SCOOP: Largest Cryobank in the U.S. to Offer Ancestry Testing
Sharon Kochlany and Vanessa Colimorio's four-year-old twin girls had a classic school assignment recently: make a family tree. They drew themselves and their one-year-old brother branching off from their moms, with aunts, uncles, and grandparents forking off to the sides.
The recently-gained sovereignty of queer families stands to be lost if a consumer DNA test brings a stranger's identity out of the woodwork.
What you don't see in the invisible space between Kochlany and Colimorio, however, is the sperm donor they used to conceive all three children.
To look at a family tree like this is to see in its purest form that kinship can supersede biology—the boundaries of where this family starts and stops are clear to everyone in it, in spite of a third party's genetic involvement. This kind of self-definition has always been synonymous with LGBTQ families, especially those that rely on donor gametes (sperm or eggs) to exist.
But the world around them has changed quite suddenly: The recent consumer DNA testing boom has made it more complicated than ever for families built through reproductive technology—openly, not secretively—to maintain the strong sense of autonomy and privacy that can be crucial for their emotional security. Prospective parents and cryobanks are now mulling how best to bring a new generation of donor-conceived people into this world in a way that leaves open the choice to know more about their ancestry without obliterating an equally important choice: the right not to know about biological relatives.
For queer parents who have long fought for social acceptance, having a biological relationship to their children has been revolutionary, and using an unknown donor as a means to this end especially so. Getting help from a friend often comes with the expectation that the friend will also have social involvement in the family, which some people are comfortable with, but being able to access sperm from an unknown donor—which queer parents have only been able to openly do since the early 1980s—grants them the reproductive autonomy to create families seemingly on their own. That recently-gained sovereignty stands to be lost if a consumer DNA test brings a stranger's identity out of the woodwork.
At the same time, it's natural for donor-conceived people to want to know more about where they come from ethnically, even if they don't want to know the identity of their donor. As a donor-conceived person myself, I know my donor's self-reported ethnicity, but have often wondered how accurate it is.
Opening the Pandora's box of a consumer DNA test as a way to find out has always felt profoundly unappealing to me, however. Many people have accidentally learned they're donor-conceived by unwittingly using these tools, but I already know that about myself going in, and subsequently know I'll be connected to a large web of people whose existence I'm not interested in learning about. In addition to possibly identifying my anonymous donor, his family could also show up, along with any donor-siblings—other people with whom I share a donor. My single lesbian mom is enough for me, and the trade off to learn more about my ethnic ancestry has never seemed worth it.
In 1992, when I was born, no one was planning for how consumer DNA tests might upend or illuminate one's sense of self. But the donor community has always had to stay nimble with balancing privacy concerns and psychological well-being, so it should come as no surprise that figuring out how to do so in 2020 includes finding a way to offer ancestry insight while circumventing consumer DNA tests.
A New Paradigm
This is the rationale behind unprecedented industry news that LeapsMag can exclusively break: Within the next few weeks, California Cryobank, the largest cryobank in the country, will begin offering genetically-verified ancestry information on the free public part of every donor's anonymous profile in its database, something no other cryobanks yet offer (an exact launch date was not available at the time of publication). Currently, California Cryobank's donor profiles include a short self-reported list that might merely say, "Ancestry: German, Lebanese, Scottish."
The new information will be a report in pie chart form that details exactly what percentages of a donor's DNA come from up to 26 ethnicities—it's analogous to, but on a smaller scale than, the format offered by consumer DNA testing companies, and uses the same base technology that looks for single nucleotide polymorphisms in DNA that are associated with specific ethnicities. But crucially, because the donor takes the DNA test through California Cryobank, not a consumer-facing service, the information is not connected in a network to anyone else's DNA test. It's also taken before any offspring exist so there's no chance of revealing a donor-conceived person's identity this way.
Later, when a donor-conceived person is born, grows up, and wants information about their ethnicity from the donor side, all they need is their donor's anonymous ID number to look it up. The donor-conceived person never takes a genetic test, and therefore also can't accidentally find donor siblings this way. People who want to be connected to donor siblings can use a sibling registry where other people who want to be found share donor ID numbers and look for matches (this is something that's been available for decades, and remains so).
"With genetic testing, you have no control over who reaches out to you, and at what point in your life."
