- Wayne State technology licensed by RetroSense Therapeutics gets green light for orphan drug designation for retinitis pigmentosa treatment
In Headlines on October 30, 2014
Zhuo-Hua Pan, Ph.D.
Technology to restore vision through the use of a component of green algae developed by Zhuo-Hua Pan, Ph.D., scientific director of the Ligon Research Center of Vision at the Kresge Eye Institute, the Wayne State University School of Medicine's Edward T. and Ellen K. Dryer Endowed Professor in Vision and Blindness Research in the Department of Ophthalmology, and professor of anatomy and cell biology, was granted Orphan Drug designation for the treatment of retinitis pigmentosa, or RP, by the United States Food and Drug Administration. RP, a genetic condition, leads to the progressive degeneration of rod and cone photoreceptors in the retina and results in severe vision loss and blindness. Currently there are no FDA-approved drugs to treat the disease.
RetroSense Therapeutics, LLC, a privately-held biopharmaceutical company, is developing RST-001 as a first-in-class gene therapy application of optogenetics – a means of conferring light sensitivity to cells that were not previously, or natively light sensitive – designed to restore vision to those affected by RP.
By applying optogenetics to retinas in which rod and cone photoreceptors have degenerated, RetroSense is conferring new light sensitivity to the retina, with the expectation of improved or restored vision. RST-001 is expected to have application to all forms of RP, independent of causative gene or mutation.
“We are pleased that the FDA has granted Orphan Drug status to our lead product, RST-001,” said Sean Ainsworth, CEO of Retrosense Therapeutics. “This significant milestone will enable us to continue to develop new and innovative treatments for retinitis pigmentosa, a truly debilitating condition. We are hopeful that the benefits associated with Orphan Drug status will better enable us to advance RST-001 through development and ultimately into the marketplace where it may benefit many who are suffering from blindness due to retinitis pigmentosa.”
The FDA Office of Orphan Products Development supports the evaluation and development of products that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions. The FDA awards Orphan Drug designation as an incentive to develop drugs and biological therapeutics for diseases that affect fewer than 200,000 people in the United States. The benefits of Orphan Drug designation include a seven-year period of market exclusivity following FDA approval, certain tax credits for clinical testing expenses conducted after orphan designation is received, and reduced regulatory fees.
“RetroSense’s Orphan Drug designation from the FDA is a major step forward in the development of RST-001,” said Joan Dunbar, associate vice president for technology commercialization at Wayne State University. “Sean Ainsworth has dedicated years of extensive efforts to champion this technology to the next level, and because of his motivation and the extensive research of Dr. Pan and his research team, we are closer to a potential treatment for retinitis pigmentosa.”
- Retina study results will enhance, speed treatment decisions for eye diseases
In Headlines on October 29, 2014
Bruce Berkowitz, Ph.D.
Wayne State University School of Medicine scientists have discovered a new way to determine two important aspects of retinal health.
Professor of Anatomy and Cell Biology and of Ophthalmology Bruce Berkowitz, Ph.D., who is working to prevent vision loss and blindness, is the principal investigator of a study published in the Federation of American Societies for Experimental Biology journal outlining a test that measures how well retina blood vessels and photoreceptors -- the specialized retinal cells that detect light -- are working.
“Before this study, it was not possible to measure these two parameters together in living animals,” Dr. Berkowitz said.
The approach could lead to new possibilities for understanding the molecular changes that occur in retinal disease and evaluating the benefits of treatment earlier in the disease course.
“We believe these findings will enhance and speed decisions about treatment in patients with specific diseases of the eye. This, in turn, may slow the progression of such diseases and help save sight in patients,” he added.
“Development of an MRI Biomarker Sensitive to Tetrameric Visual Arrestin 1 and its Reduction via Light-Evoked Translocation In Vivo” was published in the FASEB Journal online.
The WSU research team, in collaboration with scientists at Vanderbilt University, Washington University and the University of Southern California, spent two years taking and comparing photos of the retina in dark and light. They saw that the front of the retina responded differently from the back of the retina.
“We hypothesized that this difference in behavior to light and dark was due to changes in blood flow in the front of the retina and photoreceptor metabolism in the back of the retina. This hypothesis was confirmed by comparing normal mice to mice whose retinas were genetically altered,” Dr. Berkowitz said.
The new MRI technology takes a molecular photo of the retina and other tissues. The technology can potentially be applied to patients, and will help doctors understand diseases that affect patients with certain genetic defects that can cause loss of sight, such as Oguchi’s disease, an inherited form of Retinitis Pigmentosa linked to night blindness, Dr. Berkowitz said. The study also is expected to help improve ongoing clinical trials of genetic treatment of diseases like Leber’s congenital amaurosis, or LCA, the first disease successfully treated with gene therapy.The project was supported by grants from the National Institutes of Health Animal Models of Diabetic Complications Consortium and Mouse Metabolic Phenotyping Centers Pilot and Feasibility Programs; Research to Prevent Blindness; National Eye Institute (EY015851, EY011500, EY021126 and EY019312); Department of Ophthalmology and Visual Sciences at Washington University (EY002687) and the Mary D. Allen Foundation.
