 |
|
Intravenous nanoparticle gene therapy shows activity in stage IV lung cancer
April 18, 2007LOS ANGELES-A cancer-suppressing gene has been successfully delivered into the tumors of stage 4 lung cancer patients via an intravenously administered lipid nanoparticle in a phase I clinical trial at The University of Texas M. D. Anderson Cancer Center. The gene, FUS1, also was found to be active in the metastatic non-small cell lung cancer tumors.
"We've treated 13 patients in this first-in-human study and we've seen an exciting proof of concept with no significant drug-related toxicity," says principal investigator Charles Lu, M.D., associate professor in M. D. Anderson's Department of Thoracic, Head and Neck Medical Oncology.
Blinded analysis of pretreatment and post-treatment biopsies of three patients' tumors show that expression of FUS1 was absent from pretreatment samples while a high level of FUS1 was expressed in tumors after treatment. FUS1 can induce apoptosis-programmed cell death-in cancer cells but is frequently lost when normal cells become cancerous.
Lu presented a poster on the study on April 17 at the late-breaking abstract session of the American Association for Cancer Research annual meeting in Los Angeles.
Other attempts at gene therapy have employed an adenovirus to deliver the therapeutic gene. "Here we are using a non-viral, non-infectious delivery system," Lu says.
The only clinically significant side effect so has been fever, but Lu says premedication with a steroid and diphenhydramine has eliminated that so far.
Previous gene therapy clinical trials also involved direct injection into tumors. "This is the first time anyone has shown that a gene can be injected and then be taken up and expressed in cancer cells at distant sites," said Jack Roth, M.D., professor of the M. D. Anderson Department of Thoracic and Cardiovascular Surgery and a pioneer in the field of gene therapy.
FUS1 can induce apoptosis-programmed cell death-in cancer cells but is absent in those cells. The FUS1 nanoparticle formulation was developed and tested in Roth's lab. It advanced to phase I clinical trial after a promising test on human non-small cell lung cancer in a mouse model.
FUS1 was discovered by a research team led by Roth at M. D. Anderson and by John Minna, M.D., of the Department of Internal Medicine and Pharmacology, Hamon Center for Therapeutic Oncology Research, at The University of Texas Southwestern Medical Center at Dallas. Roth and Minna are the co-principal investigators of a National Cancer Institute Specialized Program of Research Excellence in Lung Cancer.
"As a clinician, I am very cautious about saying that we have shown clinical activity at this point. We have some encouraging data. The number of patients is too small to draw any definite conclusions, however," Lu said.
Three patients of eight who received two or more doses experienced stable disease for three to seven months. Median survival time for all patients is 14.6 months, which Lu notes compares favorably to a seven-month median survival time for patients receiving second line therapy.
All patients on the trial had been treated with front line cisplatin combination chemotherapy, which failed to halt their disease. The clinical trial continues. No maximum tolerated dose has been established, Lu says.
The nanoparticle delivery system consists of a plasmid gene expression cassette loaded with DNA that encodes the FUS1 protein. This is wrapped tightly in a form of cholesterol to protect it from the body's defense mechanisms. The nanoparticles accumulate mainly in the lungs, particularly in the tumors, where the genes repeatedly express FUS1 tumor-suppressing proteins.
Lung cancer is the leading cause of cancer death in the United States, causing 160,000 deaths annually. About 80 percent of lung cancer is of the non-small cell type.
University of Texas M. D. Anderson Cancer Center
Lu presented a poster on the study on April 17 at the late-breaking abstract session of the American Association for Cancer Research annual meeting in Los Angeles.
Other attempts at gene therapy have employed an adenovirus to deliver the therapeutic gene. "Here we are using a non-viral, non-infectious delivery system," Lu says.
The only clinically significant side effect so has been fever, but Lu says premedication with a steroid and diphenhydramine has eliminated that so far.
Previous gene therapy clinical trials also involved direct injection into tumors. "This is the first time anyone has shown that a gene can be injected and then be taken up and expressed in cancer cells at distant sites," said Jack Roth, M.D., professor of the M. D. Anderson Department of Thoracic and Cardiovascular Surgery and a pioneer in the field of gene therapy.
FUS1 can induce apoptosis-programmed cell death-in cancer cells but is absent in those cells. The FUS1 nanoparticle formulation was developed and tested in Roth's lab. It advanced to phase I clinical trial after a promising test on human non-small cell lung cancer in a mouse model.
FUS1 was discovered by a research team led by Roth at M. D. Anderson and by John Minna, M.D., of the Department of Internal Medicine and Pharmacology, Hamon Center for Therapeutic Oncology Research, at The University of Texas Southwestern Medical Center at Dallas. Roth and Minna are the co-principal investigators of a National Cancer Institute Specialized Program of Research Excellence in Lung Cancer.
