Many have taken rides from Lyft (LYFT) and Uber (UBER), and soon they may finally be able to invest in both, Daren Fonda and Jon Swartz write in this week’s edition of Barron’s. An expected wave of initial public offerings by giant, well-known companies like these could open opportunities for investors to jump into businesses with leading positions in winner-take-all markets, potentially becoming the next Amazon (AMZN) or Facebook (FB), the publication notes. Yet investors should temper their expectations as steep private valuations could make the stocks disappointing, at least in the near-term, they add.
Saturday, December 22, 2018
J&J selloff overdone, still more discounted stocks to consider, Barron’s says
A selloff in Johnson & Johnson stock this month over potential legal damages from the alleged health risks of baby powder looks overdone, but the stock was expensive to start with, Jack Hough writes in this week’s edition of Barron’s. There are more deeply discounted health-care stocks for investors to consider, the report adds.
Stem cell-derived neurons stop seizures, improve cognitive function
About 3.4 million Americans, or 1.2 percent of the population, have active epilepsy. Although the majority respond to medication, between 20 and 40 percent of patients with epilepsy continue to have seizures even after trying multiple anti-seizure drugs. Even when the drugs do work, people may develop cognitive and memory problems and depression, likely from the combination of the underlying seizure disorder and the drugs to treat it.
A team led by Ashok K. Shetty, Ph.D., a professor in the Department of Molecular and Cellular Medicine at the Texas A&M College of Medicine, associate director of the Institute for Regenerative Medicine and a research career scientist at the Olin E. Teague Veterans’ Medical Center, part of the Central Texas Veterans Health Care System, is working on a better and permanent treatment for epilepsy. Their results published this week in the Proceedings of the National Academy of Sciences(PNAS).
Seizures are caused when the excitatory neurons in the brain fire too much and inhibitory neurons—the ones that tell the excitatory neurons to stop firing—aren’t as abundant or aren’t operating at their optimal level. The main inhibitory neurotransmitter in the brain is called GABA, short for gamma-Aminobutyric acid.
Over the last decade, scientists have learned how to create induced pluripotent stem cells from ordinary adult cells, like a skin cell. These stem cells can then be coaxed to become virtually any type of cells in the body, including neurons that use GABA, called GABAergic interneurons.
“What we did is transplant human induced pluripotent stem cell-derived GABAergic progenitor cells into the hippocampus in an animal model of early temporal lobe epilepsy,” Shetty said. The hippocampus is a region in the brain where seizures originate in temporal lobe epilepsy, which is also important for learning, memory and mood. “It worked very well to suppress seizures and even to improve cognitive and mood function in the chronic phase of epilepsy.”
Further testing showed that these transplanted human neurons formed synapses, or connections, with the host excitatory neurons. “They were also positive for GABA and other markers of specialized subclasses of inhibitory interneurons, which was the goal,” Shetty said. “Another fascinating aspect of this study is that transplanted human GABAergic neurons were found to be directly involved in controlling seizures, as silencing the transplanted GABAergic neurons resulted in an increased number of seizures.”
“This publication by Dr. Shetty and his colleagues is a major step forward in treating otherwise incurable diseases of the brain,” said Darwin J. Prockop, MD, Ph.D., the Stearman Chair in Genomic Medicine, director of the Texas A&M Institute for Regenerative Medicine and professor at the Texas A&M College of Medicine. “One important aspect of the work is that the same cells can be obtained from a patient.” This type of process, called autologous transplant, is patient specific, meaning that there would be no risk of rejection of the new neurons, and the person wouldn’t need anti-rejection medication.
“We will need to make sure that we’re doing more good than harm,” Shetty said. “Going forward, we need to make sure that all of the cells transplanted have turned into neurons, because putting undifferentiated pluripotent stem cells into the body could lead to tumors and other problems.”
The development of epilepsy often happens after a head injury, which is why the Department of Defense is interested in funding the development of better treatment and prevention options.
“A great deal of research is required before patients can be safely treated,” Prockop said. “But this publication shows a way in which patients can someday be treated with their own cells for the devastating effects of epilepsy but perhaps also other diseases such as Parkinsonism and Alzheimer’s disease.”
