AACR, Author Interviews, Cancer Research / 19.12.2019

MedicalResearch.com Interview with: [caption id="attachment_52532" align="alignleft" width="130"]Dr. Samir Parekh, MBBS Associate Professor Medicine, Hematology and Medical Oncology Icahn School of Medicine at Mount Sinai Dr. Parekh[/caption] Dr. Samir Parekh, MBBS Associate Professor Medicine, Hematology and Medical Oncology Icahn School of Medicine at Mount Sinai MedicalResearch.com: What is the background for this study? Would you briefly describe what is meant by 'neoantigens'? How might they be used to stimulate immunity in a multiple myeloma patients?  Response: Myeloma is considered a “cold” tumor for immunotherapy (as compared to some solid tumors such as melanoma) given the relatively fewer DNA mutations in an average myeloma patient. Our clinical experience suggests that this may not be totally correct.  Our findings focus on mutations that can become antigens (neo-antigens) and challenges the stereotype. We can create vaccines based on peptides resulting from these mutations to stimulate immune responses.
Author Interviews, Epilepsy / 19.12.2019

MedicalResearch.com Interview with: [caption id="attachment_52529" align="alignleft" width="160"]Steve S. Chung MD Epilepsy Neurology Banner University Medical Center University of Arizona, Phoenix, AZ Dr. Chung[/caption] Steve S. Chung MD Epilepsy Neurology Banner University Medical Center University of Arizona, Phoenix, AZ MedicalResearch.com: What is the background for this study? Response: Prior studies have shown that midazolam nasal spray (MDZ-NS) is superior to placebo in providing rapid, sustained seizure control and a favorable safety profile when administered to patients experiencing seizure clusters, a type of seizure emergency impacting an estimated 150,000 patients in the U.S. with uncontrolled epilepsy. To explore the influence of concomitant antiepileptic drugs (AEDs) on treatment outcomes, we conducted a post hoc analysis of a Phase III trial to evaluate the tolerability and efficacy of midazolam nasal spray in patients 12 years of age and older with seizure clusters, according to concomitant enzyme-inducing AED (EIAED)/non-enzyme-inducing AED (NEIAED) status and by the number of concomitant AEDs.
Author Interviews, Epilepsy / 19.12.2019

MedicalResearch.com Interview with: [caption id="attachment_52524" align="alignleft" width="200"]Eun Jung Choi, MD, PhD; Senior Director, New Product Launch, Neurology UCB Dr. Eun Jung Choi[/caption] Eun Jung Choi, MD, PhD; Senior Director, New Product Launch, Neurology UCB MedicalResearch.com: What is the background for this study? Response: Patients experiencing seizure clusters, a type of seizure emergency impacting an estimated 150,000 patients in the U.S. with uncontrolled epilepsy, are at risk for potentially serious consequences including hospitalization, mortality, physical injury, neurological damage, and status epilepticus. Although unmet needs of seizure clusters are intuitively well accepted, the burden of seizure clusters has not been well characterized and evaluated in a systemic matter. As a result, the seizure cluster patients are overlooked, and burden of seizure clusters is under-recognized. So we systematically assessed the clinical, humanistic, and economic burden associated with seizure clusters to provide the comprehensive body of evidence about burden of seizure clusters.
Author Interviews, Hematology / 19.12.2019

MedicalResearch.com Interview with: [caption id="attachment_52512" align="alignleft" width="80"]Tadeusz Robak MD, Ph.D. Professor of Hematology Medical University of Lodz, Poland Dr. Robak[/caption] Tadeusz Robak MD, Ph.D. Professor of Hematology Medical University of Lodz, Poland MedicalResearch.com: What is the background for this study? What are the main findings?
  • Rozanolixizumab is an advanced SC anti-neonatal FcRn therapy currently in clinical development which has the potential to provide a targeted, convenient option to optimize individualized patient care.
  • The rozanolixizumab Phase II (TP0001) study was specifically designed to explore multiple dose regimens in order to inform the dosing strategy for further development in primary immune thrombocytopenia (ITP). Patients received either a single dose (1 x 15 mg/kg or 1 x 20 mg/kg) or multiple doses (5 x 4 mg/kg, 3 x 7 mg/kg, 2 x 10 mg/kg) of subcutaneous (SC) rozanolixizumab. The total dose was similar in all treatment groups, ranging from 15 to 21 mg/kg.
  • Rozanolixizumab was well tolerated by patients with primary ITP across all dose groups, consistent with previous rozanolixizumab studies. Additionally, improved platelet counts and reduced immunoglobin G (IgG) levels were seen at all doses and regimens of rozanolixizumab treatment, with higher response rate, higher percentage of responders and shorter time to response achieved by the 1 x 15 mg/kg, 1 x 20 mg/kg and the 3 x 7 mg/kg rozanolixizumab dose groups. These safety, tolerability and efficacy data support the Phase III development in patients with primary ITP.
Author Interviews, JAMA, Opiods, Pediatrics, University of Michigan / 19.12.2019

