Category Archives: Cell Therapy

UCLA has received grant funding from the Center for the Advancement of Science in Space (CASIS) to lead a research mission that will send rodents to the International Space Station (ISS). The mission will allow astronauts on the space station and scientists on Earth to test a potential new therapy for accelerating bone growth in humans.

The research will be led by Dr. Chia Soo, a UCLA professor of plastic and reconstructive surgery and orthopaedic surgery, who is member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research. Soo is also research director for UCLA Operation Mend, which provides medical care for wounded warriors. The study will test the ability of a bone-forming molecule called NELL-1 to direct stem cells to induce bone formation and prevent bone degeneration.

Other members of the UCLA research team are Dr. Kang Ting, a professor in dentistry who discovered NELL-1 and is leading efforts to translate NELL-1 therapy to humans, Dr. Ben Wu, a professor of bioengineering who modified the NELL-1 molecule to make useful for treating osteoporosis, and Dr. Jin Hee Kwak, an assistant professor of dentistry who will manage daily operations.

Based on results of previous studies supported by the NIH, the UCLA-ISS team will begin ground operations in early 2015. They hope that the study will provide new insights into the prevention of bone loss or osteoporosis as well as the regeneration of massive bone defects that can occur in wounded military personnel. Osteoporosis is a significant public health problem commonly associated with "skeletal disuse" conditions such as immobilization, stroke, cerebral palsy, muscular dystrophy, spinal cord injury and jaw resorption after tooth loss.

"NELL-1 holds tremendous hope, not only for preventing bone loss but one day even restoring healthy bone," Ting said. "For patients who are bed-bound and suffering from bone loss, it could be life-changing."

The UCLA team will oversee the ground operations of the mission in tandem with a flight operation coordinated by CASIS and NASA.

"A group of 40 rodents will be sent to the International Space Station U.S. National Laboratory onboard the SpaceX Dragon capsule, where they will live for two months in a microgravity environment during the first ever test of NELL-1 in space," said Dr. Julie Robinson, NASA's chief scientist for the International Space Station program at the Johnson Space Center.

"CASIS is proud to work alongside UCLA in an effort to promote the station as a viable platform for bone loss inquiry," said Warren Bates, director of portfolio management for CASIS. "Through investigations like this, we hope to make profound discoveries and enable the development of therapies to counteract bone loss ailments common in humans."

Prolonged space flights induce extreme changes in bone and organ systems that cannot be replicated on Earth.

"Besides testing the limits of NELL-1's robust bone-producing effects, this mission will provide new insights about bone biology and could uncover important clues for curing diseases such as osteoporosis," Wu said.

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Growing bone in space: Study to test therapy for bone loss on the International Space Station

Stem cell therapy may have helped patients with a form of multiple sclerosis, according to a preliminary study.

Patients with relapsing-remitting multiple sclerosis showed signs of improvement after being treated with their own, or autologous "nonmyeloablative hematopoietic stem cells," a class of blood-forming stem cells, the study found. It was published Tuesday in the Journal of the American Medical Association.

Half, or 41 patients, tested two years after treatment experienced significant improvement on the Expanded Disability Status Scale, a measure of disability. And of patients tested at 4 years, 23, or 64 percent, showed significant improvement. Four-year relapse-free survival was 80 percent and progression-free survival was 87 percent.

"To our knowledge, this is the first report of significant and sustained improvement in the EDSS score following any treatment for MS," stated the study. It was led by Dr. Richard K. Burt of Northwestern University in Chicago.

However, only limited conclusions can be drawn from the uncontrolled study, according to scientists who examined the results. While the therapy was associated with improvement, the stem cell transplant may not have been key. A conditioning regimen that partially depleted the stem cells before transplantation may have been responsible, said Dr. Stephen L. Hauser in a JAMA article accompanying the study.

