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Category Archives: Molecular Medicine

Bio-inspired Materials Give Boost to Regenerative Medicine – Bioscience Technology

Posted: August 22, 2017 at 5:43 am

What if one day, we could teach our bodies to self-heal like a lizards tail, and make severe injury or disease no more threatening than a paper cut?

Or heal tissues by coaxing cells to multiply, repair or replace damaged regions in loved ones whose lives have been ravaged by stroke, Alzheimers or Parkinsons disease?

Such is the vision, promise and excitement in the burgeoning field of regenerative medicine, now a major ASU initiative to boost 21st-century medical research discoveries.

ASU Biodesign Institute researcher Nick Stephanopoulos is one of several rising stars in regenerative medicine. In 2015, Stephanopoulos, along with Alex Green and Jeremy Mills, were recruited to the Biodesign Institutes Center for Molecular Design and Biomimetics (CMDB), directed by Hao Yan, a world-recognized leader in nanotechnology.

One of the things that that attracted me most to the ASU and the Biodesign CMDB was Haos vision to build a group of researchers that use biological molecules and design principles to make new materials that can mimic, and one day surpass, the most complex functions of biology, Stephanopoulos said.

I have always been fascinated by using biological building blocks like proteins, peptides and DNA to construct self-assembled structures, devices and materials, and the interdisciplinary and highly collaborative team in the CMDB is the ideal place to put this vision into practice.

Yans research center uses DNA and other basic building blocks to build their nanotechnology structures only at a scale 1,000 times smaller than the width of a human hair.

Theyve already used nanotechnology to build containers to specially deliver drugs to tissues, build robots to navigate a maze or nanowires for electronics.

To build a manufacturing industry at that tiny scale, their bricks and mortar use a colorful assortment of molecular Legos. Just combine the ingredients, and these building blocks can self-assemble in a seemingly infinite number of ways only limited by the laws of chemistry and physics and the creative imaginations of these budding nano-architects.

Learning from nature

The goal of the Center for Molecular Design and Biomimetics is to usenatures design rulesas an inspiration in advancing biomedical, energy and electronics innovation throughself-assembling moleculesto create intelligent materials for better component control and for synthesis intohigher-order systems, said Yan, who also holds the Milton Glick Chair in Chemistry and Biochemistry.

Prior to joining ASU, Stephanopoulos trained with experts in biological nanomaterials, obtaining his doctorate with the University of California Berkeleys Matthew Francis, and completed postdoctoral studies with Samuel Stupp at Northwestern University. At Northwestern, he was part of a team that developed a new category of quilt-like, self-assembling peptide and peptide-DNA biomaterials for regenerative medicine, with an emphasis in neural tissue engineering.

Weve learned from nature many of the rules behind materials that can self-assemble. Some of the most elegant complex and adaptable examples of self-assembly are found in biological systems, Stephanopoulos said.

Because they are built from the ground-up using molecules found in nature, these materials are also biocompatible and biodegradable, opening up brand-new vistas for regenerative medicine.

Stephanopoulos tool kit includes using proteins, peptides, lipids and nucleic acids like DNA that have a rich biological lexicon of self-assembly.

DNA possesses great potential for the construction of self-assembled biomaterials due to its highly programmable nature; any two strands of DNA can be coaxed to assemble to make nanoscale constructs and devices with exquisite precision and complexity, Stephanopoulos said.

Proof all in the design

During his time at Northwestern, Stephanopoulos worked on a number of projects and developed proof-of-concept technologies for spinal cord injury, bone regeneration and nanomaterials to guide stem cell differentiation.

Now, more recently, in a new studyin Nature Communications, Stephanopoulos and his colleague Ronit Freeman in the Stupp laboratory successfully demonstrated the ability to dynamically control the environment around stem cells, to guide their behavior in new and powerful ways.

In the new technology, materials are first chemically decorated with different strands of DNA, each with a unique code for a different signal to cells.

To activate signals within the cells, soluble molecules containing complementary DNA strands are coupled to short protein fragments, called peptides, and added to the material to create DNA double helices displaying the signal.

By adding a few drops of the DNA-peptide mixture, the material effectively gives a green light to stem cells to reproduce and generate more cells. In order to dynamically tune the signal presentation, the surface is exposed to a soluble single-stranded DNA molecule designed to grab the signal-containing strand of the duplex and form a new DNA double helix, displacing the old signal from the surface.

This new duplex can then be washed away, turning the signal off. To turn the signal back on, all that is needed is to now introduce a new copy of single-stranded DNA bearing a signal that will reattach to the materials surface.

