AEGiS-PRn: Aastrom Biosciences Awarded Two NIH Grants for Umbilical Cord Blood Transplants and AIDS Gene Therapy; Grants Support Development of New Applications for Aastrom's Enabling Technologies for the Transplantation and Gene Modification of Human Stem Cells PRNewswireImportant note: Information in this article was accurate in 1997. The state of the art may have changed since the publication date.
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Aastrom Biosciences Awarded Two NIH Grants for Umbilical Cord Blood Transplants and AIDS Gene Therapy; Grants Support Development of New Applications for Aastrom's Enabling Technologies for the Transplantation and Gene Modification of Human Stem Cells

PR Newswire, 810 Seventh Avenue, New York, NY 10019 - Tuesday October 7 8:40 AM EDT


ANN ARBOR, Mich., Oct. 7 /PRNewswire/ -- Aastrom Biosciences, Inc. (Nasdaq:ASTM) announced today that it was awarded two grants from the National Institutes of Health (NIH), totaling approximately $460,000. A Phase I Small Business Innovation and Research (SBIR) grant, received from the National Heart, Lung and Blood Institute (NHLBI), supports Aastrom's further development of processes for the expansion of therapeutic stem and progenitor cells sourced from umbilical cord blood (UCB), using the Aastrom(TM) Cell Production System (CPS). The second, a four year grant, received from the National Institute of Diabetes and Digestive and Kidney Disease (NIDDK), supports the development of a novel AIDS therapy involving optimized gene transfer into human stem cells, through the use of Aastrom's patented stem cell replication and gene loading technologies.

"These grants reflect support by the NIH of the clinical relevance of Aastrom's core product technologies," said Walter C. Ogier, Vice President Marketing at Aastrom. "The umbilical cord blood transplant grant marks recognition of the Aastrom(TM) CPS' potential expanded utility for stem cell therapy in treating patients with leukemia and related types of cancer. The AIDS grant recognizes the potential complementary value of the Aastrom(TM) CPS and the Aastrom(TM) Gene Loader technologies to enable a promising new stem cell gene therapy for AIDS."

The goal of the research to be carried out under the NHLBI grant is to develop novel processes for the production of stem and progenitor cells sourced from the UCB remaining in the placenta after childbirth. UCB is highly enriched in human stem cells and has been used to achieve potentially curative stem cell transplants over the past four years in several hundred patients, most of whom are children. The new technique, which is being increasingly used in place of bone marrow or blood stem cell transplantation, may overcome the limitations of immune system incompatibility between donor and patient for the treatment of many forms of leukemia and other malignant blood disorders.

The widespread clinical use of UCB transplantation is hampered by the comparatively small total quantity of cells available from a placenta. The Aastrom(TM) CPS represents a potentially enabling technology for cord blood transplantation through the production of a large quantity of UCB cells sourced from a placenta. This capability may increase the number of patients who could benefit from UCB transplantation, including offering the procedure to adult cancer patients as well as children.

Umbilical cord blood transplantation is being practiced by an increasing number of hospitals in North America and Europe, as an alternative to more conventional methods of bone marrow transplantation. The NIH recently funded the establishment of three new cord blood banks at Duke University, Children's Hospital of Orange County (California), and the University of California, Los Angeles, to support practicing hospitals with transplantable cells. The world's largest UCB bank, at the New York Blood Center, has been in operation for five years and maintains more than 6,000 UCB collections for use in UCB transplantation worldwide.

A primary goal of the second grant, awarded by the NIDDK, is to confer human resistance to HIV infection by placing cell destruction genes into the stem cells of AIDS patients. Aastrom will collaborate with Gail Harrison, Ph.D. and Elizabeth J. Shpall, M.D. at the University of Colorado to achieve this goal. If the gene transfer research is successful, it may potentially lead to a clinical trial of a new "suicide" gene therapy. When infected by the AIDS-causing virus, the patient's otherwise normal cells would activate the cell destruction gene and die before the AIDS virus could replicate, potentially interfering with the progression of the disease. AIDS patients would receive transplants of gene modified cells.

"Many gene therapy programs are stalled at the laboratory bench because they are unable to achieve efficient transfer of the therapeutic gene into target human cells," said Alan K. Smith, Ph.D., Vice President Research at Aastrom. "This problem is further compounded when the target cells are stem cells, which do not ordinarily replicate outside the body and thus do not offer the opportunity for new genetic material to be integrated into the DNA of the cell. Aastrom's(TM) Gene Loader technology has been shown to overcome a major obstacle in transferring genes into cells, while the Aastrom(TM) CPS enables gene integration into the DNA of stem cells by enabling ex vivo stem cell replication. We are enthusiastic about the application of these complementary technologies to achieve the therapeutic goals of the AIDS project."

Gene therapies are being developed by biopharmaceutical companies and university laboratories worldwide. These therapies are based on the principle of inserting a normal gene into a human cell or tissue to correct a genetic defect, or inserting a novel gene to cause a desired pharmacological or biological effect. In ex vivo gene therapy, these procedures are performed outside the body, and the gene modified cells or tissue are then returned to the patient to achieve therapeutic benefit.

Aastrom Biosciences, Inc. is pioneering the development and commercialization of proprietary medical product systems, including the Aastrom(TM) CPS and Aastrom(TM) Gene Loader. These technology platforms are expected to enable the practice of stem cell therapy and other cell and gene therapies at clinical sites worldwide. In 1993, Aastrom entered into a marketing alliance with COBE BCT, a world leader in blood cell processing products, to assist Aastrom with the distribution and customer service of the Aastrom(TM) CPS for stem cell therapy.

This document contains forward-looking statements, including without limitation statements concerning clinical trials, regulatory reviews and product commercialization programs, which involve certain risks and uncertainties. Actual results may differ significantly from the expectations contained in the forward-looking statements. Among the factors that may result in the differences are the results obtained from clinical trials, uncertainties associated with the regulatory approval process, competitive developments, and the availability of resources. These and other significant factors are discussed in greater detail in Aastrom's Form 10-K and other filings with the Securities and Exchange Commission.

SOURCE Aastrom Biosciences, Inc.

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