Using stem cells to treat Parkinson’s disease

by on September 26, 2011 at 7:13 am | 0 Comments
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Neurons derived from embryonic stem cells

Neurons derived from embryonic stem cells

When neurons that make a chemical called dopamine are slowly destroyed, nerve cells in that part of the brain cannot properly send the messages that would normally control muscle function. As the damage gets worse with time, a person experiences tremors and movement becomes difficult. This is Parkinson’s disease.

In short, Parkinson’s patients need more dopamine. Or, better yet, new neurons that produce dopamine on their own. In a paper published August 25 in the journal PLoS ONE, a team led by Dr. Stuart Lipton, director of Sanford-Burnham’s Del E. Webb Neuroscience, Aging, and Stem Cell Research Center, demonstrates how this therapeutic approach might be possible.

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10 years of science & counterterrorism

by on September 11, 2011 at 6:41 am | 0 Comments
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Crystal Structure of Anthrax Lethal Factor complexed with a small molecule inhibitor

Crystal Structure of Anthrax Lethal Factor complexed with a small molecule inhibitor

As the United States pauses to observe the 10th anniversary of the September 11th terrorist attacks, we reflect on the research advances that contribute to new counterterrorism measures—understanding anthrax, for example—and the health of our soldiers in Iraq and Afghanistan, including under-studied conditions such as traumatic brain injury (TBI) and post-traumatic stress disorder (PTSD). Here are a few examples, and these only cover discoveries made at Sanford-Burnham since September 11, 2001. Can you think of more? Please share your thoughts in the comments below.

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How to make to new neurons

by on August 31, 2011 at 9:55 am | 0 Comments
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Neurons (Image courtesy of the Lipton lab)

Neurons (Image courtesy of the Lipton lab)

Imagine the ability to take skin cells from a patient with Alzheimer’s disease, convert them directly into brain cells, and then study how the disease progresses in those cells—which still contain the patient’s DNA—all in the lab, with minimal invasiveness on the part of the patient. Then imagine taking those same brain cells and testing novel but risky drugs that could cure the devastating disease—again, in the safety of a dish in the lab.

Researchers are on their way to achieving this remarkable milestone. Dr. Stuart Lipton at Sanford-Burnham, Dr. Sheng Ding at the Gladstone Institutes, and their collaborators recently figured out how to reprogram skin cells directly into functioning neurons. The study was published online July 28 in the journal Cell Stem Cell.

“This technology should allow us to very rapidly model neurodegenerative diseases in a dish by making nerve cells from individual patients in just a matter of days, rather than the months required previously,” Dr. Lipton says in a statement released by the Gladstone Institutes.

The paper is one of several recent studies that are all zeroing in on a long-sought-after advance in stem cell science: the potential to obtain unlimited numbers of brain cells from an easily accessible tissue such as the skin.

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Getting to the root of Alzheimer’s disease

by on August 17, 2011 at 5:47 am | 0 Comments
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Dr. Stuart Lipton

Dr. Stuart Lipton

Alzheimer’s disease is characterized by abnormal proteins that stick together in little globs, disrupting cognitive function (thinking, learning, and memory). These sticky proteins are mostly made up of beta-amyloid peptide. A better understanding of these proteins, how they form, and how they affect brain function will no doubt improve the diagnosis and treatment of Alzheimer’s disease. To this end, a research team led by Dr. Stuart Lipton‘s group found that beta-amyloid-induced destruction of synapses—the connections that mediate communication between nerve cells—is driven by a chemical modification to an enzyme called Cdk5. The team found that this altered form of Cdk5 (SNO-Cdk5) was prevalent in human Alzheimer’s disease brains, but not in normal brains. These results, published August 15 in the Proceedings of the National Academy of Sciences of the USA, suggest that SNO-Cdk5 could be targeted for the development of new Alzheimer’s disease therapies.

Cdk5 is an enzyme known to play a role in normal neuronal survival and migration. In this study, Dr. Lipton and colleagues found that beta-amyloid peptides, the hallmark of Alzheimer’s disease, trigger Cdk5 modification by a chemical process called S-nitrosylation. In this reaction, nitric oxide (NO) is attached to the enzyme, producing SNO-Cdk5 and disrupting its normal activity.

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Serendipity in science

by on July 28, 2011 at 12:46 pm | 0 Comments
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Stephen Hawking, famous English physicist, lives with amyotrophic lateral sclerosis (Lou Gehrig's disease), a debilitating disese that affects motor neurons.

