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Rejected Grants May Be Sent Back for More Review

July 29th, 2012 3:52 pm


California stem cell agency chairman J.T. Thomas has raised the possibility of sending some of the disease team applications back for additional review if the board feels that is necessary to consider new information and resolve scientific disputes. 

Thomas discussed such a move at the beginning of the discussion of the $243 million disease team round. Nine out of 15 rejected applicants have appealed to the full board.  Some have presented new information. Others have disputed the scientific work of reviewers. 
The board used such a referral process for one application in the past. That grant was ultimately approved.

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Stem Cell Directors Order More Consideration on $20 Million UCLA Research Application

July 29th, 2012 3:52 pm


Directors of the California stem cell
agency today deferred action on a $20 million proposal that was
rejected by its grant reviewers and sent it back for more consideration. 

The move involved an application by
Stanley Nelson and M. Carrie Miceli of UCLA dealing with Duchenne
muscular dystrophy. They are parents of a child with the affliction,
which is usually fatal by age 25. The CIRM review summary said it is
“a devastating and incurable muscle-wasting disease caused by
genetic mutations in the gene that codes for dystrophin, a protein
that plays a key role in muscle cell health.”
Nelson had filed an appeal seeking to
overturn reviewers' action. Five  mothers and two fathers with
children suffering from the disease made emotional appeals to
CIRM directors on behalf of the application. Following their presentation, Art
Torres
, co vice chairman of the CIRM board, responded equally
emotionally that the board is dedicated to finding therapies for such
afflictions as Duchenne muscular dystrophy
The board approved more review for the
application after it was disclosed that a company issued a press
release two days ago that showed that  a drug involved in the
proposal was more effective than reviewers believed. Philip Pizzo, a
member of the board and dean of the Stanford medical school,
expressed caution about the press release, given its timing and
source. His comments came prior to the appearance of the parents.
The application is scheduled to be
brought back to the full board in September for further action.

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California Stem Cell Directors Open Meeting

July 29th, 2012 3:52 pm


Today's session of the governing board of the $3 billion California stem cell agency has begun. The major item on the agenda is a $243 million grant round that has triggered a record pace for appeals by rejected applicants. At the request of the California Stem Cell Report, the agency has provided the conflict of interest list used by the agency to determine which directors will not be allowed today to vote or participate in the discussion of specific applications.  The list can be found below. Conflict of Interest List  -- CIRM Directors Meeting 7-26-12ound below.  

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Short-term Borrowing to Continue to Provide California Stem Cell Cash

July 29th, 2012 3:52 pm


The California stem cell agency will continue to be funded with short-term borrowing -- commercial paper -- provided through the state treasurer's office, J.T. Thomas, chairman of the agency's governing board, said today. 

Until late last year, the $3 billion agency was funded through state bond funds, but California Gov. Jerry Brown is trying to reduce the state's long-term debt load, which has skyrocketed in the last decade. 
Thomas told directors this morning that short-term funding comes at "the lowest possible interest rate." He said the arrangement leaves the agency in "very good shape."
The state will provide the funding to CIRM on a month-to-month basis in addition to providing a two month cushion, Thomas said. 
At some point, the short-term debt is likely to covered by state bonds.  As of June 30, CIRM
had $50.9 million on hand, down $42 million from April 30. During the fiscal year ending June 30, the agency paid out $232.7 million compared to $201.4 million in the previous fiscal year. 

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Blood vessels from liposuction stem cells

July 29th, 2012 1:11 am

NEW ORLEANS, July 28 (UPI) -- Adult stem cells extracted during liposuction can be used to grow new, small-diameter blood vessels for use in heart bypass surgery, U.S. researchers say.

Lead author Matthias Nollert -- an associate professor at the University of Oklahoma School of Chemical, Biological and Materials Engineering, in Norman, Okla. -- said the liposuction-derived vessels, grown in a laboratory, could help solve major problems associated with grafting blood vessels from elsewhere in the body or from using artificial blood vessels that are not living tissue.

In the study, adult stem cells derived from fat were turned into smooth muscle cells in the laboratory, and then "seeded" onto a very thin collagen membrane.

As the stem cells multiplied, the researchers rolled them into tubes matching the diameter of small blood vessels. In three to four weeks, they grew into usable blood vessels, Nollert said.

"Current small-diameter vessel grafts carry an inherent risk of clotting, being rejected or otherwise failing to function normally," Nollert said in a statement. "Our engineered blood vessels have good mechanical properties and we believe they will contract normally when exposed to hormones. They also appear to prevent the accumulation of blood platelets -- a component in blood that causes arteries to narrow."

The findings were presented at the American Heart Association's Basic Cardiovascular Sciences scientific sessions in New Orleans.

