Today, a slight curling of her hands is the only trace of Molly Foley's severe illness …Shortly after Molly Foley and her husband returned from a 2006 trip to Ireland, she noticed that her hands were stiff and tingly. Darn. Carpal tunnel, Foley thought, blaming the eight hours a day she spent at her computer as an economic development consultant in Rock Island, IL. Foley, then 33, made an appointment with her general practitioner, who agreed that Foley probably had a repetitive-stress injury and prescribed wrist splints. Foley wore them religiously, but her fingers quickly became so stiff, she couldn't straighten out her hands. Worse, the rigidity was spreading to other parts of her body - her shoulders, her back, even her face - and her skin was becoming hard and shiny. Foley returned to her doctor, who now suspected rheumatoid arthritis and referred her to a rheumatologist, a physician specializing in autoimmune disorders.
The rheumatologist took one look at Foley's skin and said he didn't think it was arthritis. He thought what she had was worse - much worse. Blood work, a skin biopsy, a CT scan of Foley's chest, and an echocardiogram of her heart confirmed his diagnosis. "I had systemic scleroderma," Foley says.
Scleroderma is a chronic connective tissue disease. It occurs when the body turns on itself, destroying the smallest blood vessels and smooth muscle cells and replacing them with collagen and scar tissue. The collagen builds up, and affected organs harden. Scleroderma has no known cause and no conventional cure. Without treatment, Foley was given less than a 50% chance of surviving five years. And even with treatment - aggressive immunosuppressants - more than a third of patients die anyway, she learned. "So three years after my wedding, I started planning my funeral," Foley says.
But as she pecked out mourning instructions that she hoped would help her loved ones - a service at the church where she and her husband had been married, a big "happy" wake, bagpipes playing "My Wild Irish Rose" - Foley made appointments with two doctors working along what appeared to be a fine line between science and science fiction. "They were experimental stem cell studies, where the doctors were 'reprogramming' the immune system," Foley says. "Of course that sounded great. And of course I wanted hope. But I felt like I had to be very, very careful. Because I really wasn't interested in false hope - for me or my family."
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Both doctors Foley consulted were well-credentialed and involved in research at universities with rigorous oversight. She discussed the studies with her physicians and her family, and she got info from staff at the Scleroderma Foundation, a group she had contacted shortly after her diagnosis. Then she looked up the medical literature on both, checking that the work had been published in peer-reviewed journals (meaning it had been vetted by other doctors). "I'm just a super-thorough person," Foley says. "I can't help it."
After her considerable research, which included making sure that she met all the criteria to participate, Foley chose to enroll in a study conducted by Richard K. Burt, M.D., chief of the Feinberg School of Medicine's division of immunotherapy at Northwestern University in Chicago. The main reason Foley picked it: Patients in the control group (who did not get the new treatment) were able to "cross over" and receive a stem cell transplant if after one year the disease progressed. Since assignments are always done anonymously and randomly, "there was a 50% chance of winding up in the control group," Foley says. "And I didn't see the point of signing up to help test an experimental treatment if I wasn't assured of the chance to at least try it."
Dr. Burt agrees that all patients should have that option. Right now, he explains, the standard is that you're given a drug, and then you have a life of managing your illness. "But stem cell treatment is a paradigm shift in medicine. You treat one time, then use medication, and eventually stop all therapy unless the disease comes back."
Sure enough, Foley wound up in the control group. For six months, she received high doses of a conventional treatment for scleroderma, the immunosuppressant Cytoxan. She did not get any better. Instead, she got worse. She couldn't walk more than a few feet without stopping. She couldn't bend to put on her own socks. Her lung function dropped below 50%, and she was facing heart trouble.
Finally, in March 2009, Foley got to try stem cell therapy. "I couldn't believe how simple it was," she says. First, in a procedure that took two and a half hours, doctors used a dialysis-type machine to extract stem cells from Foley's blood. Then, two weeks later, she returned to the hospital and underwent five days of high-dose immune suppressant therapy to completely knock out her malfunctioning immune system. A week after that, doctors brought Foley's stem cells back to her room - the same cells, not treated in any way. She asked a hospital minister to bless what looked to be a pint-size bag of blood. As soon as the prayer was over, a nurse hooked Foley up for an ordinary transfusion. The whole procedure took about 20 minutes.
"I started feeling better immediately," Foley says. "An occupational therapist came to work with me during the 10 days I had to stay in the hospital. Right away, we both noticed that the skin on my hands loosened up. I could move my fingers. I could stand and sit without joint pain. When I got out of the hospital, I started physical therapy. By June, I could ride a stationary bike for 10 minutes. By July, I could get my hands over my head. And late that summer, I got out my golf clubs and started chipping balls in my backyard."
