Embryonic Stem Cell Research and Therapeutic Cloning Policy Issues

Embryonic Stem Cell Research and Therapeutic Cloning Policy Issues, their Significance to the MPS and ML Community and Practical Reasons for an Honest ResolutionMr. Les Sheaffer, Board of Directors National MPS Society, Chairman Committee on Federal LegislationRevised March 11th 2003


Embryonic Stem Cell Research (ESCR) is an issue not easily broached due in part to the polarization of many of the involved parties. This polarization results from divergent ethical, scientific and spiritual beliefs and perspectives, and the ever present human component, emotion. What I hope to accomplish with this document is to express the viewpoint of the National MPS Society and MPS / ML community and to support specific public policy. These viewpoints are based upon numerous discussions with both researchers and families struggling with these deadly genetic disorders on a daily basis.

A Perspective on MPS and ML Disorders

To gain an adequate perspective on what is to follow the reader must gain a basic understanding of the disorders that plague our children and loved ones. Mucopolysaccharidosis (MPS) and Mucolipodisis (ML) disorders are genetic Lysosomal Storage Disorders caused by the bodies’ inability to produce certain enzymes. Normally the body uses these enzymes to break down and recycle cellular debris. In affected individuals, the missing or insufficient enzyme prevents the normal breakdown and recycling of debris resulting in storage of these deposits in virtually every cell in the body. As a result of the storage, cells do not perform properly and may cause progressive damage throughout the body, including the heart, bones, joints, respiratory system and central nervous system. While the disease may not be apparent at birth, signs develop with age as more cells become damaged by the accumulation of the deposits. It must be noted that the resulting damage to the various organ systems results in a severely diminished quality of life and drastically shortened lifespan for children suffering from these disorders.

Embryonic Stem Cell Research and the Promise it Holds for Development of Effective Therapies

MPS and ML are complex disorders that present a number of very significant obstacles to treatment. Therapies used in the past and those currently under development may offer varying degrees of improved quality of life to affected individuals. However these therapies do not address the damage to the central nervous system nor do they completely alleviate or correct the systemic effects of the disease.

The established view of the MPS research community holds that ESCR and stem cell research in general represents a critically important avenue that must be aggressively pursued. The ways in which stem cells could be used in Lysosomal Storage Disorder research are many. The great potential benefits of ESCR may be best articulated by those most involved in MPS and ML research. To illustrate this point I have included the following paragraphs authored by Mark Sands PhD, Division of Bone Marrow Transplantation and Stem Cell Biology of the Washington University School of Medicine

The use of embryonic stem (ES) cells as therapeutic tools has enormous potential for a wide variety of diseases. Pluripotent ES cells are very primitive cells that have the potential to differentiate into virtually any cell type in the body. Under the appropriate conditions, neurons, cardiac myocytes, cells of the hematopoietic lineage, adipocytes and a wide variety of other cell types have been generated from ES cells in vitro. In the case of murine ES cells, every tissue in a live mouse can be derived from these pluripotent cells. The recent development of human ES-like cells opens up the possibility of replacing damaged or diseased cells in humans.

In the case of lysosomal storage diseases where the enzyme deficiencies can be complemented by exogenous enzyme supplied by donor cells, ES cells may be particularly useful. It has been shown that the biochemical and clinical defects associated with lysosomal storage diseases can be corrected by transplanting normal donor cells into affected patients or animal models of these diseases. A systemic reduction of lysosomal storage can be accomplished by replacing the affected patient’s hematopoietic system with that of a normal individual by bone marrow transplantation. In addition, lysosomal storage in the central nervous system of animal models of lysosomal storage disease has been reduced or eliminated following transplantation of neuronal progenitor cells into the brain. These cells could also be used in conjunction with gene therapy approaches. Primitive ES cells could be genetically modified to over produce a deficient enzyme, differentiated into a particular cell type and then transplanted into the site where the enzyme was needed.
In addition to simply supplying the deficient enzyme progenitor cells may also have the ability to replace cells that have already died or become senescent. Results from these studies highlight the potential of using stem cell and transplantation technologies for the treatment of inborn errors of metabolism.

Although recent reports suggest that many different cell types can be generated from bone marrow cells, those studies have not been widely confirmed. It remains unclear whether cells residing in the bone marrow of adults will have the same potential to differentiate into as wide a variety of authentic cell types as primitive ES cells derived from blastocysts. Therefore, ES cells still offer the most potential for the replacement of a wide variety of cell types in disease situations.

