New Autism Care Rules Require Some To Pay For Once Free Services

PITTSBURGH (KDKA) Parents of children with autism are protesting new rules that took effect today in Pennsylvania, which will require parents at certain income levels to partially pay for services that used to be free.

Nicki Gratton, of Plum, is one of them. She has three children. Jeremy has cerebral palsy and Riley has pervasive developmental disorder, which is a type of autism.

Gratton told KDKA-TVs Dr. Maria Simbra that three home visits per week to help her with her childrens behavior issues have been a God-send.

The Pennsylvania Department of Public Welfare used to provide these services at no charge for 48,000 children with mental and physical disabilities.

However, families earning more than $45,000 a year will now have to pay co-pays ranging from $1 to $3 per service. Items such as medical equipment, physical, occupational and speech therapy and outpatient services are exempt.

Cathy Hughes with Family Behavioral Resources, which provides services now subject to co-pays, said the change could be devastating.

Im taking calls and e-mails night and day from families that are going to pay between $400 to $700 a month and theyre going to have to make huge sacrifices, Hughes said.

She also worries about her agency and others having to lay off staff, or parents quitting their jobs so they fall under the poverty level and qualify for free services.

Welfare Secretary Gary Anderson said adding the co-pays was necessary to keep services afloat.

Were growing in the department at eight percent a year, and state revenues are only growing at two percent. In order for us to be able to sustain it for the long haul, the families need to start to pay something for their health care, said Anderson.

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New Autism Care Rules Require Some To Pay For Once Free Services

Understanding aging: Stem cell dysfunction links cancer and aging

ScienceDaily (Oct. 1, 2012) Aging is a complex biological process whereby the functional capacity of the body diminishes with time, ultimately leading to the death of the individual. However, aging is also associated with the onset of many diseases, including cancer, which is often called a "disease of aging." While aging has major effects on the individual, it also represents a significant burden on society as a major healthcare cost. Therefore, it is of chief importance to understand the normal process of aging to help improve not only the lifespan of individuals, but also their healthspan; in other words, to enable people to live longer, healthier lives.

Despite significant worldwide research, the causes of aging remain poorly understood. In particular, why the body undergoes a functional decline over the course of time is not entirely clear. Now, a new study from researchers at the CRG has uncovered a significant clue in understanding how aging may occur, and how this may promote the development of diseases such as cancer.

In this study, the researchers studied the skin of young and old mice, as the skin is one of the most obvious tissues to undergo aging. Indeed many of the visible features of aging are the result of skin aging, including loss of hair growth, wrinkling and thinning of the skin and a reduced wound-healing ability.

In the skin, as in the rest of the body, the tissue is constantly in a state of turnover, replenishing itself by replacing dead and damaged cells with new healthy ones. To achieve this, each tissue relies on populations of specialized cells known as stem cells. "These cells are unique in their ability, as they are able to grow and differentiate into all the other different cells types in the tissue, as well as tolerating stress and damage better than non-stem cells. This process of rejuvenation and renewal is something that was thought to occur all throughout life" says Jason Doles, the first author on the study and a postdoctoral researcher at the CRG.

In this work, the researchers have studied skin stem cells during the aging process to see if changes in stem cell function might contribute to aging. Their major finding is that during the aging process, skin stem cells actually lose their ability to function properly. "We have discovered that major changes occur in these stem cells during aging, whereby stem cells exhibit impaired growth in older animals as compared to their more youthful counterparts. We also found that the aged stem cells are not able to tolerate stress as well as young stem cells, strongly supporting the idea that changes in stem cell function might actually drive the aging process" says Bill Keyes, group leader of the Mechanisms of Cancer and Aging lab at the CRG and lead author of the study.

The report goes further, uncovering novel processes driving skin stem cell aging, and linking the aging process with diseases such as cancer. In fact, a recent study from the same group, demonstrated that these same stem cells become deregulated during the development of squamous cell carcinoma, a deadly type of skin cancer. The current study performed high-throughput profiling of the aging stem cells and identified a likely cause of the loss of function during aging. They demonstrated that during normal aging, the entire skin changes and produces many different proteins that mediate inflammation, and that it is the abnormal production of these inflammatory-mediators that contributes to the decline of stem cell function. Given that the link between inflammation and the development of cancer has been long known, the current study uncovers important findings on how the two might be linked.

Altogether, these findings help to explain what is likely a major cause of the aging process and how this develops, opening the door for future studies that may help to alleviate aspects of the aging process. But in addition, with the identification of inflammation as a cause of stem cell dysfunction, the study also uncovers likely causes in the development of cancer.

The research has been funded by the Spanish Ministry for Science and Innovation and the Centre for Genomic Regulation (CRG).

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Understanding aging: Stem cell dysfunction links cancer and aging

Stem cells improve visual function in blind mice

ScienceDaily (Oct. 1, 2012) An experimental treatment for blindness, developed from a patient's skin cells, improved the vision of blind mice in a study conducted by Columbia ophthalmologists and stem cell researchers.

The findings suggest that induced pluripotent stem (iPS) cells -- which are derived from adult human skin cells but have embryonic properties -- could soon be used to restore vision in people with macular degeneration and other diseases that affect the eye's retina.

"With eye diseases, I think we're getting close to a scenario where a patient's own skin cells are used to replace retina cells destroyed by disease or degeneration," says the study's principal investigator, Stephen Tsang, MD, PhD, associate professor of ophthalmology and pathology & cell biology. "It's often said that iPS transplantation will be important in the practice of medicine in some distant future, but our paper suggests the future is almost here."

The advent of human iPS cells in 2007 was greeted with excitement from scientists who hailed the development as a way to avoid the ethical complications of embryonic stem cells and create patient-specific stem cells. Like embryonic stem cells, iPS cells can develop into any type of cell. Thousands of different iPS cell lines from patients and healthy donors have been created in the last few years, but they are almost always used in research or drug screening.

No iPS cells have been transplanted into people, but many ophthalmologists say the eye is the ideal testing ground for iPS therapies.

"The eye is a transparent and accessible part of the central nervous system, and that's a big advantage. We can put cells into the eye and monitor them every day with routine non-invasive clinical exams," Tsang says. "And in the event of serious complications, removing the eye is not a life-threatening event."

In Tsang's new preclinical iPS study, human iPS cells -- derived from the skin cells of a 53-year-old donor -- were first transformed with a cocktail of growth factors into cells in the retina that lie underneath the eye's light-sensing cells.

The primary job of the retina cells is to nourish the light-sensing cells and protect the fragile cells from excess light, heat, and cellular debris. If the retina cells die -- which happens in macular degeneration and retinitis pigmentosa -- the photoreceptor cells degenerate and the patient loses vision. Macular degeneration is a leading cause of vision loss in the elderly, and it is estimated that 30 percent of people will have some form of macular degeneration by age 75. Macular degeneration currently affects 7 million Americans and its incidence is expected to double by 2020.

In their study, the researchers injected the iPS-derived retina cells into the right eyes of 34 mice that had a genetic mutation that caused their retina cells to degenerate.

In many animals, the human cells assimilated into mouse retina without disruption and functioned as normal retina cells well into the animals' old age. Control mice that got injections of saline or inactive cells showed no improvement in retina tests.