MedicalResearch.com Interview with: [caption id="attachment_52464" align="alignleft" width="200"]Kao-Ping Chua, MD PhD Assistant Professor, Department of Pediatrics Susan B. Meister Child Health Evaluation and Research Center University of Michigan Dr. Kao-Ping Chua[/caption] Kao-Ping Chua, MD PhD Assistant Professor, Department of Pediatrics Susan B. Meister Child Health Evaluation and Research Center University of Michigan MedicalResearch.com: What is the background for this study? Response: Opioids are frequently prescribed to adolescents and young adults aged 12-21 years – in a recent study, 1 in 8 patients in this population were prescribed opioids during the year. At the same time, almost 30% of the 3000 opioid-related overdose deaths in 2016 among adolescents and young adults involved prescription opioids. Given the frequency of opioid prescribing and the risk of overdose, it is important to understand how to prescribe opioids safely to adolescents and young adults. However, there have been few studies that examine which opioid prescribing patterns are associated with prescription opioid overdose in adolescents and young adults. Prior studies examining these patterns have focused on older adults, particularly U.S. Veterans, so the generalizability of these findings to younger populations is unclear.
Author Interviews, Breast Cancer, Mammograms / 19.12.2019

MedicalResearch.com Interview with: [caption id="attachment_52515" align="alignleft" width="60"]Nancy R. Kressin, PhD VA Medical Center, Jamaica Plain Campus Boston, MA  Dr. Kressin[/caption] Nancy R. Kressin, PhD VA Medical Center, Jamaica Plain Campus Boston, MA   MedicalResearch.com: What is the background for this study? Response: State-level legislation requires informing women about breast density (BD) with mammogram results, to increase awareness of BD’s tendency to mask cancers on mammography, its association with increased breast cancer risk, and to encourage women to discuss personal risk and supplemental screening with physicians. The Food and Drug Administration is currently developing dense breast notification (DBN) language for use nationwide; information about effects of state DBNs could be informative for FDA’s language.
Author Interviews, Brain Injury, Exercise - Fitness, JAMA / 18.12.2019

MedicalResearch.com Interview with: [caption id="attachment_52509" align="alignleft" width="133"]John Breck, DO,  CAQSM Medical Services at University of Colorado, Boulder Dr. Breck[/caption] John Breck, DO,  CAQSM Medical Services at University of Colorado, Boulder MedicalResearch.com: What is the background for this study? Response: To date, most concussion research has focused on sport-related concussion, though it is known that a substantial number of concussions occur outside of participating in sport. Focusing solely on sport-related concussion may result in an underestimation of the underlying incidence and prevalence of concussion in the general population and leads to the widely held perception that most concussions are sport-related. An understudied population at risk for concussion are United States undergraduate college students. 
Author Interviews, Cancer Research, Genetic Research / 18.12.2019

MedicalResearch.com Interview with: [caption id="attachment_52506" align="alignleft" width="200"]Dr. Aaron Elliott, PhD CEO Ambry Genetics Dr. Aaron Elliott[/caption] Dr. Aaron Elliott, PhD CEO Ambry Genetics Dr. Elliott discusses The New York State Clinical Laboratory Evaluation Program (CLEP) approval of +RNAinsight™, a new genetic test for hereditary cancer risk.  MedicalResearch.com: What is the background for this announcement? What types of cancers can be genetically tested for predisposition? To whom should the testing be offered?  Response: The New York State Clinical Laboratory Evaluation Program (CLEP) has approved +RNAinsight™, which enables clinicians – for the first time ever – to conduct both DNA and RNA genetic testing at the same time. This is the first genetic testing advancement in over a decade to significantly increase the diagnostic yield (meaning the number of patients identified with a specific hereditary risk for cancer) in genes like BRCA1 and BRCA2. With +RNAinsight, Ambry is the first and only lab to offer this paired RNA and DNA genetic testing. Genetics may contribute to individuals’ risk of developing a number of cancers, including breast, ovarian, prostate, colorectal, and others. Approximately five to 10 percent of cancer cases are hereditary, according to the National Cancer Institute. The National Society of Genetic Counselors (NSGC) guidelines indicate who should receive genetic testing to learn whether they have increased risks to develop hereditary cancer. For example, someone with close family members who developed cancer at young ages may be a good candidate.
Author Interviews, JAMA, USPSTF / 18.12.2019