Clearly there exists a substantial body of thought to support the view not only of the National MPS Society, but the majority of the rare disease and genetics community. In our opinion the identification of embryonic stem cells represents a great scientific achievement and ongoing stem cell research to explore the therapeutic benefits of ES cells must be strongly supported to ensure the opportunity for innovation and discovery in treating MPS as well as other deadly diseases.

The American Public, the National MPS Society and Opinion Regarding ESCR

The majority of Americans support federal funding of Embryonic Stem Cell Research according to available polling data. In a June 2001 ABC News poll 60% of Americans supported federal funding of ESCR with 31% opposing it. A July 2001 Washington Post poll reports that 63% support ESCR and 33% of those polled opposed. Finally, in a June 2001 Wall Street Journal poll 69% of respondents were in favor of ESCR and 23% were opposed to the use of surplus embryos for procurement of stem cells for research.

Americans whose children or family members are affected by MPS and ML disorders or one of 6000 other rare diseases far exceed the national average in terms of supporting federal funding of ESCR. This is understandable due to the unique perspective provided by having a loved one suffering from a serious chronic or fatal disease. These Americans do not have the luxury of debating the issue of ESCR as an interesting abstract issue serving as fodder for discussion of their theoretical, ethical and moral views.

To these people ESCR represents an additional avenue to the development of potential treatments for their family members affected by these debilitating and fatal diseases.
In the case of MPS and other complex rare disorders federal funding for research including ESCR is a vital component in scientific discovery and understanding. To illustrate this point MPS researchers on average obtain 85% of their research dollars from the National Institutes of Health with the remaining 15% being derived from non profit organizations such as the National MPS Society and Children’s Medical Research Foundation.

Clearly, without significant federal funding MPS research would not be where it is today. Continued federal investment in the development of novel therapeutic approaches including the use of stem cells will continue to be a critical factor for the foreseeable future.

Commonsense Federal Policy Provisions Supported by the National MPS Society

The public policy aspects of this issue must be framed in the following context, Embryonic Stem Cell as well as adult and umbilical cord stem cell research must be federally funded and ESCR requires practical regulatory controls to establish uniform guidelines and protections from potential abuses.

The commonsense policy provisions that we anticipate will be needed immediately to provide a reasonable regulatory structure and expedite research activity would includeFull federal funding of ESCR utilizing stem cells derived from surplus embryos created for infertility treatment that would normally be disposed of, providing consent of the donors has been obtained. We fully support federal funding of research on the 64 recognized stem cell lines available. In addition, the rapid implementation of the above stated policy is essential and will provide for expanded access to critical new stem cell lines that will not be available under current administration policy.

It should be noted that the National Institutes of Health in its paper titled “Strategies for Implementing Human Embryonic Stem Cell Research” dated February 28, 2002 predicted that they will encounter supply related problems in providing NIH funded investigators with sufficient numbers of ES cells. This is due to the length of time it requires to refine and characterize these cells and the lack of ability to predict the level of interest that will present itself. It is quite evident that this situation could be significantly reduced by access to additional lines that would be available from IVF donated material.

These cells may provide treatments to people with a variety of serious diseases with a greatly reduced frequency of rejection and reduce the need for immunosuppressive drugs. This resource (donated embryos from IVF clinics) continues to be discarded on a daily basis, this practice is clearly in no ones best interest.

In our view this policy represents the ideal immediate approach to ensuring enhanced availability of stem cells for potentially life saving research. We fully support the advancement of this policy on its own merits regardless of its real or perceived political linkage to the therapeutic cloning issue.

Prohibition of commercial sale of embryos and of compensation to the involved parties at any time for donation of surplus embryos. This provision, if viewed honestly, should serve to void the fears of ’embryo farming’ that have been suggested in the recent past by removing financial incentives for donation of frozen embryos from in vitro fertilization clinics. It could be effectively argued that prohibiting the voluntary donation of surplus frozen embryos from IVF clinics for therapeutic research could serve to establish the ‘black market’ that is feared by those who oppose expanding the federal funding of ESCR and expansion of available cell lines. This provision should not prohibit reasonable payments associated with transportation, preservation and storage.

Strong support for and promotion of extensive animal studies involving ESCR to facilitate the establishment of the significant scientific knowledge and evidence to support human ESCR studies. The importance of this provision has been born out in numerous areas of biomedical research. Preclinical animal studies are indispensable components in the formulation of solid scientific knowledge and understanding needed to bring safe and effective products and treatment strategies to everyday people in dire need of medical treatment options.