Shetty cautioned that these tests were early interventions after the initial brain injury induced by status epilepticus, which is a state of continuous seizures lasting more than five minutes in humans. The next step is to see if similar transplants would work for cases of chronic epilepsy, particularly drug-resistant epilepsy. “Currently, there is no effective treatment for drug-resistant epilepsy accompanying with depression, memory problems, and a death rate five to 10 times that of the general population,” he said. “Our results suggest that induced pluripotent stem cell-derived GABAergic cell therapy has the promise for providing a long-lasting seizure control and relieving co-morbidities associated with epilepsy.”
Explore further: Some types of epilepsy pose more risks during pregnancy
More information: Dinesh Upadhya et al, Human induced pluripotent stem cell-derived MGE cell grafting after status epilepticus attenuates chronic epilepsy and comorbidities via synaptic integration, Proceedings of the National Academy of Sciences (2018). DOI: 10.1073/pnas.1814185115
New drug cocktail increases human beta cell proliferation at rapid rates
Researchers at the Icahn School of Medicine at Mount Sinai have discovered a novel combination of two classes of drugs that induces the highest rate of proliferation ever observed in adult human beta cells—the cells in the pancreas that produce insulin. The result is an important step toward a diabetes treatment that restores the body’s ability to produce insulin.
The finding involved one drug that inhibits the enzyme dual specificity tyrosine-regulated kinase 1A (DYRK1A) and another that inhibits transforming growth factor beta superfamily members (TGFβSF). Together, they caused the cells to proliferate at a rate of 5 to 8 percent per day. The study, titled “Combined Inhibition of DYRK1A, SMAD and Trithorax Pathways Synergizes to Induce Robust Replication in Adult Human Beta Cells,” was published today in Cell Metabolism.
“We are very excited about this new observation because for the first time, we are able to see rates of human cell beta cell replication that are sufficient to replenish beta cell mass in human beings,” said Andrew Stewart, MD, Director of the Mount Sinai Diabetes, Obesity, and Metabolism Institute and lead author of the study. “We have discovered a drug combination that makes beta cells regenerate at rates that are suitable for treatment. The next big hurdle is figuring out how to deliver them directly to the pancreas.”
According to Dr. Stewart, none of the diabetes drugs currently on the market can induce beta cell regeneration in people with diabetes. In parallel with the Mount Sinai work, other researchers are studying pancreatic transplantation, beta cell transplantation, and stem cell replacement of beta cells for people with diabetes, but none of these approaches is in widespread use. Approximately 30 million people in the United States have diabetes and nearly 50 to 80 million more are living with prediabetes (also called “metabolic syndrome”). Diabetes occurs when there are not enough beta cells in the pancreas, or when those beta cells secrete too little insulin, the hormone required to keep blood sugar levels in the normal range. Diabetes can lead to major medical complications: heart attack, stroke, kidney failure, blindness, and limb amputation.
Loss of insulin-producing beta cells has long been recognized as a cause of type 1 diabetes, in which the immune system mistakenly attacks and destroys beta cells. In recent years, researchers have concluded that a deficiency of functioning beta cells is also an important contributor to type 2 diabetes, the most common type that occurs in adults. Thus, developing drugs that can increase the number of healthy beta cells is a major priority in diabetes research.
This current paper builds upon a study that Dr. Stewart and his team published in Nature Medicine in 2015, showing that a drug called harmine drove sustained division and multiplication of adult human beta cells in culture. They also learned that harmine treatment led to normal control of blood sugar in mice whose beta cells had been replaced with human beta cells. While this was a major advance, the proliferation rate was lower than needed to rapidly expand beta cells in people with diabetes.
In 2017, Dr. Stewart and his team published a second paper, in Nature Communications, which revealed the genetic abnormalities in insulinomas, a benign type of human beta cell tumor, and served as a “genetic recipe” to reveal targets for new drugs that can make beta cells regenerate.
