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Autism Stem-Cell Therapy to Be Tested in Kids in Trials

August 21st, 2012 4:14 pm

By Ryan Flinn - 2012-08-21T04:01:00Z

Researchers are recruiting autistic children for a study that will test whether injecting stem cells banked from their umbilical cords can lessen symptoms and provide insights into the nature of the disorder.

While stem cells have been promoted, and sold, as a treatment for autism, few clinical trials have been conducted to see whether theyre effective. The study, which begins enrolling patients today, is the first of its kind approved by the U.S. Food and Drug Administration to assess the use of stem cells as a potential autism therapy, said Michael Chez, director of pediatric neurology at Sutter Medical Center in Sacramento, California, and the principal investigator.

About 1 in 88 children in the U.S. are diagnosed with an autism-related condition. The disorder hurts brain development and is linked to poor social interaction and communication skills, repeated body movements, and unusual attachments to objects.

With this study well be able to answer in a firm way that this is truly an observed effect, or we didnt get an observed effect, Chez said in a phone interview.

Thirty children with autism, ages 2 to 7, will be divided in two groups, with one getting the stem cell injection and the other receiving a placebo shot. After six months, the groups will switch. Patients will be monitored for improvement in language as well as irritability and other autism rating scales.

Ricardo Dolmetsch, a neurobiologist at Stanford University in California whose laboratory is studying autism, said he doesnt think the trial will yield much in usable results, though hes glad the idea of using stem cells is being testing.

I commend them for having the guts to actually do it, given that there are all kinds of people out there trying to sell it, he said. On the other hand I dont think its big enough to provide an answer.

Chez theorizes that autism, which has no known cause or cure, may be spurred on by damaged nerve cells. Stem cells, the building blocks of life that can grow into any type of tissue in the body, could repair the damage or create new cells, he said. Such a mechanism would yield results in six to 12 months, the time it takes to create new cells.

Another possibility may be that autism is related to a signaling issue, where cells in the body arent connecting properly. Stem cells may help repair that problem, he said, and would be evident if results are seen within weeks of the injection. A third and more exploratory possibility is the disorder is related to inflammation, an immune system response.

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Autism Stem-Cell Therapy to Be Tested in Kids in Trials

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Stem Cells Can Become Anything – but Not Without This Protein

August 21st, 2012 4:14 pm

Newswise ANN ARBOR, Mich. -- How do stem cells preserve their ability to become any type of cell in the body? And how do they decide to give up that magical state and start specializing?

If researchers could answer these questions, our ability to harness stem cells to treat disease could explode. Now, a University of Michigan Medical School team has published a key discovery that could help that goal become reality.

In the current issue of the prestigious journal Cell Stem Cell, researcher Yali Dou, Ph.D., and her team show the crucial role of a protein called Mof in preserving the stem-ness of stem cells, and priming them to become specialized cells in mice.

Their results show that Mof plays a key role in the epigenetics of stem cells -- that is, helping stem cells read and use their DNA. One of the key questions in stem cell research is what keeps stem cells in a kind of eternal youth, and then allows them to start growing up to be a specific type of tissue.

Dou, an associate professor of pathology and biological chemistry, has studied Mof for several years, puzzling over the intricacies of its role in stem cell biology.

She and her team have zeroed in on the factors that add temporary tags to DNA when its coiled around tiny spools called histones. In order to read their DNA, cells have to unwind it a bit from those spools, allowing the gene-reading mechanisms to get access to the genetic code and transcribe it. The temporary tags added by Mof act as tiny beacons, guiding the reader mechanism to the right place.

Simply put, Mof regulates the core transcription mechanism without it you cant be a stem cell, says Dou. There are many such proteins, called histone acetyltransferases, in cells but only MOF is important in undifferentiated cells.

Dou and her team also have published on another protein involved in DNA transcription, called WDR5, that places tags that are important during transcription. But Mof appears to control the process that actually allows cells to determine which genes it wants to read a crucial function for stem-ness. Without Mof, embryonic stem cells lost their self-renewal capability and started to differentiate, she explains.

The new findings may have particular importance for work on induced pluripotent stem cells the kind of stem cells that dont come from an embryo, but are made from adult tissue.

IPCS research holds great promise for disease treatment because it could allow a patient to be treated with stem cells made from their own tissue. But the current way of making IPSCs from tissue involves a process that uses a cancer-causing gene a step that might give doctors and patients pause.

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Stem Cells Can Become Anything - but Not Without This Protein

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New marker for identifying precursors to insulin-producing cells in pancreas

August 21st, 2012 4:13 pm

Public release date: 21-Aug-2012 [ | E-mail | Share ]

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 x2156 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, August 21, 2012For the millions of people worldwide with type 1 diabetes who cannot produce sufficient insulin, the potential to transplant insulin-producing cells could offer hope for a long-term cure. The discovery of a marker to help identify and isolate stem cells that can develop into insulin-producing cells in the pancreas would be a critical step forward and is described in an article in BioResearch Open Access, a new bimonthly peer-reviewed open access journal from Mary Ann Liebert, Inc. (http://www.liebertpub.com) The article is available free online at the BioResearch Open Access website (http://www.liebertpub.com/biores).

Pancreatic stem cells, the precursors of insulin-producing cells, have not yet been identified in humans or animals, and there is much debate about where they may reside. Ivka Afrikanova, Ayse Kayali, Ana Lopez, and Alberto Hayek, University of California, San Diego, CA, have identified a biochemical markerstage-specific embryonic antigen 4 (SSEA4)that they propose can be used to identify and purify human pancreatic stem cells. The article "Is Stage-Specific Embryonic Antigen 4 a Marker for Human Ductal Stem/Progenitor Cells" (http://online.liebertpub.com/doi/full/10.1089/biores.2012.0235) reports that when grown in culture with high levels of glucose and B27, these SSEA4+ stem cells can differentiate into insulin-producing pancreatic cells.

###

About the Journal

BioResearch Open Access (http://www.liebertpub.com/biores) is a bimonthly peer-reviewed open access journal that provides a new rapid-publication forum for a broad range of scientific topics including molecular and cellular biology, tissue engineering and biomaterials, bioengineering, regenerative medicine, stem cells, gene therapy, systems biology, genetics, biochemistry, virology, microbiology, and neuroscience. All articles are published within 4 weeks of acceptance and are fully open access and posted on PubMedCentral. All journal content is available online at the BioResearch Open Access website (http://www.liebertpub.com/biores).

