TIME Reproductive Health

Why We Still Need Fetal-Tissue Research

Planned Parenthood President Feldt
Mario Tama—Getty Images

Two sting videos that claim to implicate Planned Parenthood in the illegal practice to selling fetal tissue for a profit prompted a Congressional investigation of the organization. But it doesn’t mean that research on fetal tissue is wrong. Or that it should be stopped.

The first video released in mid-July that was secretly made by the Center for Medical Progress, a group that includes well known anti-abortion activists, centered around one question: What happens to the fetuses that result from abortions performed at Planned Parenthood? A second surreptitiously filmed video by the same group was released Tuesday. Its central question: Whether Planned Parenthood profits from the sale of such tissue. (The group insists it does not.)

Fetal tissue is valuable for medical research; the National Institutes of Health spent $76 million on fetal research in 2014, and fetal tissue has contributed to vaccines for polio, rubella and chicken pox. While recent efforts to transplant fetal tissue to treat conditions like Parkinson’s haven’t been as consistently successful, it’s still critical to scientific progress.

In the video, Dr. Deborah Nucatola, senior director of medical services, notes that the fees Planned Parenthood charges are within laws that govern fetal tissue procurement; the fees cover the expenses of handling, storing and shipping the material, not for the material itself. But in calling for the Congressional investigation into Planned Parenthood’s practices, House Speaker John Boehner said: “When an organization monetizes an unborn child — and with the cavalier attitude portrayed in this horrific video — we must all act.”

But there’s a blurring of the ethical and political lines here that is both intentional — and intentionally misleading. It’s one that’s always shadowed anything involving fetal tissue in this country. Fetal tissue research was initially allowed under specific conditions and approval by a government Ethics Advisory Board (EAB). During the 1980s, however, as controversy over the source of the fetal tissue — mostly abortions, and primarily elective ones — became increasingly politicized, a moratorium was placed on fetal tissue studies, and the EAB was disbanded. The restriction was lifted in 1993, but the work continued to be a challenge.

The ethical and political conflicts erupted again in 1998, when researchers studying excess IVF embryos and fetuses from elective abortions made breakthroughs in understanding stem cells, the pre-cells of everything that develops in the human body. The promise represented by these stem cells, which because of their developmental potential can possibly be manipulated to replace diseased or ailing cells, raised anew the questions of whether studying tissues from unused embryos and aborted fetuses was ethically — and politically — acceptable.

MORE: Why Planned Parenthood Provides Fetal Cells to Scientists

The resulting debate hampered stem cell research in the U.S. for nearly a decade, after the George W. Bush Administration prevented federal research money from being used to study excess embryos that couples had donated after IVF. Researchers wanting to pursue this work had to find private funding or leave the country, which some did. President Obama lifted the restriction in 2009 — and now, the controversy has erupted again. And as in times past, science is getting muddied by politics.

“This video is primarily aimed not at fetal tissue research but at Planned Parenthood,” says David Magnus, director of the Stanford University Center for Biomedical Ethics. “I don’t think this is about the use of tissue that is already discarded. I think it’s about abortion itself. The fact that it’s not clear whether there is any actual problem in terms of [Planned Parenthood’s] behavior highlights the fact that this is politically motivated.” Several Republican presidential candidates have also criticized Planned Parenthood’s practices, invoking the organization’s “disregard for the culture of life” and it’s “penchant for profiting off the tragedy of a destroyed human life.”

There’s no evidence on the video that Planned Parenthood makes a profit from fetal tissue. Nucatola is recorded as saying the organization pays anywhere from $30 to $100 per specimen, and that those fees cover administrative and handling costs, not the cost of the tissue itself. (Those costs are far lower than what other companies that broker exchange of tissues from hospitals and abortion clinics to those who want to study them charge.)

Women who decide to have abortions are asked after they make their decision about whether they want to donate the fetus to research. But not every woman is even given the choice. Similar to marijuana laws, in which there is a disconnect between federal and state policies governing its legality, federal law allows donation of fetal tissue if there is no payment involved, and it doesn’t influence the woman’s decision to have an abortion, while state policies may differ.

