Dr. Alessandro Iannaccone Invited to Write Editorial in Lancet to Comment on Phase 1B Trial of Hereditary Retinal Degeneration

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Dr. Alessandro Iannaccone

Despite a tremendous increase in the research efforts and progress to find treatments and cures for hereditary retinal degenerations, therapeutic options able to restore significantly vision remain to date limited. But the scenario is changing.

Alessandro Iannaccone, MD, MS, Director of the Retinal Degenerations and Ophthalmic Genetics Service and Associate Professor of Ophthalmology at the Hamilton Eye Institute of the University of Tennessee Health Science Center (UTHSC), was invited by Lancet to comment, alongside with Dr. Marco Zarbin, MD, PhD, FARVO, Professor and Chair of the Department of Ophthalmology at the Rutgers-New Jersey Medical School, on a recent Phase 1B clinical trial of a special form of retinal degeneration due to a specific enzymatic defect in the retina that reflects thing changing scenario.

Dr. Iannaccone, who also serves on the Scientific Advisory Board of Foundation fighting Blindness (FFB) and is the President of the FFB Mid-South chapter of Memphis, was asked to comment on a new approach to treating Leber congenital amaurosis (LCA) with mutations in either the RPE65 or the LRAT genes. LCA is a very early onset form of a condition known better as retinitis pigmentosa (RP). These forms of LCA are characterized by enzymatic defects that cause early-onset severe visual loss and nystagmus due to a deficiency of the vitamin A metabolite, 11-cis-retinal, in photoreceptors. The 11-cis-retinal molecule is the so-called “chromophore” of the rod visual pigment, rhodopsin. “When the retina does not receive enough 11-cis-retinal or cannot recycle effectively the one it has, our night vision is greatly incapacitated”, commented Iannaccone. “In the case of these forms of LCA, the protein portion of the visual pigment, called opsin, can reach normally the outer segment of the photoreceptor, but the cells cannot use it because it is not complexed with enough 11-cis-retinal to form the actual visual pigment, rhodopsin, and night vision is very poor despite only limited degeneration of the photoreceptors themselves. The at least partial structural integrity of photoreceptors in these forms of LCA is behind the success that gene therapy has also had in the RPE65-linked form of LCA”. Patients from the UTHSC Retinal Degenerations and Ophthalmic Genetics Service at the Hamilton Eye Institute have been successful participants in such trial.

Now a pharmacological approach to bypassing the enzymatic defects in these forms of LCA has been devised and tested in a human Phase 1B trial. An orally administered synthetic retinoid, 9-cis-retinyl acetate, was used to provide an alternate form of the chromophore to photoreceptors and tested to assess its safety and its preliminary efficacy. The results have been very encouraging. “This trial is extremely encouraging and points to a rapidly changing scenario in the development of therapeutic approaches for RP, LCA and related diseases.”

But some notes of caution are warranted. “Interpreting Phase 1B clinical trials that are not designed to assess efficacy can prove tricky, so we need to be careful not to overstate the results of this otherwise very promising study,” remarked Iannaccone. “This is especially important when a largely pediatric population is treated, and visual function testing is performed on children. Some of the tests of visual function one needs to use to assess efficacy can be particularly variable, especially in children, whose attention span and comprehension of the test can be limited and can increase significantly during the trial. In these specific forms of LCA in which there is an enzymatic deficiency in the retina precluding proper provision of 11-cis-retinal to photoreceptors, there is also a very delayed ability to adapt to changes in lighting conditions. This too can impact greatly our ability to measure correctly changes in visual function in these patients, and potentially confound the outcome of such trials. Emphasis is usually placed on letting patients adjust to darkness for long enough, but not as much emphasis is placed on letting them adjust long enough to light.”

Dr. Iannaccone highlighted this important issue in the Lancet editorial, showing how increasing durations of light adaptation before running a visual field test can impact dramatically the size of a patient’s visual field and the quality of the vision measured within it. “We have to be extremely careful at controlling as precisely as possible these variables. The last thing anyone wants to do is to claim huge therapeutic successes when, in fact, the perceived benefits may have been due, at least in part, to a poorly controlled confounding variable as essential as the time spent adjusting to the light of the testing instrument before running the test. This is not as critical in normal subjects, but in patients with retinal degenerations, and especially in these specific LCA patients, it is essential.” Dr. Iannaccone noted how, in his experience, this issue is often seen in RP patients in his clinic, and especially in patients with another unique related disorder, known as choroideremia. “Since 9-cis-retinyl acetate carries also a potential risk for a neuro-ophthalmological complication known as pseudotumor cerebri, it is especially important that claims of efficacy for this treatment be made with a great deal of caution.”