In this current paper, Dr. Stewart and his team took advantage of the insulinoma “genetic recipe” which suggested that a combination of two classes of drugs—a DYRK1A inhibitor such as harmine with a TGFβSF inhibitor drug—would be able to synergistically increase beta cell regeneration. This proved to be true. However, this new drug combination is not without its hurdles. “Since these drugs have effects on other organs in the body, we now need to develop methods to deliver these drugs specifically to the beta cell in humans,” said Dr. Stewart. “We have the packages to deliver, but now we need a courier system to deliver them to the exact beta cell address.”
“Beta cell regeneration is a ‘holy grail’ for the treatment of diabetes,” said Peng Wang, Ph.D., Associate Professor of Medicine (Endocrinology, Diabetes, and Blood Disease) at Mount Sinai and first author on the study. “We are excited to finally have drugs that can induce beta cell proliferation at rates that are likely to be effective in people with type 1 and type 2 diabetes.”
“This is one of the most exciting series of discoveries in the field of diabetes and is a key next step in drug development for this disease,” said Dennis S. Charney, MD, Anne and Joel Ehrenkranz Dean, Icahn School of Medicine at Mount Sinai. “In a very short time, Dr. Stewart and his team of researchers have made incredible progress. Their important work truly holds promise for so many people.”
“We know that in order to achieve a cure for type 1 diabetes and to bring people to insulin independence, we will have to find ways to increase the numbers of functional beta cells,” said Francis J. Martin, Ph.D., Associate Director of Research and leader of the JDRF Beta Cell Regeneration and Survival Program. “Now, through the work of Drs. Stewart and Wang, we see that we can increase the rates of human beta cell reproduction to levels that were previously thought to be impossible. There are still challenges ahead, but this work brings us a little closer to therapies that can restore insulin production in people with the disease, and ultimately produce a cure.”
Explore further: Scientists discover mechanisms behind neonatal diabetesDiabetes-inducing blood vessel damage could be prevented by a growth factor
Scientists have identified a growth factor found in the kidneys that could minimise the diabetes-inducing effects of blood vessel damage.
The findings come in the wake of recent data from Diabetes UK which revealed that almost 4.6 million people nationwide have either type 1 or type 2 diabetes. Coupled with the recent NHS announcement that diabetes prescriptions now cost the UK £1bn a year, this latest research offers fresh hope for preventing an increasingly prevalent disease.
The research, led by experts in renal disease and translational health sciences from the University of Bristol and funded by the British Heart Foundation, focused on a group of growth factors categorised as VEGFs, or vascular endothelial growth factors. These are growth factors produced by cells that stimulate the formation of blood vessels.
In health, the subtype VEGFA plays a vital role in the kidney ensuring that blood vessels can filter and so allow the kidneys to effectively remove waste products and excess fluid from the body, while retaining other vital substances. However, too much VEGFA can damage blood vessels, causing them to leak substances that should be retained, which reduces their functionality and so exacerbates disease.
“We wanted to know whether we could protect the kidneys by using another growth factor from the same family of proteins to balance this effect,” explains Dr. Rebecca Foster, from Bristol University’s Translational Health Sciences, part of the Bristol Medical School.
Researchers were able to express this other growth factor subtype (VEGFC) in the kidneys of a mouse model as diabetes developed. The mice expressing VEGFC were protected from the development of diabetic kidney disease.
The study found that the growth factor VEGFC can counterbalance the effects of VEGFA by protecting the blood vessels. Pending further research, these findings could ultimately prove instrumental in how clinicians may protect those with diabetes from the development of diabetic kidney disease
The findings are published today [20 December] in the journal Diabetes.
“We now have a better understanding of how to protect filtering blood vessels in the kidney,” added Dr. Foster. “Blood vessel damage is an early event in the development of diabetic kidney disease. By activating the VEGFC signalling pathway, we can potentially prevent the development of diabetic kidney disease.
“The next step is to identify the key part of the VEGFC pathway that leads to reduced blood vessel leakiness. Then we need to demonstrate how this aspect could be selectively activated to protect people from diabetic kidney disease.”
Professor Metin Avkiran, Associate Medical Director from the British Heart Foundation, which funded the research, said: “In the UK, around 4.6 million people are living with diabetes. The damage this condition does to our blood vessels can cause diabetic kidney disease, as well as a whole range of heart and circulatory diseases. A third of adults with diabetes will die from one of these conditions.
