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Scientist for gene therapy regulation

While highlighting the need to define ethical principles to guide research studies with human subjects, K. Kasturirangan, member (science), Planning Commission, Government of India, sounded a word of caution on gene therapy, and called for regulation and legislation to prevent misuse.

Over the years, we are witnessing that the trend of medical science is to shift away from treating the evident symptoms or organ failures in diagnosis and treatment at the molecular level. This revolution, this ability to intervene at the genetic level, has the potential to permanently change the human gene pool, and has also triggered moral debates, he said, delivering the address at the 19 convocation of Sri Ramachandra University on Thursday.

He called for careful regulation, legislation and social consensus to guide actions against any misuse of this capability.

With Indias plans for manned space missions, he said, a new dimension needed to be created with respect to medical education to understand the physiological and psychological effects on humans in space environment, and to adopt necessary counter measures.

Mr. Kasturirangan distributed gold medals to meritorious students. MBBS graduate D. Rajmadhangi bagged four gold medals.

Chancellor of the university V.R. Venkataachalam and vice-chancellor J.S.N. Murthy were present.

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Stem Cell Therapy Could Transform Parkinson’s

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Stem cell therapy is emerging as a promising treatment for Parkinson's disease

Neurosurgeon Ivar Mendez of the University of Saskatchewan often shows a video clip to demonstrate his work treating Parkinson's disease. It features a middle-aged man with this caption: Off medications. The man's face has the dull stare typical of Parkinson's. Asked to lift each hand and open and close his fingers, he barely manages. He tries but fails to get up from a chair without using his hands. When he walks, it is with the slow, shuffling gait that is another hallmark of Parkinson's, a progressive neurological disorder that afflicts an estimated one million Americans, most of them older than 60.

Then the video jumps forward in time. The same man appears, still off medications. It is now eight years since Mendez transplanted dopamine cells from a fetus into the patient's brain. These neurons, which live in a midbrain region called the substantia nigra and secrete the neurotransmitter dopamine to initiate movement, are the ones that die off in Parkinson's. The man has aged, but his energy and demeanor are characteristic of a much younger man. Asked to do the same tasks, he smoothly raises his arms high and flicks his fingers open and shut rapidly. Arms crossed on his chest, he rises from a chair with apparent ease. Then he struts down the hall.

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NIH Stem-Cell Program Closes

The director of the agency's Center for Regenerative Medicine resigned on March 28 after just one clinical-trial award had been made

Therapies based on induced pluripotent stem cells, here differentiating into retinal cells on a scaffold, were the focus of the Center for Regenerative Medicine. Credit: NIH

Stem-cell researchers at the National Institutes of Health (NIH) have been left frustrated and confused following the demise of the agencys Center for Regenerative Medicine (CRM). The intramural programs director, stem-cell biologist Mahendra Rao, left the NIH, in Bethesda, Maryland, on 28March, and the centers website was taken down on 4 April. Although no official announcement had been made at the timeNaturewent to press, NIH officials say that they are rethinking how they will conduct in-house stem-cell research.

Researchers affiliated with the center say that they have been left in the dark. When contacted byNatureon 7April, George Daley, a stem-cell biologist at Harvard Medical School in Boston, Massachusetts, and a member of the centers external advisory board, said that he had not yet been told of Raos departure or the centers closure.

The CRM was established in 2010 to centralize the NIHs stem-cell program. Its goal was to develop useful therapies from induced pluripotent stem (iPS) cells adult cells that have been converted into embryonic-like stem cells and shepherd them towards clinical trials and regulatory approval. Its budget was intended to be $52million over seven years.

Rao took the helm in 2011. Relations seem to have soured last month owing to an NIH decision to award funding to only one project aiming to move iPS cells into a clinical trial. Rao says he resigned after this became clear. He says that he had hoped that five trials would be funded, especially because the center had already sorted out complex issues relating to tissue sources, patents and informed consent.

James Anderson, director of the NIHs Division of Program Coordination, Planning, and Strategic Initiatives, which administered the CRM, counters that only one application that made by Kapil Bharti of the National Eye Institute in Bethesda and his colleagues received a high enough score from an external review board to justify continued funding. The team aims to use iPS cells to treat age-related macular degeneration of the retina, and hopes to commence human trials within a few years. Several other proposals, which involved the treatment of cardiac disease, cancer and Parkinsons disease, will not receive funding to ready them for clinical trials. Anderson stresses that Bhartis trial will not be affected by the CRMs closure.

