Category Archives: Cell Therapy

SATURDAY, March 24 (HealthDay News) -- An innovative approach using patients' own bone marrow cells to treat chronic heart failure came up short in terms of effectiveness, researchers report.

Use of stem cell therapy to repair the slow, steady damage done to heart muscle and improve heart function is safe, but has not been shown to improve most measures of heart function, the study authors said.

"For the measures we paid most attention to, we saw no effect, there is no question about that," said researcher Dr. Lemuel Moye, a professor of biostatistics at the University of Texas School of Public Health in Houston.

"Ultimately, this is going to pay off handsomely for individuals and for public health in general, but it's going to take years of work," Moye said. "We are the vanguard looking for new promising lines of research."

While the hoped-for results didn't materialize, there appeared to be a small improvement in some patients, he said. "When we looked at another commonly used measure of heart function called ejection fraction, or the strength of the heart's pumping, that's where all the action was," Moye noted.

It's hard to know which measures of heart function to look at, Moye explained. "We have had some difficulty with that," he said.

Future research will look at other measures of heart function, pay more attention to the characteristics of the cells that are injected and determine which cells are best, he added.

Cardiac cells and other types of specially prepared cells are available now that were not accessible when this study started in 2009, Moye pointed out.

The results of the trial, which was sponsored by the U.S. National Heart, Lung, and Blood Institute, were to be presented Saturday at the American College of Cardiology's annual meeting in Chicago. The report was also published online March 24 in the Journal of the American Medical Association.

For the study, Moye and colleagues worked with 92 patients, average age 63 and mostly male, who had heart failure with and without chest pain. They were randomly assigned to receive either an injection of 100 million bone marrow cells from their own bone marrow, or an inactive placebo. Patients in both groups also received aggressive medical therapy.

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Stem-Cell Trial Failed to Treat Heart Failure

Public release date: 24-Mar-2012 [ | E-mail | Share ]

Contact: Kristin Wincek kwincek@mhif.org 612-863-0249 Minneapolis Heart Institute Foundation

CHICAGO Cell therapy may present an option for patients with ischemic heart disease to use their own bone marrow cells to repair the damaged areas of their hearts, and may pave the way for future treatment options, according to the FOCUS trial, which will be presented as a late-breaking clinical trial March 24 at the 61st annual American College of Cardiology (ACC) scientific session.

This is the largest study to date to look at stem cell therapy, using a patient's own stem cells, to repair damaged areas of the heart in patients with chronic ischemic heart disease and left ventricular dysfunction. Researchers found that left ventricular ejection fraction (the percentage of blood leaving the heart's main pumping chamber) increased by a small but significant amount (2.7 percent) in patients who received stem cell therapy. The study also revealed that the improvement in ejection fraction correlated with the number of progenitor cells (CD34+ and CD133+) in the bone marrow; and this information will help in evaluating and designing future therapies and trials.

"FOCUS is an incredibly important trial, as it has informed the cell therapy community how to better treat this high-risk patient population, and allows us to enter into an exciting, next generation of stem cell therapy armed with more data," said study investigator Timothy D. Henry, MD, an interventional cardiologist at the Minneapolis Heart Institute (MHI) at Abbott Northwestern Hospital in Minneapolis and director of research with the Minneapolis Heart Institute Foundation.

This multicenter study was conducted by the Cardiovascular Cell Therapy Research Network (CCTRN), which is supported through a research grant from the National Institutes of Health's National, Heart, Lung and Blood Institute (NHLBI), with the goal to evaluate novel stem cell-based treatment strategies for individuals with cardiovascular disease.

FOCUS will be presented at ACC.12 by its lead investigator Emerson C. Perin, MD, PhD, director of clinical research for cardiovascular medicine at the Texas Heart Institute, one of the five sites in the CCTRN. The Minneapolis Heart Institute is another site of the five in the network, and a large number of CCTRN patients were enrolled in Minnesota.

For this study, which took place between April 2009 and April 2011, the five sites randomly selected 92 patients to receive stem cell treatment or placebo. The symptomatic patients, with an average age 63, all had chronic ischemic heart disease and an ejection fraction of less than 45 percent (baseline 34 percent) along with heart failure and/or angina and were no longer candidates for revascularization. "These patients had no other options, as medical management failed to improve their symptoms," explained the study's co-investigator Jay Traverse, MD, an interventionalist cardiologist at the Minneapolis Heart Institute at Abbott Northwestern Hospital and physician researcher with the Minneapolis Heart Institute Foundation.