There is still much appeal for treatments that can provide across-the-board help for these patients. Although hereditary retinal degenerations as a whole are estimated to affect only one in 3,500 to 4,000 people and fall in the category of the orphan diseases, there are over 200 genes that can cause these conditions. This is a huge genetic heterogeneity for such rare diseases. Thus, broad-spectrum, non-specific treatments that can favorably impact these conditions remain essential to treat these diseases. But to cure them,” remarked Iannaccone, “we now understand that a molecularly driven and genetically specific approach may be indispensable, and this Phase 1B trial with 9-cis-retinyl acetate is an excellent example of that. This is the fruit of bed-to-bench-to-bed translational research at its best, and represents the future of retinal degenerations therapeutics.” Trials and studies that Dr. Iannaccone is presently involved with at the Hamilton Eye Institute fall in the same conceptual framework. “I am very excited about the latest developments in this field. The first gene for RP was cloned just in 1990, and we have come a very long way in a short time. I have been working in this field for nearly 25 years, and the fruit of hard, at times unrewarding, work are truly coming to fruition now. It comes as no surprise to me that the National Eye Institute has also identified finding treatments and cures for retinal degenerations as one of its top programmatic priorities, and it is great to be at the forefront of this field.”

 

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The link to the Lancet editorial in question can be found at:

 

http://www.ncbi.nlm.nih.gov/pubmed/25030841

 

Hohmeier Named NACDS Faculty Scholar

The National Association of Chain Drug Stores Foundation has selected Kenneth C. Hohmeier, PharmD, assistant professor of Clinical Pharmacy, as one of the 2014-15 NACDS Foundation Faculty Scholars. As a NACDS Foundation Faculty Scholar, Dr. Hohmeier will participate in a program focused on community pharmacy-based patient research.

After earning his Doctor of Pharmacy degree at the University of Toledo in Ohio, he began his post-graduate year 1 (PGY1) community pharmacy residency training with Kroger Pharmacy and the University of Toledo. Upon completion of his residency year, Dr. Hohmeier began practicing at an independent pharmacy chain in Northern Ohio. After three years, Dr. Hohmeier had built a profitable pharmacy including compounding and medication therapy management services, a disease state management program with a local employer group, and he worked collaboratively with University Hospitals and the Cleveland Clinic.  He also gained appointment as a clinical assistant professor of Pharmacy Practice at Northeast Ohio Medical University, where he taught in both the MTM and pharmacy leadership courses, and provided experiential education instruction to introductory and advanced pharmacy practice experience student pharmacists.

In addition to his practice responsibilities, Dr. Hohmeier has also served on several American Pharmacist Association standing committees, and recently served as an elected member of the Ohio Pharmacist Association Board of Trustees. Dr. Hohmeier also has had several publications, including grant-funded research. His research interests include clinical services in the community pharmacy, pharmacy leadership and entrepreneurship, and innovative health care reimbursement models.

Professor Donna K. Hathaway of The University of Tennessee Health Science Center Receives $2.5 Million Grant for Kidney Transplant Medication Research

Dr. Donna Hathaway

Donna K. Hathaway, PhD, RN, FAAN, University Distinguished Professor in the Department of Advanced Practice and Doctoral Studies in the College of Nursing at the University of Tennessee Health Science Center (UTHSC), has received a grant totaling $2,507,759 from the National Institute of Diabetes and Digestive and Kidney Diseases, a subsidiary of the National Institutes of Health. The award will be used to support a project titled, “An RCT for Medication Adherence in Kidney Transplant Recipients.” The award will be distributed over a four-year period. [Read more...]

Inaugural Raza A. Dilawari DVP to be Held on Saturday, September 20

The Inaugural Raza A. Dilawari Endowed Distinguished Visiting Professorship will be held on Saturday, September 20, from 8am until 1:30pm at the FedEx Institute of Technology. The topic is: Multidisciplinary Cancer Symposium: Contemporary Insights into the Management of Pancreatic Neoplasms, featuring Dr. James Yao, Associate Professor, Department of Gastrointestinal (GI) Medical Oncology, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, TX

To register for the DVP, please go to www.utconferences.com/dilawaridvp to reserve your spot soon. The full PDF brochure for the Dilawari DVP can be viewed online by clicking here: www.tinyurl.com/dilawaridvp.