Other human iPS-cell trials are further along. For example, one on macular degeneration designed by Masayo Takahashi at the RIKEN Center for Developmental Biology in Kobe, Japan, began recruiting patients last August.

Anderson says that the CRM will not continue in its current form. The field is moving so fast that we need to rethink. To that end, the NIH plans to hold a workshop in May to gather stem-cell researchers together and decide what to do with the program and its remaining budget. To me thats just smart science, he says. If somethings not on track you dont keep spending money on it.

One option could be to allow CRM projects to be absorbed by the National Center for Advancing Translational Sciences, an NIH institute established in 2011 to translate basic research into therapies. But Anderson says that participants at the workshop will also discuss whether the NIH needs to replace the CRM with another dedicated stem-cell program.

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NIH Stem-Cell Program Closes

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Mahendra Rao Joins The New York Stem Cell Foundation Research Institute

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Newswise NEW YORK, NY (April 9, 2014) Dr. Mahendra Rao, who has directed the Center for Regenerative Medicine at the National Institutes of Health (NIH CRM) since 2010, will join The New York Stem Cell Foundation (NYSCF) Research Institute as its Vice President for Regenerative Medicine, a newly created position, Susan Solomon, NYSCF Chief Executive Officer, announced today.

Dr. Rao, who holds an MD degree and a PhD in developmental neurobiology, is one of the nations most prominent stem cell scientists. He has over twenty years of experience in all aspects of the stem cell field including government, academia, and business. Before joining the NIH, Dr. Rao spent six years as the vice president of Regenerative Medicine at Life Technologies, Inc. (now Thermo Fisher Scientific) after serving as the chief of Neurosciences at the National Institute on Aging and co-founding Q Therapeutics, a neural stem cell company based in Utah. Dr. Rao is tenured at the University of Utah School of Medicine in both Neurobiology and Anatomy and has over twenty submitted and ten issued patents.

Dr. Raos expertise in translational research, academia, and industry make him a valuable asset in our mission to take stem cell research from the laboratory to the clinic in order to find cures for the diseases that affect those we love, Solomon said. We are delighted to have him on board.

Solomon said that recruiting Dr. Rao is a major coup for NYSCF as it builds on its existing successes and carries out its strategic goals. Dr. Raos expertise and experience in setting up a company and in leading the translational effort at NIH will complement their expertise in automation and high-throughput induced pluripotent stem (iPS) cell generation.

I am enthused about NYSCFs efforts to generate high-quality stem cell lines and partner with the pharma and academic communities. I am excited to be joining them to advance their goals, said Dr. Rao.

In addition to his business career, Dr. Rao has served on scientific advisory boards, editorial boards and review panels and on committees including as the U.S. Food and Drug Administrations Cellular, Tissue, and Gene Therapies Advisory Committee chair and as the California Institute of Regenerative Medicine and International Society for Stem Cell Research liaison to the International Society for Cellular Therapy. Currently, he sits on the board of Cesca Therapeutics, Inc. and serves as the Chief Strategy Officer and Chairman of the Scientific Advisory Board at Q Therapeutics.

"Mahendra is a widely-recognized and accomplished leader in stem cell research. He will be a major asset for NYSCF as we continue to develop new therapeutics for patients," said Dr. Zach Hall, NYSCF Board Member and former Director of National Institute of Neurological Disorders and Stroke.

About The New York Stem Cell Foundation

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Study by prominent Brigham scientists retracted due to compromised data

A Brigham and Womens Hospital stem cell study, which raised the possibility that the human heart could repair itself, has been retracted after an internal investigation showed the researchers used compromised data.

The retraction comes just a week after a Japanese scientist was accused of fabricating data in a major stem cell paper that was led by a different Brigham scientist.

The authors of the retracted paper claimed they had found evidence that heart muscle can regenerate at a higher rate than previously thought. The work was part of a broad effort to discover the bodys natural regenerative abilities and harness them to create therapies that could repair damaged or diseased hearts.

The paper, published in 2012 in the journal Circulation, was withdrawn Tuesday by the journals publisher, the American Heart Association. An ongoing institutional review by Harvard Medical School and Brigham and Womens Hospital has determined that the data are sufficiently compromised that a retraction is warranted, the journal said.