One of the findings of this work is the possibility of using the synthetic material to signal neural stem cells to proliferate, then at a specific time selected by the scientist, trigger their differentiation into neurons for a while, before returning the stem cells to a proliferative state on demand.

One potential use of the new technology to manipulate cells could help cure a patient with neurodegenerative conditions like Parkinsons disease.

The patients own skin cells could be converted to stem cells using existing techniques. The new technology could help expand the newly converted stem cells back in the lab and then direct their growth into specific dopamine-producing neurons before transplantation back to the patient.

People would love to have cell therapies that utilize stem cells derived from their own bodies to regenerate tissue, Stupp said. In principle, this will eventually be possible, but one needs procedures that are effective at expanding and differentiating cells in order to do so. Our technology does that.

In the future, it might be possible to perform this process entirely within the body. The stem cells would be implanted in the clinic, encapsulated in the type of material described in the new work, and injected into a particular spot. Then the soluble peptide-DNA molecules would be given to the patient to bind to the material and manipulate the proliferation and differentiation of transplanted cells.

Scaling the barriers

One of the future challenges in this area will be to develop materials that can respond better to external stimuli and reconfigure their physical or chemical properties accordingly.

Biological systems are complex, and treating injury or disease will in many cases necessitate a material that can mimic the complex spatiotemporal dynamics of the tissues they are used to treat, Stephanopoulos said.

It is likely that hybrid systems that combine multiple chemical elements will be necessary; some components may provide structure, others biological signaling and yet others a switchable element to imbue dynamic ability to the material.

A second challenge, and opportunity, for regenerative medicine lies in creating nanostructures that can organize material across multiple length scales. Biological systems themselves are hierarchically organized: from molecules to cells to tissues, and up to entire organisms.

Consider that for all of us, life starts simple, with just a single cell. By the time we reach adulthood, every adult human body is its own universe of cells, with recent estimates of 37 trillion or so. The human brain alone has 100 billion cells or about the same number of cells as stars in the Milky Way galaxy.

But over the course of a life, or by disease, whole constellations of cells are lost due to the ravages of time or the genetic blueprints going awry.

Collaborative DNA

To overcome these obstacles, much more research funding and recruitment of additional talent to ASU will be needed to build the necessary regenerative medicine workforce.

Last year, Stephanopoulos research received a boost with funding from the U.S. Air Forces Young Investigator Research Program (YIP).

The Air Force Office of Scientific ResearchYIP award will facilitate Nicks research agenda in this direction, and is a significant recognition of his creativity and track record at the early stage of his careers, Yan said.

Theyll need this and more to meet the ultimate challenge in the development of self-assembled biomaterials and translation to clinical applications.

Buoyed by the funding, during the next research steps, Stephanopoulos wants to further expand horizons with collaborations from other ASU colleagues to take his research teams efforts one step closer to the clinic.

ASU and the Biodesign Institute also offer world-class researchers in engineering, physics and biology for collaborations, not to mention close ties with the Mayo Clinic or a number of Phoenix-area institutes so we can translate our materials to medically relevant applications, Stephanopoulos said.

There is growing recognition that regenerative medicine in the Valley could be a win-win for the area, in delivering new cures to patients and building, person by person, a brand-new medicinal manufacturing industry.

Stephanopoulos recent research was carried out at Stupps Northwesterns Simpson Querrey Institute for BioNanotechnology. The National Institute of Dental and Craniofacial Research of the National Institutes of Health (grant 5R01DE015920) provided funding for biological experiments, and the U.S. Department of Energy, Office of Science, Basic Energy Sciences provided funding for the development of the new materials (grants DE-FG01-00ER45810 and DE-SC0000989 supporting an Energy Frontiers Research Center on Bio-Inspired Energy Science (CBES)).

The paper is titled Instructing cells with programmable peptide DNA hybrids. Samuel I. Stupp is the senior author of the paper, and post-doctoral fellows Ronit Freeman and Nicholas Stephanopoulos are primary authors.

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Molecular Medicine Research – Wake Forest School of Medicine

Posted: August 17, 2017 at 3:46 am

The Section on Molecular Medicinefocuses on performing cutting-edge research in cellular and molecularmechanisms of human disease and supports graduate and postgraduate leveleducational programs within the Department of Internal Medicine. The Sectionserves as the administrative home for the largest PhD graduate program(Molecular Medicine and Translational Science) in the Biomedical Sciences atWake Forest University and an NIH-sponsored institutional predoctoral trainingprogram (T-32) in Integrative Lipid Sciences, Inflammation, and ChronicDiseases.

A major goal of the section is toserve as a nidus for translational research by providing an environment whereclinical and basic science faculty interact to make new discoveries and toeducate future scientists.