“State and local policies, as best I can tell, are patchwork, and there is no consistency across states with regard to how [fetal] tissues are used, whether or not they are allowed to be used, etcetera,” says Debra Mathews, assistant director for science programs at the Berman Institute of Bioethics at Johns Hopkins University. According to the Guttmacher Institute, six states currently prohibit fetal tissue research on aborted fetuses; three states have introduced similar statues that were struck down. And adding to the confusion, some states prohibit experimentation on “live” fetuses, attempting to make distinctions between the state of the fetus following the procedure.

Such opaque policies, and the highly contentious nature of discussing anything involving fetuses, makes it nearly impossible to fully inform women and discuss their choices in an objective way. With embryonic stem cell research, which involves use of embryos that couples donate for research, Mathews notes that there were discussions about the ethical and moral questions involved. “I don’t know that we have had robust conversations about fetal tissue,” she says. “It’s very difficult to talk about. Abortion politics in this country make it very difficult to have discussions about the use of these tissues.”

And that’s led to a situation that’s far from open when it comes to the fate of fetal tissue from abortions. “There is important research, good research, involving fetal tissues,” says Mathews. “But we have not been transparent about it. In so far as this increases the transparency, and helps us to have a conversation about the research being done, and folks are following the rules that do exist, I think that’s important.”

MORE: Here’s What Planned Parenthood President Cecile Richards Had to Say to David Koch

That may be nearly impossible, however, if conservative politicians continue to corral abortion positions and fetal research positions into the same ethical pen. Magnus notes that those opposed to abortion can still support fetal tissue research, and that the two stances aren’t as mutually exclusive from an ethical perspective. “The analogy is often made of organ procurement. ‘I’m not in favor of car accidents or people shooting each other. But if tragedies happen, and somebody is shot or there is a car accident, then being able to have something good come out of that is seen largely as a good thing.’”

One question the Congressional investigation will consider is whether the decision to donate the tissue influences the way in which abortions are performed at Planned Parenthood — if it does, that too is unlawful. But it would only be unethical if it compromises the health of the woman in any way. In the video, Nucatola discusses the fact that the way the abortion is performed should be the same for every woman, regardless of whether she agreed to donate the fetal tissue or not. But she does admit that “some people will actually try to change the presentation [of the fetus]” and that “you’re just kind of cognizant of where you put your graspers, …we’ve been very good at getting heart, lung, liver, because we know that, so I’m not gonna crush that part…and I’m gonna see if I can get it all intact.”

Planned Parenthood’s president Cecile Richards issued an apology for the tone of the discussion, acknowledging that “This is unacceptable, and I personally apologize for the staff member’s tone and statements.”

But Richards defends the way that Planned Parenthood performs abortions as ethical and legal. “Our donation programs, like any other high-quality health care providers, follows all laws and ethical guidelines. [Women and families’] commitment to life-saving research, developing treatments for diseases like Parkinson’s and Alzheimer’s is important and compassionate. And it should be respected, not attacked,” she said in a video responding to the allegations.

But as long as the dialogue about the science and the medical potential of fetal research is entwined in the political debate over abortion, that respect — and the lives that can potentially be saved from these studies — will be hard to come by.

TIME Cancer

Researchers Grow a Breast In a Dish

Technically, it’s breast tissue but it develops in a lab culture the same way it would in a teen hitting puberty. And it could help scientists to better understand how the breast develops and what happens when things go awry in breast cancer

For the first time, scientists have taken healthy breast cells from women and isolated the stem cells that can recreate major breast structures—including the milk-feeding ducts and structures that actually produce breast milk. In a new paper in the journal Development, they report that they’ve set up a model for studying how normal breast tissue develops during puberty, and, in coming months, expect to introduce mutations in these cells to study how they might develop cancer.

Starting with breast tissue from women who have had breast reduction surgery, Dr. Christina Scheel, from the Helmholtz Center for Health and Environmental Research, and her colleagues managed to isolate the few stem cells within them that are responsible for generating the new breast tissue that results in the breast’s constant remodeling during puberty, at each menstrual cycle and with each pregnancy.