Treatments like Foley's are at the cutting edge of medicine, taking these most basic biological units - essentially, cells that don't yet have a specific identity (muscle, skin, hair) - and using them to create a healthy immune system or, in other cases, to repair diseased tissue. But patients have actually been getting stem cell treatments for more than 40 years in the form of bone marrow transplants, bone marrow cells being the only kind doctors knew how to retrieve and easily transplant for many years. While the approach became highly successful, it was limited to treating a few cancers and blood disorders. Starting in the late 1980s and 1990s, however, scientists developed techniques to collect stem cells from a variety of sources (including newborns' cord blood) as well as to modify an individual's own stem cells and grow them into new tissue; over the past decade, doctors have been trying out this technology on a host of different diseases and conditions, from diabetes to Parkinson's disease. Today there are nearly 1,800 FDA-approved stem cell clinical trials being conducted in the United States.
But as research expanded, so did the controversy. Some stem cells are obtained from human embryos created for in vitro fertilization use; the embryos are destroyed in the process of harvesting the stem cells. In 2001, to quell the controversy, President George W. Bush offered a compromise, restricting federal funding for research to already-existing lines of stem cells. But many scientists found that too restrictive and sought private funding to continue their work. Then, in 2009, President Barack Obama lifted the restriction.
So far, only a handful of clinical trials have used embryonic stem cells, though many scientists believe they're more promising than those obtained from other sources because they're the most adaptive, able to grow into virtually any type of body cell - nerve, heart, cartilage - explains Doug Melton, Ph.D., codirector of the Harvard Stem Cell Institute. Others are less flexible: Liver stem cells, for example, can grow only into liver cells.
There's also controversy about some practitioners of stem cell medicine. Beyond the legitimate doctors testing experimental treatments, there are many charlatans who are offering nothing more than false hope - and possibly danger. It can be extraordinarily difficult to discern the hope from the hype, and desperate patients and their families of-ten wind up making life-altering medical decisions - and spending huge amounts of money - based only on Google searches and misplaced faith.
A "Miracle" in China?
Macie Morse (left), with mom Rochelle, has had three stem cell treatments in ChinaRochelle Morse first learned about stem cells seven years ago. She was doing her quarterly Internet search - "Optic Nerve Hypoplasia" and "Treatment" - when she saw a story about frogs' ability to regenerate optic nerves. Oh, wow, Morse thought. Maybe someday stem cells will help my daughter!
Morse's older child, Macie, was born with optic nerve hypoplasia (ONH), a condition in which the optic nerve - which transmits visual information from the retina to the brain - is underdeveloped. Macie was diagnosed in 1993, when she was 2 months old. "The ophthalmologist said, 'She is blind. She will always be blind. It is what it is, and there is nothing you can do.' I remember it all very well," Morse says, "because it was the first time I used the F word in front of my mother. I said, 'F - - - you. I believe in God, and I believe in miracles.' I'm sure the doctor thought I was in denial."
It would not have been an unreasonable conclusion. Macie had her first formal sight test when she was just a toddler, and her vision turned out to be 20/400 in her right eye; in her left eye, she could detect only light.
Macie's parents, Rochelle and Greg, as well as her younger brother, Easton, tried to keep the girl's home life happy and normal. "We watch TV, play games, and have too much to clean up like every family," says Morse, a high school office manager in Fort Collins, CO.
But even wearing her monocular - a sort of telescopic eyepiece - Macie bumped into things and fell. "We had lots of 'owies,' " says Morse. "At school, kids would trick her. So, outside our home, things could be very hard."
But Morse never wanted her daughter to have a crutch or a label: "That's why I didn't have Macie learn Braille or get a guide dog or use a cane." She didn't even tell her daughter she was blind. "I just told her that she didn't see as well as most people."
The explanation worked reasonably well until January 25, 2008, Macie's 15th birthday. That morning, her giggly, dimpled daughter bounded down the stairs and shouted, "Yay! Now I can get my driver's permit!"
Morse recalls having had to work very hard not to cry. "I carefully said, 'Macie, I know I've told you that you don't see like the rest of us. But what I haven't said is that it is pretty bad. So the whole driving thing is going to be postponed.' I didn't say no, because I'd never done that," Morse says. Still, she could tell that her daughter was crushed, trying to distract herself by watching TV the only way she could - about six inches from the family's 46-inch screen.
So Morse attempted to distract herself, too. As she had so many times before, she went to her computer and typed in her two search terms. Astonishingly, this time something new came up. A 6-year-old girl from Missouri with optic nerve hypoplasia had gone to China for a transplant of stem cells derived from umbilical cord blood and administered by spinal infusion and IV. Before her treatment, the child hadn't even been able to detect light. Now she was telling her mother she was beautiful.