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Medical Megatrends – Stem Cells – Part II of III

July 29th, 2012 1:10 am

Imagine a man with a recent severe heart attack who has the muscle repaired with stem cells or a child with a severe bladder defect repaired with stem cells grown on a biodegradable scaffold. Sounds like science fiction but these are actual clinical studies in progress today. Stem cell therapies promise to be one of those scientific breakthroughs that will have an enormous impact on health care in the future. Stem cells will bring us closer to the goal of personalized medicine, just as genomics is doing. The course of a disease will change once we have the technology to develop and then insert stem cells into the human body to actually create a tissue. For example, a person with a heart attack will not go on to live the rest of his or her life with damaged heart muscle and resultant heart failure. Instead, stem cells will repopulate the heart muscle and make it whole again. Similarly, a person with Parkinsons disease will recover full faculties thanks to the ability of stem cells to regenerate the damaged area of the brain. The person with type I diabetes will be free of the disease because of the formation of new pancreatic islet cells. The athlete will play again because new cartilage will be created for the worn knee. This is the promise of regenerative medicine. I have written the above as though each will definitely happen, a promise that will be kept. They probably will, but it may be a long time before the science of stem cells is sufficiently developed that these types of incredible results will be commonplace. Adult stem cells are being used today for treatment of a few diseases and there are studies ongoing and planned for many additional possibilities. Lets consider a few of them. Each of our tissues has a population of cells that can divide as needed to keep the organ or tissue functional as cells die or are injured. We see this with our skin as it constantly lays down new cells which make their way to the surface as the dead cells on the surface are rubbed off in the shower. We also see it when we cut ourselves and yet in a few days the wound is completely healed that was stem cells at work. It appears that essentially every organ has its own pool of such cells. There are cells in the bone marrow that can become stem cells for many different tissues. These cells circulate in the blood and can be called to assist a tissue or organ to rebuild itself after injury or damage. So for example, if a surgeon takes one half of a fathers liver for transplantation into his son, we know that the fathers liver will grow back to normal size within about 6 to 8 weeks. Some of the stem cells will have been those already in the liver but some will have come from the blood stream to assist. Of course, the liver is the exception to the rule that if a portion of an organ is removed by trauma or surgery, it will not grow back. Cut off your finger and stem cells will help it to heal but not to grow back to its original state. Adult stem cells are the ones used for treating leukemia, myeloma and other cancers and for correcting certain childhood immune deficiencies. Most often is the use of allogeneic hematopoietic stem cell transplantation, meaning the use of stem cells obtained from a closely matched individual. An identical twin is ideal but few have such a potential donor. Only 25% of siblings will likely match completely. This leaves the use of the National Marrow Donor Registry to find as close a match as possible from unrelated individuals. The Registry has markedly improved the chances for a close match and thus for successful transplantation outcomes. Many parents are now having umbilical cord blood saved and frozen to have available in the unlikely event that their child requires a transplant many years later. Although these cells are identical they usually are not sufficient in numbers to lead to engraftment and often the white blood cells (neutrophils) recover only very slowly leaving a prolonged period of infection risk. Perhaps a technique will be found to get the umbilical stem cells to multiply in the laboratory so that a larger number would be available. Adult stem cells are being used in studies of myocardial infarction and heart failure. Current guidelines of immediate angioplasty and stent insertion as appropriate help protect the heart from permanent damage after an infarct. Still, about 400,000 new cases of heart failure are developing in the USA each year. Long term survival is limited once overt failure develops. Could the damaged heart muscle be fixed? The concept is to use stem cells to repopulate the muscle fibers and to have those cells divide over and over and differentiate into new muscle fibers or perhaps also the small vessels that carry blood to the muscle cells. So far there are some exciting animal studies and even some trials in patients that are encouraging enough to warrant further evaluations. For example, one study uses adult mesenchymal stem cells derived from the bone marrow and infused intravenously within 7 days after a heart attack. 42 centers are collaborating in this double blind, randomized trail in conjunction with Osiris Therapeutics. 220 patients will receive either the stem cells or a placebo and then be monitored with various imaging and functional studies. So, stay tuned. Another common albeit less lethal problem is loss of bladder control leading to incontinence. There are studies in progress to determine if stem cells placed into the bladders sphincter muscle will help it regain control. The adult stem cells are obtained from a leg muscle biopsy. Stem cells are isolated and allowed to grow in tissue culture. These are then injected into the weakened bladder sphincter muscle. Once again, these are studies just beginning but with intriguing early results. Here is another bladder repair concept. When the bladder muscle is weak or largely missing in children it may be possible to literally rebuild the bladder by tissue engineering. A biopsy of the bladder yields cells that can be grown in the laboratory to large numbers. They can then be placed on a biodegradable scaffold and grown further. In time they seem to create a new bladder muscle wall complete with blood vessels. This layer of cells can be implanted in the bladder of children with a defect. Once more I need to note that it is still early days in these studies but they do raise exciting possibilities. The message here is that adult stem cells are being used today for life threatening and life impairing diseases with excellent success and are being studied in other diseases with exciting prospects for the future.

Stephen C Schimpff, MD is an internist, professor of medicine and public policy, former CEO of the University of Maryland Medical Center and is chair of the advisory committee for Sanovas, Inc. and senior advisor to Sage Growth Partners. He is the author of The Future of Medicine Megatrends in Healthcare and The Future of Health Care Delivery- Why It Must Change and How It Will Affect You from which this post is partially adapted. Updates are available at http://medicalmegatrends.blogspot.com

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Medical Megatrends – Stem Cells – Part II of III

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Stem cell 'makeovers' provide a way to get rid of wrinkles

July 29th, 2012 1:10 am

MIAMI (WTVJ/NBC) - It is widely known that stem cells can be used in life-saving treatments for deadly diseases.

Now they are being used in the fight against wrinkles.

Donna Pritchit recently had a "stem cell" makeover.

The 64-year-old headed into the operating room wanting to turn back the hands of time without it being totally obvious.

"I don't want someone to stop and go by and say Oh, she had a facelift.' I want to have someone say Donna went on vacation she must be having a great life,'" she said before the $5,000 procedure began.

