Much of their hope, they say, rides on the possibility that human embryonic stem cells may generate insulin-producing cells that could be used to reverse the disease. Others are optimistic such cells may someday treat those with spinal cord injuries, heart problems, Parkinson's disease or Alzheimer's disease.
The fragile, controversial cells are, scientists say, the new frontier of medicine.
"It reminds me of our country's quest for space many years ago," Utah Sen. Orrin Hatch told the U.S. Senate in April. "Yet, what was only a vision when it was conceived, yielded wonders beyond anything we could have imagined."
Hatch is among those leading the charge to make the cells more available to scientists.
Yet even if the entrenched political debate were resolved - and federal restrictions on using the cells were lifted immediately - scientists and politicians say it could take another 10, even 20 years before commercial embryonic stem cell therapies are widely available.
Many factors, they say - including the restrictions, a lack of trained scientists and sluggish private investment - have slowed efforts to clinically develop embryonic stem cell therapies, placing the U.S. at risk of lagging behind other countries.
"When you've told the world stem cells are the answer, the world is like, 'Well, let's have it today or tomorrow,' '' said G. Russell Reiss, a cardiothorasic surgeon at the Veterans Affairs Medical Center in Salt Lake City. "I don't think scientists are intentionally misleading anybody about cures or the potential therapies that are going to come out of stem cells.
"It's just that the public at this point desperately wants to speed up that scientific progress."
Cells of controversy, promise. The restrictions date to Aug. 9, 2001, when President Bush announced researchers who accept federal funds would be limited to using 60 or so embryonic stem-cell lines already in existence.
Embryonic stem cells used for research begin with in vitro fertilization: a human egg that is fertilized and begins to grow in a laboratory. Five days later, embryonic stem cells are extracted from the resulting blastocyst - a sphere of about 150 cells - terminating the embryo.
That destruction is the source of anti-abortion opposition to such research.
Embryonic stem cells' promise comes from their ability to become a heart cell, a brain cell or any cell in the body. Scientists believe they may be used to test new drugs, provide insight into birth defects and, perhaps, treat injury or disease.
Such cells might be injected into an injured spinal cord, replacing damaged nerve cells and allowing a paraplegic to walk. Or injected into the brain of a person with Parkinson's, restoring communication between nerve cells and reversing the effects of the disease.
"Stem cells have an extraordinary potential for revolutionizing medicine," said James Battey, a physician and vice chairman of the National Institutes of Health's Stem Cell Task Force.
Stalled federal research. But since 2001, the genetic quality of some of the presidentially approved embryonic stem-cell lines have deteriorated after duplication. Others couldn't be kept alive. Just 21 lines remain.
All the approved lines, scientists worry, have been cultivated in lab dishes that once contained mouse feeder cells, which may have infected the stem cells with animal viruses.
Also, the surviving lines aren't representative of the general population, making it difficult for scientists to gauge how effective a particular therapy might be.
Other effects of the restrictions: American universities studying embryonic stem cells from both the Bush-approved lines and those cultivated in the private sector must segregate their research, Battey said. Equipment purchased with federal money, for instance, cannot be used to perform research on privately developed stem cells.
No Utah universities are working with the federally approved cells. Many institutions have bought duplicate equipment.
"They're terrified of being audited," Battey explained, since loss of federal funding would be "disastrous for universities."
The political challenge. In April, the Senate passed a bill that would override Bush's restrictions and support federal funding for embryonic stem cell research. The House is expected to take it up after Memorial Day, and if the measure is passed, it will go to the president. But Bush is expected to veto it - sending it back to the Senate for an override vote, which is not expected to succeed.
The Parker family had hoped to help lift the restrictions in 2005, when they traveled to the nation's capital. The Parkers told Utah's delegation that treatment advances in the past decade have made type 1 diabetes more manageable. But long-term risks, including eye, heart and kidney complications, remain. Embryonic stem cell therapies may be their daughters' best option, they said.
Also in 2005, a poll conducted for The Salt Lake Tribune showed nearly 53 percent of adults backed legislation sponsored by Hatch that would have removed Bush's restrictions. Another 35 percent disagreed, while 12 percent didn't know or declined to answer.
Two years later, the on-going political debate frustrates the Parkers.
