When Braxton Tackett turned 18 months old, he began playing with his toys differently. He would line them up and continuously spin them around. He would tap them on tables, walls and floors, so they made sounds. As these distinct behaviors, known as stimming, began, Tackett’s eye contact and talking faded.
“We noticed things were different,” explained Carrie Tackett, Braxton’s mother. One year later, he was diagnosed with autism spectrum disorder, a range of brain development conditions that affects communication skills and social interaction. According to the Centers for Disease Control and Prevention, one in 59 children in the U.S. is diagnosed with the condition.
“We have since been living with him at that level,” said Carrie Tackett, who resides in Twinsburg, Ohio, with her family of three. “In my head, I keep picturing my son being able to talk to me, or eating food he has never eaten before, or using the bathroom on his own.”
In 2017, Carrie Tackett applied to enroll her son in the only U.S. clinical stem cell trial for autism at the time, which was run by Duke University in North Carolina. While waiting almost two years to find out if Braxton, now 7, would be accepted, she joined a group of parents on Facebook discussing stem cell therapy for autism.
Some studies have suggested that autism has been correlated to increased inflammation in the brain. Duke’s research was exploring this theory by targeting inflammation with stem cells to examine whether it would improve behavioral outcomes in children with the condition. Parents in the Facebook group began posting about getting rejected from the clinical trial at Duke and turning to the Stem Cell Institute in Panama City for treatment, which was also using stem cells in an attempt to decrease inflammation and symptoms of autism.
After hearing that Duke was only accepting children who demonstrated the ability to communicate verbally, Carrie Tackett knew she had to do more and so booked two flights for $1400 to Panama on Jan. 5.
Although stem cells are heralded as having the potential to instigate lifesaving medical interventions, political regulations regarding the moral dilemma of stem cell use, such as the destruction of embryos, have slowed the research and approval progress in the U.S. As a result, only one stem cell type – cord blood – is approved for treatment in the States, which has prompted some parents to travel to different countries in pursuit of other stem cells treatments for conditions such as autism.
“There are some places in the U.S. to get unapproved stem cell therapies, but it is more common in countries like Panama, Mexico and China,” said Lorenz Studer, the founder and director of the Center for Stem Cell Biology at Memorial-Sloan Kettering Cancer Center in New York City. He refers to this trend as “stem cell tourism.” Not surprisingly, as regulations on stem cell research in the U.S. become stricter, the industry of “stem cell tourism” continues to grow.
The Stem Cell Institute in Panama has been studying and performing stem cell treatments for autism for about nine years. The five day-long therapy consists of a physical examination and blood testing on the first day followed by four days of injections of human umbilical cord mesenchymal stem cells. Treatments range from $15,825 to about $17,000 for children and include a stay at the Hilton hotel with transportation to and from the airport, VIP airport gate services and accelerated customs clearance upon arrival to Panama.
The number of stem cells delivered in the 20-minute procedure is based on the child’s age, for which there is no restriction, and weight. When administered intravenously, mesenchymal stem cells can produce anti-inflammatory effects, which in turn can ameliorate some symptoms of autism, such as lack of social interaction and loss of expressive vocabulary.
In the treatment office at the Stem Cell Institute, which overlooks the sparkling Panama Bay, Tackett’s son received 60 million stem cells with a price tag of $16,850. “You’re in the office for maybe 10 minutes if you’re kid isn’t having a meltdown or screaming their head off,” said Tackett. By the third and fourth day, Tackett said, she didn’t have to hold his second arm down anymore. There were no painful side or after effects, other than Braxton having an irritable mood on the first day. On the other days, Tackett explored the city and went swimming with her son. Two months later, Carrie Tackett sees cheerful changes in his behavior. Braxton now eats foods he previously wouldn’t touch, like pretzels and cheese. He purposely looks at other people in the eye and is even starting to pronounce words including “hi.”
