The average stem cell count per unit in a recovered umbilical cord blood sample is 867 million stem cells. Right now, more than 150 research institutions and biotech companies around the world are researching stem cell expansion to increase the number of cells available within each unit. Though stem cell transplants are an effective treatment for a variety of blood diseases and immune deficiencies, increasing the number of cells in a cord blood sample would provide valuable new treatment options to doctors and patients.
Clinical Trials
In order to make stem cell expansion available to your sample, this new technology must be approved by the FDA. Three phases of clinical trials are required by the FDA before any new drug or medical treatment can be approved for use in the general population. These clinical trials evaluate the effectiveness and safety of new treatments and are strictly monitored to protect the safety of participants. Ultimately, only 5% of all new drugs and treatments successfully complete all three trials and gain FDA approval.
- Phase I – Treatment is applied to a small number of subjects(20-80) to evaluate safety, dosage range, and possible side effects.
- Phase II – Treatment is applied to a larger group of subjects (100-300) and is used to collect data on safety and refine research methods for succeeding trials
- Phase III - Treatment is extended to a larger group of subjects (1,000-3,000) to confirm effectiveness, evaluate side effects and compare the treatment to those already in standard use. Less common side effects are more likely to be detected with a larger number of subjects being tested.
- Phase IV - Trials in this phase can be conducted after the treatment is approved for use in the general public to evaluate the treatments long-term risks, benefits and optimal use and to test the treatment on populations that were not included in the first initial phases.
Currently, stem cell expansion studies have reached Phase III of clinical trials. However, no company has yet gained FDA approval.
For additional information on the FDA and clinical trials:
www.FDA.gov/fdac/features/2003/503_trial.html
www.ClinicalTrials.gov
The Science of Expansion
Stem cells have the capability to self renew and differentiate. Research has been focused on identifying the mechanisms that control this process. Though several elements have been identified in this process, self-renewal in stem cells is not completely understood. The major challenge of stem cell expansion is increasing the number of undifferentiated stem cells in a sample. Undifferentiated cells give rise to specialize cells. Stem cells that are undifferentiated have not yet taken on the identity of another cell and are necessary for successful engraftment in any transplant.
Currently, some researchers use culture approaches to proliferate the number of undifferentiated stem cells in a sample. Culture approaches for stem cell expansion utilize cytokines and stroma cells. Cytokines are basically a wide variety of regulatory proteins produced and secreted by cells that are used to communicate with other cells. These proteins can signal renewal in a stem cell culture. Stoma cells are non blood cells that are derived from blood organs, such as bone marrow and fetal liver that are capable of supporting the growth of blood cells.
Successful Expansion in the United States
Regenetech, Inc
Utilizing a 3D cell culture/growth model developed by NASA for microgravity cell expansion, this Texas based company has technology capable of expanding a quantity of viable stem cells by 107 times, or more. The expansion can occur in a relatively short period of time and the resulting stem cells have no chromosomal mutations. The companies primary research is focused on the heart and pancreas (diabetes), while continuing research efforts on the liver, skin, muscle, bone and cartilage
To read more on Regenetech
www.regenetech.com
Cytomatrix
This Massachusetts based biotechnology company is working on a more natural approach stem cell expansion without the use of chemicals. Their Cytomatrix® grows and replicates cells three fold in three dimensions without the use of cytokines. This Cytomatrix® provides an environment similar to the inside of the body for cells to multiply. This research may lead to treatments for illness' such as heart attacks, diabetes, cirrhosis, and the repair of damaged tissue throughout the body.
To read more about Cytomatrix
www.cytomatrix.com
Availability – Will this technology be made available to my sample?
A common question is whether or not stem cell expansion will be made available to your sample once it is approved by the FDA. The answer is YES! Stem cell expansion will be made available to your sample. To put this in perspective, could you imagine a world in which penicillin was only made available to a fraction of the population who needed it for medical treatment? Neither can we. Companies are researching expansion to increase treatment options for doctors and patients everywhere.
What is Cord Partners doing to prepare for stem cell expansion?
In anticipation of amplification, Cord Partners stores your sample in dual chamber Med-Sep cryobags. These cryobags preserve your sample in a 5ml compartment and a 20ml compartment designed to accommodate expansion. This means, the 5ml compartment can be accessed and expanded without disturbing the 20ml bag.
Summary
Stem cell expansion will greatly increase the treatment potential of your cord blood sample. Over 150 companies are researching and developing this technology. Though progress has been made, no company has yet gained approval from the FDA. When this technology is finally approved for use in the general population it will be made available to your sample.
Additional Reading
http://www.vrc.nih.gov/research/hsc_summary.pdf
http://www.medicalnewstoday.com/printerfriendlynews.php?newsid=23934
http://stemcells.nih.gov/info/scireport/chapter5.asp
Works Cited
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