**Photo courtesy of Max Aguilera-Hellweg, M.D. of National Geographic
This website was orignially published in 2005 by National Geographic, but it has since been updated to reflect the latest scientific advances in the field of stem cell research. The interactive article page is informative and insightful, perfect for the layman to understand. The photographs are also eye-catching. Those interested in what research is currently being conducted and what the future holds for stem cells can find information here. There is also a discussion forum where one can see others' opinions on the various topics and voice their own. Take a look and tell us what you think!
The California Institute for Regenerative Medicine (CIRM) was created by California’s Proposition 71 in 2004 which authorized it to issue $3 billion in grants, funded by bonds, over ten years for embryonic stem cell and other biomedical research. It is claimed to be the world’s largest single supporter of research in stem cells. The incentives for California include royalties from new therapies, more jobs, and access to cheaper medicines for state residents.
In the current field of research, researchers are encouraged to acquire patents and this has raised the bar and drives researchers to avoid the field. CIRM aims to support collaboration by requiring grant recipients to donate the exclusive license to any insight or technology to a common patent supervised by a nonprofit organization. This will serve as a place where scientists can receive low-cost licenses for further research. Combined with this patent pool will be a prize incentive for companies that collaborate to produce a successful stem cell therapy. Not only will CIRM reduce the cost of research in stem cell therapy and encourage innovation, it will provide affordable medicine. The “shared prize model” eliminates 30%-40% of pharmaceutical industry revenue generated by wasteful marketing costs. It also does not fund research and development of duplicates to medicines that are out on the market. California hopes to reform the biomedical system by changing how government, medical researchers, and the private sector interact.
Do California taxpayers need assurances that they will benefit from any products developed from the research? The institutions getting grants have ties to drug companies have the public at doubt. There is potential for fear that the stem-cell effort could wind up benefiting private companies at taxpayers’ expense. It is almost necessary that the state requires that drugs developed by stem-cell researchers and their affiliated companies be affordable. However, it is essential for universities to have ties with biomedical companies to enhance the research effort. The real solution will come when the interactions between researchers, the government, and private companies. If CIRM is successful, it will pave the way for much more innovation in the biomedical world in the 21st century. A revolution in the biomedical field will be a major breakthrough in healthcare policy.
Paddock, Catharine. "Stem Cells May Provide Vaccine For Colon Cancer." Medical News Today. MediLexicon International Ltd, 8 Oct. 2009. Web. 15 Nov. 2009. <http://www.medicalnewstoday.com/articles/166696.php>.
The field of stem cell research is advancing, slowly but surely. While the changes in stem cell policy are (as of yet) small, they are paving the way for fantastic future applications and discoveries involving stem cells. One potential future use of stem cells is a vaccine preventing cancer.
The University of Connecticut Stem Cell Institute has shown that human embryonic stem cells injected into mice consistently promote an anti-tumor immune response against colon cancer cells. Other scientists have used stem cells in an attempt to vaccinate against cancer, but the University of Connecticut vaccine is the first to “trick” the host’s immune system into believing it had cancer and trigger a cancer-fighting response. In the past, scientists have attempted to target antigens, such as proteins, on the surface of the cancer cells. So far, the University of Connecticut’s study has only been performed in mice (with promising results), but in the future, who knows what will happen? Perhaps someday scientists will have the technology and knowledge to create a universal vaccine for cancer.
Cancer is the number-two cause of death in America. More than 500,000 Americans die from cancer each year. If a universal vaccine for cancer becomes a reality, more than half a million lives will be saved each year. An effective cancer vaccine would be significantly less expensive than providing for the millions of people fighting cancer each year. Billions of dollars could be saved annually. Funding for a universal cancer vaccine makes economic sense. Now the technology (and regulatory practices in the US) just needs to catch up.
Lovell-Badge, Robin. "The future for stem cell research." Nature 414. (2001): 88-91. Web. 15 Nov 2009. Article Link.
Stem cell research has come a long way in the last decade. In the future, scientists look toward times when stem cells can be used to regenerate organs, facilitate regrowth of neural tissue in devastating neurodegenerative diseases like Alzheimer’s and Parkinson’s and as a treatment therapy for diabetes. With such a hopeful future and with everything that has already been discovered, there are still tremendous strides to be made – especially in such a politically charged, ethically sensitive arena.
