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How Big Is Vertex's News for Its Potential Diabetes Cure? – Motley Fool

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Returns as of 11/22/2021
Returns as of 11/22/2021
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Vertex Pharmaceuticals (NASDAQ:VRTX) recently announced positive results from an early stage clinical study of cell therapy VX-880 in treating type 1 diabetes. In this Motley Fool Live video recorded on Oct. 20, Fool.com contributors Keith Speights and Brian Orelli discuss the significance of this news for an experimental drug that has been referred to as a potential cure for type 1 diabetes.

Keith Speights: Well, Brian, let’s switch to some non-COVID news. Vertex Pharmaceuticals announced encouraging results from a phase 1 study of its experimental cell therapy that has been touted to hold the potential to effectively cure type 1 diabetes in some patients. What’s your take on these results, and how big is this data for Vertex?
Brian Orelli: This was an early stage study. It just had one patient with type 1 diabetes. That’s the type that typically you get diagnosed with as a child. You don’t have functional cells in the pancreas to make insulin. This patient was injected with cells that can make insulin, and then they acted fairly much like normal. They weren’t producing insulin all the time. They responded to the fact that you ate food, and then that triggered production of insulin, which is exactly what the pancreas would do.
The patient went from needing 34 units of insulin per day to needing just 3 units. That’s whatever 90-something percent drop in the need for insulin. Looking at HbA1c, which is a measure of long-term sugar levels, that went from 8.6% to 7.2%. Under 7% is basically the gold standard. Pretty close there, and the patient was obviously really high at 8.6. Getting close to 7.2 is definitely a step in the right direction.
As promising as this is, it’s really only the first step. This was a half a dose and only 90-day data. They are obviously going to test the full dose, and then we obviously need longer data to know if the cells stick around and does it continue to help the patient. Do they even get better than this, or does it go down? Does the insulin production go down, and that means they need to start taking more insulin on their own?
Then obviously, it’s not realistic to be a cure for most patients since by doing this, you have to undergo lifelong immunosuppressants, which cause their own risk. In this case, the patient had five potentially life-threatening hypoglycemics. That’s low blood sugar level episodes. Obviously, the risk of not doing anything was counteracted by the risk of potentially getting an infection while on immunosuppressants. But the average type 1 diabetic who has their blood sugar levels fairly under control with their insulin probably isn’t going to be able to justify taking immunosuppressants for the rest of their lives.
Vertex is working on an encapsulating version of the cells. That would allow them to evade the immune system, and they’re looking to start a clinical trial with the encapsulated cells next year. I think that’ll be the really telling thing here. This is very promising, but Vertex’s shares were basically flat on the news, and I think that’s reasonable. I think it’s definitely good news and is a step in the right direction, but I think it’s still too early to really put a valuation on it in terms of Vertex’s share price.
Speights: While type 1 diabetes is not a rare disease, the initial focus for Vertex here is rare, and so they’re going after a relatively small market. I think what you’re talking about with the trial that they hope to start next year for encapsulated cells, if that’s successful, then that could open up a really big market opportunity for Vertex.
Orelli: I agree.
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Health and Lifestyle