California Cryobank will require all new donors to consent to this extra level of genetic testing, setting a new standard for what information prospective parents and donor-conceived people can expect to have. In the immediate, this information will be most useful for prospective parents looking for donors with specific backgrounds, possibly ones similar to their own.
It's a solution that was actually hiding in plain sight. Two years ago, California Cryobank's partner Sema4, the company handling the genetic carrier testing that's used to screen for heritable diseases, started analyzing ethnic data in its samples. That extra information was being collected because it can help calculate a more accurate assessment of genetic risks that run in certain populations—like Ashkenazi Jews and Tay Sachs disease—than relying on oral family histories. Shortly after a plan to start collecting these extra data, Jamie Shamonki, chief medical officer of California Cryobank, realized the companies would be sitting on a goldmine for a different reason.
"I didn't want to use one of these genetic testing companies like Ancestry to accomplish this," says Shamonki. "The whole thing we're trying to accomplish is also privacy."
Consumer-facing DNA testing companies are not HIPAA compliant (whereas Sema4, which isn't direct-to-consumer, is HIPAA compliant), which means there are no legal privacy protections covering people who add their DNA to these databases. Although some companies, like 23andMe, allow users to opt-out of being connected with genetic relatives, the language can be confusing to navigate, requires a high level of knowledge and self-advocacy on the user's part, and, as an opt-out system, is not set up to protect the user from unwanted information by default; many unwittingly walk right into such information as a result.
Additionally, because consumer-facing DNA testing companies operate outside the legal purview that applies to other health care entities, like hospitals, even a person who does opt-out of being linked to genetic relatives is not protected in perpetuity from being re-identified in the future by a change in company policy. The safest option for people with privacy concerns is to stay out of these databases altogether.
For California Cryobank, the new information about donor heritage won't retroactively be added to older profiles in the system, so donor-conceived people who already exist won't benefit from the ancestry tool, but it'll be the new standard going forward. The company has about 500 available donors right now, many of which have been in their registry for a while; about 100 of those donors, all new, will have this ancestry data on their profiles.
Shamonki says it has taken about two years to get to the point of publicly including ancestry information on a donor's profile because it takes about nine months of medical and psychological screening for a donor to go from walking through the door to being added to their registry. The company wanted to wait to launch until it could offer this information for a significant number of donors. As more new donors come online under the new protocol, the number with ancestry information on their profiles will go up.
For Parents: An Unexpected Complication
While this change will no doubt be welcome progress for LGBTQ families contemplating parenthood, it'll never be possible to put this entire new order back in the box. What are such families who already have donor-conceived children losing in today's world of widespread consumer genetic testing?
Kochlany and Colimorio's twins aren't themselves much older than the moment at-home DNA testing really started to take off. They were born in 2015, and two years later the industry saw its most significant spike. By now, more than 26 million people's DNA is in databases like 23andMe and Ancestry; as a result, it's estimated that within a year, 90 percent of Americans of European descent will be identifiable through these consumer databases, by way of genetic third cousins, even if they didn't want to be found and never took the test themselves. This was the principle behind solving the Golden State Killer cold case.
The waning of privacy through consumer DNA testing fundamentally clashes with the priorities of the cyrobank industry, which has long sought to protect the privacy of donor-conceived people, even as open identification became standard. Since the 1980s, donors have been able to allow their identity to be released to any offspring who is at least 18 and wants the information. Lesbian moms pushed for this option early on so their children—who would obviously know they couldn't possibly be the biological product of both parents—would never feel cut off from the chance to know more about themselves. But importantly, the openness is not a two-way street: the donors can't ever ask for the identities of their offspring. It's the latter that consumer DNA testing really puts at stake.
"23andMe basically created the possibility that there will be donors who will have contact with their donor-conceived children, and that's not something that I think the donor community is comfortable with," says I. Glenn Cohen, director of Harvard Law School's Center for Health Law Policy, Biotechnology & Bioethics. "That's about the donor's autonomy, not the rearing parents' autonomy, or the donor-conceived child's autonomy."
Kochlany and Colimorio have an open identification donor and fully support their children reaching out to California Cryobank to get more information about him if they want to when they're 18, but having a singular name revealed isn't the same thing as having contact, nor is it the same thing as revealing a web of dozens of extended genetic relations. Their concern now is that if their kids participate in genetic testing, a stranger—someone they're careful to refer to as only "the donor" and never "dad"—will reach out to the children to begin some kind of relationship. They know other people who are contemplating giving their children DNA tests, and feel staunchly that it wouldn't be right for their family.