Stephen Hawking, famous English physicist, lives with amyotrophic lateral sclerosis (Lou Gehrig's disease), a debilitating disese that affects motor neurons.

Many people are familiar with the story of Alexander Fleming’s accidental discovery of penicillin produced by mold growing in a bacterial culture. These same people would probably be surprised at how often carefully planned scientific experiments yield unexpected (and even unwanted!) results, usually leading to repetition of the experiment to discover where things went wrong. However, one mark of a really good investigator (like Fleming) is the ability to recognize when the “error” may actually be a truth that provides a key new insight. The phenomenon of looking for one thing and serendipitously finding another plays a surprisingly frequent role in the process of scientific discovery.

A case in point can be found in studies of motor neuron degeneration being carried out in the laboratory of Dr. Dongxian Zhang, associate professor at Sanford-Burnham. The death of motor neurons in the spinal cord is responsible for lethal diseases such as spinal muscular atrophy and amyotropic lateral sclerosis (Lou Gehrig’s disease), neither of which is treatable or curable. Dr. Zhang’s group hypothesized that motor neuron death might be caused by the absence or malfunction of a specific type of membrane receptor called MNR. To test their theory more directly, they paid a commercial company to create a mouse in which MNR was genetically deleted. Sure enough, motor neurons in these mice degenerated a few days after birth. To further prove their point, the group attempted to rescue the lethal defect by genetically adding back the MNR gene. To their consternation, these transgenic rescue mice still died shortly after birth.

“At that point we were completely stumped and discouraged,” confesses Dr. Zhang.

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Renowned biochemist joins Sanford-Burnham

by on July 19, 2011 at 3:01 pm | 0 Comments
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Dr. Randal J. Kaufman

Dr. Randal J. Kaufman

This month we welcomed Sanford-Burnham’s newest faculty member, Dr. Randal J. Kaufman. Dr. Kaufman joins the Institute as  professor and director of the Degenerative Disease Research Program, in the Del E. Webb Neuroscience, Aging and Stem Cell Research Center.

“I am looking forward to the opportunities for collaboration that Sanford-Burnham affords,” Dr. Kaufman says. “This promises to be a very productive environment for my area of research.”

Dr. Kaufman’s current research is focused on understanding the fundamental mechanisms that regulate protein folding and the cellular responses to the accumulation of unfolded proteins within the endoplasmic reticulum (ER). When proteins fail to fold correctly, they don’t work properly. Certain types of misfolded proteins defy eradication by the cellular protein degradation machinery and accumulate with age, causing cellular toxicity. In many degenerative diseases, including neurological, metabolic, genetic and inflammatory diseases, it’s thought that the accumulation of misfolded proteins leads to cellular dysfunction and death.

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Join us for a congressional briefing on Alzheimer’s disease

by on July 8, 2011 at 3:25 pm | 1 comment
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U.S. Capitol Building, Washington, D.C.

The United States Capitol, Washington, D.C.

What: Congressional Briefing on Alzheimer’s Disease Research and Therapeutic Advancements, Innovations and Treatments

CHI-The California Healthcare Institute and the Healthcare Institute of New Jersey (HINJ) invite you to a congressional briefing that will facilitate a dialogue about Alzheimer’s disease research. This program will include an overview of advances stemming from California’s and New Jersey’s life sciences sectors, with an expert panel discussing current research and drug development, as well as future discoveries, followed by a brief question and answer session.

When: July 14, 2011, 9:30 a.m. – 11 a.m.
Where: Capitol Visitor’s Center, Congressional Meeting Room South, Washington, D.C.

Why: According to a 2011 report released by the Alzheimer’s Association, an estimated 5.4 million people are living with Alzheimer’s disease, and someone develops the disease every 69 seconds. The United States, like many other countries, has an aging population with nearly one in five residents reaching the age of 65 or older by 2030. Additionally, in 2010, 14.9 million family members and friends provided 17 billion hours of unpaid care to those living with Alzheimer’s and other dementias — care valued at approximately $202 billion. With the imminent increase in dementia caused by Alzheimer’s disease and other conditions — and without a cure — the development of new innovations and treatments remains all the more critical to assist in improving the quality of life for those affected. CHI members Genentech, Pfizer, Sanford-Burnham and University of California, Irvine and HINJ members Merck, Lundbeck, Bayer and Pfizer are all working to advance important new studies and therapeutics.