What Causes Narcolepsy? These Factors May Play a Role | Health.com – Health.com

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In many cases, chronic sleepiness is tied to low levels of certain brain chemicals.
When diagnosed with a new condition, the first question is almost always "How?" We naturally want to know exactly what brought us to that moment. This curiosity may be even stronger with something like narcolepsy, a chronic sleep disorder that is both under-recognized and misunderstood, according to the nonprofit Project Sleep.
While scientists have yet to pinpoint the exact cause of narcolepsy, a majority of cases are tied to low levels of a brain chemical involved in regulating our sleep-wake cycle, according to the Cleveland Clinic. And other factors are thought to play a role in triggering the disease process.
Here's how sleep experts explain the causes of narcolepsy.
Before delving into the causes, let's consider what narcolepsy looks like.
Narcolepsy is characterized by excessive daytime sleepiness, hallucinations, sleep paralysis, vivid dreams, and more, says Steven Thau, MD, division chief of the Pulmonary and Sleep Medicine Department and medical director of the Sleep Center at Phelps Hospital/Northwell Health.
It can present at any point in a person's life, but most commonly it initially occurs in a person's teens or 20s, Dr. Thau tells Health.
While each case is different, excessive daytime sleepiness is generally the first symptom to surface. Symptoms such as hallucinations, sleep paralysis, and cataplexy may follow, says Manjamalai Sivaraman, MD, FAASM, a sleep medicine specialist and neurologist at the University of Missouri. The latter may not happen for a few years, if at all. 
RELATED: What Are the Types of Narcolepsy? Sleep Experts Explain the Differing Presentations of This Sleep Disorder
There are two main types of narcolepsy: types 1 and 2. There's also a third known as secondary narcolepsy. (More on that one below.)
Narcolepsy type 1 covers anyone who has low levels of hypocretin (a brain chemical that controls wakefulness) and experiences cataplexy (sudden muscle loss), according to the Mayo Clinic. Type 1 makes up about 70% of narcolepsy cases, says Richard Bogan, MD, a medical officer at SleepMed, Inc. and associate clinical professor at the University of South Carolina School of Medicine and the Medical University of South Carolina in Charleston.
People with narcolepsy type 2 may experience all the symptoms of narcolepsy except cataplexy—and their symptoms are often less severe, says the National Institute of Neurological Disorders and Stroke (NINDS). And their hypocretin levels are usually normal.
While there are no known ways to prevent or cure type 1 or type 2 narcolepsy, NINDS notes that lifestyle changes and medications may be helpful for maintaining alertness and managing other symptoms.
RELATED: Is Narcolepsy Genetic? What Sleep Experts Say About Inheriting This Chronic Disorder
While the science is still evolving, here's what's known so far.
People with type 1 narcolepsy have very low levels of brain chemicals called hypocretins. These chemicals, first discovered in 1998, are important for a couple of reasons, per the Division of Sleep Medicine at Harvard Medical School. For one thing, they keep people awake and alert. They also prevent people from drifting off into REM (rapid-eye movement) sleep while they're awake.
In people type 1 narcolepsy, however, the nerve cells that produce hypocretins die off, and the resulting dearth of these chemicals leads to sleepiness and poorly regulated REM sleep, per Harvard's Sleep Medicine Division.
Research by two separate investigative teams suggests that type 1 narcolepsy is caused by a severe loss of neurons that produce these chemicals, per a 2015 review in the New England Journal of Medicine.
As for what causes type 2 narcolepsy? It's possible that people who have this form of the disorder may sustain less injury to their neurons than those with type 1, according to that same review, which references a 2009 Sleep study. But data on the disease process involved in type 2 narcolepsy "are quite limited," notes the New England Journal.
Bottom line: Scientists don't fully understand what triggers the loss of hypocretin-producing cells, although it appears that one or more of the following factors may be involved:
Most people with narcolepsy, especially type 1, have a gene variation known as HLA-DQB1*06:02. It is a variation of the HLA-DQB1 gene, which "provides instructions for making part of a protein that plays an important role in the immune system," according the US National Library of Medicine. The risk of narcolepsy associated with this variation and related genes is unclear to researchers at this time.
That same gene variation is found in 50% of people with narcolepsy type 2, but only 12-30% of the general population, according to the New England Journal.
Speaking of risk factors, narcolepsy isn't a disorder that tends to run in the family. According to NINDS, just up to 10% of people with type 1 narcolepsy have a close family member who presents with similar symptoms. If a parent has narcolepsy, the odds of passing it down to a child is only about 1%, says Mayo Clinic.
People with the HLA-DQB1*06:02 gene variation may be at increased risk of developing narcolepsy after being exposed to a trigger, such as an infection, says NINDS. That's based on studies of people after they developed narcolepsy.
Upper airway infections such as streptococcus pyogenes and influenza A (including H1N1) are strongly associated with narcolepsy, per a 2011 study in the Annals of Neurology, especially in cases where it begins in childhood, notes Dr. Sivaraman.
We know that people with narcolepsy type 1 have low hypocretin levels—but why? A leading theory considers narcolepsy to be an autoimmune disorder.
"There are supporting evidences for autoimmune destruction—the immune system in one's body attacking its own healthy cells—of hypocretin neurons in the hypothalamus of the brain," says Dr. Sivaraman. To break it down, if this theory is true, then a person's own immune system is responsible for the brain lacking in hypocretin.
As Dr. Thau puts it, in this case, "the cells that control wakefulness are damaged."
Currently, researchers are working on using immunotherapy to reverse this loss, Dr. Bogan tells Health. According to a 2020 review published in Current Treatment Options in Neurology, small studies have shown an improvement in symptoms for narcolepsy patients after using immunotherapy treatment, especially those who recently presented with the disease. However, the experiments were uncontrolled and did not have clear endpoints, requiring more research to achieve any definitive answer on the treatment's benefits.  
RELATED: 7 Narcolepsy Symptoms to Know, According to Sleep Specialists
Unlike narcolepsy types 1 and 2, doctors do know the "why" behind secondary narcolepsy. This form of narcolepsy occurs when the brain's hypothalamus region gets damaged, according to Harvard's Division of Sleep Medicine.
These people can experience all of the same symptoms as those with types 1 and 2. However, they might also have severe neurological problems and require a large amount of sleep—typically 10 hours or more.
"In rare cases, brain lesions or diseases such as tumors, vascular malformations, strokes or inflammatory diseases of the brain can result in the destruction of the signaling pathways that increase brain activity and promote wakefulness," says Dr. Thau.
According to the National Health Service, secondary narcolepsy causes include:
As Dr. Thau notes, "a healthy lifestyle and avoiding smoking or the use of illicit drugs decrease the risk of some of the disorders that cause secondary narcolepsy."
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Mount Laurel police asks public's help in finding child – Courier Post

