The California stem cell agency faces
no easy task in trying to translate basic research findings into
something that can be used to treat patients and be sold commercially.

Elizabeth Iorns Science Exchange Photo

“One goal of scientific publication
is to share results in enough detail to allow other research teams to
reproduce them and build on them. However, many recent reports have
raised the alarm that a shocking amount of the published literature
in fields ranging from cancer biology to psychology is not
reproducible.”

“First, I think it is important to
accept that there is a crisis affecting preclinical research. Recent
studies estimate that 70% of preclinical research cannot be
reproduced. This is the research that should form the foundation upon
which new discoveries can be made to enhance health, lengthen life,
and reduce the burdens of illness and disability. The
irreproducibility of preclinical research is a significant impediment
to the achievement of these goals. To solve this problem requires
immediate and concrete action. It is not enough to make
recommendations and issue guidelines to researchers. Funders must act
to ensure they fund researchers to produce high quality reproducible
research. One such way to do so, is to reward, or require,
independent validation of results. The reproducibility initiative
provides a mechanism for independent validation, allowing the
identification of high quality reproducible research. It is vital
that funders act now to address this problem, to prevent the wasted
time and money that is currently spent funding non-reproducible
research and to prevent the erosion of public trust and support for
research.”

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

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View original post here:
Probe sought into status of stem cell therapy in the Philippines

According to Aristotle, only the Pentacosiomedimnoi were eligible for election to high office as archons and therefore only they gained admission into the Areopagus. A modern view affords the same privilege to the hippeis. The top three classes were eligible for a variety of lesser posts and only the Thetes were excluded from all public office.

Depending on how we interpret the historical facts known to us, Solon's constitutional reforms were either a radical anticipation of democratic government, or they merely provided a plutocratic flavour to a stubbornly aristocratic regime, or else the truth lies somewhere between these two extremes.

Solon's reforms can thus be seen to have taken place at a crucial period of economic transition, when a subsistence rural economy increasingly required the support of a nascent commercial sector. The specific economic reforms credited to Solon are these: Fathers were encouraged to find trades for their sons; if they did not, there would be no legal requirement for sons to maintain their fathers in old age. Foreign tradesmen were encouraged to settle in Athens; those who did would be granted citizenship, provided they brought their families with them. Cultivation of olives was encouraged; the export of all other produce was prohibited. Competitiveness of Athenian commerce was promoted through revision of weights and measures, possibly based on successful standards already in use elsewhere, such as Aegina or Euboia or, according to the ancient account but unsupported by modern scholarship, Argos

It is generally assumed, on the authority of ancient commentators that Solon also reformed the Athenian coinage. However, recent numismatic studies now lead to the conclusion that Athens probably had no coinage until around 560 BC, well after Solon's reforms.

Solon's economic reforms succeeded in stimulating foreign trade. Athenian black-figure pottery was exported in increasing quantities and good quality throughout the Aegean between 600 BC and 560 BC, a success story that coincided with a decline in trade in Corinthian pottery. The ban on the export of grain might be understood as a relief measure for the benefit of the poor. However, the encouragement of olive production for export could actually have led to increased hardship for many Athenians since it would have led to a reduction in the amount of land dedicated to grain. Moreover an olive produces no fruit for the first six years. The real motives behind Solon's economic reforms are therefore as questionable as his real motives for constitutional reform. Were the poor being forced to serve the needs of a changing economy, or was the economy being reformed to serve the needs of the poor?

Solon's reform of these injustices was later known and celebrated among Athenians as the Seisachtheia (shaking off of burdens). As with all his reforms, there is considerable scholarly debate about its real significance. Many scholars are content to accept the account given by the ancient sources, interpreting it as a cancellation of debts, while others interpret it as the abolition of a type of feudal relationship, and some prefer to explore new possibilities for interpretation. prohibition on a debtor's person being used as security for a loan. release of all Athenians who had been enslaved.

The removal of the horoi clearly provided immediate economic relief for the most oppressed group in Attica, and it also brought an immediate end to the enslavement of Athenians by their countrymen. Some Athenians had already been sold into slavery abroad and some had fled abroad to escape enslavement Solon proudly records in verse the return of this diaspora. It has been cynically observed, however, that few of these unfortunates were likely to have been recovered. It has been observed also that the seisachtheia not only removed slavery and accumulated debt, it also removed the ordinary farmer's only means of obtaining further credit.

