For stem cell case plaintiff, faith and science go hand-in-hand

Yesterday, we shared a Q&A with the lead plaintiffs in the controversial federal lawsuit challenging federal funding for embryonic stem cell (eSC) research, Dr. James L. Sherley, a biological engineer at Boston Biomedical Research Institute, and Dr. Theresa Deisher, research and development director at AVM Biotechnology LLC in Seattle.

Both researchers agreed to field questions about their beliefs regarding eSC research. Deisher, who has been especially vocal about her Catholic faith and how it informs her research, also agreed to take a few questions about the connection she sees between her beliefs and science.

According to Deisher’s bio, she has 17 years of experience in scientific and corporate leadership positions involving research, discovery, production and commercialization of human therapeutics. She obtained her Ph.D. in molecular and cellular physiology from Stanford University. Prior to founding AVM Biotechnology in 2007, Deisher held positions at Repligen Corp. in Cambridge, Mass., ZymoGenetics Inc., Immunex and Amgen in Seattle and CellCyte Genetics Corp. in Bellevue, Wash. She has had 23 patents issued and has published numerous scientific manuscripts.

ddn: Do you find any conflict between your faith and the scientific research you engage in?

Deisher: I do not find any conflict between my faith, which is Catholic, and my research. My faith enhances my work. My Christian faith calls me to focus on drugs and treatments that are affordable so that the greatest number of people will benefit. My faith calls me to use reason and the order of natural law to determine, for instance, the stem cell most optimal for clinical use. My faith calls me to focus only on those treatments that will be effective. My faith also calls me to respect the intrinsic dignity of human life in my work.

ddn: How does your faith impact your research approach?

Deisher: My faith is completely complementary to my research, which focuses currently on stem cells for regenerative medicine and alternative vaccines.

Adult “self” stem cells, meaning a patient’s own stem cells, are affordable, compared to all other stem cell therapies. For the most part, therapies using adult stem cells will cost about $25,000 compared to Geron’s projected $500,000 for embryonic stem cell-based therapies. Adult “self” stem cells are found naturally in every organ, in each of us, and they are “preprogrammed’” to perform the functional regeneration that patients require. They also lack the issues of immune rejection or tumor formation that plague pluripotent stem cells such as embryonic stem cells. Adult “self” stem cells are far advanced in clinical trials, and in comparison to “patented” stem cell lines, they show more effectiveness in patients. Whether one believes in God or Darwin, one can arrive at an optimal stem cell for patients using objective measures, common sense and business criteria to generate the greatest good for the most people.

I would apply these same criteria to any type of treatment that I would work on, including biologics and small molecules: Will the therapy be affordable, or will only the very few benefit? Will the therapy be effective or merely enhance my stock price or financing temporarily? Will the therapy be undermined by adverse side effects? These criteria are sound business objectives and compatible with my faith.

Q+A with the stem cell case plaintiffs

On the cover of our November issue (which you can download here), you can read about a controversial lawsuit that is at the center of one of our nation’s hottest current debates: embryonic stem cell (eSC) research. (See the story here.)

The lawsuit, Sherley, et al., v. Sebelius, et al., alleges that the order signed by President Barack Obama in March 2009 lifting a previous ban on federal funding for eSC research violates the Dickey-Wicker amendment, a 1995 law prohibiting the government from appropriating funds for research that involves the creation or destruction of human embryos for research purposes. The lawsuit also contends that President Obama’s order has intensified competition for government research funds—which are already in short supply—for researchers engaged in other kinds of stem cell research.

The case has been making its way through the courts for a while, but it made headlines in August when the U.S. District Court for the District of Columbia issued a preliminary injunction in the case, essentially bringing funding for embryo-destructive research to a screeching halt while the case is under consideration.

With the research community in uproar over the decision, less than a month later, the U.S. Court of Appeals temporarily suspended the injunction, arguing that it would cause irreparable harm to researchers, taxpayers and scientific progress while the case is appealed.

While Sherley v. Sebelius concerns the interpretation of law and competitive issues, the case has renewed the debate over the practice of stem cell research in the United States, a political and moral tug-of-war that has been waged for decades.