This retraction is highly significant. In my 30 years in cardiovascular science I cannot recall a paper of similar prominence being retracted from Circulation, Dr. Charles Murry, codirector of the Institute for Stem Cell and Regenerative Medicine at the University of Washington, wrote in an e-mail. This appears to settle the controversy about the rate of cell replacement in the human heart.

Dr. Rose Marie Robertson, chief science officer for the American Heart Association, said that the journal received the retraction request from Harvard Medical School, in a letter that described concerns about several figures in the paper. She declined to elaborate on what the specific problems were.

The journals retraction notice does not specify whether the data irregularities were accidental or intentional, or which researchers were at fault. The authors include several high-profile scientists, including Dr. Piero Anversa, a cardiologist whose research has often raised questions from other scientists, and Dr. Joseph Loscalzo, chief of medicine at the Brigham.

Robertson said that based on the information provided by Harvard, the Heart Association did not have concerns about the role Loscalzo played in the paper. Loscalzo is the editor of the journal Circulation and recused himself from the retraction process, she said.

The study was supported by funds from the National Institutes of Health. In 2013, Anversas lab received $6.9 million from the agency, according to an NIH website. The federal Office of Research Integrity, which reviews allegations of scientific misconduct on federally sponsored research, said because of privacy reasons, it could not confirm or deny an investigation.

The key authors of the paper did not respond to direct requests for comment, and a Brigham spokeswoman declined to make them available. The hospital released a statement saying, Any questions, concerns, or allegations regarding research conducted at BWH are confidentially evaluated per the hospitals policies and federal regulations.

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Stem Cell Therapy for Chronic Pain – Video


Stem Cell Therapy for Chronic Pain

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Gloria Arroyos stem cell therapy doc blamed for cancer patient’s death

A doctor offering stem cell therapy may face charges for the death of a cancer patient who allegedly underwent treatment similar to that administered to former president and incumbent Pampanga Rep. Gloria Macapagal-Arroyo.

This was after it was found out that she is not a licensed doctor in the Philippines.

A report on GMA News TV's "News To Go" on Wednesday said a complaint has been filed against Dr. Antonia Carandang-Park at the National Bureau of Investigation (NBI) by Bernard Tan, who claims that his daughter, Kate, died after going through the said alternative treatment.Cancer patient Kate Tan received stem cell therapy from Dr. Antonia Carandang-Park, Gloria Arroyo's doctor.

Park owns the Tagaytay-based Green & Young Health and Wellness Center where Arroyoburdened by persistent trouble with her cervical spinesought treatment in 2012.

In an interview with GMA News, Tan said his daughter, who had Hodgkin's lymphoma (a type of cancer of the blood), was given "the same treatment that [Park] did with Gloria," which included "juicing diet, vegetable diet... acupuncture coffee enema, at 'yun na nga, stem cell."

Stem cell therapy introduces new adult stem cells into damaged tissue in order to treat disease or injury.

"Ang sabi niya, 'Give me three months, magaling na 'yan,'" Tan told GMA News. He added that his family was easily convinced to take their daughter to Park's wellness center because "Presidente na ng Pilipinas ang pumunta doon."

"Siguro naman na-scrutinize na nila 'yan, na-background check na nila 'yan," he said. "Kumbaga, 'yung credibility no'n, nag-build up na."

Kate was fed nothing but bananas and vegetable juices for three months, and had eight rounds of "embryonic" stem cell treatment, he said.

However, the 23-year-old lost even more weight, prompting the family to seek the assistance of a different doctor. Kate had eight rounds of 'embryonic' stem cell treatment, her father Bernard Tan said.Seven months later, in July 2013, Kate passed away.

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Stem Cell Institute Public Seminar on Adult Stem Cell Therapy Clinical Trials in New York City May 17th, 2014

New York, NY (PRWEB) April 09, 2014

The Stem Cell Institute, located in Panama City, Panama, will present an informational umbilical cord stem cell therapy seminar on Saturday, May 17, 2014 in New York City at the New York Hilton Midtown from 1:00 pm to 4:00 pm.

Speakers include:

Neil Riordan PhD Clinical Trials: Umbilical Cord Mesenchymal Stem Cell Therapy for Autism and Spinal Cord Injury

Dr. Riordan is the founder of the Stem Cell Institute and Medistem Panama Inc.

Jorge Paz-Rodriguez MD Stem Cell Therapy for Autoimmune Disease: MS, Rheumatoid Arthritis and Lupus

Dr. Paz is the Medical Director at the Stem Cell Institute. He practiced internal medicine in the United States for over a decade before joining the Stem Cell Institute in Panama.