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MOUNT LAUREL – Police are asking the public’s help in finding a 6-year-old girl who was allegedly abducted by her non-custodial mother.
The girl, Grace Craytor of Pennsauken, was last seen around 7:10 p.m. Monday with her mother, Kristina Maletteri, at Lifetime Fitness in Mount Laurel, according to township police.
The girl’s father, who has a full custody order for Grace, had invited Maletteri to swim with the child during a supervised visit at the facility at Church and Fellowship roads, said a police account.
 “At some point, Ms. Maletteri is said to have taken her daughter and left the area without consent,” the account said.
Maletteri is known to drive a 2017 silver Audi Q3 with New Jersey license plates “S64MPY.”
The missing child is 46 inches tall, 70 pounds, with blonde hair and hazel eyes, police said.
Anyone with information is asked to call Mount Laurel police at 856-234-8300 or the confidential tip line 856-234-1414, extension 1599.
Tips can also be emailed to Lamaro@mountlaurelpd.org.
Jim Walsh covers public safety, economic development and other beats for the Courier-Post, Burlington County Times and The Daily Journal.
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Healthy Snacks for the Office – How to Pack Food for Work – menshealth.com

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Our product picks are editor-tested, expert-approved. We may earn a commission through links on our site.
Allow these experts to help pack your lunchbox.
Buh-bye, vending machine. Here are four easy ways to boost your energy at work. Plus, three moves to make any lunch meeting extra appetizing.
Combine carbs and protein for long-lasting energy, says Marisa Moore, R.D.N., an integrative dietitian. Mix roasted, lightly salted sunflower seeds and dried blueberries in a small jar for a snack that’s sweet, salty, and crunchy. Bonus: The unsaturated fats in the seeds will keep you feeling full.
A favorite of Cara Harbstreet, R.D., of Street Smart Nutrition, is protein- and omega-3-rich tuna or salmon (StarKist makes packaged versions) spread on sliced cucumbers or mini bell peppers. Drizzle with your favorite hot sauce for a tiny yet protein-packed meal.
Jordan Mazur, R.D., director of nutrition for the San Francisco 49ers, suggests these key ingredients: shredded rotisserie chicken for lean protein; pistachios, walnuts, pumpkin seeds,dried tart cherries, and dark chocolate chips for a healthy trail mix; and antioxidant-rich blueberries or grapes.
Don’t go more than three to four hours without eating, to help keep your blood sugar steady. You can avoid mindless snacking by setting an alarm to get up every hour instead of reaching for the chips, says Kelly Hogan Laubinger, R.D
As we head back to the office, those DIY outdoor lunches can still be the thing to do.
TRY A HEARTY SALAD IN A JAR, says Moore. Build it from the bottom up: Start with a vinaigrette, then add chickpeas, carrots, tomatoes, olives, and cucumbers. Add feta to the top for a salty, tangy finish. Close, and shake when ready to eat.
REINVENT YOUR SANDWICH. Slapping protein and a salad’s worth of greens between whole-grain bread works well, too: Try sliced turkey or canned tuna, topped with sprouts, cucumbers, leafy greens, avocado, and tomato.
MAKE A HEALTHY CHEESE BOARD, says Harbstreet. Go with hard cheeses like cheddar and Gouda and a soft cheese like cottage. Pair pita bread or crispy crackers with jerky or low-sodium deli meats. Then toss in pistachios and blueberries.
This article appears in the October 2021 issue of Men’s Health.

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