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Stem cells improve visual function in blind mice

Houston Stem Cell Summit Announces Extraordinary Lineup of Keynote Speakers

HOUSTON, Oct. 1, 2012 /PRNewswire/ --The Houston Stem Cell Summit will host an extraordinary lineup of keynote speakers who represent the most accomplished stem cell scientists, clinicians and entrepreneurs in the United States. Joining these distinguished speakers will be Governor of Texas, Rick Perry, consistent champion of adult stem cell therapies.

(Logo: http://photos.prnewswire.com/prnh/20120831/NY66463LOGO )

The Houston Stem Cell Summit will be held October 26 27 in its namesake city and will highlight the latest therapeutic research regarding the use of adult stem and progenitor cell therapies. The Summit will also provide a forum for entrepreneurs to discuss their latest efforts to commercialize stem cell therapies, and to debate and discuss FDA and other legal and regulatory issues impacting stem cell research and commercialization.

Opening Keynote Address October 26, 2012 Arnold I. Caplan, PhD, Professor of Biology and Professor of General Medical Sciences (Oncology) Case Western Reserve University

Dr. Caplan has helped shape the direction and focus of adult stem cell research and commercialization. Virtually every adult stem cell company and literally tens of thousands of research papers are based on Dr. Caplan's original and ground breaking research. Professor Caplan is considered to be the "father" of the mesenchymal stem cell and first described this progenitor cell in his landmark paper; "Mesenchymal stem cells", Journal of Orthopaedic Research 1991;9(5):641-650. Since that foundational study, Dr. Caplan has published over 360 manuscripts and articles in peer reviewed journals. Dr. Caplan has been Chief Scientific Officer at OrthoCyte Corporation since 2010. In addition, Dr. Caplan co-founded Cell Targeting Inc. and has served as President of Skeletech, Inc. as its founder. He is the recipient of several honors and awards from the orthopedic research community. Dr. Caplan holds a Ph. D. from Johns Hopkins University Medical School and a B.S. in chemistry from the Illinois Institute of Technology.

Summit Keynote Address October 26, 2012 Texas Governor Rick Perry

Governor Perry is the 47th and current Governor of Texas. Governor Perry has long championed the role of medical technologies in building the future of not only Texas, but also the United States. In many ways, his strong advocacy on behalf of research and advanced medical technologies is one of his strongest and as yet underappreciated legacies. In addition to his service to the state of Texas, Governor Perry has also served as Chairman of the Republican Governors Association in 2008 and again in 2011. Despite a rigorous schedule, particularly in the teeth of this election season, Governor Perry has graciously made time to speak and encourage the researchers, patients, companies and physicians who form the fabric and future of the stem cell therapy community.

Texas Medical Center Keynote Address, October 27, 2012 James T. Willerson, MD

Over the course of his career, Dr. James T. Willerson has served as a medical, scientific and administrative leader for each of the major institutions that are the foundation of the Texas Medical Center. Dr. Willerson is currently President and Medical Director, Director of Cardiology Research, and Co-Director of the Cullen Cardiovascular Research Laboratories at Texas Heart Institute (THI). Dr. Willerson was appointed President-Elect of THI in 2004 and became President and Medical Director in 2008. He is also an adjunct professor of Medicine at Baylor College of Medicine and at The University of Texas MD Anderson Cancer Center. He is the former chief of Cardiology at St. Luke's Episcopal Hospital and the former chief of Medical Services at Memorial Hermann Hospital.

Dr. Willerson has served as a visiting professor and invited lecturer at more than 170 institutions.

The rest is here:
Houston Stem Cell Summit Announces Extraordinary Lineup of Keynote Speakers

World Renowned Scientists and Advocates to Celebrate and Shine Light on Stem Cell Breakthroughs

IRVINE, CA--(Marketwire - Oct 1, 2012) - Oct. 3 marks International Stem Cell Awareness Day, a global celebration where leading scientists, researchers and supporters will acknowledge the scientific advances of stem cell research and its ability to potentially treat a variety of diseases and injuries in the 21st century. This dedicated community is committed to unlocking the potential of stem cells and has made significant strides since the discovery of a method to grow human stem cells less than 15 years ago.

"This is a critical and historic time for stem cell research," said Peter Donovan, Ph.D., director, Sue & Bill Gross Stem Cell Research Center, UC Irvine. "We're literally on the brink of developing new treatments for some of the world's most devastating diseases and injuries. The act of simply raising awareness about this research is one of the best things people can do to help accelerate the process. This event is a great opportunity for everyone to help spread the word and build momentum through a timely mass effort."

Scientists at UC Irvine and other research facilities around the globe continue to work diligently to develop therapies to treat life threatening and debilitating conditions such as Alzheimer's disease, multiple sclerosis, macular degeneration, cancer, Huntington's disease, Parkinson's disease, brain disorders and paralysis caused by spinal cord injuries. These efforts continue to give hope to millions who suffer from these devastating conditions by offering revolutionary treatments and potential cures.

There are several research programs taking place at the Sue & Bill Gross Stem Cell Research Center at UC Irvine that continue to break down barriers and open doors to new treatments for major diseases and injuries:

Spinal Cord and Traumatic Brain Injuries: Neurobiologist Hans Keirstead, Ph.D., as well as husband and wife scientists Aileen Anderson, Ph.D., and Brian Cummings, Ph.D., are conducting stem cell studies to develop treatments for the more than 1.3 million Americans who suffer from spinal cord injuries. Their advancements have led to the world's first clinical trial of human neural stem cell-based therapy for chronic spinal cord injuries (Anderson/Cummings) and the first FDA approved clinical trials using embryonic stem cells (Keirstead). Their research is significant because no drug or other forms of treatment have been able to restore function for those suffering from paralysis. In addition, Cummings and Anderson are applying their stem research to traumatic brain injury, a leading cause of death and disability worldwide, especially in children and young adults.

Alzheimer's Disease: An estimated 35 million people worldwide suffer from Alzheimer's disease, five million of whom live in the U.S. Frank LaFerla, Ph.D., director of UC Irvine's Institute for Memory Impairments and Neurological Disorders, and Matthew Blurton-Jones, Ph.D., of the Sue & Bill Gross Stem Cell Research Center, UC Irvine, have shown for the first time that neural stem cells can rescue memory in mice with advanced Alzheimer's disease, raising hope for a potential treatment in humans. Their work is expected to move to clinical trials in less than five years.

Huntington's Disease: Huntington's disease is a degenerative and ultimately fatal brain disorder that takes away a person's ability to walk, talk and reason. It affects about 30,000 people in the U.S. with another 200,000 or more likely to inherit the disorder. Leslie Thompson, Ph.D., and her team of researchers are currently investigating new stem cell lines and techniques to support the area of the brain that is susceptible to the disease with the hope of developing a cure for future generations.

Macular Degeneration, Retinitis Pigmentosa and Inherited Blindness: Henry Klassen, M.D., Ph.D. has focused his stem cell research on regenerating damaged retinal tissue to restore sight to people suffering from retinitis pigmentosa (an inherited form of degenerative eye disease) and macular degeneration which usually affects older people and leads to loss of vision. Macular degeneration affects millions of Americans. His work hopes to find cures and treatments for corneal and retinal eye disease.

New Website Helps Spread the Word Online To commemorate International Stem Cell Awareness Day and encourage support of stem cell research, an interactive website has been created. Advocates are asked to visit http://www.StemCellsOfferHope.com and share online a wide range of key facts, downloadable images and links to other valuable resources within their social networks.