“Understanding how we can protect blood vessels in people with diabetes could help us to find ways to prevent and treat many conditions, including coronary heart disease, stroke and vascular dementia.”
Explore further: Study questions certain treatments for diabetic patients at high risk for heart disease
More information: Karen L. Onions et al. VEGFC Reduces Glomerular Albumin Permeability and Protects Against Alterations in VEGF Receptor Expression in Diabetic Nephropathy, Diabetes (2018). DOI: 10.2337/db18-0045
FDA casts shadow on hemp win, calling CBD products illegal
The hemp industry still has work ahead to win legal status for hemp-derived cannabidiol, or CBD oil, as an ingredient in food or dietary supplements despite the big farm bill President Donald Trump signed this week designating hemp as an agricultural crop.
CBD oils have become increasingly popular in lotions, tinctures and foods, but their legal status has been murky and the Food and Drug Administration has sent warning letters to some companies making health claims for CBD.
In a statement following Thursday’s bill signing in Washington, FDA Commissioner Scott Gottlieb restated his agency’s stance that CBD is a drug ingredient and therefore illegal to add to food or health products without approval from his agency.
“Selling unapproved products with unsubstantiated therapeutic claims is not only a violation of the law, but also can put patients at risk, as these products have not been proven to be safe or effective,” Gottlieb wrote.
CBD is a non-psychoactive compound found in hemp, a version of the cannabis plant that is low in THC, the part of cannabis that gives pot its high.
An FDA-approved drug for the treatment of seizures, Epidiolex, contains cannabis-derived CBD. GW Pharmaceuticals’ syrup became the first prescription drug derived from the cannabis plant in June.
The FDA statement also specified parts of hemp that are safe as food ingredients, but the CBD stance disappointed advocates. Courtney Moran, a lobbyist for Oregon hemp farmers, said she plans to work with U.S. Sen. Ron Wyden, an Oregon Democrat, to nudge the FDA toward greater acceptance of CBD.
“We do hope the FDA does clear a pathway for these products that have already hit store shelves and are out in the marketplace,” Moran said. She said it’s an “opportunity for industry to educate the FDA.”
The FDA statement said three ingredients derived from hemp—hulled hemp seeds, hemp seed protein and hemp seed oil—are safe as foods and won’t require additional approvals, as long as marketers do not make claims that they treat disease.
Hemp, like marijuana, already was legal in some states before Trump signed the farm bill. But now hemp farmers will be able to buy crop insurance, apply for loans and grants, and write off their business expenses on their taxes like any other farmer.
Explore further: With OK from Congress, US hemp market set to boom (Update)AFM Has Doctors, Families Trying ‘Whatever Works’
His story starts out like many other patients’, but it has ended with a far more positive outcome than most. His mother is convinced that’s because Billy got the right treatment early from doctors who recognized what was happening to her son, who was then 13.
“We felt that our health care providers were very proactive in recognizing Billy’s situation and very aggressive in his treatment,” says Dawn Sticklen of Joplin, MO. “They already had a protocol in place if a child came in exhibiting the symptoms Billy did, so they knew exactly what they were going to do to try and help him.”
“He continues to improve.” Billy Sticklen spent 2 months in the hospital with AFM but has largely recovered.
Billy was among the lucky ones. The number of AFM cases confirmed in the United States this week hit a record high of 158 in 36 states. The disease has been on the national radar only since 2014 and remains a mystery. There is little to no consensus on what causes it, how to diagnose it, or how to treat it.
AFM is a rare but serious condition. It attacks the nervous system, specifically the area of the spinal cord called gray matter, which weakens muscles and reflexes. Almost all AFM patients — almost all are young children — first had a mild respiratory illness or fever akin to a viral infection.
In the absence of solid diagnostics, testing, and treatments, many families and doctors are relying on unproven remedies or word-of-mouth accounts of what has worked for others.