The section consists of ten (10) primary faculty members and one (1) Emeritus faculty member who use cellular and molecular approaches to gain abetter understanding of the basic mechanisms underlying several chronic humanconditions including: asthma, atherosclerosis, hepatosteatosis, obesity andinsulin resistance, autoimmunity, and age-related pathology (arthritis,Alzheimers disease).

A particular research focus isthe role of inflammation in the pathogenesis of acute and chronic humandiseases. Faculty research strengths are in areas of cell signaling, cellbiology, proteomics, regulation of gene expression, and the use of genetically-modifiedmouse models of human disease. The research in the section is supported bygrants from the NIH, from the Department of Defense, from foundations including the Avon Foundation and theAmerican Heart Association, and from partnerships with industry.

The section also provides acenter for laboratory research training and education in translational researchfor medical students, residents, and postdoctoral fellows includingsubspecialty fellows in the Department of Internal Medicine. A seminar seriesis held weekly in conjunction with the graduate program in Molecular Medicineand Translation Science.

John S. Parks, PhDProfessor of Internal Medicine, Biochemistry, and Translational ScienceChief, Section on Molecular Medicine

Molecular Medicine Journal Club

Faculty News

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MS in Molecular Medicine – Drexel University College of …

Posted: August 17, 2017 at 3:46 am

The Master of Science in Molecular Medicine (MMED) program provides training in the academic, research and entrepreneurial aspects of the biomedical sciences with an emphasis on translational research in the development of therapeutics and vaccines.

Participation in the program will provide enhanced educational credentials through a flexible curriculum, with most classes offered in the early evening to maximize accessibility. Classes can be attended at two Drexel University College of Medicine locations: Center City and Queen Lane Campuses in Philadelphia. State-of-the-art videoconferencing provides real-time interactive learning at both locations.The program now can also be completed online, with all required courses and many elective courses available.

The Master of Science in Molecular Medicine program is designed to provide academic and practical biotechnological knowledge in translational research, particularly in the areas of molecular therapeutics and vaccine development.

If you prefer an online learning experience, you can still earn a Drexel master's degree in the field of molecular medicine. The online Master of Science in Molecular Medicine program features the same curriculum, flexibility, course content, and instructors as the traditional, face-to-face degree program.

Learn more about the online Master of Science in Molecular Medicine program!

In addition to broad geographic access, the curriculum provides flexibility in content and course load. Most students will complete the program in two years through completion of required courses and electives selected from two menus: research theory and laboratory research. The research experience can be in an academic environment or a company setting, as best fits the individual student's goals and interests.Some students may opt to complete the program on a part-time basis, taking up to four years. In either sequence, no dissertation is required. Program directors and course faculty will work closely with each student to best achieve his or her specific goals.

Learn more about the curriculum

The molecular medicine program is ideally suited for enhancing the scientific credentials of the following groups:

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Spotlight Innovation Enters into Sponsored Research Agreement with Indiana University to Develop New Therapies for … – Markets Insider

Posted: August 17, 2017 at 3:46 am

URBANDALE, Iowa, Aug. 16, 2017 /PRNewswire/ --Spotlight Innovation Inc. (OTCQB: STLT) today announced that the Company has entered into a Sponsored Research Agreement with Indiana University to support research directed by Elliot Androphy, M.D., aimed at developing safe and effective drugs to treat patients with spinal muscular atrophy (SMA). Dr. Androphy is a member of Spotlight Innovation's Scientific Advisory Board and a co-inventor of STL-182, the Company's lead product candidate for SMA.

Geoffrey Laff, Ph.D., Spotlight Innovation's Senior Vice President of Business Development, commented, "Dr. Androphy is a prolific researcher and highly-respected thought leader. We are privileged to work with him to develop novel therapies for SMA."

Dr. Androphy is the Chair of the Department of Dermatology of Indiana University School of Medicine and has published widely in high-impact journals including Science, Nature, EMBO Molecular Medicine, Human Molecular Genetics, Journal of Virology, and Molecular Cell. He served as Vice Chair for Research of the Department of Medicine and Director of the M.D./Ph.D. Program at the University of Massachusetts Medical School where his lab characterized the disease-causing mechanism of alternative splicing of the SMN2 gene. At Indiana University School of Medicine, Dr. Androphy has used a novel, cell-based high throughput screen for compounds that increase levels of the SMN protein. This work has led to the identification of pre-clinical drug candidates for SMA.

About Spotlight Innovation Inc.