Dr. Sharon McQuillan at the Ageless Institute in Aventura, FL marked the areas where she would take fat out of Pritchit's belly - and place it back into her face.

The retired teacher also hoped it would be her last step in getting rid of embarrassing acne scars.

The outpatient procedure began with traditional liposuction, and then McQuillan and her team processed that fat and concentrated the stem cells so they could be injected into Pritchit's wrinkles and in places where she has lost fullness.

"Stem cells in general are the cells in your body that regenerate tissue and heal tissue, and they make the skin look beautiful and younger," McQuillan explained.

While there are not many long-term studies on the procedure, McQuillan said the results are permanent.

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State stem cell research funding agency awards $20 million to UCI, StemCells Inc.

July 28th, 2012 2:14 am

Public release date: 27-Jul-2012 [ | E-mail | Share ]

Contact: Cathy Lawhon clawhon@uci.edu 949-824-1151 University of California - Irvine

Irvine, Calif., July 26, 2012 Efforts to begin human clinical trials using stem cells to treat cervical spinal cord injury in the U.S. received a $20 million boost Thursday, July 26, from the state's stem cell research funding agency, the California Institute for Regenerative Medicine.

The award will be shared by Aileen Anderson and Brian Cummings, associate professors of physical medicine & rehabilitation at UC Irvine's Sue & Bill Gross Stem Cell Research Center, and Nobuko Uchida of StemCells Inc. in Newark, Calif. Anderson and Cummings proved that transplanting human neural stem cells discovered and developed by Stem Cells, Inc. into rodents with thoracic spinal cord injury could restore mobility. The CIRM award announced Thursday will fund the collection of data necessary to establish human clinical trials in the U.S.

"Our therapeutic approach is based on the hypothesis that transplanted human neural stem cells integrate into the injured spinal cord to repair the protective myelin sheath and spinal circuitry," Anderson said. "Any therapy that can partially reverse some of the effects of spinal cord injury could substantially change the quality of life for patients by altering their dependence on assisted living and medical care."

CIRM's governing board on Thursday gave authorized $150 million for eight teams at five institutions statewide. The projects backed are considered critical to the institute's mission of translating basic stem cell discoveries into clinical cures.

"CIRM's support for UC Irvine's efforts to advance novel stem cell-based therapies for a variety of diseases is extremely gratifying," said Peter Donovan, director of the university's Sue & Bill Gross Stem Cell Research Center. "This latest award for spinal cord treatment holds great promise. We are delighted."

About 1.3 million Americans suffer chronically from spinal cord injuries. In California, nearly 147,000 individuals are living with such damage, which can severely impair the movement, sensation and autonomic function of otherwise healthy people. Recovery from spinal cord injury is often limited, even after aggressive emergency intervention with steroids and surgery, followed by rehabilitation.

"That's crushing for anyone," Anderson noted. "It's very tough for patients and their families. We believe stem cell therapies could provide significant functional recovery, improve quality of life and reduce the cost of care for those with spinal cord injury. That's our goal."

Anderson's and Cummings' laboratory has a long history of collaboration with StemCells Inc. in addressing spinal cord injury, including studies that led to the world's first clinical trial of a neural stem cell therapy for chronic spinal cord injury. This Phase I/II clinical trial, currently under way in Zurich, recently reported positive safety data from the first cohort of treated patients and continues to enroll subjects.

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Stem Cells Extracted During Liposuction Help Create Blood Vessels

July 28th, 2012 2:14 am

July 27, 2012

Lee Rannals for redOrbit.com Your Universe Online

Findings presented at the American Heart Associations Basic Cardiovascular Sciences 2012 Scientific Sessions show that adult stem cells extracted during liposuction can be used to grow small-diameter blood vessels that could be used in heart bypass surgery.

Matthias Nollert, the lead author of the study, said these liposuction-derived vessels could help solve major problems associated with grafting blood vessels from other places in the body, or form using artificial blood vessels that are not living tissue.

Current small-diameter vessel grafts carry an inherent risk of clotting, being rejected or otherwise failing to function normally, Nollert said in a press release. Our engineered blood vessels have good mechanical properties and we believe they will contract normally when exposed to hormones. They also appear to prevent the accumulation of blood platelets a component in blood that causes arteries to narrow.

Adult stem cells derived from fat are turned into smooth muscle cells in the laboratory, and then are seeded into a thin collagen membrane. As the cells multiply, the researchers rolled them into tubes matching the diameter of small blood vessels.

Within three to four weeks, the cells brew into healthy, usable small-diameter blood vessels. Millions of people with heart disease need small blood vessel replacements or grafts to restore function to damaged arteries.

Nolbert said that creating blood vessels through this technique has potential for off-the-shelf replacement vessels that can be used in graft procedures.

The researchers hope that within six months, they will have a working prototype to start testing in animals.

Although it is still just a preliminary study, further successful results in deeper studies could eventually lead to the stem cell derived small blood vessels being used during heart bypass surgery and other procedures when blood needs to be re-routed.

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Cancer Drug Unmasks HIV in Immune Cells

July 28th, 2012 2:13 am

WASHINGTON People infected with HIV, the virus that causes AIDS, must take anti-retroviral drugs for the rest of their lives in order to control their disease.