For the first time in 15 years, Morse had hope for Macie. "But I didn't want to get her excited about something that might turn out to be bogus," Morse says. So with the help of Easton, then 13, she quietly investigated the stem cell treatment being offered by China's Beike Biotech. Morse says that she spoke to someone at the University of Pittsburgh Medical Center who told her that the worst that could happen was nothing. But a spokesman for the university's Stem Cell Research Center says the institution has a policy of not commenting on any outside research or procedures. Morse also examined the online testimonials of the child from Missouri and another from Romania, then read further on the Beike site and contacted the center directly. The center did not offer "gold-standard" randomized study results or peer-reviewed articles in medical journals about its procedure. In fact, the site was vague about how the infusions (given in the spine and into a vein) even worked. "They said that other patients had had good results," Morse says, "but they made no guarantees."
Morse acknowledges that the company could seem "shady." But she points out that she has never had the one thing that Foley had - a treatment option in the United States. "I knew it was experimental. I knew there was no promise. I knew some people thought it was 'out there.' But finally someone was giving us hope."
"Mom! I Can See!"
As soon as Morse told Macie about the China treatment, Macie wanted to start packing. The issue was money. Beike Biotech charged $24,000 for the treatment. In spring 2008, the Morses began fund-raising. They hosted golf tournaments and poker events. They passed a bucket during hockey games. And the kicker on the local indoor football team accepted pledges for every goal he made that season.
The Morses wound up receiving about half of what they needed. They came up with the rest themselves, and in July 2008, Macie got her first passport and flew with her mother to Qingdao, China. Both took up residence in a single room in a local hospital, and every few days for four weeks, Macie spent about an hour getting an infusion of stem cells harvested from Chinese umbilical cord blood. She also had acupuncture and something called electric-wave therapy around her temples - for energy to "draw in" the stem cells.
One afternoon shortly after her third dose, Macie ran into the room where Morse was on Skype with her own mother back home. "Mom! Mom!" she screamed.
Morse turned to scold her daughter for interrupting, but Macie yelled again. "You have to see this!"
Macie had been journaling in a lounge area when she suddenly realized she was sitting like a normal person. Before, in order to see what she was writing, Macie had had to hunch over so her nose was just a few inches from the paper. But she wanted her mom to test her to make sure it wasn't "some placebo thing," a phrase she'd heard a doctor use on a TV show.
In regular-size letters, Morse printed Macie's nickname - "Chicken Legs" - adding, "We're going home soon!"
Macie read it from about a foot away. Mother and daughter screamed and fell into each other's arms. Suddenly, Macie pulled back, peered into her mother's face, and said, "Mom, your eyes are green."
Both started sobbing. Macie, they believe, was seeing her mother's eyes clearly for the first time.
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Barrett Katz, M.D., the Frances De Jur chair in ophthalmology at the Albert Einstein College of Medicine in New York City and a professor of ophthalmology, neurology, and neurosurgery, does not believe that is likely. "Stem cells do not have a homing device that allows them to be injected into the cerebrospinal space and travel to where they are needed," he explains. "Current stem cell therapies are very site-specific. To treat the optic nerve, you would have to go into the eye." Scientists have shown in mouse studies that transplanted stem cells may migrate into the retina and perhaps evolve into mature photoreceptors, Dr. Katz adds. "But whether such implants can be made in humans or whether they would improve vision has not been demonstrated."
During the Morses' six-week stay in China - they remained there two weeks after the treatments - the doctors at Beike did some tests on Macie. But they were unlike any she'd had in the United States, and Morse did not know what the exams had shown. So they were eager to get home to Macie's ophthalmologist in order to verify her improvement. In his office, the doctor dilated Macie's eyes, peered at her optic nerve, and then gave his verdict: "Just what I expected. No difference."
Macie and her mom were crushed, but not for long. "I decided to look for another doctor, one who was at least open," Morse says. They wound up in the office of a local optometrist, a woman specializing in treatment for visually impaired people, but not a medical doctor. "I don't really know anything about this," she told Morse. "But I am here to support you and your daughter." She tested Macie's vision as 20/80 in her right eye (20/30 with her monocular), a big improvement from the 20/400 Morse reported from earlier testing. In her left eye, where Macie had had only light detection, the optometrist measured 20/800. But Dr. Katz is skeptical. Although he doesn't know the particulars of Macie's case, he points out that "measured vision can be highly subjective and difficult to quantitate with precision." He compares it to weighing oneself, stepping on a scale several times. "You can gain or lose a pound or two by standing a certain way on the scale, but you are not really losing weight by doing so." Vision-test results, he says, vary according to light, distance, fatigue, even unconscious cues from the examiner.
Based on her testing, the local optometrist signed a form certifying that Macie met the Colorado vision requirement to operate a car. A day after her 16th birthday, she got her driver's permit.
Continue reading more about the new science behind stem cell research and its ability to treat incurable diseases and the fraud and false hope that also surrounds this miracle medicine.
-by Amanda Robb
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