Spotlight Innovation Inc. (OTCQB: STLT) identifies and acquires rights to innovative, proprietary technologies designed to address unmet medical needs, with an emphasis on rare, emerging and neglected diseases. To find and evaluate unique opportunities, we leverage our extensive relationships with leading scientists, academic institutions and other sources. We provide value-added development capability to accelerate development progress. Whenscientifically significantbenchmarkshave been achieved, we will endeavor to partner with proven market leaders via sale, out-license or strategic alliance. For more information, visit http://www.spotlightinnovation.com or follow us on http://www.twitter.com/spotlightinno.

Forward-Looking Statements

Statements in this press release that are not purely historical are forward-looking statements. Forward-looking statements herein include statements regarding Spotlight Innovation's efforts to develop and commercialize various product candidates, including STL-182, and to achieve its stated benchmarks. Actual outcomes and actual results could differ materially from those in such forward-looking statements. Factors that could cause actual results to differ materially include risks and uncertainties, such as: the inability to finance the planned development of STL-182; the inability to hire appropriate staff to develop STL-182; unforeseen technical difficulties in developing STL-182; the inability to obtain regulatory approval for human use; competitors' therapies proving to be more effective, cheaper or otherwise more preferable; or, the inability to market a product. All of which could, among other things, delay or prevent product release, as well as other factors expressed from time to time in Spotlight Innovation's periodic filings with the Securities and Exchange Commission (SEC). As a result, this press release should be read in conjunction with Spotlight Innovation's periodic filings with the SEC. The forward-looking statements contained herein are made only as of the date of this press release and Spotlight Innovation undertakes no obligation to publicly update such forward-looking statements to reflect subsequent events or circumstances.

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Master of Science (MSc) in Molecular Medicine – NTNU

Posted: August 17, 2017 at 3:46 am

International students are encouraged to attend NTNU'sOrientation Week14 - 20 August 2017.

OnMonday 21 August at 10:15there will be a welcome meeting for all new master's students at the Faculty of Medicine and Health Sciences. This meeting takes place in auditorium KA12 in theKnowledge Centre at Campus ya.

After the welcome meeting (Monday 21 August at 12:00)there will be an orientation meeting for the MSc in Molecular Medicine. This meeting takes place in room Ls42 in the Laboratory Centre at Campus ya. It iscompulsory to attendthis meeting.

The field of molecular medicine is often referred to as "tomorrow's medicine". It aims to provide a molecular understanding of how normal cellular processes change, fail or are destroyed by disease. The purpose of the MSc programme is to develop knowledge and skills in cellular and molecular biology. These have applications in both research and practical clinical work, and will contribute to an increased understanding of processes, diagnostics and treatment of diseases.

The application deadline for for applicants from non-EU/non-EEA students is 1 December. The application deadline for students from EU/EEA countries is 1 March. You submit your application electronically.

The MSc in Molecular Medicine qualifies graduates for a wide range of careers, including practical clinical work and technical executive positions in hospital laboratories, and positions in pharmaceuticals and MedTech/BioTech companies.

The MSc is a two-year, full-time programme starting in the autumn semester. There are two main components: a master's thesis worth 60 credits, and theoretical and methodological courses totalling a further 60 credits.

Contact one of our student counsellors if you have any questions about the MSc programme. Email: lbk-post@medisin.ntnu.no / Telephone: +47 72 82 07 00

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A New Method of 3D Printing Living Tissues – Technology Networks

Posted: August 17, 2017 at 3:46 am

Scientists at the University of Oxford have developed a new method to 3D-print laboratory-grown cells to form living structures.

The approach could revolutionise regenerative medicine, enabling the production of complex tissues and cartilage that would potentially support, repair or augment diseased and damaged areas of the body.

Printing high-resolution living tissues is hard to do, as the cells often move within printed structures and can collapse on themselves. But, led by Professor Hagan Bayley, Professor of Chemical Biology in Oxfords Department of Chemistry, the team devised a way to produce tissues in self-contained cells that support the structures to keep their shape.

The cells were contained within protective nanolitre droplets wrapped in a lipid coating that could be assembled, layer-by-layer, into living structures. Producing printed tissues in this way improves the survival rate of the individual cells, and allowed the team to improve on current techniques by building each tissue one drop at a time to a more favourable resolution.

To be useful, artificial tissues need to be able to mimic the behaviours and functions of the human body. The method enables the fabrication of patterned cellular constructs, which, once fully grown, mimic or potentially enhance natural tissues.

Dr Alexander Graham, lead author and 3D Bioprinting Scientist at OxSyBio (Oxford Synthetic Biology), said: We were aiming to fabricate three-dimensional living tissues that could display the basic behaviours and physiology found in natural organisms. To date, there are limited examples of printed tissues, which have the complex cellular architecture of native tissues. Hence, we focused on designing a high-resolution cell printing platform, from relatively inexpensive components, that could be used to reproducibly produce artificial tissues with appropriate complexity from a range of cells including stem cells.