About the Publisher

Mary Ann Liebert, Inc., publishers (http://www.liebertpub.com) is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research, including Tissue Engineering, Stem Cells and Development, Human Gene Therapy and HGT Methods, and AIDS Research and Human Retroviruses. Its biotechnology trade magazine, Genetic Engineering & Biotechnology News (GEN), was the first in its field and is today the industry's most widely read publication worldwide. A complete list of the firm's 70 journals, books, and newsmagazines is available at the Mary Ann Liebert, Inc. website (http://www.liebertpub.com).

Mary Ann Liebert, Inc. 140 Huguenot St., New Rochelle, NY 10801-5215 http://www.liebertpub.com Phone: (914) 740-2100 (800) M-LIEBERT Fax: (914) 740-2101

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New marker for identifying precursors to insulin-producing cells in pancreas

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Saving a penny: Stem cell therapy shows promise in repairing stress urinary incontinence

August 21st, 2012 5:20 am

ScienceDaily (Aug. 17, 2012) Stress urinary incontinence (SUI) can occur due to sneezing, coughing, exercising or even laughing and happens because the pelvic floor muscles are too weak causing leakage when the bladder is put under pressure. New research published in BioMed Central's open access journal BMC Medicine shows that a new technique, using stem cells isolated from amniotic fluid, can regenerate damaged urethral sphincter muscles and prevent pressure incontinence in mice.

Although SUI is more common during and after pregnancy, and after the age of 40, one in three women will experience it at some point in their lives. Men can also be affected, especially after prostate surgery. SUI is treatable and in many cases losing weight, reducing caffeine intake, pelvic floor exercises, and bladder training can have very beneficial effects. If this does not work more invasive treatments are necessary, however there can be serious side effects associated with surgery.

Using stem cells to regenerate the damaged or weak muscles has been proposed as an alternative to surgery. But most protocols for harvesting stem cells also require invasive procedures, and often produce very low numbers of viable cells. In contrast amniotic stem cells can be collected easily, and have very low immunogenicity, reducing chances of rejection. Researchers from Kyungpook National University, Korea, investigated the ability of stem cells isolated from human amniotic fluid obtained during routine amniocentesis to regenerate damaged urethral sphincter muscles in mice.

James Yoo and Tae Gyun Kwon, who led this research, explained, "These stem cells are mesenchymal and consequently have the ability to become muscle cells when grown under the right conditions. We found that the stem cells were able to survive for seven days inside the mice but by 14 days they had all disappeared. Nevertheless they were able to induce regeneration of the mouse's own urethral sphincter muscle."

Quite how stem cells are able to retrain the body's own cells is still not fully understood. Not only was muscle regenerated, but this muscle had proper connections to nerves, and was able to improve the pressure required in the bladder before incontinence begins and stops. Humans are already being treated with stem cell therapy for diseases, including diabetes, and since Since amniotic stem cells appeared to cause no immune response or tumour formation, these cells may provide an avenue for future stem cell therapy for humans.

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The above story is reprinted from materials provided by BioMed Central Limited.

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Press Release

August 21st, 2012 5:20 am

Advanced Cell Technology Inc.Posted on:20 Aug 12

Advanced Cell Technology, Inc. (ACT; OTCBB: ACTC), a leader in the field of regenerative medicine, announced today that Scotlands NHS Lothian has been confirmed as a site for its Phase I/II human clinical trial for Stargardts Macular Dystrophy (SMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs).

NHS Lothian should be a superb partner for our EU clinical trial for SMD, said Gary Rabin, chairman and CEO of ACT. We are particularly pleased to be working with the Principal Investigator, Professor BaljeanDhillon, and his team. Additionally, we would like to thank the men and women of the Scottish Development Authority and Scottish National Blood Transfusion Service (SNBTS) for their tireless efforts to help make this history-making clinical trial a reality.

This approved, Phase I/II clinical trial for SMD is a prospective, open-label study designed to determine the safety and tolerability of RPE cells derived from hESCs following sub-retinal transplantation to patients with advanced SMD. It is similar in design to the companys US trials for SMD and dry age-related macular degeneration initiated in July 2011.

SMD represents an important unmet need in the wider clinical arena of macular degeneration, said Professor Dhillon, BMed Sci, BM BS, FRCS, Consultant Ophthalmic Surgeon, at the Princess Alexandra Eye Pavilion, NHS Lothian andHonorary Professor of Ophthalmology at the University of Edinburgh. This trial will evaluate a promising potential new treatment for this condition, using hESC-derived RPE cells.

Professor Marc Turner, Medical Director of SNBTS continued, hESC-derived RPE cells represent one of the first of a new generation of regenerative therapies and is an example of the high quality clinical research being conducted in, and supported by, NHS Scotland which we hope will help to transform medicine over the coming decades.

On July 30, the company announced that the third patient in this SMD clinical trial had been treated.

More information on the companys clinical trials will be posted today on Mr. RabinsChairmans blog.

About Stargardts Disease

Stargardts disease or Stargardts Macular Dystrophy is a genetic disease that causes progressive vision loss, usually starting in children between 10 to 20 years of age. Eventually, blindness results from photoreceptor loss associated with degeneration in the pigmented layer of the retina, called the retinal pigment epithelium, which is the site of damage that the company believes the hESC-derived RPE may be able to target for repair after administration.

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Press Release

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ACT Announces Scotland’s NHS Lothian as Additional Site for EU Clinical Trial Using hESC-Derived RPE Cells for Macular …

August 20th, 2012 8:14 pm

MARLBOROUGH, Mass.--(BUSINESS WIRE)--

Advanced Cell Technology, Inc. (ACT; OTCBB: ACTC), a leader in the field of regenerative medicine, announced today that Scotlands NHS Lothian has been confirmed as a site for its Phase I/II human clinical trial for Stargardts Macular Dystrophy (SMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs).