International Stem Cell Awareness Day Events at UC Irvine The Sue & Bill Gross Stem Cell Research Center at UC Irvine will celebrate International Stem Cell Awareness Day by hosting three special events. An open house will take place on Oct. 1 for high school students. A UC Irvine student, faculty and staff open house will take place on Oct. 2. Finally, an all-day science symposium on Oct. 3 will feature a "Meet the Scientist" interactive forum. The forum and symposium are open to all UC Irvine scientists, clinicians, graduate students, post-docs and members of the community. To RSVP for any these events or for more information, include the name of the event in the subject line and email stemcell@research.uci.edu.

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World Renowned Scientists and Advocates to Celebrate and Shine Light on Stem Cell Breakthroughs

Commentary on Progeria Therapy Trials at the SENS Foundation

Over at the SENS Foundation, you'll find fairly detailed commentary from Michael Rae on the recent news of progress towards a viable therapy for the rare accelerated aging condition progeria. As I've noted in recent years, one of the things learned about the mechanisms of progeria is that they seem to be a greatly exaggerated version of processes that happen in all of us - in the same sense that the runaway mechanisms of Alzheimer's or Parkinson's disease (and many other age-related conditions) take place at low levels in all of us.

So should we do more than keep a weather eye on progeria research? Probably not:

All of us at SENS Research Foundation are inspired by the rapid progress that was made against this tragic disease ... However, it is also important not to read too much into this apparent advance in regards to its implications for the development of new medicines against the diseases and disabilities of aging. In particular, the common characterization of HGPS "progeria" as a disease of "premature aging" leads some to expect that this research has direct implications for the development of rejuvenation biotechnologies, targeting the damage and disabilities of aging.

It is true that the splicing defect responsible for formation of progerin is sporadically active in wild-type cells, and that number of cells in which progerin is present and the level at which it appears do appear to rise with aging. However, such cells are rare enough, and their progerin levels low enough, as to seem highly unlikely to meaningfully contribute to tissue dysfunction with aging, at least within the bounds of a currently-normal lifespan. Additionally, there is evidence that progerin can be turned over in the nuclear lamina, and the causal relationship between the higher prevalence of progerin in aging cells and cellular senescence or disease are not clear, leaving open the possiblity that repair of well-established forms of aging damage may in turn lead to the reversal or obviation of this phenomenon.

Notably, the need to remove "senescent" cells as part of a comprehensive panel of rejuvenation biotechnologies is already clear from first principles, and its potential to ameliorate aspects the frailty and disability of aging has been demonstrated in proof-of-concept rejuvenation research, rendering the specific role of progerin in the process moot. That is, removing "senescent" cells is essential whether progerin accumulation is a cause or a consequence of cellular senescence, and will be equally effective as a regenerative medical therapy against age-related disability in either case.

It is absolutely the case that we'd expect new and interesting challenges to show up once people are living well past the normal human life span. We'd expect to see forms of biological damage that are generally irrelevant over the course of a century turn out to be lethal at two centuries, for example - perhaps nuclear DNA damage, perhaps progerin accumulation, perhaps the fact that some important macromolecules are never normally replaced, perhaps more obscure aggregated metabolic waste products. So largely things we presently know about, can presently ignore, and will have a great deal of time to work on should it turn out to be problem down the line.

Source:
http://www.longevitymeme.org/newsletter/latest_rss_feed.cfm

Shorter People Tend to Live Longer

It is thought that size in humans relates to life expectancy via aspects of metabolism such as growth hormone - less growth hormone means a smaller size but longer life in mammal species. Ames dwarf mice are an example of this taken to an extreme through genetic engineering, lacking growth hormone but living more than 60% longer than their peers.

From an evolutionary perspective, an abundance of food and good health in early life or gestation is thought to trigger a more aggressive front-loading of growth and fertility - which comes at the cost of faster decline once an individual is beyond their reproductive lifespan:

Sardinians have been studied extensively looking for clue to long lifespan. In the current study researchers analyzed the role of a person's height in their eventual lifespan. The researchers analyzed the height of men when they entered the military at age 20 between the years of 1866 and 1915. A total of 685 subjects were analysed. These heights were then related to the persons eventual age at death. It was found that shorter people (shorter than 161.1 cm) lived significantly longer on average than taller people (taller 161.1cm). Furthermore at age 70, taller people lived on average 2 years less than shorter people. At age 70 each quarter inch of height reduced lifespan by one year.

The authors write: In conclusion, shorter people and taller people exhibit differences in longevity. Although a tall body generally reflects abundant nutrition and good living conditions during the growth period, this height has negative ramifications as well. Biological mechanisms indicate that a larger body places greater stress on cells, tissues, and organs, which can reduce longevity.

Link: http://extremelongevity.net/2012/09/26/shorter-people-live-longer/

Source:
http://www.longevitymeme.org/newsletter/latest_rss_feed.cfm

Rate of Increase of Short Telomeres Predicts Longevity in Mammals

Telomeres are the protective caps at the end of chromosomes. They shorten with cell division, and so are part of the clock which decides when a cell reaches the Hayflick limit and ceases dividing. There is much more to it than this, however: telomere length across all the cells in a piece of living tissue is dynamic, as there are processes that lengthen telomeres as well - such as the activity of telomerase.

In general average telomere length erodes with age, reflecting the progressive breakdown of the body's ability to maintain itself - but this proceeds quite differently in different tissues and different species. It can even be reversed in the short term if the health of an individual improves, though in the long term the overall progression is still downhill.

Shorter average telomere length has been correlated with measures of health in statistical studies, but data allowing prediction of longevity for an individual has proven elusive to date. Here, however, a more sophisticated measure of telomere dynamics is show to be predictive of life span in individual mice:

Aberrantly short telomeres result in decreased longevity in both humans and mice with defective telomere maintenance. Normal populations of humans and mice present high interindividual variation in telomere length, but it is unknown whether this is associated with their lifespan potential. To address this issue, we performed a longitudinal telomere length study along the lifespan of wild-type and transgenic telomerase reverse transcriptase mice.

We found that mouse telomeres shorten ?100 times faster than human telomeres. Importantly, the rate of increase in the percentage of short telomeres, rather than the rate of telomere shortening per month, was a significant predictor of lifespan in both mouse cohorts, and those individuals who showed a higher rate of increase in the percentage of short telomeres were also the ones with a shorter lifespan. These findings demonstrate that short telomeres have a direct impact on longevity in mammals, and they highlight the importance of performing longitudinal telomere studies to predict longevity.

Link: http://dx.doi.org/10.1016/j.celrep.2012.08.023

Source:
http://www.longevitymeme.org/newsletter/latest_rss_feed.cfm

An Update on Myostatin Research

Based on what we know today, inhibition of myostatin in muscle tissue looks like one of the few win-win, all-round beneficial alterations that could be made to human metabolism. Lacking myostatin, a mutation that occurs naturally in very rare cases, an individual has much more muscle, less fat, and resistance to some of the common issues that occur with aging - though it is unclear as to how much of that latter benefit stems from an extended ability to exercise and the comparative lack of visceral fat. A sedentary lifestyle and excess visceral fat are both very bad for you over the long term, causing a shorter life expectancy and greater risk of many forms of age-related disease and disability.

Myostatin inhibitors are under investigation as the potential basis for therapies to slow or reverse the progressive loss of muscle mass and strength that occurs with age, a condition known as sarcopenia. The physical frailty of aging is something of a self-reinforcing downward spiral, and addressing even just the muscle strength component of this decline could bring noteworthy benefits.