MedicalResearch.com Interview with: [caption id="attachment_42708" align="alignleft" width="143"]Dr. Michael Barry MD Director of the Informed Medical Decisions Program Health Decision Sciences Center at Massachusetts General Hospital Physician at Massachusetts General Hospit Professor of Medicine,Harvard Medical School Dr. Barry[/caption] Dr. Michael Barry MD Director of the Informed Medical Decisions Program Health Decision Sciences Center at Massachusetts General Hospital Physician at Massachusetts General Hospit Professor of Medicine,Harvard Medical School  MedicalResearch.com: What is the background for this study? Response: An abdominal aortic aneurysm (AAA) is a bulge in the wall of the main artery that carries blood from the heart to the lower part of the body. While AAA often has no signs or symptoms, the aneurysms can grow silently and burst without warning, which can be deadly. 
Author Interviews / 18.12.2019

MedicalResearch.com Interview with: Dr Timothy Brown BSc, MRes, PhD Senior Lecturer University of Manchester MedicalResearch.com: What is the background for this study? Response: Over the past 20+ years multiple lines of scientific evidence have indicated that short wavelength light is especially effective at re-setting the body clock and driving associated physiological responses in humans and other mammals. This reflects the involvement of a specialised light sensing protein in the retina, melanopsin, which more efficiently detects photons in the short wavelength region of the visible spectrum and has therefore commonly been reported as indicating a major role for ‘blue’ light. If fact, however, melanopsin does not detect colour. Instead, our perception of colour comes from comparing signals detected by the different types of cone cells in the retina. Importantly then, except under very specific circumstances, there is no correlation between perceived colour and the brightness signals detected by the melanopsin system.
Author Interviews, Diabetes, JAMA, Nutrition / 18.12.2019

MedicalResearch.com Interview with: Dr Mathilde Touvier, MPH, PhD Head of the Nutritional Epidemiology Research Team Dr Bernard Srour, PharmD, PhD Inserm, Inrae, University of Paris MedicalResearch.com: What is the background for this study? Response: We found that for an increase of 10 in the percentage of ultra-processed food quantity in the diet, we had a significant 15% increase in Type 2 diabetes risk. We have already shown, in the NutriNet-Santé cohort, associations between the consumption of ultra-processed foods and overall cancer, breast cancer, cardiovascular, coronary heart diseases risk, mortality and depressive symptoms. However, no prior study had studies the associations between ultra-processed food and Type 2 diabetes risk. We suspected that we would find these associations since some components of ultra-processed foods light have metabolic interactions with human health (some food additives for instance).
Author Interviews, Breast Cancer, Exercise - Fitness / 16.12.2019

MedicalResearch.com Interview with: [caption id="attachment_52481" align="alignleft" width="200"]Tiffany Won-Shau Chen MD Internal Medicine Residency Mount Sinai Beth Israel Dr. Chen[/caption] Tiffany Won-Shau Chen MD Internal Medicine Residency Mount Sinai Beth Israel MedicalResearch.com: What is the background for this study? Response: The research I presented on details a randomized, prospective study done to evaluate whether it would be feasible and effective to implement a yoga program for breast cancer patients receiving chemotherapy that could reduce patients' chemotherapy-related symptoms and improve their quality of life. 50 patients were recruited, half of whom underwent a 12-week long yoga program with weekly courses, while the other half did not participate in the program. Surveys were completed at baseline, 6 weeks, and 12 weeks assessing patients' functional wellbeing, sleep quality, and anxiety/depression levels.
Author Interviews, Brigham & Women's - Harvard, Cognitive Issues, JAMA, Supplements / 16.12.2019