"With genetic testing, you have no control over who reaches out to you, and at what point in your life," Kochlany says. "[People] reaching out and trying to say, 'Hey I know who your dad is' throws a curveball. It's like, 'Wait, I never thought I had a dad.' It might put insecurities in their minds."
"We want them to have the opportunity to choose whether or not they want to reach out," Colimorio adds.
Kochlany says that when their twins are old enough to start asking questions, she and Colimorio plan to frame it like this: "The donor was kind of like a technology that helped us make you a person, and make sure that you exist," she says, role playing a conversation with their kids. "But it's not necessarily that you're looking to this person [for] support or love, or because you're missing a piece."
It's a line in the sand that's present even for couples still far off from conceiving. When Mallory Schwartz, a film and TV producer in Los Angeles, and Lauren Pietra, a marriage and family therapy associate (and Shamonki's step-daughter), talk about getting married someday, it's a package deal with talking about how they'll approach having kids. They feel there are too many variables and choices to make around family planning as a same-sex couple these days to not have those conversations simultaneously. Consumer DNA databases are already on their minds.
"It frustrates me that the DNA databases are just totally unregulated," says Schwartz. "I hope they are by the time we do this. I think everyone deserves a right to privacy when making your family [using a sperm donor]."
"I wouldn't want to create a world where people who are donor-conceived feel like they can't participate in this technology because they're trying to shut out [other] information."
On the prospect of having a donor relation pop up non-consensually for a future child, Pietra says, "I don't like it. It would be really disappointing if the child didn't want [contact], and unfortunately they're on the receiving end."
You can see how important preserving the right to keep this door closed is when you look at what's going on at The Sperm Bank of California. This pioneering cryobank was the first in the world to openly serve LGBTQ people and single women, and also the first to offer the open identification option when it opened in 1982, but not as many people are asking for their donor's identity as expected.
"We're finding a third of young people are coming forward for their donor's identity," says Alice Ruby, executive director. "We thought it would be a higher number." Viewed the other way, two-thirds of the donor-conceived people who could ethically get their donor's identity through The Sperm Bank of California are not asking the cryobank for it.
Ruby says that part of what historically made an open identification program appealing, rather than invasive or nerve-wracking, is how rigidly it's always been formatted around mutual consent, and protects against surprises for all parties. Those [donor-conceived people] who wanted more information were never barred from it, while those who wanted to remain in the dark could. No one group's wish eclipsed the other's. The potential breakdown of a system built around consent, expectations, and respect for privacy is why unregulated consumer DNA testing is most concerning to her as a path for connecting with genetic relatives.
For the last few decades in cryobanks around the world, the largest cohort of people seeking out donor sperm has been lesbian couples, followed by single women. For infertile heterosexual couples, the smallest client demographic, Ruby says donor sperm offers a solution to a medical problem, but in contrast, it historically "provided the ability for [lesbian] couples and single moms to have some reproductive autonomy." Yes, it was still a solution to a biological problem, but it was also a solution to a social one.
The Sperm Bank of California updated its registration forms to include language urging parents, donor-conceived people, and donors not to use consumer DNA tests, and to go through the cryobank if they, understandably, want to learn more about who they're connected to. But truthfully, there's not much else cryobanks can do to protect clients on any side of the donor transaction from surprise contact right now—especially not from relatives of the donor who may not even know someone in their family has donated sperm.
A Tricky Position
Personally, I've known I was donor-conceived from day one. It has never been a source of confusion, angst, or curiosity, and in fact has never loomed particularly large for me in any way. I see it merely as a type of reproductive technology—on par with in vitro fertilization—that enabled me to exist, and, now that I do exist, is irrelevant. Being confronted with my donor's identity or any donor siblings would make this fact of my conception bigger than I need it to be, as an adult with a full-blown identity derived from all of my other life experiences. But I still wonder about the minutiae of my ethnicity in much the same way as anyone else who wonders, and feel there's no safe way for me to find out without relinquishing some of my existential independence.
The author and her mom in spring of 1998.
"People obviously want to participate in 23andMe and Ancestry because they're interested in knowing more about themselves," says Shamonki. "I wouldn't want to create a world where people who are donor-conceived feel like they can't participate in this technology because they're trying to shut out [other] information."