The debate centers on the creation of a human embryonic stem cell (eSC) line, which requires the destruction of a human embryo. Advocates of the pro-life movement are concerned with the rights and status of an embryo as an early-aged human life and equate eSC research with murder. Those opposing this view argue that these embryos are to be destroyed or stored for long periods of time past their viable storage life, and point out that stem cell research has the potential to dramatically alter approaches to understanding and treating diseases, and to alleviate suffering.

Science holds pros and cons for both sides of the moral debate. Adult stem cell research has achieved great levels of success and potential, but these stem cells are generally limited to differentiating into different cell types of their tissue of origin. Although some researchers are of the opinion that the differentiation potential of eSCs is broader than most adult stem cells, they may be rejected by the immune system—a problem that would not occur if a patient received his or her own stem cells.

Dr. James Sherley

As the case continues to play out in court, media attention is turning to the lead plaintiffs in the case, Dr. James L. Sherley, a biological engineer at Boston Biomedical Research Institute, and Dr. Theresa Deisher, research and development director at AVM Biotechnology LLC in Seattle. Although the pair admit they did not know each other prior to the case unfolding—they were reportedly “recruited” by attorneys seeking to challenge President Obama’s order—they both have certain beliefs about the ethics involved in eSC research, and strongly support a focus on adult stem cells.

Sherley, whose research focuses on the molecular and biochemical mechanisms of adult stem cells, has long been vocal about his opposition to human eSC research. He has described the position that eSCs hold the promise to cure or treat debilitating diseases “misinformation,” maintaining that adult stem cell research is “a viable and vibrant path to new medical therapies.” Sherley also once made headlines for protesting a decision by the Massachusetts Institute of Technology (MIT) to deny him tenure by going on a 12-day hunger strike. Sherley, who is African-American, has also publicly said he believes that MIT did not give him the freedom to challenge scientific orthodoxy the way the institution would have for a white colleague.

Dr. Theresa Deisher

Deisher, who works exclusively with adult stem cells, founded AVM Biotechnology “in response to growing concerns about the need for safe, effective, affordable and ethical medicines and therapeutic treatments,” with help from private donors. According to the company’s website, “The use of aborted fetal tissue and embryonic cells in the discovery, development and production of vaccines and pharmaceuticals … make it difficult for many physicians, pharmacists, scientists and healthcare professionals to navigate their fields of expertise without sacrificing their consciences.” You can see some of Deisher’s writing on the subject here.

As media scrutiny has intensified in the wake of the case, Sherley and Deisher have backed away from many interviews, but were gracious enough to take questions from ddn regarding their views about eSC research. Here are their responses:

ddn: What should the goal of stem cell research be?

Sherley: Like other NIH-funded research, the goal should be to increase our scientific knowledge about the natural world through the conduct of ethical scientific research towards the goal of improving human health.

Deisher: The goal should be to develop safe, effective and affordable therapies for human disease, adhering to the highest scientific ethical standards.

ddn: Are you against embryonic stem cell research as a practice, or the federal funding of it, or both? Why?

Sherley: Specifically, I wish to educate and alert the public that, first and foremost, human embryonic stem cell research is unethical according to pre-existing NIH guidelines for human subjects research; that its funding by NIH is illegal according to U.S. law as articulated by the Dickey-Wicker amendment; and that it continues to be misrepresented by many of its proponents who misstate its potential for providing medical advances and present it as if there were no alternatives, when if fact both adult stem cell research and traditional disease research are not only effective alternatives, but better ones in many respects.

Deisher: Human embryonic stem cell research does not meet the high ethical standards test. A human being is necessarily destroyed in the process of obtaining human embryonic stem cells, which is not an acceptable outcome according to U.S. human subjects guidelines. Additionally, the research is not necessary, another requirement of our human subjects guidelines, as superior alternatives are available using adult stem cells. According to human subjects research guidelines, even if parents give consent, research cannot be done if it is not necessary. Human subjects guidelines should be applied to all research, regardless of funding source, and should be applied to regulate research on human embryos.

ddn: What are viable alternatives to embryonic stem cells? How soon will they bring about therapies? What is their commercial potential?

