Category Archives: Cell Therapy

--Study lends support to safe use for therapy

Newswise A team of researchers from Johns Hopkins University and the National Human Genome Research Institute has evaluated the whole genomic sequence of stem cells derived from human bone marrow cellsso-called induced pluripotent stem (iPS) cellsand found that relatively few genetic changes occur during stem cell conversion by an improved method. The findings, reported in the March issue of Cell Stem Cell, the official journal of the International Society for Stem Cell Research (ISSCR), will be presented at the annual ISSCR meeting in June.

Our results show that human iPS cells accrue genetic changes at about the same rate as any replicating cells, which we dont feel is a cause for concern, says Linzhao Cheng, Ph.D., a professor of medicine and oncology, and a member of the Johns Hopkins Institute for Cell Engineering.

Each time a cell divides, it has the chance to make errors and incorporate new genetic changes in its DNA, Cheng explains. Some genetic changes can be harmless, but others can lead to changes in cell behavior that may lead to disease and, in the worst case, to cancer.

In the new study, the researchers showed that iPS cells derived from adult bone marrow cells contain random genetic changes that do not specifically predispose the cells to form cancer.

Little research was done previously to determine the number of DNA changes in stem cells, but because whole genome sequencing is getting faster and cheaper, we can now more easily assess the genetic stability of these cells derived by various methods and from different tissues, Cheng says. Last year, a study published in Nature suggested higher than expected cancer gene mutation rates in iPS cells created from skin samples, which, according to Cheng, raised great concerns to many in the field pertaining to usefulness and safety of the cells. This study analyzed both viral and the improved, nonviral methods to turn on stem cell genes making the iPS cells

To more thoroughly evaluate the number of genetic changes in iPS cells created by the improved, non-viral method, Chengs team first converted human blood-forming cells or their support cells, so-called marrow stromal cells (MSCs) in adult bone marrow into iPS cells by turning on specific genes and giving them special nutrients. The researchers isolated DNA from--and sequenced--the genome of each type of iPS cells, in comparison with the original cells from which the iPS cells were derived.

Cheng says they then counted the number of small DNA differences in each cell line compared to the original bone marrow cells. A range of 1,000 to 1,800 changes in the nucleic acid letters A, C, T and G occurred across each genome, but only a few changes were found in actual genes--DNA sequences that act as blueprints for our bodys proteins. Such genes make up two percent of the genome.

The blood-derived iPS cells contained six and the MSC-derived iPS cells contained 12 DNA letter changes in genes, which led the researchers to conclude that DNA changes in iPS cells are far more likely to occur in the spaces between genes, not in the genes themselves.

Next, the investigators examined the severity of the DNA changes--how likely each one would disrupt the function of each gene. They found that about half of the DNA changes were silent, meaning these altered blueprints wouldnt change the nucleic acid building code for its corresponding protein or change its function.

Originally posted here:
Improved Adult-Derived Human Stem Cells Have Fewer Genetic Changes Than Expected

Newswise HOUSTON (April 16, 2012) A researcher at The University of Texas Health Science Center at Houston (UTHealth) is studying a novel cell therapy that could help avoid autoimmune problems after stem cell transplantation, as well as potentially treat other autoimmune diseases.

The preclinical study, funded with a $1.9 million grant from the National Institutes of Health (NIH) National Heart, Lung, and Blood Institute (NHLBI), is being conducted in collaboration with the NIH and The University of Texas MD Anderson Cancer Center.

The therapy centers on regulatory T cells, which are central to the control of autoimmunity in the body. Dat Tran, M.D., assistant professor in the Pediatric Research Center at the UTHealth Medical School, said the therapy could help prevent graft-versus-host disease (GVHD), an autoimmune disorder that occurs in up to 80 percent of cancer patients receiving bone marrow stem cell transplants. It could also potentially treat other autoimmune diseases such as type 1 diabetes.

Leukemia and lymphoma are blood cell cancers, immune system cancers. You have to wipe out the whole immune system and then give the person a new system through a stem cell transplant, said Tran, who holds a dual appointment in the UTHealth Department of Pediatrics Division of Allergy, Immunology and Rheumatology. But the immune system is designed to detect foreign particles and attack them. Depending on how close the match is, you can have a reaction between the graft donor and the host patient that results in an autoimmunity problem.

According to the National Institutes of Health, GVHD occurs in 30 to 40 percent of recipients using related donors and 60 to 80 percent in recipients using unrelated donors. Acute symptoms include abdominal pain, diarrhea, fever, jaundice, skin rash, vomiting and weight loss. Chronic symptoms include dry eyes and mouth, hair loss, hepatitis, lung and digestive tract disorders and skin thickening. Some cases can lead to death.

The most effective treatments are high-dose corticosteroids, which often have severe side effects. Thats not good enough, Tran said.