The Raza A. Dilawari DVP features a world-class lineup of presenters and speakers, featuring:

  • “Management of Pancreatic Neuroendocrine Tumors” – James Yao, MD, Inaugural Raza A. Dilawari Distinguished Visiting Professor
  • “Genetics and Management of Familial Pancreatic Cancer” – Teresa Brentnall, MD
  • “Multidisciplinary Management of Borderline Resectable Pancreatic Cancer:”
    Endoscopic Evaluation – Mohammed Ismail, MD
    Cracking the Code on Chemotherapy for Pancreatic Cancer – Margaret Tempero, MD
    Radiation Therapy – Brian Czito, MD
    Surgical Strategies and Outcomes – Matthew Katz, MD
  • “Palliation in Pancreatic Cancer and End of Life Issues” – Clay Jackson, MD and Nia Zalamea, MD
  • “Debate: Management of Intraductal Papillary Mucinous Neoplasm
    in Young Patient: Partial vs Total Pancreatectomy” – Stephen Behrman, MD and Matthew Katz, MD

The University of Tennessee College of Medicine is accredited by the Accreditation Council for Continuing Medical Education to provide continuing medical education for physicians. The University of Tennessee College of Medicine designates this live activity for a maximum of 4.75 AMA PRA Category 1 Credits. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

 

ITS Continues Replacement of NAC with ISE Thursday, July 17th

Faculty & Staff,

On Thursday, July 17th, ITS will continue the replacement of the Cisco NAC with ISE (Identity Services Engine) in the Pharmacy Bldg & the RBL (Regional Biocontainment Laboratory) facility. We expect on the following Tuesday (7/22), additional buildings will be transitioned, to complete this project in all primary teaching facilities before the August semester starts. Please check the ISE rollout schedule at http://www.uthsc.edu/ise/schedule.php for a complete list of buildings & scheduled rollout dates.

Thanks in advance for your cooperation…

UTHSC Information Technology Services

 __________________________________________

ITS is replacing the NAC with ISE across the UTHSC campus.

We need your help to ensure a smooth transition on your building’s rollout date.

NOTE: ALL users must update their NAC agent & Sophos AntiVirus at www.uthsc.edu/ise

 Macintosh Users:      http://www.uthsc.edu/ise/macos.php

Wired-LAN Mac OS X desktop/laptop users will have to manually configure their machine. The instructions for configuring your computer vary depending on which version of the Mac OS X operating system you are running. Please enter the link below for your Mac OS version for further instructions.

Mac OSX 10.5.x     http://www.uthsc.edu/ise/docs/installOSX-V10.5.8.pdf
Mac OSX 10.6.x     http://www.uthsc.edu/ise/docs/installOSX-V10.6.8.pdf
Mac OSX 10.7.x     http://www.uthsc.edu/ise/docs/installOSX-V10.7-9.pdf
Mac OSX 10.8.x     http://www.uthsc.edu/ise/docs/installOSX-V10.7-9.pdf
Mac OSX 10.9.x     http://www.uthsc.edu/ise/docs/installOSX-V10.7-9.pdf

Windows desktop/laptop computers should be re-booted 1st thing in the morning on the day your building is switched over, then the user will be prompted to download and update the ISE agent. This updated agent is required for continued network access.

LINUX Users:      http://www.uthsc.edu/ise/linux.php

Complete LINUX instructions provided at the link above.

IF YOU HAVE QUESTIONS, PLEASE CALL THE HELP DESK AT #8-2222

Smith Presents to National Vaccine Advisory Committee

Dr. Cindy Smith, Assistant Professor of Clinical Pharmacy, from Reeves-Sain Pharmacy in Murfreesboro presented to the National Vaccine Advisory Committee in Washington, DC on June 11, 2014. This committee is advisory to the Assistant Secretary of Health of the US Public Service to assist in making decisions about the government’s role concerning vaccines. Cindy’s presentation was titled “Human Papilloma Virus Vaccine-The Pharmacists’ Perspective.” She was asked to help the committee understand how a practicing pharmacist deals with the HPV vaccine. She was listed on the agenda as representing the University of Tennessee. If you would like more information, please follow the NVAC link http://www.hhs.gov/nvpo/nvac/meetings/upcomingmeetings/060102014agenda.html

The University of Tennessee Health Science Center to Operate West Tennessee Regional Forensic Center

West Tennessee Regional Forensic Center

The West Tennessee Regional Forensic Center

The University of Tennessee Health Science Center (UTHSC) has been awarded a $3.1 million contract to operate the West Tennessee Regional Forensic Center (WTRFC) and the Shelby County Medical Examiner’s Office. [Read more...]

Joan C. Han, MD, Named Associate Professor in Department of Pediatrics at UTHSC and Founding Director for the UT-Le Bonheur Pediatric Obesity Center

Joan C. Han, MD

Joan C. Han, MD

David M. Stern, MD, executive dean of the College of Medicine at the University of Tennessee Health Science Center (UTHSC), and Meri Armour, CEO of Le Bonheur Children’s Hospital, have announced the joint appointment of pediatric endocrinologist Joan C. Han, MD. In July, Dr. Han will join UTHSC as associate professor in the Department of Pediatrics, Division of Endocrinology, and founding director of the new UT-Le Bonheur Pediatric Obesity Center. Dr. Han will also be director of the Pediatric Obesity Program on Le Bonheur’s clinical staff. [Read more...]