Bone marrow was aspirated from the patients and processed to obtain just the mononuclear fraction of the marrow. In patients randomly selected to receive stem cell therapy, physicians inserted a catheter into the heart's left ventricle to inject 100 million stem cells in more than 15 sites that showed damage on the electromechanical mapping image of the heart.

"Studies such as these are able to be completed much faster because of the team approach of the network" said Sonia I. Skarlatos, PhD, NHBLI's deputy director of the division of cardiovascular sciences and program director of CCTRN.

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Cell therapy using patient's own bone marrow may present option for heart disease

SOUTH SAN FRANCISCO, CA--(Marketwire -03/21/12)- VistaGen Therapeutics, Inc. (OTC.BB: VSTA.OB - News) (OTCQB: VSTA.OB - News), a biotechnology company applying stem cell technology for drug rescue and cell therapy, and Vala Sciences, Inc., a biotechnology company developing and selling next-generation cell image-based instruments, reagents and analysis software tools, have entered into a strategic collaboration. Their goal is to advance drug safety screening methodologies in the most clinically relevant human in vitro bioassay systems available to researchers today.

Cardiomyocytes are the muscle cells of the heart that provide the force necessary to pump blood throughout the body, and as such are the targets of most of the drug toxicities that directly affect the heart. Many of these drug toxicities result in either arrhythmia (irregular, often fatal, beating of the heart) or reduced ability of the heart to pump the blood necessary to maintain normal health and vigor.

"Our collaboration with Vala directly supports the core drug rescue applications of our Human Clinical Trials in a Test Tube platform," said Shawn K. Singh, JD, VistaGen's Chief Executive Officer. "Our high quality human cardiomyocytes combined with Vala's high throughput electrophysiological assessment capabilities is yet another example of how we are applying our stem cell technology platform within a strategic ecosystem of complementary leading-edge companies and technologies. We seek to drive our drug rescue programs forward and generate a pipeline of new, cardiosafe drug candidates."

Through the collaboration, Vala will use its Kinetic Image Cytometer platform to demonstrate both the suitability and utility of VistaGen's human pluripotent stem cell derived-cardiomyocytes for screening new drug candidates for potential cardiotoxicity over conventional in vitro screening systems and animal models. VistaGen's validated human cardiomyocyte-based bioassay system, CardioSafe 3D, will permit Vala to demonstrate the quality, resolution, applicability and ease of use of its new instrumentation and analysis software to make information-rich, high throughput measurements and generate fundamentally new insights into heart cell drug responses. Accurate, sensitive and reproducible measurement of electrophysiological responses of stem cell-derived cardiomyocytes to new drug candidates is a key element of VistaGen's CardioSafe 3D drug rescue programs. VistaGen's strategic collaboration with Vala is directed towards this goal.

About VistaGen Therapeutics

VistaGen is a biotechnology company applying human pluripotent stem cell technology for drug rescue and cell therapy. VistaGen's drug rescue activities combine its human pluripotent stem cell technology platform, Human Clinical Trials in a Test Tube, with modern medicinal chemistry to generate new chemical variants (Drug Rescue Variants) of once-promising small-molecule drug candidates. These are drug candidates discontinued due to heart toxicity after substantial development by pharmaceutical companies, the U.S. National Institutes of Health (NIH) or university laboratories. VistaGen uses its pluripotent stem cell technology to generate early indications, or predictions, of how humans will ultimately respond to new drug candidates before they are ever tested in humans, bringing human biology to the front end of the drug development process.

Additionally, VistaGen's small molecule drug candidate, AV-101, is in Phase 1b development for treatment of neuropathic pain. Neuropathic pain, a serious and chronic condition causing pain after an injury or disease of the peripheral or central nervous system, affects approximately 1.8 million people in the U.S. alone. VistaGen is also exploring opportunities to leverage its current Phase 1 clinical program to enable additional Phase 2 clinical studies of AV-101 for epilepsy, Parkinson's disease and depression. To date, VistaGen has been awarded over $8.5 million from the NIH for development of AV-101.

About Vala Sciences

Vala Sciences is a San Diego-based biotechnology company that develops and sells cell-image-based instrumentation, reagents and analysis software tools to academic, pharmaceutical and biotechnology scientists. Vala's IC 200 class of instrumentation, and CyteSeer Automated Image Cytometry software convert labor-intensive qualitative observations of biological changes that can take from days to months, into accurate measurements delivered automatically in minutes.