Only one in about 2,000 of these cells are stem cells, but by mixing up a more nurturing culture solution, they were able to increase the growth of these cells by five-fold, and before their eyes the cells began to form the branchlike structures that serve as the duct network of the breast. With other adjustments, Scheel was also able to promote the growth of the cluster-like cells that produce milk. By labeling the initial stem cell, they saw that all of the complex structures in the breast remarkably arose from a single cell, guided by the right developmental instructions.

“[During puberty,] the normal breast tissue grows [aggressively] into the surrounding connective tissue,” says Scheel. “The cells push forward into the surrounding tissue almost like an invasive tumor but in a very controlled process.”

The fact that the normal breast tissue growth is so intense is leading Scheel to next study whether breast cancer might result from some loss of this very controlled regulation of breast tissue growth, similar to a car without brakes.

She and her team also found that when they grew the breast stem cells on a more rigid platform, the cells grew more aggressively and acted more tumor-like compared to when they were grown on a more flexible, softer framework. That may explain why women with dense breasts, which contain more connective tissue, tend to have higher rates of breast cancer. “This model will allow us to better study normal breast development, and then to understand the first steps that predispose women to developing tumors,” she says.

TIME medicine

Exclusive: Meet the World’s First Baby Born With an Assist from Stem Cells

This newborn is the first baby in the world born using a breakthrough IVF treatment

Doctors in Canada have begun a new chapter in medical history, delivering the first in a wave of babies expected to be born this summer through a technique that some experts think can dramatically improve the success rate of in vitro fertilization (IVF).

Now 22 days old, Zain Rajani was born through a new method that relies on the discovery that women have, in their own ovaries, a possible solution to infertility caused by poor egg quality. Pristine stem cells of healthy, yet-to-be developed eggs that can help make a woman’s older eggs act young again. Unlike other kinds of stem cells, which have the ability to develop into any kind of cell in the body, including cancerous ones, these precursor cells can only form eggs.

In May 2014, Zain’s mother, Natasha Rajani, now 34, had a small sliver of her ovarian tissue removed in a quick laproscopic procedure at First Steps Fertility in Toronto, Canada, where she lives. Scientists from OvaScience, the fertility company that is providing Augment, then identified and removed the egg stem cells and purified them to extract their mitochondria.

Mitochondria are the powerhouses of the cell, a molecular battery that energizes everything a cell does. Adding the mitochondria from these egg precursor cells to Natasha’s poor-quality eggs and her husband Omar’s sperm dramatically improved their IVF results. In the Rajanis’ first traditional-IVF attempt, Natasha produced 15 eggs, but only four were fertilized—just one of those matured to the point were Natasha’s doctor felt comfortable transferring it. “I knew it wasn’t the best-quality embryo, but it was what she had,” says. Dr. Marjorie Dixon, of First Steps Fertility.

With Augment, the Rajanis produced four embryos, two of which have been frozen should the couple decide to have more children. Another one became baby Zain.

It’s not currently available in the U.S., since the Food and Drug Administration (FDA) considers the process of introducing mitochondria a form of gene therapy, which it regulates. So far, some three dozen women in four countries have tried the technique, and eight are currently pregnant. All of the women have had at least one unsuccessful cycle of IVF; some have had as many as seven.

“We could be on the cusp of something incredibly important,” says Dr. Owen Davis, president of the American Society of Reproductive Medicine (ASRM). “Something that is really going to pan out to be revolutionary.”

The Next Big Thing in IVF

The technique is indeed poised to usher in the next big advance in IVF; since the first baby, Louise Brown, was born using the process in 1978, the procedure has changed little. Scientists have made incremental advances in fine-tuning the procedure, but taken together, these improvements have nudged pregnancy rates upward by only a percent or two over the course of 35 years. As it stands, the IVF success rate is about 38% for women in their late 30s and 18% for those in their early 40s. Natasha’s first IVF cycle differed little from the one that produced Brown more than 35 years ago.

Augment emerged from a breakthrough made in 2004 by biologist Jonathan Tilly, then at Harvard Medical School and now chair of biology at Northeastern University. He found that cells scraped from the outer surface of the ovary contain the precursor cells that can provide a more reliable source of energy to older eggs. “The technique addresses a void now in IVF,” says Tilly. “No cell culture can circumvent poor egg quality or an egg that is simply too tired to execute what it is capable of doing. We are taking patients with a zero percent pregnancy rate, patients who have failed IVF because of poor egg quality, and getting them pregnant.”