After all, it was the allure of that exact conceit—knowing more about oneself—that seemed to magnetically draw in millions of people to these tools in the first place. It's an experience that clearly taps into a population-wide psychic need, even—perhaps especially—if one's origins are a mystery.
Big Data Probably Knows More About You Than Your Friends Do
Data is the new oil. It is highly valuable, and it is everywhere, even if you're not aware of it. For example, it's there when you use social media. Sharing pictures on Facebook lets its facial recognition software peg you and your friends. Thanks to that software, now anywhere you visit that has installed cameras, your face can be identified and your actions recorded.
The big data revolution is advancing much faster than the ones before, and it carries both promises and perils for humanity.
It's there when you log into Twitter, posting one of the 230 million tweets per day, which up until last month were all archived by the Library of Congress and will be made public for research. These social media data can be used to predict your political affiliations, ethnicity, race, age, how close you are with your family and friends, your mental health, even when you are most likely to be grumpy or go to the gym. These data can also predict when you are apt to get sick and track how diseases are spreading.
In fact, tracking isn't limited to what you decide to share or public spaces anymore. Lab experiments show Comcast and other cable companies may soon be able to record and monitor movements in your house. They may also be able to read your lips and identify your visitors simply by assessing how Wi-Fi waves bounce off bodies and other objects in houses. In one study, MIT researchers used routers and sensors to monitor breathing and heart rates with 99% accuracy. Routers could soon be used for seemingly good things, like monitoring infant breathing and whether an older adult is about to take a big tumble. However, it may also enable unwanted and unparalleled levels of surveillance.
Some call the first digital pill a snitch pill, medication with a tattletale, and big brother in your belly.
Big data is there every time you pick up your smartphone, which can track your daily steps, where you go via geolocation, what time you wake up and go to bed, your punctuality, and even your overall health depending on which features you have enabled. Are you close with your mom; are you a sedentary couch potato; did you commit a murder (iPhone data was recently used in a German murder trial)? Smartphone-generated data can be used to label you---and not just you, your future and past generations too.
Smartphones are not the only "things" gathering data on you. Anything with an on and off switch can be connected to the internet and generate data. The new rule seems to be, if it can be, it will be, connected. Washing machines, coffee makers, medical appliances, cars, and even your luggage (yes, someone created a self-driving suitcase) can and are often generating data. "Smart" refrigerators can monitor your food levels and automatically create shopping lists and order food for you—while recording your alcohol consumption and whether you tend to be a healthy or junk food eater.
Even medicines can monitor behaviors. The first digital pill was just approved by the FDA last November to track whether patients take their medicines. It has a sensor that sends signals to a patient's smartphone, and others, when it encounters stomach acid. Some call it a snitch pill, medication with a tattletale, and big brother in your belly. Others see it as a major breakthrough to help patients remember to take their medications and to save payers millions of dollars.
Big data is there when you go shopping. Credit card and retail data can show whether you pay for a gym, if you are pregnant, have children, and your credit-worthiness. Uber and Lyft transactional data reveal what time you usually go to and leave work and who you regularly visit (Uber data has been used to catch cheating spouses).
Amazon now sells a bedroom camera to see your fashion choices and offer advice. It is marketing a more fashionable you, but it probably also wants the video feed showing your body measurements—they're "a newly prized currency," according to the Washington Post. They help retailers create more customized and better fitting clothes. Amazon also just partnered with Berkshire Hathaway and JPMorgan Chase, the largest bank in the United States by assets, to create an independent health-care company for their employees--raising privacy concerns as Amazon already owns so much data about us, from drones, devices, the AI of Alexa, and our viewing, eating, and other purchasing habits on Amazon Prime.
Data generation and storage can also be used to make the world better, safer and fairer.
Big data is arguably a new phenomenon; almost all the world's data (90%) were produced within the last 2 years or so. It is a result of the fusion of physical, digital, and biological technologies that together constitute the fourth industrial revolution, according to the World Economic Forum. Unlike the last three revolutions, involving the discoveries of steam power, electrical energy, and computers—this revolution is advancing much faster than the ones before and it carries both promises and perils for humanity.
Some people may want to opt out of all this tracking, reduce their digital footprint and stay "off the grid." However, it is worth noting that data generation and storage can be used for great things --- things that make the world better, safer and fairer. For example, sharing electronic health records and social media data can help scientists better track and understand diseases, develop new cures and therapies, and understand the safety and efficacy profiles of medicines and vaccines.
