Author Archives: admin


Stem Cell Research in New York State | NYSTEM

(Complete report available in pdf format - 1.6 MB)

Executive Summary I. Introduction II. Methodology and Scope III. The Potential of stem cell research and NYSTEM IV. Findings V. Observations and Recommendations from the NY stem cell research community

Directory of Principal Investigators (available in the PDF above) An updated directory of stem cell scientists in New York State is now available.

This report summarizes data obtained from responses to written surveys and structured personal interviews with stem cell scientists in New York State between June and October, 2007. The findings are accompanied by a directory of the scientists interviewed. The objectives of this initial inquiry were threefold: (i) identify institutions and scientists in New York State with ongoing stem cell research programs; (ii) develop an overview of the scope and directions of these researchers' activities and (iii) solicit the views of the stem cell science community in New York regarding the potential scope and mechanisms of funding by NYSTEM. Letters were sent to 42 institutions that were identified through publicly available funding and publication records as having relevant research efforts. Overall, from 28 responding institutions we received feedback from 162 principal investigators (PI) at 23 institutions in time for inclusion in this report. The first interviews took place July 20 and the most recent visit was October 4; in this span of 10 weeks we interviewed investigators from 21 institutions representing all geographic areas of the state.

Importantly, this inquiry identified a strong community of stem cell scientists across the state, as judged by publications and external funding, with diverse interests and expertise, who are well positioned to take immediate advantage of the opportunities that will be provided by NYSTEM. Within the limits of our surveys and interviews, we estimate that more than 200 scientists head laboratories conducting stem cell related research, and that roughly two-thirds of these have a major focus in some aspect of stem cell science. Our survey demonstrated that 52% of PIs have NIH funding. Based on an examination of public databases in 2006, the research scientists have attracted $39.5 million in National Institutes of Health (NIH) funding for stem cell research, as well as substantial foundation, industry and other types of support for which no comprehensive figures are available. Based on survey responses, their work has resulted in at least 115 patents and 16 licenses. Moreover, we estimate that approximately 1,000 scientists, trainees and support staff are currently employed in their academic and private laboratories conducting stem cell research. The data provide fuel for the need for traineeships. At present, only 58% of PIs have graduate students working on stem cells. The situation is similar but slightly better with postdocs as 67% of PIs report having a postdoc.

Stem cell research in New York is broad in scope and highly collaborative, as about 80% of the investigators reported at least one collaboration. One objective of our inquiry was to ascertain the scope of this research within New York State and determine if there are dominant themes or specific areas of strength. Based on data from 162 scientists, the largest fractions had a focus on cancer, neural disease or aging. Other major topics included hematopoietic and musculoskeletal disease and diabetes. The data also showed that many investigators are engaged in studies of fundamental aspects of stem cell biology. Of the 162 respondents, nearly half reported that their research significantly concerned basic stem cell biology.

The types and sources of stem cells that researchers use in their studies are diverse. The majority of investigators use rodent or other non-human sources to supply stem cells for their research. However, nearly half of the investigators use stem cells of human origin, most of whom employ non-embryonic derived cells of a variety of types: hematopoietic and mesenchymal stem cells from marrow and umbilical cord blood, amniotic stem cells, and organ-specific cells derived from skin, cardiac, liver, kidney and other sources. Also included in this list are cancer stem cells. A smaller but still significant fraction of investigators use human embryonic stem cells (hESCs) in their work, or hold approved protocols and plan to use hESCs in the immediate future. Of 39 investigators, 24 reported using only NIH-approved ("registry") hESC lines and 15 reported using "non-registry" lines. Investigators planning to derive new stem cell lines from embryos deemed non-viable, were included in the "non-registry" hESC group. Several of those involved in hESC work were doing so only through collaborations with investigators at other institutions, and only about half of those using hESCs in their work reported that it represented a large fraction of their effort.

