Top Stories - Translational Medicine

New TRI Facility in Downtown Orlando
Translational Research...

Florida Hospital and Sanford-Burnham announce the opening of the Florida Hospital –...

NeuroMap was founded by (left to right): Daniel Norton, Dr. Alexey Terskikh, Dr. Dmitriy Sivtsov and Dr. Andrew Rabinovich. (Photo by Sean M. Haffey, courtesy of San Diego Union-Tribune)
NeuroMap wins Entrepreneur...

Sanford-Burnham spin-out company NeuroMap, founded by Dr. Alexey Terskikh and his collaborators,...

Dr. Robert Wechsler-Reya
New insights into...

San Diego’s Rady Children’s Hospital recently brought together an all-star cast of speakers for...

Dr. Steven Smith and Dr. Stephen Gardell leave their marks on the new TRI building.
Building translational...

The  Translational Research Institute for Metabolism and Diabetes (TRI), a collaboration between...

Florida Department of Health and Sanford-Burnham to kick off collaborative research program

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Last week was a great one for medical researchers across the state of Florida. The state legislature and governor approved funding for the Collaborative Research Grant program between the Florida Department of Health and Sanford-Burnham Medical Research Institute. Starting in July, the program will provide scientists at universities and non-profit institutes throughout Florida with access to Sanford-Burnham scientists and our state-of-the-art technologies for drug discovery. This includes access to the Institute’s Conrad Prebys Center for Chemical Genomics.

Together with the Florida Department of Health, Sanford-Burnham will develop a competitive grant program, based on peer-review that will provide funds for collaborative projects between Florida-based research scientists and Sanford-Burnham’s fully operational, state-of-the-art drug discovery technology center based at Lake Nona.

Presidential advisor John P. Holdren visits Sanford-Burnham at Lake Nona

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We’re always thrilled to have public officials visit our facilities in California and Florida, but last Friday was an especially exciting day for scientists and staff at Sanford-Burnham’s Lake Nona campus in Orlando, Fla. Dr. John P. Holdren, advisor to President Barack Obama, toured Orlando’s Medical City and spent time at the Sanford-Burnham site to learn about the promising research that is being conducted in our Diabetes and Obesity Research Center.

Dr. Holdren is assistant to President Obama for science and technology, director of the White House Office of Science and Technology Policy (OSTP), and co-chair of the President’s Council of Advisors on Science and Technology (PCAST). Congress established the OSTP in 1976 to advise the President and others within the Executive Office of the President on the effects of science and technology on domestic and international affairs. The OSTP also makes recommendations on the annual NIH budget.

A “twisted” grand opening ceremony

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“My goal is to cure diabetes,” Steven Smith, M.D., scientific director of the Florida Hospital – Sanford-Burnham Translational Research Institute for Metabolism and Diabetes (TRI), said boldly at the opening ceremony of the TRI’s new state-of-the-art facility in downtown Orlando on March 27. “We believe that personalized medicine is our best shot at discovering cures for our most serious health problems like diabetes.”

The ceremony’s highlight was the unveiling of a spectacular nine-foot double-helix DNA structure that will be placed at the main entrance of the building, symbolizing the fundamental research being conducted at the TRI, as well as the synergies and collaborations the TRI represents. Selected board members and presenters each added one illuminated “bar,” representing a nucleotide, to the double helix.

“This is one of those rare times when the reality far exceeds the dream,” said John Reed, M.D., Ph.D., CEO of Sanford-Burnham. “The TRI is a wonderful opportunity for our organization, which will bring more and more to life our slogan From Research, the Power to Cure. We’re very excited about this opportunity to take our relationship with Florida Hospital to the next level.”

Translational Research Institute establishes new research paradigm for metabolic diseases

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Florida Hospital and Sanford-Burnham today celebrate the opening of the Florida Hospital – Sanford-Burnham Translational Research Institute for Metabolism and Diabetes’ (TRI) new state-of-the-art facility in downtown Orlando, Fla., dedicated to the advancement of a new paradigm of personalized approaches to researching and treating diabetes and obesity.

“We are witnessing the rise of personalized medicine, most notably in cancer. Our goal at the TRI is to accelerate the advancement of personalized medicine in diabetes and obesity,” said Steven Smith, M.D., Sanford-Burnham professor and scientific director of the TRI.  “We are working to rapidly expand knowledge of complex genetic and molecular causes of diabetes and obesity so that we can better define disease subpopulations. By working independently and in partnership with industry, we hope to develop therapies and treatment approaches tailored to those subpopulations. Our ultimate goal is that our discoveries will someday lead to cures for certain patients.”

Obesity research advances to clinical testing

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Discoveries made in the laboratories of Sanford-Burnham will, for the first time, advance to the clinical research stage involving human studies at the Florida Hospital – Sanford-Burnham Translational Research Institute for Metabolism and Diabetes (TRI). The research will focus on orexin, an appetite-inducing hormone produced in the brain, which appears to resolve obesity without requiring a reduction in food consumption or elevation in physical activity. This research exemplifies the translational research focus at Sanford-Burnham and the TRI – advancing science from laboratory bench to patient bedside. The studies will provide insight into individual responses and contribute to the development of personalized therapies for treating metabolic diseases – a focus area for both the TRI and Sanford-Burnham.

