You Saw the Grammys, But You Missed the More Important Awards
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.

The 2018 Stem Cell Action Awards, honoring recipients for advocacy, leadership, and inspiration, presented by the Regenerative Medicine Foundation.
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.
They received retinal implants to restore their vision. Then the company turned its back on them.
A company called Second Sight made an implant that partially restored vision to people who'd been blind for decades. But when Second Sight pivoted, it stopped servicing its product, leaving many in the dark.
The first thing Jeroen Perk saw after he partially regained his sight nearly a decade ago was the outline of his guide dog Pedro.
“There was a white floor, and the dog was black,” recalls Perk, a 43-year-old investigator for the Dutch customs service. “I was crying. It was a very nice moment.”
Perk was diagnosed with retinitis pigmentosa as a child and had been blind since early adulthood. He has been able to use the implant placed into his retina in 2013 to help identify street crossings, and even ski and pursue archery. A video posted by the company that designed and manufactured the device indicates he’s a good shot.
Less black-and-white has been the journey Perk and others have been on after they were implanted with the Argus II, a second-generation device created by a Los Angeles-based company called Second Sight Medical Devices.
The Argus II uses the implant and a video camera embedded in a special pair of glasses to provide limited vision to those with retinitis pigmentosa, a genetic disease that causes cells in the retina to deteriorate. The camera feeds information to the implant, which sends electrical impulses into the retina to recapitulate what the camera sees. The impulses appear in the Argus II as a 60-pixel grid of blacks, grays and whites in the user’s eye that can render rough outlines of objects and their motion.
Smartphone and computer manufacturers typically stop issuing software upgrades to their devices after two or three years, eventually rendering them bricks. But is the smartphone approach acceptable for a device that helps restore the most crucial sense a human being possesses?
Ross Doerr, a retired disability rights attorney in Maine who received an Argus II in 2019, describes the field of vision as the equivalent of an index card held at arm’s length. Perk often brings objects close to his face to decipher them. Moreover, users must swivel their heads to take in visual data; moving their eyeballs does not work.
Despite its limitations, the Argus II beats the alternative. Perk no longer relies on his guide dog. Doerr was uplifted when he was able to see the outlines of Christmas trees at a holiday show.
“The fairy godmother department sort of reaches out and taps you on the shoulder once in a while,” Doerr says of his implant, which came about purely by chance. A surgeon treating his cataracts was partnered with the son of another surgeon who was implanting the devices, and he was referred.
Doerr had no reason to believe the shower of fairy dust wouldn’t continue. Second Sight held out promises that the Argus II recipients’ vision would gradually improve through upgrades to much higher pixel densities. The ability to recognize individual faces was even touted as a possibility. In the winter of 2020, Doerr was preparing to travel across the U.S. to Second Sight’s headquarters to receive an upgrade. But then COVID-19 descended, and the trip was canceled.
The pandemic also hit Second Sight’s bottom line. Doerr found out about its tribulations only from one of the company’s vision therapists, who told him the entire department was being laid off. Second Sight cut nearly 80% of its workforce in March 2020 and announced it would wind down operations.
Ross Doerr has mostly stopped using his Argus II, the result of combination of fear of losing its assistance from wear and tear and disdain for the company that brought it to market.
Jan Doerr
Second Sight’s implosion left some 350 Argus recipients in the metaphorical dark about what to do if their implants failed. Skeleton staff seem to have rarely responded to queries from their customers, at least based on the experiences of Perk and Doerr. And some recipients have unfortunately returned to the actual dark as well, as reports have surfaced of Argus II failures due to aging or worn-down parts.
Product support for complex products is remarkably uneven. Although the iconic Ford Mustang ceased production in the late 1960s, its parts market is so robust that it’s theoretically possible to assemble a new vehicle from recently crafted components. Conversely, smartphone and computer manufacturers typically stop issuing software upgrades to their devices after two or three years, eventually rendering them bricks. Consumers have accepted both extremes.
But is the smartphone approach acceptable for a device that helps restore the most crucial sense a human being possesses?
Margaret McLean, a senior fellow at the Markkula Center for Applied Ethics at Santa Clara University in California, notes companies like Second Sight have a greater obligation for product support than other consumer product ventures.
“In this particular case, you have a great deal of risk that is involved in using this device, the implant, and the after care of this device,” she says. “You cannot, like with your car, decide that ‘I don’t like my Mustang anymore,’ and go out and buy a Corvette.”
And, whether the Argus II implant works or not, its physical presence can impact critical medical decisions. Doerr’s doctor wanted him to undergo an MRI to assist in diagnosing attacks of vertigo. But the physician was concerned his implant might interfere. With the latest available manufacturer advisories on his implant nearly a decade old, the procedure was held up. Doerr spent months importuning Second Sight through phone calls, emails and Facebook postings to learn if his implant was contraindicated with MRIs, which he never received. Although the cause of his vertigo was found without an MRI, Doerr was hardly assured.