In terms of funding preferences, there was strong, but not universal agreement for the use of an investigator-initiated NIH R01-like grant mechanism that would provide substantial funding to individual laboratories for multiple years. Many also favored an additional mechanism of investigator-initiated funding analogous to the NIH R21 vehicle which encourages higher risk with the promise of greater reward. Many interviewees supported institution-based multi-investigator grants in which several researchers at one institution, or investigators at several institutions, collaborate on complementary aspects of a particular research problem. There was considerable support for individual postdoctoral fellowships or young investigator grants as a mechanism for bringing new talent into the stem cell field. Likewise there was considerable enthusiasm for short -term funding for "sabbaticals" in which investigators could visit another laboratory (inside or outside NYS) to acquire specific training in stem cell science or a field that would benefit particular aspects of stem cell research. In contrast, there was little support for institutional training grants, in which graduate students or postdocs are supported en masse, often to work in assigned labs.

It is clear that the area of hESC research has been constrained by inadequate federal support, and that an important focus of NYSTEM should be to enhance opportunities for hESC studies within appropriate ethical guidelines as established by the Board. Concurrent with this opinion, there was unanimity among these researchers that NYSTEM funding should not be restricted to hESC work, since it is unknown at this time which human stem cell types (embryonic or adult) will be best suited for application to particular diseases. Several investigators involved in translational research noted that there is a major gap in available federal funding for pre-clinical studies that move important findings from animal models to human systems. New York State funding for advanced biotechnology core facilities was identified as important by a number of individuals. In particular, researchers working with non-registry hESC indicated that the duplication of equipment required by current federal funding restrictions was a hardship.

Read this article:
Stem Cell Research in New York State | NYSTEM

Stem cell heart failure treatment advances

Stem cells grown under low oxygen. These stem cells from Stemedica are licensed to CardioCell.

CardioCell, a San Diego stem cell company, has started a Phase 2a trial of its treatment for chronic heart failure.

The companys special stem cells will be injected into patients with heart failure not caused by a heart attack. Nearly 2 million Americans have that kind of heart failure.

CardioCell is also testing these stem cells on heart attack patients to help their recovery. The cells are licensed from Stemedica, CardioCell's parent company.

Taken from bone marrow, the stem cells produce chemicals intended to heal malfunctioning heart cells. They are grown under low oxygen conditions, or hypoxia. CardioCell says hypoxia reflects the conditions under which natural stem cells exist. Histogen, also of San Diego, is developing its own kind of low-oxygen stem cells.

Growing stem cells with abundant oxygen reduces their "stemness," and they become prone to differentiate, said Sergey Sikora, CardioCell's president and chief executive.

Sergey Sikora, president and CEO of CardioCell / CardioCell

More than 20 patients are being sought to take part in the study, which is taking place at three locations. These are Emory University in Atlanta, Northwestern University in Chicago, and the University of Pennsylvania in Philadelphia.

Patients will receive injections of the stem cells, and a control group will receive a saline injection. After 90 days, the groups will be reversed. Patients who had received the stem cells will get a saline injection, and the control group will get the stem cells.

The stem cells last for about a month, after which they disappear, Sikora said.

Originally posted here:
Stem cell heart failure treatment advances

Lung Institute Reveals New Stem Cell Therapy Case Study

Tampa, FL (PRWEB) July 10, 2014

The Lung Institute works to help people who have received a diagnosis of debilitating lung disease get their quality of life back. The latest case study demonstrates how stem cell therapy can be used effectively to treat interstitial lung disease. After his recent stem cell treatment at the Lung Institute, Al Corter can now complete his daily tasks on his horse farm much faster, and finally attend the Silver Spur Riding Club Open Horse Show the weekend of July 12th in Fonda, NY.

Twelve years ago, Al was exposed to toxic fumes in the workplace and subsequently diagnosed with interstitial lung disease and bronchiectasis, a form of chronic obstructive pulmonary disorder (COPD). Living in upstate New York and running his horse farm, Als serious pulmonary conditions had a major effect on his life. Shortness of breath, coughing, reliance on supplemental oxygen and fatigue were taking a toll. Al needed a new solution.

Al decided to travel to Florida to undergo stem cell treatment at the Lung Institute facility in Tampa. He was seeking an alternative treatment to help with his symptoms. Stem cell therapy is a minimally invasive process that involves extracting stem cells, and then reintroducing them to cue the bodys natural healing processes. The stem cells are taken from the patients own body, so there is no controversy or risk of rejection.

Stem cell therapy is a viable option for many people with lung disease, said Dr. Burton Feinerman, Medical Director of the Lung Institute. Our patients are breathing easier, walking further and depending less on supplemental oxygen.

