TAG: "ALS"

UCSF expands access for people with ALS


Santa Rosa satellite clinic, pilot telemedicine initiative offer new options to patients, families.

The ALS Treatment and Research Center, a clinical practice of the Department of Neurology at UC San Francisco and an ALS Association-certified Center of Excellence, is expanding its support for the community of people facing amyotrophic lateral sclerosis (ALS).

In partnership with The ALS Association Golden West Chapter, the center is expanding direct service through a new satellite clinic in Santa Rosa. In addition, the UCSF Department of Neurology is launching a pilot program in telemedicine technology that will eventually allow people with ALS throughout California to access the center’s services from their homes.

ALS, also known as Lou Gehrig’s disease, is a fatal neurodegenerative disease that attacks nerve cells in the spinal cord and brain. People with ALS progressively lose their ability to move, speak, swallow, and eventually their ability to breathe, while all five senses continue to function normally.

ALS can strike men or women of any age, though, for reasons that are not fully understood, military veterans are at twice the risk as the general population. The average life expectancy of a person with ALS is two to five years from diagnosis. There is no known cause and no cure. In the later stages of the disease, the annual costs for home care, coupled with the cost of necessary equipment, can exceed $200,000 per year.

There are only 34 Centers of Excellence certified by The ALS Association in the United States. Each employs multidisciplinary clinic teams of healthcare professionals from several fields— including neurology, nursing, nutrition, physical therapy, occupational therapy, speech therapy and social work — to provide the highest standard of care for people with ALS.

With key support from The ALS Association Golden West Chapter, UCSF began offering a satellite multidisciplinary clinic in Monterey in 2005, to better serve the southern Bay Area and Central Coast ALS community. A second satellite clinic, which will offer services at six-month intervals, has begun operations at 100 Brookwood Ave. in Santa Rosa, to help people with ALS in the northern Bay Area receive services closer to home. The first Santa Rosa clinic was held in March, and the next will be held Oct. 23 and 24.

“Multidisciplinary clinics such as ours at UCSF Medical Center improve the quality of life for people with ALS and prolong survival, but it can be very difficult for families to travel extended distances to come to these clinics,” said Catherine Lomen-Hoerth, M.D., Ph.D., medical director of the ALS Treatment and Research Center at UCSF. “This support from The ALS Association Golden West Chapter for our satellite clinics greatly increases access to care for people throughout the Bay Area living with ALS.”

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New therapeutic target ID’d for ALS


New approach also holds promise for developing treatment of frontotemporal degeneration.

A team of scientists led by researchers from the UC San Diego School of Medicine and Ludwig Institute for Cancer Research have identified a novel therapeutic approach for the most frequent genetic cause of ALS, a disorder of the regions of the brain and spinal cord that control voluntary muscle movement, and frontotemporal degeneration, the second most frequent dementia.

Published ahead of print in last week’s online edition of the journal PNAS, the study establishes using segments of genetic material called antisense oligonucleotides – ASOs – to block the buildup and selectively degrade the toxic RNA that contributes to the most common form of ALS, without affecting the normal RNA produced from the same gene.

The new approach may also have the potential to treat frontotemporal degeneration or frontotemporal dementia (FTD), a brain disorder characterized by changes in behavior and personality, language and motor skills that also causes degeneration of regions of the brain.

In 2011, scientists found that a specific gene known as C9orf72 is the most common genetic cause of ALS. It is a very specific type of mutation which, instead of changing the protein, involves a large expansion, or repeated sequence of a set of nucleotides – the basic component of RNA.

A normal C9orf72 gene contains fewer than 30 of the nucleotide repeat unit, GGGGCC. The mutant gene may contain hundreds of repeats of this unit, which generate a repeat containing RNA that the researchers show aggregate into foci.

“Remarkably, we found two distinct sets of RNA foci, one containing RNAs transcribed in the sense direction and the other containing anti-sense RNAs,” said first author Clotilde Lagier-Tourenne, M.D., Ph.D., UC San Diego Department of Neurosciences and Ludwig Institute for Cancer Research.

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UC awarded $21M in stem cell grants


Diseases targeted include prostate cancer, autism, ALS and AIDS/HIV.

Alysson Muotri, UC San DIego

Alysson Muotri, UC San DIego

The University of California and its affiliates received seven grants totaling more than $21 million in the latest round of funding from the state’s stem cell agency.