Otherwise, reservoirs of dormant virus hiding within the immune system can become active, and the infection can reemerge. Now, researchers have discovered that a cancer drug can dislodge these latent copies of the AIDS virus. They view the development as a critical step toward curing HIV-infected people.

HIV has evolved a way to survive inside the human body by integrating itself into the genetic architecture of immune-system T-cells, the specialized white blood cells targeted by the AIDS virus. Anti-retroviral drugs can suppress HIV to near undetectable levels, giving the immune system a chance to repair itself. But the AIDS virus is always lurking in miniscule numbers - roughly one in every million T cells - and threatening to come back to life should an individual ever stop taking the anti-retroviral cocktail.

Now, researchers have succeeded in flushing this latent virus out of its hiding place, with a drug used to treat lymphoma, a rare and potentially deadly cancer of the lymphatic system.

David Margolis, a professor of microbiology and immunology at the University of North Carolina Chapel Hill has been studying how HIV hides, dormant, within immune-system cells, says that in some lymphomas, the drug, vorinostat, makes cancer cells die. But Margolis adds that in HIV-infected cells, the cancer drug causes the latent virus to show itself.

Theoretically, doing this clinically would be a way to sort of unmask the hidden virus; flush the virus out of hiding," he says. "And that might then allow us to develop ways to get rid of the leftover virus in people that are on treatment so they could stop treatment and there would be nowhere for the virus to come back from.

Margolis and his colleagues studied eight HIV-infected patients who were medically stable on anti-retroviral therapy. Their levels of HIV CD4 T cells, which the virus uses to reproduce itself, were measured both before and after the men were given vorinostat.

What we saw in every single person was a tiny amount of virus detectable before the dose of the drug," he says. "And the amount of virus that was detectable went up on average about five-fold, five times, after a single exposure to the drug.

Margolis says his so-called proof of concept experiment demonstrates that HIV can be flushed out of hiding with vorinostat and then targeted for destruction by anti-AIDS drugs.

But none of the participants was cured, he adds.

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Dormant HIV gets rude awakening

July 28th, 2012 2:13 am

Following the success of antiretroviral therapy for HIV, some researchers are now focusing their attention on a loftier goal a cure. That means targeting viral reservoirs, primarily the long-lived cells of the immune system in which the virus lies dormant. Eliminating these reservoirs isnt easy, but recent research offers glimmers of hope that it may one day be possible.

The strongest proof that HIV can be cured comes from the case of Timothy Brown, who was infected with HIV until he received a stem-cell transplant in 2007 to treat leukaemia1. He has remained free of HIV since then. Browns transplant helped cure his HIV, in part, because the donor's stem cells lacked a key receptor that the virus needs to enter cells.

Particles of HIV that are invisible to the immune system must be flushed out before the disease can be said to be cured.

SCIEPRO/Getty Images

But at this week's XIX International AIDS Conference in Washington DC, Timothy Henrich, an infectious-disease physician at the Brigham and Womens Hospital in Boston, Massachusetts, reported a study of two HIV-infected men who received transplants of stem cells that did have the HIV receptor. Since they received a milder dose of chemotherapy than Brown prior to their transplants, they were able to continue taking antiretrovirals throughout the procedure. The transplants did not immediately eliminate the mens infected immune cells, but roughly ten months later, the men had no evidence of HIV in their blood.

After their transplants, both men developed graft-versus-host disease, in which donor immune cells attack the transplant patients cells. Henrich and his colleagues speculate that the antiretroviral drugs protected the donor cells from infection with HIV. These healthy donor cells then destroyed the HIV-infected cells, leaving the men free of virus.

Theoretically, they could be cured because the immune system was rebuilt under the coverage of antiviral therapy, says Steven Deeks, an HIV researcher at the University of California, San Francisco, who wasnt involved in the research. The ultimate test, however, will be to see whether the men remain HIV-free when they stop taking antiretroviral medicines. Henrich is working with the patients, their physicians and an ethics board to determine whether that is feasible.

But stem cell transplants are too risky to be used on people who dont have a life-threatening illness. This is not scalable or affordable or reasonable or ethical in anyone else, Deeks says.

A more palatable tactic would be to purge the virus from its main hiding spot the long-lived memory cells of the immune system, called CD4+ memory T cells. A paper published this week in Nature provides the first evidence that this may be possible in humans2 (see 'Drug brings HIV out of hiding'). David Margolis, an HIV expert at the University of North Carolinas Center for Infectious Diseases in Chapel Hill, and his colleagues administered a cancer drug called vorinostat (suberoylanilide hydroxamic acid) to eight people in an attempt to coax dormant HIV out of hiding.

A single dose of the medicine produced a 4.8-fold increase in HIV RNA expression. The hope is that this results in HIV particles being made and released, so that they are visible to the patient's immune system again. However, it is still unclear to scientists whether this increased expression will lead to the destruction of HIV-infected cells and shrink the viral reservoir. But it's a positive signal, says Nicolas Chomont, an HIV researcher at the Vaccine & Gene Therapy Institute of Florida in Port St Lucie.

