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Sanford-Burnham Science Blog

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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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DTW

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:

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On the Cutting Edge

by Josh Baxt on April 13, 2011 at 8:55 am | 0 Comments
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Neural stem cells, image by Dr. Ilyas Singec, Snyder laboratory

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.

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Frontiers in Biomedical Science

by Heather Buschman, Ph.D. on April 1, 2011 at 10:43 am | 0 Comments
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Scenes from a symposium

Scenes from a symposium

Sanford-Burnham’s East Coast facility is located on a modern-day frontier – Orlando’s emerging Medical City at Lake Nona, where a life science campus with a medical school and two hospitals has sprung from wild pastures. Because they joined Sanford-Burnham at Lake Nona at its inception and work together at the forefront of diabetes and obesity research, the 185 scientists and staff who work there are often called “pioneers”. And on March 11, Lake Nona also hosted Frontiers in Biomedical Science: Metabolic Networks and Disease Signatures. This second annual symposium brought together more than 200 people from the state (especially the University of Florida and the University of Central Florida) and other research institutions and pharmaceutical companies around the country, including Merck, Eli Lilly and GlaxoSmithKline.

According to Dr. Daniel Kelly, scientific director of Sanford-Burnham at Lake Nona, “The speakers at this year’s annual symposium spoke about metabolic research and discovery, as well as disease applications relevant to our focus at Sanford-Burnham at Lake Nona. But innovation was a central theme. From Dr. David Botstein’s seminal work with yeast genetics to Dr. Leslie Leinwand’s novel investigations with Burmese pythons, the audience learned about groundbreaking approaches.”

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Filling the Drug Pipeline

by Josh Baxt on March 29, 2011 at 12:18 pm | 0 Comments
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Dr. Paul Laikind

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

Quick peek at Sanford-Burnham

by Josh Baxt on March 23, 2011 at 7:19 am | 0 Comments
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Sanford-Burnham's La Jolla campus. Photo by Nadia Borowski Scott

A video by high school student Daniel Osterman, son of Sanford-Burnham investigator Dr. Andrei Osterman, takes a quick look at the basic biomedical research being conducted at the Institute. In particular, the piece focuses on Dr. Hudson Freeze’s research. Dr. Freeze recently organized Sanford-Burnham’s 2nd Annual Rare Disease Symposium, and studies a group of rare conditions called Congenital Disorders of Glycosolation (CDG), in which sugars fail to attach properly to proteins.

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Japanese signing ceremony initiates new partnership

by Deborah Robison on February 7, 2011 at 9:41 am | 1 comment
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On January 31, Sanford-Burnham, Florida Hospital and Takeda Pharmaceuticalcelebrated their new research alliance with a signing ceremony steeped in Japanese tradition. Representatives from each organization met at Sanford-Burnham’s Lake Nona campus in Orlando to sign the agreement and exchange gifts.The drawing of one “eye” on a Daruma doll held special significance for the Japanese scientists. As Takeda executives explained, at the start of a new undertaking, partners color in one eye of the doll. Later, if discovery efforts are successful  –  isolating a new target or a good lead compound — the team will fill in the remaining eye. Dr. Paul Chapman, general manager of Takeda’s research division, joked that the particularly large doll was symbolic of the big challenges ahead. Takeda, the largest pharmaceutical company in Japan, is committed to discovering new therapeutics to treat obesity and diabetes.

“We are delighted to have found the world-class talent that we are seeking here in Central Florida,” Dr. Chapman said.

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A different path to fat-related heart disease

by Heather Buschman, Ph.D. on January 18, 2011 at 3:09 pm | 2 Comments
Full Article

Heart disease is the leading cause of death for both men and women in the United States. But heart disease is more than just one disease; there are many different ‘flavors’ that can result from a heart attack, high blood pressure, diabetes or other causes. In lipotoxic cardiomyopathy, for example, heart function is disrupted by fat accumulation in heart cells. Obesity and high-fat diets are major risk factors for lipotoxic cardiomyopathy, but scientists recently unraveled an alternative pathway to lipotoxic cardiomyopathy in fruit flies – a genetic mechanism that occurs independently of a diet high in fat. Their study lays the foundation for the development of new ways to combat lipotoxic cardiomyopathy and other types of heart disease.