Prostate cancer, autism, ALS and AIDS/HIV are among the diseases targeted by the California Institute for Regenerative Medicine, whose governing board awarded a total of more than $40 million in funding for this round.

Overall, CIRM’s governing board has awarded more than $1.8 billion in stem cell grants, with half of the total going to the University of California or UC-affiliated institutions.

CIRM Early Translation Awards IV:

  • UC Irvine: $4.3 million: Magdalene Seiler
  • UCLA: $13 million: Donald Kohn, Gerald Lipshutz, Robert Reiter, Jerome Zack
  • UC San Diego: $1.8 million: Alysson Muotri
  • UCSF-affiliated J. David Gladstone Institutes: $2.3 million: Steven Finkbeiner

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Scientists ID compounds that target amyloid fibrils in Alzheimer’s


UCLA study is first to use “structural” approach in hunt for amyloid-inhibiting agents.

Compounds binding to amyloid fibrils

Compounds binding to amyloid fibrils

UCLA chemists and molecular biologists have for the first time used a “structure-based” approach to drug design to identify compounds with the potential to delay or treat Alzheimer’s disease, and possibly Parkinson’s, Lou Gehrig’s disease and other degenerative disorders.

All of these diseases are marked by harmful, elongated, rope-like structures known as amyloid fibrils, linked protein molecules that form in the brains of patients.

Structure-based drug design, in which the physical structure of a targeted protein is used to help identify compounds that will interact with it, has already been used to generate therapeutic agents for a number of infectious and metabolic diseases.

The UCLA researchers, led by David Eisenberg, director of the UCLA–Department of Energy Institute of Genomics and Proteomics and a Howard Hughes Medical Institute investigator, report the first application of this technique in the search for molecular compounds that bind to and inhibit the activity of the amyloid-beta protein responsible for forming dangerous plaques in the brain of patients with Alzheimer’s and other degenerative diseases.

In addition to Eisenberg, who is also a professor of chemistry, biochemistry and biological chemistry and a member of UCLA’s California NanoSystems Institute, the team included lead author Lin Jiang, a UCLA postdoctoral scholar in Eisenberg’s laboratory and Howard Hughes Medical Institute researcher, and other UCLA faculty.

The research was published July 16 in eLife, a new open-access science journal backed by the Howard Hughes Medical Institute, the Max Planck Society and the Wellcome Trust.

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Regulating single protein prompts fibroblasts to become neurons


Findings could have implications for developing new treatments for Parkinson’s, Alzheimer’s.

Confocal micrograph of a primary human fibroblast cell grown in culture stained blue for actin, a highly abundant protein that makes up the cytoskeleton of cells.Energy-producing mitochondria are shown in green.

Repression of a single protein in ordinary fibroblasts is sufficient to directly convert the cells – abundantly found in connective tissues – into functional neurons. The findings, which could have far-reaching implications for the development of new treatments for neurodegenerative diseases like Huntington’s, Parkinson’s and Alzheimer’s, will be published online in advance of the Jan. 17 issue of the journal Cell.

In recent years, scientists have dramatically advanced the ability to induce pluripotent stem cells to become almost any type of cell, a major step in many diverse therapeutic efforts. The new study focuses upon the surprising and singular role of PTB, an RNA-binding protein long known for its role in the regulation of alternative RNA splicing.

In in vitro experiments, scientists at the UC San Diego School of Medicine and Wuhan University in China describe the protein’s notable regulatory role in a feedback loop that also involves microRNA – a class of small molecules that modulate the expression of up to 60 percent of genes in humans. Approximately 800 miRNAs have been identified and characterized to various degrees.

One of these miRNAs, known as miR-124, specifically modulates levels of PTB during brain development. The researchers found that when diverse cell types were depleted of PTB, they became neuronal-like cells or even functional neurons – an unexpected effect. The protein, they determined, functions in a complicated loop that involves a group of transcription factors dubbed REST that silences the expression of neuronal genes in non-neuronal cells.

According to principal investigator Xiang-Dong Fu, Ph.D., professor of cellular and molecular medicine at UC San Diego, it’s not known which neuronal signal or signals turn on the loop, which in principle can happen at any point in the circle. But the ability to artificially manipulate PTB levels in cells, inducing them to become neurons, offers tantalizing possibilities for scientists seeking new treatments for an array of neurodegenerative diseases.

It is estimated that over a lifetime, 1 in 4 Americans will suffer from a neurodegenerative disease, from Alzheimer’s and Parkinson’s to multiple sclerosis and amyotrophic lateral sclerosis (Lou Gehrig’s disease).