Sherley: The viable alternatives are both adult stem cell research and traditional disease research. In particular, adult stem cell research addresses the development of new therapies based on repairing tissues or replacing tissues with regenerative cells. Such therapies based on adult stem cells are already a part of standard clinical practice. Bone marrow reconstitution with blood stem cells is one of the best known examples, but there is a large body of clinical research underway both to make blood stem cell treatments even more effective and to develop new therapies based on other types of adult stem cells. This clinical research includes commercial development of therapies for diseases like diabetes based on developing adult stem cells that renew the cells that make insulin. It is important that the public understands that proposed treatments based on human embryonic stem cells invariably require that they be converted into either adult cells or adult stem cells, which are ultimately required for any therapy that will repair, replace, or treat tissues in children and adults. When the public knows this, it then becomes imperative for it to ask proponents of human embryonic stem cell research, “What is your motivation?”

Deisher: Adult stem cell therapies are in late-stage clinical trials, having advanced extremely rapidly since their discovery in the late 1990s, entering clinical trials by 2002.  Prior to the late 1990s, scientific dogma held that adult stem cells did not exist outside of the blood system. These novel discoveries have brought promise to many patients suffering from grievous illness; unfortunately, the United States lags behind the rest of the first world in advancing these therapies because our academic scientists and elected officials have preferentially advanced human embryonic stem cell research, which is currently helping no one. Other alternatives include therapies that block tissue destructive pathways. For instance, Enbrel is a drug for rheumatoid arthritis that blocks the joint destroying action of a molecule called TNF. Once the destructive pathway is blocked, naturally present regenerative processes are able to replace the damaged joints.

ddn: Describe some of your current work in this area.

Sherley: My laboratory’s research is focused on developing methods for production of adult stem cells, discovering biomarkers that can be used to quantify adult stem cells, and elucidating cellular mechanisms that are important for adult stem cell function, health and longevity.

Deisher: My research focuses on the efficient delivery and retention of stem cells in diseased organs.

ddn: What should the government’s role be in stem cell research being done in the United States?

Sherley: Through its agent the NIH, its role should be the same as for other areas of research: Adhering to U.S. law, foster and fund ethical, high-quality research that has potential to positively impact human health.

Deisher: (References the NIH’s mission statement, which states, “NIH’s mission is to seek fundamental knowledge about the nature and behavior of living systems and the application of that knowledge to enhance health, lengthen life and reduce the burdens of illness and disability. The goals of the agency are: To foster fundamental creative discoveries, innovative research strategies, and their applications as a basis for ultimately protecting and improving health; To develop, maintain and renew scientific human and physical resources that will ensure the nation’s capability to prevent disease; To expand the knowledge base in medical and associated sciences in order to enhance the nation’s economic well-being and ensure a continued high return on the public investment in research; and to exemplify and promote the highest level of scientific integrity, public accountability and social responsibility in the conduct of science.”)

Highlighted in red are NIH mandates that are not met by NIH funding of human embryonic stem cell research. The government’s role should be to actively promote research and clinical trials in areas that would not be adequately advanced by private industry. Adult stem cells from a patient’s own blood, bone marrow or other organs are not patentable, and therefore, absolutely require federal funding to bring these remarkable treatments to U.S. citizens. As taxpayer funds are used by the government for these purposes, one would hope that affordability would also be an important criterion for federal funding of clinical trials. Adult stem cell therapies are affordable, and will cost on average about $25,000, while embryonic stem cell therapies, if ever available, will be expensive and beyond the means of almost all U.S. taxpayers. Geron Corp. issued public statements in January 2009 estimating the cost of human embryonic stem cell-based therapies for spinal cord injury to be just under $500,000. Geron received significant NIH funding for this work.

ddn: How does the 2009 executive order from President Obama impact your access to research funds?

