Foundation Continues Pursuit Of Ethical Stem Cell Use…Babies for Life Foundation…(BFL- www.babiesforlife.org) collects donations of this “diamond” mine of stem cell–rich umbilical cord blood, linking new mothers, researchers and patients in need.
For the past five years Dr. Gerry Sotomayor of BFL has collected umbilical cord blood from newborns, sending it to cord blood public registries to help patients worldwide with the 65 diseases now successfully treated with umbilical cord adult stem cells—not to mention the at least 97 diseases that can be treated or cured by the various types of adult stem cells found throughout the body.
The foundation, established by Sotomayor, has developed a systematic way to collect units at 10 participating Georgia hospitals from women who agree to donate cord blood at no risk to themselves or their babies, thus facilitating a newborn’s first act of charity…each birth provides 1.5-2.5 million cord blood stem cells. These and other adult stem cells are regenerative, unspecialized cells that are able to differentiate into various specialized cells that form tissues.
“It’s the byproduct of a delivery that can be utilized to give life to someone else,” said Sotomayor.
Through the BFL nonprofit organization, this obstetrician-gynecologist from Puerto Rico is “developing relations with several labs” and is also seeking funding in hopes of helping set up a public storage facility at the Medical College of Georgia.
Sotomayor is eager to collaborate with companies and universities involved in various types of research on these virile adult stem cells—hoping one day to replace the embryonic stem cell research that involves destroying embryos to extract just a small number of stem cells to grow in a lab. [A]dult stem cell research [is ethically supported, and] opposes research involving the destroying of embryos, the earliest form of human life.
BFL has also begun collaborating with researcher Mariusz Ratajczak, Ph.D., director of the stem cell biology program at the James Graham Brown Cancer Center at the University of Louisville, Ky. BFL is supplying cord blood to this researcher who has identified a rare type of very small embryo-like stem cell (VSEL) in cord blood and bone marrow very different from the other stem cells found there. VSELs are pluripotent and very similar to embryonic stem cells.
Embryonic (embryo-destructive) research proponents argue that human embryonic stem cells have more potential for medical applications than adult stem cells because they have high plasticity and can turn into any cell in the body. However, Dr. Ratajczak has molecular and morphological evidence that these VSELs have the same cell structures and protein markers as are generally found in embryonic stem cells, which makes them more flexible and with the potential to turn into liver, pancreas, neural tissue, skeletal muscles or heart tissue — [so that they are about equal in performance to embryo-destructive stem cells without the ethical dilemma of destroying human embryos].
“They’re like real diamonds in bone marrow and cord blood,” Ratajczak said. “For the first time we’ve purified these cells from adult bone marrow and cord blood at the single cell level and shown their morphology and markers.”
Georgia Commission To Establish Cord Blood Bank Network
Sotomayor is excited about sharing this “national treasure” of cord blood.
“BFL has expanded into not only just being a collection of stem cells, but we’re identifying people doing the right kind of research. … We need more time for research before we start talking about more treatments. … We’re not there yet, but we will get there soon if we continue ethical stem cell research,” he said. “We’re solving the issues of destroying the human embryos and providing cord blood with (very these small embryo-like stem cells – VSELs). … That’s why we’re excited about pushing the agenda of the (Georgia) Senate bill that failed that would provide a cord blood collection across the state.”
So Sotomayor, Foundation lobbyist B.J. Van Gundy, executive director Simon Moldenhawer and other volunteers are happy that the Senate bill—which didn’t pass during this legislative session in the Georgia General Assembly—has found new life through an executive order by Gov. Sonny Perdue. BFL had advised State Sen. David Shafer in drafting legislation to establish a network of cord blood storage facilities at Georgia universities and to require doctors to inform expectant mothers on options for cord blood private storage or donation. The legislation had ample support, but legislators ran out of time to pass it. On April 14 Gov. Perdue announced plans to establish the Governor’s Commission for Newborn Umbilical Cord Blood Research and Medical Treatment, which for the first time will establish a network of postnatal tissue [placental and umbilical cord] and fluid banks in partnership with universities, hospitals, nonprofit organizations and private firms in Georgia for the purpose of collecting and storing postnatal tissue and fluid.