"Among our circle of associations with others with the disease, it [stem cell therapies] is very widely discussed," Chad said. "It is something that is providing a great deal of hope."
The scientific challenge. Tucked into freezers in Mario Capecchi's University of Utah School of Medicine lab are thousands of tiny vials full of embryonic stem cells taken from mice.
Capecchi, a professor of human genetics, began working with the cells in the mid-1980s and discovered how to isolate and modify genes, place them in an early embryo and propagate the change in a new generation of mice.
The breakthrough, for which he won the Albert Lasker Award for Basic Medical Research in 2001, means scientists can now "modify any gene in the genome and ask what the consequences of that gene are," he said.
Capecchi, a scientific adviser to Hatch, says the biggest challenge facing scientists is how to guide the cells down a specific path - cajoling a stem cell to become a blood cell, for instance. And like organ transplant patients, embryonic stem-cell transplant patients may face a lifetime of immuno-suppressant drugs to prevent rejection. One possible alternative, he said, involves cloning.
Embryonic stem cells taken instead from an unfertilized egg and transplanted with a nucleus from a patient's skin cell, for example, could produce cells with a patient's own genes.
"Under certain conditions that cell will divide and grow and will make a little embryo and from that you can make embryonic stem cells," he said. "Because it's getting all of your genetic material, which will be running this system, you won't reject it."
Help wanted. Another obstacle is the U.S. has "a real shortfall of people who are well-trained and well-versed in the culture of stem-cell growing," said Battey of the NIH Stem Cell Task Force.
"Technically, the limits are getting enough cells and enough experience to understand precisely what the molecular cues are to drive an undifferentiated cell [or a cell that hasn't been assigned a function yet] into another cell type," he said.
Scientists are leaving the U.S. to pursue opportunities in countries that do not have embryonic stem cell restrictions, and "young people are very hesitant to get into this area because they simply don't know where it's going," Capecchi said.
Laments Hatch: "We are definitely losing the scientists who will carry the critical research into the future."
The financial challenge. At Q Therapeutics in Salt Lake City, private researchers are using technology licensed from the University of Utah to find treatments for degenerative diseases of the central nervous system, such as cerebral palsy and multiple sclerosis.
So far, however, it is doing so with adult stem cells, not embryonic ones.
Deborah Eppstein, its president and CEO, said while there is optimism about embryonic stem cell therapies, "there's frustration because there is no funding to do the work here."
Another side effect of the federal restrictions is less investment in biotechnology companies developing embryonic stem-cell therapies.
"I can tell you very clearly the lack of federal funding has certainly impacted the willingness of venture capitalists to fund stuff because venture capitalists don't like to fund research," Eppstein said.
Investors are waiting for the field to mature, said Reiss, of the Veterans Affairs Medical Center. "They've kind of waited to see, is this real?" he said.
"Intellectual infrastructure." Several states, including California and Massachusetts, are creating ways to tap into the 400 embryonic stem cell lines that exist worldwide. Californians have approved a $3 billion bond to fund such research for the next 10 years.
Voters "wanted to make California a global leader in this emerging biomedical field, to provide support to life science companies," said Dale Carlson, a spokesman for the state agency created to oversee the funding and research.
The bond is "essentially an investment in intellectual infrastructure," he said.
At a recent Utah Technology Council meeting, Eppstein said, she pitched a similar idea to the Governor's Office of Economic Development (GOED).
"We're very fortunate this pioneering technology came out of the University of Utah," she said, "but we need funding to carry it forward."
Nicole Toomey Davis of GOED said it's unlikely Utah will follow California's lead. While economically healthy on a per-capita basis, Utah doesn't have the tax revenue to fund large-scale basic research, she said.
"I just can't even imagine," Davis said, "how we would go about asking the community for that kind of money."
Coming Monday: Unwilling to wait. On a cold January day, Tim Schmanski, his wife, Maria, and their 16-year-old daughter, Tori, left Orem for an adult stem-cell clinic in Hangzhou, China.
With few other treatment options available for Tori - who suffered a brain injury after nearly drowning in a canal in June 2005 - the Schmanskis decided to place their bets on a month-long therapy that has not yet been proved safe and effective in the U.S.
lrosetta@sltrib.com