Billy’s medical challenges started with a respiratory illness that didn’t improve. Then one night he complained of neck pain, and the next morning he couldn’t use a spoon at breakfast. “As he was eating, he was having difficulty scooping cereal from the bowl into his mouth. His arms were shaking,” Sticklen recalls. She rushed him back to their primary care doctor, who sent them on to the local ER.
As Billy began to have trouble moving the full length of his arms, he was sent by ambulance to Children’s Mercy in Kansas City, MO.
“They explained Billy was showing polio-like symptoms they suspected were caused by 68. They’d had two other patients that summer with the same symptoms and same virus,” Sticklen says.
Billy’s condition worsened quickly. His legs began to weaken, and an MRI showed inflammation running the length of his spinal cord. Doctors immediately started him on high doses of oral steroids and plasmapheresis, a process that cleans the blood to get rid of proteins or antibodies that could be causing harm. Still, improvement didn’t come right away.
“By the next day, he could not even hold his head up on his own. He tried to stand, and he fell and he couldn’t raise his arms,” Sticklen recalls.
Doctors continued with the treatment — five rounds of plasmapheresis over 10 days, then 2 weeks of steroids — and Sticklen says in time, her son’s condition stabilized. “Damage had already been done by the time they started the treatments,” she says. “But I think treatment kept the paralysis from getting worse.”
Billy spent 2 months in the hospital and another 2 months in a rehabilitation facility. Once he was sent home, he still had weakness in his left arm and shoulder and his right glute, but he could walk with the aid of a walker. The family says intensive physical, occupational, and aqua therapy, plus regular sessions with a physical trainer, brought continued improvements, and 8 months later, Billy was walking on his own.
Sticklen says her son, now a 17-year-old high school senior, is nearly back to normal. “He can’t run like he did before. He can’t lift his left arm higher than chest height, and he has a hard time holding things that are heavy in his left hand. But he continues to improve,” she says. “If you were to see him, you would not notice his deficiencies without me pointing them out to you.”
Sticklen has since befriended several other AFM parents and knows that her son’s recovery has been more complete than many others. This illness appears to be very individualized, so she says it’s hard to know the exact reasons why. But she now lobbies hard to make parents and doctors know that it’s vital to get an early diagnosis of AFM and better understand treatment options. She believes it’s the best way to help children who get this mystery illness.
“I know it’s only anecdotal, but I think early intervention is what made the difference for Billy. I think it stopped the inflammation from doing the most damage to my son, preventing more paralysis and possibly even saving his life, because it didn’t deeply impact his breathing,” Sticklen says. “I am aware this isn’t scientifically proven though, which is why the CDC needs to do even more to understand this illness and how best to treat it.”
Lack of Guidelines
The CDC says it’s investigating AFM on several fronts — working to get more data, monitoring AFM activity, confirming cases, and working with national experts to explore treatments. But the agency stresses there are no firm recommendations or official guidelines on how to treat it.
That message is echoed by the American Academy of Pediatrics (AAP), which says the group’s Red Book, a guide on how to diagnose and treat infectious diseases, does not have anything definitive on how to best treat children who have AFM symptoms.
The CDC says there continues to be a “paucity of published evidence for treatment of AFM, limited to case reports and case-series of patients with AFM.” So it says that when doctors see AFM cases, they must consult with experts treating AFM patients.
In the hopes of learning more about what is and isn’t working, the CDC now has a comprehensive website for assessing AFM patients. It contains tips for doctors to collect blood, stool, spinal fluid, and respiratory specimens from potential patients. And it outlines what it calls interim considerations for treatment that have been updated.
The therapies referenced include:
- Corticosteroids (anti-inflammatories)
- Intravenous immunoglobulin (IVIG), which is a mixture of antibodies prepared from many donors
- Plasmapheresis
- Antiviral medications
- Fluoxetine (Prozac)
- Interferon, a protein naturally produced by cells to fight viruses
- And immunosuppressive medications or biological modifiers
The CDC has been hesitant to declare any one treatment as being a proper protocol because the numbers aren’t there yet.
But the agency stresses there’s no indication any of these treatments should be preferred or avoided. And while it says there’s no evidence IVIG could harm patients, it warns there is some evidence that corticosteroids, interferon, and immunosuppressants could cause harm, and there are risks with plasma exchange. The agency also points out that a recent study on a mouse model didn’t show fluoxetine to be effective.