"According to Carl Sagan, 'extraordinary claims require extraordinary evidence,' a standard that is not always met in this report, and not claimed by the authors. Even though the authors appropriately acknowledge many of the limitations associated with their case series, their statement that 'to our knowledge, this is the first report of significant and sustained improvement in the EDSS score following any treatment for MS' could be challenged," Hauser wrote.

Jeanne Loring, a stem cell researcher who studies multiple sclerosis and other neurodegenerative diseases, agreed that the results are far from conclusive.

"Multiple sclerosis is an autoimmune disease, meaning that the patients' own immune cells attack their own nervous systems," Loring said by email after examining the study. "The authors of the JAMA article treated MS patients with their own blood stem cells in the hope that these cells would replace some of the self-destructive immune cells."

However, the uneven course of MS makes it hard to draw conclusions, wrote Loring, who heads the Center for Regenerative Medicine at The Scripps Research Institute in La Jolla.

"Most patients with MS have attacks, followed by recovery, followed by another attack. In a few of these patients, the blood stem cell treatment seemed to extend their time between attacks. It's important to understand that other treatments, including drugs, have shown similar modest improvements, so it's too soon to celebrate a stem cell therapy."

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MS patients given stem cells improve

Results from a preliminary study indicate that among patients with relapsing-remitting multiple sclerosis (MS), treatment with nonmyeloablative hematopoietic stem cell transplantation (low intensity stem cell transplantation) was associated with improvement in measures of disability and quality of life, according to a study in the January 20 issue of JAMA.

Fifty percent of patients with MS are unable to continue employment by 10 years from diagnosis or are unable to walk by 25 years. Despite an annual cost of approximately $47,000 per patient to treat MS, no therapy approved by the U.S. Food and Drug Administration has been shown to significantly reverse neurological disability or improve quality of life, according to background information in the article.

Multiple sclerosis is thought to be an immunemediated disorder of the central nervous system. Autologous (the use of one's own cells) hematopoietic (blood) stem cell transplantation (HSCT) is a form of immune suppression but unlike standard immune-based drugs, autologous HSCT is designed to reset rather than suppress the immune system. Richard K. Burt, M.D., of the Northwestern University Feinberg School of Medicine, Chicago, and colleagues studied the association of nonmyeloablative HSCT with neurological disability and other clinical outcomes in patients with relapsing-remitting MS (defined as acute relapses followed by partial or complete recovery and stable clinical manifestations between relapses; n = 123) or secondary-progressive MS (defined as a gradual progression of disability with or without superimposed relapses; n = 28) treated between 2003 and 2014.

Outcome analysis was available for 145 patients with an average follow-up of 2.5 years. On a measure of disability (Expanded Disability Status Scale [EDSS] score), there was significant improvement in 41 patients (50 percent of patients tested at 2 years) and in 23 patients (64 percent of patients tested at 4 years). "To our knowledge, this is the first report of significant and sustained improvement in the EDSS score following any treatment for MS," the authors write.

Receipt of HSCT was also associated with improvement in physical function, cognitive function and quality of life. There was also a reduction on another measure of clinical disease severity, volume of brain lesions associated with MS seen on magnetic resonance imaging (MRI). Four-year relapse-free survival was 80 percent and progression-free survival was 87 percent.

Patient selection is important in determining outcome, the researchers write. "In the post hoc analysis, the EDSS score did not improve in patients with secondary-progressive MS or in those with disease duration longer than 10 years."

The authors note the results are limited because this was an observational study without a control group. "Definitive conclusions will require a randomized trial; however, this analysis provides the rationale, appropriate patient selection, and therapeutic approach for a randomized study."

(doi:10.1001/jama.2014.17986; Available pre-embargo to the media at http://media.jamanetwork.com)

Editor's Note: This study was made possible by financial support from the Danhakl family, the Cumming Foundation, the Zakat Foundation, the McNamara Purcell Foundation, and Morgan Stanley and Company. Please see the article for additional information, including other authors, author contributions and affiliations, financial disclosures, etc.