"As a clinician, I am very cautious about saying that we have shown clinical activity at this point. We have some encouraging data. The number of patients is too small to draw any definite conclusions, however," Lu said.
Three patients of eight who received two or more doses experienced stable disease for three to seven months. Median survival time for all patients is 14.6 months, which Lu notes compares favorably to a seven-month median survival time for patients receiving second line therapy.
All patients on the trial had been treated with front line cisplatin combination chemotherapy, which failed to halt their disease. The clinical trial continues. No maximum tolerated dose has been established, Lu says.
The nanoparticle delivery system consists of a plasmid gene expression cassette loaded with DNA that encodes the FUS1 protein. This is wrapped tightly in a form of cholesterol to protect it from the body's defense mechanisms. The nanoparticles accumulate mainly in the lungs, particularly in the tumors, where the genes repeatedly express FUS1 tumor-suppressing proteins.
Lung cancer is the leading cause of cancer death in the United States, causing 160,000 deaths annually. About 80 percent of lung cancer is of the non-small cell type.
University of Texas M. D. Anderson Cancer Center
Gene Therapy Current Events and Gene Therapy News RSSResearch reveals lipids' unexpected role in triggering death of brain cells
The lipid that accumulates in brain cells of individuals with an inherited enzyme disorder also drives the cell death that is a hallmark of the disease, according to new research led by St. Jude Children's Research Hospital investigators.
No-entry zones for AIDS virus
The AIDS virus inserts its genetic material into the genome of the infected cell. Scientists of the German Cancer Research Center have now shown for the first time that the virus almost entirely spares particular sites in the human genetic material in this process. This finding may be useful for developing new, specific AIDS drugs.
Cornell researchers identify a weak link in cancer cell armor
The seeming invincibility of cancerous tumors may be crumbling, thanks to a promising new gene therapy that eliminates the ability of certain cells to repair themselves.
Treatment to improve degenerating muscle gains strength
A study appearing in Science Translational Medicine puts scientists one step closer to clinical trials to test a gene delivery strategy to improve muscle mass and function in patients with certain degenerative muscle disorders.
Iowa State University researcher discovers key to vital DNA, protein interaction
A researcher at Iowa State University has discovered how a group of proteins from plant pathogenic bacteria interact with DNA in the plant cell, opening up the possibility for what the scientist calls a "cascade of advances."
Scientists successfully reprogram blood cells
Researchers have transplanted genetically modified hematopoietic stem cells into mice so that their developing red blood cells produce a critical lysosomal enzyme -preventing or reducing organ and central nervous system damage from the often-fatal genetic disorder Hurler's syndrome.
Immune therapy can protect against or treat later lymphoma
Specially developed immune system cells that target the common Epstein-Barr virus can protect immune-suppressed bone marrow transplant recipients against lymph system disease and cancers that arise from the viral infection.
Caltech researchers show efficacy of gene therapy in mouse models of Huntington's disease
Researchers at the California Institute of Technology (Caltech) have shown that a highly specific intrabody (an antibody fragment that works against a target inside a cell) is capable of stalling the development of Huntington's disease in a variety of mouse models.
Immunotherapy demonstrates long-term success in treating lymphoma
Targeted immunotherapy has been an attractive new therapeutic area for a number of cancers because it has the potential to destroy tumor cells without damaging surrounding normal tissue. New study results demonstrate high success rates using specialized white blood cells to prevent or treat lymphoma associated with the Epstein-Barr virus (EBV-lymphoma) in patients who have received a hematopoietic stem cell transplant (HSCT).
Toward bold new anti-cancer medicines
Bold new strategies in the battle against cancer may turn forms of the disease that presently are incurable into manageable conditions that can be controlled for long periods of time.