Research into myostatin dovetails with research into the decline of stem cells with aging, such as the satellite cells in muscle. The fading activity of the satellite stem cell populations that support muscle tissue is thought to be one contributing cause of sarcopenia. Others range from chronic inflammation through to a progressive inability to make proper use of leucine in the diet.

There is no claim that inhibition of myostatin will address the root causes of sarcopenia: it is more a matter of dialing up the "build muscle" switch to levels that do not normally occur as a way of compensation. As a method of doing so it seems to cause no undue complications - which is a good thing and sadly very rare due to the overwhelming complexity of our biology - but it is nonethless far from ideal. In that ideal world, we'd want all therapies (for aging or otherwise) to tackle root causes rather that patch over symptoms, but sometimes you take what you can get.

In any case, here is an update from the world of myostatin research with some additional information on how things tie together under the hood:

Blocking myostatin function in normal mice causes them to bulk up by 25 to 50 percent. What is not known is which cells receive and react to the myostatin signal. Current suspects include satellite cells and muscle cells themselves. In this latest study, researchers used three approaches to figure out whether satellite cells are required for myostatin activity. They first looked at specially bred mice with severe defects in either satellite cell function or number. When they used drugs or genetic engineering to block myostatin function in both types of mice, muscle mass still increased significantly compared to that seen in mice with normal satellite cell function, suggesting that myostatin is able to act, at least partially, without full satellite cell function.

...

to further confirm their theory that myostatin acts primarily through muscle cells and not satellite cells, the team engineered mice with muscle cells lacking a protein receptor that binds to myostatin. If satellite cells harbor most of the myostatin receptors, removal of receptors in muscle cells should not alter myostatin activity, and should result in muscles of normal girth. Instead, what the researchers saw was a moderate, but statistically significant, increase in muscle mass. The evidence once again, they said, suggested that muscle cells are themselves important receivers of myostatin signals. ... since the results give no evidence that satellite cells are of primary importance to the myostatin pathway, even patients with low muscle mass due to compromised satellite cell function may be able to rebuild some of their muscle tone through drug therapy that blocks myostatin activity.

"Everybody loses muscle mass as they age, and the most popular explanation is that this occurs as a result of satellite cell loss. If you block the myostatin pathway, can you increase muscle mass, mobility and independence for our aging population? [Our] results in mice suggest that, indeed, this strategy may be a way to get around the satellite cell problem."

So myostatin inhibition continues to look like a promising form of patch, in that it fails to address root causes but nonetheless produces meaningful benefits with few if any unwanted side-effects - which is more than can be said for many other forms of patch either in operation or under development in the world of medicine.

Source:
http://www.longevitymeme.org/newsletter/latest_rss_feed.cfm

Overexpressing Fatty-Acid-?-Oxidation-Related Genes Extends Fly Lifespan

Researchers here investigate another portion of the mechanisms of metabolism that are influenced by calorie restriction and many of the known longevity genes. This sort of discovery helps to fill in a very complicated landscape of intertwining effects and controllers of effects - at some point in the not too distant future the research community will be able to set out a complete map of how all of the longevity genes and known ways to extend life in laboratory animals relate to one another and work through an overlapping set of mechanisms:

In this study, we demonstrated that the overexpression of fatty-acid-?-oxidation-related genes extended median and maximum lifespan [in flies] and increased stress resistance, suggesting that the level of fatty-acid ?-oxidation regulates lifespan.

Consistent with our results, many investigations have suggested fatty-acid ?-oxidation as a lifespan determinant. One of the well-known longevity-candidate genes, AMPK reportedly regulates fatty-acid synthesis and oxidation. Moreover, calorie restriction and [insulin/insulin-like growth factor (IGF) signaling (IIS)] have been reported to promote fatty-acid ?-oxidation. In addition, enigma mutant, which exhibits oxidative stress resistance and a longevity phenotype, was found to encode a fatty-acid-?-oxidation related enzyme. ... However, the present study is the first to provide direct evidence that the modulation of fatty-acid-?-oxidation components extends lifespan.

Our data showed that lifespan extension by dietary restriction decreased with the overexpression of fatty-acid ?-oxidation-related genes, indicating that lifespan extension by fatty-acid-?-oxidation components is associated with dietary restriction. It was previously reported that calorie restriction increased whole-body-fat oxidation. Energy deprivation subsequent to calorie restriction activates AMPK, which subsequently enables the increase of fatty-acid oxidation necessary to utilize the energy resource. These findings suggested that fatty acid oxidation and dietary restriction are related by same underlying mechanisms.

Link: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3446750/

Source:
http://www.longevitymeme.org/newsletter/latest_rss_feed.cfm

The cost of clinical trial data bias/loss, FDA's new job and the need for bold leadership.


The scandal of clinical trial data loss is eroding the fundamentals of evidence-based research and clinical medicine.


Before you right this post off as the stuff of conspiracy theories, fear-mongering, and 'alternative world views' consider that this view is shared by the likes of the FDA, the International Committee of Medical Journal Editors, the Cochrane Collaboration, and researchers at institutions like Johns Hopkins School of Medicine.


Here's the underlying premise as succinctly described by author Ben Goldacre:

"Drugs are tested by the people who manufacture them, in poorly designed trials, on hopelessly small numbers of weird, unrepresentative patients, and analysed using techniques that are flawed by design, in such a way that they exaggerate the benefits of treatments. Unsurprisingly, these trials tend to produce results that favour the manufacturer.

When trials throw up results that companies don't like, they are perfectly entitled to hide them from doctors and patients, so we only ever see a distorted picture of any drug's true effects. Regulators see most of the trial data, but only from early on in a drug's life, and even then they don't give this data to doctors or patients, or even to other parts of government. This distorted evidence is then communicated and applied in a distorted fashion."

Authors M. Todwin and J. Abramson summarize it thusly:

"Trials with positive results generally are published more frequently than studies that conclude that a new drug poses greater risks or is no more effective than standard therapy or a placebo. Furthermore, some articles may distort trial findings by omitting important data or by modifying prespecified outcome measures. Lack of access to detailed information about clinical trials can undermine the integrity of medical knowledge."

Here is a great list of very recent resources that may convince you of the merits of this concern:

Yesterday, the US Secretary of Health and Human Services announced (in an FR notice) that the FDA was now charged with ensuring all organizations comply with the heretofore enacted but relatively unenforced  requirement to submit all relevant clinical trial data to http://www.clinicaltrials.gov

For further commentary on this move see the following reports from:
What is abundantly clear to me is that the FDA is left almost powerless - and if not powerless than certainly without sufficient resources - to successfully enforce its new power.  This requires collective industry leadership.  Bold, industry-initiated standards, infrastructure and old-fashioned peer pressure.

Here's what I wish.  

I wish that as a cell therapy industry we - through organizations like ISSCR, ARM, ISCT, etc and leading publishers of some of our leading journals like Regenerative Medicine, Cytotherapy, Cell Stem Cell, Stem Cells, etc - would take a leadership position on an issue like this.

I believe that as a relatively small and nascent sector of the biopharma industry we are more likely capable of collaborating on something important like this than larger, more established [entrenched] and diverse sectors.  Of course it requires the political will and cajones.