The commission will be made up of five members and will encourage cord blood stem cell donation and promote awareness of options available to expectant mothers; create a network of cord blood banks to provide safe and secure storage of newborn stem cells; and ensure the availability of these cells for research and life-saving medical procedures and research, according to a press release from the governor’s office. It will report its findings by December 2007.
“I’ve personally collected over 1,000 units by now, not just for transplants but also for research purposes, so we have the experience and know-how to collect quality units with adequate volume with carefully selected patients and with an efficient delivery system—because that’s important. You can’t just sit around with the unit because it goes bad on you after 48 hours,” Sotomayor said in an interview in his Tucker office April 12 along with his daughter, Stephanie, BFL’s marketing director.
For that reason “ideally we’d like to keep everything in-state instead of shipping because we have the technology. We have the people. We have the resources to make it become a leader in the nation in terms of umbilical cord blood,” he continued. “It
’s the future of medicine. This is where we’re going to be going forward in the next few years. We’re going to be talking about less surgeries … and more stem cell treatments. That’s why we’d like to position Georgia ahead of everybody by providing an ethical solution to the stem cell issue.”
BFL volunteer Joe O’Farrell said it’s a “miracle” that the governor is enacting a number of the bill’s provisions. “The work of BFL will continue during next year’s legislative session, and we will once again work to put forward legislation that will further promote the myriad of economic and public health benefits to universal umbilical blood collection. We will also work with Gov. Perdue and the newly created Newborn Stem Cell Commission to help promote public awareness and save lives,” he reported.
The legislation was named Keone’s law, after Keone Penn of Georgia who, because of sickle cell disease, had to endure monthly blood transfusions to survive and was unable to find the match needed for a bone marrow stem cell transplant. So in 1998 doctors with Children’s Health Care of Atlanta and Emory University successfully performed the first unrelated umbilical cord blood stem cell transplant on Penn through umbilical cord blood from a New York public registry that matched his type, and in 2003 he testified before Congress to his cure.
Researchers Unleashing Power Of Adult Stem Cells
According to the Stem Cell Research Foundation, more than 100 million Americans and two billion other people worldwide suffer from diseases that may eventually be treated more effectively or even cured with stem cells, as they could become a renewable source of replacement cells and tissues. Research using non-embryonic stem cells from postnatal tissue and fluid has already resulted in treatment for diseases, including anemia, leukemia, lymphoma, lupus, multiple sclerosis, rheumatoid arthritis, spinal cord injury and Crohn’s disease, and they are being studied for diseases as wide-ranging and diverse as corneal degeneration, heart disease, stroke, Parkinson’s disease and Alzheimer’s. Adult stem cells are also found in the placenta and the amniotic fluid, the septum of the nose, body fat, bone marrow, and other parts of the body in small amounts, in a five- to nine-week-old fetus—which can be obtained ethically following a miscarriage—and in cadavers. They are found in the brain, although they are limited in number and difficult to extract.
There are increasing numbers of examples of tangible ways to unleash the power of adult stem cells. Adult stem cells from the various sources have helped to avert corneal degeneration and to restore vision in cases of blindness and have restored proper cardiac function to heart attack sufferers and improved movement in spinal cord injury patients. One spinal cord injury case example is that of Susan Fajt of Austin, Texas, who became paralyzed in her lower body from a spinal cord injury and went to Portugal in June 2003 to undergo experimental surgery involving transplanting stem cells from her nasal area into the spinal cord injury site. By 2004 she was able to walk with the aid of braces and was testifying before Congress.
And while embryonic stem cells, which in theory can turn into any cell in the body, have been successfully coaxed into many human tissues in the lab, researchers are facing the problem that they form tumors in animal lab experiments. A technique to grow embryonic stem cells in the lab was first developed in 1998.
“To this day there is not a single experiment that has worked in providing therapies or a solution to cancers or other chronic diseases [using embryo-destructive stem cells],” Sotomayor said.
Many adult stem cells are multipotent and are already tweaked in a certain direction and are more restricted in the kinds of specialized cells they can generate. Tad Pacholezyk, Ph.D., of the NCBC, explained in a talk on stem cells in Atlanta that proponents of embryonic stem cell research are quick to point out this disadvantage, but that quality is what also makes them now more applicable for treatment without the tumor problem.