Editorial: Hematopoietic Stem Cell Transplantation for MS

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Stem cell transplantation shows potential for reducing disability in patients with MS

Stem Cell Therapy | Recent Strides Quell Stem Cell Debate
Ethical concerns for stem cells for arthritis could be mute...maybe. Reports show that adult stem cells (Autologous) have been shown in recent studies to hav...

By: Nathan Wei

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Stem Cell Therapy | Recent Strides Quell Stem Cell Debate - Video

A Winnipeg-based company that has touted its ability to improve the lives of Multiple Sclerosis patients through stem cell therapy is now under the microscope after allegations of fraud from a client.

The CEO of Regenetek Research Inc. has been collecting thousands of dollars from Canadian patients looking for help. Some of the patients are now questioning the research and credentials of the man they know as Dr. Doug.

One of them is Lee Chuckry, 47. He has been living with MS for nearly two decades.

MS just keeps progressing, thats what it does. Hopefully I could stop it. That was my ultimate goal, Chuckry said in an interview with CTV News.

His efforts led him to Regenetek, and its CEO: Doug Broeska.

In testimonials, MS patients attributed miraculous medical improvement to experimental stem cell therapy. For $35,000, Regenetek patients were flown to India for the procedure.

Chuckry was one of the participants. But when he returned home, he says his symptoms worsened.

When he started digging deeper, he said, he found the doctor hed put his faith in wasnt what he claimed to be.

Im going to call Doug a con artist, Chuckry said. You are preying on people who are desperate. They are looking for hope of any sort.

Chuckry and at least one other patient have gone to the RCMP. They allege Broeska, who claims to hold a PhD and a Bachelor of Science, is a fraud who is operating as a medical researcher without proper credentials.

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Winnipeg company offering stem cell therapy is fraudulent, MS sufferer alleges

IMAGE:BioResearch Open Access is a bimonthly peer-reviewed open access journal led by Editor-in-Chief Robert Lanza, MD, Chief Scientific Officer, Advanced Cell Technology, Inc. and Editor Jane Taylor, PhD. The Journal... view more

Credit: Mary Ann Liebert, Inc., publishers

New Rochelle, NY, January 12, 2014--A promising new therapeutic approach to treat a variety of diseases involves taking a patient's own cells, turning them into stem cells, and then deriving targeted cell types such as muscle or nerve cells to return to the patient to repair damaged tissues and organs. But the clinical effectiveness of these stem cells has only been modest, which may be due to the advanced age of the patients or the effects of chronic diseases such as diabetes and cardiovascular disease, according to a probing Review article published in BioResearch Open Access, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers . The article is available on the BioResearch Open Access website.

Anastasia Yu. Efimenko, TN Kochegura, ZA Akopyan, and YV Parfyonova, Moscow State University (Russia), analyze how aging and chronic diseases might affect the regenerative potential of autologous stem cells and explain the differences between the promising results reported in preclinical studies using stem cells derived from healthy young donors and the more modest success of clinical studies in aged patients. The authors propose strategies to test for and enhance to regenerative properties and therapeutic potential of stem cells in the article "Autologous Stem Cell Therapy: How Aging and Chronic Diseases Affect Stem and Progenitor Cells".

"This review discusses a very important issue in regenerative medicine, how aging and chronic pathologies such as cardiovascular diseases and metabolic disorders affect adult stem/progenitor cells," says BioResearch Open Access Editor Jane Taylor, PhD, MRC Centre for Regenerative Medicine, University of Edinburgh, Scotland. "Future therapies are discussed by the authors in terms of overcoming or correcting the limitations of these cells in order to enhance their therapeutic potential."

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About the Journal

BioResearch Open Access is a bimonthly peer-reviewed open access journal led by Editor-in-Chief Robert Lanza, MD, Chief Scientific Officer, Advanced Cell Technology, Inc. and Editor Jane Taylor, PhD. The Journal provides a new rapid-publication forum for a broad range of scientific topics including molecular and cellular biology, tissue engineering and biomaterials, bioengineering, regenerative medicine, stem cells, gene therapy, systems biology, genetics, biochemistry, virology, microbiology, and neuroscience. All articles are published within 4 weeks of acceptance and are fully open access and posted on PubMed Central. All journal content is available on the BioResearch Open Access website.

