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'Bubble babies' immune systems restored

Posted: Published on September 11th, 2012

LOS ANGELES UCLA stem cell researchers have found that a gene therapy regimen can safely restore immune systems to children with so-called "bubble boy" disease, a life-threatening condition that if left untreated can be fatal within one to two years. In the 11-year study, researchers were able to test two therapy regimens for 10 children with ADA-deficient severe combined immunodeficiency (SCID), which has come to be known as "bubble boy" disease because some of its victims have been forced to live in sterile environments. During that time, the researchers refined their approach to include a light dose of chemotherapy to help remove many of the blood stem cells in the bone marrow that were not creating the enzyme adenosine deaminase (ADA), which is critical for the production and survival of healthy white blood cells, said study senior Dr. Donald Kohn, a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA. The refined gene therapy and chemotherapy regimen proved superior to the other method tested in the study, restoring immune function to three of the six children who received it, said Kohn, who is also a professor of pediatrics and of microbiology, immunology … Continue reading

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Stem cell researchers use gene therapy to restore immune systems in 'Bubble Boy' disease

Posted: Published on September 11th, 2012

ScienceDaily (Sep. 11, 2012) UCLA stem cell researchers have found that a gene therapy regimen can safely restore immune systems to children with so-called "Bubble Boy" disease, a life threatening condition that if left untreated can be fatal within one to two years. In the 11-year study, researchers were able to test two therapy regimens for 10 children with ADA-deficient severe combined immunodeficiency (SCID). During the study, they refined their approach to include a light dose of chemotherapy to help remove many of the blood stem cells in the bone marrow that are not creating an enzyme called adenosine deaminase (ADA), which is critical for the production and survival of healthy white blood cells, said study senior Dr. Donald Kohn, a professor of pediatrics and of microbiology, immunology, and molecular genetics in Life Sciences and a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA. The refined gene therapy and chemotherapy regimen proved superior to the other method tested in the study, restoring immune function to three of the six children who received it, Kohn said. Going forward, an even further refined regimen using a different type of virus delivery system will … Continue reading

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After stem cell therapy, Arroyo thanks supporters

Posted: Published on September 11th, 2012

MANILA, Philippines Former President and now Pampanga Rep. Gloria Macapagal Arroyo thanked the public for their support as she undergoes medical treatment to address complications from a dislodged titanium implant along her cervical spine. Hi everyone. Thank you for your support, Arroyo posted on her official Twitter account on Tuesday. Arroyo also expressed her appreciation for the government doctors for allowing her to seek alternative medical care to address complications from her ailment. Replying to reporters queries on the micro-blogging site Twitter, Arroyo described the stem cell procedure she took on Monday. Procedure was not only intravenous injection of the stem cell material but also other naturalist treatment therapies. It has to be done under very hygienic conditions, she said. Arroyo said that after her stem cell treatment, she proceeded to her therapy at the Veterans Memorial Medical Center. Then I proceeded to my 3x weekly physical therapy at Veterans. I'm glad my Veterans' attending physicians recognize my right to seek alternative treatment and don't consider it in conflict with the conventional treatment they're giving me. In earlier tweets on Sunday, Arroyo explained to netizens that her stem cell therapy is not meant to address the mechanical problem directly but … Continue reading

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UCLA stem cell researchers use gene therapy to restore immune systems in 'bubble babies'

Posted: Published on September 11th, 2012

Public release date: 11-Sep-2012 [ | E-mail | Share ] Contact: Kim Irwin kirwin@mednet.ucla.edu 310-435-9457 University of California - Los Angeles Health Sciences UCLA stem cell researchers have found that a gene therapy regimen can safely restore immune systems to children with so-called "Bubble Boy" disease, a life threatening condition that if left untreated can be fatal within one to two years. In the 11-year study, researchers were able to test two therapy regimens for 10 children with ADA-deficient severe combined immunodeficiency (SCID). During the study, they refined their approach to include a light dose of chemotherapy to help remove many of the blood stem cells in the bone marrow that are not creating an enzyme called adenosine deaminase (ADA), which is critical for the production and survival of healthy white blood cells, said study senior Dr. Donald Kohn, a professor of pediatrics and of microbiology, immunology, and molecular genetics in Life Sciences and a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA. The refined gene therapy and chemotherapy regimen proved superior to the other method tested in the study, restoring immune function to three of the six children who received … Continue reading

