At four months old, Anthony Dones was diagnosed with Osteopetrosis, a rare bone disease that requires the replacement of the bone marrow in order to be treated. His doctors decided to try a new therapy, a cord blood transplant, which utilizes stem cells extracted from the placenta following childbirth, to try and treat him. Within a week, a suitable match was found and, a month after he was diagnosed, Dones had the procedure done. Thanks to the work of his doctors, the National Cord Blood Program, and the research of stem cell scientists, young Anthony, now four, is leading a normal childhood.
Patients have been facing increasingly better odds of overcoming formerly debilitating and even deadly diseases as science has progressed over the years. Research with stem cells has opened up new treatment options, but the field is even now only scratching the surface of what’s possible.
Newer and more versatile embryonic stem cell research carries the promise of being able to treat some of the most destructive of conditions, such as heart disease and cancer, but ethical and moral arguments between groups on both sides of the issue have slowed further advancement and funding for this fledgling field. This leaves the fate of persons afflicted by diseases such as Dones’ in the balance.
Recently, the findings of Professor Robert Lanza, medical director of Advanced Cell Technology in Massachusetts, were reported in the scientific journal Nature. Lanza claims that he and his team extracted single cells from human embryos early in their development and created new stem cell lines without destroying the embryo.
This news has the potential to give embryonic stem cell research a boost. There is currently a prohibition against federal funding for new stem cell research that destroys the embryo. If Lanza’s research proves to be viable, stem cell lines created in this manner may qualify for crucial federal approval and funding while also easing the qualms of some ethical opponents.
Some critics have contended that Lanza’s research, while promising, did not actually save the embryos, as all those used in his experiment were destroyed. Additionally, others say that the removed cell had the potential to develop into a twin. Regardless, his findings and the reports of several other teams in late August (see sidebar), have once again brought embryonic stem cell research into the limelight as the new frontier of modern medicine.
These developments are only the newest in the field of stem cell research. The use of stem cells began with advances made with cord blood stem cells in the late 1980s. Cord blood stem cells are collected from umbilical cords and placentas following childbirth. This blood contains not only the normal elements of blood, but also stem cells that can differentiate into various new and different blood cells. According to the National Cord Blood Program website, over 70 different diseases, including Dones’ Osteopetrosis, have already been treated with cord blood stem cells. These range from multiple forms of leukemia and immune system maladies to a variety of rare childhood diseases.
Since late 1998 however, stem cell research has centered more around embryonic stem cells, which requires stem cells collected from human embryos. Embryonic stem cells are much more useful to scientists than cord blood stem cells due to the fact they can differentiate into any kind of body cell, as opposed to only blood cells. Embryos used in stem cell research are created in vitro, that is outside of the body, a process performed largely by fertility clinics. Unused embryos are donated with the consent of donor parents and then cultivated for four to five days, forming what is known as a blastocyst.
These blastocysts contain stem cells, which are then collected for research purposes, a process during which the embryo is destroyed. After the stem cells are extracted from the blastocyst, researchers put them through a rigorous testing and culturing procedure in hopes of creating a viable stem cell line, a collection of embryonic stem cells that can replicate themselves for long periods of time in the absence of a human host.
In 2001, President Bush made first ever federal funding available for embryonic stem cell research. Prior to this time, there was a ban on embryonic research in the United States, stemming from the attachment of a clause to the 1995 appropriations bill for the National Institutes for Health (NIH). The clause, known as the Dickey Amendment after the bill’s presenter, Representative Jay Dickey, stated “none of the funds made available in this Act may be used for…research in which a human embryo or embryos are destroyed, discarded, or knowingly subjected to risk of injury or death…”
In an address to the nation on stem cell research given in 2001, President Bush stated that he would make available federal aid for “more than 60 genetically diverse stem cell lines.” According to the NIH Human Embryonic Stem Cell Registry, which lists all embryonic stem cell lines eligible for federal funding, there are only 21 embryonic stem cell lines eligible for federal funding.
The NIH, which has final say on federal funding for stem cell research, made $609 million available for stem cell research in 2005. Of that amount, only $40 million, a mere 6.6 percent, was allocated to human embryonic stem cell research. This shortage of federal assistance may be a contributing factor as to why no treatments have been made using embryonic stem cells.