The researchers hope that, with further development, the materials could have a wide impact on healthcare worldwide. Potential applications include shaping reproducible human tissue models that could take away the need for clinical animal testing.

Over the coming months they will work to develop new complementary printing techniques, that allow the use of a wider range of living and hybrid materials, to produce tissues at industrial scale. Dr Sam Olof, Chief Technology Officer at OxSyBio, said: There are many potential applications for bioprinting and we believe it will be possible to create personalised treatments by using cells sourced from patients to mimic or enhance natural tissue function. In the future, 3D bio-printed tissues maybe also be used for diagnostic applications for example, for drug or toxin screening.

Dr Adam Perriman from the University of Bristols School of Cellular and Molecular Medicine, added: The bioprinting approach developed with Oxford University is very exciting, as the cellular constructs can be printed efficiently at extremely high resolution with very little waste. The ability to 3D print with adult stem cells and still have them differentiate was remarkable, and really shows the potential of this new methodology to impact regenerative medicine globally.

This article has been republished frommaterialsprovided by the University of Oxford. Note: material may have been edited for length and content. For further information, please contact the cited source.

Reference:

Graham, A. D., Olof, S. N., Burke, M. J., Armstrong, J. P., Mikhailova, E. A., Nicholson, J. G., . . . Bayley, H. (2017). High-Resolution Patterned Cellular Constructs by Droplet-Based 3D Printing. Scientific Reports, 7(1). doi:10.1038/s41598-017-06358-x

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Cancer Treatment Centers of America and Foundation Medicine Join Forces to Advance Precision Cancer Treatment – Markets Insider

Posted: August 17, 2017 at 3:46 am

CAMBRIDGE, Mass., Aug. 15, 2017 /PRNewswire/ --Cancer Treatment Centers of America (CTCA) and Foundation Medicine today announced a new element to their longstanding partnership to increase awareness of advancements in genomic testing and precision medicine in oncology. The educational initiative directed toward physicians, other caregivers and patients will highlight the importance of integrating comprehensive genomic testing of solid tumors early in an individual's care plan as a model to inform personalized care and improve clinical outcomes for individuals with cancer.

"Precision cancer treatment using advanced genomic testing is changing the science of cancer care," said Maurie Markman, M.D., President of Medicine & Science at CTCA. "As oncologists, we have an obligation to the patients we serve to keep pace with, and, whenever possible, lead the way in the application of the latest diagnostic tools that may help inform treatment decisions. Our partnership with Foundation Medicine empowers our physicians to customize treatment plans according to the individual patient's clinical profile right down to the molecular level, and therefore furnish care in a much more comprehensive and effective manner."

The partnership brings together CTCA, a national network of five cancer treatment hospitals at the forefront of delivering precision cancer treatment to address individual patients' unique treatment needs, and Foundation Medicine, a leader in molecular information that offers a suite of comprehensive genomic profiling (CGP) assays that identifies the molecular alterations in an individual's cancer and matches them with potentially relevant targeted therapies, including immunotherapies.

Through their shared patient-centered philosophy, CTCA and Foundation Medicine will educate the medical community about the successful approach CTCA is using to incorporate FoundationOne for solid tumors into clinical care. Specifically, the educational initiative will feature several patients with cancer, chronicling each person's journey from cancer diagnosis to tumor profiling to treatment. Through this case-based approach, the program aims to provide insights into precision medicine treatment approaches based on an individual's unique cancer, including the selection of targeted therapies, appropriate clinical trials and responses to immunotherapy.

"Precision medicine, and a move to a more personalized, targeted approach to cancer care, is becoming ever more ubiquitous as the published data continues to validate this approach as leading to better clinical outcomes for patients," said Vincent Miller, M.D., Chief Medical Officer for Foundation Medicine. "As such, it's critical that every stakeholder in a patient's care planphysician, patient and care teamis knowledgeable about the benefits of genomic profiling, and importantly, that they have the right tools at the ready to implement such an approach. We applaud CTCA leadership in this area and we're delighted to collaborate with them on this educational initiative."

To learn more about genomics and precision cancer treatment, visit cancercenter.com. To learn more about genomic testing and FoundationOne, visit FoundationMedicine.com.