MedicalResearch.com Interview with: [caption id="attachment_52478" align="alignleft" width="160"]Pieter A. Cohen, MD Department of Medicine, Cambridge Health Alliance Somerville, Massachusetts Harvard Medical School Boston, Massachusetts Dr. Cohen[/caption] Pieter A. Cohen, MD Department of Medicine, Cambridge Health Alliance Somerville, Massachusetts Harvard Medical School Boston, Massachusetts MedicalResearch.com: What is the background for this study? Response: There has been increasing interest in the use of over-the-counter supplements to help improve memory and cognitive function.  However, prior studies have suggested that these types of supplements might contain unapproved investigational drugs.
Author Interviews, Endocrinology, Geriatrics, Thyroid Disease / 15.12.2019

MedicalResearch.com Interview with: [caption id="attachment_52473" align="alignleft" width="200"]Carol Chiung-Hui Peng, MD Department of Internal Medicine University of Maryland Medical Center Baltimore, MD Dr. Chiung-Hui Peng[/caption] Carol Chiung-Hui Peng, MD Department of Internal Medicine University of Maryland Medical Center Baltimore, MD  MedicalResearch.com: What is the background for this study? Response: In recently published meta-analyses, focusing on the general population, showed that both overt hypothyroidism and subclinical hypothyroidism were linked to higher all-cause and cardiovascular mortality. However, there is still debate and conflicting evidence on managing overt and subclinical hypothyroidism in the elderly. This study aimed to evaluate and confirm the association between hypothyroidism and mortality in the elderly population.
Author Interviews, JAMA, Ophthalmology, Rheumatology / 15.12.2019

MedicalResearch.com Interview with: [caption id="attachment_52467" align="alignleft" width="130"]Robert Ritch, MD, FACS, FARVO Shelley and Steven Einhorn Distinguished Chair Professor of Ophthalmology Surgeon Director Emeritus Chief, Glaucoma Services Emeritus The New York Eye and Ear Infirmary of Mount Sinai New York, NY 10003 Founder, Medical Director and Chairman, Scientific Advisory Board The Glaucoma Foundation Dr. Ritch[/caption] Robert Ritch, MD, FACS, FARVO Shelley and Steven Einhorn Distinguished Chair Professor of Ophthalmology, Surgeon Director Emeritus Chief, Glaucoma Services Emeritus The New York Eye and Ear Infirmary of Mount Sinai New York, NY 10003 Founder, Medical Director and Chairman, Scientific Advisory Board The Glaucoma Foundation  MedicalResearch.com: What is the background for this study? Response: Nailfold capillaroscopy (NFC), long used in rheumatology is a new approach to investigation of glaucoma. Posterior to the nailbed and just anterior to the proximal nailfold is the cuticle, which has no structural elements visible to the naked eye. NFC is a non-invasive imaging modality that provides a highly magnified view of the capillaries at the nailfold of digits. It has also been used in ophthalmology to show morphological changes at the nailfold capillaries of POAG and XFG/XFS patients, helping to confirm the systemic nature of these diseases. [caption id="attachment_52469" align="alignleft" width="400"]Nailfold Capillaroscopy Dr. Ritch Nailfold Capillaroscopy / Dr. Ritch[/caption] With nailfold capillaroscopy, an extensive array of capillaries can be seen greatly enlarged on a monitor screen. Capillary loops can be imaged, stored, recorded with videoscopy, and blood flow actively imaged and measured.. The first series of papers on glaucoma were written by Prof Josef Flammer’s group at the turn of the 21st century, looking at vasospasm, blood flow in normal-tension and high-tension glaucoma, and relating ocular blood flow alterations to systemic vascular regulation and relating laser Doppler flowmetry to NFC. Studies from Korea later associated nailbed hemorrhages and loss of nailbed capillaries to the presence of optic disc hemorrhages and investigated correlation of of heart rate variability with visual field defects and nailfold capillaroscopy. Studies by our group began with the publication in 2015 of a paper by Pasquale et al (Nailfold Capillary Abnormalities in Primary Open-Angle Glaucoma: A Multisite Study. IOVS;56:7021) using NFC video microscopy, associating dilated capillaries, avascular zones, and hemorrhages with primary open-angle glaucoma. Successive manuscripts and presentations at conferences have indicated differences between capillary loop patterns in high-tension and normal-tension POAG and exfoliation syndrome/exfoliation glaucoma. Our goal in this paper was to compare nailfold peripheral blood flow in XFG, which had not previously been compared to control subjects using NFC. We explored the peripheral blood flow at the nailfold of patients with high-tension glaucoma, normal-tension glaucoma, exfoliation glaucoma (XFG) and compared it to control subjects further evaluate the possible differences between these glaucoma entities. We examined the morphology and extent of nailfold capillary loops, vascular tortuosity, blood flow, and nailfold hemorrhages.
Author Interviews, Breast Cancer, Genetic Research / 15.12.2019