Appetite-suppressing drugs have traditionally been the basis of weight-loss treatments since obesity is thought to be caused by excessive energy intake and low physical activity. However, appetite suppressants can produce unacceptable side effects and, after the treatment ends, patients usually the weight they lost. Recent data indicate that orexin leads to weight loss by releasing excess energy as heat instead of storing it.

Taking stock: obesity research progress with Takeda

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Scientists from the Diabetes and Obesity Research Center and their colleagues from Florida Hospital recently returned from Japan, where they reviewed the progress that has been made at the mid-point of a research partnership with Takeda Pharmaceutical. The two-year collaboration focuses on the discovery and evaluation of new therapeutic approaches to obesity. In Japan, Sanford-Burnham scientists reported benchmark data that sets the stage for a key element in future drug development—the testing of obesity drug candidates.

“The data generated thus far lays the groundwork for analysis of how individuals respond differently to disease,” said Steven R. Smith, M.D., director of the Florida Hospital – Sanford-Burnham Translational Research Institute for Metabolism and Diabetes (TRI), where the clinical studies are being performed with volunteers. “This partnership with Takeda, TRI, and Sanford-Burnham establishes a model to accelerate the development of safe and effective therapies.”

Collaborating with Scripps Health to advance new cancer treatments

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Sanford-Burnham has announced a collaboration to advance cancer research and treatment. We are teaming up with Scripps Health to bridge the gap between laboratory scientists uncovering new approaches for treating cancer and the physicians caring for patients with cancer.

Basic medical research often struggles to achieve the financing and support to convert significant breakthroughs into medical practice. The field of translational medicine— aimed at “translating” scientific research into treatments for patients— has developed to help ensure promising early-stage discoveries don’t languish, but instead move into the drug development pipeline.

“San Diego is known for its scientific research and for its premier health care. This collaboration is the latest example of how Scripps and Sanford-Burnham are bringing the two together,” said Chris Van Gorder, president and CEO of Scripps Health.

Sanford-Burnham CEO meets with FDA Commissioner

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Sanford-Burnham CEO Dr. John Reed met with U.S. Food and Drug Administration (FDA) commissioner Dr. Margaret Hamburg last month at the FDA headquarters in Silver Spring, Maryland to discuss the FDA’s new initiatives in innovation. The meeting was a great opportunity to exchange ideas on how to make the process of reviewing new drug applications more effective and efficient—in other words, how to get more new medicines to the people who need them.

Dr. Hamburg was appointed as the 21st FDA commissioner by President Obama and assumed her new role in May 2009. She brings a fresh perspective to the FDA and a commitment to improving the organization’s performance. The FDA is the regulatory authority that determines how new medicines, diagnostics, and medical devices will be tested on humans. They set the performance and safety criteria required for commercialization in the United States.

The San Diego Foundation supports science

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At a time when scientists are having increasing difficulty acquiring financial support from federal sources, alternative sources of funding are becoming more important for maintaining the momentum of critical research at universities, research institutes and even industrial laboratories. At Sanford-Burnham, research assistant professor Dr. Kazuki N. Sugahara was recently awarded a one-year, $75,000 grant from The San Diego Foundation, via the The Blasker-Rose-Miah Fund. This marks one of the few times that a Sanford-Burnham investigator has received funding from this source, underscoring the novelty and importance of the project. This key piece of local funding will allow Dr. Sugahara to continue his research on the use of tissue-penetrating peptides that can detect developing tumors and enhance the delivery of cancer therapeutic drugs.

NeuroMap wins Entrepreneur Challenge

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By Peijean Tsai

When a person is diagnosed with depression, pinpointing the right treatment is typically a trial-and-error process that frustrates both doctors and patients.  Chronic symptoms interrupt everyday life while the patient seeks an effective remedy.

To address this challenge, NeuroMap, an early-stage company, is developing assays using induced pluripotent stem cells (iPSCs) to accurately predict how individuals with major depressive disorder (MDD) will respond on a personal level to medications, such as selective serotonin re-uptake inhibitors (SSRIs), the most commonly prescribed antidepressants.

“Some will have to go for months or years to find the right drug, and that’s what we’re trying to eliminate,” says Sanford-Burnham’s Dr. Alexey Terskikh, who founded NeuroMap with Dr. Dmitriy Sivtsov, a psychiatrist at the University of California, San Diego (UCSD) School of Medicine, computer scientist Dr. Andrew Rabinovich and Daniel Norton of UCSD’s Rady School of Management.

This novel concept – personalized  depression therapeutics based on Sanford-Burnham technology – is what catapulted NeuroMap to win first prize earlier this month at the 5th Annual UCSD Entrepreneur Challenge’s Business Plan Competition, one of three contests the organization holds each year. The competition was judged by professionals from San Diego’s technology and entrepreneurial communities and presented before a public audience. The honor also awarded the startup company $57,000 in cash and entrepreneurial services, which Dr. Terskikh says will help move the company forward with its efforts to secure funding from government and private sources.