The payoff from our sector in taking a leadership role on this issue could potentially be enormous in terms of providing our sector with truly transparent and useful data.  Perhaps even more important would be the public profile such leadership would provide the sector.  Such a move requires bold leadership, pain, and cost but this is the kind of stuff that moves the needle and goes down as critical pivot points in history. 

Just my thought for the day...

--Lee

Source:
http://feeds.feedburner.com/CellTherapyBlog

Anticipated short-term cell therapy industry clinical milestones


What follows is an interesting but not exhaustive list of cell therapy industry clinical milestones we anticipate in the next 3-9 months as selected from the list of cell therapy products we are tracking in late-stage or post-commercial development.  


There are other commercial milestones we are monitoring as well as other clinical milestones we expect to see related to cell therapy products in earlier stages of the development pipeline that are not included below.


CellCoTec (http://www.cellcotec.com)
  • Having completed a trial in Europe of their device to enable POC production of an autologous chondrocyte cellular product in/with a biodegradable, load-bearing scaffold for the treatment of articular cartilage defects, they have now submitted their CE market application.  The CE mark application is under review and they anticipate a response in October.  
  • This device and the potential emergence of Sanofi's MACI in the European market next year may have an impact on Tigenix (EBR:TIG) most directly.



ERYtech Parma (http://www.erytech.com)

  • Their 'pivotal' phase 2/3 trial in Europe of lead product, GRASPA, for the treatment of Acute Lymphoblastic Leukemia (ALL) is scheduled for completion 2H 2012. 


GamidaCell (http://www.gamidacell.com)

  • Their 'pivotal' phase 2/3 trial in the US, Israel, and Europe of lead product, StemEx, for the treatment of leukemia and lymphoma, in joint development with Teva, completed enrollment in February and is scheduled for completion 2H 2012.  They have not been shy about the fact they expect to be in the market in 2013.


Innovacell (http://www.innovacell.com)

  • They raised over 8m Euro in April for a phase 3 trial in Europe for their lead product, ICES13, for the treatment of stress-urinary incontinence which was scheduled for a preliminary clinical data readout in Q4 2012 and be ready for market authorization in 2013. Since announcing the capital raise the company has been stone silent and no clinical trial registry has been filed.  Status unknown.


Miltenyi Biotec (www.miltenyibiotec.com)

  • Their phase 3 trial in Germany of CD133+ cells as an adjunct to CABG surgery for myocardial ischemia or coronary artery disease is scheduled for completion in January.


NovaRx (http://www.novarx.com)

  • Their phase 3 trial in US, Europe, and India of their lead product, Lucanix, for the treatment of advanced Non-small Cell Lung Cancer (NSCLC) following front-line chemotherapy is scheduled in clnicaltrials.gov for completion in October but we have learned they expect their next 'interim analysis' in February.


NuVasive (http://www.nuvasive.com)

  • They have a series of trials scheduled to complete 2H 2012 intended to provide additional clinical data to support its marketing of Osteocel Plus for the treatment of a growing number of orthopedic applications.


Sanofi's Genzyme (http://www.genzyme.com)

  • Having completed their phase 3 trial in Europe of MACI for knee repair (symptomatic articular cartilage defects of the femoral condyle including the trochlea), they expect to file their market authorization application (MAA) in 1H 2013.


Hope that's helpful and gives you a sense some of the late-stage things to watch for in the coming weeks and months.  



--Lee

Source:
http://feeds.feedburner.com/CellTherapyBlog

The cost of clinical trial data bias/loss, FDA’s new job and the need for bold leadership.


The scandal of clinical trial data loss is eroding the fundamentals of evidence-based research and clinical medicine.


Before you right this post off as the stuff of conspiracy theories, fear-mongering, and 'alternative world views' consider that this view is shared by the likes of the FDA, the International Committee of Medical Journal Editors, the Cochrane Collaboration, and researchers at institutions like Johns Hopkins School of Medicine.


Here's the underlying premise as succinctly described by author Ben Goldacre:

"Drugs are tested by the people who manufacture them, in poorly designed trials, on hopelessly small numbers of weird, unrepresentative patients, and analysed using techniques that are flawed by design, in such a way that they exaggerate the benefits of treatments. Unsurprisingly, these trials tend to produce results that favour the manufacturer.

When trials throw up results that companies don't like, they are perfectly entitled to hide them from doctors and patients, so we only ever see a distorted picture of any drug's true effects. Regulators see most of the trial data, but only from early on in a drug's life, and even then they don't give this data to doctors or patients, or even to other parts of government. This distorted evidence is then communicated and applied in a distorted fashion."

Authors M. Todwin and J. Abramson summarize it thusly:

"Trials with positive results generally are published more frequently than studies that conclude that a new drug poses greater risks or is no more effective than standard therapy or a placebo. Furthermore, some articles may distort trial findings by omitting important data or by modifying prespecified outcome measures. Lack of access to detailed information about clinical trials can undermine the integrity of medical knowledge."

Here is a great list of very recent resources that may convince you of the merits of this concern:

Yesterday, the US Secretary of Health and Human Services announced (in an FR notice) that the FDA was now charged with ensuring all organizations comply with the heretofore enacted but relatively unenforced  requirement to submit all relevant clinical trial data to http://www.clinicaltrials.gov

For further commentary on this move see the following reports from:
What is abundantly clear to me is that the FDA is left almost powerless - and if not powerless than certainly without sufficient resources - to successfully enforce its new power.  This requires collective industry leadership.  Bold, industry-initiated standards, infrastructure and old-fashioned peer pressure.

Here's what I wish.  

I wish that as a cell therapy industry we - through organizations like ISSCR, ARM, ISCT, etc and leading publishers of some of our leading journals like Regenerative Medicine, Cytotherapy, Cell Stem Cell, Stem Cells, etc - would take a leadership position on an issue like this.

I believe that as a relatively small and nascent sector of the biopharma industry we are more likely capable of collaborating on something important like this than larger, more established [entrenched] and diverse sectors.  Of course it requires the political will and cajones.

The payoff from our sector in taking a leadership role on this issue could potentially be enormous in terms of providing our sector with truly transparent and useful data.  Perhaps even more important would be the public profile such leadership would provide the sector.  Such a move requires bold leadership, pain, and cost but this is the kind of stuff that moves the needle and goes down as critical pivot points in history. 

Just my thought for the day...

--Lee

Source:
http://feeds.feedburner.com/CellTherapyBlog

Fortune Magazine on California Stem Cell Agency: Warm, Personal and Favorable


California's $3 billion stem cell
research effort today garnered a handsome dollop of favorable
national news coverage– a lengthy piece in Fortune magazine that
spoke of looming stem cell cures and the leading role of the state
stem cell agency.

The article led the Fortune web page online at one point this morning and
likely will be read by tens of thousands of persons, although it was not the cover story on the print product. 
Written by a former senior editor of
the magazine, Jeffrey O'Brien of Mill Valley, Ca., the piece was warm
and personal. He began with the story of his 95-year-old
grandmother and her health issues, ranging from arthritis to macular
degeneration. And he wrote,

“The citizens of California have
spoken. If my grandmother and I had the power to get the rest of the
country to follow, we would.”

O'Brien also discussed the science and
finances of the stem cell business. He said,

“To be clear, the earliest stem cell
therapies are almost certainly years from distribution. But so much
progress has been made at venerable research institutions that it now
seems possible to honestly discuss the possibility of a new medical
paradigm emerging within a generation. Working primarily with rodents
in preclinical trials, MDs and Ph.D.s are making the paralyzed walk
and the impotent virile. A stem cell therapy for two types of macular
degeneration recently restored the vision of two women. Once they
were blind. Now they see!