Light snacks will be served afterwards. Our speakers and stem cell therapy patients will also be on hand to share their personal experiences and answer questions.

Admission is free but space in limited and registration is required. For venue information and to register and reserve your tickets today, please visit: http://www.eventbrite.com/e/stem-cell-institute-seminar-tickets-11115112601 or call Cindy Cunningham, Patient Events Coordinator, at 1 (800) 980-7836.

About Stem Cell Institute Panama

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Gloria Arroyos stem cell therapy doc blamed for cancer patient's death

A doctor offering stem cell therapy may face charges for the death of a cancer patient who allegedly underwent treatment similar to that administered to former president and incumbent Pampanga Rep. Gloria Macapagal-Arroyo.

This was after it was found out that she is not a licensed doctor in the Philippines.

A report on GMA News TV's "News To Go" on Wednesday said a complaint has been filed against Dr. Antonia Carandang-Park at the National Bureau of Investigation (NBI) by Bernard Tan, who claims that his daughter, Kate, died after going through the said alternative treatment.Cancer patient Kate Tan received stem cell therapy from Dr. Antonia Carandang-Park, Gloria Arroyo's doctor.

Park owns the Tagaytay-based Green & Young Health and Wellness Center where Arroyoburdened by persistent trouble with her cervical spinesought treatment in 2012.

In an interview with GMA News, Tan said his daughter, who had Hodgkin's lymphoma (a type of cancer of the blood), was given "the same treatment that [Park] did with Gloria," which included "juicing diet, vegetable diet... acupuncture coffee enema, at 'yun na nga, stem cell."

Stem cell therapy introduces new adult stem cells into damaged tissue in order to treat disease or injury.

"Ang sabi niya, 'Give me three months, magaling na 'yan,'" Tan told GMA News. He added that his family was easily convinced to take their daughter to Park's wellness center because "Presidente na ng Pilipinas ang pumunta doon."

"Siguro naman na-scrutinize na nila 'yan, na-background check na nila 'yan," he said. "Kumbaga, 'yung credibility no'n, nag-build up na."

Kate was fed nothing but bananas and vegetable juices for three months, and had eight rounds of "embryonic" stem cell treatment, he said.

However, the 23-year-old lost even more weight, prompting the family to seek the assistance of a different doctor. Kate had eight rounds of 'embryonic' stem cell treatment, her father Bernard Tan said.Seven months later, in July 2013, Kate passed away.

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Gloria Arroyos stem cell therapy doc blamed for cancer patient's death

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Blocking DNA repair mechanisms could improve radiation therapy for deadly brain cancer

UT Southwestern Medical Center researchers have demonstrated in both cancer cell lines and in mice that blocking critical DNA repair mechanisms could improve the effectiveness of radiation therapy for highly fatal brain tumors called glioblastomas.

Radiation therapy causes double-strand breaks in DNA that must be repaired for tumors to keep growing. Scientists have long theorized that if they could find a way to block repairs from being made, they could prevent tumors from growing or at least slow down the growth, thereby extending patients' survival. Blocking DNA repair is a particularly attractive strategy for treating glioblastomas, as these tumors are highly resistant to radiation therapy. In a study, UT Southwestern researchers demonstrated that the theory actually works in the context of glioblastomas.

"This work is informative because the findings show that blocking the repair of DNA double-strand breaks could be a viable option for improving radiation therapy of glioblastomas," said Dr. Sandeep Burma, Associate Professor of Radiation Oncology in the division of Molecular Radiation Biology at UT Southwestern.

His lab works on understanding basic mechanisms by which DNA breaks are repaired, with the translational objective of improving cancer therapy with DNA damaging agents. Recent research from his lab has demonstrated how a cell makes the choice between two major pathways that are used to repair DNA breaks -- non-homologous end joining (NHEJ) and homologous recombination (HR). His lab found that enzymes involved in cell division called cyclin-dependent kinases (CDKs) activate HR by phosphorylating a key protein, EXO1. In this manner, the use of HR is coupled to the cell division cycle, and this has important implications for cancer therapeutics. These findings were published April 7 in Nature Communications.