My moment with Corinna

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This video of three year-old Corinna is remarkable because it would seem so unremarkable if you didn’t know Corinna’s story—she looks just like any other kid. I had the pleasure of meeting this very special attendee at Sanford-Burnham’s 2nd Annual Rare Disease Day Symposium last February. Corinna was born with hypophosphatasia (HPP), a rare inherited disease that affects bone development, leaving her fragile and unable to walk. Lauren, Corinna’s mother, had brought her along to the symposium and was excited to meet the scientists studying HPP and hear about the latest research.

The family traveled from Philadelphia to be there that day – no small task, considering Corinna’s special needs and lack of mobility. I first talked to Lauren on the phone, helping her with directions from her hotel to the Sanford-Burnham campus. Then I waited for them outside and helped her get the stroller out of the taxi. I set it up while Lauren got Corinna out of the car. Corinna was blond, adorable, friendly—and just about the same size as my own daughter. My heart went out to them. I know how trying it can be to travel with a toddler, even under the best of circumstances.

Building translational research

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The  Translational Research Institute for Metabolism and Diabetes (TRI), a collaboration between Sanford-Burnham and Florida Hospital, is one step closer to opening its research facility. The 54,000 square-foot building recently reached its final height, and a special ceremony was held during which researchers gathered to sign one of the interior columns. Construction to enclose the building will now begin.The TRI, which studies diabetes, obesity and the metabolic origins of cardiovascular disease, will  help bridge the gap between the scientist’s laboratory and the patient’s bedside. The TRI will unite scientists, clinicians and advanced technologies to spur translational research and rapidly create new, more effective treatments.

“We are witnessing an epidemic of obesity and diabetes in the United States,” said Dr. Steven R. Smith, scientific director of the TRI. “The main goal of the TRI is to generate new knowledge to improve lives through innovative research. By ‘topping out’ the TRI, we are one step closer towards developing Orlando as a medical destination.”

Where will new medicines come from?

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In December 2010, the National Institutes of Health (NIH) announced a plan to create a new center, known as the National Center for Advancing Translational Sciences (NCATS), to speed development and testing of novel diagnostics and therapeutics aimed at a wide range of human diseases. In a recent opinion piece published in Genetic Engineering & Biotechnology News (GEN), Dr. Henry I. Miller, policy fellow at Stanford University’s Hoover Institution, argues against NCATS, claiming that drug development is better left in the hands of private pharmaceutical and biotechnology companies.In a rebuttal published online May 13, Sanford-Burnham CEO Dr. John Reed picks apart Dr. Miller’s argument, explaining why the public sector must participate in the search for new medicines and how NCATS will further catalyze these efforts. According to Dr. Reed, private companies are increasingly reluctant to fund the crucial early stages of pre-clinical development – the research necessary to “translate” promising discoveries made in laboratories into potential therapeutics ready for testing in human clinical trials. He writes:

“This situation leaves us with the aptly named “Valley of Death” – the large research and funding gap that sets federally-funded basic researchers (those of us in non-profit research institutions, academia, hospitals and federal laboratories) on one side and the pharmaceutical industry on the other. Few companies are able to reach far enough backward to bridge that gap – the costs and risks are just too high for organizations that are responsible for delivering financial results to their investors and shareholders. Enter the NIH’s newly proposed center for translational sciences – just the shot in the arm basic research needs to reach forward across that valley.”

For more, read “NCATS Could Mitigate Pharma Valley of Death“, Genetic Engineering & Biotechnology News, online May 13, in print May 15, 2011.

On the Cutting Edge

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On April 12, Dr. Evan Snyder, who directs the Stem Cells and Regenerative Biology program at Sanford-Burnham, was interviewed by Shally Zomorodi of Fox 5 News about recent advances in stem cell research. Dr. Snyder singled out four different areas where researchers are making great progress: diseases in a dish; using stem cells to protect other cells; recreating organs for transplant and using stem cells to treat diseased tissues or cancers (particularly in the brain) with targeted gene therapy. Dr. Snyder noted that all these approaches are fairly advanced.

Filling the Drug Pipeline

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On March 27, Sanford-Burnham’s chief business officer, Dr. Paul Laikind, appeared on BioCentury This Week, along with venture capitalist Brian Atwood, to examine how a major downturn in research and development spending is affecting drug development. Specifically, they discussed how pharmaceutical companies and venture capitalists are investing less in early stage development, potentially starving the pipeline for new drugs. Dr. Laikind noted that Sanford-Burnham is working to help fill this research gap:

“We are doing cutting-edge science, that’s always what we’ve been focused on,” said Dr. Laikind. “What we’ve done in the last five to ten years has invested significantly in the translational part of the equation. Not to become a pharmaceutical company…but to be able to push it [the science] further down the pipeline so that we can do collaborations…work with venture capitalists and work with big pharma to take projects farther forward.”

Watch R & D Goes Flatline: Part II to learn more about this burgeoning crisis and potential solutions.

Related Beaker content:
Academia Jumps into Drug Discovery
The Promise of Chemical Genomics
Laboratory to Pharmacy
From Research, the Power to Cure