NHS Lothian should be a superb partner for our EU clinical trial for SMD, said Gary Rabin, chairman and CEO of ACT. We are particularly pleased to be working with the Principal Investigator, Professor BaljeanDhillon, and his team. Additionally, we would like to thank the men and women of the Scottish Development Authority and Scottish National Blood Transfusion Service (SNBTS) for their tireless efforts to help make this history-making clinical trial a reality.

This approved, Phase I/II clinical trial for SMD is a prospective, open-label study designed to determine the safety and tolerability of RPE cells derived from hESCs following sub-retinal transplantation to patients with advanced SMD. It is similar in design to the companys US trials for SMD and dry age-related macular degeneration initiated in July 2011.

SMD represents an important unmet need in the wider clinical arena of macular degeneration, said Professor Dhillon, BMed Sci, BM BS, FRCS, Consultant Ophthalmic Surgeon, at the Princess Alexandra Eye Pavilion, NHS Lothian and Honorary Professor of Ophthalmology at the University of Edinburgh. This trial will evaluate a promising potential new treatment for this condition, using hESC-derived RPE cells.

Professor Marc Turner, Medical Director of SNBTS continued, hESC-derived RPE cells represent one of the first of a new generation of regenerative therapies and is an example of the high quality clinical research being conducted in, and supported by, NHS Scotland which we hope will help to transform medicine over the coming decades.

On July 30, the company announced that the third patient in this SMD clinical trial had been treated.

More information on the companys clinical trials will be posted today on Mr. RabinsChairmans blog.

About Stargardts Disease

Stargardts disease or Stargardts Macular Dystrophy is a genetic disease that causes progressive vision loss, usually starting in children between 10 to 20 years of age. Eventually, blindness results from photoreceptor loss associated with degeneration in the pigmented layer of the retina, called the retinal pigment epithelium, which is the site of damage that the company believes the hESC-derived RPE may be able to target for repair after administration.

Excerpt from:
ACT Announces Scotland’s NHS Lothian as Additional Site for EU Clinical Trial Using hESC-Derived RPE Cells for Macular ...

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Building Body Parts: Ears, Muscles and More!

August 20th, 2012 8:14 pm

BACKGROUND: According to the U.S. Department of Health and Human Services, regenerative medicine is the next evolution of medical treatments. Regenerative medicine offers the potential for the body to heal itself. Scientists at the Wake Forest Institute for Regenerative Medicine in Winston-Salem, N.C., were the first in the world to engineer lab-grown organs that were successfully implanted into humans. Now, the team of researchers is working to engineer more than 30 different replacement tissues and organs to develop cell therapies with the goal of curing a variety of diseases. (SOURCE: Wake Forest Institute for Regenerative Medicine)

LAB-GROWN URETHRAS: Researchers from Wake Forest were the first in the world to use patients own cells to build tailor-made urine tubes in the lab and successfully replace damaged tissue in five boys in Mexico. The boys were unable to urinate due to a pelvic injury. After receiving the lab-grown urethras, all the boys continue to do well with normal or near-normal urinary flow. The urethras were grown on biodegradable mesh scaffolds made of a polyester compound. The scaffolds were seeded with cells taken from the patients own bladders and incubated in the lab for four to seven weeks. They were then used to repair damaged segments of the boys urethras. For us, really, our goal here at the Institute is really to try to complete technologies that we can get to patients to make their lives better, so anytime that were able to do that, improve the quality of patients lives, we feel like thats part of our mission, Anthony Atala, M.D., Director, Wake Forest Institute for Regenerative Medicine, told Ivanhoe.

(SOURCE: Ivanhoe interview with Dr. Atala and WebMD article)

GROWING EARS: Scientists are working on printing ears in the lab. What we can do is we can take any three dimensional image of an ear, and it can be put into the computer, and that will generate an image within the printer that then prints that specific three dimensional structure, John Jackson, Ph.D., Associate Professor, Wake Forest Institute for Regenerative Medicine, told Ivanhoe. Right now, implants that are commercially-available are hard and rigid. They also cause problems with erosion through the skin. The new, tailor-made ears are flexible and patient-specific. In animal studies, the lab-grown ears have been shown to cause less erosion. The next step is to print the ears for use in humans. To be able to take a structure, generate a 3D implant and have that as a potential treatment for a patient who has lost an ear, thats very exciting, Dr. Jackson told Ivanhoe.

(SOURCE: Ivanhoe interview with Dr. Jackson)

ENGINEERING MUSCLE: Researchers are also looking to see if they can engineer tissue that resembles muscle to repair small injuries in the body. They take biopsies from skeletal muscles and culture out the stem cells from the muscle. They then seed the cells onto a scaffold and condition the scaffold and a bioreactor to exercise muscle in-vitro. Then, they use that construct as an implant to accelerate regeneration and repair of injured muscle in the body. Scientists have been studying the engineered muscle in animals, and the next step is to try it in humans. For me, personally, its fantastic because you dont often get an opportunity to do research thats not only compelling but that can result in therapies that can help people on a daily basis and really improve their quality of life, George Christ, Ph.D., Professor of Regenerative Medicine, Wake Forest Institute for Regenerative Medicine, told Ivanhoe.

(SOURCE: Ivanhoe interview with Dr. Christ)

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Building Body Parts: Ears, Muscles and More!

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CSU vets seeking cats with chronic kidney disease

August 20th, 2012 8:12 pm

Colorado State University veterinarians are looking for cats with chronic kidney disease to participate in a clinical trial involving stem cell therapy.

Felines with chronic kidney disease may benefit from the clinical trial. Kidney disease, or renal failure, is a highly common ailment particularly in older cats.

Currently, other than kidney transplantation, only supportive care home treatments are available to try and slow the progression of the disease. Recent studies have shown that stem cell therapy has the potential to improve kidney function in rodents with kidney failure. In laboratories, stem cells improve renal function, decrease inflammation and scarring in the kidney and improve levels of excess protein in the urine.

What the study involves: Stem cells that have been grown from the fat of young healthy specific-pathogen free cats (the cats are not harmed during the collection process) will be slowly injected intravenously every two weeks for three treatments. A small group of cats will receive a placebo treatment during the trial, but have the option to receive stem cell treatment after finishing the trial. The study involves a minimum of five visits to the Veterinary Teaching Hospital, so cats that are stressed or become agitated during veterinary visits, or are not local to the CSU area, are not ideal candidates.

