Translational Medicine

Partnering with Asia’s largest pharmaceutical company to find obesity treatments

by Patrick Bartosch on February 11, 2013 at 11:00 pm | 0 Comments

Drs. Smith (TRI), Izumo (Takeda), and Kelly (Sanford-Burnham; left to right) in traditional Japanese coats at the signing ceremony for the renewed partnership

We held a special signing ceremony today at our Lake Nona, Orlando, campus to renew our research agreement with Florida Hospital and Takeda Pharmaceutical Company Limited. The renewal extends our collaboration to discover and evaluate new therapeutic approaches to obesity. The collaboration uses a research and drug-development model that creates an early feedback loop in the discovery pipeline. We and our partners expect this model will shorten the time to develop new therapeutics.

Interest in the development of obesity treatments remains strong, as the regulatory approval of two new obesity therapeutics in 2012 offered a proven pathway for drug candidates. “As the worldwide obesity crisis continues to escalate, we are seeing a rise in the prevalence of severe obesity—defined by BMI greater than 40—and we know that this subset of the obese population experiences increased mortality and associated diseases, such as heart disease, diabetes and cancer,” said Steven R. Smith, M.D., scientific director of the Florida Hospital – Sanford-Burnham Translational Research Institute for Metabolism and Diabetes (TRI) and president-elect of The Obesity Society. “These statistics are staggering and clearly demonstrate the need to rapidly develop treatment strategies for obesity.”

Cancer drug targets hard-to-reach leukemia stem cells

by Heather Buschman, Ph.D. on January 18, 2013 at 5:31 am | 0 Comments

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Potential cancer drug sabutoclax blocks Bcl-2 protein family members that help keep cancer cells alive. This image shows the structure of one Bcl-2 protein, known as Bcl-Xl. (Image courtesy of the Pellecchia laboratory)

Researchers find that certain types of drug-resistant leukemia stem cells are vulnerable to sabutoclax, a novel cancer stem cell-targeting drug based on Sanford-Burnham research.

New experiments show that sabutoclax, a novel cancer stem cell-targeting drug that grew out of research at Sanford-Burnham Medical Research Institute, in combination with other therapies, could effectively treat diseases like chronic myeloid leukemia (CML). Sabutoclax might also lower the chance of relapse.

“The demonstration of sabutoclax’s preclinical activity in mouse models of CML is exciting and encourages further evaluation of this promising drug candidate for aggressive leukemias. We look forward to continuing our collaborative studies of sabutoclax, as we move this drug closer to the clinic,” said John Reed, M.D., Ph.D., professor and Donald Bren Chief Executive Chair at Sanford-Burnham.

Sabutoclax was first discovered as a result of research in the laboratories of Reed and his Sanford-Burnham colleague, Maurizio Pellecchia, Ph.D. The pair is now working with biotechnology company Oncothyreon Inc to develop sabutoclax into a potential anti-cancer drug. This latest study of sabutoclax’s efficacy, published January 17 in the journal Cell Stem Cell, was led by Catriona Jamieson, M.D., Ph.D., at UC San Diego Moores Cancer Center, in collaboration with Reed, Pellecchia and others.

Sanford-Burnham and Intrexon Corporation establish collaboration to accelerate stem cell research

by Heather Buschman, Ph.D. on January 3, 2013 at 6:00 am | 0 Comments

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Sanford-Burnham's Stem Cell Research Center provides resources and expertise to the entire scientific community. They are also building the world's largest collection of human induced pluripotent stem cells (iPSCs).

New collaboration combines Sanford-Burnham’s renowned scientific team and Intrexon’s proprietary discovery platforms to accelerate human induced pluripotent stem cell (iPSC) research

Today, we announced a new collaboration with Intrexon Corporation, a leading synthetic biology company, aimed at accelerating stem cell research. Under the agreement, Sanford-Burnham will gain access to sophisticated proprietary cellular selection and gene regulation technologies that are not currently on the market, including Intrexon’s Laser-Enabled Analysis and Processing (LEAP^™) instrument and RheoSwitch Therapeutic System^® (RTS^®). As part of the agreement, Intrexon may obtain commercial and intellectual property rights resulting from technological advances made under the collaboration.

