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Boston Children’s Hospital Stem Cell Program Newsroom

NIH grant to compare ES and iPS cells

A major question in stem cell biology is whether human induced pluripotent stem cells (iPS cells) are the functional and molecular equivalent to human embryonic stem cells (ES cells), and whether they can serve as an effective resource for research and for cell-based therapies. Under a new, $1.7 million Recovery Act grant from the National Institutes of Health, the laboratory of George Q. Daley, MD, PhD, will comprehensively compare iPS cells with ES cells isolated from embryos, ES cells produced through nuclear transfer, and ES cells created through parthenogenesis.

From fishtank to bedside: turbo-charging blood stem cell production

A new drug that boosts numbers of blood stem cells, originally discovered in zebrafish in the Boston Children’s Hospital laboratory of Leonard Zon, MD, goes to clinical trial in patients with leukemia and lymphoma. Read more from Dana Farber Cancer Institute, one of the sites of the trial, which is sponsored by Fate Therapeutics. For more on the trial itself, visit ClinicalTrials.gov.

iPS cells from patients with a rare premature aging disorder bring surprises

In patients with dyskeratosis congenita, genetic mutations impair a key enzyme, leaving the body’s cells unable to maintain the tips of their chromosomes, known as telomeres. This in turn makes the chromosomes more susceptible to insults from their environment, leading to degradation of multiple tissues and much-shortened lifespans.

Now, Suneet Agarwal, MD, PhD, and George Q. Daley, MD, PhD, investigators in Children’s Stem Cell Transplantation Program, have discovered that the process of creating iPS cells from patients with this disease actually reactivates the enzyme, allowing telomeres to be maintained — and potentially correcting the disease, without the need for gene therapy to correct the original mutation. The findings have broad-reaching implications, not just for understanding iPS cells, but also for the aging and cancer fields. And for patients with dyskeratosis congenita, they suggest the possibility of developing drugs to help maintain their telomeres, prolonging their lives, and of making blood stem cells from patients’ iPS cells to provide a safer bone marrow transplant. Read the reports from Reuters, Technology Review and USA Today.

The secret lives of stem cells

Through a novel technique using genetic tags as “barcodes,” Fernando Camargo, PhD, of Children’s Stem Cell Program is tracking the live workings of adult stem cells in mice, under a National Institutes of Health New Innovator Award. Camargo is monitoring the day-to-day activity and differentiation of individual stem cells and their offspring in their natural living environment over time. Although the work will track stem cells in the blood-forming system, the model is applicable to a variety of tissues and will yield many insights applicable to human disease and potential treatment by regenerating diseased or damaged tissues.

Matching melanoma tactic for tactic: taking on cancer stem cells

Melanoma, if not caught early, transforms into a highly deadly, treatment-resistant cancer. The immune system initially mounts anti-tumor attacks, but these assaults generally fail, allowing more advanced melanomas to metastasize. Now, research led by Markus Frank, MD, of the Transplantation Research Center of Children’s and Brigham and Women’s Hospital, provides the first evidence that this ability to dodge the immune system lies in the cancer stem cell. It also suggests several possible lines of counter-attack.

Publishing online January 12, 2010 in the journal Cancer Research, Frank and colleagues detail how melanoma stem cells actually lull the immune system into shielding the cancer from immune attack. In 2008, the same team made headlines when they showed that melanoma stem cells drive melanoma progression, enabling the cancer to resist chemotherapy drugs. Using the very molecule that endows this drug resistance, they selectively targeted melanoma stem cells for destruction, inhibiting tumor growth in mice. That strategy is now in clinical drug development.

Worldwide distribution of iPS lines

In an effort to support the research community, Children’s Stem Cell Program investigator George Daley, MD, PhD, has made more than 20 iPS lines developed at Children’s Hospital Boston available for use by other scientists through the Harvard Stem Cell Institute. To date, cell lines have been distributed to over 65 laboratories worldwide.

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