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Top Stories - Drug Discovery

New TRI Facility in Downtown Orlando
Translational Research...

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

In Sanford-Burnham’s Conrad Prebys Center for Chemical Genomics, scientists use robots like this one to search hundreds of thousands of chemical compounds for the few that might become tomorrow’s new medicines.
Collaborating with Pfizer to...

Sanford-Burnham is the latest research organization to partner with Pfizer, Inc. as part of...

Michael Jackson, Ph.D., vice president of drug discovery and development in Sanford-Burnham's Conrad Prebys Center for Chemical Genomics (photo by Mark Dastrup)
Collaborating with Mayo Clinic...

Researchers at the two organizations will work together to identify and validate...

The Florida Translational Research Program provides Florida-based scientists with access to Sanford-Burnham's drug-discovery technology and expertise.
First five organizations...

Sanford-Burnham at Lake Nona announced today the selection of the first five research organizations...

NIH director Francis Collins calls for continued funding of basic medical research

by Patrick Bartosch on September 5, 2012 at 5:51 am | 0 Comments
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Dr. Francis Collins makes a compelling case for continued funding of basic medical research (Photo by National Institutes of Health)

Dr. Francis Collins makes a compelling case for continued funding of basic medical research (Photo by National Institutes of Health)

In an editorial for Science, Dr. Francis Collins, director of the U.S. National Institutes of Health (NIH), makes a convincing case for continued funding of basic medical research. In the editorial, Dr. Collins writes that the NIH will continue to support basic research, which it defines as systematic study directed toward fuller knowledge or understanding of the fundamental aspects of phenomena and of observable facts without specific applications in mind. According to the article:

“In this time of severe budget constraints, Americans need to know that today’s basic research is the engine that powers tomorrow’s therapeutic discoveries,” says Dr. Francis Collins. “They need to know that basic research is the type of science that the private sector, which requires rapid returns on investment, cannot afford to fund. They need to know that, because it is impossible to predict whence the next treatment may emerge, the nation must support a broad portfolio of basic research.”

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Xconomy: the state of Sanford-Burnham’s drug discovery pipeline

by Patrick Bartosch on September 4, 2012 at 6:35 am | 0 Comments
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JohnReed1

Earlier this month, Dr. John Reed, CEO and the Donald Bren Chief Executive Chair at Sanford-Burnham, spoke with Bruce Bigelow of Xconomy—a network of biotechnology-related blogs, events, and other initiatives—about how our drug discovery capabilities and the Institute as a whole have developed in the decade since he became CEO. The article points out that Dr. Reed led the development and implementation of a 10-year plan to extend the Institute’s work beyond basic research, with a focus on discovering and developing new drug candidates. Today, Sanford-Burnham identifies between two and four compounds each year that are considered valid clinical candidates.

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Researchers find new anti-malarial drug target

by admin on July 20, 2012 at 1:16 pm | 0 Comments
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Ring forms of the Plasmodium falciparum (malaria) parasite, inside red blood cells (Image by Michael Zahniser)

Ring forms of the Plasmodium falciparum (malaria) parasite, inside red blood cells (Image by Michael Zahniser)

An international team of scientists, including researchers at the University of California, San Diego (UCSD) and Sanford-Burnham Medical Research Institute, have identified the first reported inhibitors of a key enzyme involved in survival of the parasite responsible for malaria. Their findings, which may provide the basis for anti-malarial drug development, were published July 19 in the Journal of Medicinal Chemistry.

According to the World Health Organization, there were 216 million cases of malaria worldwide in 2010. Severe forms of the disease are mainly caused by the parasite Plasmodium falciparum, transmitted to humans by female Anopheles mosquitoes. Malaria eradication has not been possible due to the lack of vaccines and the parasite’s ability to develop resistance to most drugs.

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Congresswoman Sandy Adams visits Sanford-Burnham’s Lake Nona campus in Orlando

by Patrick Bartosch on July 11, 2012 at 5:29 am | 0 Comments
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U.S. Congresswoman Sandy Adams holding a 5-pound model of human fat cells

U.S. Congresswoman Sandy Adams holding a 5-pound model of human fat cells

We hosted a special visitor last week – U.S. Representative Sandra (Sandy) Adams, of Florida’s 24th Congressional District, where our Lake Nona, Orlando, campus is located. The congresswoman was elected to the U.S. House of Representatives in 2010 and will run again for the newly redrawn 7th District this year. She took a tour of the facility and spoke to Drs. Steve Gardell, Phil Wood, and Sheila Collins about the exciting research taking place in Lake Nona.