“Put that into context for a minute. I get into a serious car accident. I end up in the emergency room, and I have a tag saying I have an implanted medical device,” he says. “You can’t do an MRI until you get the proper information from the company. Who’s going to answer the phone?”
Second Sight’s management did answer the call to revamp its business. It netted nearly $78 million through a private stock placement and an initial public offering last year. At the end of 2021, Second Sight had nearly $70 million in cash on hand, according to a recent filing with the Securities and Exchange Commission.
And while the Argus II is still touted at length on Second Sight’s home page, it appears little of its corporate coffers are earmarked toward its support. These days, the company is focused on obtaining federal approvals for Orion, a new implant that would go directly into the recipient’s brain and could be used to remedy blindness from a variety of causes. It obtained a $6.4 million grant from the National Institutes of Health in May 2021 to help develop Orion.
Presented with a list of written questions by email, Second Sight’s spokesperson, Dave Gentry of the investor relations firm Red Chip Companies, copied a subordinate with an abrupt message to “please handle.” That was the only response from a company representative. A call to Second Sight acting chief executive officer Scott Dunbar went unreturned.
Whether or not the Orion succeeds remains to be seen. The company’s SEC filings suggest a viable and FDA-approved device is years away, and that operational losses are expected for the “foreseeable future.” Second Sight reported zero revenue in 2020 or 2021.
Moreover, the experiences of the Argus II recipients could color the reception of future Second Sight products. Doerr notes that his insurer paid nearly $500,000 to implant his device and for training on how to use it.
“What’s the insurance industry going to say the next time this crops up?” Doerr asks, noting that the company’s reputation is “completely shot” with the recipients of its implants.
Perk, who made speeches to praise the Argus II and is still featured in a video on the Second Sight website, says he also no longer supports the company.
Jeroen Perk, an investigator for the Dutch customs service, cried for joy after partially regaining his sight, but he no longer trusts Second Sight, the company that provided his implant.
Nanda Perk
Nevertheless, Perk remains highly reliant on the technology. When he dropped an external component of his device in late 2020 and it broke, Perk briefly debated whether to remain blind or find a way to get his Argus II working again. Three months later, he was able to revive it by crowdsourcing parts, primarily from surgeons with spare components or other Argus II recipients who no longer use their devices. Perk now has several spare parts in reserve in case of future breakdowns.
Despite the frantic efforts to retain what little sight he has, Perk has no regrets about having the device implanted. And while he no longer trusts Second Sight, he is looking forward to possibly obtaining more advanced implants from companies in the Netherlands and Australia working on their own products.
Doerr suggests that biotech firms whose implants are distributed globally be bound to some sort of international treaty requiring them to service their products in perpetuity. Such treaties are still applied to the salvage rights for ships that sunk centuries ago, he notes.
“I think that in a global tech economy, that would be a good thing,” says McLean, the fellow at Santa Clara, “but I am not optimistic about it in the near term. Business incentives push toward return on share to stockholders, not to patients and other stakeholders. We likely need to rely on some combination of corporately responsibility…and [international] government regulation. It’s tough—the Paris Climate Accord implementation at a slow walk comes to mind.”
Unlike Perk, Doerr has mostly stopped using his Argus II, the result of combination of fear of losing its assistance from wear and tear and disdain for the company that brought it to market. At 70, Doerr says he does not have the time or energy to hold the company more accountable. And with Second Sight having gone through a considerable corporate reorganization, Doerr believes a lawsuit to compel it to better serve its Argus recipients would be nothing but an extremely costly longshot.
“It’s corporate America at its best,” he observes.
Rehabilitating psychedelic drugs: Another key to treating severe mental health disorders
A recent review paper found evidence that using psychedelics such as MDMA can help with treating a variety of common mental illnesses, but experts fear that research might easily be shut down in the future.
Lori Tipton's life was a cascade of trauma that even a soap opera would not dare inflict upon a character: a mentally unstable family; a brother who died of a drug overdose; the shocking discovery of the bodies of two persons her mother had killed before turning the gun on herself; the devastation of Hurricane Katrina that savaged her hometown of New Orleans; being raped by someone she trusted; and having an abortion. She suffered from severe PTSD.
“My life was filled with anxiety and hypervigilance,” she says. “I was constantly afraid and had mood swings, panic attacks, insomnia, intrusive thoughts and suicidal ideation. I tried to take my life more than once.” She was fortunate to be able to access multiple mental health services, “And while at times some of these modalities would relieve the symptoms, nothing really lasted and nothing really address the core trauma.”
Then in 2018 Tipton enrolled in a clinical trial that combined intense sessions of psychotherapy with limited use of Methylenedioxymethamphetamine, or MDMA, a drug classified as a psychedelic and commonly known as ecstasy or Molly. The regimen was arduous; 1-2 hour preparation sessions, three sessions where MDMA was used, which lasted 6-8 hours, and lengthy sessions afterward to process and integrate the experiences. Two therapists were with her every moment of the three-month program that totaled more than 40 hours.