About the Publisher

Mary Ann Liebert, Inc., publishers is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in many areas of science and biomedical research, including DNA and Cell Biology, Tissue Engineering, Stem Cells and Development, Human Gene Therapy, HGT Methods, and HGT Clinical Development, and AIDS Research and Human Retroviruses. 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 80 journals, books, and newsmagazines is available on the Mary Ann Liebert, Inc., publishers website.

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Is stem cell therapy less effective in older patients with chronic diseases?

Religious groups have concerns that the ALS Association, which has been the recipient of millions of dollars in donations through its ice bucket challenge, supports embryonic stem cell research.

The facts: The ALS Association which fights amyotrophic lateral sclerosis, commonly known as Lou Gehrigs Disease primarily is involved with adult stem cell research, which the Catholic Church does not oppose, according to the U.S. Conference of Catholic Bishops.

The bishops statement on stem cells states that the use of adult stem cells and umbilical-cord blood have been shown to offer a better way to produce cells that can benefit patients.

There is no moral objection to research and therapy of this kind, when it involves no harm to human beings at any stage of development and is conducted with appropriate informed consent, the statement says. Catholic foundations and medical centers have been, and will continue to be, among the leading supporters of ethically responsible advances in the medical use of adult stem cells.

The ALS Association said it does, however, fund one study that uses embryonic stem cell research with money provided by one specific donor who is committed to this area of research, the Record reported.

The association added that donors could designate that they do not want their embryos used to fund any stem cell research.

Most embryonic stem cells are derived from embryos that develop from eggs that have been fertilized in vitro and then donated for research with the consent of the donors, according to the National Institutes of Health. They are not derived from eggs fertilized in a womans body.

After a couple completes the in vitro process, they either continue to freeze the leftover embryos or allow them to thaw, which destroys the cells. In some states, couples do have the choice to donate the embryos to research or to adoptive families, TruthOrFiction.com notes.

Embryonic stem cell research does not appear to rank as a major issue for most Catholics, according to a 2013 survey by the Pew Researchs Religion and Public Life Project. In the survey, 72 percent of Catholics said embryonic stem cell research was not a moral issue or was morally acceptable.

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Fact Check: Adult stem cell research not opposed by church

Doctors, nurses and anesthesiologist's care for a horse that will be receiving stem cells to help repair a meniscal tear in the stifle at the CSU Veterinary Teaching Hospital in Fort Collins. (Joe Amon, The Denver Post)

Stem-cell research by Colorado State University staffers using bone marrow from horses to heal joint injuries on the same animal is making strides, and researchers have great hope that the project will lead to human medical applications.

A team with CSU's Equine Orthopaedic Research Center reports that adding stem-cell therapy to traditional arthroscopic surgery on horses has significantly increased success rates.

Horses that had follow-up, stem-cell treatment were twice as likely to return to normal activity as those that did not, said David Frisbie, an associate professor of equine surgery with CSU and part of the research team.

"We've doubled it, conservatively," in treating cartilage damage in the knee, Frisbie said.

The team had results of its work published last year in the journal Veterinary Surgery.

Some lesions in the meniscus of horses that could not be treated by surgery have been successfully mended using stem cells alone.

"Western performance horses, reining and cutting horses, and barrel horses are very prone to meniscal injuries," Frisbie said.

Beyond meniscus damage, researchers also have focused on tendon lesions in the lower leg, which typically strike race horses.

Horses that suffered a tendon lesion had about a 66 percent chance of reinjury after surgery. Add stem-cell treatment and the reinjury rate drops to 21 percent, Frisbie said.

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CSU research on horse injuries, stem-cell recovery, may help humans