“As research continues to identify the cause of AFM and how to best manage AFM, we hope that we will be able to manage patients with AFM even better,” says Mobeen H. Rathore, MD, a spokesman for the American Academy of Pediatrics and chief of infectious diseases and immunology at Wolfson Children’s Hospital in Jacksonville, FL.
“From what I have observed, I think the CDC has been hesitant to declare any one treatment as being a proper protocol because the numbers aren’t there yet,” Sticklen says. “But the problem of waiting for numbers to show a pattern is that too many kids have to suffer irreparable damage before they finally decide on an official protocol. It’s a Catch-22 situation, and children are stuck in the middle.”
Treatment on the Front Lines
In the absence of clear-cut treatment guidelines, hospitals are coming up with their own approaches.
“We don’t have good consensus or good data on the best therapies for AFM at this point,” says Samuel Dominguez, MD, PhD, a pediatric infectious disease expert with Children’s Hospital Colorado who has helped treat more than 20 AFM patients. “We need better studies to understand which of these is the best way forward. So for now, different institutions are taking different approaches.”
He says doctors at his hospital have done research on several therapies, including fluoxetine, which showed no effectiveness, and IVIG, which showed some benefits in a mouse model. So for now, his institution is mainly using IVIG as a first-line treatment.
“We think it is safe, and we think it may be beneficial. We think IVIG can neutralize the virus, although we’re not entirely sure,” Dominguez says. “IVIG is pooled antibodies from multiple blood donors, so if you have enough antibodies against a pathogen causing disease, it makes sense that it could neutralize it.”
Cynthia Wang, MD, an assistant professor of pediatrics and neurology at UT Southwestern Medical Center in Dallas, who’s treated patients with AFM since 2014, says her institution generally uses a combination of high-dose IV steroids, IVIG, and/or plasma exchange, even though she says none of these therapies has been shown to significantly improve the outcome of children with AFM.
“The existing data has not demonstrated that any acute treatment can halt or improve the course of AFM,” Wang says.
But, she says, it’s often difficult to know for sure what treatment will work for a child in need of medical care.
“There are autoimmune disorders of the spinal cord and nerves that can resemble AFM but respond favorably to immunotherapies,” she says.
It’s better to try a treatment that historically has shown little or no benefit against AFM if it turns out the child has an autoimmune disorder unrelated to AFM.
“Sometimes we do see stabilization of weakness or mild improvements in strength following immunotherapies in children with AFM,” Wang says. “However, it is difficult to discern if these treatments are responsible for the positive changes or if they are related to the supportive care that a child receives when he or she is hospitalized.”
Parents Back Particular Treatments
Mason Smith’s family says they do think IVIG helped him. He woke up one morning in 2017 unable to stand — days after contracting a respiratory illness — and was rushed to a hospital in San Antonio, TX. Once there, his paralysis progressed quickly, moving up the teen’s legs and then up his back.
At first, doctors diagnosed him with Guillain-Barre syndrome, a condition where the immune system attacks the nerves. Doctors ordered treatment similar to what’s used to help many AFM patients — a 5-day course of IVIG, followed by 5 days of steroids and 5 days of plasmapheresis. Ultimately, his diagnosis was AFM.
“We started to see hope.” Cami Axton was 2 when she got AFM. Her grandmother, Lamay, says a combination of IVIG, steroids and fluoxetine saved her life.
“As a parent, knowing what I know now, I wonder if giving him steroids with the IVIG would not have been a better approach,” says his mother, Sherri Smith. “But I do think the IVIG had something to do with the halting of the progression of the illness. We got that within 24 hours, and in the same time frame, the paralysis stopped. I think that made a difference.”
LaMay Axton thinks treatment helped her 2 1/2-year-old granddaughter, Cami, too. The toddler, whom she is raising, was affected in 2016. Axton says while the first hospital they went to didn’t seem to recognize the urgency of the situation, even as the girl’s paralysis stretched along her legs and arms and up to her diaphragm, she was eventually transferred to a military hospital that jumped into action.