Cats with stable chronic kidney disease can participate in the stem cell study. Those with other illnesses or heart disease, kidney infection, stones or other renal complications cannot be enrolled in the study.

All visits, lab work, stem cell treatments and a $200 stipend for the owner's expenses are funded by CSU's Frankie's Fund for Feline Stem Cell Research and the Morris Animal Foundation.

For more information about enrolling a cat in this study, contact Dr. Jessica Quimby at jquimby@colostate.edu.

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CSU vets seeking cats with chronic kidney disease

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CSCR Reading List: A Look at the Grant Appeal Process at the California Stem Cell Agency

August 19th, 2012 3:54 pm


Here is a list of articles from the California Stem Cell Report as well as CIRM documents dealing with the grant appeal process at the California stem cell agency. The list was prepared on Aug. 16, 2012. To read the entire articles, click on the links.

Articles from the California Stem Cell Report

Aug. 7, 2012
Researcher
Alert: Opportunity for Fresh Appeal in $243 Million Disease Team
Round
A tiny opening exists for scientists
who failed to win approval last month of their bids for $20 million
research awards from the California stem cell agency.
July 26, 2012
Stem
Cell Directors Approve $151 Million to Commercialize Stem Cell
Research
Directors of the California stem cell
agency today approved $151 million in research awards aimed at
commercializing stem cell research and pushing therapies into
clinical treatment....Five of the applications involving appeals were
sent back by the board for more review. (See herehere and here.)
They will be considered again in early September or October.
July 24, 2012
Record
Appeals by Researchers in Huge California Stem Cell Agency Round
The California stem cell agency's
latest grant round – which is budgeted for $243 million – has
drawn an extraordinary and record outpouring of appeals from more
than half of the scientists rejected by the grant reviewers. Nine of
the 15 applicants who were turned down have filed appeals to the
governing board for its meeting
Thursday
 in Burlingame. No other CIRM grant round
has drawn as high a percentage of appeals, formally known as
extraordinary petitions. (See here
for a story on the previous record 
for percentage of
appeals.)
Aug. 10, 2010

Emotionalism and Potential Favoritism Cited as Need for Changes in CIRM Grant Appeals
Passion and favoritism, democracy and gamesmanship – all are part of the ongoing discussion among directors of the $3 billion California stem cell agency as they try to fix what some of them call a “broken” grant appeal process.

July 19, 2010
UC Davis Scientist Praises CIRM Appeals Change
A stem cell researcher at UC Davis today said a change in the CIRM grant appeals procedure makes “a lot of sense.” Writing on his blog in regard to "extraordinary petitions," Paul Knoepfler said, “I think the proposed change makes a lot of sense and would greatly improve the process. Sometimes the reasons in the petitions are clearly not meritorious and as it now stands, they end up wasting CIRM's time. The last time CIRM received 9 petitions as well, which represented a remarkably large fraction of the total applications. A stricter process would discourage the submission of large numbers of petitions, an important issue given that the number of petitions received by CIRM continues to grow.”

CIRM Finally Discloses Grant Appeal Proposals
The California stem cell agency early today belatedly posted a two-page memo on proposed changes in how it will deal with appeals by scientists whose grant applications have been rejected by reviewers.

July 18, 2010
Sticky, Troubling Appeals by Rejected Researchers Targeted by Stem Cell Agency
A key step in the process for awarding billions of dollars in research grants is “broken,” according to many directors of the California stem cell agency, and major changes are looming that will affect hundreds of scientists.

June 22, 2010
Immunology Grants: CIRM Gives $25 Million to 19 Researchers
Directors of the California stem cell agency today approved $25 million for immunology research, overturning four negative decisions by its grant reviewers. Directors faced a record nine public petitions to reverse its reviewers. After some grumbling, the directors, who see only a summary of the application and reviewer comments, okayed the four.

June 19, 2010
More Grant Appeals Filed: Yamanaka Invoked
The California stem cell agency has set another benchmark, although this is one that it may not want to trot out at international stem cell gatherings. Eight scientists whose applications were rejected for funding by the CIRM grants working group and scientific reviewers are seeking to overturn those decisions at the agency's board meeting in San Diego on Tuesday. It is the largest number of “extraordinary petitions” ever filed and amounts to more than one out of every four applications that were turned down. The total number of applications received was 44. Fifteen were approved. Some of the researchers are likely to appear at the board meeting and make a personal pitch.

May 18, 2010
Competing for California Stem Cell Cash: Rules of the Game Coming Under Scrutiny
Every California stem cell scientist and researcher looking to join the field – be they from academia or business – should pay very close attention to a meeting next week of a key group of directors of the $3 billion California stem cell agency. They plan to discuss possible changes in how scientists compete for stem cell cash, which is no small matter since CIRM has another $2 billion to hand out over the next several years.

CIRM documents

Pre-application review – CIRM report (Jan. 2010) on the process

Extraordinary petition policy – Version as of 5/25/10

Appeal policy – Version as of 5/25/2010

Transcript of July 20, 2010, meeting of CIRM directors Science Subcommittee. Discussion of petitions begins on page 40.

Transcript of the June 22, 2010, CIRM directors meeting. Discussions of extraordinary petitions begin on pages 24 and 67.

Transcript of 5/25/10 Science Subcommittee meeting dealing with appeals issue. Discussion begins on page 99.

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

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$70 Million Research Proposal Up Next Week at California Stem Cell Agency

August 19th, 2012 3:54 pm


Directors of the $3 billion California
stem cell agency will hold a special, teleconference meeting next
Tuesday to deal with business that was put off last month, including
a new, $70 million research round.
The meeting is necessary because directors could not finish their business July 26 after they lost the supermajority quorum required to do business. They delayed action on a number of
matters, including the translational research proposal, which is
scheduled to be posted as an RFA next month.
The governing board also had discussed
dealing with changes in its intellectual property rules at next week's meeting, but that proposal is not on Tuesday's agenda. The next meeting of the board is Sept. 5 and 6 in San Francisco. The
agency has confirmed that it will be a two-day session.
At least one new appeal is expected to
come up in September in the $243 million disease team round that
consumed so much time in July.
Next week meeting involves a host of
locations throughout California. The public is entitled to
participate in the session from any of those sites. The specific
addresses can be found on the agenda.