“Some experts assert that AMD could
be eradicated within a decade. Other scientists are heralding a
drug-free fix for HIV/AIDS. Various forms of cancer, Parkinson's,
diabetes, heart disease, stroke, and ALS have already been eradicated
in mice. If such work translates to humans, it will represent the
type of platform advancement that comes along in medicine only once
in a lifetime or two. The effect on the economy would be substantial.
Champions of stem cell research say it would be on the order of the
Internet or even the transistor.”

O'Brien continued,

“The obstacles along the road from
lab rat to human patients are many, of course, but the biggest by far
is money. With the dramatic events in the lab, you might think that a
gold rush would be under way. That's far from true. Long time
horizons, regulatory hurdles, huge R&D costs, public sentiment,
and political headwinds have all scared financiers. Wall Street isn't
interested in financing this particular dream. Most stem cell
companies that have dared go public are trading down 90% or more from
their IPOs. Sand Hill Road is AWOL. The National Venture Capital
Association doesn't even have a category to track stem cell
investments.”

As for the California stem cell agency
itself, the article contained remarks from its Chairman J.T.Thomas,
President Alan Trounson and former chairman Robert Klein about the origins and progress of the California Institute for Regenerative Medicine (CIRM).
O'Brien wrote, 

“The $1.7 billion awarded so far has made one obvious mark on the state: a dozen gleaming research institutions. CIRM has proved adept at getting billionaires to donate funds to the cause.”

O'Brien interviewed a several
prominent businessmen who have contributed tens of millions of
dollars to stem cell research “about the prospects of a legitimate industry emerging.” One was “bond genius” Bill Gross, who has
contributed to UC Irvine. Gross replied.

“Goodness, you're talking to the
wrong guy. Our donation had nothing to do with business.”

Eli Broad, another big stem cell donor,
said pretty much the same thing. And Andy Grove, the former chairman
of Intel, was “surprisingly full of doom and gloom.” O'Brien
wrote,

“For close to two hours, Grove argues
passionately about how the FDA is enabling predatory offshore
industries by impeding progress and the many reasons financiers want
no part of stem cells. "VCs aren't interested because it's a
shitty business," he says. Big Pharma? Forget it. CIRM? "There
are gleaming fucking buildings everywhere. That wasn't necessary."
When I press him to be constructive, he wearily offers one possible
solution. Rather than courting billionaires to put their names on
buildings, we need a system of targeted philanthropy in which the 99%
can sponsor the individual stem cell lines that matter to them.”

O'Brien said, however,

“It was clear during our talk that
Grove wants an economic model for stem cell research and development
to emerge, even if he's not willing to bet money on its happening.
And that puts him in good company.”

While the Fortune article has its
negative points about stem cell research, it is about as laudatory as
it is going to get at this point for the California stem cell agency.
The piece recognizes and even celebrates much of the work of the
agency. The article clearly details the void in financing
for commercialization of stem cell research, bolstering support for
efforts like those in California. Importantly, it also helps to push
the activities of the stem cell agency more fully into the national
discussion of stem cell research and its future. That should pay off
again and again in future news coverage and also benefit the stem
cell agency as it explores the possibility of additional funding –
either private or public – after the cash for new awards runs out
in 2017.

(The story is in the Oct. 8, 2012, edition of Fortune.)

Source:
http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss

October CIRM Board Meeting Moved to Burlingame


The location of the October meeting of
the governing board of the California stem cell agency has been
changed from Irvine to Burlingame, near San Francisco International
Airport, in an effort to save travel costs.  

CIRM Chairman J.T. Thomas said the
one-day meeting is being moved because the session will require the
attendance of a large number of CIRM staffers who are based in the
agency's San Francisco headquarters. They will be involved in
presentations involving the agency's new strategic partnership fund and other matters.
The date of the meeting remains
unchanged – Oct. 25. Look for posting of the agenda on the CIRM web
site on Oct. 15. The site of the meeting is the Hilton Bayfront
Hotel
, 600 Airport Blvd.

Source:
http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss

$700,000 Blue-ribbon Study of CIRM All But Finished


The $700,000 study of the $3 billion
California stem cell agency is nearly concluded and is expected to be
released sometime in November.

A draft of the report has been sent out
for “peer review” and no additional public meetings are
scheduled, according to a spokeswoman for the Institute of
Medicine(IOM)
, which is conducting the study. The IOM did not respond
to questions from the California Stem Cell Report about the number of peer reviewers or how they were selected.
The study began last year under a contract with the stem cell agency, which commissioned the effort, in
part, because agency directors hoped the findings by the blue-ribbon
panel would bolster efforts to win voter approval of another multi-billion dollar state bond issue. More recently the agency has
explored the possibility of private financing to continue operations.
The agency is expected to run out of
funds for new awards in 2017. It currently has something in the
neighborhood of $700 million for awards that is not already committed
in one fashion or another.
Christine Stencel, senior media
relations officer for the IOM, said in an email,

There will be no
further information-gathering meetings. The committee members have
finished drafting their report and it is now undergoing peer review.
Reviewers are anonymous to study staff and committee members; they
will be listed in the front matter of the report when it’s finished
and released.”

She said the stem
cell agency will not be given an opportunity to comment further.
Stencel said,

Sponsors are not
treated as peer reviewers; that is, they’re not afforded an
opportunity to comment on IOM draft reports prior to public release.
IOM is aiming for a public release in November (the exact time frame
will hinge on the duration of the peer review, which is influenced by
people’s schedules and adherence to deadlines). IOM is looking at
options for how best to hold this release, whether there will be an
event of some sort. Once plans are set, they’ll be noted on the
project web pages and IOM will alert the various stakeholders and
interested parties of the plans. The study is moving along and we’re
looking forward to the report’s debut in the not too distant
future.”

Source:
http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss

$1.5 Billion in Stem Cell Awards Goes to Directors' Institutions


The Sacramento Bee today published an article that reported that $1.5 billion, more than 90 percent of the amount dispensed by the California stem cell agency, has gone to institutions linked to past and present directors of the agency.

The piece was carried on the front page of the newspaper's Sunday Forum section and was written by David Jensen, publisher-editor of the California Stem Cell Report.

The text of the Forum article is below. The Bee also carried a chart listing the top 10 recipient institution. The full text of the comments from Alan Trounson, president of the California stem cell agency,  and two other persons quoted in the article can be found here.

Stem cell cash mostly aids directors' interests

Special to The Bee

By David Jensen

With its latest round of awards earlier this month, California's stem cell agency has now handed out $1.5 billion to enterprises linked to its directors.

The figure amounts to 92 percent of the $1.7 billion awarded by the agency. The grants and loans range from $261 million to Stanford University, whose medical school dean, Philip Pizzo, sits on the agency's governing board, to $170,500 to Children's Hospital in Oakland, whose president, Bert Lubin, also is a member of the board.

The University of California, Davis, has received $128 million. Claire Pomeroy, chief executive officer of UC Davis Health System, is another one of the 29 board members. In all, 27 institutions with past or present representatives on the agency board have received funding.

None of this is illegal. And none of it is likely to change. The situation was created by Proposition 71, the 2004 ballot measure that established the state's $3 billion stem cell agency, formally known as the California Institute for Regenerative Medicine, or CIRM. The initiative was crafted so that virtually all of the institutions that stood to benefit from the state's largesse had seats at the table where the money is handed out.