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StemCells, Inc. Awarded $20 Million From the California Institute for Regenerative Medicine

July 27th, 2012 8:22 am

Award to Fund IND-Enabling Activities for the Company's HuCNS-SC(R) Neural Stem Cells in Cervical Spinal Cord Injury

Decision on Funding Alzheimer's Program Deferred to CIRM's September Board Meeting

NEWARK, Calif., July 26, 2012 (GLOBE NEWSWIRE) -- StemCells, Inc. (STEM) today announced that the California Institute for Regenerative Medicine (CIRM) has approved an award to the Company and its collaborators for up to $20 million under CIRM's Disease Team Therapy Development Award program (RFA 10-05). The award is to fund preclinical development of StemCells' proprietary HuCNS-SC(R) product candidate (purified human neural stem cells) as a potential treatment for cervical spinal cord injury. The award will provide funding over a maximum four-year period, with the goal of filing an investigational new drug (IND) application to begin clinical testing in that time. CIRM deferred a decision on the Alzheimer's disease application submitted by StemCells and referred the application back to CIRM's Grants Working Group for further consideration. CIRM is expected to review the application again at the next meeting of its governing board currently scheduled for September 6th.

"We understand that this was a very competitive process and we are extremely grateful to CIRM for its support," commented Martin McGlynn, President and CEO of StemCells, Inc. "We view this decision by CIRM as a strong vote of confidence in our neural stem cell technology and the world class team of scientists and clinicians who will be collaborating to translate this exciting research into potential treatments and cures for patients with spinal cord injury. We are currently conducting a Phase I/II trial in thoracic spinal cord injury. This funding now allows us the opportunity to expand testing of our cells for cervical spinal cord injury, the most common form of spinal cord injury."

StemCells will evaluate its HuCNS-SC cells as a potential treatment for cervical spinal cord injury in collaboration with a team led by Aileen Anderson, Ph.D., Associate Professor in the Departments of Physical Medicine and Rehabilitation, and Anatomy and Neurobiology at University of California, Irvine. Dr. Anderson's laboratory has a long history of collaboration with StemCells in spinal cord injury, including the studies which led to the world's first clinical trial for a neural stem cell therapeutic in chronic spinal cord injury. This Phase I/II clinical trial, currently underway in Zurich, Switzerland, recently reported positive safety data from the first cohort of treated patients, and continues to enroll patients from Europe, the United States and Canada.

About Spinal Cord Injury

Spinal cord injury affects approximately 1.3 million people in the United States, for which there are no effective treatment options. Moreover, spinal cord injuries are a significant financial drain on the public health system. Cervical spinal cord injuries represent approximately half of all spinal cord injuries, for which lifetime healthcare costs range from $1.8 to $3.3 million per patient, depending upon severity of the injury.

About CIRM

CIRM was established in November 2004 with the passage of Proposition 71, the California Stem Cell Research and Cures Act. The statewide ballot measure, which provided $3 billion in funding for stem cell research at California universities and research institutions, was overwhelmingly approved by voters, and called for the establishment of an entity to make grants and provide loans for stem cell research, research facilities, and other vital research opportunities. A list of grants and loans awarded to date may be seen here: http://www.cirm.ca.gov/for-researchers/researchfunding.

The two applications submitted by StemCells, Inc. under CIRM's RFA 10-05 for cervical spinal cord injury and for Alzheimer's disease, as well as the feedback on each application from CIRMS's grants working group, can be viewed on the CIRM website at http://www.cirm.ca.gov/research-summaries-rfa-10-05-cirm-disease-team-therapy-development-awards.

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First ever child recipient of novel stem cell trachea 'doing well'

July 26th, 2012 11:21 pm

Thirteen-year-old Ciaran Finn-Lynch, the teenager who made medical history in 2010 by having his throat rebuilt with his own stem cells, is making a successful recovery according to his doctors, BBC News reported.

Finn-Lynch, from Castleblayney in North Ireland,was hailed as the first child to undergo the novel tracheal transplant.

Born with a condition known as long-segment tracheal stenosis when more than two-thirds of the tracheas cartilage are misshapen and do not grow Finn-Lynch had a very difficult time breathing. He underwent the surgery in a desperate attempt to save his life, BBC News said.

Since undergoing the operation at Londons Great Ormond Street Hospital, Finn-Lynch has grown more than four inches and has returned to school, according to his doctors. Since the stem cells used to build the trachea were his own, he is able to live a normal life without having to take medication to prevent rejection of his transplant.

A follow-up report in the Lancet detailed the procedure and explained how the new organ had strengthened over the years.

The original procedure involved seeding stem cells taken from Finn-Lynchs bone marrow into a collagen skeleton of windpipe from a donor, BBC news reported. These stem cells formed a brand new trachea that was then implanted into his body, allowing its cells to grow and mature naturally.

Click for more from BBC News.

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Stunning Recovery for First Child to Get Stem Cell Trachea

July 26th, 2012 11:21 pm

The first child in history to receive a trachea fashioned by his own stem cells has shown remarkable progress since the initial transplant two years ago, marking a new record for the novel procedure.

Ciaran Finn-Lynch, the now 13-year-old boy from the UK who the world's first child to receive the stem cell trachea transplant, is breathing normally and no longer needs anti-rejection medication, researchers reported in a paper published Wednesday in the journal Lancet.

The organ itself is strong, has not shown signs of rejection, and has even grown 11 centimeters since it had been transplanted, according to the researchers.

Ciaran was born with a rare condition known as Long Segment Tracheal Stenosis, marked by a small windpipe that does not grow and can restrict breathing. He underwent the stem cell transplant in March 2010 after a standard trachea transplant did not work.

Researchers at the Great Ormond Street Hospital for Children, the Karolinska Institute in Stockholm and the University College London, stripped cells from a donor trachea and then used Ciaran's own bone marrow stem cells to rebuild the airways in the body. They also infused growth proteins to generate the tissue lining.