“It’s a well-accepted notion that if you eat too much fatty food and your body can’t metabolize it properly, you can become obese and this can lead to lipotoxic cardiomyopathy. Our study shows that there is also an alternative cause of obesity and associated heart problems – an imbalance in the fats that normally make up the basic structure of our cells,” explained Dr. Hui-Ying Lim, post-doctoral researcher and lead author of the study.

In this study, the researchers analyzed mutant fruit flies (called easily shocked mutants) that have abnormally low levels of phosphatidylethanolamine (PE), a type of fat that makes up a major component of cellular membranes in both flies and mammals. They found that these flies compensate for low PE levels by initiating a mechanism for synthesizing fat. In this mechanism, a protein called sterol regulatory element-binding protein (SREBP) turns on genes encoding metabolic enzymes that synthesize more fat.

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What is “Disease in a Dish?”

by Heather Buschman, Ph.D. on January 12, 2011 at 11:34 am | 6 Comments
Full Article
"Disease in a dish" has great potential to accelerate drug discovery.

"Disease in a dish" has great potential to accelerate drug discovery.

“Disease in a dish” is a cutting-edge, stem cell-based strategy that allows researchers to study an individual patient’s cells in a laboratory dish. Traditionally, scientists interested in a particular disease have used a standard cell line that has been grown in the lab for years or a mouse model (if one exists) that has been engineered to mimic the disease. Although extremely valuable, these techniques have obvious limitations. Animal models never entirely reflect the actual human condition – they don’t capture the complicated interplay between an individual patient’s genetics and the environmental factors that might influence the development of the disease or that patient’s response to a new therapy.

Read below to find out how diseases in a dish are made, how they’re being used to study and treat disease and how Sanford-Burnham researchers are applying the technique.

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Four ways patient advocates help drive research

by Heather Buschman, Ph.D. on December 16, 2010 at 2:53 pm | 5 Comments
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How can patient advocates help drive basic research?

How can patient advocates help drive basic research?

Last week I attended the Stem Cell Meeting on the Mesa, an annual event organized by CONNECT. The meeting included all the stellar scientific panels I expected and one I didn’t expect: “Patient Advocacy 2.0 – Can they participate?”

The panel discussed opportunities for patient participation and the ethics involved. I was captivated by panel member Dani Grady’s story of surviving breast cancer and her advocacy for increased cancer research funding, education, improved patient care and more patient participation in clinical trials. It was interesting to hear how a patient’s perspective can improve clinical trials and the drug approval process. But as I sat there, I couldn’t help wondering… how can patients participate in basic research – the earliest phase of biomedical discovery, during which the molecular underpinnings of disease are only just beginning to be understood?

So I did a little research of my own.

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The couch potato effect

by Heather Buschman, Ph.D. on December 1, 2010 at 8:06 am | 2 Comments
Full Article

A surprising new model for studying muscle function was unveiled this week: the couch potato mouse. While these mice maintain normal activity and body weight, they don’t have the energy to exercise. In the December 1 issue of the journal Cell Metabolism, Dr. Daniel Kelly, Dr. Christoph Zechner and their colleagues reportwhat happens when muscle tissue lacks PGC-1, a protein coactivator that muscles need to convert fuel into energy.“Part of our interest in understanding the factors that allow muscles to exercise is the knowledge that whatever this machinery is, it becomes inactive in obesity, aging, diabetes and other chronic conditions that affect mobility,” explains Dr. Kelly, scientific director at Sanford-Burnham’s Lake Nona campus.

Normally, physical stimulation boosts PGC-1 activity in muscle cells, which switches on genes that increase fuel storage, ultimately leading to “trained” muscle (the physical condition most people hope to attain through exercise). In obese people, PGC-1 levels drop, possibly further reducing a person’s capacity to exercise – creating a vicious cycle. In this study, mice without muscle PGC-1 looked normal and walked around without difficulty, but could not run on a treadmill.