While full of promise, big data is not without its pitfalls. Data are often not interoperable or easily integrated. You can use your credit card practically anywhere in the world, but you cannot easily port your electronic health record to the doctor or hospital across the street, for example.
Data quality can also be poor. It is dependent on the person entering it. My electronic health record at one point said I was male, and I was pregnant at the time. No doctors or nurses seemed to notice. The problem is worse on a global level. For example, causes of death can be coded differently by country and village. Take HIV patients: they often develop secondary infections, like TB. Do you record the cause of death as TB or HIV? There isn't global consistency, and political pressure from patient groups can exert itself on death records. Often, each group wants to say they have the most deaths so they can fundraise more money.
Data can be biased. More than 80 percent of genomic data comes from Caucasians. Only 14 percent is from Asians and 3.5 percent is from African and Hispanic populations. Thus, when scientists use genomic data to develop drugs or lab tests, they may create biased products that work for only some demographics. Take type 2 diabetes blood tests; some do not work well for African Americans. One study estimates that 650,000 African Americans may have undiagnosed diabetes, because a common blood test doesn't work for them. Using biased data in medicine can be a matter of life and death. Moreover, if genomic medicine benefits only "a privileged few," the practice raises concerns about unequal access.
Large companies are selling data that originated from you and you are not sharing in the wealth.
We need to think carefully and be transparent about the values embedded in our data, data analytics (algorithms), and data applications. Numbers are never neutral. Algorithms are always embedded with subjective normative values--sometimes purposely, sometimes not. To address this problem, we need ethicists who can audit databanks and algorithms to identify embedded norms, values and biases and help ensure they are addressed or at least transparently disclosed. Additionally, we need to determine how to let people opt out of certain types of data collection and uses—and not just at the beginning of a system, but also at any point in their lifetimes. There is a right to be forgotten, which hasn't been adequately operationalized in today's data sphere.
What do you think happens to all of these data collected about us? The short answer is the public doesn't really know. A lot of it looks like what is in a medical record—i.e. height, weight, pregnancy status, age, mental health, pulse, blood pressure, and illness symptoms--- yet, it isn't protected by HIPPA, like your medical record information.
And it is being consolidated into the hands of fewer and fewer big players. Large companies are selling data that originated from you and you are not sharing in the wealth.
A possible solution is to create an app, managed by a nonprofit or public benefit corporation, through which you could download and manage all the data collected about you. For example, you could download your credit card statements with all your purchasing habits, your Uber rides showing transit patterns, medical records, electric bills, every digital record you have and would like to download--into one application. You would then have the power to license pieces or the collection of your data to users for a small fee for one year at a time. Uses and users could be monitored and audited leveraging blockchain capabilities. After the year is up, you can withdraw access.
You could be your own data landlord. We could democratize big data and empower people to better control and manage the wealth of information collected about us. Why should only the big companies like Amazon and Apple profit off the new oil? Let's create an app so we can all manage our data wealth and maybe even become data barons—an app created by the people for the people.
You Saw the Grammys, But You Missed the More Important Awards
Last week in Miami, more than 450 researchers, physicians, lawyers, ethicists, and executives gathered from far-flung corners of the globe to share the latest updates in stem cell research and regenerative medicine. Sure, a science conference might not seem as glamorous as a celebrity-filled Madison Square Garden, but it's the place to be if you care about breakthroughs that could give you a longer and healthier life. Here are our top ten takeaways about what's hot and what's happening worldwide:
"The places you least expect will turn up to produce some really extraordinary things."
1) The future of stem cell treatment may involve the creation of a universal cell line that is genetically modified so every patient's immune system will accept it.
One of the leading scientists at the convention, Japanese stem cell pioneer Dr. Norio Nakatsuji, dubbed this quest a "very hot topic" right now. Being able to produce one safe cell line for everyone would be much cheaper and faster than having to create and grow patient-specific cells. "It is theoretically possible to genetically modify the lines so everyone can accept them," said Nakatsuji. A Seattle-based biotech company aptly named Universal Cells is leading the way in this promising area.
2) Japan was the world leader in stem cell research 10 years ago, but has since fallen behind the United States for reasons that some researchers find frustrating.