While the above basic study describes how the cell chooses between NHEJ and HR, a translational study from the Burma lab demonstrates how blocking both repair pathways can improve radiotherapy of glioblastomas. Researchers in the lab first were able to show in glioblastoma cell lines that a drug called NVP-BEZ235, which is in clinical trials for other solid tumors, can also inhibit two key DNA repair enzymes, DNA-PKcs and ATM, which are crucial for NHEJ and HR, respectively. While the drug alone had limited effect, when combined with radiation therapy, the tumor cells could not quickly repair their DNA, stalling their growth.

While excited by the initial findings in cell lines, researchers remained cautious because previous efforts to identify DNA repair inhibitors had not succeded when used in living models -- mice with glioblastomas. Drugs developed to treat brain tumors also must cross what's known as the blood-brain-barrier in living models.

But the NVP-BEZ235 drug could successfully cross the blood-brain-barrier, and when administered to mice with glioblastomas and combined with radiation, the tumor growth in mice was slowed and the mice survived far longer -- up to 60 days compared to approximately 10 days with the drug or radiation therapy alone. These findings were published in the March 1 issue of Clinical Cancer Research.

"The consequence is striking," said Dr. Burma. "If you irradiate the tumors, nothing much happens because they grow right through radiation. Give the drug alone, and again, nothing much happens. But when you give the two together, tumor growth is delayed significantly. The drug has a very striking synergistic effect when given with radiation."

The combination effect is important because the standard therapy for glioblastomas in humans is radiation therapy, so finding a drug that improves the effectiveness of radiation therapy could have profound clinical importance eventually. For example, such drugs may permit lower doses of X-rays and gamma rays to be used for traditional therapies, thereby causing fewer side effects.

"Radiation is still the mainstay of therapy, so we have to have something that will work with the mainstay of therapy," Dr. Burma said.

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Blocking DNA repair mechanisms could improve radiation therapy for deadly brain cancer

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Biomarkers in HPV negative squamous-cell carcinomas of head, neck

A quartet of proteins that play critical roles in cell replication, cell death, and DNA repair could lead to better targets for therapy against treatment-resistant head-and-neck squamous cell cancers.

In a study to be presented at the AACR Annual Meeting 2014 on Tuesday, April 8, Ranee Mehra, MD, a medical oncologist who specializes in head and neck cancers at Fox Chase Cancer Center, and colleagues, showed a correlation between the expression levels of these proteins in head-and-neck cancers negative for human papilloma virus (HPV). These tumors have a poorer prognosis than HPV-positive head-and-neck cancers.

The cancers arise in the cells that line moist surfaces in the mouth, including the lips, tongue, and gums, as well as the throat, larynx, and sinuses. The National Cancer Institute reports that some 75 percent of these cancers are caused by tobacco and alcohol use -- and that use of the two together are a greater risk than use of either separately.

Mehra's research could help determine potential treatments for head and neck cancers. "The ultimate goal would be to better understand a tumor's protein signature and underlying biology so that, in the future, we can better understand treatments are more likely to be beneficial to our patients with head and neck cancer."

She and colleagues looked at protein expression levels in samples from 101 cases of head-and-neck cancer banked from 1990 to 2002 in the Fox Chase tissue repository. One advantage of using this tissue, Mehra says, is that samples are cross-reference with patient treatment and survival data. Using tissue microarrays, which allow researchers to examine at a large number of samples at one time, the team looked for expression of ERCC-1, a DNA repair protein; survivin, a protein that inhibits programmed cell death or apoptosis; and two proteins active during cell division, Aurora A and phospho-Aurora A.

The research showed positive associations between expression of the repair protein ERCC1 and each cell-division protein, AuroraA (P < .0001) and phospho-Aurora (P = .0027). It also showed an association between both Aurora proteins (P < .0001). There was also an association between the apoptosis-regulator, survivin, and Aurora A (P = .0064), as well as survivin and ERCC1 (P = .0084).

A review of a publically available database examining mRNA expression levels for the four proteins showed a highly significant correlation between Aurora A and survivin (P = .002), confirming the protein microarray findings.

Survivin expression may prove a marker for improved survival, especially in patients who were treated with surgery plus radiation, Mehra said. She found that tumors with less than a median level of survivin expression were associated with improved patient survival compared to tumors with more than a median level of survivin (P = .03).

ERCC1 is already a potential prognostic biomarker for survival among patients treated with radiation after surgery. In a study published in Clinical Cancer Research last year, Mehra and colleagues found lower levels of ERCC1 predicted improved survival among patients treated with radiation.

"We hope to understand how these various pathways and mechanisms interrelate with each others. Understanding those pathways would help guide future research," Mehra said.

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