Collaborating with Pfizer to speed drug discovery

by on November 3, 2011 at 10:51 am | 2 Comments
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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.

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.

Sanford-Burnham is the latest research organization to partner with Pfizer, Inc. as part of Pfizer’s commitment to transforming research and development through a focus on translational medicine. The Centers for Therapeutic Innovation (CTI) is a research unit at Pfizer dedicated to open innovation through establishing global partnerships with academic research institutions to bridge the gap between discovery science and clinical applications.

“Pfizer is truly excited to work in this new partnership with leading experts from Sanford-Burnham Medical Research Institute to understand more about the mechanisms that drive diseases with high unmet medical need,” said Dr. Anthony Coyle, vice president and head of Pfizer’s Global Centers for Therapeutic Innovation.

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Shared resources, shared successes

by on August 8, 2011 at 1:27 pm | 0 Comments
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Microscope available in Sanford-Burnham's Cell Imaging facility

Microscope available in Sanford-Burnham's Cell Imaging facility

Editor’s note: This is the first in a series of posts highlighting Shared Resources available at Sanford-Burnham. Future posts will further explore some of the individual capabilities found in these core facilities.

Suppose you’re a new assistant professor just starting your career at Sanford-Burnham, and you need to perform some high-resolution fluorescence microscopy to finish your first big paper as a principal investigator. How do you afford that $400,000 confocal microscope for the key experiments? For that matter, how does anyone afford a $400,000 microscope? Here’s where Shared Resources saves the day. Just down the stairway sits the Zeiss Laser Scanning Confocal Microscope that Sanford-Burnham’s Cell Imaging facility has thoughtfully provided for you. How did you get so lucky?

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CARing for pulmonary arterial hypertension

by on July 22, 2011 at 9:57 am | 0 Comments
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Dr. Masanobu Komatsu

Dr. Masanobu Komatsu

Pulmonary arteries carry blood from the heart to the lungs, where they pick up fresh oxygen for distribution to the rest of the body. Since almost every cell in the human body needs oxygen in order to convert nutrients into energy, pulmonary artery function is crucial. Pulmonary arterial hypertension (PAH) occurs when pressure builds up in these blood vessels, impairing this function. People with PAH experience shortness of breath, fatigue and chest pain. As the condition worsens, the heart has to work harder and harder to pump blood, sometimes leading to heart failure.

Despite eight approved clinical therapies for PAH and additional therapies currently in trials, there is no cure. What’s more, current treatments don’t specifically target pulmonary arteries, which can lead to severe side effects.

Sanford-Burnham scientists, led by Drs. Masanobu Komatsu and Dr. Takeo Urakami, in collaboration with VBS Pharmaceuticals, recently discovered a peptide (a short protein) that selectively targets and penetrates lung blood vessels affected by PAH. When the team tested this peptide, called CARSKNKDC (or CAR for short) in a rodent model of PAH, it homed in on hypertensive lungs, but spared healthy lungs and other organs. CAR also accumulated in other regions of the respiratory system that play crucial roles in PAH development and progression.

Published in the June 2011 issue of the American Journal of Pathology, these findings indicate that CAR could be used to deliver therapeutic compounds and imaging probes directly to PAH lungs.

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NeuroMap wins Entrepreneur Challenge

by on June 27, 2011 at 11:22 am | 0 Comments
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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 was founded by (left to right): Daniel Norton, Dr. Alexey Terskikh, Dr. Dmitry Sivtsov and Dr. Andrew Rabinovich. (Photo by Sean M. Haffey, courtesy of San Diego Union-Tribune)

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.

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My moment with Corinna

by on May 27, 2011 at 6:39 am | 0 Comments
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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.

