Your Questions Answered About Kids, Teens, and Covid Vaccines
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.
This virtual event convened leading scientific and medical experts to address the public's questions and concerns about Covid-19 vaccines in kids and teens. Highlight video below.
DATE:
Thursday, May 13th, 2021
12:30 p.m. - 1:45 p.m. EDT
Dr. H. Dele Davies, M.D., MHCM
Senior Vice Chancellor for Academic Affairs and Dean for Graduate Studies at the University of Nebraska Medical (UNMC). He is an internationally recognized expert in pediatric infectious diseases and a leader in community health.
Dr. Emily Oster, Ph.D.
Professor of Economics at Brown University. She is a best-selling author and parenting guru who has pioneered a method of assessing school safety.
Dr. Tina Q. Tan, M.D.
Professor of Pediatrics at the Feinberg School of Medicine, Northwestern University. She has been involved in several vaccine survey studies that examine the awareness, acceptance, barriers and utilization of recommended preventative vaccines.
Dr. Inci Yildirim, M.D., Ph.D., M.Sc.
Associate Professor of Pediatrics (Infectious Disease); Medical Director, Transplant Infectious Diseases at Yale School of Medicine; Associate Professor of Global Health, Yale Institute for Global Health. She is an investigator for the multi-institutional COVID-19 Prevention Network's (CoVPN) Moderna mRNA-1273 clinical trial for children 6 months to 12 years of age.
About the Event Series
This event is the second of a four-part series co-hosted by Leaps.org, the Aspen Institute Science & Society Program, and the Sabin–Aspen Vaccine Science & Policy Group, with generous support from the Gordon and Betty Moore Foundation and the Howard Hughes Medical Institute.
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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.
Why Blindness Will Be the First Disorder Cured by Futuristic Treatments
Stem cells and gene therapy were supposed to revolutionize biomedicine around the turn of the millennium and provide relief for desperate patients with incurable diseases. But for many, progress has been frustratingly slow. We still cannot, for example, regenerate damaged organs like a salamander regrows its tail, and genome engineering is more complicated than cutting and pasting letters in a word document.
"There are a number of things that make [the eye] ideal for new experimental therapies which are not true necessarily in other organs."
For blind people, however, the future of medicine is one step closer to reality. In December, the FDA approved the first gene therapy for an inherited disease—a mutation in the gene RPE65 that causes a rare form of blindness. Several clinical trials also show promise for treating various forms of retinal degeneration using stem cells.
"It's not surprising that the first gene therapy that was approved by the FDA was a therapy in the eye," says Bruce Conklin, a senior investigator at the San Francisco-based Gladstone Institutes, a nonprofit life science research organization, and a professor in the Medical Genetics and Molecular Pharmacology department at the University of California, San Francisco. "There are a number of things that make it ideal for new experimental therapies which are not true necessarily in other organs."
Physicians can easily see into the eye to check if a procedure worked or if it's causing problems. "The imaging technology within the eye is really unprecedented. You can't do this in someone's spinal cord or someone's brain cells or immune system," says Conklin, who is also deputy director of the Innovative Genomics Institute.
There's also a built-in control: researchers can test an intervention on one eye first. What's more, if something goes wrong, the risk of mortality is low, especially when compared to experimenting on the heart or brain. Most types of blindness are currently incurable, so the risk-to-reward ratio for patients is high. If a problem arises with the treatment their eyesight could get worse, but if they do nothing their vision will likely decline anyway. And if the treatment works, they may be able to see for the first time in years.
Gene Therapy
An additional appeal for testing gene therapy in the eye is the low risk for off-target effects, in which genome edits could result in unintended changes to other genes or in other cell types. There are a number of genes that are solely expressed in the eye and not in any other part of the body. Manipulating those genes will only affect cells in the eye, so concerns about the impact on other organs are minimal.
Ninety-three percent of patients who received the injection had improved vision just one month after treatment.
RPE65 is one such gene. It creates an enzyme that helps the eye convert light into an electrical signal that travels back to the brain. Patients with the mutation don't produce the enzyme, so visual signals are not processed. However, the retinal cells in the eye remain healthy for years; if you can restore the missing enzyme you can restore vision.
The newly approved therapy, developed by Spark Therapeutics, uses a modified virus to deliver RPE65 into the eye. A retinal surgeon injects the virus, which has been specially engineered to remove its disease-causing genes and instead carry the correct RPE65 gene, into the retina. There, it is sucked up by retinal pigment epithelial (RPE) cells. The RPE cells are a particularly good target for injection because their job is to eat up and recycle rogue particles. Once inside the cell, the virus slips into the nucleus and releases the DNA. The RPE65 gene then goes to work, using the cell's normal machinery to produce the needed enzyme.