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

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CSCR Reading List: A Look at the Grant Appeal Process at the California Stem Cell Agency

August 19th, 2012 3:52 pm


Here is a list of articles from the California Stem Cell Report as well as CIRM documents dealing with the grant appeal process at the California stem cell agency. The list was prepared on Aug. 16, 2012. To read the entire articles, click on the links.

Articles from the California Stem Cell Report

Aug. 7, 2012
Researcher
Alert: Opportunity for Fresh Appeal in $243 Million Disease Team
Round
A tiny opening exists for scientists
who failed to win approval last month of their bids for $20 million
research awards from the California stem cell agency.
July 26, 2012
Stem
Cell Directors Approve $151 Million to Commercialize Stem Cell
Research
Directors of the California stem cell
agency today approved $151 million in research awards aimed at
commercializing stem cell research and pushing therapies into
clinical treatment....Five of the applications involving appeals were
sent back by the board for more review. (See herehere and here.)
They will be considered again in early September or October.
July 24, 2012
Record
Appeals by Researchers in Huge California Stem Cell Agency Round
The California stem cell agency's
latest grant round – which is budgeted for $243 million – has
drawn an extraordinary and record outpouring of appeals from more
than half of the scientists rejected by the grant reviewers. Nine of
the 15 applicants who were turned down have filed appeals to the
governing board for its meeting
Thursday
 in Burlingame. No other CIRM grant round
has drawn as high a percentage of appeals, formally known as
extraordinary petitions. (See here
for a story on the previous record 
for percentage of
appeals.)
Aug. 10, 2010

Emotionalism and Potential Favoritism Cited as Need for Changes in CIRM Grant Appeals
Passion and favoritism, democracy and gamesmanship – all are part of the ongoing discussion among directors of the $3 billion California stem cell agency as they try to fix what some of them call a “broken” grant appeal process.

July 19, 2010
UC Davis Scientist Praises CIRM Appeals Change
A stem cell researcher at UC Davis today said a change in the CIRM grant appeals procedure makes “a lot of sense.” Writing on his blog in regard to "extraordinary petitions," Paul Knoepfler said, “I think the proposed change makes a lot of sense and would greatly improve the process. Sometimes the reasons in the petitions are clearly not meritorious and as it now stands, they end up wasting CIRM's time. The last time CIRM received 9 petitions as well, which represented a remarkably large fraction of the total applications. A stricter process would discourage the submission of large numbers of petitions, an important issue given that the number of petitions received by CIRM continues to grow.”

CIRM Finally Discloses Grant Appeal Proposals
The California stem cell agency early today belatedly posted a two-page memo on proposed changes in how it will deal with appeals by scientists whose grant applications have been rejected by reviewers.

July 18, 2010
Sticky, Troubling Appeals by Rejected Researchers Targeted by Stem Cell Agency
A key step in the process for awarding billions of dollars in research grants is “broken,” according to many directors of the California stem cell agency, and major changes are looming that will affect hundreds of scientists.

June 22, 2010
Immunology Grants: CIRM Gives $25 Million to 19 Researchers
Directors of the California stem cell agency today approved $25 million for immunology research, overturning four negative decisions by its grant reviewers. Directors faced a record nine public petitions to reverse its reviewers. After some grumbling, the directors, who see only a summary of the application and reviewer comments, okayed the four.

June 19, 2010
More Grant Appeals Filed: Yamanaka Invoked
The California stem cell agency has set another benchmark, although this is one that it may not want to trot out at international stem cell gatherings. Eight scientists whose applications were rejected for funding by the CIRM grants working group and scientific reviewers are seeking to overturn those decisions at the agency's board meeting in San Diego on Tuesday. It is the largest number of “extraordinary petitions” ever filed and amounts to more than one out of every four applications that were turned down. The total number of applications received was 44. Fifteen were approved. Some of the researchers are likely to appear at the board meeting and make a personal pitch.

May 18, 2010
Competing for California Stem Cell Cash: Rules of the Game Coming Under Scrutiny
Every California stem cell scientist and researcher looking to join the field – be they from academia or business – should pay very close attention to a meeting next week of a key group of directors of the $3 billion California stem cell agency. They plan to discuss possible changes in how scientists compete for stem cell cash, which is no small matter since CIRM has another $2 billion to hand out over the next several years.

CIRM documents

Pre-application review – CIRM report (Jan. 2010) on the process

Extraordinary petition policy – Version as of 5/25/10

Appeal policy – Version as of 5/25/2010

Transcript of July 20, 2010, meeting of CIRM directors Science Subcommittee. Discussion of petitions begins on page 40.

Transcript of the June 22, 2010, CIRM directors meeting. Discussions of extraordinary petitions begin on pages 24 and 67.

Transcript of 5/25/10 Science Subcommittee meeting dealing with appeals issue. Discussion begins on page 99.

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$70 Million Research Proposal Up Next Week at California Stem Cell Agency

August 19th, 2012 3:52 pm


Directors of the $3 billion California
stem cell agency will hold a special, teleconference meeting next
Tuesday to deal with business that was put off last month, including
a new, $70 million research round.
The meeting is necessary because directors could not finish their business July 26 after they lost the supermajority quorum required to do business. They delayed action on a number of
matters, including the translational research proposal, which is
scheduled to be posted as an RFA next month.
The governing board also had discussed
dealing with changes in its intellectual property rules at next week's meeting, but that proposal is not on Tuesday's agenda. The next meeting of the board is Sept. 5 and 6 in San Francisco. The
agency has confirmed that it will be a two-day session.
At least one new appeal is expected to
come up in September in the $243 million disease team round that
consumed so much time in July.
Next week meeting involves a host of
locations throughout California. The public is entitled to
participate in the session from any of those sites. The specific
addresses can be found on the agenda.

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Protecting prion protein keeps stem cells young

August 18th, 2012 3:11 am

COULD we stem the tide of ageing by delaying the deterioration of stem cells? A new compound that appears to do just that could help us find ways to protect our organs from age-related wear and tear, experiments in mice suggest.