The Rajanis had tried for four years to get pregnant, turning to fertility drugs, intrauterine insemination, and a naturopath before trying their first attempt at IVF. Natasha became pregnant once, but miscarried a few weeks later. “I tried to remain positive, thinking there is a light at the end of the tunnel, and that a baby will be there at the end,” she says of all the misses.

What finally made the difference wasthe population of her own egg stem cells. What makes these cells so enticing to scientists is that they come from the mother herself. Mitochondria contain their own DNA, and in a controversial decision the U.K. government recently approved so-called “three-person babies,” where mitochondrial DNA from a donor is introduced into the egg of a woman with mitochondrial disease. When the egg is then fertilized and results in a live birth, it can raise ethical questions, biological concerns and conflicts about parenthood.

With Augment, the cells used—and their mitochondrial genes—are from the mother’s own ovaries. Still, the FDA requested more studies on the effect of adding mitochondria, even from the mother who provides the egg, to the IVF process. OvaScience plans to conduct 1000 cycles using Augment this year, and generate more data that will help bring the procedure to the U.S.

Because the procedure is so new, some reproductive science experts are skeptical. What’s lacking, they say, is convincing evidence comparing pregnancy rates of women undergoing Augment to those with similar infertility problems who didn’t use the technique. So far, no formal clinical trials have been conducted; the only data on the procedure comes from recent presentations by Dr. Robert Casper of University of Toronto and Dr. Kutluk Oktay from Gen-ART IVF in Ankara, Turkey, both of whom are advisors to OvaScience.

“We’re not yet sure the scientific model has proven what the outcomes would be if you use the mitochondria of a younger egg, or from an egg stem cell,” says Davis of ASRM. “It’s a fascinating concept but we just haven’t seen the studies yet.”

In the world of infertility, however, such data are historically hard to come by. A lack of regulation of most reproductive technologies—the ones that don’t fall under the jurisdiction of the FDA as either drugs, devices or gene therapy—and the dominance of business-minded scientists has rushed new methods to clinics, often before their effectiveness has been fully proven.

Tilly counters doubters with evidence from other species that these cells can do what OvaScience has said they can. Egg precursor cells extracted from ovarian tissue from rats, mice, monkeys, pigs and women, for instance, have developed into immature eggs and, in the case of rats and mice, those eggs have mature and produced viable offspring. “Mitochondria from egg precursors rejuvenate the egg to bring it back to a high quality state,” says Tilly.

That appears to be the case with the Rajanis, and time will tell whether that ends up holding true for the other women trying Augment, too. “We see Zain as a symbol of hope for all couples struggling with infertility,” says Natasha. “While the process is long, emotional and physically draining, there is light at the end of the tunnel—and that light for us is Zain.”

For more on Zain and this new approach for infertility, see the May 18, 2015, issue of TIME.

TIME Innovation

Five Best Ideas of the Day: February 17

The Aspen Institute is an educational and policy studies organization based in Washington, D.C.

1. Is the Taliban’s fracturing a sign of its demise or a possible turn to a more lethal strategy?

By Sundarsan Raghavan in the Washington Post

2. To fight cybercrime, President Obama needs Silicon Valley.

By Katie Benner in Bloomberg View

3. The FDA needs updated systems to review drugs — made from our own cells — that target cancer and more.

By Peter W. Huber in City Journal

4. Our high incarceration rate no longer reduces crime.

By Lauren-Brooke Eisen in USA Today

5. Better than an action movie: Catch a college lecture on your next commercial flight.

By Kim Clark in Money

The Aspen Institute is an educational and policy studies organization based in Washington, D.C.

TIME Ideas hosts the world's leading voices, providing commentary and expertise on the most compelling events in news, society, and culture. We welcome outside contributions. To submit a piece, email ideas@time.com.

TIME Innovation

Five Best Ideas of the Day: January 15

The Aspen Institute is an educational and policy studies organization based in Washington, D.C.

1. India and the U.S. have much to gain from strengthening their “unique but sometimes frustrating partnership.”

By Nicholas Burns in the Boston Globe

2. Big energy is betting on power storage tools that let customers take advantage of variable energy prices and stock up when rates are low.