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VistaGen Therapeutics Enters Strategic Drug Screening Collaboration With Vala Sciences

NEW YORK, March 20, 2012 (GLOBE NEWSWIRE) -- NeoStem, Inc. (NYSE Amex:NBS) ("NeoStem" or "the Company") is a leader in the cell therapy industry, developing cell based therapeutics supported by the Company's expertise in contract manufacturing. This strategic combination and depth of experience in cell therapy development and manufacturing provide NeoStem with unique capabilities to develop its own cell therapies and that sets the Company apart from others in the cell therapy landscape. 2011 represented a major year of strategic transition for NeoStem, and the Company plans to build upon that in 2012 and in the years ahead.

NeoStem reported its audited results for 2011. Consolidated revenues for the year ended December 31, 2011 were $73.7 million compared to $69.8 million for 2010. The Company's consolidated net loss for 2011 was $56.6 million, which included $10.3 million of non-cash equity-based compensation expense, $19.4 million of goodwill impairment charges and $9.0 million of depreciation and amortization. Overall, the Company's consolidated cash loss for 2011 was $15.5 million (see reconciliation below). Net loss attributable to NeoStem common shareholder interests for 2011 was $47.8 million, or $0.54 per share.

As of December 31, 2011, the Company had consolidated cash and cash equivalents of $12.7 million, and an additional $2.5 million in cash held in escrow (classified in Other Assets).

NeoStem believes that the opportunities that exist today in cell therapy are robust and growing despite a persistently difficult financial environment, making this an opportunistic time to pursue the monetization of the Company's 51% ownership of Suzhou Erye Pharmaceutical Co., Ltd. and bolster its cell therapy business. In June 2011, the Company engaged a financial advisor to lead the effort to pursue the possible divestiture of the Company's interest in Erye. Marketing efforts are underway and have generated interest from both financial and strategic buyers.

On the therapeutics side of the business NeoStem now has a pipeline of assets that includes Amorcyte (Phase 2 trial for preservation of heart function after a heart attack), Athelos (physician sponsored Phase 1 trials for a range of auto-immune conditions) and pre-clinical development work on its VSEL(TM) technology. The Company's most advanced asset is AMR-001 for the treatment of acute myocardial infarction for which enrollment for a Phase 2 study in the United States commenced in January. The study is a multicenter, randomized, double-blind, placebo-controlled clinical trial to evaluate the safety and efficacy of infarct-related artery infusion of AMR-001, an autologous bone marrow derived cell therapy enriched for CD34+ cells. AMR-001 is administered 5 to 11 days post-stent placement in patients diagnosed with an ST segment elevation myocardial infarction ("STEMI") with ejection fraction less than or equal to 48%. The study will include 160 subjects, age 18 and older, randomized 1:1 between treatment and control. The manufacturing, product supply, and logistics for the trial will be supported by Progenitor Cell Therapy, LLC, NeoStem's contract manufacturing company.

Amorcyte currently has ten activated clinical trial sites for its Phase 2 AMI clinical trial with the initial patients enrolled. Trial enrollment is expected to be completed in approximately one year with data read out six months following the last treated patient. The Amorcyte franchise is supported by a strong patent portfolio which includes both composition of matter and methods of treatment around use of these hematopoietic stem cells for treatment of cardiac ischemia and other ischemic tissue that result from vascular insufficiency. The Company sees Amorcyte as a pipeline of therapeutics with potential in multiple indications from STEMI to congestive heart failure and other related vascular insufficiencies. The Amorcyte product addresses both an unmet medical need and a large potential market.

"One of the most important attributes of AMR-001 is that it's 'natural.' We are enhancing the body's normal and natural response to ischemic injury," said Dr. Robin Smith, CEO of NeoStem. "Ample historical evidence, published literature and our own compelling Phase 1 data give us confidence that this product will ultimately make it to the marketplace. Our next most advanced asset is held by Athelos Corporation, (a NeoStem company, partnered with Becton, Dickinson and Company) which is developing a novel T-cell platform for immunological disorders. The Athelos T-cell technology represents an innovative approach to restoring immune balance with potential applications in graft vs. host disease (GvHD), solid organ transplant (SOT) and autoimmune diseases, such as asthma and diabetes. Multiple physician sponsored phase 1 studies are expected to report results that will be used to determine the direction of clinical development.

"NeoStem is also developing pre-clinical assets, including its VSEL(TM) Technology platform for regenerative medicine, which NeoStem believes is an endogenous pluripotent non-embryonic cell that has the potential to change the paradigm of cell therapy as we know it today. These activities have received awards in excess of $2.5 million which funds support the work of prestigious researchers who are pioneering this science with NeoStem.