About Cancer Treatment Centers of AmericaCancer Treatment Centers of America Global, Inc. (CTCA), headquartered in Boca Raton, Fla., is a national network of five hospitals that serves adult patients who are fighting cancer. CTCA offers an integrative approach to care that combines advancements in genomic testing and precision cancer treatment, surgery, radiation, immunotherapy and chemotherapy, with evidence-informed supportive therapies designed to help patients physically and emotionally by enhancing their quality of life while managing side effects both during and after treatment. CTCA serves patients from around the world at its hospitals in Atlanta, Chicago, Philadelphia, Phoenix and Tulsa. Reflecting our patient-centered approach to cancer care, our patient satisfaction scores consistently rank among the highest in the country for cancer care providers, and CTCA is also rated one of the most admired hospital systems in the country in national consumer surveys. For more information, visit cancercenter.com, Facebook.com/cancercenter and Twitter.com/cancercenter.

About Foundation MedicineFoundation Medicine(NASDAQ:FMI) is a molecular information company dedicated to a transformation in cancer care in which treatment is informed by a deep understanding of the genomic changes that contribute to each patient's unique cancer. The company offers a full suite of comprehensive genomic profiling assays to identify the molecular alterations in a patient's cancer and match them with relevant targeted therapies, immunotherapies and clinical trials.Foundation Medicine'smolecular information platform aims to improve day-to-day care for patients by serving the needs of clinicians, academic researchers and drug developers to help advance the science of molecular medicine in cancer. For more information, please visithttp://www.FoundationMedicine.comor followFoundation Medicineon Twitter (@FoundationATCG). Foundation Medicineand FoundationOne are registered trademarks ofFoundation Medicine, Inc.

Cautionary Note Regarding Forward-Looking StatementsThis press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding the objectives of any educational initiatives between CTCA and Foundation Medicine; the importance of integrating comprehensive genomic testing of solid tumors early in an individual's care plan to improve clinical outcomes for individuals with cancer; and the value and performance capabilities of Foundation Medicine's comprehensive genomic profiling assays. All such forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include the risk thateducational initiatives are not developed or launched in the anticipated manner; Foundation Medicine'sCGP andservices will not be able to identify genomic alterations in the same manner as prior clinical data or prior experience; and the risks described under the caption "Risk Factors" inFoundation Medicine'sAnnual Report on Form 10-K for the year endedDecember 31, 2016, which is on file with theSecurities and Exchange Commission, as well as other risks detailed inFoundation Medicine'ssubsequent filings with theSecurities and Exchange Commission.All information in this press release is as of the date of the release, andFoundation Medicineundertakes no duty to update this information unless required by law.

Contact: Michael MyersCancer Treatment Centers of America rel="nofollow">michael.myers@ctca-hope.com 561-923-3179

Lee-Ann MurphyFoundation Medicine 617-245-3077 rel="nofollow">pr@foundationmedicine.com

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What can genetic testing really tell you? – Popular Science

Posted: August 17, 2017 at 3:46 am

Once difficult and expensive even for the most technologically advanced labs, genetic testing is fast becoming a cheap and easy consumer product. With a little spit and 200 dollars, you can find out your risk for everything from cystic fibrosis to lactose intolerance.

But its important to remember that not all genetic tests are created equal. And even the best clinical genetic test, carried out in a medical lab under a doctor's supervision, isn't perfectgenes are important, but they don't seal your fate.

Genetic tests are diagnostic, so anyone who is curious about their health can get one done. But they're more informative if you think you might be at risk for a genetic disorder.

Heavy-duty genetic tests have been used as a clinical tool for almost half a centurylong before 23andMe and Ancestry.com began offering direct-to-consumer tests. Lets say that many women in your family have had breast cancer. You can get a genetic test to see if you may have inherited an abnormal version of the BRCA gene, known to increase your risk for breast cancer.

Heidi Rehm, associate professor of pathology at Harvard Medical School, is the director of the Laboratory for Molecular Medicine, where patients get tested for diseases that can be traced to specific genetic roots. She says it is most common for people to get tested when they either suspect or know that they have a genetic disease; it may have affected multiple people in their family or they could show symptoms of something widely known to be genetic, like sickle cell anemia. For these people, genetic tests can provide a much-needed explanation for an illness and help doctors determine the best course of treatment. Babies are often tested for genetic diseases, either while they are still fetuses or shortly after birth.

Others get genetic tests if they and their partner both have family histories of an inherited diseaseeven if they dont have the disease themselves. For example, cystic fibrosis is linked to one particular gene, but you have to inherit the abnormal version of the gene from both your parents to get the disease. If you only inherit one copy, you may never knowyou wont display any of the symptoms. But if you and your partner both carry one copy of the faulty gene, your child could still inherit two copies. Genetic tests can forewarn you of that possibility.