The seisachtheia however was merely one set of reforms within a broader agenda of moral reformation. Other reforms included: the abolition of extravagant dowries. legislation against abuses within the system of inheritance, specifically with relation to the epikleros (i.e. a female who had no brothers to inherit her father's property and who was traditionally required to marry her nearest paternal relative in order to produce an heir to her father's estate). entitlement of any citizen to take legal action on behalf of another. the disenfranchisement of any citizen who might refuse to take up arms in times of civil strife, a measure that was intended to counteract dangerous levels of political apathy.

The personal modesty and frugality of the rich and powerful men of Athens in the city's subsequent golden age have been attested to by Demosthenes. Perhaps Solon, by both personal example and legislated reform, established a precedent for this decorum. A heroic sense of civic duty later united Athenians against the might of the Persians. Perhaps this public spirit was instilled in them by Solon and his reforms. Also see Solon and Athenian sexuality

The literary merit of Solon's verse is generally considered unexceptional. Solon the poet can be said to appear 'self-righteous' and 'pompous' at times and he once composed an elegy with moral advice for a more gifted elegiac poet, Mimnermus. Most of the extant verses show him writing in the role of a political activist determined to assert personal authority and leadership and they have been described by the German classicist Wilamowitz as a "versified harangue" (Eine Volksrede in Versen). According to Plutarch however, Solon originally wrote poetry for amusement, discussing pleasure in a popular rather than philosophical way. Solon's elegiac style is said to have been influenced by the example of Tyrtaeus. He also wrote iambic and trochaic verses which, according to one modern scholar, are more lively and direct than his elegies and possibly paved the way for the iambics of Athenian drama.

Read the original here:
Solon urges Congress to conduct cursory check on the status of stem cell therapy in the country (15966897)

SHANGHAI doctors announced the success of a novel technology that uses people's own skin via stem cells to grow a new face for seriously disfigured patients.

It's an alternative to the surgery used in the West in which doctors transplant the face from a dead body to a patient.

Facial tissue developed with the new technology is more readily accepted physically and psychologically by patients and has no ethical issues, doctors from Shanghai No. 9 People's Hospital said yesterday.

Since adopting the new technology, doctors have used it on more than 60 patients, including seven who needed their whole face replaced or major facial changes.

Of the seven, six were a success, while one case failed as skin on part of the face died, doctors said.

Patients include women disfigured by having sulfuric acid splashed in their faces, people who lost their nose during a fight and a person whose face was seriously burned in a fire.

Under the technology, doctors remove certain blood vessels from the patient's leg to build a small vessel net and transplant it into a place on the body to grow the new face, usually on the a patient's upper chest.

Then doctors use a skin dilator to expand the skin like a bulging ball. Later they inject the patient's own stem cells to help the skin grow stronger and stimulate the growth of blood vessels.

Soft bones which are shaped into facial features like a nose and upper jaw bone in line with the patient's own facial skeleton are then transplanted under the new facial skin.

Finally, the new face is transplanted onto the disfigured face. The new face, which is thin and comprised of a whole piece of living skin, will join with the facial muscles, thus giving a patient natural facial expressions and function to the greatest extent possible.

More:
Eastday-Shanghai doctors reveal face-change leap

Queenstown Regenerative Medicine - http://www.queenstownRM.co.nz

Professor Richard Boyd and Dr Dan Bates Latest developments of Stem Cell Therapy and Regenerative Medicine

Queenstown Regenerative Medicine, in association with Monash University Immunology and Stem Cell Centre (MISCL), has the pleasure of requesting your attendance at an evening lecture by Prof Richard Boyd, Head of MISCL and Dr Dan Bates, Sports Medicine Physician from Melbourne AFL Club.

Professor Richard Boyd is a world leader in the research and development of potential uses of stem cells to treat disease in both human and animal. He is the Director of Australia's largest and most prestigious Stem Cell Laboratory and a recipient of numerous International Awards for unique research into how stem cells and the immune system develop and how they have their effects in the body.