Prior to stem cell therapy, Al was needing more and more supplemental oxygen. His quality of life had taken a sharp turn downward. Following adipose stem cell treatment, Al is feeling better. He is getting back to the routine at the farm. Al used to use 5 to 6 liters of continuous oxygen to get his outdoor farm work done. Now, he is able to do these daily chores faster, and uses only 4 to 5 liters of oxygen on a pulsing regulator.

Im getting everyday tasks done quicker, said Al. Im using about half the amount of oxygen as I was before to do the same activities. My quality of life has definitely improved.

The Lung Institute has treated hundreds of patients with lung disease from around the country and the world. Regardless of the stage of the disease, patients are able to undergo stem cell therapy, which helps damaged lung tissue, and can lessen their symptoms.

About Lung Institute At the Lung Institute, we are changing the lives of hundreds of people across the nation through the innovative technology of regenerative medicine. We are committed to providing patients a more effective way to address pulmonary conditions and improve quality of life. Our physicians, through their designated practices, have gained worldwide recognition for the successful application of revolutionary minimally invasive stem cell therapies. With over a century of combined medical experience, our doctors have established a patient experience designed with the highest concern for patient safety and quality of care. For more information, visit our website at LungInstitute.com, like us on Facebook, follow us on Twitter or call us today at (855) 469-5864.

###

Read this article:
Lung Institute Reveals New Stem Cell Therapy Case Study

A new genome editing method brings the possibility of gene therapies closer to reality

PUBLIC RELEASE DATE:

11-Jul-2014

Contact: Jia Liu liujia@genomics.cn BGI Shenzhen

July 3, 2014, Shenzhen, China Researchers from Salk Institute for Biological Studies, BGI, and other institutes for the first time evaluated the safety and reliability of the existing targeted gene correction technologies, and successfully developed a new method, TALEN-HDAdV, which could significantly increased gene-correction efficiency in human induced pluripotent stem cell (hiPSC). This study published online in Cell Stell Cell provides an important theoretical foundation for stem cell-based gene therapy.

The combination of stem cells and targeted genome editing technology provides a powerful tool to model human diseases and develop potential cell replacement therapy. Although the utility of genome editing has been extensively documented, but the impact of these technologies on mutational load at the whole-genome level remains unclear.

In the study, researchers performed whole-genome sequencing to evaluate the mutational load at single-base resolution in individual gene-corrected hiPSC clones in three different disease models, including Hutchinson-Gilford progeria syndrome (HGPS), sickle cell disease (SCD), and Parkinson's disease (PD).

They evaluated the efficiencies of gene-targeting and gene-correction at the haemoglobin gene HBB locus with TALEN, HDAdV, CRISPR/CAS9 nuclease, and found the TALENs, HDAdVs and CRISPR/CAS9 mediated gene-correction methods have a similar efficiency at the gene HBB locus. In addition, the results of deep whole-genome sequencing indicated that TALEN and HDAdV could keep the patient's genome integrated at a maximum level, proving the safety and reliability of these methods.

Through integrating the advantages of TALEN- and HDAdV-mediated genome editing, researchers developed a new TALEN-HDAdV hybrid vector (talHDAdV), which can significantly increase the gene-correction efficiency in hiPSCs. Almost all the genetic mutations at the gene HBB locus can be detected by telHDAdV, which allows this new developed technology can be applied into the gene repair of different kinds of hemoglobin diseases such as SCD and Thalassemia.

###

About BGI

The rest is here:
A new genome editing method brings the possibility of gene therapies closer to reality

No Extra Mutations in Modified Stem Cells, Study Finds

Contact Information

Available for logged-in reporters only

Newswise LA JOLLA-The ability to switch out one gene for another in a line of living stem cells has only crossed from science fiction to reality within this decade. As with any new technology, it brings with it both promise--the hope of fixing disease-causing genes in humans, for example--as well as questions and safety concerns. Now, Salk scientists have put one of those concerns to rest: using gene-editing techniques on stem cells doesn't increase the overall occurrence of mutations in the cells. The new results were published July 3 in the journal Cell Stem Cell.

"The ability to precisely modify the DNA of stem cells has greatly accelerated research on human diseases and cell therapy," says senior author Juan Carlos Izpisua Belmonte, professor in Salk's Gene Expression Laboratory. "To successfully translate this technology into the clinic, we first need to scrutinize the safety of these modified stem cells, such as their genome stability and mutational load."

