First FDA-Approved Drug Derived from Marijuana

An FDA advisory committee has recommended the approval of a new drug, called Epidiolex, for the treatment of several types of epilepsy in children . If the FDA accepts the recommendation, Epidiolex will be the first FDA-approved drug to contain cannabidiol (CBD), a non-psychoactive substance purified from marijuana (cannabis).

Following the discovery that CBD had certain medical benefits, a number of states (30 at last count) legalized marijuana for certain medical purposes. Several other states legalized just the CDB extract. Relatively pure CBD is now widely sold over the internet for a host of medical and quasi-medical conditions, including as a sleep aid, an anti-anxiety treatment, an anxiety reliever, and even as a cure for certain types of cancer. But while some of these alleged benefits of CBD may be true, none has been proven by rigorous scientific studies. In addition, the purity and consistency of non-FDA-approved drugs sold over the internet can be suspect.

What makes Epidiolex different is that its developer and manufacturer has gone through the necessary testing to prove its medical efficacy. As a result, Epidiolex is poised to receive FDA approval for the treatment of two rare types of epileptic seizures. A distinct advantage of FDA approval is that patients with the forms of epilepsy targeted by the drug can be assured of the drug's purity and consistency of dose from one prescription to the next, because the drug will be produced according to best pharmaceutical practices.

With further testing, the FDA could expand the approved use of CBD to include other medical conditions. In the meantime (even if the FDA does eventually approve CBD, or even marijuana, for other uses), I doubt that marijuana-users will give up their favorite form of self-medication.

Fitness and Dementia

More than 5 million people in the U.S. are living with Alzheimer's disease, the most common form of dementia. There is no cure or prevention for Alzheimer's disease, despite all the research into its causes.

Now a new study spanning 46 years offers some tantalizing clues. Way back in 1968, researchers in Sweden enrolled 191 women 38-60 years old into a study of cardiovascular fitness, as measured by their capacity to exercise on a bicycle until they became so fatigued that they had to stop. Based on their workload achieved, they separated the women into "low", "medium" and "high" fitness groups. Then they followed the women for the next 44 years, looking for signs of dementia, among other health issues. Surprisingly, they found that compared to the "medium" fitness group, those in the "high" fitness group were 88% less likely to develop dementia. Among those who did develop dementia, high fitness delayed the time to onset of dementia by 5 years, compared to the medium fitness group.

A word of caution; these results show a correlation only; they do not prove that high fitness per se is what is reducing the risk of dementia. Perhaps the high fitness group also has a better diet, or just better genetics to begin with. Nevertheless, the results are intriguing, and may point the way to additional research to tease out the actual cause. Anything we can do to solve this baffling and debilitating disease would be welcome.

A Blood Test for Concussions

Traditionally, people who are suspected of having a concussion are first evaluated by a series of neurological tests known as the Glasgow Coma Scale. If the results of the Glasgow test warrant it, a computed tomography (CT) scan is conducted to try to detect intracranial lesions requiring treatment. Problem is, most CT scans are negative for lesions, meaning that a lot of CT scans are done unnecessarily.

Now, however, there's a blood test for concussions. The test measures the levels of certain proteins that are released into the blood by damaged brain tissue in the first 12 hours after a concussion. The test is highly accurate; it correctly identifies a concussion 97.5% of the time in patients also identified by a CT scan, and correctly identifies a person as not having a concussion 99.6% of the time when a CT scan is also negative. Aside from being less expensive, the blood test could be done in the field (perhaps even on the sidelines of a sporting event), allowing better decisions about who does and doesn't need a CT scan.

The blood test is currently approved only for use on adults, but the company that makes the testing device is working toward getting it approved for children as well. That would make it available for the more than a million boys who play high school football each year, as well as athletes in other contact sports.

The F.D.A. Cracks Down on Fake Opioid Addiction Treatments

Once again, the largely unregulated dietary supplements industry earns itself another black eye. The latest scam is products that purport to ease the symptoms of opioid withdrawal. Who stoops so low as to prey on victims of opioid addiction?

Case in point; a product called Opiate Detox Pro. The company's website claims that its ingredients ease opioid withdrawal symptoms, despite the fact that the company cites no research to back up its claim and has no intention of ever conducting any research. The company says that if the product doesn't work, the user can get his/her money back. Big deal!

The F.D.A. shouldn't have to keep ferreting these guys out and cracking down on them, but such is the current lack of effective regulation of dietary supplements in general. Anyone is free to market dietary supplements, as long as they don't claim to treat a specific medical condition.

With any dietary supplement, its buyer beware.