As we age, so do our mesenchymal stem cells (MSCs): their numbers in our bone marrow decline, and those that are left lose the ability to differentiate into the distinct cell types - such as bone, cartilage, fat and possibly muscle cells - that help in the healing process.

"We think this ageing of stem cells may be linked to the onset of some age-related disorders, such as osteoporosis," says Ilaria Bellantuono at the University of Sheffield in the UK.

Earlier research in mice had suggested that the prion protein expressed by MSCs might play a role in holding back stem cell ageing. Mice lacking the prion protein were less able to regenerate blood cells. The study provided more evidence that correctly folded prions serve a useful purpose in the body, despite the role that misfolded prions play in BSE and vCJD.

Bellantuono and her colleagues have now found that the prion protein performs a similar function in humans - older MSCs from human bone marrow expressed less of the protein than younger ones.

In a bid to find a compound that might slow MSC ageing, the team tested numerous molecules known to target prion proteins on dishes of human stem cells. One molecule emerged as a potential candidate - stem cells treated with it produced 300 times the number of cells over 250 days than untreated stem cells. The treated cells kept on dividing for longer.

The team then injected treated cells into the thigh bones of mice, and three days later found that they had produced three times as many new cells as they would normally produce. After five weeks, there were 10 times as many cells.

The new cells appeared to be of higher quality, too, and readily differentiated into bone and fat cells, as well as those that support the tissue and blood vessels.

Bellantuono's team think the molecule works by helping the prions protect the stem cells from the DNA damage associated with normal ageing. When they exposed both treated and untreated cells to hydrogen peroxide - a compound known to cause DNA damage - they found that the treated cells were protected from damage (Stem Cells, DOI: 10.1002/stem.1065).

"You can delay the loss of stem cells' function by manipulating the prion protein," says Bellantuono, who presented the findings at the Aging Online Symposium last month. "In the long term, you may go a long way to maintaining tissue health in [old] age."

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Stem Cells as Blood Vessels=Heart Tx?

August 18th, 2012 3:11 am

Researchers at Kings College London have developed the first artificial functioning blood vessel outside of the body, according to a release from the college. The vessels are made from reprogrammed stem cells from human skin. The team also saw the cells develop into a blood vessel inside the body for the first time.

The hope is that this new technique will eventually lead to a treatment for patients with heart disease. The plan is that the reprogrammed cells would be injected into a leg or even directly into the heart to restore blood flow. Another possibility would be to graft one of the artificially developed vessels into the body as a replacement for blocked or damaged vessels. The research team also believes the newly created vessels could be used to prevent leg amputation in diabetic patients with poor circulation.

The study, which was published in the journal Proceedings of the National Academy of Sciences, reports that the reprogrammed vascular cells have no risk turning into tumors.

The release quotes Professor Qingbo Xu of the British Heart Foundation as saying, "This is very exciting research . . . If we can develop this approach as personalized treatments for patients with the condition, it will be a significant step forward."

The researchers cautioned that this is an early study and that more research needs to be done regarding how this approach will works in patients, but Dr Hlne Wilson, Research Advisor at the British Heart Foundation, said: "The discovery could help lead towards future therapies to repair hearts after they are damaged by a heart attack. As well as playing a part in a possible future regenerative treatment, these cells might also be used in drug screening to find new treatments to tackle inherited diseases."

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New research uses stem cells as possible treatment for arthritis

August 18th, 2012 3:11 am

CTVNews.ca Staff Published Friday, Aug. 17, 2012 8:36AM EDT

A Toronto research team hopes to make hip and knee replacements a thing of the past as it explores the growth of new human cartilage using stem cells.

With an estimated four million Canadians suffering from arthritis, and that number expected to grow to seven million by 2031, doctors are hoping to use the stem cells to treat the deterioration of cartilage in joints. Although hip and knee replacements are a great operation, they improve patients lives in terms of pain, quality and function, theyre not your own joint, Dr. Nizar Mahomed told CTVs Canada AM on Friday. They dont last forever and they bring risks and limitations.

Mahomed, an orthopedic surgeon at Torontos Western Hospital, said 45,000 hip and knee replacement surgeries are performed in Canada each year. Many of the surgeries are to treat the damage left by arthritis, which he said is caused by aging, obesity and injuries.

The incident of arthritis increases with age, so as our population ages the prevalence of arthritis is going to continue to increase.

Mahomed and his colleagues are one of the first research teams in the world that have been able to grow human cartilage.

The team is now embarking on the next stage of the study, which will see the new tissue used in animals.

If we actually make it work in animals then one day well be able to bring it back into patients, said Mahomed.

He added that stem cells hold much hope for medicine in the future as studies are looking at using the cells to regenerate cardiac tissue and in the treatment of nerve and spinal cord injuries.

Mahomed said he hopes within five to 10 years the new technology can be used in human patients while putting an end to joint replacement surgeries.

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Embryonic Stem Cells Survive Freezing

August 17th, 2012 9:16 am

Even after 18 years of frozen storage, human embryos can still produce viable stem cells for drug screening and biomedical research.

By Hayley Dunning | August 16, 2012

Cryopreservation of embryos in fertility centers is common, and concerns over damage to the embryo during thawing were largely allayed by the birth of a healthy boy in 2010 from 20-year old cryopreserved embryo. Last week (August 10), researchers in Thailand reported in BioResearch Open Access that they successfully induced the growth of stem cells from a set of 17- and 18-year-old frozen embryos.

The embryos were thawed, then cultured to the blastocyst stage and co-cultured with human foreskin fibroblasts which acted as feeder cells to maintain the growth of embryonic stem cells. The team used the same method to induce stem cells to grow from fresh embryos for comparison, and in cells from both sources they found similar levels of pluripotency.

The importance of this study is that it identifies an alternative source for generating new embryonic stem lines, using embryos that have been in long-term storage, BioResearch Open Access Editor-in-Chief Jane Taylor told Asian News International.

By Cristina Luiggi

A postdoctoral research fellow at Emory University falsifies stem cell research data.

By Megan Scudellari

Human embryonic stem cells swiftly kill themselves in response to DNA damage.

By Edyta Zielinska

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Research and Markets: Personalized Medicine – A Global Market Overview

August 17th, 2012 9:16 am

DUBLIN--(BUSINESS WIRE)--

Research and Markets (http://www.researchandmarkets.com/research/vxwrkb/personalized_medic) has announced the addition of the "Personalized Medicine - A Global Market Overview" report to their offering.

