Belmont, MA-Researchers at Harvard-Affiliated McLean Hospital have been awarded a $1.9 million National Institutes of Health Director's Opportunity Award to continue their research into creating human induced pluripotent (iPS) stem cells using a method aimed at eliminating the risk of cancer and other problems associated with other options such as genome-integrating viral methods.
The three-year grant, made possible through the American Recovery and Reinvestment Act of 2009, was awarded to a research team headed by Kwang-Soo Kim, PhD, director of the Molecular Neurobiology Laboratory at McLean, and Robert Lanza at Stem Cell & Regenerative Medicine International (SCRMI), which is a joint venture with Advanced Cell Technology (ACT) of Worcester and CHA Biotech of Korea.
The joint venture involving ACT has been active in researching the use of these protein-induced pluripotent stem cells as a source of universal red blood cells and platelets for transfusion.
"This was really perfect timing for us because this work is a little bit beyond the capability of a single laboratory. It is technically demanding, labor intensive and expensive research," said Kim. "The award will support our continued research into iPS cell use that both avoids the risk of cancer growth and genetic dysfunction and provides a potential source of unlimited blood supply."
In 2009, Kim's team published an important study in the journal Cell Stem Cell, showing the ability to create stem cells out of human skin cells, eliminating the risk of cancer and removing some of the ethical concerns associated with embryonic stem cells. The paper described using proteins rather than genes or viruses to reprogram human skin cells, eliminating the risk of cancers or other abnormalities developing in the reprogrammed cells associated with the use of genes or viruses.
"The technique is hoped to result in the availability of the first clinic-ready human induced iPS cells, which could ultimately result in the development of treatments for a variety of illnesses, including Alzheimer's and Parkinson's," said Kim. "The technique of using a patient's own skin cells also eliminates the problem of immune rejection that is associated with using embryonic stem cells."
The researchers not only want to apply the technology for stem cell therapy but also for disease modeling, a process that would allow for their use in the laboratory to study disease mechanisms and in pharmaceutical companies to screen new medications.
"The ultimate goal is to use them in personalized cell therapy," explained Kim.
At the same time, the grant money will be used to see whether the iPS cells can be turned into blood lineage cells, including platelets. The SCRMI lab, headed by Lanza, has already shown the ability to make blood lineage cells from human embryonic stem cells.
If the same can be done from human iPS cells, it might be able to generate blood cells for certain patients so they can receive transfusions of blood and platelets from themselves, Kim said.
It would eliminate the problem of rejection and would provide blood lineage cells such as platelets that will be extremely useful in potentially life-threatening situations of blood shortages, he said.
A third goal of the research is to seek to make iPS cells from tissues of patients who have the rare universal blood types Rh- and O-, he added. "If we can make an unlimited amount of blood from these iPS cells, the blood could be used universally, particularly in crisis situations such as war," he said.
"Dr. Lanza's team has the world's top expertise in how to differentiate human embryonic stem cells or iPS cells to blood lineage cells," Kim said. "That is why they have combined with my lab, where we have the expertise to generate iPS cells using the protein method."
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