Professor Boyd's talk will give an overall background to stem cells and the work going on around the world to put these cellular therapies and regenerative medicine into the clinic.

Doctor Dan Bates is a Sports Medicine Physician working with Professor Boyd in the development and use of cellular medicine applications in the field of Sports Medicine and musculoskeletal injuries. Dan is the current team doctor of the Melbourne AFL club and will speak on his experiences using Platelet Rich Plasma to treat musculoskeletal injuries and the opening of stem cell treatment centres in conjunction with MISCL in Australia.

This is a unique opportunity to get first- hand knowledge from some of the best people in the field. These talks will be aimed at the practical applications of how you can use these therapies currently, as well as giving an idea of what the near future holds.

Date: Friday 21 September 2012 Time: from 6 pm 7.30 pm Location: Heritage Hotel, 91 Fernhill Road, Queenstown (Icon Conference Room) Cost: Free of charge

Scoop Media

Read this article:
Developments of Stem Cell Therapy and Regenerative Medicine

NEW YORK, Sept. 19, 2012 (GLOBE NEWSWIRE) -- NeoStem, Inc. (NYSE MKT:NBS) ("NeoStem" or the "Company"), a rapidly emerging market leader in the fast growing cell therapy market, today announced that Company management has been invited to participate at BIOX, the Noble Financial Capital Markets' Life Sciences Exposition on Monday, September 24. Company management will make a webcasted company presentation and participate in a cell therapy panel.

Noble Financial Capital Markets Investor Conference - BIOX Life Sciences Exposition

For more information about the conference, please visit http://www.nobleresearch.com/BIOX.htm.

About NeoStem, Inc.

NeoStem, Inc. continues to develop and build on its core capabilities in cell therapy, capitalizing on the paradigm shift that we see occurring in medicine. In particular, we anticipate that cell therapy will have a significant role in the fight against chronic disease and in lessening the economic burden that these diseases pose to modern society. We are emerging as a technology and market leading company in this fast developing cell therapy market. Our multi-faceted business strategy combines a state-of-the-art contract development and manufacturing subsidiary, Progenitor Cell Therapy, LLC ("PCT"), with a medically important cell therapy product development program, enabling near and long-term revenue growth opportunities. We believe this expertise and existing research capabilities and collaborations will enable us to achieve our mission of becoming a premier cell therapy company.

Our contract development and manufacturing service business supports the development of proprietary cell therapy products. NeoStem's most clinically advanced therapeutic, AMR-001, is being developed at Amorcyte, LLC ("Amorcyte"), which we acquired in October 2011. Amorcyte is developing a cell therapy for the treatment of cardiovascular disease and is enrolling patients in a Phase 2 trial to investigate AMR-001's efficacy in preserving heart function after a heart attack. Athelos Corporation ("Athelos"), which is approximately 80%-owned by our subsidiary, PCT, is collaborating with Becton-Dickinson in the early clinical exploration of a T-cell therapy for autoimmune conditions. In addition, pre-clinical assets include our VSELTM Technology platform as well as our mesenchymal stem cell product candidate for regenerative medicine. Our service business and pipeline of proprietary cell therapy products work in concert, giving us a competitive advantage that we believe is unique to the biotechnology and pharmaceutical industries. Supported by an experienced scientific and business management team and a substantial intellectual property estate, we believe we are well positioned to succeed.

For more information on NeoStem, please visit http://www.neostem.com.

Forward-Looking Statements for NeoStem, Inc.