“The very flexibility is the liability of embryonic stem cells, and if you are trying to treat me for a particular ailment you don’t need a stem cell that can be anything. You want a stem cell that is going to go into my liver and behave himself in that environment. So the adult stem cells actually, because they are further down the pathway of differentiation, represent a more stable and controlled cell type.”
Some disadvantages of adult stem cells are that they are harder to make to divide and multiply when they come from older organs and tissues and that they may not live as long as embryonic ones. They generally lose some of their ability to be stem cells over a long period of time in the body. But most adult stem cell types can be taken from the patient’s own body, which eliminates the risk of immune system rejection [patient tissue rejection].
Stephanie Sotomayor explained that for outside donor transplants, umbilical cord stem cells require a lower HLA protein match with the recipient than the perfect match needed for a bone marrow stem cell transplant…
‘Very Small Embryo-Like Stem Cells’
Ratajczak hopes the discovery of the very small embryo-like stem cells will offer a solution to the controversy over embryonic research. He and his team have developed a way to multiply these VSELs in mice and to turn them into murine heart, nerve and pancreatic cells. Details of his study appeared in the journal Leukemia in February 2006. He and his team first identified the VSELs in adult bone marrow that act differently than other bone marrow stem cells, which he reported in 2004 in Leukemia. But earlier research showed the problem of their rarity and the difficulty of growing them in a laboratory.
His next step is to develop strategies to expand VSELs isolated from human cord blood and bone marrow into spheres containing pluripotent stem cells and subsequently differentiate them in a controlled way into different tissues. Thus a strategy to expand human VSELs in the lab would solve the problem of their rarity.
“We expect to develop soon a similar strategy for human cells as we have developed recently for murine VSELs.”
Ratajczak explained that there has been a lot of controversy, indefinite evidence and conflicting data about whether the already-known adult cord blood and bone marrow hematopoietic stem cells are pluripotent, meaning whether they could turn into any cell typ
e. For years researchers have been trying to identify pluripotent adult stem cells but had not until now being able to isolate and purify them to study their single cell structure.
“We succeeded to purify them and show that these cells are pluripotent,” he said. With the murine (mouse) bone marrow VSELs, “we have developed a strategy to amplify these very rare cells both to allow for therapeutic applications in animals and later in clinics.”
If these stem cells were eventually able to be applied in medical treatment on people, they would not be rejected by the patient if taken from the person’s own body. On the other hand, a patient’s immune system would reject donated embryonic stem cells from unmatched tissue donors.
But the scientist did acknowledge that VSELs create the same challenge as do embryonic stem cells: after injection into laboratory animals, the cells isolated from the VSEL-derived spheres behave similarly to cells isolated from embryos—and tend to grow tumors in mice.
“We need also to control development of these cells so that they will not be at risk to grow tumors. … There’s always at every stage a danger these cells can grow tumors,” he said. “We have to continue to work to turn them safely into tissues and not grow tumors when we unleash their potential.”
The Polish native said he has never believed, like others, that hematopoietic stem cells found in the bone marrow that turn into hematopoietic blood cells to treat blood diseases can also turn into other cell types, for which he used to think embryonic research held more potential. But he followed his hypothesis that bone marrow contains other rare stem cells.
“I always thought that probably we have in the bone marrow, in addition to hematopoietic stem cells, another rare population of stem cells that can turn into other tissues,” he said. This hypothesis led him to discover a population of the embryo-like stem cells that are VSELs. “I realized that these cells are a real alternative to embryonic tissues, and what is most important is they are less controversial from an ethical point of view.”
He also affirmed the importance of Babies for Life.
“It is very important to collect and preserve umbilical cord blood as a source of stem cells, including VSELs, for potential therapeutic applications. This material is unfortunately wasted so far, and we need to preserve cord blood stem cells as an important resource of stem cells for regeneration therapies.”