Among several research elements still in need of more investigation is the use of single cells versus the use of a population of stem cells. In fact, one of the major limitations of recent experiments is that they are rarely performed with single cells. By using a population of cells, a scientist is testing a number of different cells, all carrying various potentials. By using a single cell, a scientist will be able to determine if that specific cell carries an unusual potential to differentiate into some tissue type (as opposed to whether a number of cells in a population have low to no probability of differentiating into that same tissue type). The solution for identifying single cells? Better stem cell markers.
And what about identifying optimal environments that facilitate stem cell differentiation? There are several components that determine ‘self renewal’ of a cell and its path towards determination – mainly membrane contact and influence of growth factors. If scientists could control for these factors, they could manipulate the ratio of stem to differentiated cells, as well as the rate of cellular proliferation. This caveat is problematic partly because scientists don’t yet fully understand the cellular mechanisms of embryonic stem cells – a wall that will transiently prevent the advance of any cell-based therapy.
As previously mentioned, molecular mechanisms of stem cells and their genetic properties continue to elude scientists. In fact, what are the cellular mechanisms underlying embryonic stem cells? How are these cells related to cancer cells? What molecular factors influence cellular differentiation? What if scientists could identify specific genes that permitted self-renewal and proliferation, or genes that promoted differentiation? These questions are so vast, yet we are forced to look to the future of stem cell therapy and research to yield the answers …
And what if we begin to answer these questions? The future of stem cell research will be in regenerative, cellular-based therapy. Scientists can further study neurodegenerative disorders, limb regeneration and reprogrammable cells. If it weren’t for policy, ‘evil’ scientists might turn toward human cloning and non-medicinal therapies. For this reason, and for the ambiguity withheld in the future of stem cells, research and policy will have to proceed hand-in-hand – as long as they share a collaborative relationship that promotes innovation and novelty within the field.
Now that Annie, Vaishali, Courtney and I have given you a background on not only the biological processes and history of stem cell research, but also the political and ethical concerns surrounding the research and therapies of such biotechnology, we would like to now shift the focus of this blog to the meat of the matter: examining the current and future roles that stem cells have in science and health care. For example, what is stem cell technology used for today? What potential future does it hold not only in our country but also the world? How does this technology fit into our current health care system, FDA and NIH regulatory and governing bodies, as well a future business potential?
The article/Fox News Video story I chose as one of my last posts is about a girl named Chloe who suffered from a stroke while still in the womb. As a result, she suffers from Cerebral Palsy. Her parents, the Levine’s, noticed symptoms when Chloe was around nine months old. As she grew, her motor and speech skills seemed increasingly odd and the symptoms became more apparent (such as rigid and weak motion in one side of the body and speech pathology). Only when they took her to a specialist, they realized how severe these deficits were and what the cause was. Initial treatment included physical, speech and occupational therapy. The family looked into all their options to offer Chloe the best treatments and therapies possible.
The Levine’s, being forward-thinking and well-prepared, had banked Chloe’s umbilical cord blood around the time of her birth. Umbilical cord blood, especially one's own, is a viable source of healthy young stem cells absent of viruses or chemicals. The chances of rejection of one's own cord blood are nearly zero. According to David Zitlow of the Cord Blood Registry, “umbilical cord cells are smart – once they are re-infused into the body, they migrate to the exact spot of injury and help the damaged cells or tissue repair itself.”
With Chloe, they took stem cells from her banked umbilical cord blood and re-infused it back into her via IV transfusion, followed by a 4-hour fluid flush. Within days, her family noticed her symptoms taper. Chloe is part of a pre-clinical trial at Duke University, examining the effectiveness of umbilical cord blood in treating Cerebral Palsy in children.
I really like this article because it is a perfect example of why it should be mandatory that hospitals bank children’s umbilical cord blood for future use. The potential for this technology is seemingly endless. Moreover, this is truly something everyone can support: it is a painless procedure, the only associated costs are storage and retrieval, and it is ethically sound (compared to embryonic stem cell research as discussed in our previous posts). This blood can be used as a natural alternative to chemical therapies and should not just be thrown away! It only seems right that our government fund research on the potential of umbilical cord blood stem cells, as this technology is not particularly controversial and is supported by many, such as scientists, health care workers, policy makers, and those opposed to other types of stem cell research.