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements reflect management's current expectations, as of the date of this press release, and involve certain risks and uncertainties. Forward-looking statements include statements herein with respect to the successful execution of the Company's business strategy, including with respect to the Company's or its partners' successful development of AMR-001 and other cell therapeutics, the size of the market for such products, its competitive position in such markets, the Company's ability to successfully penetrate such markets and the market for its CDMO business, and the efficacy of protection from its patent portfolio, as well as the future of the cell therapeutics industry in general, including the rate at which such industry may grow. Forward looking statements also include statements with respect to satisfying all conditions to closing the disposition of Erye, including receipt of all necessary regulatory approvals in the PRC. The Company's actual results could differ materially from those anticipated in these forward- looking statements as a result of various factors, including but not limited to (i) the Company's ability to manage its business despite operating losses and cash outflows, (ii) its ability to obtain sufficient capital or strategic business arrangement to fund its operations, including the clinical trials for AMR-001, (iii) successful results of the Company's clinical trials of AMR-001 and other cellular therapeutic products that may be pursued, (iv) demand for and market acceptance of AMR-001 or other cell therapies if clinical trials are successful and the Company is permitted to market such products, (v) establishment of a large global market for cellular-based products, (vi) the impact of competitive products and pricing, (vii) the impact of future scientific and medical developments, (viii) the Company's ability to obtain appropriate governmental licenses and approvals and, in general, future actions of regulatory bodies, including the FDA and foreign counterparts, (ix) reimbursement and rebate policies of government agencies and private payers, (x) the Company's ability to protect its intellectual property, (xi) the company's ability to successfully divest its interest in Erye, and (xii) matters described under the "Risk Factors" in the Company's Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 20, 2012 and in the Company's other periodic filings with the Securities and Exchange Commission, all of which are available on its website. The Company does not undertake to update its forward-looking statements. The Company's further development is highly dependent on future medical and research developments and market acceptance, which is outside its control.

Link:
NeoStem to Present at Noble Capital Markets' Life Sciences Exposition on September 24

Public release date: 17-Sep-2012 [ | E-mail | Share ]

Contact: Nicole White nicole.white@cshs.org 310-423-5215 Cedars-Sinai Medical Center

LOS ANGELES Sept. 17, 2012 Leading scientists and clinicians from across the nation will discuss the latest findings on potential stem cell treatments for diabetes and eye diseases at the second Cedars-Sinai Regenerative Medicine Scientific Symposium.

WHO: Stem cell scientists, clinicians and industry leaders.

The symposium is being hosted by the Cedars-Sinai Regenerative Medicine Institute, led by Clive Svendsen, PhD. The institute brings together basic scientists with specialist clinicians, physician scientists and translational scientists across multiple medical specialties to convert fundamental stem cell studies to therapeutic regenerative medicine.

FEATURED RESEARCH: The symposium's morning session will feature an overview of the current state of stem cells and diabetes, including efforts to start the first clinical trials with stem cells for the treatment of diabetes. Other research to be presented includes an update on regenerative medicine approaches to treating macular degeneration, a progressive deterioration of the eye that causes gradual loss of vision. This will include an update from Gad Heilweil , MD, on a key, stem-cell clinical trial on macular degeneration at the University of California Los Angeles.

WHEN: Sept. 21, 2012 8:30 a.m. to 6 p.m. Thomson's lecture begins at 8:40 a.m.

WHERE: Harvey Morse Auditorium Cedars-Sinai Medical Center 8700 Beverly Boulevard Los Angeles, CA 90048

How to register: http://www.cedars-sinai.edu/RMI

###

Read the rest here:
Leading stem cell scientists to focus on diabetes, eye diseases at Cedars-Sinai symposium

Los Angeles Times

Prop. 37: Another example of the perils of the initiative process Los Angeles Times Genetic engineering, or genetic modification, which involves manipulating DNA or transferring it from one species to another, is increasingly common in agriculture and food processing, and wouldn't be banned or even regulated by the measure. Genetic ... Barnridge: Prop. 37 promises a big food fightDaily Democrat Food Fight: New ads about Proposition 37, the labeling effort for GMOs89.3 KPCC (blog) Students show support for Proposition 37Lariat Saddleback College EI Tecolote -Forbes -New York Times (blog) all 45 news articles »

Source:
http://news.google.com/news?q=genetic-engineering&output=rss

Forbes

Move Over Genetic-Engineering; Biomimicry Seems The Better Bet For Solving ... Forbes Move Over Genetic-Engineering; Biomimicry Seems The Better Bet For Solving Global Hunger. 0 comments, 0 called-out. + Comment now. + Comment now. Biomimicry is maybe the best idea you haven't heard too much about. The term, for those unfamiliar, ...