The built-in conflicts of interest at CIRM have perturbed some experts in California government, but concerns have also reached into the scientific community. The prestigious journal Nature, in 2008, editorialized against what it called cronyism at CIRM. It said the agency "must fight the tendency of the academic institutions on the board to hoard dollars."

Some California scientists, wary of offending those who control the lifeblood of their research, privately grumble about an "old boys network."

Joe Mathews, co-author of "California Crackup," a study of major issues in state government, said last week: "California ballot initiatives are a terrible way to make public policy. And they are even worse as a method for making scientific policy."

The stem cell agency has a different view. Alan Trounson, president of the San Francisco-based enterprise, said: "There is no evidence that any of CIRM's funding decisions have been driven by conflicts of interest. Indeed, CIRM rigorously enforces its conflict of interest rules at each stage of the funding process to ensure that all decisions are made on the merits of the proposal for funding and not as a result of any conflicts of interest."

Mathews, California editor of Zocalo Public Square, and others point to the creation of the California stem cell agency as an example of abuse of the initiative process by special interests. The 10,000 words in Proposition 71 were written in private by Bay Area real estate investment banker Robert Klein and a handful of associates, who quietly determined the composition of the board. Klein later served six years as the first chairman of the stem cell agency, leaving in June 2011.

Klein later argued publicly that placing medical school deans and university and research institution executives on the board provided the expertise needed to make the decisions about how to spend the research money. However, the makeup of the board also served to win the support of institutions that envisioned the prospect of fresh cash – in this case money that the state borrows via bonds.

Mathews described the state's initiative process this way: "Essentially, to win the support of various groups whose money and backing is important to passage of a bond, a sponsor of an initiative bond will set up rules and include money specifically intended for each group. This is a form of pay-to-play. Agree to back the initiative, and you're in."

Bob Stern, who co-wrote the California Political Reform Act, said, "It would have been better had institutions receiving grants not to have had their representatives on the board awarding grants."

Trounson said the board follows "best practices" when it comes to grants and legal conflicts of interest. The agency has worked out an unusual procedure to prevent its directors from violating conflict of interest laws as they vote on applications that seek as much as $20 million each. Before each public session, agency attorneys determine which board members cannot vote on a proposal because of legal conflicts of interest. Applications to be approved are considered as a group. Each board member then votes on the entire group by saying, "Yes, on all those except with which I have a conflict."

No final tally is announced. The public can ferret out the overall vote a month or two later in the minutes of the meeting on the CIRM website (http://www.cirm.ca.gov). But the minutes do not list individual votes or conflicts of interest.

Domination of the board by academics and nonprofit institutions has led to bitter complaints from business. Less than 7 percent of all awards have gone to for-profit enterprises. Currently, however, the agency is embracing industry more warmly in an effort to commercialize stem cell research, which raises another set of coziness problems. They surfaced in July and again this month.

Klein, who led the stem cell ballot campaign before becoming chairman of the agency, appeared before his old board to lobby on behalf of a $20 million request from StemCells Inc. of Newark. The California firm was founded by the eminent Stanford stem cell scientist Irv Weissman. He sits on StemCells Inc.'s board, and he and his wife hold 273,821 shares of stock in the firm. Weissman was also an important backer of Proposition 71, working the "billionaire circuit" and raising more than $1 million for the campaign, according to an article in San Francisco magazine.

CIRM's reviewers had rejected StemCells Inc.'s application. After Klein made his pitch in July, the board sent the application back for re-review, an unusual procedure.

When the application returned to the board early this month, reviewers again rejected it. Klein again importuned his former colleagues, and – following a closed door session – the board approved the award, 7-5.

Eleven members were disqualified from voting because of legal conflicts of interest. It was the first time in the board's eight-year history that it approved an application twice rejected by reviewers.

Mathews said no likelihood exists of changing the board structure at CIRM. He said it is "baked in" by Proposition 71. That's because Klein and company wrote into the initiative a requirement for a super, super-majority vote – 70 percent – of each house of the Legislature to make any modifications.

Another initiative could be mounted, but that possibility is also exceedingly remote. 

Source:
http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss

CIRM Sponsoring Online Session with FDA on Thursday


One of the lesser known activities of
the California stem cell agency is webinars that put researchers
together with the folks who make the federal decisions about whether
stem cell research will be turned into therapies.

One of those sessions is coming up on Thursday, and it is not too late for scientists and other interested
parties to get on board.
Writing on the stem cell agency's blog,
Cynthia Schaffer, CIRM's contract administrator and compliance officer
had this to say today about the webinars.

“The FDA very graciously donates
their time to speak on these webinars because they too have pledged
to maintain an active dialogue with the industry and provide
education on their regulatory expectations for product development in
the regenerative medicine field. CIRM science officer Kevin
Whittlesey
 recently
wrote a paper
with Celia Witten of the FDA about the role of the
FDA in reaching out to regenerative medicine community, including
webinars such as these. 

“In that paper they point out that
the communication goes both ways:

“'Appropriate regulation requires a
strong understanding of the latest scientific developments to meet
current and future regulatory needs and challenges.'

“So the FDA benefits by learning from
the other speakers in the webinar – what is the current state of
the technology, what are investigator’s current thoughts on best
practices and the latest research findings, etc. They also learn what
the industry is facing by listening to the questions asked and the
discussion of the challenges during the Q&A sessions. A group of
FDA employees attend each of these CIRM sponsored webinars, and the
wide variety of other workshops and meetings that CIRM hosts
throughout the year.”  

(Editor's note: An earlier version of this item incorrectly identified Cynthia Schaffer as Cynthia Adams.)

Source:
http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss

$1.5 Billion in Stem Cell Awards Goes to Directors’ Institutions


The Sacramento Bee today published an article that reported that $1.5 billion, more than 90 percent of the amount dispensed by the California stem cell agency, has gone to institutions linked to past and present directors of the agency.

The piece was carried on the front page of the newspaper's Sunday Forum section and was written by David Jensen, publisher-editor of the California Stem Cell Report.

The text of the Forum article is below. The Bee also carried a chart listing the top 10 recipient institution. The full text of the comments from Alan Trounson, president of the California stem cell agency,  and two other persons quoted in the article can be found here.

Stem cell cash mostly aids directors' interests

Special to The Bee

By David Jensen

With its latest round of awards earlier this month, California's stem cell agency has now handed out $1.5 billion to enterprises linked to its directors.

The figure amounts to 92 percent of the $1.7 billion awarded by the agency. The grants and loans range from $261 million to Stanford University, whose medical school dean, Philip Pizzo, sits on the agency's governing board, to $170,500 to Children's Hospital in Oakland, whose president, Bert Lubin, also is a member of the board.

The University of California, Davis, has received $128 million. Claire Pomeroy, chief executive officer of UC Davis Health System, is another one of the 29 board members. In all, 27 institutions with past or present representatives on the agency board have received funding.

None of this is illegal. And none of it is likely to change. The situation was created by Proposition 71, the 2004 ballot measure that established the state's $3 billion stem cell agency, formally known as the California Institute for Regenerative Medicine, or CIRM. The initiative was crafted so that virtually all of the institutions that stood to benefit from the state's largesse had seats at the table where the money is handed out.