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Improved nanoparticles deliver drugs into brain

Posted: Published on September 11th, 2012

ScienceDaily (Sep. 11, 2012) The brain is a notoriously difficult organ to treat, but Johns Hopkins researchers report they are one step closer to having a drug-delivery system flexible enough to overcome some key challenges posed by brain cancer and perhaps other maladies affecting that organ. In a report published online on Aug. 29 in Science Translational Medicine, the Johns Hopkins team says its bioengineers have designed nanoparticles that can safely and predictably infiltrate deep into the brain when tested in rodent and human tissue. "We are pleased to have found a way to prevent drug-embedded particles from sticking to their surroundings so that they can spread once they are in the brain," says Justin Hanes, Ph.D., Lewis J. Ort Professor of Ophthalmology, with secondary appointments in chemical and biomolecular engineering, biomedical engineering, oncology, neurological surgery and environmental health sciences, and director of the Johns Hopkins Center for Nanomedicine. After surgery to remove a brain tumor, standard treatment protocols include the application of chemotherapy directly to the surgical site to kill any cells left behind that could not be surgically removed. To date, this method of preventing tumor recurrence is only moderately successful, in part, because it is hard to … Continue reading

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Novelos Therapeutics Successfully Completes Second Cohort In Phase 1b Solid Tumor Trial With I-131-CLR1404 (HOT …

Posted: Published on September 11th, 2012

MADISON, Wis., Sept. 11, 2012 /PRNewswire/ -- Novelos Therapeutics, Inc. (NVLT), a pharmaceutical company developing novel drugs for the treatment and diagnosis of cancer, today announced that it has successfully completed the second cohort in a U.S. multi-center Phase 1b dose-escalation trial of its cancer-targeted molecular radiotherapeutic compound I-131-CLR1404 (HOT) in cancer patients with advanced solid tumors. The second two-patient cohort was successfully dosed with approximately 40 mCi of HOT, triggering enrollment into the third cohort at approximately 60 mCi. Details of the trial design are available on http://www.clinicaltrials.gov ID: NCT01495663, or at http://www.novelos.com in the 'Clinical Trials' section. Glenn Liu, M.D., Associate Professor of Medicine and Director of the Cancer Therapy Discovery and Development (Phase I) Program at the University of Wisconsin Carbone Cancer Center, is the trial's principal investigator. Detailed trial results are expected to be presented at a scientific venue at a later date. "Data from this second cohort indicates that HOT was well-tolerated, without any dose-limiting or sub-dose-limiting toxicities, enabling enrollment of the third cohort as planned," said Dr. Liu. "We continue to observe selective uptake of HOT in cancerous tumors where it persists for at least 21 days." "We are pleased with HOT's safety profile … Continue reading

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GMA tweets thanks to docs for allowing stem cell treatment