“It was clear to me that [the therapists] weren't going to heal me, that I was going to have to do the work for myself, but that they were there to completely support my process,” she says. “But the effects of MDMA were really undeniable for me. I felt embodied in a way that I hadn't in years. PTSD had robbed me of the ability to feel safe in my own body.”
Tipton doesn’t think the therapy completely cured her PTSD. “But when I completed the trial in 2018, I no longer qualified for the diagnosis, and I still don't qualify for the diagnosis today,” she told an April workshop on psychedelics as mental health treatment by the National Academies of Sciences, Engineering and Medicine, or NASEM.
A Champion
Rick Doblin has been a catalyst behind much of the contemporary research into psychedelics. Prior to the DEA clamp down, the Boston psychotherapist had seen that MDMA and other psychedelics could benefit some of his patients where other measures had failed. He immediately organized efforts to question the drug rescheduling but to little avail. In 1986, he created the nonprofit Multidisciplinary Association for Psychedelic Studies (MAPS), which slowly laid the scientific foundation for clinical trials, including the one that Tipton joined, using psychedelics to treat mental health conditions.
Now, only slowly, have researchers been able to explore the power of these drugs to treat a broad spectrum of severely debilitating mental health conditions, including trauma, depression, and PTSD, where other available treatments proved inadequate.
“Psychedelic psychotherapy is an attempt to go after the root causes of the problems with just a relatively few administrations, as contrasted to most of the psychiatric drugs used today that are mostly just reducing symptoms and are meant to be taken on a daily basis,” Doblin said in a 2019 TED Talk. Most of these drugs can have broad effect but “some are probably more effective than others for certain conditions,” he added in a recent interview with Leaps.org. Comparative head-to-head studies of psychedelic therapies simply have not been conducted.
Their mechanisms of action are poorly understood and can vary between drugs, but it is generally believed that psychedelics change the activity of neurons so that the brain processes information differently, says Katrin Preller, a neuropsychologist at the University of Zurich. A recent important study in Nature Medicine by Richard Daws and colleagues used functional magnetic resonance imaging (fMRI) of the brain and found that “functional networks became more functionally interconnected and flexible after psilocybin treatment…implying that psilocybin's antidepressant action may depend on a global increase in brain network integration.”
Rosalind Watts, a clinical investigator at the Imperial College in London, believes there is “an overestimation of the importance of the drug and an underestimation of the importance of the [therapeutic] context” in psychedelic research. “It is unethical to provide the drug without the other,” she says. Doblin notes that “psychotherapy outcomes research demonstrates that the therapeutic alliance between the therapist and the patients is the single most predictive factor of outcomes. [It is] trust and the sense of safety, the willingness to go into difficult spaces” that makes clinical breakthroughs possible with the drug.
Excitement and Challenges
Recurrent themes expressed at the NASEM workshop were exciting glimpses of the potential for psychedelics to treat mental health conditions combined with the challenges of realizing those potentials. A recent review paper found evidence that using psychedelics can help with treating a variety of common mental illnesses, but the paper could identify only 14 clinical trials of classic psychedelics published since 1991. Much of the reason is that the drugs are not patentable and so the pharmaceutical industry has no interest in investing in expensive clinical trials to bring them to market. MAPS has raised about $135 million over its 36-year history to conduct such research, says Doblin, the vast majority of it from individual donors and none from foundations.
The workshop participants’ views also were colored by the history of drug crackdowns and a fear that research might easily be shut down in the future. There was great concern that use of psychedelics should be confined to clinical trials with high safety and ethical standards, instead of doctors and patients experimenting on their own. “We need to get it right this time,” says Charles Grob, a psychiatrist at the UCLA School of Medicine. But restricting access to psychedelics will become even more difficult now that Oregon and several cities have acted to decriminalize possession and use of many of these drugs.
The experience with ketamine also troubled Grob. He is hoping to “mitigate the rush of rapid commercialization” that occurred with that drug. Ketamine technically is not a psychedelic though it does share some of their potentially euphoric properties. In 2019, soon after the FDA approved a form of ketamine with a limited label indication to treat depression, for profit clinics sprang up promoting off label use of the drug for psychiatric conditions where there was little clinical evidence of efficacy. He fears the same thing will happen when true psychedelics are made available.
If these therapies are approved, access to them is likely to be a problem. The drugs themselves are cheap but the accompanying therapy is not, and there is a shortage of trained psychotherapists. Mental health services often are not adequately covered by health insurance, while the poor and people of color suffer additional burdens of inadequate access. Doblin is committed to health care equity by training additional providers and by investigating whether some of the preparatory and integration sessions might be handled in a group setting. He says it is important that the legal aspects of psychedelics also be addressed so that patients “don't have to go underground” in order to receive this care.