“All of these diseases are currently incurable. Existing therapies focus on simply trying to preserve neurons or slow the rate of degeneration,” said Fu. “People are working with the idea of replacing lost neurons using embryonic stem cells, but there are a lot of challenges, including issues like the use of foreign DNA and the fact that it’s a very complex process with low efficiency.”

Fu explained that REST is expressed in cells everywhere except in neurons. PTB is itself a target of miR-124, but also acts as a break for this microRNA to attack other cellular targets that include REST, which is responsible for repressing miR-124.

In non-neuronal cells, REST keeps miR-124 down and PTB enforces this negative feedback loop, but during neural induction, miR-124 is induced, which diminishes PTB, and without PTB as a break, REST is dismantled, and without REST, additional miR-124 is produced. This loop therefore becomes a positive feed forward, which turns non-neuronal cells into neurons.

“If we learn how to manipulate PTB, which appears to be a kind of master regulator, we might eventually be able to avoid some of these problems by creating new neurons in patients using their own cells adjacent deteriorating neurons,” said Fu.

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Scientists block toxic protein that plays key role in ALS


Findings suggest therapeutic target for treating fatal disease.

Robert Farese Jr.

Scientists at the UC San Francisco-affiliated Gladstone Institutes and the Stanford University School of Medicine have discovered how modifying a gene halts the toxic buildup of a protein found in nerve cells. These findings point to a potential new tactic for treating a variety of neurodegenerative conditions, including amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease) — a fatal disease for which there is no cure.

The Gladstone and Stanford scientists began their experiments independently before realizing that combining their efforts could strengthen their results. Their discovery — which involved the work of both neuroscientists and geneticists — underscores the importance of collaborative and cross-disciplinary research when dealing with complex neurodegenerative diseases such as ALS.

ALS usually strikes between the ages of 40 and 75, ravaging the body’s motor neurons — nerve cells that control muscle movement. This causes muscle weakness, difficulty swallowing and breathing, paralysis and, ultimately, death — often just three to five years after diagnosis. At any given time, as many as 30,000 Americans are living with ALS — which afflicts physicist Stephen Hawking and which killed baseball legend Lou Gehrig.

In a paper published today (Oct. 29) online in Nature Genetics, researchers in the laboratories of Aaron D. Gitler, Ph.D., associate professor at Standord, and Gladstone senior investigators Robert V. Farese Jr., M.D. and Steve Finkbeiner, M.D., Ph.D., describe how shutting off a gene called Dbr1 in yeast cells and in neurons obtained from rats can protect both cell types from the toxic effects of TDP-43 — a protein that plays a key role in ALS.

“Mutations in the gene that produces TDP-43 can cause this protein to build up in cells,” said Farese, who is also a professor at UCSF. “Over time, TDP-43 accumulation inside motor neurons can reach toxic levels and bind to RNAs — small bits of genetic material that act as an intermediary between genes and proteins. One theory is that this binding interferes with the RNAs’ normal functions and impairs the overall health of cells. Eventually, the neurons degrade and die, contributing to the rapid progression of ALS symptoms.”

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Common RNA pathway found in ALS, dementia


Discovery reveals set of target genes that could lead to development of new drug treatments.

Principal investigator Gene Yeo, UC San Diego

Two proteins previously found to contribute to ALS, also known as Lou Gehrig’s disease, have divergent roles. But a new study, led by researchers at the Department of Cellular and Molecular Medicine at the UC San Diego School of Medicine, shows that a common pathway links them.

The discovery reveals a small set of target genes that could be used to measure the health of motor neurons, and provides a useful tool for development of new pharmaceuticals to treat the devastating disorder, which currently has no treatment or cure.

Funded in part by the National Institutes of Health and the California Institute for Regenerative Medicine (CIRM), the study is published in the advance online edition of Nature Neuroscience on Sept. 30.

ALS is an adult-onset neurodegenerative disorder characterized by premature degeneration of motor neurons, resulting in a progressive, fatal paralysis in patients.

The two proteins that contribute to the disease — FUS/TLS and TDP-43 — bind to ribonucleic acid (RNA), intermediate molecules that translate genetic information from DNA to proteins. In normal cells, both TDP-43 and FUS/TLS are found in the nucleus where they help maintain proper levels of RNA. In the majority of ALS patients, however, these proteins instead accumulate in the cell’s cytoplasm — the liquid that separates the nucleus from the outer membrane, and thus are excluded from the nucleus, which prevents them from performing their normal duties.