Source:
http://news.google.com/news?q=genetic-engineering&output=rss

Daily Mail

Biotechnology Stock in Focus; Neuralstem Inc. (CUR) SmallCap Network Shares of Neuralstem Inc. (AMEX: CUR) surged in trading on Thursday after the biotechnology company announced that its neural stem cells were part of a study published online in a leading scientific journal CELL. In the study, called Long Distance ... Neuralstem Gains on Stem Cell Therapy for Paralyzed RatsBusinessweek all 100 news articles »

Source:
http://news.google.com/news?q=biotechnology&output=rss

Times of Swaziland

Biotechnology Park to boost economy - King Times of Swaziland He called upon every Swazi citizen to impart knowledge, skills and expertise to fulfil government's endeavour of attaining First World status through the construction of the much anticipated Science and Biotechnology Park, among other priorities. The ... and more »

Source:
http://news.google.com/news?q=biotechnology&output=rss

Proactive Investors UK

Angel Biotechnology expects to complete Materia Medica jv by end of 2012 Proactive Investors UK Biopharmaceutical contract manufacturer Angel Biotechnology (LON:ABH) expects its joint venture with Russian firm Materia Medica to be completed before the end of the year. The firm is still dealing with the details of the agreement, it said in a wide ...

Source:
http://news.google.com/news?q=biotechnology&output=rss

I always read WIRED magazine.  Aside from GEN it's the only magazine I read.  Just reading something outside of cell therapy or biotech often infuses me with an idea that otherwise would have never occurred to me like the need for a cell therapy X Prize or cellular aggregates as microcarriers or tissue-engineered memory and processing devices or even just the conviction to better represent cell therapy to the broader world out there of scientists, engineers, journalists, policy-makers, or perhaps people with too much money looking to be inspired and wanting to make a difference.

http://www.celltherapyblog.com hosted by http://www.celltherapygroup.com

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

Looking for a good job at an
enterprise that is on the cutting edge of biotechnology?

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

StemCells, Inc., said yesterday that it
will come up with the $40 million needed to match loans from the California
stem cell agency through “existing infrastructure and overhead”
and will not be issuing stocks or warrants to the agency.

“We have incurred significant
operating losses since inception. We expect to incur additional
operating losses over the foreseeable future. We have very limited
liquidity and capital resources and must obtain significant
additional capital and other resources in order to provide funding
for our product development efforts....”

Martin McGlynn StemCells, Inc., Photo

“To be clear, we do not interpret the
diligence requirement as an obligation to raise a specific amount of
money in a particular period of time, and we wish to correct the
misstatements made by some uninformed third parties that the ICOC is
requiring us to raise $20 million in matching funds. In
point of fact, we expect that a substantial amount of our
contribution towards these projects will come from existing
infrastructure and overhead, salaries for our existing personnel, and
other contributions in kind. Furthermore, we will soon be
reviewing the budgets for both projects in detail with CIRM
staff. Because each disease team budget was prepared on a
stand-alone basis, we expect to see significant economies and
efficiencies now that the company has in fact been awarded funding
for both.”

"Under this particular CIRM
program (RFA 10-05), funding for companies will be in the form of
unsecured, non-recourse, interest-bearing, term loans, which will be
forgivable in the event the funded research fails to result in a
commercialized product. On the other hand, should the product be
successfully commercialized, CIRM would earn milestone payments
depending on how successful the product becomes. Because CIRM
shares the downside risk, and could participate handsomely on the
upside, the structure makes the loan about as close to 'equity' as one could, without having to dilute existing shareholders in order
to gain access to significant amounts of capital.  The company
will not issue stock, warrants or other equity to CIRM in connection
with these awards. 

"Of course, we realize that CIRM
prefers that applicants from industry provide evidence of their
ability to secure whatever additional funds may be needed to complete
any CIRM-funded project, in this case the filing of an IND for each
indication. This is stated in the text of RFA 10-05 itself and
was repeated in various comments by CIRM staff during the application
process. When making the second award on September 5, the
ICOC naturally recognized the sizeable commitment it was making
to StemCells, so it instructed CIRM staff to satisfy themselves
of the company's ability to access the capital needed to fund the
project, namely the Alzheimer's program through to the filing of the
IND.”