But Rehm says there has been a recent trend of healthy people getting tested to predict whether theyll get certain diseases. I do think there are settings where predictive genetic testing is incredibly important and useful, Rehm says; for example, knowing that youre at risk for breast cancer gives you the opportunity for early intervention (remember when Angelina Jolie got a double mastectomy upon finding out she had a mutated BRCA gene?)

But Rehm also points out that genetic tests may not be as straightforward as they seem. For example, some genes are thought to increase risk of getting a certain disease, but it might only happen if you have specific family history, or you might be able to reduce your risk with lifestyle changes. So remember that a genetic test isnt the final verdictthere are other factors at play too.

Not entirelyits scope is limited. For starters, not all diseases are caused by genes. Plenty of conditions stem from environmental and lifestyle factors; they may interact with your genes, but the external factors are the real trigger.

But even if a disease is caused solely by faulty instructions written in your genes, you wont necessarily be able to test for it. Thats because genetic tests are mainly used for diseases that are penetrant, a term that scientists use to describe a strong connection between having a certain gene (or multiple genes) and getting a disease.

Genetic tests are surprisingly simple on the surface. All thats required of you is a small sample of cells, like a blood sample or saliva (which doesnt have DNA itself, but picks up cheek cells during its journey out of your mouth). It get sent to a lab where sequencing machines match up small pieces of synthetic DNA with your DNA to figure out the overall sequence.

Once they have your sequence, geneticists can compare it with "normal" or disease-causing sequences. In the end, they might give you a yes or no answer, or sometimes youll get a probabilitya measure of how much your genes increase your risk of developing the disease. Then, its up to your doctor to figure out what these genes (in combination with your lifestyle, family history and other risk factors) mean for your health.

With penetrant diseases, theres a very, very high ability to explain the disease, Rehm says. For example, the breast cancer-related gene BRCA1 can give you a 60 percent chance of getting breast cancer (in Jolies case, with her family history, the risk was 87 percent.)

This makes genetic tests better at detecting so-called rare diseases, says Steven Schrodi, associate research scientist at the Marshfield Clinic Research Institutes Center for Human Genetics, but theyre less useful when it comes to more common diseases, like heart disease or diabetes. Genetics can increase your likelihood of getting these disease, but scientists still dont know quite how much. Part of the problem is that there may be dozens or hundreds of genes responsible for these diseases, Schrodi says.

We have an incomplete understanding of why people get diseases, Schrodi says. A large part of it hinges on how we define diseases. Perhaps physicians have inadvertently combined multiple diseases together into a single entity.

Consumer genetic teststhe ones where you send in samples from homesometimes claim to test for these more complex traits, but be careful: Their results might not be very medically relevant, Rehm says. If they tell you that your genes make you twice as likely to develop diabetes, for example, that's a marginal increase that doesn't significantly affect your risk, especially when you take into account lifestyle factors.

Genes do seem to play a role in determining lifespan. After all, some family reunions stretch from great-great-grandparents all the way down to infants. Scientists have studied centenarianspeople who lived to be 100 years oldand found that people with certain versions of genes involved in repairing DNA tend to live longer.

This makes sense because aging leaves its mark on your DNA. Environmental factors can damage DNA, and even the routine chore of replicating cells can introduce errors as the three billion units of your DNA are copied over and over. Long-lived individuals have different sequences that seem to make their cells better at keeping DNA in mint condition.

But figuring out your expiration date is more complex than just testing for a few genes, says Jan Vijg, professor of genetics at Albert Einstein College of Medicine. In theory, you could design a test that looks at specific genes that might measure your risk for developing Alzheimers Disease or other age-related diseases, or your risk for aging quickly. To some extent, yes: Biomarkers will tell you something about your chances of living a long life, Vijg says. Still, that will only work if you live a careful life. And that means no accidents, infections, or cancers.

Aging also affects the exposed ends of your DNA, called "telomeres." DNA is stored as chromosomes, those X-like structures that you may have seen in biology textbooks. The most vulnerable parts of the chromosome are the chromosomes tips, which get shorter as you age because they arent properly replicated. But while telomere length might let you compare your DNA now with your DNA from a decade ago, you cant compare your own telomeres with other peoples telomeres. Theres a lot of variation between individuals, Vijg says. Some of us are just old souls (on the genomic level, that is.)

The methylation test, which looks at how the presence of small chemical groups attached to your DNA changes as you age, might be a better bet. A study at UCLA showed that changes were slower in longer-lived people. But Vijg is hesitant: I would not put my hopes on that as a marker to predict when exactly youre going to die.

For now, just enjoy your life, because you cant predict death. And if you decide to unlock the secrets of your DNA with an at-home test, don't take those results for more than their worth.