When scientists want to change the sequence of a stretch of DNA inside cells--either for research purposes or to fix a genetic mutation for therapeutic purposes--they have their choice of two methods. They can use an engineered virus to deliver the new gene to a cell; the cell then integrates the new DNA sequence in place of the old one. Or scientists can use what's known as custom targeted nucleases, such as TALEN proteins, which cut DNA at any desired location. Researchers can use the proteins to cut a gene they want to replace, then add a new gene to the mix. The cell's natural repair mechanisms will paste the new gene in place.

Previously, Belmonte's lab had pioneered the use of modified viruses, called helper-dependent adenoviral vectors (HDAdVs) to correct the gene mutation that causes sickle cell disease, one of the most severe blood diseases in the world. He and his collaborators used HDAdVs to replace the mutated gene in a line of stem cells with a mutant-free version, creating stem cells that could theoretically be infused into patients' bone marrow so that their bodies create healthy blood cells.

Before such technologies are applied to humans, though, researchers like Belmonte wanted to know whether there were risks of editing the genes in stem cells. Even though both common gene-editing techniques have been shown to be accurate at altering the right stretch of DNA, scientists worried that the process could make the cells more unstable and prone to mutations in unrelated genes--such as those that could cause cancer.

"As cells are being reprogrammed into stem cells, they tend to accumulate many mutations," says Mo Li, a postdoctoral fellow in Belmonte's lab and an author of the new paper. "So people naturally worry that any process you perform with these cells in vitro--including gene editing--might generate even more mutations."

To find out whether this was the case, Belmonte's group, in collaboration with BGI and the Institute of Biophysics, Chinese Academy of Sciences in China, turned to a line of stem cells containing the mutated gene that causes sickle cell disease. They edited the genes of some cells using one of two HDAdV designs, edited others using one of two TALEN proteins, and kept the rest of the cells in culture without editing them. Then, they fully sequenced the entire genome of each cell from the four edits and control experiment.

While all of the cells gained a low level of random gene mutations during the experiments, the cells that had undergone gene-editing--whether through HDAdV- or TALEN-based approaches--had no more mutations than the cells kept in culture.

More:
No Extra Mutations in Modified Stem Cells, Study Finds

Keck Medicine pushing into O.C. with oncology network acquisition

Keck Medicine of USC has acquired a small oncology network, Orange Coast Oncology Hematology, to expand its growing presence in Orange County.

Keck intends to change the name of the newly acquired network to USC Oncology/Hematology, which will operate out of offices in Newport Beach and Irvine.

Orange County cancer patients will now have access to university-based treatment, including clinical trials and genetic stem cell research, without having to drive to Los Angeles, said Thomas Jackiewicz, chief of Keck Medicine of USC.

The acquisition is part of Keck Medicines ongoing expansion into Orange County, Jackiewicz said. Keck Medicine has previously affiliated with Hoag Memorial Hospital Presbyterian in Newport Beach as part of its Orange County outreach.

We realized a lot of people were leaving Orange County for their cancer care, Jackiewicz said. We really wanted to make it about the patient and try to bring cancer care closer to home.

Under the acquisition, which was announced Wednesday, physicians with the former Orange Coast Oncology Hematology will become faculty at Keck School of Medicine. The physicians joining Keck include Greg Richard Angstreich, Minh D. Nguyen, George B. Semeniuk III, Dilruba Haque and Louis VanderMolen.

More:
Keck Medicine pushing into O.C. with oncology network acquisition

Stem cell patient has NOSE grows on her back after 8 years

Unnamed woman had tissue from her nose implanted in her spine in the hope the cells would help repair nerve damage causing paralysis Treatment failed and woman complained of increasing pain in the area Eight years later, a 3cm growth made of nasal tissue and bones appeared

By Emily Payne

Published: 07:58 EST, 9 July 2014 | Updated: 12:55 EST, 9 July 2014

1,333 shares

296

View comments

Nose-like: A 3cm growth formed from nasal tissue, bone and nerve branches was discovered

A woman has developed a nose-like growth eight years after a stem cell treatment to cure her paralysis failed.