Sherley: Because the president’s order led to NIH instituting the illegal funding of human embryonic stem cell research, it injures all U.S. scientists working in other fields who compete for the same limited NIH resources. However, the greater injury is sustained by scientists whose research focuses on the recognized alternative to human embryonic stem cells, adult stem cells, because of the juxtaposition of all stem cell research applications in the NIH review process. At each phase of the NIH review process, bias and contempt, inspired by an illegal implementation of the president’s order, can occur against their research applications, even though they may be of the highest quality.

Deisher: The executive order initiated a process where preference in NIH requests for grant proposals and ultimate grant funding is now directed towards embryonic stem cell research. The 2009 executive order amplified congressional obsession with funding embryonic stem cell research, apparent since 2006. Every adult stem cell scientist and clinician is injured by the preferential emphasis and funding of embryonic stem cell research. Most egregiously, U.S. citizens are being denied adult stem cell therapies that are in development in Europe and other countries for heart attack, blindness, paralysis, stroke, diabetes, multiple sclerosis, lupus and other grievous human illness.

ddn: What can Americans do to get past the moral arguments in favor of or against embryonic stem cell research, in order to make sure that we are still working toward finding cures for devastating diseases and conditions?

Sherley: Americans would have no dilemma to ponder, if they were better informed that human embryonic stem cells have very little potential to lead to cures for devastating diseases and conditions. The No. 1 sales pitch pushed for these cells, the “ability to make any cell in the body,” is their fatal flaw for providing new medical therapies. For effective tissue and organ therapies, regenerative cells are needed that can continuously replace the mature cells found in just the sick or ailing tissue or organ. Human embryonic stem cells cannot do this. Adult stem cells can. Since human embryonic stem cells make other cells that are not needed, if they were used, they would give the patient another well-known, very difficult-to-treat disease.

Deisher: Science should never be above moral scrutiny and one only needs a brief history lesson to know this. On another note, I hear arguments regularly that we should not deter human embryonic stem cell research because of the moral objections of a minority. This is absurd, as our entire scientific research ethical oversight is predicated on respect for the moral objections of a minority in our country. Respect for animal rights and concern for ethical research conduct using animals in experiments regularly deters experiments that are scientifically and economically expedient. Respect for ethical concerns as they relate to human subjects research should receive no less deference. However, Americans do not need moral arguments to oppose embryonic stem cell research; embryonic stem cells are known to form tumors, they would require lifelong immune suppression that itself can cause hypertension, osteoporosis and other nasty side-effects, and human embryonic stem cell products would be too expensive for Americans to afford.

Deisher was also kind enough to field questions about how her Catholic faith informs her approach to stem cell research. We’ll bring you that Q&A very soon.

Unintended advantages?

Random odd thoughts are the bane of my existence sometimes. They’ll come visit, and flit in and out of my consciousness for weeks or months like some bug trying to find its way out of the house and buzzing around annoyingly until it can.

Well, this blog will at least finally provide me a place to unload some of those thoughts (as long as they are pharma-related in some way) and eliminate some of those buzzing sounds.

In this case, after posting on our website this story on a genome-wide study of human stem cells, I was reminded of a thought that has been nagging me more and more since the study of adult human stem cells has picked up in recent years and I’ve seen increasing numbers of breakthroughs related to them. And that thought was: Did the bans related to the use of embryonic human stem cells that marked much of the start of the 21^st century actually have an unintended positive effect?

Now, don’t get me wrong. I’m not laying down moral judgments about the use of embryonic stem cells in research and perhaps therapeutics one day, and I’m neither defending nor decrying policies against the use of embryonic cells (though I admit I lean heavily toward allowing their use—I understand the value of such cells, as they are much more malleable, if you will, than their adult counterparts. Also, it seems questionable to let embryonic stem cells go to waste over a philosophical argument over abortion).

And yet…

By forcing researchers to have to look harder at adult stem cells instead—regardless of whether the policy was right or wrong—have we perhaps seen more advances in manipulating and understanding adult human stem cells than we would have if access to embryonic cells had been greater?

I don’t really have an answer, but it’s a thought worth considering. Sometimes, even decisions that may be wrong can have positive results that we never expected.