Japan is not a particularly religious society, so their culture does not object on principle to using donated human embryos for the creation of stem cells, and federal money can fund such research, unlike in the U.S. But the irony, according to Nakatsuji, is that the regulations for researchers are still very cumbersome. "We need to clear many probably unnecessary steps," he said. For example, before starting work in the field, new graduate students need special training and ethics lectures, and must be cleared by a committee; the process could take six months before an experiment can start, whereas in a country like Britain, scientists can immediately begin.
Also: back in 2006, a Japanese researcher who later won the Nobel Prize managed to reprogram 4 genes in adult cells and essentially turn back time, reversing the cells back to an embryonic state. The implications of this breakthrough were enormous, because destroying an embryo was no longer required to generate blank cells with unlimited potential—and these cells could now be created directly from a patient.
But then "a very unfortunate situation" happened in Japan, says Nakatsuji. There was a fever for these induced pluripotent (iPS) cells, and many Japanese researchers thought embryonic stem cell research was no longer important.
"This is a misconception," Nakatsuji lamented. "You do need both cell types." Embryonic stem cells, unlike their artificially made alternatives, are still safer and more reliable. A symbolic example, he said, is that groups in the U.S. and Europe are starting trials for Parkinson's disease that require dopamine-secreting neurons from stem cells. The researchers could have chosen iPS cells, but went with embryonic stem cells.
The main advantage now of iPS cells, Nakatsuji said, is not for therapeutic purposes, but for drug discovery and creating models of disease based on specific patient profiles.
Dr. Norio Nakatsuji receiving an award for international leadership from Bernard Siegel, the founder and director of the Regenerative Medicine Foundation.
3) In China, rampant stem cell tourism in 2009 led to disaster and a total government shutdown, from which the research field is only recently starting to recover.
Stem cell therapy in China "used to be totally unethical but then took a shock and is still recovering from that shock," said Dr. Wenchun Qu, a physician-researcher at the Mayo Clinic. Scam clinics profited off unapproved and unproven treatments which killed some patients until the total ban set in. Now, the research field is slowly coming back on board under strict regulation; there were only 35 clinical trial with stem cells in 2016, whereas in the U.S, there were more than 2000.
"A lack of public trust and deception is the number one factor" in China's falling behind, said Dr. Yen-Michael Hsu of Weill Cornell. "China is catching up trying to rebuild trust with the taxpayers."
As of last November, 102 designated institutions in China can conduct stem cell research only--not offer commercialized treatments. Bottom line: China is advancing fast in basic science and even leading in some areas, yet is trailing other countries in translational studies and clinical practice.
4) The Bahamas is emerging as a hub of legitimate research that is attracting innovative new trials.
A regulatory framework and National Stem Cell Ethics Committee were established around 2013, and since then, clinical research in the Bahamas has begun; the focus is on safety and efficacy, with standards high enough to satisfy the FDA, but also streamlined enough to allow for trials to proceed faster than they might in other countries.
One U.S.-based company, Advanced Regen Medical Technologies, is pursuing a proprietary cell culture that rejuvenates old cells by exposing them to young donor cells, with the goal of extending healthy living. On May 24th, 2017, the company presented to the National Stem Cell Ethics Committee, and on December 15th, they treated their first patient.
"Here's an indication that would be frankly impossible to get through the FDA and certainly not without many years of pain," said Marc Penn, a leader of the company's executive team. "We were able to get through the National Stem Cell Ethics Committee with all of us feeling good about the level of rigor within a seven-to-eight month span."
Desiree Cox, the chairwoman of the Committee, stressed the selectiveness and rigor with which the Bahamas is approaching new trial applications. Of 20 proposed stem cell trials, they have approved only four.
"We're interested in first-in-man studies, things that are breaking the boundaries, going beyond what is already done elsewhere, linking to predictive analytics," she said. "The places you least expect will turn up to produce some really extraordinary things."
Another active clinical trial there is a phase 1 study for Aging Frailty run by a Miami-based start-up called Longeveron. "Our experience is it comes as a huge relief to many people to have the opportunity to go to such a program rather than wait for a drug to be approved in the U.S.," said Dr. Joshua Hare, the director of the Interdisciplinary Stem Cell Institute at the University of Miami and the co-founder and Chief Science Officer at Longeveron.
"The challenge right now is the effective translation and development of viable stem-cell based therapies."
5) Researchers are working on building an artificial heart with stem cells, but technology is not the only hurdle.