Who:
Rep. Chris Smith (R-NJ)
Rep. Linda Sanchez (D-CA)

David Gollaher, Ph.D., President & CEO, CHI
Dean J. Paranicas, President & CEO, HealthCare Institute of New Jersey (HINJ)
Joseph Hammang, Ph.D., Senior Director, Worldwide Science Policy, Pfizer
Stuart Lipton, M.D., Ph.D., Director, Del E. Webb Neuroscience, Aging and Stem Cell Research Center, Sanford-Burnham Medical Research Institute
Bob Nelson, Ph.D., Research Fellow, Lundbeck Research USA
Wayne Poon, Ph.D., Director, UCI MIND Brain Bank and Tissue Repository
Kimberly Scearce-Levie, Ph.D., head of in vivo neurobiology at Genentech Inc.

RSVP to Caitlin Doyle at doyle@chi.org or (202) 974-6323

Coming soon: Medscape’s “Developments to Watch”

by on July 1, 2011 at 7:08 am | 0 Comments
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Dr. Evan Snyder (right) interviews his colleague, Dr. Steven Smith, on the Medscape set.

Dr. Evan Snyder interviews his colleague, Dr. Steven Smith, on the Medscape set.

Last week, Sanford-Burnham’s Fishman Auditorium, on the Institute’s La Jolla campus, was transformed into a temporary television studio. It was hardly recognizable under the bright lights and set dressing. Medical website Medscape recorded interviews with three Sanford-Burnham researchers for a new video series called “Developments to Watch.” The talk show-like discussions were hosted by Dr. Evan Snyder, who directs the Stem Cells and Regenerative Biology Program at Sanford-Burnham. Dr. Snyder is both a medical doctor who regularly sees patients and a scientist who conducts research in his own lab – the perfect person to help explain how discoveries made today might one day help patients.

Medscape is part of the network of sites run by WebMD. With this newest video series, Sanford-Burnham scientists will be providing expert commentary and information to help keep Medscape’s audience – primary care physicians, specialists and other health professionals – up-to-date on the latest medical research and what it means for their patients.

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New stem cell techniques make more neurons, faster

by on May 20, 2011 at 8:24 am | 1 comment
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Dr. Stuart Lipton

Dr. Stuart Lipton

Stroke, Alzheimer’s, Parkinson’s, ALS—these neurological conditions strip people of their abilities to think, move or both. One of the goals driving regenerative medicine research is to use stem cells to create neural cells to treat these and other neurodegenerative conditions. Two papers recently published in Proceedings of the National Academy of Sciences USA (PNAS) bring us measurably closer to this goal.In the first paper, Dr. Stuart Lipton, Professor and Director of the Del E. Web Center for Neuroscience, Aging and Stem Cell Research, collaborated with lead authors Dr. Sheng Ding of the Gladstone Institutes and Dr. Kang Zhang of the UC San Diego School of Medicine to create large quantities of self-renewing, neural stem cells from human embryonic stem cells. These cells can become many types of brain cells and showed no increased risk of forming tumors, a problem that has plagued other efforts.

This is a big deal. Stem cells have the enticing potential to heal injuries and treat disease, but it has been difficult to produce enough stable cells for clinical use. The ability to create large quantities of neural stem cells brings us a step closer to treatments.

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Diagnosing Alzheimer’s Earlier

by on April 27, 2011 at 9:59 am | 0 Comments
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Dr. Stuart Lipton

Dr. Stuart Lipton

For the first time in nearly 30 years, new criteria will guide the diagnosis of Alzheimer’s disease. The current approach focuses on an individual’s cognitive decline – primarily thinking, learning and memory. But new research has shown that changes in the brain happen long before these symptoms emerge, perhaps even decades earlier.The new guidelines, issued in April by the National Institute on Aging and the Alzheimer’s Association, spotlight the disease’s progression from its earliest onset (molecular changes in the brain). The goal is to detect the disease faster in at-risk patients. As research advances, these new diagnostic tools could allow doctors to treat patients proactively to prevent the emergence of physical symptoms.

“These guidelines should help us diagnose Alzheimer’s earlier, which eventually will be very important as new treatments for early intervention are developed,” stated Dr. Stuart Lipton, director of Sanford-Burnham’s Del E. Webb Neuroscience, Aging and Stem Cell Research Center. Dr. Lipton is also a neurologist who sees many Alzheimer’s disease patients in his own clinical practice and is credited with developing memantine (marketed in the United States as Namenda®), the latest FDA-approved Alzheimer’s drug.

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