“I’m looking forward to merging and melding our expertise,” said Evan Y. Snyder, M.D., Ph.D., professor and director of Sanford-Burnham’s Stem Cell Research Center and Stem Cell and Regenerative Biology Program. “We’ll bring our iPSC and gene therapy expertise to the table. Likewise, our colleagues at Intrexon will share their knowledge of how best to use the technologies. We envision we’ll be meeting with them frequently and sharing insights to further advance the platforms for stem cell applications.”

Sanford-Burnham is currently building the world’s largest collection of human iPSCs generated from individual patients and healthy volunteers. The Stem Cell Research Center’s expertise and resources are available to all Sanford-Burnham scientists, as well as other researchers at nonprofit and for-profit research organizations around the world.

Translational Research Institute establishes new research paradigm for metabolic diseases

Originally published March 27, 2012

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.”

Disease in a dish: the ultimate personalized medicine

by Heather Buschman, Ph.D. on December 7, 2012 at 5:15 am | 0 Comments

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The latest episode of Developments to Watch, our collaborative video series produced by Medscape, is now available online: Disease in a Dish: The Ultimate Personalized Medicine.

In the video, Sanford-Burnham CEO John Reed, M.D., Ph.D., talks to Michael Jackson, Ph.D., vice president of drug discovery and development, about the Institute’s work on creating personalized “disease in a dish” models using stem cells derived from patients. They also talk about drug repurposing—finding new applications for existing therapeutic drugs in order to get treatments to patients faster.

Here’s an excerpt:

#SBsymposium 2012: Stem cells & drug discovery

by Heather Buschman, Ph.D. on October 30, 2012 at 5:52 am | 0 Comments

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Symposium speakers Christine Mummery, Ph.D. (Leiden University Medical Center) and Mark Mercola, Ph.D. (Sanford-Burnham) both discussed their work on generating new heart muscle tissue from stem cells

Scientists from all over San Diego—and beyond—gathered last Friday for Sanford-Burnham’s 34th annual symposium. This year’s theme: Frontiers in Stem Cell Biology for Drug Discovery. The topic was timely, given the recently announced 2012 Nobel Prize in Physiology or Medicine, awarded to John B. Gurdon and Shinya Yamanaka for their “discovery that mature cells can be reprogrammed to become pluripotent.” Yamanaka figured out how to turn a normal adult cell, such as a skin cell, into a stem cell that has the potential to become any other type of cell in the body. These special, laboratory-made stem cells are called induced pluripotent stem cells (iPSCs).

The symposium’s discussions centered on the idea that stem cells –especially iPSCs—can be used to model an individual’s own unique disease in a laboratory dish. These human cell-based models can then be used to test new and existing drugs for their toxicity and efficacy against disease.

Speakers came from Sanford-Burnham, Harvard, UT Southwestern, Mass General Hospital, UC San Diego, Stanford, and more. They talked about using stem cells to study and develop new therapies for conditions such as motor neuron disease, heart disease, autism, brain injury, Huntington’s disease, and spinal muscular atrophy.

We live-tweeted the event. For a snapshot of the day, including interesting tidbits, pictures, quotes, and links for more information, check out the Storify version of our tweets below. Then join the discussion on Twitter — look for us at @SanfordBurnham and #SBsymposium.

For more on stem cells and Sanford-Burnham’s work in the field, see:
Stem Cells 101
What is “Disease in a Dish”?
More stem cell blog posts
California Institute for Regenerative Medicine

Steven Smith named president-elect of The Obesity Society

by Patrick Bartosch on October 25, 2012 at 5:24 am | 0 Comments

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Dr. Steven Smith was appointed president-elect of TOS in September

Steven R. Smith, M.D., co-director of translational research at Sanford-Burnham and scientific director of the Translational Research Institute for Metabolism and Diabetes (TRI), recently began his term as president-elect of The Obesity Society (TOS), following the organization’s September meeting. As the leading scientific society dedicated to the study of obesity, TOS is committed to encouraging research into the causes and treatment of obesity, and to keeping the medical community and public informed of new advances.