MedicalResearch.com Interview with: [caption id="attachment_52448" align="alignleft" width="200"]Dr. Julia Blanter, MD MS Icahn School of Medicine at Mount Sinai First author of the study Dr. Blanter[/caption] Dr. Julia Blanter, MD MS Icahn School of Medicine at Mount Sinai First author of the study MedicalResearch.com: What is the background for this study? Response: The Oncotype DX Breast Cancer Assay was developed to genetically profile patients with early stage, hormone positive breast cancer and predict their 10-year risk of distant recurrence. A high-risk recurrence score is associated with a benefit from adjuvant chemotherapy whereas a low risk recurrence score is associated with little to no benefit. BRCA mutated tumors have been associated with higher risk recurrence scores as compared to BRCA negative breast cancer patients. However, there have been minimal studies relating discordance to BRCA mutations. Discordance refers to a poorly differentiated or high-grade tumor with a low risk recurrence score. Prior studies demonstrated 7-19% discordance, or difference between recurrence score and tumor grade in breast cancer patients regardless of BRCA mutation status. It has been concluded that patients who exhibit discordance may benefit from additional therapy in conjunction with endocrine therapy.
Author Interviews, Hematology, Lymphoma / 12.12.2019

MedicalResearch.com Interview with: Genentech Priscilla White, spokesperson Senior Manager, Corporate Relations Genentech MedicalResearch.com: What is the background for this study? What are the main findings?
  • Response: Mosunetuzumab is a T-cell engaging bispecific antibody designed to target CD20-positive B-cell blood cancers, by binding to both CD20 (on the surface of B-cells) and CD3 (on the surface of T-cells).
  • Analyses from the ongoing Phase I/Ib GO29781 study indicate that mosunetuzumab can produce durable responses in patients who have relapsed or who are refractory to prior treatment(s), including those who have relapsed or who are resistant to CAR T-cell therapy.
  • Results from this dose-escalation study showed encouraging efficacy with an objective response rate (ORR) of 62.7 percent (n=42/67) in slow-growing Non-Hodgkin Lymphoma and 37.1 percent (n=46/124) in aggressive NHL across all dose levels assessed.
  • Additionally, data demonstrated a complete response (CR) rate of 43.3 percent (n=29/67) in slow-growing NHL and 19.4 percent (n=24/124) in aggressive NHL. CRs showed durability, with 82.8 percent (n=24/29) of patients with slow-growing NHL remaining in remission up to 26 months off initial treatment and 70.8 percent (n=17/24) of patients with aggressive NHL, remaining in remission up to 16 months off initial treatment.
  • Of the participants who received prior CAR T-cell therapy, the ORR was 38.9 percent (n=7/18), and 22.2 percent (n=4/18) achieved a CR.
  • Adverse reactions included cytokine release syndrome (CRS) in 28.9 percent of patients with 20.0 percent at Grade 1 and 1.1 percent at Grade 3. Grade 3 neurological adverse events occurred in 3.7 percent of patients.
Author Interviews, Medical Imaging, Technology / 11.12.2019

MedicalResearch.com Interview with: Dr. David Steiner, MD PhD Google Health, USA MedicalResearch.com: What is the background for this study? Response: Advances in artificial intelligence raise promising opportunities for improved interpretation of chest X-rays and many other types of medical images. However, even before researchers begin to address the critical question of clinical validation, there is important work to be done establishing strategies for evaluating and comparing different artificial intelligence algorithms. One challenge is defining and collecting the correct clinical interpretation or “label” for the large number of chest X-rays needed to train and evaluate these algorithms. Another important challenge is evaluating the algorithm on a dataset that actually represents the diversity of the cases encountered in clinical practice. For example, it might be relatively easy to make an algorithm that performs perfectly on a few hundred or so “easy” cases, but this of course might not be particularly useful in practice.
Author Interviews, Cancer Research, Fertility, OBGYNE / 11.12.2019

MedicalResearch.com Interview with: [caption id="attachment_52421" align="alignleft" width="200"]Marie Hargreave, PhD Senior Researcher Danish Cancer Society Research Center Copenhagen Dr. Hargreave[/caption] Marie Hargreave, PhD Senior Researcher Danish Cancer Society Research Center Copenhagen MedicalResearch.com: What is the background for this study? Response: Very few studies have examined the association between frozen embryo transfer and the risk of childhood cancer and most of them have been too small to show any effects. In our large nationwide population based study we found that frozen embryo replacement was associated with an increased risk of childhood cancer and especially for leukemia and neuroblastomas.
Author Interviews, Cancer Research, Dermatology, JAMA / 11.12.2019

