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Stem Cell Research: Heart Stem Cells May Help Heal Hearts After Heart Attack

Posted: August 22, 2012 at 2:10 am

Two men in landmark heart stem cell study tell their stories.

Jim Dearing of Louisville, Ky., one of the first men in the world to receive heart stem cells, might have helped start a medical revolution that could lead to a cure for heart failure.

Three years after getting the experimental stem cell procedure, following two heart attacks and heart failure, Dearings heart is working normally.

2012 WebMD, LLC. All rights reserved.

The difference is clear and dramatic -- and it's lasting, according to findings now being made public for the first time.

Dearing first showed "completely normal heart function" on an echocardiogram done in 2011, says Roberto Bolli, MD, who is leading the stem cell trial at the University of Louisville. Those results have not been published before.

That was still true in July 2012, when Dearing again showed normal heart function on another echocardiogram.

Based on those tests, Bolli says, "Anyone who looks at his heart now would not imagine that this patient was in heart failure, that he had a heart attack, that he was in the hospital, that he had surgery, and everything else."

It's not just Dearing who has benefited. His friend, Mike Jones, who had even more severe heart damage, also got the stem cell procedure in 2009. Since then, scarred regions of his heart have shrunk. His heart now appears leaner and stronger than it was before.

"What's striking and exciting is that we're seeing what appears to be a long-lasting improvement in function," Bolli says. If larger studies confirm the findings, "potentially, we have a cure for heart failure because we have something that for the first time can actually regenerate dead tissue."

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Stem Cell Treatments: False Hope Warning Signs

Posted: August 22, 2012 at 2:10 am

Unproven, Risky Treatments Mislead Patients to Seek Cutting-Edge Therapy

There's a dark side to stem cells: bogus treatments that prey on patients' hopes when mainstream medicine has little to offer.

Stephen Byer stepped far outside typical medical care when his son, Ben, had ALS. He took Ben to China for stem cell-like treatments, and later helped hundreds of people do the same, believing it would help them.

The unproven procedure could have killed Ben. It didn't -- but it also didn't work. Ben later died of ALS. So did the ALS patients Byer now regrets helping get the treatment.

Why take the chance? For Byer, it started with misleading promises online.

"The Internet, while increasing communication, has spawned a horde of charlatans and creeps," Byer says. "We were suckered into one of the earlier forms of stem cell chicanery."

But not everyone who seeks unapproved stem cell treatments feels ripped off. Even though the stem cell treatments Dawn Gusty got in Tijuana, Mexico, didn't ease her multiple sclerosis, she doesn't look back with regret.

That moment -- when hope surpasses science, and when someone claims to be able to bridge that gap -- may be one of the riskiest for patients to handle. And it's one of the most alarming for stem cell experts.

"It is a very dangerous situation," says Joshua Hare, MD, director of the Interdisciplinary Stem Cell Institute at the University of Miami.

Make no mistake: Hare is all for scientific stem cell research. His concern, he says, is "hype" that glosses over an inconvenient fact: There are no new approved stem cell therapies.

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Heart Stem Cell Trial: Interview With Researcher Roberto Bolli, MD

Posted: August 22, 2012 at 2:10 am

An interview with Roberto Bolli, MD.

University of Louisville cardiologist Roberto Bolli, MD, led the stem cell study that tested using patients' own heart stem cells to help their hearts recover from heart failure. Though that trial was preliminary, the results look promising -- and may one day lead to a cure for heart failure.

Here, Bolli talks about what this work means and when it might become an option for patients.

2012 WebMD, LLC. All rights reserved.

"Realistically, this will not come... for another three or four years, at least," Bolli says. "It may be longer, depending on the results of the next trial, of course."

Larger studies are needed to confirm the procedure's safety and effectiveness. If those succeed, it could be "the biggest advance in cardiovascular medicine in my lifetime," Bolli says.

A total of 20 patients took part in the initial study.

All of them experienced significant improvement in their heart failure and now function better in daily life, according to Bolli. "The patients can do more, there's more ability to exercise, and the quality of life improves markedly," Bolli says.

Bolli's team published its findings on how the patients were doing one year after stem cell treatment in November 2011 in the Lancet, a British medical journal.

Each patient was infused with about 1 million of his or her own cardiac stem cells, which could eventually produce an estimated 4 trillion new cardiac cells, Bolli says. His team plans to follow each patient for two years after their stem cell procedure.

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

Posted: August 21, 2012 at 9:10 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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Sutter Neuroscience Institute launching trial of cord blood stem cells in autistic children

Posted: August 21, 2012 at 9:10 am

The Sutter Neuroscience Institute in Sacramento plans to launch groundbreaking research today to discover whether infusing umbilical cord stem cells into the bloodstreams of autistic children will help them overcome debilitating characteristics of the condition.

The clinical trial is the first of its kind to be approved by the U.S. Food and Drug Administration, said Dr. Michael Chez, the principal investigator and Sutter's director of pediatric neurology.

One of the world's foremost autism experts at the institute, which is recognized for its practice of pediatric neuroscience, Chez said the research may help identify a valuable new tool in the struggle against autism spectrum disorders, which now affect about one in 88 children nationwide, and one in 54 boys.

"This is an exciting trial, because it's exposing us to the new frontier of stem cells and whether they may have some positive effect on this disease," Chez said. "This is the start of a new age of research in stem cell therapies for chronic diseases such as autism."

Recent research has revealed robust new uses for stem cells. Stem cells, for example, can now be developed from heart muscle in cardiac patients and injected back into the heart for improved functioning. Doctors are also having success using stem cells to treat leukemia and bone marrow diseases.

Autism is the leading cause of delayed development in children, typically surfacing before 3 years of age. The condition is characterized by impaired communication, repetitive thoughts and behavior and difficulty in socialization.

The likelihood of a child's being given a diagnosis of an autism spectrum disorder increased more than 20 percent from 2006 to 2008, according to a report this year by the Centers for Disease Control and Prevention. Locally, more than 3,830 students in the four-county Sacramento region were counted as autistic in December 2011, up 13 percent from the previous year, according to state data.

The Sutter clinical trial follows promising research in using cord blood stem cells to help children with cerebral palsy, a brain disorder that shares some characteristics with autism, to improve motor function.

Chez became the point man for developing protocols for the clinical trial over a year ago, after being contacted by Cord Blood Registry, the world's largest cord blood bank. The Bay Area company has collected and frozen 400,000 samples of umbilical cord blood for individuals and families.

The umbilical cord that links a mother's placenta to her fetus is imbued with blood stem cells. Since the 1990s, parents increasingly have opted to have their babies' umbilical cords frozen in storage as a sort of insurance against future disease. The trend now extends to about 5 percent of parents.

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CSU vets seeking cats with chronic kidney disease

Posted: August 20, 2012 at 11:17 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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Building Body Parts: Ears, Muscles and More!

Posted: August 20, 2012 at 2:11 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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ACT Announces Scotland’s NHS Lothian as Additional Site for EU Clinical Trial Using hESC-Derived RPE Cells for Macular …

Posted: August 20, 2012 at 2:11 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.

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

Posted: August 19, 2012 at 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
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
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
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

Posted: August 19, 2012 at 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.

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