Since the proteins are in the wrong location in the cell, they are unable to perform their normal function, according to the study’s lead authors, Kasey R. Hutt, Clotilde Lagier-Tourenne and Magdalini Polymenidou. “In diseased motor neurons where TDP-43 is cleared from the nucleus and forms cytoplasmic aggregates,” the authors wrote, “we saw lower protein levels of three genes regulated by TDP-43 and FUS/TLS. We predicted that this, based on our mouse studies, and found the same results in neurons derived from human embryonic stem cells.”

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How immune system, inflammation may play role in ALS


UCLA findings may offer new approach to reducing inflammation in Lou Gehrig’s disease.

ALS macrophages

FINDINGS:
In an early study, UCLAresearchers found that the immune cells of patients with amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease, may play a role in damaging the neurons in the spinal cord. ALS is a disease of the nerve cells in the brain and spinal cord that control voluntary muscle movement.

Specifically, the team found that inflammation instigated by the immune system in ALS can trigger macrophages — cells responsible for gobbling up waste products in the brain and body — to also ingest healthy neurons. During the inflammation process, motor neurons, whether healthy or not, are marked for clean-up by the macrophages.

In addition, the team found that a lipid mediator called resolvin D1, which is made in the body from the omega-3 fatty acid DHA, was able to “turn off” the inflammatory response that made the macrophages so dangerous to the neurons. Resolvin D1 blocked the inflammatory proteins being produced by the macrophages, curbing the inflammation process that marked the neurons for clean-up. It inhibited key inflammatory proteins like IL-6 with a potency 1,100 times greater than the parent molecule, DHA. DHA has been shown in studies to be neuroprotective in a number of conditions, including stroke and Alzheimer’s disease.

For the study, the team isolated macrophages from blood samples taken from both ALS patients and controls and spinal cord cells from deceased donors.

IMPACT:

The study findings on resolvin D1 may offer a new approach to attenuating the inflammation in ALS. Currently, there is no effective way of administering resolvins to patients, so clinical research with resolvin D1 is still several years away. The parent molecule, DHA, is available in stores, although it has not been tested in clinical trials for ALS. Studies with DHA are in progress for Alzheimer’s disease, stroke and brain injury and have been mostly positive.

AUTHORS:  

Senior author Dr. Milan Fiala, a researcher in the department of surgery at the David Geffen School of Medicine at UCLA, and first author Guanghao Liu, a UCLA undergraduate student, are available for interviews.

FUNDING:

The study was privately funded by ALS patients.

JOURNAL:

The research appeared in the May 30 edition of the peer‑reviewed American Journal of Neurodegeneration. A copy of the full study is available.

IMAGES:
Color images are available showing how a patient’s own immune cells impact neurons, as seen in the spinal cord of an ALS patient.

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Stem cell-derived neurotransmissions measured


UCLA research could shed light on a variety of neurodegenerative diseases, such as ALS.

Bennett Novitch, UCLA

In an effort to identify the underlying causes of neurological disorders that impair motor functions such as walking and breathing, UCLA researchers have developed a novel system to measure communication between stem cell-derived motor neurons and muscle cells in a Petri dish.

The study provides an important proof of principle that functional motor circuits can be created outside the body using these neurons and cells and that the level of communication, or synaptic activity, between them can be accurately measured by stimulating the motor neurons with an electrode and then tracking the transfer of electrical activity into the muscle cells to which the neurons are connected.

When motor neurons are stimulated, they release neurotransmitters that depolarize the membranes of muscle cells. This allows calcium and other ions to enter the cells, causing them to contract. By measuring the strength of this activity, one can get a good estimation of the overall health of motor neurons.

That estimation could shed light on a variety of neurodegenerative diseases, such as spinal muscular atrophy and amyotrophic lateral sclerosis (Lou Gehrig’s disease), in which communication between motor neurons and muscle cells is thought to unravel, said the study’s senior author, Bennett G. Novitch, an assistant professor of neurobiology and a scientist with the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA.

The findings of the study appear May 4 in PLoS ONE, a peer-reviewed journal of the Public Library of Science.