This report review, analyze and projects the personalized medicine market for global and the regional markets including the United States, Europe and Rest of World. The market numbers illustrated in this report only represent the market exclusively for the product segments and technologies enunciated above. The market, in this report, does not include the associated hardware equipment or software technologies that are used to manage patient data. The study includes recent and current trends related to technology and the market along with the key industry developments.

The market for personalized medicine product types analyzed in this study includes Targeted Biologics, Proteomics & Genomics, Genetically Modified (GM) Products, Wellness & Disease Management, Other Molecular Diagnostics and Self/Other Diagnostics. The report also includes the market analysis for application technologies of personalized medicine - Pharmacogenomics, Point-of-Care Testing, Stem Cell Therapy, Pharmacoproteomics, Pharmacogenetics and Other Technologies. The report analyses the global market in terms of USD Million.

This 350 page global market report includes 43 charts (includes a data table and graphical representation for each chart), supported with meaningful and easy to understand graphical presentation, of the market. The statistical tables represent the data for the global market by geographic region, product type and application technology.

The report covers the brief business profiles of 56 key global players and 77 major players across the United States - 45; Europe - 24; and Rest of World - 8.

The report also provides the listing of the companies engaged in research and development, manufacturing, processing, supplies and distribution of personalized. Also enlisting the academic institutions engages in personalized medicine, the global list covers the addresses, contact numbers and the website addresses of 395 companies.

For more information, including table of contents and list of companies mentioned, please visit http://www.researchandmarkets.com/research/vxwrkb/personalized_medic

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Research and Markets: Personalized Medicine - A Global Market Overview

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Personalized Medicine – A Global Market Overview

August 17th, 2012 9:16 am

NEW YORK, Aug. 16, 2012 /PRNewswire/ -- Reportlinker.com announces that a new market research report is available in its catalogue:

http://www.reportlinker.com/p0955290/Personalized-Medicine---A-Global-Market-Overview.html#utm_source=prnewswire&utm_medium=pr&utm_campaign=In_Vitro_Diagnostic

`Personalized Medicine can mean a lot of things to a lot of people. For some, it may relate to doctors having knowledge about their case history and the treatment received, which is a morale boosting factor, since everybody wants caregivers who can comprehend an individual's problems. The day is not very far when this level of personal or individual understanding between a patient and a doctor would be much deeper than hitherto anticipated. The coming decade is expected to witness an increase in the use of companion diagnostics and personalized medicines, with pricing incentives and efficiency improvement propelling the market. Current market leaders with diagnostic divisions focusing on biomarker identification would be at an advantage.

This report review, analyze and projects the personalized medicine market for global and the regional markets including the United States, Europe and Rest of World. The market numbers illustrated in this report only represent the market exclusively for the product segments and technologies enunciated above. The market, in this report, does not include the associated hardware equipment or software technologies that are used to manage patient data. The study includes recent and current trends related to technology and the market along with the key industry developments.

The market for personalized medicine product types analyzed in this study includes Targeted Biologics, Proteomics & Genomics, Genetically Modified (GM) Products, Wellness & Disease Management, Other Molecular Diagnostics and Self/Other Diagnostics. The report also includes the market analysis for application technologies of personalized medicine Pharmacogenomics, Point-of-Care Testing, Stem Cell Therapy, Pharmacoproteomics, Pharmacogenetics and Other Technologies. The report analyses the global market in terms of USD Million.

This 350 page global market report includes 43 charts (includes a data table and graphical representation for each chart), supported with meaningful and easy to understand graphical presentation, of the market. The statistical tables represent the data for the global market by geographic region, product type and application technology. The report covers the brief business profiles of 56 key global players and 77 major players across the United States 45; Europe 24; and Rest of World 8. The report also provides the listing of the companies engaged in research and development, manufacturing, processing, supplies and distribution of personalized. Also enlisting the academic institutions engages in personalized medicine, the global list covers the addresses, contact numbers and the website addresses of 395 companies.

PART A: GLOBAL MARKET PERSPECTIVE

1. INTRODUCTION1.1 Product Outline1.1.1 Personalized Medicine's Influence on Large Scale Studies1.1.2 Gazing into the Crystal Ball: What the Future Holds for Personalized Medicine1.1.3 Ramifications of Personalized Medicine for Healthcare Systems1.1.3.1 Pharmaceutical Industry1.1.3.2 Diagnostics Industry1.1.3.3 Insurers1.1.3.4 Physicians1.1.3.5 Government Agencies1.1.3.6 Patients1.1.4 Analysis of Personalized Medicine by Segment1.1.4.1 Targeted Biologics1.1.4.1.1 Overview1.1.4.1.2 Targeted Biologics for Breast Cancer: An Illustration1.1.4.2 Proteomics & Genomics1.1.4.2.1 Proteomics1.1.4.2.1.1 A Complex Problem1.1.4.2.1.2 Post-Translational Modifications1.1.4.2.1.3 Phosphorylation1.1.4.2.1.4 Ubiquitination1.1.4.2.1.5 Other Modifications1.1.4.2.2 Genomics1.1.4.2.2.1 Pharmacogenomics1.1.4.3 Genetically Modified (GM) Products1.1.4.3.1 The Genetic Engineering Process1.1.4.3.1.1 Applications of Genetic Engineering1.1.4.4 Wellness & Disease Management1.1.4.4.1 Wellness Defined1.1.4.4.2 Disease Management Defined1.1.4.5 Molecular Diagnostic Technologies1.1.4.5.1 DNA Sequencing1.1.4.5.2 Biochips and Microarrays1.1.4.5.3 Cytogenetics1.1.4.5.3.1 Personalized Medicine Based on Molecular Cytogenetics1.1.4.5.3.2 Personalized Medicine Based on Cytomics1.1.4.5.4 Single Nucleotide Polymorphism (SNP) Genotyping1.1.4.5.4.1 Applications of SNPs Pertinent to Personalized Medicine1.1.4.5.5 Haplotyping1.1.4.5.6 Application of Proteomics In Molecular Diagnosis1.1.4.5.7 Gene Expression Profiling1.1.4.5.8 Personalized Medicine and Molecular Imaging1.1.4.5.9 Diagnostics Based On Glycomics1.1.4.5.10 Combining Diagnostics and Therapeutics1.1.4.5.11 Point-Of-Care (POC) Diagnosis1.1.4.5.12 Genetic Testing For Disease Predisposition1.1.5 Analysis of Personalized Medicine by Technology1.1.5.1 Pharmacogenomics1.1.5.1.1 Drug Metabolism1.1.5.1.2 Applications1.1.5.2 Point-of-Care Testing1.1.5.2.1 Tests that are Most Apt for Specific Scenarios1.1.5.2.2 Advantages1.1.5.3 Stem Cell Therapy1.1.5.3.1 Treatment with Stem Cells1.1.5.3.2 Current Therapies1.1.5.3.3 Future Treatments1.1.5.4 Pharmacoproteomics1.1.5.5 Pharmacogenetics1.1.5.5.1 Prediction of Drug-Drug Interactions1.1.5.5.2 Integration of Pharmacogenetics with the Healthcare System1.1.5.5.3 Pharmacogenetic Tests1.1.5.6 Other Personalized Medicine Technologies1.1.5.6.1 Biochips1.1.5.6.2 Genetic Screening1.1.5.6.3 Metabolomics1.1.5.6.4 Molecular Diagnostics1.1.5.6.5 Pharmacodynamics1.1.5.6.6 Pharmacokinetics1.1.5.6.7 SNP Genotyping1.1.6 The Rationale Behind Personalized Medicine: "One Size no Longer Fits All"1.1.7 The Human Genome: What is It?

