Exciting news has reached The Manila Times regarding the latest and most significant breakthrough in the field of stem cell therapy from the United States.
Stem cell therapy in its simplest definition is the medical use of stem cells to treat or prevent a disease or condition. While its most widely used form is known as the bone marrow transplant for leukemia patients, research going back a decade ago gave rise to more effective procedures for more wide-ranging diseases, which unfortunately came with just as much controversy.
From the moral and ethical issues over the use of human embryonic stem cells, to alarm over the safety of later alternatives in fresh or live cell therapy, usually from sheep, the potential of this highly effective procedure has generally been impeded from helping sick people all over the world.
Thankfully, researchers over at San Diego’s Allele Biotechnology and Pharmaceuticals, headed by chief executive officer Dr. Jiwu Wang—who earned his Ph.D. in Molecular Biology from the University of Southern California (USC)— persisted in finding what they believe to be the best, safest and most ethical way of administering stem cell therapy beginning with one’s own skin cells.
In an interview with The Manila Times, Dr. Wang said that he and his team worked and improved upon Nobel Prize Winner Shinya Yamanaka’s discovery of reprogramming mature cells into pluripotent stem cells.
“To have pluripotent stem cells means having a source of cells capable of changing into any type of cell like the lung cells, liver cells neurons, pancreatic beta cells, and so on,” Dr. Wang explained.
From there, Allele discovered a more efficient way to reprogram cells since research began in 2009, a process which Allele Biotechnology has since been able to file for multiple patents under the company’s name.
Findings throughout this period of development have in fact been so promising that the research has received funding from the federal government, which has long been very careful in providing assistance to stem cell therapy related research, precisely for the many controversies its kind has raised through the years.
Best of all, Allele’s stem cell therapy is already in the process of generating personalized stem cells under the high standard termed current Good Manufacture Practice, or cGMP, defined by the USA FDA aimed for application in clinical cell therapy just as they began banking cells from patients this August in their San Diego laboratories.
Below is The Manila Times Q&A with Dr. Jiwu Wang, which should provide the most accurate understanding of this new method of administering stem cell therapy, and more importantly which diseases will benefit this treatment most.
How long has it taken Allele to discover the science of reprogramming cells and developing a new way of administering stem cell therapy?
Allele has been working on this type of self-induced Pluripotent Stem Cells, or iPSCs, since 2009, two years after Dr. Yamanaka reported that human adult cells could be reprogrammed into pluripotent stem cells. Allele’s researchers have published our own method first in 2012, followed with several papers to show how these cells can be used, and more importantly for the clinical development at this stage, be generated and manufactured under cGMP, which means extremely high quality for clinical use in patients. Allele’s cGMP plant was built between 2015 and 2017, already validated and the personal iPSC production is underway.
What makes the use of a patient’s own skin cells effective in treating his/her disease?
In most cases, we consider that the stem cells created from one’s own skin cells by reprogramming—like I said from Yamanaka’s principle but in an improved, method with Allele’s research—are the optimal cells to be used for cell-replacement therapy. This is because these “autologous” stem cells are perfectly matched for immune compatibility and thus will not be rejected by the body after transplantation. All other “off-the-shelf” cells, including non-human and human allogeneic stem cells, are the products of compromise.
In the Philippines, stem cell treatment is generally construed as an anti-aging procedure. However, what are the more significant conditions that would greatly benefit from this kind of stem cell therapy?
The conditions that would benefit most from stem cell therapy are the “degenerative” diseases in which healthy cells have been either lost or significantly reduced in patients’ body. Some examples of these diseases are diabetes, liver diseases, heart dysfunctions, Parkinson’s disease, stroke, Alzheimer’s disease, spinal cord injury and amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease). In each disease, cells such as pancreatic beta cells (diabetes), hepatocytes (liver disease), cardiomyocytes (heart disease), dopaminergic neurons (Parkinson’s), cortical neurons (stroke and Alzheimer’s), motor neurons (spinal cord injury and ALS), oligodendrocytes (spinal cord injury) and astrocytes (ALS) are degenerated. To cure or ameliorate these conditions, we seek to perform “regenerative” medicine, in which we will differentiate the patient’s own stem cells into the appropriate cell type and then transplant differentiated cells into his/her ailing body.
With this branch of medicine often controversial, it is worthy to note that your research has been given federal grants. How was this possible?
The previous generation of stem cell biology was controversial because it used “embryonic” stem cells that were harvested from aborted tissues and in-vitro fertilized embryos. Allele specializes in the new generation of stem-cell therapy, which produces “reprogrammed” stem cells from skin cells. This approach causes no controversy in the US, and this is why Allele had no trouble competing and winning US federal grants in this field. Allele’s continued ability to win highly competitive US National Institutes of Health grants is the result of the quality of the Allele scientific team and the nature of Allele’s iPSC work.
In as far as securing an FDA approval for eventual clinical therapy, how close is Allele toward that goal?
We recently formed a partnership with another biotech company, which has obtained an FDA permit for clinical trials for ALS cell therapy. This will be our first significant step toward the realization of stem-cell therapy using our cells. We expect that we will be performing several clinical trials by ourselves as well as with additional partners within the next one to five years.
As Allele launches the cell banking component of the treatment this month, what kind of patients do you expect to receive at the San Diego clinic?
Patients with diabetes, heart problem, or eye conditions.
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About Dr. Jiwu Wang
Dr. Jiwu Wang earned his Ph.D. degree in Molecular Biology at the University of Southern California (USC). His research focused on pre-MRA splicing, one of the most important molecular biology mechanisms related to many diseases including cancer. He was awarded a National Institutes of Health (NIH) postdoctoral fellowship and performed his postdoctoral training in RNA-related fields at the University of California at San Diego.
In 1999, through a number of NIH grants Dr. Wang and his colleagues established Allele Biotechnology and Pharmaceuticals Inc. It is a research-based company specializing in the fields of RNAi, stem cells, viral expression, camelid antibodies and fluorescent proteins. It was the first to bring cutting edge products in many fields, such as RNAi, FP, iPSC.
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About Allele Biotechnology
Allele Biotechnology and Pharmaceuticals, Inc. is a private, San Diego-based company that explores the procedures of biological processes in order to come up with technologies and products for biomedical research and therapy development.
Its mission is to increase accessibility to innovative molecular biology research tools by offering cutting-edge technologies in iproducts and fully integrated platforms.
Allele utilizes proprietary non-integrating cellular reprogramming methods to develop cell therapy and enable drug discovery through the use of human and non-human primate iPS cells, cGMP-grade human iPS cells and their derivatives. With additional expertise in genome modification and unique cell-based fluorescence sensors/reporters, Allele provides advanced cell and assay development solutions.
Allelle also has developed a wide variety of advanced research tools including superior fluorescent proteins, highly efficient luciferase assay substrates, genotyping kits, and camelid antibodies that have been validated for co-immunoprecipitation, histology, and flow cytometry. The company has also been a leader in the RNAi field with its patents in Pol 3 promoter-driven siRNA, shRNA, and miRNA (www.allelebiotech.com)