What Are Clinical Trials?
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What Are Clinical Trials?
A critical part in the process of bringing new therapies to patients is clinical development, the study of potential new therapies in humans.
It typically takes 12 years from the moment of discovery in the lab for a potential new therapy to gain approval for use and to reach patients. Clinical development is the most time intensive and expensive part of this research and development continuum accounting for approximately 45 to 75 percent of the $50 million average cost of bringing a new therapy to market.
Trials conducted during clinical development generate thousands of pieces of data and are generally used by regulatory agencies, such as The U.S. Food and Drug Administration (FDA) to determine approval for use of the therapy and in which medical indications it can be used.
Clinical trials involve hundreds to many thousands of volunteer trial participants. Every trial is governed using well-defined ethical rules and standards to protect participant safety.
For patients suffering from a condition that is currently untreatable, participation in a clinical trial may provide access to potential new therapies. While participating, volunteers obtain closely controlled care in research medical facilities, in addition to helping others by contributing to medical research.
Phases of Development
Clinical Development Process
An experimental therapy is first tested in the laboratory and in animal studies. After this “pre-clinical” testing, and only if shown to meet certain safety criteria and shown to have value as a potential new therapy, the therapy advances to clinical testing in humans. The Phases of Clinical Development (please select a Phase below to view).
WHO / WHAT REGULATES THE GIOSTAR/BIOSCIENCE AMERICAS STEM CELL TREATMENT PROTOCOL AND THERAPY?
There are two aspects to consider regarding the use of stem cell therapies in the U.S. and potential host countries outside of the U.S.
The first is the existing legal structure surrounding research and secondly, the regulatory framework regarding application of that research to treat patients.
The Global Institute of Stem Cell Therapy and Research (GIOSTAR) adheres to the guidelines promulgated by the U.S. Food and Drug Administration (FDA) under the U.S. Department of Health and Human Services, Code of Federal Regulation, Part 1271 entitled “Regulation of Human Cells, Tissues, and Cellular and Tissue Based Products”.
The FDA makes a clear distinction of minimally manipulated cells and autologous transplantation from other cell types when it states in Part 1271, that ‘you are not required to comply with the requirements of this part if you are an establishment that removes human cells, tissues and cellular and tissue-based products from an individual and implants such products into the same individual during the same surgical procedure’. What this implies is that minimally manipulated and autologous cell therapy should not have regulations that are in place for other human cells, tissues, tissue based products and drugs.
GIOSTAR follows Part 1271 for adult autologous stem cell transplant.
Adult HSC (Hematopoietic Stem Cell) transplants under practice and approved by US FDA over the last 30 years include the following examples of diseases:
- Acute myeloid leukemia (AML)
- Chronic myeloid leukemia (CML)
- Acute lymphoblastic leukemia (ALL)
- Hodgkin lymphoma (relapsed, refractory)
- Non-Hodgkin (relapsed or refractory) lymphoma
- Ewing sarcoma
- Multiple Myeloma
- Myelodysplastic syndromes
- Gliomas, other solid tumors
- Sickle Cell Anemia
- Aplastic anemia
- Fanconi anemia
- Immune deficiency syndromes
- Inborn errors of metabolism
Regarding patient application in our host countries, as indicated in a previous e-mail, prohibitions are generally in place regulating the use of embryonic stem cell. GIOSTAR does not conduct research or treat patients with embryonic stem cells.
As currently applied in Colombia, for example, patient therapies will be administered under FDA Part 1271 guidelines, as noted above, operating under the authority and license of the attending physician and associated medical professionals.