Lee: Public stem cell funding would aid U.S. market

Human embryonic stem (ES) cell research will revolutionize how myriad diseases are treated, but it would also have huge ramifications for the trillion-dollar healthcare industry. However, for the past seven years, the progress of human ES cell research in America has been stifled because of the restrictions that have been placed on how public funding can be used.

Recent rhetoric from political leadership has threatened to completely derail the meager progress of human ES cell research in America — many want to stifle the progress of the research by advocating a complete ban on both public and private funding under the belief that human ES cell research has become unnecessary.

Yet the past few years have seen rapid advancement in the field of stem cell research. Induced pluripotent stem cells (iPS), which resemble human ES cells, have captured the attention of the public as well as the scientific community. Like ES cells, they apparently can develop into any functional cell type in an organism. The discovery of iPS cells is exciting, but they are by no means mutually exclusive nor do they provide an alternative to human ES research. (The discovery of human iPS cells could not have occurred without the last decade of human ES cell research.)

Still, much work needs to be done before human iPS cells can replace human ES cells. Currently, human iPS cells can only be made by utilizing viruses and overexpressing genes that are often associated with cancers. That is, current iPS cells are unfit to ever be used for therapeutic purposes. Furthermore, there are genomic differences between iPS cells and ES cells that are unaccounted for. Many leading scientists will instead agree that the new iPS cell discoveries are complementary and synergistic and a not substitutive to human ES cell research.

Human ES cells are still the “gold standard” and benchmark for assessing the ability of cells to develop into all the functional cells in an organism. Only human ES cells (and not human iPS cells) have the current potential to eradicate the chronic use of toxic drugs for many diseases and the potential to cure what were once thought to be incurable and grossly disabling illnesses.

Treatments for diseases such as diabetes will be radically transformed by human ES cell research. Such research will make it possible to create transplantable beta secreting insulin cells, eliminating the need for anti-diabetes drugs like insulin. This will have a tremendous impact on not only the way diabetes is treated, but also on the way the $100 billion metabolism market (which includes diabetes drugs) does business.

The importance of human ES cell research to the trillion-dollar healthcare industry cannot be overlooked. America has been instrumental in creating the current healthcare industry, and every major financial institution has invested in it heavily.

Countries like Singapore are realizing the financial potential of human ES cell research, and they have promoted such research by building gigantic publicly funded biomedical research centers.

Therefore, public funding of human ES cell research in America is vital if we want to continue to dominate the biomedical field. Biotech companies based on human ES cell therapies currently find it difficult to raise capital because the horizon in which investors want to see returns on their investment is presently unknown or thought to be too long.

Publicly funding the research is the only way around investor insecurity. Besides, the first therapies that result from human ES cell research are guaranteed to be billion dollar discoveries. Given the importance of the healthcare industry to sustaining America’s now-floundering economy, the nation can not afford to let other countries take the forefront of human ES cell research.

America’s current restrictions already placed on public funding of human ES cell research in America have stifled the advancement of the field for far too long. To stifle it further by promoting a ban on even private funding of the research would be tragic. Human ES cell advancements will happen in Europe or Asia if they don’t happen here. Allowing another country to take the helm would be a monumental mistake, especially if it happens because of premature and erroneous conclusions concerning recent iPS cell breakthroughs.

Throughout world history, advancements in science have been met with public resistance—science will always incite controversy. But the most hotly debated discoveries have often turned out to be the most important, rewarding and lasting. (In recent times, many people thought that cloning genes was controversial, but now undergraduates are conducting this epochal procedure in their biology lab courses.)

As the nation graples with its financial crisis and focuses on how to spend money more shrewdly, here’s an idea: to fund human ES cell research more liberally. Doing so would be a prudent measure that would pay huge dividends in the future.

Diverting a hundred-million dollars of public funds toward human ES cell research would benefit the health and economy of America, unlike certain publicly-funded infrastructure projects that lead to nowhere. Human ES cell research is a bridge to a better economy, a better healthcare system and a better life for all Americans.

Comments

  • Eli

    Let the private sector fund it.

    Government funded research is notoriously inefficient, wasteful and comparatively unproductive while the meager results are poorly managed.

  • Dan

    The private sector has failed to cure anyone since polio; since then, the move has been to turn every ailment into a chronic disease meant to fuel ongoing pharmaceutical sales and doctors' treatments. The Federal government should be pursuing this as a means of eliminating some of this costly waste (e.g. chronic disease treatment), but for the past 8 years, has largely been hampered by failed Federal policy and lack of coordination among states which actually do fund this research. The ideal candidate to guide policy on this issue would be the U.S. Department of Health and Human Services, but don't expect anything with the current leadership.