By Ucilia Wang in Forbes

3. With class replacing race as a dividing line, some find South Africa is a “less equal place” now than under apartheid.

By Jeb Sharp at PRI’s The World

4. Preliminary research with stem cells shows how the versatile therapy could effectively cure type-1 diabetes.

By Haley Bridger in the Harvard Gazette

5. A critical piece of improving American education is improving teacher quality, and that is finally happening.

By Dan Goldhaber and Joe Walch in Education Next

The Aspen Institute is an educational and policy studies organization based in Washington, D.C.

TIME Ideas hosts the world's leading voices, providing commentary and expertise on the most compelling events in news, society, and culture. We welcome outside contributions. To submit a piece, email ideas@time.com.

TIME Cancer

Most Cancer Is Beyond Your Control, Breakthrough Study Finds

There’s a lot we can do to protect ourselves from certain cancers — don’t smoke, avoid prolonged exposure to the sun, and try not to breathe or ingest too many chemical pollutants in the air or our food. But scientists have always known that this was only part of the cancer story. There’s also heredity, but that only explains about 5% to 10% of cancer. The truth of the matter is that some tumors emerge simply at random. But how much of malignancy can be attributed to this unfortunate roll of the dice? What really causes cancer?

Christian Tomasetti and Bert Vogelstein at Johns Hopkins University believe they may have found an answer, and it’s likely to turn our understanding of cancer — and how it should be diagnosed and treated — on its head. In a groundbreaking paper published in Science, the duo describe a new factor, a tissue’s stem cells, that may explain as much as two-thirds of the difference in cancer risk among different tissues.

Many tissues in the body have stem cells that serve as factories for churning out more cells of the same kind; it’s what keeps our skin cells refreshed, and our blood and immune cells young and vigorous. This replicative power is the engine that keeps the body going, allowing tissues to replace cells as they die off. But it’s also the process behind cancer, since cancer is caused by cells that pick up mutations in their DNA when they divide — and stem cells are the only population that copy their DNA and divide to make more cells. Only a small proportion of a tissue’s cells are made up of stem cells, so Tomasetti and Vogelstein decided to map out whether the number of stem cells in a specific tissue bears any relationship to its tendency to develop cancer.

MORE Promising New Cancer Treatment Uses Immune Cells

Indeed, when they charted out the stem cell data for 31 types of tissues, they found a dramatic connection between the two — the more stem cells the tissue had, the higher its incidence of cancer over a person’s life time on average. “Think of cancer as the risk of having an accident if you are driving a car,” says Tomasetti, a biostatistician who holds positions in the department of oncology at Johns Hopkins Kimmel Cancer Center and the Johns Hopkins Bloomberg School of Public Health. “If you drive the car on a cross country trip, your risk of an accident is much higher than if you take a local trip to the grocery store. The risk correlates to the length of the trip. The trip to the grocery store might be thought of as bone cancer, which has few stem cell divisions. While the cross country trip might be more like colon cancer, which has many more cell divisions.”

In fact, the correlation held strong among cancers that were both common and more rare. The more likely those cells would divide and develop DNA errors or mutations in the process that led to uncontrolled growth, the more likely that tissue would develop tumors.

“It was quite surprising to us. We think it’s pretty big,” he says. “About 65% of cancer incidence across tissue types appears to be explained by the number of stem cell divisions.”

MORE Stem Cells That Kill

Having a detailed understanding of both how large a tissue’s stem cell population is, as well as how active it is, could be a determining factor in whether it’s likely to develop cancer. Both the brain cells that can cause glioblastoma and medulloblastoma, and the colon contain about the same number of stem cells, Tomasetti estimates — about one hundred million. But the colon stem cells divide about 6000 times on average during lifetime, compared to nearly zero for the brain stem cells. That leads to rates of colon cancer that are 22 times higher than rates of the brain tumors.

PrintCredit: C. Tomasetti, B. Vogelstein and illustrator Elizabeth Cook, Johns Hopkins

Such an explanation could also resolve some of cancer’s mysteries — why people who don’t smoke still get lung cancer in surprising numbers, or why rates of colon cancer are higher than rates of cancer in the small intestine, despite being shorter in length. One reason, says Tomasetti, could have to do with the different stem cell activity in these tissues.