The built-in conflicts of interest at CIRM have perturbed some experts in California government, but concerns have also reached into the scientific community. The prestigious journal Nature, in 2008, editorialized against what it called cronyism at CIRM. It said the agency "must fight the tendency of the academic institutions on the board to hoard dollars."

Some California scientists, wary of offending those who control the lifeblood of their research, privately grumble about an "old boys network."

Joe Mathews, co-author of "California Crackup," a study of major issues in state government, said last week: "California ballot initiatives are a terrible way to make public policy. And they are even worse as a method for making scientific policy."

The stem cell agency has a different view. Alan Trounson, president of the San Francisco-based enterprise, said: "There is no evidence that any of CIRM's funding decisions have been driven by conflicts of interest. Indeed, CIRM rigorously enforces its conflict of interest rules at each stage of the funding process to ensure that all decisions are made on the merits of the proposal for funding and not as a result of any conflicts of interest."

Mathews, California editor of Zocalo Public Square, and others point to the creation of the California stem cell agency as an example of abuse of the initiative process by special interests. The 10,000 words in Proposition 71 were written in private by Bay Area real estate investment banker Robert Klein and a handful of associates, who quietly determined the composition of the board. Klein later served six years as the first chairman of the stem cell agency, leaving in June 2011.

Klein later argued publicly that placing medical school deans and university and research institution executives on the board provided the expertise needed to make the decisions about how to spend the research money. However, the makeup of the board also served to win the support of institutions that envisioned the prospect of fresh cash – in this case money that the state borrows via bonds.

Mathews described the state's initiative process this way: "Essentially, to win the support of various groups whose money and backing is important to passage of a bond, a sponsor of an initiative bond will set up rules and include money specifically intended for each group. This is a form of pay-to-play. Agree to back the initiative, and you're in."

Bob Stern, who co-wrote the California Political Reform Act, said, "It would have been better had institutions receiving grants not to have had their representatives on the board awarding grants."

Trounson said the board follows "best practices" when it comes to grants and legal conflicts of interest. The agency has worked out an unusual procedure to prevent its directors from violating conflict of interest laws as they vote on applications that seek as much as $20 million each. Before each public session, agency attorneys determine which board members cannot vote on a proposal because of legal conflicts of interest. Applications to be approved are considered as a group. Each board member then votes on the entire group by saying, "Yes, on all those except with which I have a conflict."

No final tally is announced. The public can ferret out the overall vote a month or two later in the minutes of the meeting on the CIRM website (http://www.cirm.ca.gov). But the minutes do not list individual votes or conflicts of interest.

Domination of the board by academics and nonprofit institutions has led to bitter complaints from business. Less than 7 percent of all awards have gone to for-profit enterprises. Currently, however, the agency is embracing industry more warmly in an effort to commercialize stem cell research, which raises another set of coziness problems. They surfaced in July and again this month.

Klein, who led the stem cell ballot campaign before becoming chairman of the agency, appeared before his old board to lobby on behalf of a $20 million request from StemCells Inc. of Newark. The California firm was founded by the eminent Stanford stem cell scientist Irv Weissman. He sits on StemCells Inc.'s board, and he and his wife hold 273,821 shares of stock in the firm. Weissman was also an important backer of Proposition 71, working the "billionaire circuit" and raising more than $1 million for the campaign, according to an article in San Francisco magazine.

CIRM's reviewers had rejected StemCells Inc.'s application. After Klein made his pitch in July, the board sent the application back for re-review, an unusual procedure.

When the application returned to the board early this month, reviewers again rejected it. Klein again importuned his former colleagues, and – following a closed door session – the board approved the award, 7-5.

Eleven members were disqualified from voting because of legal conflicts of interest. It was the first time in the board's eight-year history that it approved an application twice rejected by reviewers.

Mathews said no likelihood exists of changing the board structure at CIRM. He said it is "baked in" by Proposition 71. That's because Klein and company wrote into the initiative a requirement for a super, super-majority vote – 70 percent – of each house of the Legislature to make any modifications.

Another initiative could be mounted, but that possibility is also exceedingly remote. 

Source:
http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss

Text of Comments on Awards to Stem Cell Directors' Institutions


Here is the full text of comments made
by the California stem cell agency, Joe Mathews, co-author of
California Crack-Up” and Bob Stern, former president of the
Center for Governmental Studies and co-author of the California
Political Reform Act
, in connection with the Sept. 23, 2012, article
in The Sacramento Bee headlined “Stem Cell Cash Mostly Aids Directors' Interests.” The comments were abbreviated for
publication in The Bee because of newspaper space constraints.

Comments by Alan Trounson, president of
CIRM:

“To make sure we do the best job of
managing taxpayer's money it's natural that we turn to people who
know most about stem cells and stem cell research. In fact, as the
state's own Little Hoover Commission reported in its analysis of
CIRM: “The fact that CIRM funding has gone largely to prestigious
California universities and research institutes is hardly surprising
and should be expected, given the goals of Proposition 71 and the
considerable expertise resident in these research centers.” But in
recruiting the best minds, we also adopt best practices to ensure
that there is no conflict of interest. Every board member has to
recuse themselves from voting on, or even being part of a discussion
on anything to do with their own institution, or to an institution or
company that they have any connections to. All this is done in
meetings that are open to the public. CIRM’s conflict of interest
rules have been subject to multiple reviews – by the Bureau of
State Audits, the Little Hoover Commission and the Controller – and
there is no evidence that any of CIRM’s funding decisions have been
driven by conflicts of interest. Indeed, CIRM rigorously enforces its
conflict of interest rules at each stage of the funding process to
ensure that all decisions are made on the merits of the proposal for
funding and not as a result of any conflicts of interest. 

“In addition all funding applications
are reviewed by an independent panel of scientists on our Grants
Working Groups, all of whom are out-of-state and meet strict conflict
of interest requirements, and it is their recommendations that help
guide the ICOC (CIRM governing board) on what to fund.”

Joe Mathews' comments:

“California ballot initiatives are a
terrible way to make public policy. And they are even worse as a
method for making scientific policy. 

“It's not merely that
this initiative was drafted in such a way as to benefit the
enterprises of its directors. It's that, under this initiative's own
provisions and the California constitution, it's so hard to change
Proposition 71 and fix what ails CIRM. Effectively, these provisions are
baked in, and nothing short of another vote of people can really make
the change. (Yes, there are provisions, as you know, that permit the
legislature by super-majority to do things, but supermajorities are
effectively out of reach in California). 

“Sadly, initiatives
like Proposition 71 are not uncommon. Many measures are drafted to benefit
the people who would support the measure, or oversee the program
established. This has been very common with bonds. Essentially, to
win the support of various groups whose money and backing is
important to passage of a bond, a sponsor of an initiative bond will
set up rules and include money specifically intended for each group.
This is a form of pay-to-play. Agree to back the initiative and
you're in. And it happens because there's no rule against it and
because passing initiatives in California require difficult,
expensive campaigns. 

“And this sort of thing will continue
to happen. There is no serious push to do anything about this.
Indeed, good government groups and reformers in California have
opposed changes to the initiative process -- because they want to use
the process for their own schemes.”

Bob Stern's comments:

“It would have been better had
institutions receiving grants not to have had their representatives
on the board awarding grants. On the other hand, we want to have the
most knowledgeable people on the board overseeing this very important
program. The question: Were these people the only qualified ones to
sit on the board?”

Source:
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