What is Cell Therapy?
Cell therapy is the use of human cells to prevent and treat medical disorders. Cell therapies now encompass areas such as stem cell therapy, gene therapy, immunotherapy, tissue engineering, regenerative medicine, drug delivery, and cancer vaccines.

Initially reported in the New England Journal of Medicine, the clinical benefits of stem cell therapy in the treatment of cancer have been established over the past three decades. (Thomas ED, Lochte HL, Lu WC et al., Intravenous infusion of bone marrow in patients receiving radiation and chemotherapy, New England Journal of Medicine (1957), 257; 491-496.). Stem cells are used to produce all the functional mature cell types found in normal organs of healthy individuals.

Progenitor cells are cells that have already developed from stem cells, but can still produce one or more types of mature cells within an organ. The objective of stem cell therapy is to restore a patient's hematopoietic system, which is comprised of and generates the cells of bone marrow, blood and the immune system, following chemotherapy and radiation therapy. This is accomplished through the infusion and subsequent engraftment of healthy, processed cells to replace damaged or diseased bone marrow and to promote the rapid recovery of neutrophils and platelets in patients" blood and immune systems. We expect that new sources of blood and marrow stem cells from unrelated donors, mismatched donors, and umbilical cord blood will further increase the application of this therapy for treating cancer.

Emerging cell therapies currently in development aim to:

• prevent solid organ transplant rejection;
• treat infectious and auto-immune diseases;
• prevent the recurrence of cancer;
• correct genetic deficiencies; and
• provide patients with access to cell and tissue regenerating products.

Current Cell Therapies
The most common types of cell therapy, used with regard to blood and platelet transfusions, have been used for many decades. Since the 1970s, bone marrow, and then later blood and umbilical cord-derived stem cells, have been used to restore bone marrow and blood and immune system cells damaged by chemotherapy and radiation therapy used to treat many cancers. According to the International Bone Marrow Transplant Registry, approximately 40,000 blood and marrow transplants were performed worldwide in 2000. According to Registry data, approximately 18,000, or just under half, of these procedures were performed in North America.

Established cell therapies for treating cancer (both solid tumors and hematological disorders) are also widely used and in most cases, are considered the standard of care. These therapies involve the collection of cells from a patient about to undergo chemotherapy or radiation treatment, or from a suitably matched donor. The cells require processing and storage in a sophisticated, high-quality cell therapy laboratory, followed by the delivery of the processed cells for infusion into the patient subsequent to chemotherapy or radiation therapy. Transplantation of these cells in this manner is known as stem cell therapy. As stated in the New England Journal of Medicine, stem cell transplantation is regarded as the enabling treatment for high-dose chemotherapy and radiation. (Thomas ED, Lochte HL, Lu WC et al. Intravenous infusion of bone marrow in patients receiving radiation and chemotherapy, New England Journal of Medicine (1957), 257; 491-496.).

While there are approximately 300 stem cell transplant laboratories in the United States (most of which are affiliated with, or part of major clinical centers), we estimate that fewer than a dozen service enough patients to be cost effective and able to meet increasingly stringent regulatory requirements. It is possible that many smaller transplant centers may find it difficult or even possible to operate cost effectively given the new requirements as established through 21 CFR 1271. In addition, the regulatory and physical environment required for new cell therapies beyond stem cell transplantation, i.e., therapies based on the use of dendritic cells, gene therapy and manipulated T-cells, etc are placing ever increasing demands on stem cell laboratories.

Therefore, PCT capabilities are tailored to address these needs:

• hematopoietic stem cells used for blood and marrow stem cell transplantation collected and distributed through a nationwide network using qualified transportation systems;
• pre-clinical development research including outsourcing of cell therapy manufacturing for clinical trials;
• long-term stem cell storage including umbilical cord blood stem cells; and
• consulting in the areas of GMP and GTP compliance, clinical trials design, process optimization and manufacturing.

The Future of Cell Therapies
As of 2005, the value of the collective cell therapy market was estimated at $26.6 billion. Projections for 2010 and 2015 see this figure increase to $56.2 billion and 96.3 billion respectively. (Cell Therapy-Technologies, Markets, and Companies)

The FDA intends to regulate cell-based therapies in a manner that is similar (but not identical) to the manner in which it regulates drugs and other pharmaceutical agents (FDA Regulation 21 CFR, Part 1271). However, unlike pharmaceutical-based therapies, cell-based therapies must meet a unique set of manufacturing and distribution requirements to comply with FDA standards and accommodate their limited biologic shelf life and consider the important difference from traditional pharmaceuticals since the processing of cellular tissue involves a biological donor. Therefore, the Donor Eligibility requirements, specifically established to help prevent the spread of communicable diseases from the donor to the recipient, have been established and represent a key element of the GTP rules.

Progenitor Cell Therapy, LLC has created a service-based business model that enables and optimizes cell-based therapies from pre-clinical development through clinical trials and commercialization.

PCT's business model offers product-based companies ready access to experienced and qualified personnel and facilities that shorten the time for the overall "drug” approval process, thereby reducing development cost. In addition, we leverage the cost of commercial scale facilities across several products and customers can reduce the capital risk and cost of manufacturing for the product-based company.

Unlike pharmaceutical drug developers, we have extensive experience in manufacturing and delivering cell-based therapies. Our facilities and personnel create value for our corporate clients that are developing a cell-based therapy by decreasing development time, optimizing the manufacturing process and saving capital that are usually used to build and staff cGMP facilities, for current and future clinical trials.