2. KEY MARKET TRENDS

Combating Melanoma and Lung Cancer Facilitated Using Novel Personalized Drugs

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What Your Neural Stem Cells Aren't Telling You

August 15th, 2012 9:24 pm

Neural stem cells (green) in the hippocampus huddle around a neuron (purple), listening for stray signals.

In 2000, a team of neuroscientists put an unusual idea to the test. Stress and depression, they knew, made neurons wither and die particularly in the hippocampus, a brain area crucial for memory. So the researchers put some stressed-out rats on an antidepressant regimen, hoping the mood boost might protect some of those hippocampal neurons. When they checked in a few weeks later, though, the team found that rats hippocampuses hadnt just survived intact; theyd grown whole new neurons bundles of them. But thats only the beginning of our tale.

By the time 2009 rolled around, another team of researchers was suggesting that human brains might get a similar hippocampal boost from antidepressants. The press announced the discovery with headlines like, Antidepressants Grow New Brain Cells although not everyone agreed with that conclusion. Still, whether the principle applied to humans or not, a far more basic question was begging to be answered: How, exactly, does a brain tell new cells to form?

Well, through synapses, of course, you might answer and thatd be a very reasonable guess. After all, synapses are how most neurons talk to each other: electrochemical information is squirted from a tiny tendril of one neuron into the tip of a tendril on another; and cells throughout most of the brain share essentially this same mechanism for passing signals along: The signals coming out of Neuron As synapses keep bugging Neuron B by stimulating its synapses, until finally Neuron B caves under peer pressure and bugs Neuron C with the signal and so on.

There are, however, two significant exceptions to this system.

The first exception was discovered a few years ago, as scientists got more and more curious about the role of neuroglia (also known as just glia), synapse-less cells that many had assumed were just there to serve as structural support for neurons. A 2008 study showed that glia help control cerebral blood flow, and research in 2010 demonstrated that some glia cells known as astrocytes actively listen for and respond to certain neurotransmitter messages. These so-called quiet cells are actually pretty loud talkers once you learn to tune in to their chatter.

The second exception to the synapse rule is even more mysterious in large part because its a brand-new discovery: As the journal Nature reports, a team led by Hongjun Song at the Johns Hopkins University School of Medicine have found that neural stem cells listen in on the stray chemical signals that leak from synapses.

You can imagine neural stem cells as being sort of neural embryos depending on the surrounding conditions, they can develop into neurons or into glia. And heres whats strange about the way these cells communicate: They respond not to any single synaptic signal, but to the overall chemical vibe of their environment to chronic feelings of stress, for instance. By way of response, they may morph into neurons or glia or even tell the brain to crank out some all-new cells.

Neural stem cells seem to be particularly interested in the chemical GABA (gamma-aminobutyric acid) a neurotransmitter thats known to be involved in inhibiting signals from other neurons. When scientists artificially block these stem cells GABA receptors from receiving messages, the cells wake up and start replicating but when those GABA signals are allowed to reach the receptors, the stem cells stay dormant.

In this case, Song explains, GABA communication keeps the brain stem cells in reserve, so if we dont need them, we dont use them up.

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What Your Neural Stem Cells Aren't Telling You

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Fresh cell therapy promises better health, sex and more

August 15th, 2012 12:10 am

MANILA, Philippines Celebrity hairstylist Ricky Reyes, talent manager and host Lolit Solis, actress Lorna Tolentino and even former President Joseph Estrada are only among the prominent Filipinos who swear by the healing effects of fresh cell therapy, which involves the injection of live animal cells into the body.

Reyes, who used to suffer from a rare disease which he called reading eye epilepsy, said he went to Germany last June for fresh cell therapy.

After a number of sessions, the celebrity hairstylist can now read newspapers without suffering a seizure.

It was gone immediately, he said. Pati arthritis ko. Naalis yung sakit, tapos gaganda at babata ka pa.

Solis, 65, had fresh cell therapy after experiencing knee pain, and 75-year-old Estrada opted to undergo the procedure in Germany to keep healthy.

Before them, several other well-known figures worldwide are said to have tried fresh cell treatments, among them the late English actor Charlie Chaplin.

So how is this procedure done? Dr. Robert Janson-Muller, who runs a fresh cell therapy clinic in Germany, is in town to give Filipinos the lowdown on this decades-long treatment.

Not stem cell treatment

Before starting his lecture for members of the local media on Tuesday, Janson-Muller made it clear that fresh cell therapy is different from the now controversial stem cell treatment, which aims to replace damaged organs in the body or create one from scratch.

He stressed that his methods, which do not promise miracles, have been proven effective by his predecessors for the past 60 years.

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