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.
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.”
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.
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Academia Jumps into Drug Discovery
The Promise of Chemical Genomics
Laboratory to Pharmacy
From Research, the Power to Cure
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.
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.
"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.
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.
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.
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.
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.
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.