In the most recent clinical trial, 93 percent of patients who received the injection—who range in age from 4 to 44—had improved vision just one month after treatment. So far, the benefits have lasted at least two years.
"It's an exciting time for this class of diseases, where these people have really not had treatments," says Spark president and co-founder, Katherine High. "[Gene therapy] affords the possibility of treatment for diseases that heretofore other classes of therapeutics really have not been able to help."
Stem Cells
Another benefit of the eye is its immune privilege. In order to let light in, the eye must remain transparent. As a result, its immune system is dampened so that it won't become inflamed if outside particles get in. This means the eye is much less likely to reject cell transplants, so patients do not need to take immunosuppressant drugs.
One study generating buzz is a clinical trial in Japan that is the first and, so far, only test of induced pluripotent stem cells in the eye.
Henry Klassen, an assistant professor at UC Irvine, is taking advantage of the eye's immune privilege to transplant retinal progenitor cells into the eye to treat retinitis pigmentosa, an inherited disease affecting about 1 in 4000 people that eventually causes the retina to degenerate. The disease can stem from dozens of different genetic mutations, but the result is the same: RPE cells die off over the course of a few decades, leaving the patient blind by middle age. It is currently incurable.
Retinal progenitor cells are baby retinal cells that develop naturally from stem cells and will turn into one of several types of adult retinal cells. When transplanted into a patient's eye, the progenitor cells don't replace the lost retinal cells, but they do secrete proteins and enzymes essential for eye health.
"At the stage we get the retinal tissue it's immature," says Klassen. "They still have some flexibility in terms of which mature cells they can turn into. It's that inherent flexibility that gives them a lot of power when they're put in the context of a diseased retina."
Klassen's spin-off company, jCyte, sponsored the clinical trial with support from the California Institute for Regenerative Medicine. The results from the initial study haven't been published yet, but Klassen says he considers it a success. JCyte is now embarking on a phase two trial to assess improvements in vision after the treatment, which will wrap up in 2021.
Another study generating buzz is a clinical trial in Japan that is the first and, so far, only test of induced pluripotent stem cells (iPSC) in the eye. iPSC are created by reprogramming a patient's own skin cells into stem cells, circumventing any controversy around embryonic stem cell sources. In the trial, led by Masayo Takahashi at RIKEN, the scientists transplant retinal pigment epithelial cells created from iPSC into the retinas of patients with age-related macular degeneration. The first woman to receive the treatment is doing well, and her vision is stable. However, the second patient suffered a swollen retina as a result of the surgery. Despite this recent setback, Takahashi said last week that the trial would continue.
Botched Jobs
Although recent studies have provided patients with renewed hope, the field has not been without mishap. Most notably, an article in the New England Journal of Medicine last March described three patients who experienced severe side effects after receiving stem cell injections from a Florida clinic to treat age-related macular degeneration. Following the initial article, other reports came out about similar botched treatments. Lawsuits have been filed against US Stem Cell, the clinic that conducted the procedure, and the FDA sent them a warning letter with a long list of infractions.
"One red flag is that the clinics charge patients to take part in the treatment—something extremely unusual for legitimate clinical trials."
Ajay Kuriyan, an ophthalmologist and retinal specialist at the University of Rochester who wrote the paper, says that because details about the Florida trial are scarce, it's hard to say why the treatment caused the adverse reaction. His guess is that the stem cells were poorly prepared and not up to clinical standards.
Klassen agrees that small clinics like US Stem Cell do not offer the same caliber of therapy as larger clinical trials. "It's not the same cells and it's not the same technique and it's not the same supervision and it's not under FDA auspices. It's just not the same thing," he says. "Unfortunately, to the patient it might sound the same, and that's the tragedy for me."
For patients who are interested in joining a trial, Kuriyan listed a few things to watch out for. "One red flag is that the clinics charge patients to take part in the treatment—something extremely unusual for legitimate clinical trials," he says. "Another big red flag is doing the procedure in both eyes" at the same time. Third, if the only treatment offered is cell therapy. "These clinics tend to be sort of stand-alone clinics, and that's not very common for an actual big research study of this scale."
Despite the recent scandal, Klassen hopes that the success of his trial and others will continue to push the field forward. "It just takes so many decades to move this stuff along, even when you're trying to simplify it as much as possible," he says. "With all the heavy lifting that's been done, I hope the world's got the patience to get this through."
Genetic Engineering For All: The Last Great Frontier of Human Freedom
[Editor's Note: This op/ed appears in response to January's Big Moral Question: "Where should we draw a line, if any, between the use of gene editing for the prevention and treatment of disease, and for cosmetic enhancement?" Currently, it is illegal to develop human trials for the latter in the U.S.]