This finding potentially changes the landscape of cancer. In recent decades, cancer rates have come down due to aggressive efforts to educate and motivate people to take positive steps toward preventing cancer in the first place, such as quitting smoking and avoiding the sun’s ultraviolet rays. Have those messages been wrong?

Not exactly. Tomasetti says that the study shows that it’s time to redirect that cancer strategy a bit — not abandon it. For example, he and Vogelstein propose looking at cancers in two categories, those that are primarily due to genetic bad luck, and those that are due to that unfortunate roll of the genetic dice plus environmental or hereditary factors. So melanoma, ovarian cancer, many brain cancers, lung cancer among non-smokers, the most common leukemias and bone cancers, for example, are pretty much out of people’s control. They’re the result of the random mutations caused by the stem cells dividing in these tissues — bone, blood, ovaries, brain and skin — that make mistakes that turn malignant. For these cancers, changing your lifestyle or trying other interventions to stop the cancer from occurring in the first place won’t help. But being vigilant about screening, and picking up the first signs of trouble early, can be life saving.

MORE This New Kind of Stem Cell May Revolutionize How We Treat Diseases

For the other type of cancers, those that are the product of both stem cell mutations and heredity or other exposures, continuing with proven prevention methods, which include screening in cases of inherited disease, as well as quitting smoking and reducing exposure to radiation and carcinogens, is still critical. That’s what has lowered rates of lung cancer among smokers, for example, and colon cancer among those with hereditary disease.

“Everything we know about altering lifestyles to prevent cancer from the environmental point of view we absolutely need to continue doing,” says Tomasetti. “If anything it puts more stress on the need to spend even more money on early detection. It may be the key tool for quite a few cancer types.”

Tomasetti admits that two common cancers are missing from the study — breast cancer and prostate cancer. That’s because knowledge about their stem cell populations, and how often those tissues renew, isn’t quite as solid as it is for tissues such as colon. “We are working on that,” he says. “We hope this type of work highlighting the importance of self renewal will cause others to investigate these stem cell populations in more detail as well.”

In the meantime, he stresses that while we may not be able to prevent the tumors from forming, it’s still possible to treat them and potentially save lives by finding them early and removing them or using chemotherapy or radiation to keep them under control. “My biggest fear is that people will say forget about it, and then do nothing. The opposite is true. We need to do everything we did before, but we want to do it even more than before,” he says.

Read next: Your Chances of Surviving Cancer May Depend on Where You Live

TIME Cancer

Most Types of Cancer Just ‘Bad Luck,’ Researchers Say

488635633
JUAN GARTNER—Getty Images/Science Photo Library RF Lymphocytes and cancer cell

Two thirds of cancers could be explained as biological misfortune

Researchers have found that bad luck plays a major role in determining most types of cancer, rather than genetics or risky lifestyle choices such as smoking.

The results, published in the journal Science on Thursday, found that random DNA mutations that amass in the body when stem cells divide into various tissues cause two thirds of cancers.

After examining 31 cancer types, researchers found 22 were from mutations in stem cells that could not be prevented.

Cancers that could be explained with biological bad luck included pancreatic, leukemia, bone, testicular, ovarian and brain cancer.

But the researchers say lifestyle choices such as avoiding smoking, eating healthily and staying out of the sun will help to prevent certain cancers, just not all of them.

Read next: Medicine Is About to Get Personal

Listen to the most important stories of the day.

TIME medicine

This New Kind of Stem Cell May Revolutionize How We Treat Diseases

Scientists have created a new type of stem cell that could speed treatments for diseases and make them safer

Ever since Japanese researcher Shinya Yamanaka found a way to treat skin cells with four genes and reprogram them back to their embryonic state, scientists have been buzzing over the promise of stem cell therapies. Stem cells can be coaxed to become any of the body’s cell types, so they could potentially replace diseased or missing cells in conditions such as diabetes or Alzheimer’s. And Yamanaka’s method also meant that these cells could be made from patients themselves, so they wouldn’t trigger dangerous immune rejections.