While working at the National Institutes of Health as an allergy and immunology fellow, Tran began to research ways to enhance the regulatory T cells in the body.

Regulatory T cells are extremely important because if you dont have them, your body will develop autoimmunity and attack itself, he said. We think perhaps the regulatory T cells dont develop fast enough in stem cell transplants. So I thought, Why dont we put the regulatory T cells in the body before the transplant to enhance transplantation and avoid autoimmunity?

The challenge, he said, was finding a way to grow the T cells in large enough quantities and separate out the best ones to achieve a more potent population. He found a marker they could use to isolate the good regulatory T cells and published a proof-of-theory paper in the May 2009 issue of the American Hematology Society journal Blood.

Were now testing the cells in mice to see if they are stable and potent and that they will work, he said. In the next five years, in collaboration with MD Anderson, we hope we can prevent GVHD.

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UTHealth Uses $1.9 Million Grant to Study Therapy for Autoimmunity

Hyderabad, April 8:

The Indian stem cell industry is in a nascent stage. There is potential for fast growth and global players will enter the market through mergers and acquisitions, says a study by YES Bank and the Federation of Asian Biotech Associations (FABA).

At present, the top five players in the Indian private sector space in stem cells are Reliance Life Sciences, Life Cell, Cryobanks India, Stempeutics and Trans-Scell Biologics. Most of the Indian companies are focussed on stem cell banking and research, the study released recently stated.

With the benefits of stem cell therapy growing, it is attracting several organisations and industry to invest, create and commercialise promising technologies. The global stem cell therapy market was pegged at $21 billion in 2010. It is projected to grow to $60 billion by 2015, the study pointed out.

Stem cells are unique as they have the ability to divide indefinitely and give rise to specialised types. Under specific conditions they can be induced to become tissue or organ specific cells with special functions. The main sources of these cells are embryos and adult tissues.

When it comes to treatment, it serves as an internal repair mechanism, dividing to replenish other cells. These characteristics of the stem cells are helping scientists to even tackle difficult diseases such as Alzheimer's or Parkinson's.

The market for stem cells will include specific products, services and technologies to aid in diagnosis to cure.

Though, the report did not give the size of the Indian market, it forecasts an annual growth rate of around 15 per cent, mainly driven by investments from the Government and private players.

The Indian Government has been quite proactive in promoting work in stem cells, especially in clinical trials, basic research and creating applications. The Department of Biotechnology has invested $60 million into stem cell research during 2005-2010.

The National Centre for Biological Sciences, Bangalore, is a major player. Similarly, the Hyderabad-based Centre for Cellular and Molecular Biology has set up a clinical research facility for stem cell technologies with an investment of Rs 24 crore last year. Other important centres for stem cell work are National Centre for Cell Sciences at Pune, LV Prasad Eye Institute, Hyderabad, Centre for Stem Cell Research at CMC, Vellore, National Brain Research Centre, New Delhi and so on, the report identified.

The rest is here:
Stem cells market all set to grow: Study

Public release date: 9-Apr-2012 [ | E-mail | Share ]

Contact: Jill Scoggins jill.scoggins@louisville.edu 502-852-7461 University of Louisville

LOUISVILLE, Ky. To paraphrase Yogi Berra: It's dj vu all over again with a twist.

A University of Louisville researcher known for his prowess at winning competitive grants from the National Institutes of Health has won another one his first for clinical research.

During his 18-year tenure at UofL, Dr. Roberto Bolli has generated more than $100 million in grants for basic research from the NIH. Today, Bolli joined with University of Louisville President James R. Ramsey to announce a new NIH grant he has won for clinical research, a seven-year, $3.4 million grant from the National Heart, Lung and Blood Institute to establish one of seven regional centers across the United States in the Cardiac Cell Therapy Research Network (CCTRN). The network conducts early clinical trials of adult stem cell therapies in patients with heart disease.

"Stem cell therapy holds great promise for treating heart disease, and researchers involved in CCTRN are helping determine how these promising therapies might be most beneficial to patients," said Dr. Sonia Skarlatos, deputy director of the Division of Cardiovascular Sciences in the NIH's National Heart, Lung, and Blood Institute. "This new round of funding is an important step in helping to improve cardiovascular health."

This move from basic to clinical research from "bench to bedside" in medical lingo will test the validity of new therapies by replicating studies in patients at two or more of the network's centers located at UofL, Stanford University, Texas Heart Institute, Minneapolis Heart Institute, University of Florida, University of Miami and Indiana University.

Replicating studies in several locations with a large number of patients is necessary for researchers to ultimately determine which ones can be submitted to the Food and Drug Administration for approval.

"Through the work of Dr. Bolli and his team, the UofL Health Sciences Center continues to fulfill the promise of a great metropolitan research university," Ramsey said. "Success like Dr. Bolli's in conducting basic research lays the foundation for him to conduct clinical studies that will determine the standard of care for the future.