  • BVM

    Han Lee claims: "Currently, human iPS cells can only be made by utilizing viruses and overexpressing genes that are often associated with cancers. That is, current iPS cells are unfit to ever be used for therapeutic purposes."

    Just last week, a team at Harvard University, led by Konrad Hochedlinger, published their success in iPS research using a non-cancer-inducing adenovirus, which was able to change an adult cell back into its embryonic form. The adenovirus does not merge its genes with the host's, unlike the old methods to which Han Lee objects, and therefore the new technique does not have the cancer risk.

    Source: "Scientists Find Way to Regress Adult Cells to Embryonic State" by Rob Stein, in the Washington Post. (Date: Sept. 25, 2008) (Link: http://www.washingtonpost.com/wp-dyn/content/article/2008/09/25/AR2008092502099.html)

    Source: "Stem Cells without Side Effects", by Lauren Gravitz, in MIT publication Technology Review (date: Sept 25, 2008) (Link: http://www.technologyreview.com/Biotech/21430/)

  • Anonymous

    I believe you have been misled by mass media BVM. If you were to read the actual published scientific article by Konrad Hochedlinger, you will see that there were genetic manipulations that are done in order to get iPS cells using adenoviruses (i.e. inducible Oct4). Genetic manipulations that will never be allowed in cells used for human therapies.

    The efficiency at which iPS cells are produced even with this genetic manipulation is very low using adenoviruses. Furthermore, a high percentage of these cells are of a tetraploid background, which is not seen with previous virus manipulations (retro or lenti). This is an even worse side effect than what has been observed using retro or lenti.

    The investigators also say that adenoviruses temporarily express high levels of the transcription factors, in most cases without integrating permanently into the genome. The key word here is in most cases. Therefore the risk is still there.

    I believe Han Lee is absolutely correct in his assessment of viruses still not being ideal for therapeutic purposes. If one were to decide between ES cells that have not been genetically manipulated and infected with viruses that in most cases should not integrate into the genome as opposed to iPS cells with all these potentially cancer causing effects, choosing ES cells for therapeutic reasons is the wise choice.

  • a stroke survivor

    It's sad that blogs like this are being spread across the internet because a president. It should be about the latest discovery about stem cells without the stupid restrictions placed on the research by a fool of a president. Right now I'm 48 years old my stroke robbed me of some of my eyesight 6 years ago and perversily I have more insight to the promise that this stem cell research holds than a president. Sadly politics has needlessly stoped any chance of a therapy and people have needlessly died because of a president that is more blind than me.

  • BVM

    The following is what Harvard University released about the Hochedlinger-group's results. As mentioned in the final paragraph, the research was partially funded by NIH (U.S. federal tax dollars).

    Title: Important new step toward producing stem cells for human treatment
    Date: September 25, 2008
    Author: B. D. Colen, Harvard News Office, Harvard University
    Link: http://harvardscience.harvard.edu/foundations/articles/important-new-step-toward-producing-stem-cells-human-treatment

    A team of Harvard Stem Cell Institute (HSCI) scientists has taken an important step toward producing induced pluripotent stem (iPS) cells that are safe to transplant into patients to treat diseases.

    Excitement over the ability of researchers to create this form of stem cell by inserting four genes into adults cells has thus far been tempered by the fact that the genes have been inserted using retroviruses, which have the potential to turn on cancer genes and trigger tumor growth.

    But today Konrad Hochedlinger and HSCI colleagues at Massachusetts General Hospital and Joslin Diabetes Center report having created mouse iPS cells using harmless adenoviruses that ultimately disappear from the new cells and therefore do not integrate into their DNA like the retroviruses.

    “The adenoviruses infect the cells” carrying the genes needed for cellular reprogramming, “but are cleared by the cells after a few cell divisions,” said Hochedlinger, an assistant professor in Harvard’s new inter-school Department of Stem Cell and Regenerative Biology (SCRB). “This wouldn’t be harmful in any way because the DNA of the new cells remains unaffected,” he said.

    The report by the Hochedlinger group appears in today’s on-line edition of the journal Science.

    Harvard Stem Cell Institute co-director Doug Melton, who is also co-chair of SCRB, said “this paper by the Hochedlinger’s group is another in a series by one of the leading labs, whose work is aimed at making stem cells from patient samples. In this advance, the use of viruses that integrate into the host genome have been eliminated, making the process easier and the end product, the induced pluripotent stem cell, that much more safe.”