"Finally, I can confirm that in
June of this year the Company applied for up to $10
million under CIRM's Strategic Partnership I program
(RFA 12-05). Unlike the disease team awards under RFA
10-05, if companies are approved for funding under RFA 12-05, they
may elect to take such funding in the form of a grant, not a
loan. Our application under RFA 12-05 is for a controlled Phase
II clinical trial of HuCNS-SC cells in Pelizaeus-Merzbacher disease
(PMD), a rare myelination disorder. StemCells completed a Phase
I study in PMD in February 2012 and in April announced that
all of the patients from that study showed evidence of cell-derived
myelination and three of the four patients in the study showed
measurable gains in motor and/or cognitive function.”

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

During the last few months, the $3 billion California stem cell
agency, which is approaching its eight-year anniversary, has chalked up a
number of important firsts.

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

SAN DIEGO UC San Diego researchers have used early stage stem cells to restore movement in rats that had suffered severe spinal cord injuries. Researchers say the stem cells in essence rewired the spinal cord.

Previous studies showed stem cells implanted at the site of a spinal cord injury didn't survive for long.

In this study, researchers embedded the stem cells in a gel. That allowed the cells to stick to and completely fill the injury site. Scientists also added a cocktail of growth factors.

UC San Diego's Mark Tuszynski, the study's principal investigator, said the technique generated an enormous amount of new neurons.

"That amount of growth, and the ability of this to improve function after the most severe spinal cord injury, moves this into the realm of potential human translation," Tuszynski explained.

Tuzynski said after the therapy, rats were able to move their hind legs again, but not at full strength.

Paul Lu, assistant research scientist at UC San Diego's Center for Neural Repair, contributed to the study.

Their research is published in the journal Cell.

Originally posted here:
UC San Diego Researchers Use Stem Cells To Restore Mobility

ScienceDaily (Sep. 11, 2012) By including chemotherapy as a conditioning regimen prior to treatment, researchers have developed a refined gene therapy approach that safely and effectively restores the immune system of children with a form of severe combined immunodeficiency (SCID), according to a study published online September 11 in Blood, the Journal of the American Society of Hematology (ASH).

SCID is a group of rare and debilitating genetic disorders that affect the normal development of the immune system in newborns. Infants with SCID are prone to serious, life-threatening infections within the first few months of life and require extensive treatment for survival beyond infancy.

Adenosine deaminase (ADA) deficiency, which accounts for approximately 15 percent of all SCID cases, develops when a gene mutation prohibits the production of ADA, an enzyme that breaks down toxic molecules that can accumulate to harmful levels and kill lymphocytes, the specialized white blood cells that help make up the immune system. In its absence, infants with ADA-deficient SCID lack almost all immune defenses and their condition is almost always fatal within two years if left untreated. Standard treatment for ADA-deficient SCID is a hematopoietic stem cell transplant (HSCT) from a sibling or related donor; however, finding a matched donor can be difficult and transplants can carry significant risks. An alternate treatment method, enzyme replacement therapy (ERT), involves regular injections of the ADA enzyme to maintain the immune system and can help restore immune function; however, the treatments are extremely expensive and painful for the young patients and the effects are often only temporary.

Given the limitations of HSCT and ERT, in the 1990s researchers began investigating the efficacy of gene therapy for ADA-deficient SCID. They discovered that they could "correct" the function of a mutated gene by adding a healthy copy into the cells of the body that help fight infectious diseases. Since then, there have been significant advances in gene therapy for SCID, yet successful gene therapy in patients with ADA-deficient SCID has been seen in only a small series of children due to the difficulty of introducing a healthy ADA gene into bone marrow stem cells and to engraft these cells back into the patients.

"Although the basic steps of gene therapy for patients with SCID have been known for a while, technical and clinical challenges still exist and we wanted to find an optimized gene therapy protocol to restore immunity for young children with ADA-deficient SCID," said Fabio Candotti, MD, one of the study's senior authors, senior investigator in the Genetics and Molecular Biology Branch of the National Human Genome Research Institute at the National Institutes of Health, and chair of the ASH Scientific Committee on Immunology and Host Defense.