Research on embryonic stem cell lines not appearing on the Human Embryonic Stem Cell Registry is currently underway at various universities and private institutions across the nation, but the costs of such involved and extensive research are enormous. Many research programs rely solely on donations and other privately obtained funding.
“We’re lucky to have great philanthropic support,” said B. D. Colen, a spokesman for the Harvard Stem Cell Institute (HSCI). He also stated that the HSCI had raised $50 million in alumni donations and other financial contributions, but was unable to put a precise amount on the costs of research due to its variable nature. “Doing science is expensive, not just stem cell [science],” he said.
Colen and the HSCI aren’t the only ones suffering from a shortage of funds. In September 2004, the Juvenile Diabetes Research Foundation (JDRF) wrote a letter to Congress outlining the necessity of new stem cell lines and additional federal funding. In it, they drew attention to issues that are still impeding the progress of scientists.
For starters, genetic differences can hinder the potential use of any advances made from the approved cell lines. This is due to the Human Leukocyte Antigen (HLA), which decides whether or not a cell is compatible with the recipient. As the JDRF puts it, this is comparable to blood types. In the same way that a person with type O blood will reject type A blood, someone with a different HLA may not be able to receive stem cell tissues differentiated from the existing stem cell lines.
Additionally, the JDRF points out that current stem cell lines have been developed and stored for extended periods of time. While this helps to maintain the viability of these cell lines, our storage methods are not perfect. As a result of continued storage and repeated usage, current stem cell lines are becoming harder to culture and are developing chromosomal abnormalities, a problem that will require new stem cell lines and the funding to develop them.
The JDRF also indicates that diseases such as muscular dystrophy, Huntington’s disease, and cystic fibrosis, which combined affect over 100,000 Americans, all arise from problems in a single human gene. None of the stem cell lines approved for federal funding contain this gene, however. Without it, scientists are forced to research these diseases with limited funding obtained privately or donated, severely limiting their ability to study, better understand, and treat these maladies.
Hoping to address concerns such as those raised in the JDRF’s letter, on July 18, 2006 the Senate passed HR810, otherwise known as the Stem Cell Research Enhancement Act of 2005. The act was intended to allow previously ineligible lines to qualify for federal funds. It also imposed a three-part stipulation outlining the requirements for aid eligibility, most of which addressed ethical concerns.
First, embryos must have been donated from fertility clinics and made for fertility treatment, but were unneeded by the donor parents. Second, it must have been determined the embryos were not needed before donation of an embryo was even considered. Lastly, the embryos must have been donated with informed written consent and without financial compensation.
When President Bush received the Stem Cell Research Enhancement Act the following day, he exercised the first veto of his Presidency. Despite efforts from both sides of the aisle, Congress was unsuccessful in the effort to override the President’s veto. President Bush went on to speak at a press conference later that afternoon, surrounded by children born from adopted excess embryos, saying, “I will not allow our nation to cross this moral line…Crossing the line would needlessly encourage a conflict between science and ethics that can only do damage to both.”
The President’s concerns have a moral basis, grounded by his belief that the destruction of an embryo for research destroys one life to save another. It is a stance he shares with many concerned groups, including members of the Roman Catholic Church.
“Life begins at conception, and as soon as fertilization occurs, the embryo is considered a person,” said Greg Coohan, Roman Catholic campus minister at the Newman Center here at the University at Buffalo. “The ends can never justify the means in Catholic thought—you can’t take from one to better another.”
The embryos used for research, however, are the excess of those needed by couples at fertility clinics. These embryos generally face three fates. The embryos can be used for another couple, they can also be frozen indefinitely, in case of future need, or they can be discarded as medical refuse.
“Is it somehow morally, ethically better to literally flush [embryos] down the sink than use them to learn about disease?” said Colen on the moral and ethical ramifications of embryonic research. “Frankly, the ultimate pro-life position is to cure disease for people who are suffering.”
As researchers continue work with stem cells, the day when we can reap the benefits without destroying human embryos may be fast approaching. While theologians, politicians, and researchers debate whether the human cost of embryonic research outweighs the potential payoff, hundreds of thousands of lives are left in balance, waiting for a decision to come that may afford them a second chance at life. If one thing is certain, it’s that this field is ready to advance beyond even the techniques that saved Anthony Dones’ life. “Cord blood [stem cells] may be useful,” said Colen, “but it’s not the only avenue.” It’s an avenue with an unstable, but potentially bright, future.
