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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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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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Failed J&J/Pfizer Alzheimer's drug was hitting target – studies

Posted: Published on September 11th, 2012

* Significantly cut levels of Alzheimer's related proteins * Findings suggest drug may work in earlier-stage disease * Dose, side effects may be an issue By Julie Steenhuysen CHICAGO, Sept 11 (Reuters) - Data from two large studies of Pfizer Inc and Johnson & Johnson (TLO: JNJ-U.TI - news) 's Alzheimer's drug, bapineuzumab, show the treatment reduced underlying markers of the disease in some patients, suggesting the failed medication might work at an earlier stage. The findings, presented at a European neurology meeting in Stockholm, followed the companies' announcement last month that they were scrapping large-scale clinical trials of the drug after it failed to improve memory or thinking skills in patients with mild to moderate Alzheimer's. Many researchers had long expected bapineuzumab to fail this test because they believe Alzheimer's starts years before memory problems become apparent. But they have been eagerly awaiting the so-called biomarker results - measurements of fluids and tissues in the body - to see if the drug hit its biological targets, suggesting it could work in at an earlier stage of the disease. These biomarker results show that compared with the subjects who were give a placebo, bapineuzumab significantly reduced the amount of the … Continue reading →

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Calif. company, its president plead guilty in growth hormone case

Posted: Published on September 11th, 2012

By the CNN Wire Staff updated 9:22 PM EDT, Mon September 10, 2012 STORY HIGHLIGHTS (CNN) -- A California company and its president pleaded guilty Monday to federal charges related to the distribution of human growth hormone, the Justice Department said. DNP International Company, Inc., of Santa Fe Springs pleaded guilty to three counts, including a felony charge for the wholesale distribution of a prescription drug and two misdemeanor charges, the department said in a news release. Owner David Ji, 50, of Irvine, pleaded guilty to a felony count of distributing prescription drugs in interstate commerce without a license, it said. "When he pleaded guilty (Monday), Ji admitted that his company had distributed an unapproved drug containing somatropin -- a human growth hormone product -- which had not been approved for distribution in the United States by the Food and Drug Administration because the version of the drug being distributed by DNP was manufactured in China," the release said. The company and Ji entered their pleas before U.S. District Judge George H. King, who set sentencing for December 10. Ji faced up to 10 years in federal prison and a $250,000 fine. DNP faces a $900,000 fine. But his lawyer, … Continue reading →

Posted in Wholesale Pharmacy | Comments Off on Calif. company, its president plead guilty in growth hormone case

Calif. treasurer's wife regains child visitation

Posted: Published on September 11th, 2012

By GILLIAN FLACCUS Associated Press SANTA ANA, Calif. (AP) - The estranged wife of California Treasurer Bill Lockyer has regained visitation rights with her 9-year-old son while she's facing charges of drug possession and child endangerment. An Orange County judge on Monday granted a modified protective order allowing Nadia Lockyer visitation and telephone calls with the boy after her husband agreed to the changes. Nadia Lockyer pleaded not guilty last month to felony and misdemeanor drug charges after police acting on a tip allegedly found methamphetamine in a home where she was visiting relatives. Nadia Lockyer is a former Alameda County supervisor who resigned after a string of bizarre public incidents she blamed on chemical dependency. She's out on bail on condition that she remain in a drug treatment program. Prosecutors say she could eventually qualify for a diversion program. Copyright 2012 The Associated Press. All rights reserved. This material may not be published, broadcast, rewritten or redistributed. Read the original: Calif. treasurer's wife regains child visitation … Continue reading →

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Ex-Alameda Co. Supe Facing Drug Charges Regains Child Visitation

Posted: Published on September 11th, 2012

Nadia Lockyers arrest photo. (Orange County District Attorneys Office) SANTA ANA (CBS / AP) The estranged wife of California Treasurer Bill Lockyer has regained visitation rights with her 9-year-old son while shes facing charges of drug possession and child endangerment. An Orange County judge on Monday granted a modified protective order allowing Nadia Lockyer visitation and telephone calls with the boy after her husband agreed to the changes. Nadia Lockyer pleaded not guilty last month to felony and misdemeanor drug charges after police acting on a tip allegedly found methamphetamine in a home where she was visiting relatives. Nadia Lockyer is a former Alameda County supervisor who resigned after a string of bizarre public incidents she blamed on chemical dependency. Shes out on bail on condition that she remain in a drug treatment program. Prosecutors say she could eventually qualify for a diversion program. (Copyright 2012 by CBS San Francisco. All Rights Reserved. This material may not be published, broadcast, rewritten, or redistributed.) Excerpt from: Ex-Alameda Co. Supe Facing Drug Charges Regains Child Visitation … Continue reading →