MedicalResearch.com Interview with: Lily Wang Student at University of Toronto Toronto, Ontario, Canada  MedicalResearch.com: What is the background for this study? Response:  Impaired skin barrier and aberrant immune function in atopic dermatitis (AD) may impact immune response to malignancy. Conflicting data exist on the risk of cancer in patients with AD. The purpose of our study was to determine the risk of non-cutaneous and cutaneous cancers in patients with atopic dermatitis compared to the general population (i.e. without AD). 
Author Interviews, HIV, JAMA, Sexual Health, STD / 11.12.2019

MedicalResearch.com Interview with: [caption id="attachment_52403" align="alignleft" width="200"]Jason Ong, PhD, MMed, MBBS, FAChSHM, FRACGP Twitter: @DrJasonJOng Sexual Health Physician, Melbourne Sexual Health Centre, Alfred Health Associate Professor (Hon), London School of Hygiene and Tropical Medicine, UK Central Clinical School, Monash University, Australia Melbourne School of Population and Global Health, University of Melbourne, Australia  Associate Editor, Sexually Transmitted Infections Special Issues Editor, Sexual Health Board Director, ASHM  Dr. Jason Ong[/caption] Jason Ong, PhD, MMed, MBBS, FAChSHM, FRACGP Twitter: @DrJasonJOng Sexual Health Physician, Melbourne Sexual Health Centre, Alfred Health Associate Professor (Hon), London School of Hygiene and Tropical Medicine, UK Central Clinical School, Monash University, Australia Melbourne School of Population and Global Health, University of Melbourne, Australia Associate Editor, Sexually Transmitted Infections Special Issues Editor, Sexual Health Board Director, ASHM   MedicalResearch.com: What is the background for this study? Response: HIV pre-exposure prophylaxis (PrEP) is being rolled out globally. This will protect many people from HIV, however PrEP does not protect against other sexually transmitted infections (STI). So we wanted to quantify how much STIs are in PrEP users as a means to advocate to strengthen sexual health services in these settings where PrEP is being offered.
Author Interviews, Cost of Health Care, JAMA, Primary Care / 11.12.2019

MedicalResearch.com Interview with: [caption id="attachment_52362" align="alignleft" width="200"]Julie Reiff Researcher Health Care Cost Institute Julie Reiff[/caption] Julie Reiff BA Researcher Health Care Cost Institute  MedicalResearch.com: What is the background for this study? Response: Prior studies have attempted to define primary care spending and quantify such spending. Using definitions from the Milbank Memorial Fund, we used Health Care Cost Institute data to calculate primary care utilization and spending among those age 0-64 with employer-sponsored insurance.
Author Interviews, Autism, Neurology / 11.12.2019

MedicalResearch.com Interview with: [caption id="attachment_52438" align="alignleft" width="159"]Dr. Neil Dawson PhD Senior Lecturer Lancaster University Dr. Dawson[/caption] Dr. Neil Dawson PhD Senior Lecturer Lancaster University MedicalResearch.com: What is the background for this study? Response: Deletions on chromosome 2p16.3, involving deletion of the NEUREXIN1 gene, dramatically increase the risk of developing a wide range of neurodevelopmental disorders including autism, Tourette’s syndrome and schizophrenia. We don’t fully understand the mechanisms involved. In our study we wanted to understand how the genetic deletion impacts on brain function and the ability of brain regions to communicate with one another, as these are known to be impaired in these neurodevelopmental disorders. We also wanted to determine how the genetic deletion impacts on the function of neurotransmitter systems involved in these disorders, and whether drugs targeting these neurotransmitter systems could restore some of the deficits in brain function seen.
Author Interviews, Epilepsy, NEJM, NIH / 11.12.2019