Homo sapien: a bipedal primate that is thought to be the only animal to construct a moral code. Despite the genetic differences between members of our species being less than 1 percent, we come in all shapes, sizes and colors. There is no normal for human genetics.
I believe genetic freedom is the most basic human right we all should have.
One DNA base change here, another there brings us humans with light skin, red hair and big muscles. Want to be an NBA All-Star? Your genes are by far the largest determinant of your height and well, there has never been an All-Star under 5'9". Sexual reproduction makes it so that our physical traits seem more a pinch of this and a dash of that than some precise architectural masterpiece. For the most part we have no control over whether we or our children will be the next Cristiano Ronaldo or are born with a debilitating disease--unless we use genetic engineering.
Anywhere from 64% in the US to over 82% of people in China support genetic modification of individuals to help treat diseases. I imagine that number will only increase as people become more familiar with the technology and I don't think most people need convincing that genetic modification for medical treatment is a good thing. In fact, most modern drugs are genetic regulation on a fundamental level. But cosmetic genetic modification is far more controversial with only 39% of people in the US finding it agreeable. Far fewer people support modifying the genes of babies before they are born. My question is: Where does one draw a line between cosmetic and medical genetic changes?
Modifying the genetics of individuals for medical reasons started in the late 1980s and early 1990s when scientists reprogrammed viruses so that instead of causing harm when they infected people, they changed the genetics of their cells. Much has changed and and despite the success of many gene therapy trials, people are still afraid. Perhaps because of concerns over safety, but gene therapies have been tested in over 2000 clinical trials in hundreds of thousands of people. So what are we so afraid of? I asked myself that same question in 2016 and could not find a basis for the fear and so performed the first successfully cosmetic human genetic modification by putting a jellyfish gene in my skin. The experiment was simple, the monetary cost minimal, and though my skin didn't fluoresce like a jellyfish, DNA testing showed it worked and the experiment showed me what was possible.
People are afraid because we are on the cusp of the human race changing as we know it. But isn't that change all we have been striving for?
In late 2017, I wanted to explore bigger cosmetic changes, so I did another genetic experiment on myself; I injected myself with a CRISPR/Cas9 system meant to modify myostatin, a gene responsible for muscle growth and fat loss. I didn't do it because I wanted bigger muscles but because the myostatin gene is a well-studied target that has been modified in many mammals using CRISPR. I feel a responsibility to try and push boundaries that scientists in universities and large corporations can't because of committees, regulations and social acceptability. When this cutting-edge technique was tried for the first time, it wasn't in an expensive lab and it didn't cost millions of dollars. It was done by me, prepared in my home lab, and the cost of this cosmetic treatment was under $500.
Home genetic engineering lab kits like this are sold by Zayner's company for less than $2000.
I have had many people call me crazy and worse, but they don't understand that I've undertaken these experiments with much thought and hesitation. Experimenting on oneself isn't fun; it is an unfortunate situation to be in as a Ph.D. scientist who less than two years ago was fulfilling a prestigious synthetic biology fellowship at NASA. The data points to the experiment being relatively safe, and similar experimental protocols have had success, so why wait? When so much is at stake, we need to show people what is possible so that one day we all can have genetic freedom.
Zayner's arm after attempting the first CRISPR injection showed little immune response; a small red dot in the upper left forearm can be seen at the injection site.
People are afraid because we are on the cusp of the human race changing as we know it. But isn't that change all we have been striving for yet unable to obtain? Have too much or too little hair? There is a non-gene therapy treatment for that. Want to change your appearance? The global cosmetic surgery market is over $15 billion. Tattoos, dyed hair and piercings abound. We sculpt our appearance by exercise, make-up, drugs, chemicals and invasive surgeries. We try so hard to fight against our genetics in every way except genetic modification.
Being human means freedom to be who we want to be. And at the moment, no one gets to choose their genetics. Instead, nature plays a probabilistic role in the most primitive genetic engineering experiment of sexual reproduction. This dice roll can sometimes end in tragedy. Fortunately, in my case I was born with the genetics of a healthy individual. Still, I push for everyone and though my newest genetic modification experiment is ongoing, even if it doesn't work, it is only a matter of time until it does in someone.
If you prevent someone like me from changing my genetics, where do you draw the line? Only people who can't walk can get genetic modification? Only people who can't run? Only people who are predisposed to skin cancer? Don't we all deserve a choice or to give parents better ones? I believe genetic freedom is the most basic human right we all should have. We no longer need to be slaves to genetics so let's break those bonds and embrace the change brought about by allowing human genetic engineering for all no matter the reason.
[Ed. Note: Check out the opposite perspective: "Hacking Your Own Genes: A Recipe for Disaster." Then follow LeapsMag on social media to share your opinion.]