Now scientists led by Dr. Andras Nagy at Mount Sinai Hospital Lunenfeld-Tanenbaum Research Institute in Toronto report an exciting new advance that could push stem cells even closer to the clinic. In a series of papers in the journals Nature and Nature Communications, the group describes a new class of stem cell, which they called F class, that they generated in the lab.

The F class cells, says Nagy, have a few advantages over the Yamanaka-generated induced pluripotent stem cells, or iPS cells. While the iPS cells are created by using viruses to introduce four genes that reprogram the cells, Nagy’s team relied on a technique they developed several years ago using transposons—small pieces of DNA that can insert themselves into different parts of a genome. Unlike viruses, these transposons can be popped out of the genome if they’re no longer needed, and they don’t carry the potential risk of viral infection.

MORE Stem-Cell Research: The Quest Resumes

Nagy’s team found that the transposons were much more reliable vehicles for delivering the reprogramming genes exactly where they were needed to efficiently turn the clock back on the skin cells. What’s more, they could use the common antibiotic doxycycline to turn the four genes on and off; adding doxycycline to the cell culture would trigger the transposons to activate, thus turning on the genes, while removing the antibiotic would turn them off.

In this way, says Nagy, he was able to pump up the efficiency of the reprogramming process. Using the Yamanaka method, it was hit-or-miss whether the viruses would find their proper place in a cell’s genome, and more uncertainty over how effectively it could direct the cell to activate the four reprogramming genes. “F class cells are much more similar [in the culture dish], like monozygotic twins while iPS cells are more like brothers and sisters,” he says.

That consistency is a potential advantage of the transposon method, since any stem cell-based treatment would require a robust population of stem cells which can then be treated with the proper compounds to develop into insulin-making pancreatic cells to treat diabetes, or new nerve cells to replace dying ones in Alzheimer’s, or fresh heart muscle to substitute for scarred tissue after a heart attack.

MORE Stem Cell Miracle? New Therapies May Cure Chronic Conditions like Alzheimer’s

Nagy’s team also described, with the most detail to date, exactly how mature cells like skin cells perform the ultimate molecular feat and become forever young again when exposed to the four genes. They analyzed the changes in the cells’ DNA, the proteins they made, and more. “It’s similar to high definition TV,” he says. “We see things much better with much more detail. We expect that having that high resolution characterization will allow us to better understand what is happening during this process at the molecular level. And obviously that better understanding is going to affect what we can do with these cells to make them better, safer and more efficient in cell-based treatments in the future.”

That may be years away yet, especially since some experts say that transposons may pose their own risk of wreaking DNA havoc on a cell’s genome. But having another type of stem cell that could potentially churn out healthy cells and tissues to replaced diseased ones is a welcome development.

TIME medicine

‘Bubble Boy’ Disease Cured With Stem Cells

Alysia Padilla-Vacarro and daughter Evangelina on the day of her gene therapy treatment. Evangelina, now two years old, has had her immune system restored and lives a healthy and normal life.
Courtesy of UCLA Alysia Padilla-Vacarro and daughter Evangelina on the day of her gene therapy treatment. Evangelina, now two years old, has had her immune system restored and lives a healthy and normal life.

Researchers have treated more than two dozen patients with a treatment made from their own bone marrow cells

Alysia Padilla-Vaccaro and Christian Vaccaro owe their daughter’s life to stem cells. Evangelina, now two, is alive today because she saved herself with her own bone marrow cells.

Evangelina, a twin, was born with a severe immune disorder caused by a genetic aberration that makes her vulnerable to any and all bacteria and viruses; even a simple cold could be fatal. But doctors at University of California Los Angeles (UCLA) Broad Stem Cell Research Center gave her a new treatment, using her own stem cells, that has essentially cured her disease. She’s one of 18 children who have been treated with the cutting-edge therapy, and the study’s leader, Dr. Donald Kohn, says that the strategy could also be used to treat other gene-based disorders such as sickle cell anemia.