By that time, 15 hours had passed, Cami’s diaphragm was paralyzed, and the child was in acute respiratory failure, a potentially deadly condition where the lungs rapidly fill with fluid, cutting off oxygen to the body. But the hospital treated her with IVIG, steroids, and fluoxetine, and LaMay believes that saved the girl’s life.
When Cami left the hospital, she needed a ventilator and was considered a quadriplegic. But today, her grandmother says, she has beaten all the odds. After a year, they removed her breathing tube, and Cami is now learning how to walk again after having upper and lower nerve transfer surgeries to help regenerate her dead nerves. Axton believes the fluoxetine helped make these improvements possible, even though recent studies have called it ineffective.
“When she started taking the [fluoxetine], that is when she started to heal. That is when we started to see hope. Her right hand moved a bit better and her right arm got stronger,” Axton says. “I don’t know what the IVIG did. I think it and the steroids probably played a role in keeping her body strong enough to keep on fighting.”
Looking for Better Options
Wang says the complexity and speed at which AFM progresses ultimately present very serious challenges for the medical field.
“In the past 4 years, there has been increasing awareness about AFM; however, it is not clear this has led to better outcomes for children who develop the disease,” she says. “This may be in part due to how quickly the illness progresses. By the time many children with AFM present to medical attention, they may already be at the peak of their weakness and already sustained significant damage to their spinal cord.”
Axton says she understands searching for a treatment plan is difficult, but she thinks if more attention had been paid to this illness when it emerged several years ago, there would now be a better understanding of how to treat it.
“If they had taken the appropriate measures to follow up with families and children, then they would know what protocols work,” she says. “Time is of the essence.”
So where could answers lie? The CDC says it still doesn’t know enough about AFM causes or triggers to suggest any preventive measures. Many in the medical community say an effective antiviral would be welcome. But the CDC says it’s tested several, and none has been effective against the viruses apparently at play in AFM.
While the condition is still considered rare, its numbers are increasing every other year, and Dominguez and others wonder if ultimately a vaccine might help. “If the pattern continues, what we really are going to need is a vaccine to prevent the disease as we ultimately did with polio,” he says. “I think the best course of therapy if this continues is prevention.”
But Wang wonders about the impact of a large-scale vaccination program. “Each year, the CDC receives thousands of reports of suspected severe vaccine reactions in children. Are we prepared to take on this risk to protect 100-200 children from developing AFM every other year? That is a very difficult and heartbreaking question,” she says.
For now, if you think your child has AFM, experts say it’s best to connect with pediatric hospitals that have both neurological and infectious disease experts. There is also widespread agreement that once your child has stabilized, aggressive physical, occupational, and speech therapy can help. Several AFM parents have just created an organization, AFM Association, aimed at helping other caretakers find resources if their children are affected. They’ve launched a website, afmanow.org, they hope will provide support for parents looking for more information.
Smith says she hopes her son’s case and that of other children who have gotten AFM will lead to more answers, and she would like federal health investigators to be more aggressive in reaching out and following their cases.
“We want to be part of the solution. If you can use our information to streamline treatments or cures or prevention, then ask away. There are nearly 600 AFM families connecting on a webpage. We can respond to you. Use us. Maybe that is unconventional, but this is an unconventional situation,” Smith says.
A year later, Smith’s son Mason is a paraplegic, basically immobile from the waist down. His chest is impaired too. But he is pushing ahead with his life plans. Now in a wheelchair, he started college on time and is studying to be a lawyer.
“AFM is a terrible disease, but these kids who have it are warriors, like nothing I have ever seen,” Smith says. “In one of his college essays, Mason wrote that he didn’t know if his body would heal on its own or if science would catch up and find a way to heal him, but either way, he wasn’t going to just wait around. He was going to go on with life. And he has.”
She says as challenging as this has been for her family, she still hasn’t lost hope that more answers will be found.
“This has been a really terrible chapter in our life. I’m not going to sugarcoat that. But I can’t wait to see what happens as medicine moves forward. I pray for treatment or a cure every day — for my child, the ones I’ve met, and any others that come after them.”
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