    It has previously been believed that the viruses carrying the four essential transcription factors had to be integrated into the genome of the target cell in order for adult cells to be reprogrammed into pluripotent stem cells, “but we’ve shown that you don’t need integration of the virus into the genome to produce iPS cells,” Hochedlinger said.

    Hochedlinger and colleagues Mathias Stadtfeld – the study’s lead author, Masaki Nagaya, Jochen Utikal, and Gordon Weir – head of HSCI’s Diabetes Program, have used the new technique to create iPS cells from mouse skin cell, and mouse fetal and adult liver cells.

    “We get stem cell lines,” said Hochedlinger. “They are all pluripotent” – meaning that they can become any type of cell –“and they have no traces of the adenovirus.” Even more important, he said, thus far none of the mice carrying the new cells lines have shown any signs of developing tumors – and tumors were being frequently reported in mice carrying the cell lines created using retroviruses.

    “The next step is to reproduce this work using human cells, and there’s no reason why it can’t work,” Hochedlinger said, adding that “this basically provides us with a system with which to test the question of whether iPS cells are the equivalent of human embryonic stem cells. That’s a question that, in my opinion, hasn’t been answered yet.”

    As Hochedlinger and his colleagues have been working to find viral substitutes for the use of retroviruses in the production of iPS cells, some HSCI researchers are reported to be looking for chemicals that might be used in place of viruses, and some of those experiments are said to be quite promising.

    The work reported today was supported by grants from the Harvard Stem Cell Institute, and by a National Institutes of Health New Innovator Award Hochedlinger received last year.

    [end of Harvard statement]

  • Dan#2

    "The private sector has failed to cure anyone since polio; since then, the move has been to turn every ailment into a chronic disease meant to fuel ongoing pharmaceutical sales and doctors' treatments."
    Perhaps this is because the diseases of aging (e.g. cancer, Alzheimer's, diabetes) are inherently tougher to treat. When there's a pathogen, we can target the pathogen and cure the disease. But the diseases of aging are complex, and it takes more than an antibiotic to cure them.

  • Anonymous

    I don't mean to discount the exciting advances that have been made with adenoviruses BVM. But however, the key phrase here is important new steps.

    The important finding in this research is that only temporary expression of certain genes are needed to produce iPS cells and not the continous expression of the genes. This shows that continous and prolonged expression of the factors are not needed once the cells have acquired ES cell characteristics. This makes searching for chemical compounds which can mimic the actions of these factors even more important now that only transient activation is needed of the pathways that these genes are a part of.

    The press release you are reading is cleverly written and again I reiterate, is mass media. If a company were to go to the FDA and show this press release and claim that iPS cells are safe, this company would get laughed at. These experiments were done in mouse cells. This is a very important delineation. Human ES cells are significantly different from mouse ES cells. Furthermore the conditions to get human iPS cells are significantly different from the ones needed for mouse iPS cells. Just because the experiment works in mouse cells does not mean it will work in human cells. The results can be vastly different which I hope not.

    The bottom line is that genetic manipulations were used in this exciting new paper. This genetic manipulation was needed in order to get iPS cells using adenoviruses. Plus, just because viral integration of the adenoviruses was not seen in this case, it does not mean it is not possible. The experiments were done in a controlled environment and the sample size is much too low for one to even claim this method is good enough for therapeutic purposes. Assessing 13 lines is not good enough to say that viral integration will never happen. When that number increases to thousands as in any FDA study, viral integrations will occur just by chance alone as numerous papers on adenoviruses have shown. The chance is still there. Plus the other side effects of adenoviruses which are not observed using genome integrating viruses such as retro or lenti-viruses.

  • Anonymous

    "As mentioned in the final paragraph, the research was partially funded by NIH (U.S. federal tax dollars)."

    Yes, this research was partially funded by the NIH. But these are MOUSE iPS CELLS AND NOT HUMAN iPS CELLS. THIS IS A HUGE DIFFERENCE. There is no controversy in using NIH funds for mouse ES cell research.

    The public must know that human iPS cells are significantly from mouse iPS cells. The only reason why investigators were able to discover the conditions needed for producing human iPS cells was because of the privately funded human ES cell research that has been occurring for the last 7 years. If it were not for the private funding, we might not be able to produce human iPS cells despite being able to produce mouse iPS cells.