- Dr. Tsveti Markova named chair of WSU Family Medicine and Public Health Sciences
In Headlines on October 28, 2014
Tsveti Markova, M.D.
Tsveti Markova, M.D., F.A.A.F.P., associate dean of Graduate Medical Education for the Wayne State University School of Medicine, has been appointed chair of the WSU Department of Family Medicine and Public Health Sciences effective immediately.
“She has proven to be a strong leader, and due to her outstanding performance and leadership the faculty recommended to me that Dr. Markova be appointed chair,” Dean Valerie M. Parisi, M.D., M.P.H., M.B.A., said in announcing the appointment Oct. 27. “The school’s executive committee of the faculty senate and the university administration supported this decision.”
Dr. Markova had been serving as interim chair of the department since late March.
“It is such an honor being selected to lead the department, nationally renowned for its innovative approaches to medical education, unique focus on public and environmental health, community integration, as well as its diverse research portfolio,” Dr. Markova said. “I am impressed by the commitment of leadership, faculty and staff to maintain the high standards of excellence, and culture of respect, trust and support.”
A member of the School of Medicine faculty since 1997, Dr. Markova received her medical degree from the Medical University of Varna in Bulgaria in 1992. She completed an internship at the University Hospital Varna Medical Center, followed by a family medicine residency at North Oakland Medical Centers in Pontiac, Mich., in 1996. She completed two fellowships, with the National Institute for Program Directors Development with the American Academy of Family Physicians, with the Hedwig van Ameringen Executive Leadership in Academic Medicine Program for Women. She was a scholar in the Program for Leading Innovations in Healthcare and Education at the Harvard Macy Institute in 2013.
“We will collectively reevaluate our strategic initiatives, aligned with the school of medicine and university priorities,” Dr. Markova said of her goals for the department, which include expanding and enhancing research capacity, achieving successful maintenance of Council on Education of Public Health accreditation for the master’s of public health degree program and better integrating educational programs throughout the educational continuum.
“To respond to the rapidly-changing health care environment, we will also focus on expanding our clinical presence, providing high-quality, efficient and effective patient care,” she said. “All of these goals will be enhanced by strengthening our partnerships with other schools, departments, centers and community organizations.”
In addition to receiving many teaching awards from the Department of Family Medicine and Public Health Sciences and the School of Medicine, Dr. Markova was the recipient of the 2008 Michigan Academy of Family Physicians’ Educator of the Year Award for outstanding contributions to undergraduate, graduate and continuing education. She received the prestigious 2011 Parker J. Palmer Courage to Teach Award from the Accreditation Council for Graduate Medical Education for exemplary teaching of residents and leadership of innovative and effective residency programs.
Named associate dean for Graduate Medical Education/Designated Institutional Official for Wayne State University sole-sponsored residency programs in March 2011, she will continue to oversee that office.
Dr. Markova has been actively engaged at the national, state and regional levels through the Association of American Medical Colleges Group on Resident Affairs, the Alliance of Independent Academic Medical Centers, Michigan Academy of Family Physicians and Southeast Michigan Center for Medical Education, among others. She has written more than 50 peer-reviewed publications on medical education innovation, learning environment and teamwork, quality improvement and health care disparities.
- 2014 Golden Gala: The Roaring Twenties photo gallery available
In Headlines on October 24, 2014
- Dr. Cote captures Komen grant to cross train breast cancer researchers
In Headlines on October 24, 2014
Michelle Cote, Ph.D., M.P.H.
Michele Cote, Ph.D., M.P.H., associate professor of oncology for the Wayne State University School of Medicine and member of the Population Studies and Disparities Research Program at the Barbara Ann Karmanos Cancer Institute, has received a three-year, $404,997 Graduate Training in Disparities Research Grant from Susan G. Komen®. This is Dr. Cote’s second Komen grant.
Her co-principal investigator is Manohar Ratnam, Ph.D., professor of oncology for the School of Medicine, faculty member of the WSU Cancer Biology Graduate Program and member of the Molecular Therapeutics Program of the Karmanos Cancer Institute. Other faculty members of the Cancer Biology Graduate Program will serve as research mentors.
A component of the program includes a Komen survivor advocate, who will help trainees recognize the impact of their work on women. Katrina Studvent, manager of Breast Cancer Special Programs at Karmanos and Komen Detroit Race co-chair, will serve as the Komen advocate.
Dr. Cote will lead a training program for exemplary doctoral students that will cross train them in basic science and epidemiology. The program will recruit three students from the 2015-2016 academic year and a second set of three students in the third year of the program. Students will be recruited from Wayne State University, as well as from universities across Michigan and the United States.
“We are extremely excited to receive this Komen funding so that we can train student researchers to examine breast cancer and the reasons for differences in diagnosis and survival between women with the disease,” Dr. Cote said. “By providing a solid foundation consisting of cellular and molecular biology, epidemiology and laboratory research, our trainees will be uniquely prepared to carry out original investigations in contemporary breast cancer disparities research.”
Komen GDTR grants are intended to establish and sustain training programs for graduate students seeking careers dedicated to understanding and eliminating disparities in breast cancer. By providing this type of funding, Komen seeks to build a diverse pool of highly-trained scientists who will emerge as the next generation of leaders in the field of breast cancer research focused on reducing disparities.