To determine whether an enhanced gene therapy approach would improve immunity in children with ADA-deficient SCID, the teams of Dr. Candotti and Donald B. Kohn, MD, director of the Human Gene Medicine Program at the University of California, Los Angeles (UCLA), Professor of Pediatrics and of Microbiology, Immunology, and Molecular Genetics, and a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, conducted a clinical trial in 10 patients with the disorder. For the first time, Drs. Candotti and Kohn and their team of investigators compared two different retroviral vectors, MND-ADA and GCsapM-ADA, to transport normal ADA genes into the young patients' bone marrow stem cells as well as two different treatment plans in preparation for receiving gene therapy. Following therapy, investigators found that more bone marrow stem cells were marked with the MND-ADA vector, demonstrating its superiority over the GCsapM-ADA vector.

The investigators also sought to determine whether providing a low dose of chemotherapy prior to gene therapy, known as a pre-transplant conditioning regimen, would successfully deplete the young patients' bone marrow stem cells and make room for gene-corrected stem cells. In four patients, gene therapy was performed without chemotherapy, and the patients remained on ERT throughout the entire procedure to evaluate the efficiency of ERT combined with gene therapy. While these patients did not experience any adverse effects, they also did not experience a significant increase in their levels of the ADA enzyme. They also maintained low absolute lymphocyte counts (ALC) and minimal immune system function, leading the researchers to believe that ERT may weaken the therapy's effect by diluting the number of gene-corrected lymphocytes.

The remaining six patients were treated with the chemotherapy drug busulfan prior to gene therapy and ERT was discontinued prior to the gene therapy procedure. A significant increase in ADA was observed in all six patients; half of them remain off of ERT with partial immune reconstitution -- findings that support results from prior trials in Italy and the United Kingdom using chemotherapy prior to gene therapy and discontinuting ERT. While the ALC of all six patients declined sharply in the first few months due to combined effects of busulfan administration and ERT withdrawal, their counts increased from six to 24 months, even in the three patients that remained off of ERT. After adjusting the chemotherapy dosage, investigators were able to determine an optimal level for enhancing the efficacy of the gene-therapy-corrected cells with minimal toxicity.

This study is the first to detail comparisons of ADA-deficient SCID patient outcomes between those treated with gene therapy who have not received pre-transplant conditioning while continuing to receive ERT with those receiving pre-transplant conditioning without the administration of ERT. This study is also the first to compare two different viral vectors to transport normal ADA genes into patient bone marrow.

"We were very happy that in this trial we were able to see a benefit in the patients after we modified the protocol," said Dr. Kohn. "Doctors treating ADA-deficient SCID have had too few options for too long, and we hope this will provide them with an efficient and effective treatment for this devastating disease."

Go here to see the original:
Gene therapy technique for children with immune disorder improved

ScienceDaily (Sep. 11, 2012) UCLA stem cell researchers have found that a gene therapy regimen can safely restore immune systems to children with so-called "Bubble Boy" disease, a life threatening condition that if left untreated can be fatal within one to two years.

In the 11-year study, researchers were able to test two therapy regimens for 10 children with ADA-deficient severe combined immunodeficiency (SCID). During the study, they refined their approach to include a light dose of chemotherapy to help remove many of the blood stem cells in the bone marrow that are not creating an enzyme called adenosine deaminase (ADA), which is critical for the production and survival of healthy white blood cells, said study senior Dr. Donald Kohn, a professor of pediatrics and of microbiology, immunology, and molecular genetics in Life Sciences and a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA.

The refined gene therapy and chemotherapy regimen proved superior to the other method tested in the study, restoring immune function to three of the six children who received it, Kohn said. Going forward, an even further refined regimen using a different type of virus delivery system will be studied in the next phase of the study, which already has enrolled eight of the 10 patients needed.

The study appears Aug. 30 in the advance online issue of the peer-reviewed journal Blood.

"We were very happy that in the human trials we were able to see a benefit in the patients after we modified the protocol," Kohn said. "Doctors treating ADA-deficient SCID have had too few options for too long, and we hope this will provide them with an efficient and effective treatment for this devastating disease."