“Now that we have this method to measure the strength of the communications between motor neurons and muscle cells, we may be able to begin exploring what happens in the earliest stages of motor neuron disease, before neuronal death becomes prevalent,” Novitch said. “This can help us to pinpoint where things begin to go wrong and provide us with new clues into therapeutic interventions that could improve synaptic communication and promote neuronal survival.”

Novitch said the synaptic communication activity his team was able to create and measure using muscle cells and motor neurons derived from mouse embryonic stem cells looks very similar to what is seen in a mouse, validating that their model is a realistic representation of what is happening in a living organism.

“That gives us a good starting point to try to model what happens in cells that harbor genetic mutations that are associated with neurodegenerative diseases,” he said. “To do that, we had to first define an activity profile of normal synaptic communication. Some research suggests that a breakdown in this communication can be an early indication of disease progression or possibly an initiating event. Neurons that cannot effectively transmit information to muscle cells will eventually withdraw their contacts, causing both the neurons and muscle cells to degenerate over time. Hopefully, we can now create disease models that will allow us to study what is happening.”

In this study, Novitch and his team, led by Joy Umbach, an associate professor of molecular and medical pharmacology at UCLA, used mouse embryonic stem cells to create the motor neurons, and they used previously established lines of muscle precursors to produce muscle fibers. They put both cells together in a Petri dish, and the cells were cultured in such a way as to encourage communication. Novitch said the team wanted to see if they would naturally form synaptic contacts and whether or not there was neural transmission between them.

In less than a week, the neurons had reached out to the muscle cells and assembled the protein networks needed for synaptic communication, Novitch said.

To measure the connections between the cells, the scientists used a technique called dual patch-clamp recording. Pipettes containing stimulating and recording electrodes are inserted into the membranes of the motor neurons and muscle cells, with special care being taken not to injure them. With this method, the researchers were able send an electrical current into the motor neurons and measure responses in the muscle cells, as well as visualize the muscular contractions.

“The in vitro system developed here might accordingly be expanded to assess the underlying cellular and molecular mechanisms that contribute to this decline in synaptic input to motor neurons,” the study states. “Thus, in addition to their utility for helping to answer fundamental biological questions, these co-cultures have clear applications in addressing problems of medical significance.”

Going forward, Novitch and his team hope to recreate and confirm the work using human stem cell-derived motor neurons and muscle cells and to measure the synaptic communications with newly developed optical recording methods, which are less invasive than the patch-clamp techniques used in this study.

The study was funded by the California Institute for Regenerative Medicine, the UCLA Broad Stem Cell Research Center, the Muscular Dystrophy Association, the UCLA Cellular and Molecular Biology Training Program, and the Ruth L. Kirschstein National Research Service Award.

The Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research: UCLA’s stem cell center was launched in 2005 with a UCLA commitment of $20 million over five years. A $20 million gift from the Eli and Edythe Broad Foundation in 2007 resulted in the renaming of the center. With more than 200 members, the Broad Stem Cell Research Center is committed to a multidisciplinary, integrated collaboration among scientific, academic and medical disciplines for the purpose of understanding adult and human embryonic stem cells. The center supports innovation, excellence and the highest ethical standards focused on stem cell research with the intent of facilitating basic scientific inquiry directed toward future clinical applications to treat disease. The center is a collaboration of the David Geffen School of Medicine at UCLA, UCLA’s Jonsson Cancer Center, the UCLA Henry Samueli School of Engineering and Applied Science and the UCLA College of Letters and Science.

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Insulin resistance, inflammation & a muscle-saving protein


UC San Diego explores complex interactions of lipids, inflammation in insulin resistance.

Christopher Glass, UC San Diego

In the online May 2 issue of the journal Cell Metabolism, researchers at the UC San Diego School of Medicine publish three distinct articles exploring:

  • the complex interactions of lipids and inflammation in insulin resistance
  • the roles of omega 3 fatty acids and a particular gene in fighting inflammation
  • how elevated levels of a particular protein might delay the muscle-destroying effects of amyotrophic lateral sclerosis.

Type 2 diabetes has reached epidemic proportions around the world, fueled in large part by the equally alarming expansion of obesity as a global health problem. But while it’s well-known that obesity is the most common cause of insulin resistance – the primary metabolic abnormality in type 2 diabetes – researchers have only recently begun to effectively parse the underlying, complicated relationships between lipids (fats and related molecules essential to cell structure and function) and chronic tissue inflammation (a key cause of obesity-induced insulin resistance).