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Targeting Arterial Plaque

by on April 19, 2011 at 4:00 am | 1 comment
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Dr. Erkki Ruoslahti

Dr. Erkki Ruoslahti

Atherosclerotic plaque is the fatty material that builds up on arterial walls, where it can lead to heart disease and stroke. Atherosclerosis is currently treated with dietary changes, angioplasty (which uses a balloon to move the plaque aside) or more invasive procedures. Using drugs to break up these fatty plaques would be an enticing alternative, but delivery poses a problem. How do we precisely target the therapeutic agent to the diseased areas, leaving healthy tissues unaffected?

Dr. Erkki Ruoslahti and colleagues at Sanford-Burnham and UC Santa Barbara may have found a solution. For many years, Dr. Ruoslahti has been using specially designed peptides (pieces of proteins) to target cancer and other diseases. In a paper published online on April 11 by the Proceedings of the National Academy of Sciences, the Ruoslahti lab reports the discovery of a new peptide that can guide drugs or imaging agents specifically to atherosclerotic plaques.

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Filling the Drug Pipeline

by 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

Measuring Nanoparticles

by on March 14, 2011 at 1:11 pm | 2 Comments
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One of the problems with nanoparticles is that, well, they’re just so small, making them difficult to study. Researchers may have solved that problem by building an instrument that can detect nanoparticles as small as tens of nanometers (billionths of a meter). The research team was led by Dr. Andrew Cleland, professor of physics at the University of California, Santa Barbara, and included Sanford-Burnham’s Dr. Erkki Ruoslahti. The study was published on March 7 in the journal Nature Nanotechnology.

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Rare disease symposium has uplifting moments

by on March 4, 2011 at 11:04 am | 0 Comments
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Kate Fischer with daughter Morgan, who is being treated for HPP.

Kate Fischer with daughter Morgan, who is being treated for HPP.

There was a moment at our recent Rare Disease Symposium when Dr. Michael Whyte, a pediatrician from Shriner’s Hospitals in St. Louis, presented video of a patient who is participating in a clinical trial. The patient, Amy, suffers from hypophosphatasia (HPP), a genetic bone disease similar to rickets. The trial is for an enzyme  replacement therapy developed collaboratively by Dr. Whyte, Dr. José Luis Millán and Enobia Pharma to treat HPP. Before treatment, Amy’s bones were so soft she had to be flown to the trial in an insulated box. She was weeks away from dying. In the video, she runs, jumps and kicks a ball. Hard not to be moved.

Enobia’s HPP drug is in Phase II clinical trials and looks quite promising. However, rare diseases present a difficult problem. While relatively few people suffer from any single rare disease, there are thousands of these conditions. Large pharmaceutical and biotech companies have a difficult time addressing them because they have not figured out how to make back their investments. But the issues go even deeper. How do you conduct a robust clinical trial on a new treatment when only a handful of people need to be treated? And how do you balance the regulatory environment to ensure that new, safe treatments can reach patients? In fact, how do you even diagnose a rare disease when so few physicians have any experience with it?

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Rare disease chat

by on February 28, 2011 at 9:57 am | 0 Comments
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Today is Rare Disease Day and Dr. José Luis Millán, a professor in our Sanford Children’s Health Research Center, held a live chat on hypophosphatasia(HPP), a rare, genetic bone disorder similar to rickets. HPP patients’  bones do not mineralize properly. In some cases, a patient’s bones are so soft they can barely be touched without breaking.Dr. Millán has been working for several years with Dr. Michael Whyte, of Shriners Hospitals for Children in St. Louis, and Enobia Pharma to develop an enzyme replacement therapy for HPP. Though still in clinical trials, the treatment has so far shown dramatic results. In some cases, children who probably would not have survived without treatment can now run, jump and develop normally.

The chat follows up on our Second Annual Rare Disease Symposium, which was held on February 25 and can still be viewed online. You can also view the chat transcript.