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Patient-founded DART Therapeutics names CEO, other execs

Posted: Published on September 11th, 2012

Gene Williams, co-founder and CEO of DART Therapeutics LLC Monday, September 10, 2012 DART Therapeutics isnt your ordinary therapeutics business. The Cambridge therapeutics startup focused on treatments for Duchenne muscular dystrophy(DMD), a fatal neuromuscular disorder with no current treatment affecting one in 3,500 boys worldwide, has made several new executive appointments to its leadership team. DART Therapeutics LLC has appointed its co-founder and executive chairman, Gene W. Williams as its CEO. In addition, the company has named Ernest Bush as chief scientific officer; Dr. Elliot Goldstein as its chief medical officer; and Cabot Brown as chief financial officer. Founded in 2010 by Williams and patient foundations in DMD, the Nash Avery Foundation and Charleys Fund, DART is a patient-funded biotech business focused on DMD and other rare diseases. Charleys Fund was founded by Tracy and Benjamin Seckler, parents of Charley Seckler who was diagnosed with DMD in 2004. The Nash Avery Foundation was created by Tom Wicka, whose son Nash was diagnosed with DMD. The patient-advocate groups created DART to make DMD research and drug development faster and less expensive, according to Seckler, Charleys dad and president of Charleys Fund. The families took matter into their own hands when they … Continue reading →

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Patient-founded DART Therapeutics names CEO

Posted: Published on September 11th, 2012

Gene Williams, co-founder and CEO of DART Therapeutics LLC Monday, September 10, 2012 DART Therapeutics isnt your ordinary therapeutics business. The Cambridge therapeutics startup focused on treatments for Duchenne muscular dystrophy(DMD), a fatal neuromuscular disorder with no current treatment affecting one in 3,500 boys worldwide, has made several new executive appointments to its leadership team. DART Therapeutics LLC has appointed its co-founder and executive chairman, Gene W. Williams as its CEO. In addition, the company has named Ernest Bush as chief scientific officer; Dr. Elliot Goldstein as its chief medical officer; and Cabot Brown as chief financial officer. Founded in 2010 by Williams and patient foundations in DMD, the Nash Avery Foundation and Charleys Fund, DART is a patient-funded biotech business focused on DMD and other rare diseases. Charleys Fund was founded by Tracy and Benjamin Seckler, parents of Charley Seckler who was diagnosed with DMD in 2004. The Nash Avery Foundation was created by Tom Wicka, whose son Nash was diagnosed with DMD. The patient-advocate groups created DART to make DMD research and drug development faster and less expensive, according to Seckler, Charleys dad and president of Charleys Fund. The families took matter into their own hands when they … Continue reading →

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Rare form of autism might be treatable

Posted: Published on September 11th, 2012

Researchers have identified a rare, hereditary form of autism that might be treatable. The announcement came after identifiying a similar genetic mutation among six children. The children, who lack an enzyme that prevents essential amino acides from being depleted,are all of Middle Eastern descent, and each has parents who are first cousins. Nature writes that according to the research,Mice lacking this gene developed neurological problems related to autism that were reversed by dietary changes. Although scientists are not sure why lacking this enzyme causes autism, they have found that the mice were treated within days of receiving a diet enriched in branched-chain amino acids. Nature reports that although the discovery shows potential for new treatment options, it is not definitive: This might represent the first treatable form of autism, says Joseph Gleeson, a child neurologist at the University of California, San Diego, who led the study. That is both heartening to families with autism, and also I think revealing of the underlying mechanisms of autism. He emphasizes, however, that the mutations are likely to account for only a very small proportion of autism cases. We dont anticipate this is going to have implications for patients in general with autism, says … Continue reading →

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