Researchers Continue to Look to Stem Cells for Clues for Effective Treatments of Orthopedic Trauma and Blood-Related Disorders

Jun 20, 2011   

This week, the scientific community announced two projects that may have important implications on the future of stem cell research and the development of effective treatments for orthopedic trauma and blood-related disorders. These projects signal a continued effort by researchers to improve our understanding of the bodys basic mechanisms for producing hematopoietic stem cells (HSCs), which are multipotent cells that have the ability to replenish all blood cell types. The U.S. Food and Drug Administration (FDA) regulates human cells or tissues intended for implantation, transplantation, infusion, or transfer into a human recipient, as well as the devices that process human cells and tissue. While the FDA has jurisdiction over these stem cells and has provided some guidance for related medical devices, its ability to regulate the industry is seemingly unmatched by the speed of scientific discovery and development. The continued research on stem cells and their potential therapeutic impact on patient care has pushed the boundaries of FDA regulation and has raised questions about the FDAs ability to keep up with science.

Arteriocyte, a clinical stage biotechnology company that develops novel stem cell products and medical devices, recently announced that it signed a Cooperative Research and Development Agreement (CRADA) with the United States Army Institute of Surgical Research (USAISR). The three-year CRADA, entitled “The Use of Concentrated Bone Marrow Aspirate from a Point-of-Care Device in Orthopaedic Trauma,” aims to investigate new stem cell-based therapies for orthopedic and battlefield-related injuries. Together, Arteriocyte and the USAISR will explore stem cell-based therapies for amputation prevention, burn debridement and post-surgical wound infection prevention.

The CRADA is designed to use Arteriocytes Magellan MAR01 system and NANEX technology. The FDA issued 510(k) clearance for Magellan, a bed-side medical device intended to rapidly produce platelet rich plasma from blood and bone marrow. The resulting tissue is rich in platelets and hematopoietic stem cells (HSCs), and can be applied to surgical cites to aid with tissue repair. The FDA has also approved the Magellan for a Phase I clinical trial of peripheral vascular disease, the obstruction of major arteries than can lead to ischemia of the limbs. NANEX technology is a biofunctional nanofiber-based 3-D scaffold that is designed to mimic the bone marrow environment. This technology is intended to rapidly produce HSCs. Arteriocyte is in the process of actively developing the NANEX technology for use in treatment of ischemic disease, identification and treatment of cancers of the blood system and rapid, high volume production of “universal-donor” red blood cells.

Researchers at the University of California, San Diego (UCSD) and the University of Massachusetts recently announced that they may have discovered the genetic mechanism and signaling pathway that controls the production of hematopoietic stem cells (HSCs). The research team believes finding the mechanism involved in creating cells during embryonic development is the next step toward creating blood stem cells for patient treatment.

The research study, which was published in Nature International Weekly Journal of Science, focused on a family of genes called Wnts, which have been identified as important to the process of embryogenesis. Researchers specifically isolated the Wnt16 gene and concentrated on examining its effect on production of HSCs. When researchers “turned on” Wnt16 in a population of zebrafish, whose blood is extremely similar to that of mammals, the expression of two types of ligands were triggered. Ten hours after fertilization, HSC production appeared in the zebrafish embryos. When Wnt16 was “turned off,” HSC production in the zebrafish was nonexistent. As a result, researchers believe Wnt16 is responsible for controlling a novel regulatory network for HSC production.

Researchers at UCSD and the University of Massachusetts are optimistic that continued research in the area of HSC production can have important implications for the development of blood stem cells and, eventually, stem cell-based therapies for blood disorders. This discovery adds to existing knowledge about HSC formation and the ways in which these types of cells communicate throughout the formative stages of blood cell production. “Were on the cusp of understanding something that people have been wondering about for decades,” said Wilson K. Clements, first author of the study and a postdoctoral fellow in UCSDs lab.

In spite of these new developments in the area of stem cell research and the scientific communitys prognostications about the therapeutic or curative potential of these stem cells, the FDA has only approved a few stem cell-based treatments, as we previously reported here and here. Even though the FDA has been cautious in outlining specific regulations for this burgeoning area of science, the continued research efforts to explore the mechanisms and value of stem cells may push the FDA to make difficult and more responsive decisions about its role in regulating this industry. Fuerst Ittleman will continue to monitor the progress and development of the FDAs regulation of HSC research and other stem cell-related devices and technologies. For more information, contact us at contact@fidjlaw.com.