"Behind the development of these therapeutic assets is the NeoStem cell therapy contract manufacturing business (PCT) which itself continues to grow. New clients have engaged PCT to assist them in the development of their products, including a global, diversified healthcare company who recently selected PCT to provide stem cell processing in our two GMP manufacturing facilities in the United States (California and New Jersey). PCT's prominence in the marketplace continues to grow and that is reflected by both client satisfaction and the revenues the company generates.

"As we look to the year ahead, we are excited on multiple fronts. Our capital preservation efforts are now bearing fruit as our cash burn rate is in-line with our peers. We expect to continue to carefully invest our capital in projects that meet our internal rate of return hurdle and risk parameters. We believe the PCT and Amorcyte acquisitions have created true value for our shareholders and we look forward to demonstrating that as these assets reach their respective value inflection points. We see the unmet medical need in cardiology and the treatment burden associated with chronic diseases as representing a significant challenge to modern society. We believe that cell therapy holds many of the solutions to the health crisis that societies face and have the potential to create real pharmacoeconomic benefit as well as shareholder value for our company.

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NeoStem Provides Updates and Reports Year End Results

OTTAWA A team of Ottawa researchers has been awarded $442,000 to test the worlds first experimental stem-cell therapy aimed at patients who suffer from septic shock, a runaway infection of the bloodstream thats notoriously difficult to treat.

The federal grant will allow researchers from the Ottawa Hospital Research Institute to use mesenchymal stem cells, found in the bone marrow of healthy adults, to treat as many as 15 patients with septic shock.

The deadly infection occurs when toxic bacteria spreads rapidly throughout the body and over-activates the immune system, leading to multiple organ failure and death in up to 40 per cent of cases.

One in five patients admitted to intensive-care units suffers from septic shock, making it the most common illness among a hospitals sickest of the sick.

Existing treatments focus on early diagnosis and intervention before organs start to fail. Patients with septic shock require aggressive resuscitation measures, large doses of intravenous antibiotics and, often, ventilators to help them breathe.

Yet because the infection can creep up on patients rapidly and cause unpredictable complications, death from septic shock remains relatively common.

The experimental therapy aims to use donor stem cells, grown and purified at the Ottawa laboratory, to dial down the bodys hyperactive immune response and reduce the cascade of inflammation that leads to organ failure.

Early results from animal studies even raise the possibility that mesenchymal cells could eliminate the bacteria that causes septic shock, although the impact on humans is not yet known.

Its a unique feature of the stem cells, said Dr. Lauralyn McIntyre, the intensive-care physician who is leading the trial. Certainly no other therapy in the past, other than antibiotics, has impacted the bacterial load in the system.

Like other stem cells, mesenchymal cells can turn into a variety of more specialized cells and tissues that help repair and regenerate damaged organs. And because mesenchymal cells are derived from adults, they sidestep the ethical issues arising from the destruction of human embryos needed to make embryonic stem cells.

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Ottawa researchers receive grant to test stem-cell therapy for septic shock

Public release date: 15-Mar-2012 [ | E-mail | Share ]

Contact: Jennifer Ganton jganton@ohri.ca 613-798-5555 x73325 Ottawa Hospital Research Institute

A team of researchers from the Ottawa Hospital Research Institute (OHRI) and the University of Ottawa (uOttawa) has been awarded $367,000 from the Canadian Institutes of Health Research (CIHR) and $75,000 from the Stem Cell Network to lead the first clinical trial in the world of a stem cell therapy for septic shock. This deadly condition occurs when an infection spreads throughout the body and over-activates the immune system, resulting in severe organ damage and death in 30 to 40 per cent of cases. Septic shock accounts for 20 per cent of all Intensive Care Unit (ICU) admissions in Canada and costs $4 billion annually. Under the leadership of Dr. Lauralyn McIntyre, this new "Phase I" trial will test the experimental therapy in up to 15 patients with septic shock at The Ottawa Hospital's ICU.

The treatment involves mesenchymal stem cells, also called mesenchymal stromal cells or MSCs. Like other stem cells, they can give rise to a variety of more specialized cells and tissues and can help repair and regenerate damaged organs. They also have a unique ability to modify the body's immune response and enhance the clearance of infectious organisms. They can be found in adult bone marrow and other tissues, as well as umbilical cord blood, and they seem to be easily transplantable between people, because they are more able to avoid immune rejection.

There has been a great deal of interest in using MSCs to treat disease, with most research so far focused on heart disease, stroke, inflammatory bowel disease and blood cancers. Hundreds of patients with these diseases have already been treated with MSCs through clinical trials, with results suggesting that these cells are safe in these patients, and have promising signs of effectiveness. MSCs are still considered experimental however, and have not been approved by Health Canada as a standard therapy for any disease.