In a wide-ranging perspective article published in Cell Metabolism, Christopher K. Glass, M.D., Ph.D., a professor in the departments of cellular and molecular medicine, and medicine at the UC San Diego, and Jerrold M. Olefsky, M.D., associate dean for scientific affairs and distinguished professor of medicine at UC San Diego, survey where the science stands, describing, for example, the pro-inflammatory effects of saturated fatty acids and the anti-inflammatory benefits of omega 3 fatty acids. They also discuss how inflammation impacts lipid metabolism at the cellular, tissue, organ and whole-body levels.

In a second, related article, Olefsky and colleague Da Young Oh, an assistant project scientist, discuss the critical role of a gene called GPR120 in inhibiting pro-inflammatory macrophages while simultaneously boosting the anti-inflammatory benefits of omega 3 fatty acids. They argue that new research highlights the importance of GPR120 as an attractive target for new drugs that could increase insulin sensitivity and, perhaps, have anti-obesity effects as well.

Finally, Don W. Cleveland, Ph.D., professor and chair of the Department of Cellular and Molecular Medicine and head of the Laboratory of Cell Biology at the Ludwig Institute for Cancer Research at UC San Diego and colleagues report the effects of elevated levels of a gene- regulating protein in mouse cells afflicted by a form of amyotrophic lateral sclerosis or ALS.

In humans, ALS is a progressive, adult-onset neurodegenerative disorder characterized by selective motor neuron and muscle loss that ultimately results in fatal paralysis. Among the key players in muscle function is a transcriptional activator protein called PGC-1alpha, which helps enhance various aspects of muscle cell function, including metabolism and mitochondrial biogenesis.

Cleveland and colleagues report that elevated levels of PGC-1alpha in the muscles of a mouse model of inherited ALS helps maintain health and function, though it does not extend survival time. The researchers suggest that increasing PCG-1alpha activity in muscle could be a new and attractive therapeutic target for maintaining, improving and extending physical abilities in ALS patients.

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Combined Health Agencies honors four UC San Diego health heroes


Winners include David Barba, Rohit Loomba, William Mobley, Howard Taras.

David Barba

Brain stimulation surgery for patients with Parkinson’s disease; promoting liver health on a national level; leading one of the nation’s top ALS clinics; and designing a law that protects the rights of students with epilepsy: these are significant reasons why four UC San Diego School of Medicine doctors were honored during the 18th annual Combined Health Agencies Health Hero Awards breakfast on March 15 at The Prado in Balboa Park.

Each year, the Combined Health Agencies’ 24 health nonprofit members each choose a person or company that works daily to improve the lives of local residents affected by chronic illness. This year, four winners who were recognized are UC San Diego physicians David Barba, M.D.; Rohit Loomba, M.D.; William Mobley, M.D., Ph.D.; and Howard Taras, M.D.

Since 2005, Barba, clinical professor of surgery in the Division of Neurological Surgery at UC San Diego Health System, has been involved with the Parkinson’s Association of San Diego. He routinely performs brain stimulation surgery on many patients with Parkinson’s disease and has demonstrated hisleadership by organizing a sold-out patient symposium securing top quality speakers in the field. Barba is currently establishing a UC San Diego system for those working on Parkinson’s research to be in direct contact with each other.

The American Liver Foundation considers Loomba, assistant professor of clinical medicine in the Division of Gastroenterology and the Division of Epidemiology in the Department of Family and Preventive Medicine, a collaborative partner as he serves on the National Board of Directors, and the non-profit local Speakers Bureau promoting prevention and care.

As Chair of the Department of Neurosciences at UC San Diego School of Medicine, Mobley garners national support from physicians and clinicians to join the UC San Diego ALS and Motor Neuron Treatment and Research Center team to raise the level of care and treatment of patients with ALS in San Diego. Through Mobley’s reputation and expertise, the ALS Clinic is quickly becoming known as a place where patients can receive the best care possible in their fight against what is commonly known as Lou Gehrig’s disease.

Taras, professor of pediatrics in the Division of Child Development and Community Health, is being recognized by the Epilepsy Foundation for his instrumental work in the passing of SB 161, a bill signed into law in 2011 that protects the rights of students with epilepsy. He has testified numerous times at California State Legislative hearings and spent hundreds of hours educating legislators and the public about the issue of emergency seizure rescue medications. Through this legislation, life-saving medication can be administered to students at school to prevent further brain damage or death.

“We are humbled by the service of these physicians and grateful to have UC San Diego Health System in our community,” said Susan Day, president of Combined Health Agencies.