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Reining in melanoma with MicroRNA

by Heather Buschman, Ph.D. on November 1, 2010 at 2:12 pm | 2 Comments
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Dr. Ranjan Perera (left) with post-doctoral researcher Dr. Joseph Mazar

Dr. Ranjan Perera with post-doctoral researcher Dr. Joseph Mazar

Skin cancer is the most common cancer in the United States. Melanoma is one of the rarest forms of skin cancer, but it is also the most deadly. At Sanford-Burnham’s Lake Nona campus, Dr. Ranjan Perera’s lab is studying what causes melanocytes (pigment-producing skin cells) to divide abnormally, ultimately forming melanoma. In a study published today in the journal PLoS ONE, a team led by Dr. Perera and post-doctoral researcher Dr. Joseph Mazar show that melanocyte growth and the cancer’s ability to invade other tissue is at least partially controlled by abnormal expression of microRNAs (miRNAs) – small strands of genetic material that may play a major role in numerous diseases by interfering with proteinproduction.“We’ve identified one specific miRNA, called miR-211, that could be used not only as a novel diagnostic marker for early melanoma detection, but also as a therapeutic target,” explains Dr. Perera, associate professor in Sanford-Burnham’s RNA Biology Program and senior author of the study.

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Our TEDster on Nature, Nurture and Nanomedicine

by Kristina Meek on October 22, 2010 at 10:10 am | 1 comment
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Earlier this week, the public had the chance to talk one-on-one with a renowned nanomedicine expert. Sanford-Burnham’s Dr. Jamey Marth hosted the online chat as a follow-up to his talk at TEDxAmericanRivierain Santa Barbara, Calif. on 10-10-2010.Dr. Marth is the director of the Center for Nanomedicine, a partnership between Sanford-Burnham and UC Santa Barbara. He is working to develop nano-sized “smart devices” that diagnose, target, treat and cure disease before it can cause symptoms and spread. This technology could lead to revolutionary treatments for diabetes, Parkinson’s and Alzheimer’s disease, to name a few.

The online chat attracted a record number of participants. The questions covered a range of topics within nanomedicine, showing a keen interest by the public in this fascinating field. For more info, you can read a transcript of the chat. Please join our mailing list if you are interested in hearing about future research chats.

Click below to watch Dr. Marth’s TEDx talk: “Nature, Nurture, and Nanomedicine”

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Mrs. Lillian Fishman Honored by Alma Mater

by Kristina Meek on September 22, 2010 at 2:53 pm | 0 Comments
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In April, Sanford-Burnham co-founder Mrs. Lillian Fishman was celebrating her 95th birthday when she received some fantastic news. Her alma mater, the University of Alberta was awarding her their Distinguished Alumni Award.  The awards are given each year to University of Alberta graduates whose achievements have earned them national or international prominence. Yesterday, Mrs. Fishman attended the award ceremony in Edmonton, accompanied by family and friends.

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DNA 101

by Heather Buschman, Ph.D. on September 21, 2010 at 4:04 pm | 44 Comments
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DNA is short for deoxyribonucleic acid. Two chains of four chemical bases (abbreviated A, T, C and G) make up DNA and act as a cell’s recipe book to make proteins. The particular sequence of a DNA chain – meaning the precise order of the four chemical bases – determines what protein will be made. A DNA segment beginning with ATTCGC would produce a very different protein than one that starts with CCGTAT. This can be likened to adjusting the order of letters in a word. Though the letters are the same, the meaning changes. For example,  act means something very different than cat.

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No Exit: Helping Cancer Cells Die

by Josh Baxt on May 11, 2010 at 3:50 pm | 2 Comments
Full Article

Cancer cells use anti-apoptotic (anti-cell death) proteins in the Bcl-2 family to evade treatment. Even when slammed with harsh doses of radiation or chemotherapy, cancer cells can harness these proteins to evade death. However, that escape route may be closing. In the past few days, Dr. Maurizio Pellecchia has published two papers that shed new light on how Bcl-2 proteins work and how we can defeat them.

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