In recent years, a number of animal studies have suggested that MSCs may also be able to help treat septic shock. For example, a recent study by Dr. Duncan Stewart, CEO and Scientific Director of OHRI (and also a co-investigator on the new clinical trial) showed that treatment with these cells can triple survival in a mouse model of this condition.

"Mesenchymal stem cell therapy appears promising in animal studies, but it will require many years of clinical trials involving hundreds of patients to know if it is safe and effective," said Dr. Lauralyn McIntyre, a Scientist at the OHRI, ICU Physician at The Ottawa Hospital, Assistant Professor of Medicine at uOttawa and a New Investigator with CIHR and Canadian Blood Services. "This trial is a first step, but it is a very exciting first step."

As with all "Phase I" trials, the main goal of this study is to evaluate the safety of the therapy and determine the best dose for future studies. The 15 patients in the treatment group will receive standard treatments (such as fluids, antibiotics and blood pressure control), plus a planned intravenous dose of 0.3 to 3 million MSCs per kg of body weight. The MSCs will be obtained from the bone marrow of healthy donors and purified in the OHRI's Good Manufacturing Practice Laboratory in the Sprott Centre for Stem Cell Research. The researchers also plan to evaluate 24 similar septic shock patients who will receive standard treatments only (no MSCs). All patients will be rigorously monitored for side effects, and blood samples will be taken at specific time points to monitor the cells and their activity. This trial will not be randomized or blinded and it will not include enough patients to reliably determine if the therapy is effective. It will be conducted under the supervision of Health Canada and the Ottawa Hospital Research Ethics Board, and will have to be approved by both of these organizations before commencing.

"The OHRI is rapidly becoming known as a leader in conducting world-first clinical trials with innovative therapies such as stem cells," said Dr. Duncan Stewart, CEO and Scientific Director of OHRI, Vice-President of Research at The Ottawa Hospital and Professor of Medicine at uOttawa. "This research is truly pushing the boundaries of medical science forward, and is providing the citizens of Ottawa with access to promising new therapies."

"The Canadian Institutes of Health Research (CIHR) is very pleased to support this clinical trial," said Dr. Jean Rouleau, Scientific Director of the CIHR Institute of Circulatory and Respiratory Health. "The work of Dr. McIntyre and her colleagues will not only add to our growing knowledge of the benefits of stem-cell therapies, but will hopefully lead to treatments that can help save the lives of patients where currently, our treatment options are less than optimal."

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Ottawa researchers to lead world-first clinical trial of stem cell therapy for septic shock

OTTAWA A team of Canadian researchers has been awarded $442,000 to test the world's first experimental stem-cell therapy aimed at patients who suffer from septic shock, a runaway infection of the bloodstream that's notoriously difficult to treat.

The federal grant will allow researchers from the Ottawa Hospital Research Institute to use mesenchymal stem cells, found in the bone marrow of healthy adults, to treat as many as 15 patients with septic shock.

The deadly infection occurs when toxic bacteria spreads rapidly throughout the body and over-activates the immune system, leading to multiple organ failure and death in up to 40 per cent of cases.

One in five patients admitted to intensive-care units suffers from septic shock, making it the most common illness among a hospital's sickest of the sick.

Existing treatments focus on early diagnosis and intervention before organs start to fail. Patients with septic shock require aggressive resuscitation measures, large doses of intravenous antibiotics and, often, ventilators to help them breathe.

Yet because the infection can creep up on patients rapidly and cause unpredictable complications, death from septic shock remains relatively common.

The experimental therapy aims to use donor stem cells, grown and purified at the Ottawa laboratory, to dial down the body's hyperactive immune response and reduce the cascade of inflammation that leads to organ failure.

Early results from animal studies even raise the possibility that mesenchymal cells could eliminate the bacteria that causes septic shock, although the impact on humans is not yet known.

"It's a unique feature of the stem cells," said Dr. Lauralyn McIntyre, the intensive-care physician who is leading the trial. "Certainly no other therapy in the past, other than antibiotics, has impacted the bacterial load in the system."

As with other stem cells, mesenchymal cells can turn into a variety of more specialized cells and tissues that help repair and regenerate damaged organs. And because mesenchymal cells are derived from adults, they sidestep the ethical issues arising from the destruction of human embryos needed to make embryonic stem cells.

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Canadian researchers receive grant to test stem-cell therapy for septic shock