Major Obstacle to Stem Cell Therapy Overcome in Vivo by ARIAD's Gene Regulation Technology
Animal studies demonstrate broad clinical applications of ARGENT stem cell therapy.
CAMBRIDGE, Mass., Sep 5, 2000 (BUSINESS WIRE) -- ARIAD Pharmaceuticals, Inc. (Nasdaq: ARIA chart, msgs) today announced that one of the most formidable obstacles to the clinical development of stem cell therapy - the inability to deliver genes to a therapeutically relevant number of stem cells - has been overcome using the ARIAD Regulated Gene Expression Technology (ARGENT(TM)) system.
In a paper published in the September issue of Nature Genetics, a team of ARIAD collaborators from the University of Washington, led by C. Anthony Blau, M.D., achieved this breakthrough by genetically engineering bone marrow stem cells with the ARGENT cell-growth switch, transplanting them into mice, and controlling their in vivo growth and differentiation with ARIAD's small-molecule Dimerizer Drug(TM).
The Dimerizer Drug activates the cell-growth switch, which results in substantial amplification of the population of genetically modified bone marrow cells. This produces a large number of specialized cells (e.g., red blood cells) that incorporate the desired therapeutic gene. Even using state-of-the-art gene transfer techniques (without ARGENT), only approximately one percent of stem cells can be genetically modified -- far too low to achieve beneficial therapeutic effects.
Dr. Blau, associate professor of medicine (hematology) at the University of Washington, commented, "I anticipate that our results will have important implications for the treatment of serious inherited blood cell disorders. This approach also may have direct applications in the treatment of a wide range of other diseases, especially in view of recent advances in stem cell biology. Our immediate goal is to move toward clinical studies with ARGENT stem cell therapy as quickly as possible."
Treatment of inherited blood cell disorders, such as beta-thalassemia (the most common genetic blood disease in the world) and sickle cell anemia, are among ARIAD's initial stem cell therapy product applications. None of the current therapies for these diseases are curative, which creates a substantial market opportunity for innovative products that address the underlying genetic basis of these diseases.
"The new NIH guidelines for stem cell research announced in late August greatly expand the opportunities for developing stem cell therapy products. I expect that we will benefit from these new initiatives. We have ongoing collaborative programs involving the use of the ARGENT cell-growth switch to control many important cell types, including neural cells (for Parkinson's disease), muscle cells (for heart failure), liver cells (for liver failure), and pancreatic cells (for diabetes)," said Harvey J. Berger, M.D., chairman and chief executive officer of ARIAD.
The paper by Jin et al is entitled, "In vivo selection using a cell-growth switch." Additional information on stem cell research and the NIH guidelines can be found at the websites for the National Institutes of Health (www.nih.gov/news/stemcell/index.htm) and the American Association for the Advancement of Science (www.aaas.org/spp/dspp/sfrl/projects/stem/main.htm).
ARIAD Pharmaceuticals, Inc. (www.ariad.com) is a leader in the discovery and development of gene therapy, cell therapy, stem cell therapy and protein therapy products featuring dose-dependent regulation by small-molecule drugs, as well as small-molecule inhibitors of signal transduction.
Some of the matters discussed in this news release are forward-looking statements that involve risks and uncertainties, which include, but are not limited to, risks and uncertainties regarding the Company's preclinical studies, the ability of the Company to conduct clinical trials of its products and the results of such trials, as well as risks and uncertainties relating to economic conditions, markets, products, competition, intellectual property, services and prices, key employees, future capital needs, dependence on our collaborators and other factors under the heading "Cautionary Statement Regarding Forward-Looking Statements" in ARIAD's Annual Report on Form 10-K for the fiscal year ended December 31, 1999 filed with the Securities and Exchange Commission. |
