Main Category: Lymphoma / Leukemia / Myeloma
Also Included In: Blood / Hematology; Stem Cell Research
Article Date: 01 Aug 2013 – 0:00 PDT
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Reprogramming patients’ cells offers powerful new tool for studying, treating blood diseases
Weiss, with Monica Bessler, M.D., Philip Mason, Ph.D., and Deborah L. French, Ph.D., all of CHOP, led a study on iPSCs and Diamond Blackfan anemia (DBA) published online June 6 in Blood. Another study by Weiss, French and colleagues in the same journal on April 25 focused on iPSCs in juvenile myelomonocytic leukemia (JMML).
“Ribosomal and hematopoietic defects in induced pluripotent stem cells derived from Diamond Blackfan anemia patients,” Blood, published online June 6, 2013.
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Children\’s Hospital of Philadelphia. “Reprogramming patients’ cells offers powerful new tool for studying, treating blood diseases.” Medical News Today. MediLexicon, Intl., 1 Aug. 2013. Web.
1 Aug. 2013. <http://www.medicalnewstoday.com/releases/264152.php>
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Furthermore, the cells offer a renewable, long-lasting model system for testing drug candidates or gene modifications that may offer new treatments, personalized to individual patients.
They then tested the cells with two drugs, each able to inhibit a separate protein known to be highly active in JMML. One drug, an inhibitor of the MEK kinase, reduced the proliferation of cancerous cells in culture. “This provides a rationale for a potential targeted therapy for this specific subtype of JMML,” said Weiss.
“Patient-derived induced pluripotent stem cells recapitulate hematopoietic abnormalities of juvenile myelomonocytic leukemia,” Blood, published online April 25, 2013.
In DBA, a mutation prevents a patient’s bone marrow from producing normal quantities of red blood cells, resulting in severe, sometimes life-threatening anemia. This basic fact makes it difficult for researchers to discern the underlying mechanism of the disease: “It’s very difficult to figure out what’s wrong, because the bone marrow is nearly empty of these cells,” said Bessler, the director of CHOP’s Pediatric and Adult Comprehensive Bone Marrow Failure Center.
CHOP researchers advance stem cell studies in a childhood leukemia and diamond blackfan anemia
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Weiss cautioned that this proof-of-principle finding is an early step, with many further studies to be done to verify if this approach will be safe and effective in clinical use.
“The technology for generating these cells has been moving very quickly,” said hematologist Mitchell J. Weiss, M.D., Ph.D., corresponding author of two recent studies led by The Children’s Hospital of Philadelphia (CHOP). “These investigations can allow us to better understand at a molecular level how blood cells go wrong in individual patients – and to test and generate innovative treatments for the patients’ diseases.”
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