by Ulrika G. Gerth
A year and a half after Toni Redmond was diagnosed with ALS, or Lou Gehrig’s disease, she died an excruciating death. Tonia Zampieri of Manchester watched the neuromuscular disease break down her once-so-active mother.
When Redmond, 56, passed away earlier this year, she was constrained to her bed, only left with the blink of her eyes to communicate.
The cause of the disease that ruthlessly takes away patients’ ability to walk, talk, swallow and eventually breathe yet leaves them painfully aware of their body’s degeneration, is a mystery to the medical community.
Redmond thought the answer could be found in stem cell research and so do a world of scientists.
"My mom was always very hopeful," said Zampieri, now in charge of the development of public awareness for the New England Chapter of the ALS Association.
But how to find cures for the nearly 150 million Americans who suffer from diseases and handicaps like multiple sclerosis, Parkinson’s disease, diabetes and spinal cord injury is surrounded by a cloud of controversy in which politics and big business play as large a role as science.
There is the research on human embryonic stem cells promoted by Hollywood stars like Michael J. Fox and the late Christopher Reeve, but opposed by pro-life advocates, since it requires the destruction of a living embryo.
Human embryonic stem cells are considered unique as they are the only cells proven to have the potential to give rise to any of the body’s 200 cell types, leading scientists to believe the cells could replace or repair organs and tissues damaged by some of today’s most devastating diseases and disabilities.
There are also adult stem cells, which despite their name, are found in both adults and children. They were once thought to only generate cells identical to the tissue in which they were harvested, but are now known to be much more versatile.
Brain stem cells can, for example, develop into blood and skeletal material whereas bone marrow stem cells can differentiate into cardiac muscle cells, fat, bone and cartilage.
Adult stem cells have for years been used to treat diseases like leukemia, lymphoma and immune deficiencies, and have recently shown clinical potential in the treatment of Parkinson’s, diabetes, advanced kidney cancer and spinal cord injury.
Six years after Dr. James Thomson at the University of Wisconsin-Madison developed the first human embryonic stem cell line in 1998, research has yet to produce results in humans.
But, said Lulu Pickering, president of the New Hampshire Biotechnology Council, "This is not hype. We’ve infinite potential to do something good. The industry is abuzz to use this technology and the potential is tremendous. The problem is the restriction on research."
Months after Redmond’s death, the issue of human embryonic stem cell research rose to the fore of Sen. John Kerry’s presidential campaign.
Hammering away at what Kerry often labeled Bush’s "ban" on the research, the Democratic candidate pledged to lift all restrictions.
In April of 2001, Bush made — for the first time — federal funds available for human embryonic stem cell research but limited the research to embryonic stem cell lines derived before the law was signed into effect. There are no such limits on research done with private funding.
In a rally in Newton, Iowa, Sen. John Edwards told the crowd that with Kerry in the White House "people like Christopher Reeve are going to walk, get up out of that wheelchair and walk again."
His statement provoked both outrage and support, showing how polarized the issue had become.
"It’s not a good thing to make a political football out of it," said Charles Carson, president of the Spinal Cord Society. "There is so much potential in both adult and embryonic stem cells, it should be free from all politicians monkeying around with it."
To James Kelly, a paraplegic since an auto accident in 1997, Edwards’ claim was "as cruel as cruel can get."
"I was upset over that. He was misleading and using millions of desperate humans for political gain. Embryonic stem cells offer nothing for spinal cord injury."
Kelly, who began researching the issue five years ago and is about to start a foundation called Cures First has testified before the U.S. Senate and around the country. But unlike the people who usually testify alongside him, pleading for more money in human embryonic stem cell research, Kelly takes the opposite position.
"Although I sympathize with them, they’re in my opinion cutting their own throat. There’s so much money and so much hype involved. We’re diverting massive resources away from research that could help people in the forseeable future."
Proposition 71 in California, that over the next 10 years will pour $3 billion in state bonds into mainly human embryonic stem cell research, represents to Kelly a misguided diversion.
The media focus and Hollywood’s hailing of the potential wonders of embryonic stem cells has left the progress of adult stem cell research out of the limelight even though, he said, it is saving lives right now.
"We’ve been told we need embryonic stem cell research and therapeutic cloning to find a cure, but the bottom line is that it’s science for the sake of science," said Kelly, who emphasized he is not religious as many other opponents of human embryonic stem cell research.