A group at the Texas Heart Institute in Houston is experimenting with this strategy: stripping a real heart organ of its cells, then repopulating it with blood-forming stem cells, and implanting it. In cows, this approach has worked successfully. But one problem, said Dr. Doris Taylor, the director of Regenerative Medicine Research at the Institute, is educating regulators, since this kind of treatment is not a drug and not a device.
That said, when will we see someone order a heart off the shelf?
"I think in the next two years," she said, "you will see exciting things happening at least at the level of congenital heart disease, if not adult hearts."
6) Cost is a major barrier to regenerative medicine's success.
"It's not about whether you can get enough of the cells you need, it's about whether you can get them for less than one million dollars," Taylor said wryly.
Cell therapies intended for patients must be manufactured in a special facility to generate the quantity necessary for treatment. Some experts expressed concerned that these bio-manufacturing facilities are like "the Wild West" right now because there is no standard for pricing.
Some companies are "getting away with murder," said Dr. Camillo Ricordi, director of the Diabetes Research Institute. "This doesn't happen in most of the rest of the world."
7) Media hype has caused the premature (and potentially dangerous) commercialization of unproven stem cell therapies.
There are now over 570 such clinics operating in the U.S., with hot spots in Florida and California, which offer up stem cells for everything from sports medicine and vitamins to beauty products and pet health.
In fact, according to the FDA, the only stem cell-based products currently approved for use consist of blood-forming stem cells derived from cord blood. Everything else, for now, is still experimental.
While plenty of legitimate research is moving ahead in clinical trials, consumers may be confused by the plethora of scam clinics. But since last August, the FDA has begun cracking down, issuing three enforcement actions.
Also worth noting: what the marketplace refers to as "stem cells" are in fact products that contain a very low amount of concentrated adult stem cells derived from fat or bone marrow. There are no pure stem cell products out there.
"The challenge right now is the effective translation and development of viable stem-cell based therapies," said Dr. Shane Shapiro, a sports medicine physician at the Mayo Clinic.
What constitutes a genetically modified organism? Europe is in the process of deciding.
8) An exciting coming trend is induced tissue regeneration.
The company AgeX, run by gerontologist and stem cell pioneer Dr. Mike West, is in preclinical trials for a treatment that can reset the regenerative potential of mature tissue.
This ability is lost in the early stages of life to help prevent cancer, but AgeX is interested in figuring out a way to restore it with pluripotent stem cells in adult tissue, to correct the damage incurred by aging. West said he expects the program to reach human clinical trials in the next five years.
9) Stem cells alone are not the whole story.
The future of cell therapy will involve cell derivatives—the things that cells secrete, like exosomes, microRNA, and viruses, that can be better controlled than the cells themselves.
Exosomes, which are extracellular vesicles released from cells, act as fingerprints that are useful for diagnosis and therapy, said Dr. Li Chen, the head of the Human Liver Cell Lab at the University of California-San Diego. Because exosomes are smaller than cells, they can also cross the blood-brain barrier.
Europe is the leading place for exosome research. Recently, a 21-year-old boy suffering from brain cancer there was treated with stem cell therapy, which failed, but then subsequently he received surgery with exosomes applied to his tumor, and he survived.
10) The European Union is in the process of deciding what legally constitutes a "genetically modified organism" – and the stakes are high.
The European Court of Justice, the EU's highest court, is considering this question: If a modification brought about by genetic engineering technology could also have occurred naturally, should the resulting organism be considered a GMO?
Just last week, an advocate general of the court proposed that whenever an organism is manmade that could theoretically occur naturally, it should not be considered a GMO, and therefore should not be subjected to such regulations.
If the Court agrees with the advice of its advocate general later this year, then the decision would have huge implications for biotech agriculture across Europe, paving the way for gene-edited crops to hit the market.
Kira Peikoff was the editor-in-chief of Leaps.org from 2017 to 2021. As a journalist, her work has appeared in The New York Times, Newsweek, Nautilus, Popular Mechanics, The New York Academy of Sciences, and other outlets. She is also the author of four suspense novels that explore controversial issues arising from scientific innovation: Living Proof, No Time to Die, Die Again Tomorrow, and Mother Knows Best. Peikoff holds a B.A. in Journalism from New York University and an M.S. in Bioethics from Columbia University. She lives in New Jersey with her husband and two young sons. Follow her on Twitter @KiraPeikoff.