one in adolescents (Study 1009) and one young adults (Study 1016), highlight that Trumenba can help protect teens and young adults against meningococcal group B disease. Additionally, these two large Phase 3 studies confirmed the results of earlier studies and supported the transition from Accelerated to Traditional Approval in the US; were pivotal for approvals in Europe, Australia, and Canada earlier this year; and add to the growing portfolio of research for TRUMENBA.
Allergan, Author Interviews, Bipolar Disorder, Brigham & Women's - Harvard / 20.12.2017

MedicalResearch.com Interview with: allerganGary Sachs, MD Associate Clinical Professor of Psychiatry Harvard Medical School  MedicalResearch.com: What is the background for this data milestone? Response: Bipolar disorder affects about 5.7 million adults in the United States.  It is a common, often disabling condition in which abnormal mood states impair a person’s ability to carry out everyday tasks. Bipolar disorder touches nearly every family and community in America, because periods of illness, a patient’s symptoms often impact their family, their friends, and their community. There are a limited number of products approved to treat bipolar depression and even fewer products that have been studied and approved to treat the full spectrum of bipolar disorder, from mania through depression. Having another product proven to treat the full range of bipolar disorder would be a welcome addition to the treatment options currently available to the psychiatry community and patients.
Author Interviews, Genetic Research, Pharmaceutical Companies / 17.12.2017

MedicalResearch.com Interview with: [caption id="attachment_38992" align="alignleft" width="128"]Alexander S Hauser, PhD student MRC Laboratory of Molecular Biology Cambridge UK Department of Drug Design and Pharmacology, University of Copenhagen Copenhagen, Denmark Alexander Hauser[/caption] Alexander S Hauser, PhD student MRC Laboratory of Molecular Biology Cambridge UK Department of Drug Design and Pharmacology, University of Copenhagen Copenhagen, Denmark MedicalResearch.com: What is the background for this study? What are the main findings? Response: The prevalence and impact of genetic variation among all human G protein coupled receptors (GPCRs) that are targeted by FDA-approved drugs remain unknown. In this study, we present a comprehensive analysis and map of the pharmacogenomics landscape of GPCR drug targets. The key highlights are: - GPCRs targeted by drugs show extensive genetic variation in the human population - Variation occurs in functional sites and may result in altered drug response - Understanding GPCR genetic variation may help reduce global healthcare expenses
Author Interviews, Brigham & Women's - Harvard, Infections, Leukemia, Merck, Transplantation / 12.12.2017

MedicalResearch.com Interview with: [caption id="attachment_38873" align="alignleft" width="120"]Francisco M. Marty, M.D Associate Professor, Harvard Medical School Dana–Farber Cancer Institute and Brigham and Women’s Hospital Dr. Marty[/caption] Francisco M. Marty, M.D Associate Professor, Harvard Medical School Dana–Farber Cancer Institute and Brigham and Women’s Hospital MedicalResearch.com: What is the background for this study? What are the main findings? Response: Cytomegalovirus (CMV) is the most common infection in patients who undergo allogeneic hematopoietic-cell transplantation (bone marrow transplantation with cells from donors different than the patient). Up until now, we had no antiviral agent that could be used for prophylaxis (prevention) of CMV post-transplant because of the side effects of drugs available to date (ganciclovir, valganciclovir, foscarnet, cidofovir). This trial confirmed that letermovir was highly effective in preventing CMV infection when used in the first 100 days after allogeneic HCT, was associated with minimal side effects of concern and was also associated with lower all-cause mortality by Week 24 post-HCT.
Author Interviews, Heart Disease, Lipids, Sanofi / 29.11.2017

MedicalResearch.com Interview with: [caption id="attachment_38586" align="alignleft" width="143"]Dr. Jay Edelberg VP Head of CV Development and Head Global CV Medical Affairs Dr. Edelberg[/caption] Dr. Jay Edelberg MD, PhD VP Head of CV Development and Head Global CV Medical Affairs Sanofi  MedicalResearch.com: What is the background for this study? What are the main findings? Response: Patients with heterozygous familial hypercholesterolemia (HeFH) are often not able to achieve their target low-density lipoprotein cholesterol (LDL-C) levels, and some may require lipoprotein apheresis (LA) to lower their “bad cholesterol.” Apheresis is a procedure similar to kidney dialysis, where bad cholesterol is mechanically removed from the blood. It is an invasive, expensive, and time-consuming treatment for patients, as well as physicians. The Phase III ESCAPE clinical study looked at the potential effect of LA on total Praluent, free and total PCSK9 concentrations, as well as the combined pharmacodynamics effect of total Praluent on LDL-C-lowering. Praluent levels remained unaffected by apheresis, and Praluent consistently suppressed free PCSK9 levels in patients with HeFH, regardless of LA treatment. This analysis further confirms clinical ESCAPE data that Praluent can be used in conjunction with LA and may reduce or potentially eliminate the need for LA in some patients.
Author Interviews, Pharmaceutical Companies, Technology / 29.11.2017

MedicalResearch.com Interview with: HITLab Mr. David Moore MBA Senior Vice President, Commercial Novo Nordisk Mr. David Moore presented the keynote presentation “Breaking Through Barriers in Innovation” at the 4th Annual HITLAB Innovators Summit, NYC November 28, 2017.  MedicalResearch.com: How did you become interested in incorporating technological advances into the sales and marketing end of pharmaceutical delivery? Response: Part of our message today is that we understand that there is an opportunity to improve outcomes if we have a better focus on collaboration and integration through engagement with the patient rather than through medications alone. We continue to innovate in diabetes pharmaceutical care but are also focusing more on meeting the patient in their journey through everyday life. The opportunity to connect, collaborate and integrate is what digital health is all about. As manufacturers, we are still early in the integration process. When we think about technological changes from the patient’s perspective, health care has become much different. The patient no longer just goes to the doctor’s office for a conversation or opinion, with the doctor keeping all the notes. Now the patient has access to a broad range of medical information and usually their own medical records. There is an element of needing things to be user-friendly. As with any consumer product or service with choices, if the choice isn’t user-friendly, the consumer won’t use it. We are looking for ways to make the delivery of pharmacologic products user-friendly to both the patient and provider.