-Margeaux Berroth Brown University Class of 2011 Human Biology Concentrator
Shortly after President Obama's inauguration in January of this year, the FDA approved the world's first clinical trial for a therapy derived from human embryonic stem cells. This trial will include 8 – 10 participants with severe spinal cord injuries. Stem cells are to be injected into the damaged site 1 – 2 weeks after the injury, as this is the optimal time frame believed to facilitate this type of therapy. Geron, a California based biotechnology company, announced the clearance of the clinical trial. This approval marked a research milestone. Just months before in March 2008, the trial was rejected on the basis that the proposal ‘lacked sufficient data.’ Others like to argue that there was severe ‘political overhang’ from the Bush administration.
The trial has been deemed Phase I – aimed mainly at testing the safety of the therapy. Despite the fact that Geron had identified 7 medical centers across the country that expressed interest in participating in the trial (pending approval through their Institutional Review Boards), there is still some uncertainty about whether this is an ‘appropriate’ trial to conduct. If the therapy is unsafe or does not work, there might be a public backlash against the scientific community – halting any existing embryonic stem cell research and severely crippling the field.
In defense of the notion that this first trial might not be successful, Geron argues that the purpose of the trial is safety, not efficacy. Though practical, this statement is unnerving because the scientists don’t know what results they will yield from the experiment. At best, the participants will experience some restoration of nerve tissue surrounding their spinal cord damage. At worst, the trial will be a disaster. For the sake of the scientific community, let’s hope for the former.
Geron's proposal of 22,000 pages discussed the success of the therapy in small animal models and evidence that the cells did not form tumors in these animals - one of the main concerns surrounding the use of embryonic stem cells. What about the total cost of the application and preclinical research? $45 million. For a company that has not yet been profitable, this trial is risky to say the least. But maybe it is risk and ingenuity that are necessary to get this type of research off the ground.
The approval of this trial was a remarkable benchmark set forth by the U.S. government and scientific community. Though Geron has put its company’s future in an uncertain place, it is such innovative investigation and pioneering attitude that will further the research of embryonic stem cells as a treatment therapy. I think this work is incredibly interesting and powerful. Powerful enough to change the minds of adversaries to stem cell research? This will depend on the outcome of the trial … as will the future of embryonic stem cell research, the future of Geron, the future of policy in the United States, and the future of those patients who would probably do anything for a cure.
Stem cell therapies, while still limited by legal and ethical restrictions, have advanced by leaps and bounds since the field’s inception. One of the most exciting advances in the field occurred recently: the combination of gene and cell therapy.
To treat adrenoleukodystrophy (ALD), a genetic disease that destroys the myelin coating of nerve fibers in boys’ brains, French scientists disabled the HIV virus so that it could not cause AIDS and used it to transplant bone marrow. Bone marrow transplants stop ALD by allowing new stem cells that can form myelin to take root. The problem with curing ALD, prior to now, has been finding a vector with which to transport the marrow. HIV is a disease that permanently invades all cells, making it a perfect vector.Scientists were able to genetically correct the boys’ bone marrow stem cells and implant them in the body by infecting them with deactivated HIV. Two years later, the boys’ brain damage shows no signs of worsening and 15% of their cells now produce the beneficial proteins received in the stem cell transplant.
What does this mean for the future of medicine? If this treatment can be replicated, then many similar diseases to ALD could also be halted – or even cured. The technological ability to correct patients’ bone marrow stem cells and return them to the patient via deactivated HIV is incredibly promising for treatment of cancer, as well as various genetic diseases. While ALD is too rare for a cure to have much sway in the current healthcare debates, the possibility of a cure for some strains of cancer is tantalizing. A cure for cancer would save hundreds of millions of dollars in Medicare and Medicaid every year. And if there’s one thing that lawmakers today can agree on, it is that we need to find ways to save money in these hard economic times. There is still a huge divide between policymakers on the ethical ramifications of stem cell research, but financially, stem cell research makes sense.