Sanford-Burnham’s vice president of government affairs, Elizabeth Gianini, welcomed the congresswoman to the Institute and Dr. Gardell took her on a tour of the drug discovery suite, which includes the Conrad Prebys Center for Chemical Genomics, the Pharmacology Core, and the Medicinal Chemistry Core, explaining the Institute’s role in finding targets that could aid in the development of new therapeutics. Dr. Wood walked her through his lab and spoke about his team’s research into metabolic syndrome and type 2 diabetes. Rep. Adams then met with Dr. Collins, who explained her research into brown fat cells and the role of heart hormones in fat metabolism.

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Breathing new life into old medicines

by Patrick Bartosch on May 8, 2012 at 10:24 am | 0 Comments
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High-throughput drug screening platforms in Sanford-Burnham's Conrad Prebys Center for Chemical Genomics will be used to screen existing drugs for new applications.

High-throughput drug screening platforms in Sanford-Burnham's Conrad Prebys Center for Chemical Genomics will be used to screen existing drugs for new applications.

Discovering and developing new treatments for disease is a challenging, time-consuming, and expensive endeavor. For every drug that eventually makes it to the pharmacy, hundreds of compounds fail to deliver results and millions of dollars are spent without a direct return on investment. However, in these economically challenging times, existing drugs and compounds—whether in development, already on the market, or even ones that have failed clinical trials due to lack of efficacy—are being re-examined by pharmaceutical companies and research institutions. The goal of this approach—called drug repurposing— is to find potential new uses for these drugs.

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

by Patrick Bartosch on April 25, 2012 at 1:30 pm | 0 Comments
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The Collaborative Research Grant program will provide Florida scientists with access to our screening center

The Collaborative Research Grant program will provide Florida scientists with access to our screening center

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.

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Drug discovery case study: high-throughput screening of TNAP

by Patrick Bartosch on April 11, 2012 at 11:52 am | 0 Comments
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Presence of calcium deposits in a mouse aorta, as revealed by alizarin red staining.

Presence of calcium deposits in a mouse aorta, as revealed by alizarin red staining.

Editor’s note: this is the second in a series of posts highlighting drug screening studies in our Conrad Prebys Center for Chemical Genomics. Read the first post here.

Calcification of the medial layer of arteries is increasingly recognized as an important clinical problem. Medial vascular calcification (MVC) is the major cause of morbidity and mortality in generalized arterial calcification of infancy (GACI), and contributes to cardiovascular deterioration in Kawasaki disease (KD), chronic kidney disease (CKD), as well as in diabetes, obesity, and aging. MVC is thought to result from decreased circulating levels of the mineralization inhibitor, inorganic pyrophosphate (PPi).

Researchers at Sanford-Burnham have revealed that the development of MVC in mouse and rat models is accompanied by up-regulation of tissue-nonspecific alkaline phosphatase (TNAP), an enzyme whose primary function is to hydrolyze PPi, and thus, crucial in determining where mineralization occurs. Preliminary data have proven that upregulation of TNAP is sufficient to cause MVC and Sanford-Burnham scientists have developed potent drug-like inhibitors of TNAP.

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Drug discovery case study: invadopodia and cancer metastasis

by Patrick Bartosch on April 9, 2012 at 11:21 am | 0 Comments
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Metastatic cancer cells form invadopodia, shown here as bright red spots. (Image by Begoña Díaz)

Metastatic cancer cells form invadopodia, shown here as bright red spots. (Image by Begoña Díaz)

Editor’s note: this is the first in a series of posts highlighting drug screening studies in our Conrad Prebys Center for Chemical Genomics. Read the second post here.

To metastasize, some types of cancer cells rely on invadopodia, cellular membrane projections that help them “walk” away from the primary tumor. To determine how cells control invadopodia formation, scientists at Sanford-Burnham took advantage of the technology and expertise of the Institute’s Conrad Prebys Center for Chemical Genomics to screen a collection of pharmacologically active compounds to identify those that either promote or inhibit the process.