Great Ormond Street Hospital Children's Charity

Using a patient's own stem cells not only could help to rebuild the fragile tissue, but also potentially could bypass the risk of having the organ rejected. A trachea is considered a difficult tissue to grow and transplant since it has a limited blood supply, according to Dr. Bill Putnam, professor and chair of the department of thoracic surgery at Vanderbilt University Medical Center, who was not involved in the research.

"I don't think there's anything standard about a tracheal transplant," said Putnam. "The fact that this single patient has survived for two years is worthy of notice."

Once the trachea was transplanted, the researchers continued to infuse growth proteins into the organ to continue stem cell generation. This technique allowed for researchers to transplant the organ faster instead of having to wait for the organ to grow outside of the body.

"Because the protocol used in this study was devised in an emergency, we applied empirically a new combination of technologies on the basis of previous clinical successes in non-airway settings," the researchers wrote, citing bioengineering techniques previously used to regenerate bone, nerves, and skin.

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SDSU researchers use stem cells to repair damaged heart tissue

July 26th, 2012 11:21 pm

SAN DIEGO (CNS) - Researchers from San Diego State University's Heart Institute rejuvenated damaged cardiac tissue removed from older heart-failure patients, using modified stem cells, the university announced Thursday.

University officials said the research could eventually lead to new treatments for heart-failure patients.

"Since patients with heart failure are normally elderly, their cardiac stem cells aren't very healthy," said Sadia Mohsin, a post-doctoral research scholar and one of the study's authors. "We modified these biopsied stem cells and made them healthier. It's like turning back the clock so these cells can thrive again."

Researchers used stem cells modified with a protein called PIM-1 to increase the activity of the enzyme telomerase, which can lengthen telomeres.

Telomeres --- DNA sequences on the ends of chromosomes -- keep the chromosomes from losing DNA base pairs during cellular replication but lose base pairs themselves during the process. If telomeres become too short, the chromosome can't replicate.

According to Moshin, modifying aged cardiac cells added to the cells' ability to regenerate damaged heart muscle.

"This is an especially exciting finding for heart failure patients," Moshin said in a statement. "Right now we can only offer medication, heart transplantation or stem cell therapies with modest regenerative potential. But PIM-1 modification offers a significant advance for clinical treatment."

While the research involved human cells, the work was limited to the laboratory.

"Researchers have tested the technique in mice and pigs and found that telomere lengthening leads to new heart tissue growth in just four weeks," according to a university statement.

The study, supported by the National Institutes of Health, was presented this week at the American Heart Association's Basic Cardiovascular Sciences 2012 Scientific Sessions and published in the Journal of the American College of Cardiology.

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CUR – Hope In Neurodegenerative Diseases

July 26th, 2012 11:21 pm

By Jason Napodano, CFA

Last month we published a NOTE outlining the pioneering efforts of Neuralstem (NYSE MKT:CUR) in the use ofhuman neural stem cells ("hNSC") for the treatment of central nervous system diseases and neurodegenerative disorders.Neuralstems lead development program is for Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrigs disease, named after the famous New York Yankee first baseman who was diagnosed with the disease in 1939, and passed in 1941 at the age of only 37.

The hippocampus is critically important to the control of memory and is severely impacted by the pathology of AD.Specifically, hippocampal synaptic density is reduced in AD and correlates with memory loss.Cam/Tet-DT mice mimic the substantial loss of hippocampal neurons that occur in advanced AD. StemCells Inc's data shows that one month after transplantation, HcCNS-SC engraft, migrate locally, and have begun to differentiate into neuronal and glial lineages in both models.

This resulted in observed increased synaptic density and improved memory post transplantation. Importantly, these results did not require reduction in beta amyloid or tau that accumulate in the brains of patients with AD and account for the pathological hallmarks of the disease, suggesting a new mechanism of action for the treatment of AD.

We think the data above presented by StemCells Inc. is interesting, and bodes well for Neuralstem's similar efforts focusing on hippocampal atrophy inneurodegenerativediseases. The different between StemCells Inc. and Neuralstem is that management at Neuralstem is attempting to recreate these highly encouraging results, only with a small molecule, NS-189, that the company discovered while testing preclinical candidates onstable neural stem cells lines derived from the human hippocampus.

A new hypothesis on major depressive disorder, implicates brain physiology ratherthan brain chemistry alone on disease progression. For example, research shows that depressed patients havereduced hippocampal volume. Accordingly, shrunken hippocampal volume observed in depressed patients could beattributable to a reduction in normal new neuronal generation and/or atrophying hippocampal neural stem cells.

The trial, which is designed to evaluate the safety and preliminary efficacy of BrainStorm's proprietary NurOwn cell therapy (bone marrow-derived, autologous, differentiated mesenchymal stromal cells) is being conducted at the Hadassah Medical Center in Jerusalem, Israel. The company submitted the positive interim safety report to the Israeli Ministry of Health. NurOwn has been granted Orphan-Drug designation by the U.S. FDA.

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CUR - Hope In Neurodegenerative Diseases

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Fat stem cells from liposuction used to form functioning blood vessels

July 26th, 2012 11:21 pm

Liposuction may yield more than just a leaner figure it can potentially produce stem cells for tissue reconstruction.