“It’s no longer like ‘Groundhog Day’ for me anymore,” she said, referring to the classic Bill Murray comedy about a man caught in an endless time loop of the same day, over and over. “Every day I feel like it’s not going to be the same day as him not talking, eating the same food or using a diaper,” said Tackett. “It feels good. It really does.”
Bans are preventing research
The main attraction of stem cells has been their regenerative approach to treating degenerative conditions, by rebuilding tissue where it dies off. While most medications can control symptoms and slow the dying of tissue, they don’t target the root cause of the disease.
“They don’t just treat symptoms. They have the ability to rewind the clock and help people recover lost function. It can change the way we think about medicine and the way we treat people,” said Kevin McCormack, senior director of public communications and patient advocate outreach for California’s Stem Cell Agency, or CIRM.
CIRM is an organization that provides funding for stem cell-related research projects in California. It currently offers over $40 million to fund U.S.-based stem cell projects seeking to better understand autism and finding novel therapies.
The science of stem cells is a relatively new field. It was only in 1998 that stem cells were first isolated from human embryos and grown in a lab.
In 2001, President George W. Bush allowed tax dollars to fund research on a small number of existing stem cell lines that had been derived from embryos. At the same time, he limited federal funding for research on human embryonic stem cells because embryos were being destroyed in the process.
Some of those restrictions, including federal funding of embryonic stem cell research, were lifted in 2008 under Barack Obama’s administration. However, Congress did not remove its legislative restrictions of creating new stem cells lines.
Then, in Sept. 25, 2019, President Donald Trump introduced bans on stem cell work that limit a wider realm of research not closely studied in previous years – fetal tissue – due to anti-abortion beliefs. Fetal tissue is obtained from a dead human embryo or fetus. According to the U.S. Department of Health and Human Services (HHS), in 2019, the National Institutes of Health (NIH) spent $109 million to fund human fetal tissue research.
Even though the stem cells in fetal tissue are different from embryonic stem cells and are not related to the mesenchymal cells used in Panama, bans are preventing researchers in the U.S. from advancing science to find possible treatments for a number of medical conditions, including autism. As a result, some researchers have seen their projects canceled and are worried about the consequences of running out of money.
“Stem cells are not just life changing, but lifesaving,” said McCormack. “[A ban] pushes people away from doing research in that area because funding is limited.”
The Stem Cell Institute sits in the middle of the financial district in Panama City, on the 63rd floor of a bank building. Even though it seems very accessible to Panamanians, only a five-minute drive from downtown, few receive treatments there. “Stem cell treatment in Panama is in a very gray area. In order to make a treatment, it has to go through research and studies, and we don’t have enough of that,” said Dr. Susana De León, a psychiatrist and founder of The Bridge Center, a Panama City- based team of providers focusing on mental health and education in Panama.
Dr. Neil Riordan in 2007 founded the Stem Cell Institute, which focuses on using mainly mesenchymal stem cells derived from human umbilical cords to treat human conditions including autism, spinal cord injury and autoimmune diseases. It began as a start-up in the City of Knowledge, a management platform that boosts innovation and collaboration between organizations and research institutions in Panama City.
Their treatments were the first of their kind in the stem cell field and grew fast under limited regulation in the company incubator. In 2008, Riordan established Medistem Panama, Inc., a stem cell laboratory and research facility licensed by the Panama Ministry of Health that prepares stem cells for the Stem Cell Institute.
Since the stem cell research for autism is lacking in other countries, due to conservative governments, a majority of people who visit the Stem Cell Institute are foreigners from the U.S., India, Australia and Europe. Parents come to a point where they want to do more to help their child with autism, even if it’s against government recommendation, so they decide to go to Panama. “There’s never a right or wrong answer for your child,” said Tackett. “You just have to do what you feel is best for your kid.”
However, experts in Panama believe the standard of care regarding autism spectrum disorder doesn’t have enough data yet to prove stem cells can treat the condition. “Some of the most renowned doctors in the Panamanian Medical Association are really worried about the legislation of treatment that is only experimental at this point. We need more data to prove this works and it doesn’t hurt people,” said De León.