“WSU has been highly successful recruiting students from various backgrounds, including underserved minority populations, and providing tailored mentoring to ensure they graduate from our program with the skills necessary to become highly sought-after scientists in academia and industry,” Dr. Cote said. “We expect that this program will attract ambitious, high-caliber students who will seek ways to reduce breast cancer incidence and mortality.”
Dr. Cote’s grant is one of more than 50 grants provided to early-career breast cancer researchers, accounting for almost half of Komen’s $34.7 million investment in new breast cancer research funding for 2014. The grants include more than $980,000 in new funding for research conducted in Michigan, bringing Komen’s total research investment in the state to $25.2 million since 1982.
Susan G. Komen’s research program is funded in part by contributions from Komen affiliates across the country. Those affiliates annually contribute 25 percent of net funds raised locally to Komen’s research program with the remaining 75 percent funding breast cancer community outreach programs. Karmanos Cancer Institute is the local presenting sponsor of the Susan G. Komen Detroit Race for the Cure, which has raised and invested nearly $27 million since the first Komen Detroit Race in 1992.
“We’re very proud that funds we’ve raised in metro Detroit are not only providing real-time help to our neighbors, but coming back to our universities and hospitals for research that can save lives,” said Maureen Keenan Meldrum, director of Breast Cancer Special Programs at Karmanos and leader of Komen operations in the tri-county area.
Registration is open for the May 16, 2015, Race at Chene Park: http://www.karmanoscancer.org/KomenDetroit/Default.aspx.
Community health programs funded by Komen Detroit can be found here: http://www.karmanoscancer.org/KomenDetroit/SubPage.aspx?id=2147485363
- Dr. Khan publishes first comprehensive paper on MS in minority populations
In Headlines on October 24, 2014
Omar Khan, M.D.
Omar Khan, M.D., professor and chair of neurology for the Wayne State University School of Medicine, is the lead author of the first comprehensive publication that encompasses the clinical, genetic, imaging and therapeutic investigations related to multiple sclerosis in minority populations in the United States.
The paper, “Multiple Sclerosis in U.S. Minority Populations: Clinical Practice Insights,” is scheduled to be published in the November issue of Neurology: Clinical Practice, an official journal of the American Academy of Neurology, the world’s largest organization of neurologists.
Classically considered to be a disease of young women of European heritage, multiple sclerosis is being increasingly recognized in populations not previously thought to develop the condition. This body of work has been led largely by research conducted by Dr. Khan, director of the Wayne State University Multiple Sclerosis Center, and leading several national multi-center investigations focused on African-American multiple sclerosis.
“The highlights of this important publication include several facts that deserve careful deliberation,” Dr. Khan said. “Minority populations in the United States, such as African-Americans and Hispanic-Americans, are often underrepresented in clinical research and trials, and thus it is difficult to assess treatment response in these populations. A distinct humoral immune response seems to be characteristic of African-Americans rather than Caucasians with MS, and African-Americans acquire greater gray matter injury and have a more aggressive disease course than Caucasians.”
Additional findings revealed in the paper include the fact that access and delivery of health care need to be carefully addressed in minority populations with MS so that they are not predisposed to greater disability.
“At Wayne State University, we are humbled to have established a deep bond with the community, and today, with more than 700 African-Americans with MS in our center, this is the largest African-American MS clinic in the United States,” Dr. Khan said. “This also puts us in a unique position to serve our community and advance scientific knowledge that may improve outcomes.”
For example, Dr. Khan’s imaging laboratory team showed greater CSF B-cell mediated cerebral gray matter injury in African-Americans than Caucasians with MS, one of the reasons a humanized monoclonal antibody targeting B cells is being pursued as a new approach to treat MS.
Similarly, the laboratory of Robert Lisak, M.D., professor of neurology, has reported a possible B cell secreting protein that may damage myelin forming cells in the brain.
“These types of research endeavors are classic examples of translational work that ends with a single unifying theme: improving patient outcomes,” Dr. Khan said.
The Wayne State University Multiple Sclerosis Center has collaborated with the several national institutions in the clinical, magnetic resonance imaging and genetic studies of MS in African-Americans, including the University of California San Francisco, the Massachusetts Institute of Technology and Harvard Medical School. These collaborations have been supported by multiple continuing grants from the National Institute of Neurological Disorders and Stroke and have led to more than a dozen major publications in journals such as Nature, the New England Journal of Medicine, Nature Genetics, Annals of Neurology, Neurology and Science. Most recently, the Wayne State University MS Center presented work on retinal structure injury that is significantly greater in African-Americans than Caucasians with MS. This work was presented by research investigator Jessica Chorostecki at the recently concluded American-European International Multiple Sclerosis Meeting, in Boston in September, where she was awarded the young investigators award. The meeting is the world’s largest MS meeting, attended by more than 9,000 clinicians and researchers.
“We are uniquely positioned to take serve our urban, ethnically diverse population, and the body of work we have published on African-American MS, to seek federal grants in the future,” Dr. Khan said