Researchers from the University of Oklahoma, in Norman, Okla., have successfully extracted adult stem cells during liposuction and used them to generate healthy blood vessels.

These newly formed blood vessels can be used in heart bypass surgery and other complicated procedures requiring healthy vessels, according to the researchers, who presented their findings at the American Heart Associations Basic Cardiovascular Sciences 2012 Scientific Sessions.

While stem cells are typically derived from other sources in the body, the researchers said liposuction-derived stem cells could be useful for an elderly demographic.

For doing coronary artery bypass graft surgery, people who get that are typically elderly, frequently diabetic and usually pretty sick, Matthias Nollert, associate professor at the University of Oklahoma School of Chemical, Biological and Materials Engineering and the studys lead author, told FoxNews.com. The more typical way for getting stem cells from adults for transplantation is to extract cells from the bone marrow.

However, you cant extract bone marrow very easily, Nollert explained. Its a very invasive procedure and patients dont tolerate it well, so we were looking for alternate source of adult stem cells for older, sicker patients.

Extracting adipose-derived stem cells or stem cells derived from fat tissue would be less invasive and also gets rid of unnecessary body fat in the process. According to Nollert, creating tissues from fat stem cells is a fairly new science, having only been experimented with in the past decade. Nollert and his team are the first to create a vascular graft out of fat stem cells with muscle cells making up the blood vessels wall.

To create the vascular graft, the researchers turned the stem cells into smooth muscle cells in the lab and seeded them onto a thin collagen membrane. They then rolled them into tubes with the same diameter as small blood vessels, and three to four weeks later, usable blood vessels were formed.

According to Nollert, utilizing liposuction-derived blood vessels could eliminate complications surrounding heart bypass operations when a healthy blood vessel is necessary for the procedure.

In normal cases, [doctors] would take a vein from your leg or arm to use as a bypass around the blockage, Nollert said. Well it turns out that of allthe people who are considered candidates for bypass, a third of them would like to do a bypass graft, but they have lousy vessels. So theyll do a different procedure that will last only four to five years, and then theyll be back here with same problems.

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ACT Issued Broad Patent for Human RPE Cells Derived From All Types of Pluripotent Stem Cells

July 26th, 2012 11:21 pm

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

Advanced Cell Technology, Inc. (ACT; OTCBB: ACTC), a leader in the field of regenerative medicine, announced today that it has been issued a patent in Australia, patent number 2005325753, Improved modalities for the treatment of degenerative diseases of the retina. The patent broadly covers the use of human retinal pigment epithelial (RPE) cells generated from pluripotent stem cells in the manufacture of pharmaceutical preparations of RPE cells, and the use of those preparations to treat patients with degenerative diseases of the retina such as Age-related Macular Degeneration. The patent covers the pharmaceutical formulation of human RPE cells made from a range of pluripotent stem cells, including both human embryonic stem cells (hESCs) and human induced pluripotent stem (iPS) cells.

We continue to make great progress with our patent estate covering RPE therapies, said Gary Rabin, chairman and CEO of ACT. Our ongoing success in securing broad patent protection around the world, including this newly-issued Australian patent, is a testament to our innovative chief scientific officer, Dr. Robert Lanza, and the rest of our scientific team.

The efficient production of highly pure RPE cell preparations represents a critical step in the creation of renewable sources of transplantable cells that can be used to target degenerative diseases of the eye such as Stargardts Macular Dystrophy (SMD) and dry Age-related Macular Degeneration (dry AMD).

Our current embryonic stem cell trials pave the way for other pluripotent stem cell therapies, commented Dr. Lanza. ACTs cellular reprogramming technologies using iPS cells are in an advanced stage of development, and we hope to be in a position to move toward clinical translation in the not-too-distant future. Since iPS cells can be made from the patients own cells such as skin or blood cells they may allow us to expand our cell therapies beyond immune-privileged sites such as the eye without the risk of immune rejection.

Mr. Rabin concluded, We are aggressively pursuing patent protection for a variety of aspects of our programs. Our intellectual property strategy includes both vigilance in pursuing comprehensive coverage from our initial patent filings, such as this new Australian patent, and filing for protection around our scientific teams various innovations. At the same time we are paying close attention to including within our patent coverage those ways others may wish to adapt our technology for commercial use, such as through the choice of stem cell source, or the use of solid supports or cell suspensions for delivery. Following this strategy, we are establishing both formidable barriers-to-entry for potential competitors, as well as strong potential licensing opportunities for others, translating into solid revenue generation possibilities for the company.

About Advanced Cell Technology, Inc.

Advanced Cell Technology, Inc., is a biotechnology company applying cellular technology in the field of regenerative medicine. For more information, visit http://www.advancedcell.com.

Forward-Looking Statements

Statements in this news release regarding future financial and operating results, future growth in research and development programs, potential applications of our technology, opportunities for the company and any other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words will, believes, plans, anticipates, expects, estimates, and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements, including: limited operating history, need for future capital, risks inherent in the development and commercialization of potential products, protection of our intellectual property, and economic conditions generally. Additional information on potential factors that could affect our results and other risks and uncertainties are detailed from time to time in the companys periodic reports, including the report on Form 10-K for the year ended December 31, 2011. Forward-looking statements are based on the beliefs, opinions, and expectations of the companys management at the time they are made, and the company does not assume any obligation to update its forward-looking statements if those beliefs, opinions, expectations, or other circumstances should change. Forward-looking statements are based on the beliefs, opinions, and expectations of the companys management at the time they are made, and the company does not assume any obligation to update its forward-looking statements if those beliefs, opinions, expectations, or other circumstances should change. There can be no assurance that the Companys clinical trials will be successful.