Although the stem cell treatments in Panama have undergone safety studies conducted by the Stem Cell Institute and supervised by the local Ministry of Health, Studer said nearly none of the mesenchymal stem cell therapies have gone through rigorous assessments in the U.S., therefore they are not available as approved treatments. “It’s a minimally money-related product. There’s not much science behind it,” said Studer.
This lack of approval and risk has parents worried people will accuse them of hurting their kids and some are even afraid to talk about it publicly in case it paints stem cell treatments in a negative light. But they are convinced it works, because parents like Tackett sing the positive results to their networks and on their social media accounts.
Even though there are many parents out there looking for ways to help their children, the treatments need to be both effective and not put patients at risk, experts say. “On the one hand, you want to protect people,” said Alan Regenberg, director of outreach and research support at Johns Hopkins Berman Institute of Bioethics. But he cautioned that sometimes, conditions can also get worse because of stem cell treatments. “It can hurt them,” he cautioned.
Not an easy decision
When Sue Rimovsky told her pediatrician she was considering stem cells to treat her 2-year-old daughter’s autism, she received negative feedback. “He was trying to shut me down. I wanted to believe him as a doctor but I said my kid is in there and I’m getting her back,” said Rimovsky of Manhattan, Illinois.
Similar to most children with autism, Rimovsky’s daughter, Abby, hit all her appropriate milestones up to the 15-month-old mark. Within the following three months, her vocabulary dwindled from 20 words to none. When her parents interacted with her, she no longer had a purposeful look, but a blank stare. “She was completely gone. That’s the only way I can explain it. She was a different person,” explained Rimovsky.
In the U.S., behavioral therapies, such as Applied Behavioral Analysis, or ABA, are most commonly recommended for addressing autism. ABA is based on teaching social, motor and verbal behaviors through positive reinforcement. Rimovsky tried this with her daughter but didn’t think it was enough.
Yet, the decision to go to Panama wasn’t easy for her. There were many emotions involved in making the choice, knowing that this type of stem cell treatment is not legal in the U.S. “It’s a huge headline: ‘Not FDA approved’. You start thinking to yourself ‘why do I have to go to another country?’”
Money was another factor. Even though Abby was a candidate for treatment in Panama, Rimovsky pondered the trip because her family couldn’t pay for it by themselves due to the expense. But with the help of a GoFundMe page, she was able to raise enough money within a month. In April 2018, she and Abby took the five-hour flight to Panama.
At the Stem Cell Institute, Abby received 40 million stem cells over the course of four injections in four days. “When you become a patient of theirs, you’re very well taken care of. We were treated like royalty,” said Sue Rimovsky. Only three weeks after their return to the U.S., Abby, now 4, spoke for the first time since she was 18 months old. Sue Rimovsky found the immediate results promising, so she kept the GoFundMe page open and returned to Panama six months later. Between the two trips, she raised over $33,000.
Even though Rimovsky doesn’t believe it was a magic cure, she doesn’t regret her decision to go to Panama. “Do I believe we stopped a lot of bad behaviors that could have arisen in her life? I do. Does she still have hers? Yes. Have they gotten calmer?” Rimovsky said before treatment, Abby couldn’t remain still enough to sit down to eat, but now she can sit in a chair for an hour. “It has kept her more comfortable and relaxed in her body.”
Rimovsky is one of 10,000 members in the Facebook group on which parents support one another through the process of seeking out stem cell therapy for autism in the U.S. and abroad. In addition to sharing their experiences and results, they also offer the names of clinics and stem cell studies that have been performed in the autism field.
It was in one of these groups Rachel Ortiz in Bangor, Maine, heard about treatments in Panama City for autism. When she first applied, in 2015, to one of the Stem Cell Institute’s clinical trials for her 12-year-old son, Connor, he was rejected. But one day, she received a phone call inviting Connor, now 17, to be part of a new clinical trial.