More Gene Therapy Current Events and Gene Therapy News Articles
 |
Gene Therapy: Treating Disease by Repairing Genes (New Biology) by Joseph, Ph.D. Panno (Author) Many diseases are caused by a simple point mutation - they are the result of a single defective gene. Gene therapy is the attempt to replace a defective gene with a good copy or to add a good gene to compensate for the bad. Although this procedure has proven to be extremely hazardous, of all the technologies provided by the new biology, gene therapy holds much potential for curing disease. Dealing with one of the most controversial aspects of the new biology, Gene Therapy explores the development and future of this area of research and explains why it is considered such a promising branch of science. This encyclopedia presents an overview of genetic defects, viruses, and gene therapy in a way that students and general readers will find easy to understand. Comprehensive in scope, this book... |
 |
Gene and Cell Therapy: Therapeutic Mechanisms and Strategies, Third Edition by Nancy Smyth Templeton (Editor) Since the publication of the second edition of this book in 2004, gene therapy and cell therapy clinical trials have yielded some remarkable successes and some disappointing failures. Now in its third edition, Gene and Cell Therapy: Therapeutic Mechanisms and Strategies assembles many of the new technical advances in gene delivery, clinical applications, and new approaches to the regulation and modification of gene expression. New Topics Covered in this Edition: Gene and Cell Therapies for Diabetes and Cardiovascular Diseases Clinical Trials Human Embryonic Stem Cells Tissue Engineering Combined with Cell Therapies Novel Polymers Relevant Nanotechnologies SiRNA Therapeutic Strategies Dendrimer Technologies Comprised of... |
 |
Gene Therapy and Gene Delivery Systems (Advances in Biochemical Engineering / Biotechnology) by David V. Schaffer (Editor), Weichang Zhou (Editor) 1 D.V. Schaffer, W. Zhou: Gene Therapy as Future Human Therapeutics.- 2 J. Heidel, S. Mishra, M.E. Davis: Molecular Conjugates.- 3 M. Manthorpe, P. Hobart, G. Hermanson, M. Ferrari, A. Geall, B. Goff, A. Rolland: Plasmid Vaccines and Therapeutics: From Design to Applications.- 4 S.R. Little, R. Langer: Non-Viral Delivery of Cancer Genetic Vaccines.- 5 J.C. Grieger, R.J. Samulski: Adeno-Associated Virus as a Gene Therapy Vector: Vector Development, Production and Clinical Applications.- 6 J.H. Yu, D.V. Schaffer: Advanced Targeting Strategies for Murine Retroviral and Adeno-Associated Viral Vectors.- 7 N. Loewen, E.M. Poeschla: Lentiviral Vectors.- 8 N.E. Altaras, J.G. Aunins, R.K. Evans, A. Kamen, J.O. Konz, J.J. Wolf: Production and Formulation of Adenovirus Vectors.- |
|
Gene Therapy & Molecular Biology by Intl Soc Gene Therapy Mole Bio | |
 |
Gene Therapy by Mary Colavito (Author), Michael A. Palladino (Author) KEY MESSSAGE: Will gene therapy one day become the ultimate application of genetic technology? Gene Therapy, the newest booklet in the Special Topics in Biology Series, addresses this question by exploring gene therapy as a complex technology for delivering therapeutic genes as a way to treat and cure human genetic diseases. Author Mary Colavito provides an overview of the basic science involved in gene therapy methods and chronicles the history of gene therapy by discussing successful and ongoing gene therapy treatments as well as adverse outcomes in some cases of gene therapy. Also discussed are challenges that must be overcome for gene therapy to become a more reliable and readily accessible approach for treating a multitude of genetic diseases that affect... |
 |
Jazz Therapy, Vol. 1: Smile by Gene Bertoncini & Roni Ben-Hur The first in a new series of fundraising CDs produced in association with the Jazz Foundation of America, and the first time these two popular guitarists have recorded together. Liner notes by Ira Gitler. Entire CD was played, recorded, packaged, and is being promoted on a pro-bono basis, raising funds for the Englewood Hospital and Medical Center's Dizzy Gillespie Fund. |
 |
Gene Therapy: Dawn of a New Age in Medicine (NCME Video 653) Also With: Kenneth W. Culver (Primary Contributor), Network For Continuing Medical Education (Primary Contributor) |
 |
Six Years of Gene Therapy Son of Rust (Primary Contributor) |
 |
Chromosome 11 Gene Therapy Sickle Cell Anemia Photographic Poster Print, 24x32 by Art.com Art.com is the world's largest retailer of art prints, posters, photographs, and framed artwork. With our huge selection of over 400,000 prints, you'll easily find the perfect piece for your home, office, or classroom. Our art is printed on quality paper. When you order framed artwork, the piece is built by our team of in-house professionals. Visit our Amazon store today at www.amazon.com/artdotcom to find Special Offers and search for products based on 'Artist Name' and 'Subject Categories' such as Movie, Music, Vintage, TV, Children, Travel, Kitchen, Museum Art, Animals, Floral, Motivational, and Sports. Art.com is dedicated to providing you with high quality products and service by offering you 100% satisfaction guaranteed. We ship internationally to over 80 countries. Decorate your... |
 |
Charlie Rose (August 30, 2000) A rebroadcast of an interview that originally aired on June 21, 2000. Part three of the special edition series, "Mapping the Human Genome." First, three prominent scientists on how the decoding of the genome will lead to breakthroughs in the battle against disease. They are Dr. Harold Varmus, president of Memorial Sloan-Kettering Cancer Center, Dr. Arnold Levine, president of Rockefeller University, and Dr. Savio Woo, president of the American Society of Gene Therapy.||Then, Dr. James Watson, Nobel laureate, co-discoverer of the double-helix structure of DNA and president of Cold Springs Harbor Laboratory, discusses public and private genome projects and his new book, a collection of his essays over the years called A Passion for DNA. This product is manufactured on demand using DVD-R... |
| © 2009 BrightSurf.com |