Known to doctors as adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID), it’s better known as “bubble boy” disease, since children born with the genetic disorder have immune systems so weak that they need to stay in relatively clean and germ-free environments. Until Evangelina and her sister Annabella were 11 months old, “We were gowned and masked and did not go outside,” says their mother Alysia Padilla-Vaccaro. “Our children did not physically see our mouths until then because we were masked all the time. We couldn’t take them outside to take a breath of fresh air, because there is fungus in the air, and that could kill her.”

Both parents wore masks at work to lower the chances they would be exposed to germs that they might bring back home. And they took showers and changed clothes as soon as they entered the house.

MORE: Gene-Therapy Trial Shows Promise Fighting ‘Bubble Boy’ Syndrome

SCID is caused by a genetic mutation in the ADA gene, which normally produces the white blood cells that are the front lines of the body’s defense against bacteria and viruses. The Vaccaros decided to treat Annabella in the same way that they cared for Evangelina; “They were crawling and playing with each other, and every toy they sucked on, they stuck in each other’s hands and each other’s mouth, so we couldn’t take one outside to have a grand old time and potentially bring something back that could harm her sister,” says Padilla-Vaccaro.

Courtesy of UCLAChristian and Alysia Padilla-Vaccaro and their healthy twins Annabella (left) and Evangelina. Now with a newly-restored immune system, Evangelina lives a normal and healthy life.

The only treatments for SCID are bone marrow transplants from healthy people, ideally a matched sibling; the unaffected cells can then repopulate the immune system of the baby with SCID. But despite being her twin, Annabella wasn’t a blood match for her sister, nor were her parents. Padilla-Vaccaro and her husband, Christian, were considering unrelated donors but were concerned about the risk of rejection. “We would be trying to fix one problem and getting another,” she says.

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That’s when the doctors at the Children’s Hospital at Orange County, where Evangelina was diagnosed, told her parents about a stem cell trial for SCID babies at UCLA, led by Dr. Donald Kohn. “As soon as they said trial, I thought, ‘my kid is dead,” says Padilla-Vaccaro of the last resort option. But a dozen children born with other forms of SCID—in which different mutations caused the same weak immune systems—who were successfully treated by Kohn convinced the couple that the therapy was worth trying. Kohn had one spot left in the trial and was willing to hold it for Evangelina until she matured more. Born premature, she was diagnosed at six weeks old and needed more time for what was left of her immune system to catch up to weather the procedure.

When she was two months old, Evangelina was admitted to UCLA and had bone marrow drawn from her tiny hip. It contained the stem cells that go on to develop into all of the cells in the blood and immune systems. Kohn treated them with gene therapy, co-opting a modified virus to carry the healthy ADA gene so it could infect the stem cells from Evangelina’s bone marrow. The idea was that by transplanting these healthy ADA-containing cells back into Evangelina, she would soon be making her own healthy immune cells. And because they were made from her own cells, her body wouldn’t reject them.

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“After the transplant of this miraculous tube of stem cells, which literally took five minutes, we had to just wait and see for a good six weeks,” says Padilla-Vaccaro. “The week after Christmas [in 2012], Dr. Kohn came in and told me, ‘It worked.’ It worked. Those words…besides the birth of my children, that day will always be the best day in my life.”

The success was a long time coming for Kohn as well. His group has been researching the best way to treat SCID with gene therapy for more than two decades. In the first trial, in 1993, they used cord blood, treating it with the healthy ADA gene and hoping enough of them would “take” to rebuild an immune system. It didn’t work.

In 2001, they tried a different way of delivering the precious gene in four patients. That failed as well.

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Then, in 2009, he and his team began the trial that Evangelina eventually joined. After reading about a group in Italy that completely obliterated the patients’ existing immune systems with chemotherapy first, before introducing the new bone marrow cells to repopulate the system, Kohn tried that strategy on 10 babies. “Of all the patients we treated, all have had good immune reconstitution,” he says. “Within a month or two, we start seeing cells appear in the blood that are making the missing gene. When they are six months old or so, their immune systems are good enough for them to go out and not be protected, and by age two, they are pretty stable—their immune systems are reset.”

That’s where Evangelina is now, able to finally enjoy the world outside her home and the hospital. She got her first kisses from her parents when she was 18 months old. “My worry was that I couldn’t raise my daughter without her sister,” says Padilla-Vaccaro. “Now I don’t have to.”

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