In July 2004, Dennis Turner who has suffered from Parkinson’s for 14 years testified before a Senate subcommittee about his improved condition after being treated with adult neural stem cells taken from a small tissue sample in his brain.
Extreme shaking of the right side of his body disappeared, and for four years following the treatment he was symptom-free.
At the same hearing, 19-year-old Laura Dominguez told the senators how doctors had given her no chance to ever walk again after she was left paralyzed from the neck down in a car accident three years ago.
But research and consultations with medical experts led her to Portugal, where she became the first American to have tissue removed from her olfactory sinus area and transplanted into her spinal cord, the site of injury.
Now, she said in her statement, she can contract her quadriceps and hamstring muscles, move her feet, feel down to her hip level, and with assistance and the use of braces, walk 1,400 feet.
Zampieri said one of her mother’s friends who also suffered from ALS went for a new treatment in Atlanta.
Desperate for a cure, she was one of 43 patients who paid $25,000 each to have umbilical cord blood infused. The blood is rich in adult stem cells, and in animal studies the treatment has proved to slow the progress of the disease and relieve some of the symptoms, according to Dr. Mitchell Ghen who performed the treatment.
Seventeen of the patients had their symptoms reversed although Redmond’s friend was not one of them.
But to the patients’ disappointment, the Federal Drug Administration put a stop to the treatments because Ghen had not filed an investigational new drug application that the agency had initially said he did not need. Sen. Joe Lieberman questioned the decision in a letter to the FDA.
Although there are still deficiencies in diabetes treatment with adult stems cells, 80 percent of 250 patients who received adult pancreatic stem cells from adult cadavers have been free from insulin shots or pumps for more than a year and adult stem cells have reversed diabetes in animals, according to studies cited by The Center for Bioethics and Human Dignity.
"There’re extremely interesting developments and I wouldn’t be surprised if adult stem cells turn out to be more valuable than embryonic stem cells," said Alan Cherrington, president of the American Diabetes Foundation. "But it’d be silly to restrict research to only one of them."
Alzheimer’s disease is contrary to conventional thought one of the least-likely diseases to benefit from embryonic stem cell treatments, experts on the subject conceded to the Washington Post in June this year. Ronald Reagan’s death from the disease triggered an outpouring of support that had little to do with scientific evidence.
"To start with, people need a fairy tale," Ronald D.G. McKay, a stem cell researcher at the National Institute of Neurological Disorders and Stroke, told The Post. "Maybe that’s unfair, but they need a story line that’s relatively easy to understand."
Charles Jennings, the executive director of the Harvard Stem Cell Institute, dismissed as "absurd criticism" claims that the growth of human embryonic stem cells are too difficult to control and that they are likely to be rejected by a patient’s immune system as they — unlike adult stem cells — are not matched genetically with the patient.
"This is a field in its infancy. There is clear evidence of the potential in mouse research," Jennings said. "We have to careful not to raise false hope, but the sooner we start, the sooner we’ll see results."
First he would like to see federal funding extended to research of new human embryonic stem cell lines. The 22 lines that are available for research with federal funds from the National Institutes of Health registry are not adequate, he said, as they only represent a healthy, white middle class.
Harvard scientists recently announced they had developed 17 new human embryonic stem cell lines but the effort had to be privately funded.
In 2002, 30 firms spent $208 million experimenting on embryonic and adult stem cells, according to the NIH, the federal government’s leading biomedical research organization. Last year, the NIH funded $383 million in adult stem cell research and $24 million in human embryonic stem cell research.
But with California about to pump nearly $300 million a year into the industry and with its eye set on top researchers worldwide, Jennings recognizes the institute is likely to lose a few of its scientists to the West Coast. Still, he hopes Harvard is attractive enough to retain most of them.
If scientists can figure out how human embryonic stem cells differentiate into specialized cells in the body, they may be able to reverse processes that today result in devastating diagnoses for millions of Americans, said Pickering at the Biotechnology Council.
"It’s a glorious thing," she said. "But it’s hard work."
Until a real cure materializes, the ALS Association in Manchester will keep getting calls from people willing to travel the world for the slightest chance of a miracle.
"People are desperate," said Zampieri. "This is a disease I’d never wish on my worst enemy. There’s so much possibility out there that everything must be explored."
Ulrika G. Gerth can be reached at 603-742-4455 ext. 5395 or by e-mail at [email protected]