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What can genetic testing really tell you? - Popular Science

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Spotlight Innovation Enters into Sponsored Research Agreement with Indiana University to Develop New Therapies for … – PR Newswire (press release)

Posted: August 16, 2017 at 2:42 pm

Geoffrey Laff, Ph.D., Spotlight Innovation's Senior Vice President of Business Development, commented, "Dr. Androphy is a prolific researcher and highly-respected thought leader. We are privileged to work with him to develop novel therapies for SMA."

Dr. Androphy is the Chair of the Department of Dermatology of Indiana University School of Medicine and has published widely in high-impact journals including Science, Nature, EMBO Molecular Medicine, Human Molecular Genetics, Journal of Virology, and Molecular Cell. He served as Vice Chair for Research of the Department of Medicine and Director of the M.D./Ph.D. Program at the University of Massachusetts Medical School where his lab characterized the disease-causing mechanism of alternative splicing of the SMN2 gene. At Indiana University School of Medicine, Dr. Androphy has used a novel, cell-based high throughput screen for compounds that increase levels of the SMN protein. This work has led to the identification of pre-clinical drug candidates for SMA.

About Spotlight Innovation Inc.

Spotlight Innovation Inc. (OTCQB: STLT) identifies and acquires rights to innovative, proprietary technologies designed to address unmet medical needs, with an emphasis on rare, emerging and neglected diseases. To find and evaluate unique opportunities, we leverage our extensive relationships with leading scientists, academic institutions and other sources. We provide value-added development capability to accelerate development progress. Whenscientifically significantbenchmarkshave been achieved, we will endeavor to partner with proven market leaders via sale, out-license or strategic alliance. For more information, visit http://www.spotlightinnovation.com or follow us on http://www.twitter.com/spotlightinno.

Forward-Looking Statements

Statements in this press release that are not purely historical are forward-looking statements. Forward-looking statements herein include statements regarding Spotlight Innovation's efforts to develop and commercialize various product candidates, including STL-182, and to achieve its stated benchmarks. Actual outcomes and actual results could differ materially from those in such forward-looking statements. Factors that could cause actual results to differ materially include risks and uncertainties, such as: the inability to finance the planned development of STL-182; the inability to hire appropriate staff to develop STL-182; unforeseen technical difficulties in developing STL-182; the inability to obtain regulatory approval for human use; competitors' therapies proving to be more effective, cheaper or otherwise more preferable; or, the inability to market a product. All of which could, among other things, delay or prevent product release, as well as other factors expressed from time to time in Spotlight Innovation's periodic filings with the Securities and Exchange Commission (SEC). As a result, this press release should be read in conjunction with Spotlight Innovation's periodic filings with the SEC. The forward-looking statements contained herein are made only as of the date of this press release and Spotlight Innovation undertakes no obligation to publicly update such forward-looking statements to reflect subsequent events or circumstances.

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Spotlight Innovation Enters into Sponsored Research Agreement with Indiana University to Develop New Therapies for ... - PR Newswire (press release)

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Yenepoya University to offer biotech skill enhancement programme – Hindu Business Line

Posted: August 16, 2017 at 2:42 pm

Mangaluru, August 16:

The Centre for Systems Biology and Molecular Medicine at Yenepoya University in Mangaluru has been awarded the Biotechnology Skill Enhancement Programme (BiSEP) by the Karnataka Biotechnology and Information Technology Services (KBITS).

Addressing presspersons in Mangaluru on Wednesday, T.S. Keshava Prasad, Deputy Director of the Centre for Systems Biology and Molecular Medicine, said the centre has been awarded the BiSEP to conduct a one-year postgraduate diploma in multiomics technology. (Multiomics is an interdisciplinary subject that includes genomics, proteomics, metabolomics and proteogenomics.)

He said Yenepoya University is the only centre to offer BiSEP in multiomics technology. The centre has facilities and experts in this technology to undertake such a training programme.

Candidates for BiSEP - postgraduate diploma programme - will be selected based on their performance in the Karnataka Biotechnology Aptitude Test to be held in September. Students enrolled in the programme will be provided fellowship of Rs 10,000 a month during the course.

He said 50 per cent of the tuition fee for Karnataka students will be paid by the state government.

Students will undergo a six-month hands-on training programme in different omics platforms at the Centre for Systems Biology and Molecular Medicine. This will be followed by a six-month internship.

He said graduates and postgraduates in the field of life sciences would be equipped with necessary employable skills under BiSEP. This will help make them industry-ready in the field of genomic, proteomic and metabolomic technologies. This programme will enable supply of skilled manpower required by multinational biotechnology and pharmaceutical companies, he added.

(This article was published on August 16, 2017)

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Yenepoya University to offer biotech skill enhancement programme - Hindu Business Line

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