This year’s event is possible by the generous support of community sponsors UC San Diego Health System, PhRMA, GlaxoSmithKline, Johnson & Johnson, BIOCOM, Rady Children’s Hospital-San Diego, Sonnenberg & Company CPAs, and The San Diego Business Journal.

Combined Health Agencies has been United Way’s health partner in the United Way/CHAD Campaign since 1974. As a federation of 24 local health charities, Combined Health Agencies is focused on improving the quality of life for individuals and families who are faced with chronic health conditions.

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UCSF ALS Center’s founding director dies of disease he studied


Richard Olney dies at 64.

Richard Olney

Richard K. Olney, M.D., founding director of the ALS Treatment and Research Center at UC San Francisco and a pioneer in clinical research on amyotrophic lateral sclerosis (ALS), has died at age 64, following his own eight-year battle with the disease.

Olney used the tragic irony of his diagnosis – which occurred 18 years after he began focusing on ALS as a physician and researcher – to create public awareness about the condition. He did many interviews with the national and local press beginning in 2004, the year he was diagnosed, even as his condition deteriorated dramatically.

ALS, commonly known as Lou Gehrig’s disease, is a progressive, fatal disease in which motor neurons in the brain and spinal cord degenerate. Patients gradually lose control of their muscles, while their minds generally remain intact.

Current research focuses on what genetic and environmental factors may make people susceptible to the disease, which is not contagious. Olney did not have a family history of ALS.

Throughout his 25-year career, most of it at UCSF, Olney, UCSF professor of neurology, was interested in the diagnosis and management of motor neuron diseases, which include ALS. He joined the UCSF faculty in 1985 and his increasing clinical focus on ALS led to his establishment of UCSF’s ALS clinic in 1993, with one nurse and a physical therapist.

In 1999, the clinic became the UCSF ALS Center and Olney served as its first director, a post he held until August 2004 when the disease’s progression forced him to resign.

Under his leadership, the center grew to its current caseload of more than 375 patients, providing services in multiple disciplines, involving a neurologist, a nurse, a physical therapist, a respiratory therapist, an occupational therapist, a speech pathologist, a dietician, a communication specialist, a social worker and representatives from the ALS Association and the Muscular Dystrophy Association (MDA), who advise patients and families about available support programs.

In 2001, the center received the prestigious designation of “ALS Center of Excellence,” from the ALS Association and the MDA, making it one of only 16 such centers in the United States at the time. The designation, held by only two centers in California, is granted to those programs that offer advanced diagnostics and comprehensive patient care, provide access to the latest drug trials, and conduct clinical research aimed at identifying therapies.

Olney was known internationally for his excellence as a physician, teacher and clinical researcher. In his role as an investigator, he studied potential drug therapies and the disruption in nerve signaling that occurs in ALS and other neuromotor diseases. He refined the statistical method for measuring the rate of ALS progression and, together with UCSF neurologists Catherine Lomen-Hoerth, M.D., Ph.D., and Bruce Miller, M.D., director of the UCSF Memory and Aging Center, defined the links between ALS with features of other neurodegenerative diseases, such as fronto-temporal dementia (FTD).

In the year before his diagnosis, Olney and Lomen-Hoerth – now the director of the ALS Treatment and Research Center at UCSF and Olney’s personal physician – developed a clinical trial to investigate whether two drugs used to combat AIDS and cancer might slow or halt the progression of ALS in some patients.

In January 2005, Olney became the first test subject in this clinical trial. Because it was a placebo-controlled, “blinded” study, neither he nor Lomen-Hoerth, would know for six months if he was receiving the drug.

“It was typical of Rick to put the value of the medical research before himself and not take the drugs outside the boundaries of the trial,” Lomen-Hoerth said. “He knew it was highly unlikely that a treatment would be found during his lifetime, but nothing was going to stop him from doing whatever he could to advance the research.”

When the blinded period of the clinical trial ended, Olney and Lomen-Hoerth learned he had received it. “It may have helped,” Lomen-Hoerth said. “It’s hard to know. Early-stage clinical trials like this involve low doses that are designed to test drug safety, as opposed to efficacy.”

“He’s an inspiration to a whole generation of students who studied under him,” she added. Lomen-Hoerth completed her National Institutes of Health-funded post-doctoral fellowship at UCSF under Olney’s mentorship in 1999.