MedicalResearch.com Interview with: [caption id="attachment_52433" align="alignleft" width="160"]Robin Conwit, M.D Program Director, Division of Clinical Research NIH: National Institute of Neurological Disorders and Stroke Dr. Conwit[/caption] Robin Conwit, M.D Program Director, Division of Clinical Research NIH: National Institute of Neurological Disorders and Stroke MedicalResearch.com: What is the background for this study? Response: Approximately one-third of patients with intractable status epilepticus do not respond to benzodiazepines, often the first line medications given in the emergency department, leaving doctors to decide among three commonly prescribed anti-convulsants. In this study all of the anti-convulsants were equally effective. The trial gives doctors a way to clear the air of arguments that one of these drugs works any better than the others, or that any one of them is a lot safer.  It gives doctors a reason to choose a dosing strategy in status epilepticus for levetiracetam, which has otherwise been controversial and non-standard.  
Author Interviews, Cancer Research, Leukemia / 11.12.2019

MedicalResearch.com Interview with: [caption id="attachment_52430" align="alignleft" width="167"]Bridget Marcellino, MD Icahn School of Medicine at Mount Sinai Mount Sinai Hospital Dr. Marcellino[/caption] Bridget Marcellino, MD Icahn School of Medicine at Mount Sinai Mount Sinai Hospital MedicalResearch.com: What is the background for this study? Response: Our work focuses on elucidating the mechanisms that drive the pathogenesis and progression of myeloproliferative neoplasms (MPN). Dysregulation of the TP53 pathway is associated with MPN progression evidenced by the association of TP53 loss of heterozygosity with transformation to acute myeloid leukemia (AML) and the presence of inactivating mutations of TP53 found in a proportion of MPN-related AML patients.   Studies have shown that TP53 mutations, TP53 deletions and overexpression of the negative regulator of TP53, Murine Double Minute 2 (MDM2) all contribute to TP53 downregulation in MPNs and we therefore are interested in exploring other potential means by which TP53 is downregulated. Protein Phosphatase, Mg2+/Mn2+ Dependent 1D (PPM1D) is another negative regulator of the TP53 pathway and activating mutations in this gene are present in myeloid malignancies including MPNS. We therefore hypothesized that genomic alterations in PPM1D and/or overexpression of PPM1D would be found in the hematopoietic cells of MPN patients.
Author Interviews, Breast Cancer, Cancer Research / 10.12.2019

MedicalResearch.com Interview with: [caption id="attachment_52426" align="alignleft" width="130"]Jian Jin, Ph.D. Mount Sinai Endowed Professor in Therapeutics Discovery Professor, Department of Pharmacological Sciences Professor, Department of Oncological Sciences Director, Mount Sinai Center for Therapeutics Discovery Co-leader, Cancer Clinical Investigation Program, Tisch Cancer Institute Icahn School of Medicine at Mount Sinai  Dr. Jian Jin[/caption] Jian Jin, Ph.D. Mount Sinai Endowed Professor in Therapeutics Discovery Professor, Department of Pharmacological Sciences Professor, Department of Oncological Sciences Director, Mount Sinai Center for Therapeutics Discovery Co-leader, Cancer Clinical Investigation Program, Tisch Cancer Institute Icahn School of Medicine at Mount Sinai  MedicalResearch.com: What is the background for this study? Response: Triple-negative breast cancer (TNBC), a subtype of breast cancer that lacks estrogen receptor (ER), progesterone receptor (PR) and human epidermal growth factor receptor 2 (HER2), represents 12-20% of all breast cancers. TNBC has poor prognosis, high recurrence, a low survival rate, and has higher incidence in African-American and Hispanic women. Currently, there are no effective therapies for treating a substantial portion of TNBC patients. Enhancer of zeste homolog 2 (EZH2) is the main enzymatic subunit of the polycomb repressive complex 2 (PRC2) which catalyzes trimethylation of histone H3 lysine 27 (H3K27me3) to promote transcriptional silencing. EZH2 is overexpressed in multiple types of cancer including triple negative breast cancer (TNBC) and high expression levels correlate with poor prognosis. Several EZH2 inhibitors which inhibit the enzymatic activity of EZH2 have shown promise in treating sarcoma and follicular lymphoma in clinics. However, current EZH2 inhibitors are ineffective at blocking proliferation of TNBC cells even though they effectively inhibit the enzymatic activity of EZH2. While the proteolysis targeting chimera (PROTAC) technology for selective degradation of the target protein has been rapidly gaining momentum in the drug discovery field, the hydrophobic tagging approach for selective protein degradation has received considerately less attention from the scientific community. 
Author Interviews, Hematology, Lymphoma / 10.12.2019