At the Hospital de Egas Moniz in Lisbon, Portugal, the unnamed woman, a U.S. citizen, had tissue from her nose implanted in her spine.

Doctors hoped the cells would develop into neural cells and help repair the nerve damage to the woman's spine.

Follow this link:
Stem cell patient has NOSE grows on her back after 8 years

Stem cell boss joins board he funded

Alan Trounson, then president of the California Institute for Regenerative Medicine, poses for a portrait at his offices in San Francisco, Monday, March 9, 2009. (AP Photo/Eric Risberg)

The former head of California's stem cell agency, which is handing out $3 billion of voter-approved funds for research, has joined the board of a major grant recipient one week after leaving his post.

Alan Trounson, the former president of the California Institute for Regenerative Medicine, has joined the board of StemCells Inc., the recipient of $19.4 million from the agency.

The agency has been grappling with potential conflicts of interest, some of which are built into its governance under Proposition 71, approved by voters in 2004. CIRM paid $700,000 for a report last year making recommendations on how to mitigate conflicts.

Trounson's move has reignited debate over the issue.

"The announcement raises serious and obvious concerns on a number of fronts," Chairman Jonathan Thomas wrote to his colleagues on the CIRM board. "Under state law, however, it is permissible for Dr. Trounson to accept employment with a CIRM-funded company. Nonetheless, state law does impose some restrictions on Dr. Trounsons post-CIRM employment activities.

Board members will be forbidden to discuss the company with Trounson for one year after his departure, Thomas wrote.

Randy Mills, Trounson's successor as agency president, said in a statement Wednesday that "in the interests of transparency and good governance we will be conducting a full review of all CIRM activities relating to StemCells Inc.

"We take even the appearance of conflicts of interest very seriously," Mills said in the statement.

Not only board members, but CIRM employees are being reminded of the conflict of interest rules.

View original post here:
Stem cell boss joins board he funded

NOSE grows on woman's BACK after failed stem cell treatment

A woman found a nose-like growth on her back eight years after stem cell treatment[GETTY]

The unnamed woman had tissue from her nose implanted in her spine to help cure her paralysis but the procedure failed.

Eight years later the woman complained of increasing pain in the area and doctors found a three-centimetre long growth made of nasal tissue.

It was discovered last year and was also made of bits of bone and nerve branches but it had not connected with her spinal nerves.

Surgeons at the University of Iowa Hospitals removed the growth from the US citizen, then aged 28, which they said was not cancerous.

The unnamed woman had tissue from her nose implanted in her spine [GETTY / PIC POSED BY MODEL]

Doctors discovered a nose-like growth on a woman's back eight years after stem cell treatment

However, it was secreting a "thick copious mucus-like material" which likely caused the pain on the woman's spine.

The results of the surgery were published in New Scientist today.

George Daley, a stem cell researcher at Harvard Medical School, said: "It is sobering.

Continued here:
NOSE grows on woman's BACK after failed stem cell treatment

TESTS ADVANCE FOR CARDIOCELL MEDICINE

CardioCell, a stem cell company in San Diego, has started a Phase 2a clinical trial of its treatment for chronic heart failure.

The companys special stem cells are injected into patients with heart failure not caused by a heart attack. Nearly 2 million Americans have that kind of heart failure.

Separately, CardioCell is testing these stem cells on heart attack patients.

The cells are licensed from Stemedica, the parent company of CardioCell.

Taken from bone marrow, the stem cells produce chemicals intended to heal malfunctioning heart cells. They are grown under low-oxygen conditions, or hypoxia. CardioCell said hypoxia reflects the conditions under which natural stem cells exist.

Also in San Diego, the company Histogen is developing its own type of low-oxygen stem cells.

Growing stem cells with abundant oxygen reduces their stemness, and they become prone to differentiation turning into other types of cells, said Sergey Sikora, CardioCells president and chief executive.

More than 20 patients are being sought for the Phase 2a trial, which is taking place at Emory University in Atlanta, Northwestern University near Chicago and the University of Pennsylvania in Philadelphia.

Patients receive injections of the stem cells, while a control group receives a saline injection. After 90 days, the groups treatment will be reversed.

The stem cells live for about a month, Sikora said.

Read more here:
TESTS ADVANCE FOR CARDIOCELL MEDICINE