Another research team, led by neuroscientist Dr. Fred Roisen at the University of Louisville, has also shown progress. In March 2006 his team published the findings of their research in the journal Stem Cells. Mice unable to use a front leg due to spinal cord damage were healed and able to scurry across a rope within 12 weeks after being injected with nasal stem cells that were turned into nerve cells.
Another area of significant advancement is stem cell treatment for heart disease. One example is Dr. Amit Patel, director of the Center for Cardiac Cell Therapy at the University of Pittsburgh Medical Center and the UP McGowan Institute for Regenerative Medicine, who pioneered a technique to inject bone marrow stem cells to help rebuild weakened heart muscle. It was first used in May 2005 to treat Jeannine Lewis who suffered heart failure. He accompanied her to Thailand to have the experimental procedure, and she has improved significantly, as have others who have undergone the procedure.
And Dr. David Prentice of the Family Research Council reported in the Journal of Investigative Medicine in January 2006 that to treat diabetes several examples now exist showing generation of insulin-secreting cells from various adult stem cells, including the liver, bone marrow and pancreas. He reported that using spleen cells, one group was able to achieve permanent disease reversal and now has approval from the U.S. Food and Drug Administration to begin human trials for juvenile diabetes.
Sotomayor explained that in some tissues adult stem cells appear to join with the host tissue and “they figure out what the crowd around them is doing, and with the cellular identification process it allows for cells to start working in the same capacity as those surrounding them.”
Sotomayor noted that umbilical cord blood stem cell transplants can also be a good alternative for those who could benefit from a bone marrow stem cell transplant, as it is much less expensive—about $20,000 compared to roughly $275,000—is also painless for the donor and recipient and requires three to five donors compared to 20 to 30. Currently about 10,000 candidates for a bone marrow transplant die each year waiting for a donor. However, the physician added that more research must be done to achieve better success rates comparable to that of bone marrow transplants.
BFL Launches Public Awareness, Fundraising Campaign
To increase awareness about and support for adult stem cell research, BFL is fundraising with a goal to raise $4 million by the end of the year and is launching a public awareness campaign. Stephanie Sotomayor, as one of a core team of nine staff members, has gained valuable experience educating the public and meeting with legislators at the state Capitol.
“It feels great to know that I can be part of something so much bigger than I am and help make a difference. This is really like pioneer work in this area,” said the Boston College graduate. “We want our collection system to serve as a template” for other states and internationally.
Said the proud father, “It’s been a real blessing to watch her grow and blossom as a young professional … dedicating her heart, her entire day and night to this project with such passion.”
When he’s not delivering babies or advocating for the Foundation, Dr. Sotomayor has been working on a master’s degree in bioethics, and he is traveling the globe to speak about a drug used in delivery for a drug company—with a “side agenda” on education about cord blood donation. BFL has been working with the Latin American pharmaceutical company Recalcine to help it establish a cord blood connection system across Latin America based in Brazil and is discussing collaboration with a California company to collect cord blood to treat sickle cell and other diseases that predominantly affect African-Americans.
< span style="font-size: 10pt; color: #000000; font-family: verdana,geneva">The cord blood collected now is shipped by BFL to public cord blood banks outside of Georgia, and those units deemed usable after testing are registered with the Caitlin–Raymond International Registry at the University of Massachusetts and the National Marrow Donor Program, making them available for research and those in need. Currently there are only about 80,000 units of umbilical cord blood in storage worldwide, compared with about 1.5 million needed for a suitable public pool.
“The bottom line is if we all cooperate and we can create a sufficiently large inventory of stem cells that have been ethically collected, we will have enough stem cells to meet the worldwide requirement,” said Sotomayor. “Currently the umbilical cord, the blood and the placenta are wasted. (They are discarded) as medical byproducts … because of a lack of knowledge of the uses and sometimes a lack of resources. That’s why we’re here—to fill in the gap.”
And as they fill that gap they’re glad to support the work of Ratajczak and others to advance research to alleviate suffering.
“We have great enthusiasm for the work we’re doing,” said the physician, as he began quickly eating his lunch before seeing his afternoon patients. “As more umbilical cord stem cells become available, more researchers will come, more treatments will be developed.”
[18May06, http://www.georgiabulletin.org/local/2006/05/18/stemcell/; P. Greear]