“His life and career inspires us all to redouble our efforts to find answers to this cruel neurological problem, and to continue to provide best patient care for our patients with ALS,” said Stephen L. Hauser, M.D., chairman of the UCSF Department of Neurology. “The ALS Center at Moffitt-Long Hospital, and the new Neuroscience Research Building at Mission Bay, will carry on in Rick’s magnificent tradition. He always will be remembered as an inspiration to physicians and scientists of UCSF and beyond who are dedicated to finding answers to ALS. We were very lucky to have had him in our midst.”

Lucie Bruijn, Ph.D., chief scientist of the ALS Association, remembered Olney “not only as a courageous person with ALS, but someone who was an outstanding clinician and scientist who made major contributions both for patients and the scientific field.

“He has been an inspiration to those of us who work every day toward the goal of finding meaningful therapies for ALS. It is an honor to have known him personally and the ALS Association is proud to have funded his important studies to identify genetic and environmental influences that impact the disease.”

In 2005, Olney helped to raise awareness about ALS by appearing in a public service announcement produced by the ALS Association.

Olney is survived by his wife of 38 years, Paula, and two children, Amy Koch Olney Dobbs (husband Ryan Dobbs), an occupational therapist at California Pacific Medical Center, and Nicholas T. Olney (wife Caroline Olney) and grandson Richard Knox Olney, all of the Bay Area. Nicholas, inspired by his father’s illness, decided to become a physician. He graduated from UCSF School of Medicine last spring, is carrying out his internship in internal medicine, and will conduct his residency in neurology at UCLA.

Even though Olney became significantly immobilized by his disease, having lost virtually all muscle control and communicating only by moving his pupils across a computer tablet, he remained actively engaged in his family, watching his two children marry, serving as his son’s best man, and participating in the life of his grandchild, who was born last spring and named in his honor.

Olney was the author of numerous research articles, associate editor of Muscle Nerve, and a member of the editorial boards forAnnals of Neurology, Clinical Neurophysiology, and the Journal of Clinical Neurophysiology. He also served as a member of the board of directors of the American Association of Electrodiagnostic Medicine and as a councilor of the neuromuscular section of the American Academy of Neurology.

Olney graduated Phi Beta Kappa from the University of Oklahoma with a bachelor’s degree in Chemistry, Mathematics, and Zoology with highest honors in 1968. He received his medical degree from Baylor College of Medicine in Houston in 1973. He attended UCLA for his training in psychiatry and University of Oregon Health Sciences Center for his training in neurology.

A private memorial service will be held.

Donations to the ALS Center at UCSF should be made payable to the UCSF Foundation, Box 45339, San Francisco, CA 94145-0339. The memo line should state: “Rick Olney ALS Endowment (S0406).”

UCSF is a leading university that advances health worldwide by conducting advanced biomedical research, educating graduate students in the life sciences and health professions, and providing complex patient care.

Related news:

Sampling of the many news articles featuring Richard Olney following his diagnosis:

San Francisco Chronicle
“Dr. Richard Olney in last stages, study of disease”
April 25, 2011
www.sfgate.com/cgi-bin/article.cgi?f=/c/a/2011/04/25/MNHL1J5LEV.DTL

San Francisco Chronicle
“Cruel irony – Gehrig’s disease expert stricken
Shocking diagnosis after decades caring for ALS patients”
Nov. 29, 2004
www.sfgate.com/cgi-bin/article.cgi?f=/c/a/2004/11/29/MNGQ4A35ST1.DTL

The New York Times
“Longtime expert on ALS now knows it all too well”
Feb. 22, 2005
www.nytimes.com/2005/02/22/health/22als.html

People magazine
“Stricken by the disease he was trying to cure”
March 28, 2005
www.people.com/people/archive/article/0,,20147225,00.html

CBS Sunday Morning
The Doctor and the disease
“Doctor battles ALS, the disease he spent a lifetime researching”
May 22, 3005
www.cbsnews.com/stories/2005/05/22/sunday/main697074.shtml

San Francisco Chronicle
“Researcher with ALS finds solace in expertise”
March 22, 2008
www.sfgate.com/cgi-bin/article.cgi?f=/c/a/2008/03/22/MNH7VK56V.DTL

San Francisco Chronicle
“Dying doctor’s noble choice Stricken SF neurologist enters own ‘placebo’ trial”
Jan. 17, 2005
www.sfgate.com/cgi-bin/article.cgi?f=/c/a/2005/01/17/MNG24ARHTM1.DTL

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