Establishment of a Permanent Non Partisan Cell Development and Stem Cell Research Oversight Body. This provision would serve to provide an entity to establish specific guidelines and provide adequate oversight. In our view, the body should be comprised of officials with limited terms of service appointed by the President and both houses of Congress to ensure an adequately diverse body of thought. The body would work to develop clear standards of conduct using established standards of scientific exchange, peer review and public oversight as applied to other areas of federally funded research. The body should also develop and promote policy that ensures efficient and timely funding of ESCR and consult with Congress, the National Institutes of Health and other appropriate federal agencies.

Strict Prohibition of Human Reproductive Cloning. This provision requires inclusion to prevent the production of a human being through asexual reproduction. By definition “Human” or “Reproductive” cloning requires the attempted or actual implantation of the product of nuclear transplantation into a uterus or functional equivalent of a uterus manipulated for the purposes of producing a human being. In our view the practice of human or reproductive cloning serves no legitimate purpose and should be prohibited. Enforcement of prohibition policy should include strict federal civil and criminal penalties including imprisonment.

Allowance of Somatic Cell Nuclear Transfer for Procurement of Stem Cells for Medical Research. (Therapeutic Cloning and Regenerative Medicine) This provision pertains to what has been historically referred to as “therapeutic cloning”. In our view, it is important that “Therapeutic Cloning” be adequately defined as somatic cell nuclear transfer only for the purposes of producing stem cells for research. Therapeutic cloning should not be linked in policy to “Reproductive” or “Human” cloning as they are separate issues and techniques with distinctly differing outcomes.

The National Academy of Science, Institute of Medicine and National Bioethics Advisory Commission have established that unlike human cloning, the creation of embryonic stem cells by nuclear transplantation involves the use of an oocyte (egg cell) with its nucleus removed and then replaced with somatic cell material, electronically activated and allowed to divide for a short period of time to produce stem cell clusters. The manipulated oocyte is never fertilized with sperm or implanted in a uterus. Therefore, the process cannot produce a complete ‘live-born animal’. Therefore, somatic cell transfer or therapeutic cloning for the purposes of this discussion should be considered as an avenue for procurement of stem cells for research purposes.

These provisions with appropriate federal oversight and enforcement of high standards of ethical conduct and safety with respect to human subjects in research, informed consent and privacy protections are supported by the National MPS Society Inc.

Practical Reasons for a Resolution of this Issue in Congress and Implementation of Sound Policy

The practical reasons for supporting the policies stated above are many .In future years the cost of providing care for chronically and terminally ill people would be greatly reduced due to the ability to treat currently untreatable diseases. Commonsense suggests that the rate of divorce and crippling financial hardship (common consequences of caring for severely disabled family members) would also be reduced. (Studies have shown high divorce rates among families struggling with providing care for disabled children)
In addition the social and financial burden on the general public through delivery of important programs vital to families of children with severe disabilities such as Supplemental Security Income (SSI) and Medicaid would be reduced by the availability of effective therapies. These benefits represent only a few of the potential positive outcomes of Embryonic Stem Cell Research.

The plain fact is for MPS and ML families the importance of research for improved quality of life is of the greatest possible priority. Like families struggling with any one of the thousands of diseases in existence, we are searching for hope and a future for our children and loved ones. Innovative biomedical research represents our best hope for one day making treatment options available for MPS and ML families as well as the myriad of other disorders where effective therapies are not available.

While progress on this issue is delayed and in some cases trivialized for political expedience, MPS and ML children will continue to slip further into the progression of these terrible diseases and ultimately pass away without the opportunity to experience all that life has to offer those of us blessed with good health. It is essential that every avenue that could provide the knowledge and resources to save human lives be aggressively pursued. To close the door on this approach would be a disservice to our children and future generations.

Specific Legislation Supported by the National MPS Society with Respect to Stem Cell Research and Cloning as of March 11th 2003

The National MPS Society has in the past supported S.723 and HR.2059 the Stem Cell Research Act of 2001, and S.2439 the Human Cloning Prohibition Act of 2002, we recognize the need for additional avenues with respect to access to ES cells and the importance of a clear prohibition of human reproductive cloning therefore, we fully support S.303 the “Human Cloning Ban and Stem Cell Research Protection Act of 2003”.


For additional information regarding MPS Society Legislative Policy Positions and Goals, contact

Mrs. Sissi Langford Mr. Les Sheaffer
Co-Chair Committee on Federal Legislation Chairman Committee on Federal Legislation
NoniML@att.net lessheaffermps@juno.com


Informational Resources Used in Researching this Document

National MPS Society Publications MSNBC online
The American Society of Human Genetics The Library of Congress (Thomas)
The Congressional Record ABC News Online
The Washington Times The National Society ofGenetic Counselors
The National Institutes of Health The Gallup Organization
The Department of Health and Human Services The Washington Post