Children born with SCID, an inherited immunodeficiency, are generally diagnosed at about six months. They are extremely vulnerable to infectious diseases and don't grow well. Chronic diarrhea, ear infections, recurrent pneumonia and profuse oral candidiasis commonly occur in these children. SCID cases occur in about 1 of 100,000 births

Currently, the only treatment for ADA-deficient SCID calls for injecting the patients twice a week with the necessary enzyme, Kohn said, a life-long process that is very expensive and often doesn't return the immune system to optimal levels. These patients also can undergo bone marrow transplants from matched siblings, but matches can be very rare.

About 15 percent of all SCID patients are ADA-deficient. Kohn and his team used a virus delivery system that he had developed in his lab in the 1990s to restore the gene that produces the missing enzyme necessary for a healthy immune system. To date, about 40 children with SCID have received gene therapy in clinical trials around the world, Kohn said.

Two slightly different viral vectors were tested in the study, each modified to deliver healthy ADA genes into the bone marrow cells of the patients so the needed enzyme could be produced and make up for the cells that don't have the gene. Four of the 10 patients in the study remained on their enzyme replacement therapy during the gene therapy study. There were no side effects, but their immune systems were not sufficiently restored, Kohn said.

In the next six patients, the enzyme therapy was stopped and a small dose of chemotherapy was given before starting the gene therapy to deplete the ADA-deficient stem cells in their bone marrow. Of those patients, half had their immune systems restored. The human findings confirmed another study, also published recently in Blood by Kohn and UCLA colleague Dr. Denise Carbonaro-Sarracino, which tested the techniques in parallel, using a mouse model of ADA-deficient SCID.

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Stem cell researchers use gene therapy to restore immune systems in 'Bubble Boy' disease

Public release date: 11-Sep-2012 [ | E-mail | Share ]

Contact: Kim Irwin kirwin@mednet.ucla.edu 310-435-9457 University of California - Los Angeles Health Sciences

UCLA stem cell researchers have found that a gene therapy regimen can safely restore immune systems to children with so-called "Bubble Boy" disease, a life threatening condition that if left untreated can be fatal within one to two years.

In the 11-year study, researchers were able to test two therapy regimens for 10 children with ADA-deficient severe combined immunodeficiency (SCID). During the study, they refined their approach to include a light dose of chemotherapy to help remove many of the blood stem cells in the bone marrow that are not creating an enzyme called adenosine deaminase (ADA), which is critical for the production and survival of healthy white blood cells, said study senior Dr. Donald Kohn, a professor of pediatrics and of microbiology, immunology, and molecular genetics in Life Sciences and a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA.

The refined gene therapy and chemotherapy regimen proved superior to the other method tested in the study, restoring immune function to three of the six children who received it, Kohn said. Going forward, an even further refined regimen using a different type of virus delivery system will be studied in the next phase of the study, which already has enrolled eight of the 10 patients needed.

The study appears Aug. 30 in the advance online issue of the peer-reviewed journal Blood.

"We were very happy that in the human trials we were able to see a benefit in the patients after we modified the protocol," Kohn said. "Doctors treating ADA-deficient SCID have had too few options for too long, and we hope this will provide them with an efficient and effective treatment for this devastating disease."

Children born with SCID, an inherited immunodeficiency, are generally diagnosed at about six months. They are extremely vulnerable to infectious diseases and don't grow well. Chronic diarrhea, ear infections, recurrent pneumonia and profuse oral candidiasis commonly occur in these children. SCID cases occur in about 1 of 100,000 births

Currently, the only treatment for ADA-deficient SCID calls for injecting the patients twice a week with the necessary enzyme, Kohn said, a life-long process that is very expensive and often doesn't return the immune system to optimal levels. These patients also can undergo bone marrow transplants from matched siblings, but matches can be very rare.

About 15 percent of all SCID patients are ADA-deficient. Kohn and his team used a virus delivery system that he had developed in his lab in the 1990s to restore the gene that produces the missing enzyme necessary for a healthy immune system. To date, about 40 children with SCID have received gene therapy in clinical trials around the world, Kohn said.

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UCLA stem cell researchers use gene therapy to restore immune systems in 'bubble babies'