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Replacing Diseased Immune System With A Healthy One To Cure Chrohn's Disease

July 26th, 2012 11:20 pm

Editor's Choice Main Category: Crohn's / IBD Article Date: 26 Jul 2012 - 9:00 PDT

Current ratings for: Replacing Diseased Immune System With A Healthy One To Cure Chrohn's Disease

3 (1 votes)

Crohn's disease is a chronic inflammatory condition of the gastrointestinal tract with symptoms of pain, fever, diarrhea and weight loss, which usually occurs in adolescents and young adults, but which can also occur during early childhood and older age. The Crohn's and Colitis Foundation of America estimates that more than 700,000 Americans are affected by the disease, although incident rates vary in different parts of the world, with incidence rates of 4 to 9 people per 100,000 in North America. 10% of Crohn's disease sufferers are affected by the most severe form for which there is no completely effective treatment.

Researchers have made substantial progress in the medical treatment of Crohn's disease over the past decade and a half, although even with the best immunosuppressive therapy, fewer than 50% of patients with a moderate to severe form of the disease achieve long-term relief. When Crohn's patients cease taking their medicines, their intestinal inflammation returns and patients who took prolonged courses of medicines that suppress the immune system were noted to have some severe infections.

The initial aim of the Crohn's Allogeneic Transplant Study (CATS) is to treat a small sample of patients with treatment-resistant Crohn's disease by transplanting matched bone marrow cells from a sibling or unrelated donor, which replaces a diseased or abnormal immune system with a healthy one.

The researchers hypothesized that an exchange of the immune system may be successful based on evidence that Crohn's is linked to abnormal immune responses to intestinal bacteria and to a loss of immune tolerance. CATS leading investigator George McDonald, M.D., a transplant researcher and gastroenterologist in the Hutchinson Center's Clinical Research Division says that there is solid evidence that genetic abnormalities in the immune regulatory system are related to Crohn's disease.

Even though the CATS clinical trial is a new direction for bone marrow transplantation, the procedure has already been used by Hutchinson Center researchers, who pioneered bone marrow and hematopoietic cell transplantation to treat blood cancers, and who have used allogeneic transplants to cure patients suffering from both leukemia and Crohn's disease with the result that the signs and symptoms of Crohn's disease subsequently disappeared. German studies have reported similar experiences.

Researchers have previously used autologous stem cell transplants in Crohn's disease patients, whereby the patient's own hematopoietic cells are removed and returned after high-dose chemotherapy to suppress the immune system. However, the benefits have only been partially permanent, which may be because the risk genes for Crohn's are still in the patient's body.

McDonald said:

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Should high-dose interleukin-2 continue to be the treatment of choice for metastatic melanoma?

July 26th, 2012 11:19 pm

Public release date: 26-Jul-2012 [ | E-mail | Share ]

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

New Rochelle, NY, July 26, 2012 Administering high-doses of interleukin-2 (IL-2) has been the preferred treatment for patients with stage IV metastatic melanoma. An article published in the current issue of Cancer Biotherapy and Radiopharmaceuticals, a peer-reviewed journal from Mary Ann Liebert, Inc. (http://www.liebertpub.com), explores whether or not this regimen is still the most effective. The article is available free online at the Cancer Biotherapy and Radiopharmaceuticals website (http://www.liebertpub.com/cbr).

In the article "Should High-Dose Interleukin-2 Still Be the Preferred Treatment for Patients with Metastatic Melanoma?" (http://online.liebertpub.com/doi/full/10.1089/cbr.2012.1220) Robert Dillman and colleagues at the Hoag Institute for Research and Education and Hoag Family Cancer Institute, Newport Beach, CA concluded that until long-term survival data for some of the newer drugs are available, patients with stage IV metastatic melanoma who are well enough to be given intensive IL-2 therapy should receive it initially, either alone or in combination with one of the newer therapeutic agents.

"This is an important article that puts into perspective the reasons why IL-2 should continue to be the initial therapy in patients with metastatic melanoma," says Editor Donald J. Buchsbaum, PhD, Division of Radiation Biology, Department of Radiation Oncology, University of Alabama at Birmingham.

###

About the Journal

Cancer Biotherapy and Radiopharmaceuticals (http://www.liebertpub.com/cbr), published 10 times a year in print and online, is under the editorial leadership of Editors Donald J. Buchsbaum, PhD and Robert K. Oldham, MD, Lower Keys Cancer Center, Key West, FL. Cancer Biotherapy and Radiopharmaceuticals is the only journal with a specific focus on cancer biotherapy, including monoclonal antibodies, cytokine therapy, cancer gene therapy, cell-based therapies, and other forms of immunotherapy. The Journal includes extensive reporting on advancements in radioimmunotherapy and the use of radiopharmaceuticals and radiolabeled peptides for the development of new cancer treatments. Topics include antibody drug conjugates, fusion toxins and immunotoxins, nanoparticle therapy, vascular therapy, and inhibitors of proliferation signaling pathways.

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 Journal of Interferon & Cytokine Research, Human Gene Therapy and Human Gene Therapy Methods, and Stem Cells and Development. 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 Mary Ann Liebert, Inc. (http://www.liebertpub.com)

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