"Clinical trials of new adult stem cell therapies are among the most promising and exciting areas of medical research today, and being part of a national network such as the CCTRN means UofL can bring this cutting-edge medicine to the people of Kentucky and beyond."

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From bench to bedside: NIH grant establishes cardiac clinical research center at UofL

Texas' proposed adult stem-cell regulations, up for approval this week, are under fire for circumventing the Food and Drug Administration and making the experimental therapy commercially available before it's been proven safe and effective.

The criticism of the Texas Medical Board draft policy, developed in the aftermath of Gov. Rick Perry receiving stem-cell treatment for his back problems, is coming from a host of pre-eminent scientists and institutions, including the influential scientific journal Nature and the International Society for Stem Cell Research.

"Texas officials should take the FDA's regulatory power over stem cells more seriously," editorialized Nature. "If the medical board were to act according to its stated pledge to protect patients, then it would make clear the need for clinical validation of adult stem cells before use and rescind the medical licenses of any doctors in breach of rules about using unapproved treatments."

Dr. Irving Weissman of the ISSCR called the draft "a clever way around the FDA's appropriate role overseeing clinical trials." Weissman, director of Stanford University's Institute for Stem Cell Biology and Regenerative Medicine, said the policy violates the ISSCR's guidelines for clinical use of adult stem cells.

The policy, thought to be the first of its kind, would allow Texas doctors to provide the unlicensed therapy as long as they have approval from a review panel that assesses the cells' use for patient safety, as is customary in clinical trials. But unlike most clinical trials, in which experimental therapy is provided free, the policy would allow Texas doctors to charge for it, typically tens of thousands of dollars.

Mari Robinson, executive director of the Texas Medical Board, denied that the proposed policy represents an alternative path than the FDA. She said that in the absence of definitive direction from the FDA, the board simply wants to ensure that "new and expanding areas of medicine" are safe for patients.

"The FDA has provided no definitive statement about the medical use of adult stem cells, including whether it even has jurisdiction if the cells are taken from patients and given back within the state," said Robinson. "The board wants to let the field come forward and grow but in a safe manner."

Robinson, a lawyer, said the courts ultimately may provide the answer about the FDA's jurisdiction. The federal agency last year filed an injunction to stop a Colorado clinic from treating patients with their own stem cells, arguing its failure to comply with FDA regulations "puts the public's health at risk."

Adult stem cells are cells in the body that multiply to replenish dying cells. Though they lack the ability of embryonic stem cells to morph into any kind of tissue, they have shown much promise in recent research treating a variety of conditions. Still, the therapy is mostly in early-stage clinical trials and considered five to 10 years away from FDA approval.

Clinics popping up

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Medical board's proposed stem-cell policy under fire

NEW YORK, March 28, 2012 (GLOBE NEWSWIRE) -- NeoStem, Inc. (NYSE Amex:NBS) ("NeoStem" or "the Company"), a leader in the cell therapy industry, today announced that it intends to offer and sell shares of its common stock and warrants in an underwritten public offering. All of the shares and warrants in the offering will be sold by NeoStem. The offering is subject to market conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering. The securities will be issued pursuant to a prospectus supplement filed as part of an effective registration statement on Form S-3 previously filed with the Securities and Exchange Commission (SEC).

Maxim Group LLC is acting as book-runner of the offering.

A shelf registration statement relating to the securities was filed with the SEC, which became effective on June 13, 2011. A preliminary prospectus supplement related to the offering will be filed with the SEC and will be available on the SEC's website at http://www.sec.gov. Copies of the preliminary prospectus supplement and the accompanying prospectus relating to this offering may be obtained, when available, from Maxim Group LLC, 405 Lexington Avenue, New York, NY 10174 or via telephone at (212) 895-3685.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction. NeoStem intends to file a preliminary prospectus supplement relating to the offering with the SEC, which will be available along with the prospectus filed with the SEC in connection with the shelf registration, on the SEC's website at http://www.sec.gov.

About NeoStem, Inc.