“The Obesity Society is focused on education, research, and action to reverse the obesity epidemic and to help all Americans achieve better health,” says Smith. “Society is plagued by this epidemic that threatens our health and burdens our health care delivery system. In my new role, I look forward to helping educate the public, fellow clinicians, and policy makers about the impact of obesity. It is also imperative that we advocate for increased research funding in parallel with ongoing efforts to improve health through nutrition and physical activity.” At Sanford-Burnham and the TRI, Smith and his team hope to “crack the code” of obesity and provide more individualized, tailored approaches to treatment and prevention.

Stem cells 101

by Communications Staff on October 8, 2012 at 10:52 am | 2 Comments

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Sanford-Burnham's Stem Cell Research Center

Congratulations to John B. Gurdon and Shinya Yamanaka on winning the 2012 Nobel Prize in Physiology or Medicine! They received the award today for their “discovery that mature cells can be reprogrammed to become pluripotent.” In other words, these scientists figured out how to turn a normal adult cell, such as a skin cell, into a stem cell that has the potential to become any other type of cell in the body. Read below to learn more about stem cells and how they are revolutionizing medical research.

What are stem cells?

Stem cells are special because each is like a blank slate. Once it’s given the proper instruction, a stem cell can specialize and become any type of cell in the body—brain, heart, muscle, and more. Stem cells also have the ability to reproduce themselves indefinitely, renewing the supply.

Are there different types of stem cells?

Embryonic stem cells only exist during an organism’s development, when it is an embryo. These cells are pluripotent, meaning they have the capacity to become any cell type in the body.

Adult stem cells exist in fully developed organisms. They are more limited than embryonic stem cells—they are multipotent rather than pluripotent. These stem cells usually can only become a few types of specialized cells, based on the tissue from which they originate.

Induced pluripotent stem cells (iPSCs) are pluripotent, much like embryonic stem cells. iPSCs are produced in the laboratory by genetically reprogramming any adult cell, such as a skin cell.

Meet Rebecca, Sanford-Burnham employee and obesity study participant

by Patrick Bartosch on August 3, 2012 at 5:57 am | 0 Comments

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Scientists at the TRI measure Rebecca Kaercher's oxygen levels, among other parameters

Rebecca Kaercher has been struggling with her weight for years. “Losing weight has been a very frustrating endeavor for me. I go on diets, I work out, but I just don’t lose any weight,” she says. Rebecca has been working at Sanford-Burnham at Lake Nona for three years and, together with a few of her Institute coworkers, was thrilled to volunteer as a metabolism and weight loss study subject at the Florida Hospital – Sanford-Burnham Translational Research Institute for Metabolism and Diabetes (TRI). “I am very proud to work at Sanford-Burnham and I want to be an active part of our research. Except for being overweight, I am a very healthy person. I don’t have high cholesterol, no high blood pressure, no diabetes. So it was hard for me to understand why I didn’t lose any weight.”

Rebecca is the kind of person scientists at the TRI are currently looking for as volunteers – people who have tried to lose weight by exercising and changing their diets but didn’t succeed. As with many other people who have the same problem, something in Rebecca’s body keeps her from losing weight and researchers at the TRI are trying to find out what that could be. The common perception is that overweight people only need to eat healthier and exercise more in order to lose weight. But this simple approach does not work for everyone. That is about to change with the research taking place at the TRI’s new facility in downtown Orlando.

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)

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.

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

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

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

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

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

“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

by Patrick Bartosch on March 27, 2012 at 6:00 am | 0 Comments

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New TRI Facility in Downtown Orlando

Obesity research advances to clinical testing

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

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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.

Top Stories - Translational Medicine

Partnering with Asia’s largest pharmaceutical company to find obesity treatments

Cancer drug targets hard-to-reach leukemia stem cells

Sanford-Burnham and Intrexon Corporation establish collaboration to accelerate stem cell research

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Translational Research Institute establishes new research paradigm for metabolic diseases

Disease in a dish: the ultimate personalized medicine

#SBsymposium 2012: Stem cells & drug discovery

Steven Smith named president-elect of The Obesity Society

Stem cells 101

What are stem cells?

Are there different types of stem cells?

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Meet Rebecca, Sanford-Burnham employee and obesity study participant

Researchers find new anti-malarial drug target

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

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

A “twisted” grand opening ceremony

Translational Research Institute establishes new research paradigm for metabolic diseases

Obesity research advances to clinical testing

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