Posted: Published on September 11th, 2012

By: Lira Dalangin-Fernandez, InterAksyon.com September 11, 2012 7:21 PM InterAksyon.com The online news portal of TV5 MANILA, Philippines - Former President and now Pampanga Representative Gloria Macapagal-Arroyo said Tuesday she was thankful to her doctors at the Veterans Memorial Medical Center for recognizing her right to seek stem cell treatment for her medical condition. In her Twitter account (@gmarroyo), Arroyo said, "I'm glad my Veterans attending physicians recognize my right to seek alternative treatment and don't consider it in conflict with the conventional treatment they're giving me." Arroyo on Monday underwent her fourth stem cell intravenous treatment while still attending her weekly therapy at the VMMC. "Procedure was not only intravenous injection of the stem cell material but also other naturalist treatment therapies," she said in her Twitter account. "It has to be done under very hygienic conditions. Then I proceeded to my 3x (three times) weekly physical therapy at Veterans," she added. Earlier, she said her treatment would involve cultured stem cells, which would be "much more modest in price that the one coming from sheep or one's own body." Stem cell therapy involves the use of fresh cells injected into the body to regenerate cells to reverse aging … Continue reading

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UCLA stem cell researchers use gene therapy to restore immune systems in 'bubble babies'

Posted: Published on September 11th, 2012

Public release date: 11-Sep-2012 [ | E-mail | Share ] Contact: Kim Irwin kirwin@mednet.ucla.edu 310-435-9457 University of California - Los Angeles Health Sciences UCLA stem cell researchers have found that a gene therapy regimen can safely restore immune systems to children with so-called "Bubble Boy" disease, a life threatening condition that if left untreated can be fatal within one to two years. In the 11-year study, researchers were able to test two therapy regimens for 10 children with ADA-deficient severe combined immunodeficiency (SCID). During the study, they refined their approach to include a light dose of chemotherapy to help remove many of the blood stem cells in the bone marrow that are not creating an enzyme called adenosine deaminase (ADA), which is critical for the production and survival of healthy white blood cells, said study senior Dr. Donald Kohn, a professor of pediatrics and of microbiology, immunology, and molecular genetics in Life Sciences and a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA. The refined gene therapy and chemotherapy regimen proved superior to the other method tested in the study, restoring immune function to three of the six children who received … Continue reading

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Stem cell researchers use gene therapy to restore immune systems in 'Bubble Boy' disease

Posted: Published on September 11th, 2012

ScienceDaily (Sep. 11, 2012) UCLA stem cell researchers have found that a gene therapy regimen can safely restore immune systems to children with so-called "Bubble Boy" disease, a life threatening condition that if left untreated can be fatal within one to two years. In the 11-year study, researchers were able to test two therapy regimens for 10 children with ADA-deficient severe combined immunodeficiency (SCID). During the study, they refined their approach to include a light dose of chemotherapy to help remove many of the blood stem cells in the bone marrow that are not creating an enzyme called adenosine deaminase (ADA), which is critical for the production and survival of healthy white blood cells, said study senior Dr. Donald Kohn, a professor of pediatrics and of microbiology, immunology, and molecular genetics in Life Sciences and a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA. The refined gene therapy and chemotherapy regimen proved superior to the other method tested in the study, restoring immune function to three of the six children who received it, Kohn said. Going forward, an even further refined regimen using a different type of virus delivery system will … Continue reading

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Stem cell researchers use gene therapy to restore immune systems in 'Bubble Boy' disease

Posted: Published on September 11th, 2012

ScienceDaily (Sep. 11, 2012) UCLA stem cell researchers have found that a gene therapy regimen can safely restore immune systems to children with so-called "Bubble Boy" disease, a life threatening condition that if left untreated can be fatal within one to two years. In the 11-year study, researchers were able to test two therapy regimens for 10 children with ADA-deficient severe combined immunodeficiency (SCID). During the study, they refined their approach to include a light dose of chemotherapy to help remove many of the blood stem cells in the bone marrow that are not creating an enzyme called adenosine deaminase (ADA), which is critical for the production and survival of healthy white blood cells, said study senior Dr. Donald Kohn, a professor of pediatrics and of microbiology, immunology, and molecular genetics in Life Sciences and a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA. The refined gene therapy and chemotherapy regimen proved superior to the other method tested in the study, restoring immune function to three of the six children who received it, Kohn said. Going forward, an even further refined regimen using a different type of virus delivery system will … Continue reading

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