The study identified several compounds that block invadopodia and found that many of the compounds targeted Cdks, a family of enzymes that were not previously associated with invadopodia. One of these enzymes, Cdk5, is required for the formation and function of invadopodia and for cellular invasion.

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

by Patrick Bartosch on April 4, 2012 at 2:13 pm | 0 Comments
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Dr. John P. Holdren tours Sanford-Burnham's Lake Nona site

Dr. John P. Holdren tours Sanford-Burnham's Lake Nona site

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.

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A “twisted” grand opening ceremony

by Patrick Bartosch on March 29, 2012 at 3:35 pm | 0 Comments
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TRI grand opening speakers (from left to right): John Reed, Terry Owen, Dan Kelly, Steve Smith, Lars Houmann, Des Cummings, Don Jernigan

TRI grand opening speakers (from left to right): John Reed, Terry Owen, Dan Kelly, Steve Smith, Lars Houmann, Des Cummings, Don Jernigan

“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.”

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Translational Research Institute establishes new research paradigm for metabolic diseases

by Patrick Bartosch on March 27, 2012 at 6:00 am | 0 Comments
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New TRI Facility in Downtown Orlando

New TRI Facility in Downtown Orlando

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.”

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Obesity research advances to clinical testing

by Patrick Bartosch on March 6, 2012 at 6:00 am | 8 Comments
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Brown Fat Cells

Orexin targets brown fat cells

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.

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Why the economy depends on federal funding for medical research

by Patrick Bartosch on February 22, 2012 at 3:03 pm | 0 Comments
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NIH Funding

NIH funding is crucial for medical research

When Sanford-Burnham CEO John Reed, M.D., Ph.D. traveled to Washington, D.C., in early February, he attended a variety of Capitol Hill briefings to discuss the importance of National Institutes of Health (NIH) funding for medical research. He pointed out that NIH grants account for approximately 80 percent of all funding for non-profit medical research institutions in the United States, such as Sanford-Burnham.

NIH grants contribute to the ultimate goal of developing new treatments for diseases and improving the quality of life for millions of Americans and people worldwide. The research supported by these grants also generates U.S. patents that fuel the biotechnology industry and creates thousands of jobs across the nation. NIH funding supports the training of our biomedical research workforce and strengthens the foundation of a 21st century knowledge-based economy.

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Talking research in Washington, D.C.

by Kristina Meek on February 17, 2012 at 4:22 pm | 0 Comments
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Dr. John Reed with Daniel Webster, Congressman from Florida's 8th District

Dr. John Reed with Daniel Webster, Congressman from Florida's 8th District

On February 2, Sanford-Burnham CEO Dr. John Reed traveled to Washington D.C., where he described at several Capitol Hill briefings how important NIH funding is to the life sciences sector and, in turn, to America’s economy. He and Scott Salka, CEO of CendR Inc., a biotech start-up that spun out of Sanford-Burnham in 2010, participated as part of CONNECT’s “Innovation 101” series of Capitol Hill Briefings on life sciences research.

CONNECT, a regional program that catalyzes the creation of innovative technology and life sciences products and companies in San Diego County, described the significance of this undertaking by saying, “As life science research institutions increase their focus on commercialization of discoveries and develop strategies to help start-up companies succeed, it is imperative that Congress and the Obama administration understand how federal research funding results in successful discoveries, start-ups, and job creation.”

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Cancer drug discovery leaders come together at Sanford-Burnham

by Heather Buschman, Ph.D. on February 16, 2012 at 4:52 pm | 0 Comments
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Kristiina Vuori, M.D., Ph.D., Institute president and director of Sanford-Burnham's NCI-designated Cancer Center (center), with NCI-CBC leadership and participants

Kristiina Vuori, M.D., Ph.D., Institute president and director of Sanford-Burnham's NCI-designated Cancer Center (center), with NCI-CBC leadership and participants

Where do new medicines come from? The first step in the drug discovery process often involves screening small molecules (chemicals) to determine their potential to produce innovative biological research tools. Sanford-Burnham’s Conrad Prebys Center for Chemical Genomics uses robotic technology to sift through chemical compounds by the millions to find the few that could potentially be developed into new medicines

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