Although the stem cell treatment expense was covered by the trial which involved four transfusions and two post-treatment visits, Ortiz still had to pay for six round-trip flights for two people to Panama, along with $7,200 for the lab. “For me as a half-time professor and being a caregiver of my child, I thought I had to do fundraising,” said Ortiz, who teaches Spanish at the University of Maine in Bangor. She created a GoFundMe page, which raised about $7,000, and held a pasta dinner and silent auction in her local community, which raised another $3,000.
“As a mom, you really get into this mode of leaving no stone unturned,” said Ortiz, 41. “I’m always looking for something that is going to enhance my son’s life,” she said, noting she tried everything from ABA to altering Connor’s diet before trying the treatment in Panama. “If this could in any way benefit him, then it was worth it.”
The results were positive, Ortiz said, and she saw improvements that weren’t there before, such as an increase in cognitive awareness. For example, when the bus used to pick up her son for school, Ortiz would walk him to its door. But now, she can stand on the porch as he walks to the street, looks side to side and gets on by himself.
If money isn’t an object for parents who want to have their children try the treatment, Ortiz recommends the trip. Others without the means, though, should consider the significant personal risks. “It’s devastating when the parents have to give life savings to do this. I would be very cautious because this isn’t something covered by insurance,” said Ortiz. “Families were remortgaging houses, selling and moving,” to participate in the same clinical trial, she said. “If you think there is a minute chance their life will get better, I get [why] they will do that.”
Calls to change U.S. policy
Over the past 10 years, there has been significant progress in understanding the biology of stem cells and how they evolve into distinct types of cells in the body. Scientists are now using the next five to 10 years to discover a way to translate the potential of different types of stem cells into actual treatments that can be approved in humans. “It’s not going to be for every disease, but it’s a new type of therapy. It’s going to become routine,” said Studer.
Stem cell research is continuing to move out of academic research labs as more companies help advance the stem cell field. Even though there have been opposing opinions and regulations on stem cell research between the past presidential administrations, McCormack said, it won’t wholly stymie the availability and future of stem cell research.
“The potential is enormous, regardless of who is in office. This research will go on at the state level and national level. The more support from the government, the more likely the therapies will be developed in the U.S.,” said McCormack.
However, if the government continues to restrict federal funds for certain research in the stem cell field, researchers will have to find alternative sources of funding from nonprofit organizations or private foundations, such as CIRM, where there is less money available than the NIH.
As the field of stem cell research continues to grow, the International Society for Stem Cell Research (ISSCR), an international nonprofit organization founded in 2002 and based in Illinois to regulate stem cell research in the U.S., maintains its communication with patients, the public and policymakers, so everyone can make informed decisions relating to stem cell treatments.
Julie Perlin, science communications and education manager for ISSCR, said it’s important for the organization to continue working with scientists to get the message across that stem cells can further our understanding of science and medicine as well as improve human health.
The ISSCR supports human fetal tissue research and has called for changes to President Trump’s new policies as they could slow the pace of progress toward finding new treatments for human disease. As stated in their letter to HHS on Sept. 16, 2019, the restrictions “will not only delay and interrupt biomedical research that gives patients hope but will also hamper the development of alternatives to human fetal tissue.”
Many believe the traditional medical system has failed parents of children with autism, and with a conservative government, there is less hope for stem cell advancements in the field. “It’s easy to understand someone with a condition is feeling desperate and has no other options. It’s a tough space and we haven’t come up with great answers to help people yet. We have to keep working on it,” said Regenberg. As a result, parents continue to go against government recommendation at their own personal risk and expense to help their child.
“I’ve already seen a couple things that have given me hope. But it’s scary. I’m an American going to South America for my tiny son who actually has no say in it,” said Tackett. “Am I a bad mom? Am I doing the right thing for my child, what if this doesn’t work? We just spent a fortune on a hope and chance this could improve his quality of life.”