MedicalResearch.com Interview with: [caption id="attachment_52400" align="alignleft" width="200"]Steven M. Horwitz, MD Memorial Sloan Kettering Cancer Center New York, NY Dr. Horwitz[/caption] Steven M. Horwitz, MD Memorial Sloan Kettering Cancer Center New York, NY MedicalResearch.com: What is the background for this study? Response: Relapsed or refractory Peripheral T-Cell Lymphoma (R/R PTCL) remains a disease of significant unmet medical need. Duvelisib is an oral dual inhibitor of PI3K-δ and PI3K-γ approved for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) after at least two prior therapies, relapsed or refractory follicular lymphoma (FL) after at least two prior systemic therapies, and is being developed for the treatment of additional hematologic malignancies including R/R PTCL. In early studies, we saw a suggestion of quite good activity of duvelisib as a single agent in a range of subtypes of T-cell lymphoma. The PRIMO study is an ongoing, multi-center, open-label, registration-directed Phase 2 study evaluating duvelisib in patients with R/R PTCL that is expected to enroll approximately 120 patients. The study includes both a dose optimization phase and an expansion phase. The Primo study will be sufficiently powered to give a much more precise estimate of the activity in peripheral t cell lymphomas. However, prior to initiating the main cohort we needed to first try to identify an optimal dose. That “dose optimization cohort” is the subject of our presentation here.
Author Interviews, Hematology, Lymphoma / 10.12.2019

MedicalResearch.com Interview with: Prof. John Seymour, MBBS, Ph.D Lead investigator of the MURANO Trial Director. Department of Hematology at the Peter MacCallum Cancer Centre & Royal Melbourne Hospital in Australia MedicalResearch.com: What is the background for this study? What are the main findings?
  • MURANO is an international, multicenter, open-label, randomized Phase 3 study designed to evaluate the efficacy and safety of venetoclax in combination with rituximab compared with bendamustine in combination with rituximab in patients with relapsed/refractory (R/R) chronic lymphocytic leukemia (CLL).
  • At this year’s American Society of Hematology (ASH) annual meeting, we presented results from the four-year updated analysis from the study, which showed an 81 percent reduction in the relative risk of disease progression or death in patients randomized to the chemotherapy-free, two year fixed-duration treatment course of venetoclax plus rituximab and higher rates of minimal residual disease (MRD)-negativity compared to the standard of care regimen, bendamustine plus rituximab.
  • The long-term data further support the sustained clinical benefit of fixed-duration treatment with venetoclax in combination with rituximab for this patient population.
  • The safety profile of the combination is consistent with the known safety profile of each individual therapy alone. There were no new serious safety issues observed in the MURANO study since the last update. 
Author Interviews, Health Care Systems, University of Pittsburgh / 10.12.2019

MedicalResearch.com Interview with: [caption id="attachment_52414" align="alignleft" width="188"]Hayley Drew Germack PhD Assistant Professor of Acute and Tertiary Care University of Pittsburgh School of Nursing Dr. Hayley Drew Germack[/caption] Hayley Drew Germack PhD Assistant Professor of Acute and Tertiary Care University of Pittsburgh School of Nursing MedicalResearch.com: What is the background for this study? Response: The rate of rural hospital closures has been increasing over the last ten years. Rural hospitals close for a number of reasons including poor hospital economic health tied to uncompensated care and community factors, like a local aging population. Rural hospital and unit closures have been tied to decreased access to emergency and specialty care for patients including decreased access to obstetric-gynecological services and increase travel time for appointments. A recent paper also found a 6% increase in mortality in conditions needing emergent attention after rural hospital closures. We examined the impact of hospital closures in rural counties on the counties’ supply of physicians.
Addiction, Author Interviews, Opiods, Technology / 10.12.2019

MedicalResearch.com Interview with: [caption id="attachment_52410" align="alignleft" width="148"]Anna Konova, PhD Assistant Professor, Dept. of Psychiatry & UBHC Core Faculty, Brain Health Institute Rutgers University - New Brunswick Dr. Anna Konova[/caption] Anna Konova, PhD Assistant Professor, Dept. of Psychiatry & UBHC Core Faculty, Brain Health Institute Rutgers University - New Brunswick MedicalResearch.com: What is the background for this study? Response: Opioid reuse and relapse are common outcomes even when a person is seeking treatment for their addiction. These reuse events pose many health risks, as well as risk for treatment failure. We currently lack the much needed tools to understand and predict this reuse vulnerability. In this study, we used computer games that assess a person's decision making process, to get at psychological processes related to how people make decisions involving risks, when they transitioned between lower and higher reuse vulnerability states during the first few months of opioid treatment.