This is Your Brain on Drugs

by on February 15, 2011 at 7:12 am | 1 comment
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Cocaine produces its powerful high by stimulating “reward” signals in the brain, sending users back again and again for more. Cocaine gains this effect, in part, by stimulating a receptor called the metabotropic glutamate receptor 2 (mGluR2). Dr. Nicholas Cosford’s group is currently collaborating with Dr. Athina Markou at UC San Diego and Dr. P. Jeffrey Conn at Vanderbilt University Medical Centerto design chemical compounds that bind mGluR2 in a way that dampens cocaine’s stimulatory effects on the brain. The team recently found that, when given to rats, one such compound – called BINA – reduces cocaine-seeking behavior and consumption.New treatments for drug addiction are clearly needed. Cocaine is responsible for more emergency room visits in the United States than any other illegal drug. It harms the brain, heart, blood vessels and lungs, and can even cause sudden death. With an estimated two million addicts in this country, cocaine is a major public health issue.

Yet a deeper understanding of mGluR2 might have an even greater impact on human health.

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Japanese signing ceremony initiates new partnership

by 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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New Partnership Targets Brain Conditions

by on January 18, 2011 at 9:27 am | 1 comment
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Robots in the Prebys Center screen hundreds of thousands of compounds to find the handful that could become new medicines.

Robots in the Prebys Center screen hundreds of thousands of compounds to find the handful that could become new medicines.

Sanford-Burnham has been conducting cutting-edge research on neurological and neuropsychiatric conditions for many years and recently that research received a big boost. The Institute announced a collaboration with Ortho-McNeil-Janssen Pharmaceuticals, Inc., (OMJPI), a division of Johnson & Johnson, to discover new drugs to treat Alzheimer’s diseaseand major psychiatric disorders.Under the agreement, Sanford-Burnham will look for new therapeutic targets for Alzheimer’s disease and neuropsychiatric conditions. Then, the Conrad Prebys Center for Chemical Genomics (Prebys Center) will identify chemical compounds that therapeutically alter those targets. These compounds will then be optimized and directed into OMJPI’s drug pipeline.

 

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Collaboration with Takeda seeks to translate research into treatments

by on December 28, 2010 at 4:00 am | 0 Comments
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Sanford-Burnham, Florida Hospital and Takeda Pharmaceutical have formed a collaboration to investigate new therapies for obesity, a growing worldwide health problem. The partnership leverages the three organizations’ strengths in basic biomedical research, clinical research and drug development to identify obesity-related biomarkers and other targets with therapeutic potential.“There is an epidemic of obesity in the U.S.; two-thirds of Americans are now overweight or obese. These staggering statistics serve as a call for decisive action, including innovative bench-to-bedside translational research,” says Dr. Steven R. Smith, scientific director of the Florida Hospital-Sanford-Burnham Translational Research Institute for Metabolism and Diabetes (TRI). “This partnership with Takeda, TRI and Sanford-Burnham represents a major milestone in the quest for a better understanding of obesity as a disease and a pathway forward for the development of safe and effective therapies.”

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Bayh-Dole Act: Driving Innovation for 30 Years

by on December 17, 2010 at 4:08 pm | 1 comment
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When it first passed 30 years ago this week, the Bayh-Dole Act chipped away at academia’s ivory towers. This landmark legislation smoothed the way for new innovations begun in federally funded labs at universities and independent research institutes to reach the marketplace as new therapies.A recent article published by the Biotechnology Industry Organization (BIO) calls the Bayh-Dole Act “the legislation largely responsible for the growth of the biotech sector.”

“Before the Bayh-Dole Act, discoveries made in academic laboratories were not moving forward to the patients who could benefit from them. Companies had no guarantee that they would be rewarded for huge initial investments in research and clinical trials,” explains Margaret Dunbar, senior director of intellectual property and legal affairs at Sanford-Burnham. “Now, patents are what motivate researchers and companies to move therapies forward to the public.”

Since the Institute’s founding (just four years before Bayh-Dole was enacted), Sanford-Burnham research has led to 603 patents and generated more than 90 license agreements with strategic partners. For those achievements, we celebrate 30 years of Bayh-Dole.

For more information on Sanford-Burnham technologies and commercialization opportunities, visit our Innovation Catalog.

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