NeoStem, Inc. ("we," "NeoStem" or the "Company") continues to develop and build on its core capabilities in cell therapy to capitalize on the paradigm shift that we see occurring in medicine. In particular, we anticipate that cell therapy will have a large role in the fight against chronic disease and in lessening the economic burden that these diseases pose to modern society. Our January 2011 acquisition of Progenitor Cell Therapy, LLC ("PCT") provides NeoStem with a foundation in both manufacturing and regulatory affairs expertise. We believe this expertise, coupled with our existing research capabilities and collaborations, will allow us to achieve our mission of becoming a premier cell therapy company. Our PCT subsidiary's manufacturing base is one of the few current Good Manufacturing Practices ("cGMP") facilities available for contracting in the burgeoning cell therapy industry. Amorcyte, LLC ("Amorcyte"), which we acquired in October 2011, is developing a cell therapy for the treatment of cardiovascular disease. Amorcyte's lead compound, AMR-001, represents NeoStem's most clinically advanced therapeutic and has commenced enrollment for a Phase 2 trial to investigate AMR-001's efficacy in preserving heart function after a heart attack. We also expect to begin a Phase 1 clinical trial by 2013 to investigate AMR-001's utility in arresting the progression of congestive heart failure and the associated comorbidities of that disease. Athelos Corporation ("Athelos"), which is approximately 80%-owned by our subsidiary, PCT, is engaged in collaboration with Becton-Dickinson that is exploring the earlier stage clinical development of a T-cell therapy for autoimmune conditions. In addition, our pre-clinical assets include our VSELTM Technology platform as well as our MSC (mesenchymal stem cells) product candidate for regenerative medicine.

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

Forward-Looking Statements

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 and medical strategy, including with respect to the development of AMR-001 and other cell therapies and its divestiture of its interest in Suzhou Erye Pharmaceutical Co., Ltd. about which no assurance can be given. The Company's actual results could differ materially from those anticipated in these forward- looking statements as a result of various factors. Factors that could cause future results to materially differ from the recent results or those projected in forward-looking statements include the "Risk Factors" described in the Company's Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 19, 2012 and in the Company's periodic filings with the Securities and Exchange Commission. The Company's further development is highly dependent on future medical and research developments and market acceptance, which is outside its control.

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NeoStem Announces Proposed Public Offering of Common Stock and Warrants

28-03-2012 10:17 Dr Joshua Hare is Professor of Medicine and Director of the Interdisciplinary Stem Cell Institute at the University of Miami. The interview was conducted on 25 March 2012 at the American College of Cardiology's (ACC's) 61st Annual Scientific Session & Expo in Chicago. See more ACC.12 Coverage: http://www.getinsidehealth.com

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Stem cell therapy for the repair of myocardium in heart failure patients - Video

ScienceDaily (Mar. 27, 2012) A team of researchers led by scientists at Weill Cornell Medical College has designed what appears to be a powerful gene therapy strategy that can treat both beta-thalassemia disease and sickle cell anemia. They have also developed a test to predict patient response before treatment.

This study's findings, published in PLoS ONE, represents a new approach to treating these related, and serious, red blood cells disorders, say the investigators.

"This gene therapy technique has the potential to cure many patients, especially if we prescreen them to predict their response using just a few of their cells in a test tube," says the study's lead investigator, Dr. Stefano Rivella, Ph.D., an associate professor of genetic medicine at Weill Cornell Medical College. He led a team of 17 researchers in three countries.

Dr. Rivella says this is the first time investigators have been able to correlate the outcome of transferring a healthy beta-globin gene into diseased cells with increased production of normal hemoglobin -- which has long been a barrier to effective treatment of these disease.

So far, only one patient in France has been treated with gene therapy for beta thalassemia, and Dr. Rivella and his colleagues believe the new treatment they developed will be a significant improvement. No known patient has received gene therapy yet to treat sickle cell anemia.

A Fresh Approach to Gene Therapy

Beta-thalassemia is an inherited disease caused by defects in the beta-globin gene. This gene produces an essential part of the hemoglobin protein, which, in the form of red blood cells, carries life-sustaining oxygen throughout the body.

The new gene transfer technique developed by Dr. Rivella and his colleagues ensures that the beta-globin gene that is delivered will be active, and that it will also provide more curative beta-globin protein. "Since the defect in thalassemia is lack of production of beta-globin protein in red blood cells, this is very important," Dr. Rivella says.

The researchers achieved this advance by hooking an "ankyrin insulator" to the beta-globin gene that is carried by a lentivirus vector. During the gene transfer, this vector would be inserted into bone marrow stem cells taken from patients, and then delivered back via a bone marrow transplant. The stem cells would then produce healthy beta-globin protein and hemoglobin.

This ankyrin insulator achieves two goals. First, it protects delivery of the normal beta-globin gene. "In many gene therapy applications, a curative gene is introduced into the cells of patients in an indiscriminate fashion," Dr. Rivella explains. "The gene lands randomly in the genome of the patient, but where it lands is very important because not all regions of the genome are the same." For example, some therapeutic genes may land in an area of the genome that is normally silenced -- meaning the genes in this area are not expressed. "The role of ankyrin insulator is to create an active area in the genome where the new gene can work efficiently no matter where it lands," Dr. Rivella says. He adds that the small insulator used in his vector should eliminate the kind of side effects seen in the French patient treated with beta-thalassemia gene therapy.

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New gene therapy approach developed for red blood cell disorders