Digital Pills

The FDA recently approved a pill that will send an alert when the pill is actually taken, according to a recent article in The Inquirer. The pill contains a tiny sensor about the size of a grain of sand. When the pill is swallowed, acid in the stomach activates the sensor to send a signal to a skin patch worn by the patient. The patch then notifies a cell phone that the medication has been taken.

The first digital pill is expected to contain a drug used to treat schizophrenia and other serious illnesses - conditions in which patient compliance is often an issue. But one can envision all kinds of uses of the technology. Of course the pill raises issues of privacy and of how the ability to collect data about your pill use might lead to coercion. What if, for example, health insurers were to "incentivize" you by lowering the price for pills with sensors to get you to use them? How would they use the data they could collect on you? What if they were to insist on receiving data about your medication compliance as a condition of future health care? These are issues that should be considered before digital pill use becomes widespread, not after.

On the other hand, digital pills might be a real help for people who want to be compliant but just can't remember to take their medications, such as the aged. Like any new technology, it'll be how we use it that will determine its ultimate usefulness.

Of course, pills with embedded sensors will cost more, too...

Aaron Hernandez Suffered From CTE

Remember Aaron Hernandez, the standout football player at the University of Florida who played for the New England Patriots from 2010 to 2012? He was convicted of murder in 2015 and later hanged himself in his prison cell.

Now neuropathologists at Boston University have released his autopsy report. According to CNN News, the report indicates that Hernandez had the worst case of chronic traumatic encephalopathy (CTE) ever seen by BU pathologists in someone of his age. He was only 27 years old at the time of his death. Whether his behavior in the last few years of his life was related to the CTE is unknown.

There has been a lot of discussion lately about the effects of repeated head trauma, and how it might lead to CTE. The association between head trauma and CTE is so strong now that the NFL has set up a fund to provide up to five million dollars per retired player for medical expenses related to CTE. And there have been changes to the rules of football to try to reduce incidences of head trauma in current players. Nevertheless, CTE is likely to remain a problem for football for some time to come; Boston University pathologists have found evidence of CTE in nearly all of the brains of former NFL players that have been examined so far.

According to a USAToday news report, retired sportscaster Bob Costas predicts a decline in interest in the sport of football unless something is done to make it safe. It will be interesting to see whether he is right or wrong.

Head Trauma in Adolescence is Associated with Multiple Sclerosis Risk

Multiple sclerosis (MS) is a progressive, debilitating disease in which the body's immune system attacks the fatty layer that insulates some nerves, leading to permanent nerve damage. Based on studies in animals, there has been a suspicion that trauma to the central nervous system may be a risk factor for the development of MS. Now a Swedish study provides new evidence that repeated concussions during adolescence are associated with increase the risk of MS later in life. (Sweden keeps particularly good medical records on all its citizens, making comprehensive population studies possible).

The study's authors examined the records of all Swedes who had been diagnosed with MS since 1964 - over 7,000 people in all. Then they looked at the number of concussions each person with MS had had during adolescence, comparing it to the number of concussions in 10 other people without MS matched for age, gender, and county of residence. The key finding was that persons who had had two or more concussions during adolescence were more than twice as likely to develop MS as persons who had not had any concussions.

No one knows yet how concussions might trigger the development of MS, if indeed they do. One theory is that damage to central nervous system might lead to the release of nervous tissue breakdown products that cause the immune system to attack neural tissue. But that remains to be tested.

The First Gene Therapy to Cure a Fatal Brain Disease

Gene therapy has now been used to cure a fatal brain disease called adrenoleukodystrophy (ALD), a genetic disorder that occurs in approximately one in 20,000 boys. Even more startling is that the vectors used to deliver the normal form of the ALD gene to the boy's cells are disabled viruses similar to the AIDS virus, called lentiviruses.

In the new technique, hematopoietic (blood-forming) stem cells are removed from the patient's bone marrow and exposed to lentiviruses that contain normal copies of the ALD gene. (Lentiviruses are used because like the AIDS virus, they are better than most other viruses at inserting genes into cells.) The stem cells are then returned to the patient's bone marrow, where they grow and multiply.

Now for the really interesting part. Some of the corrected stem cells make their way to the brain, where (apparently because of the environment there) they develop into neural supporting cells called glial cells, correcting the original defect in glial cell formation.

The timing of treatment is critical, however. It takes about a year between initial diagnosis and the time the treatment is effective. Unfortunately, ALD can progress so fast that patients who already have obvious symptoms of the disease may not be able to be saved. The best candidates for a cure are patients who (by virtue of hereditary history) are thought to be at risk for the disease, and whose only signs of the disease so far are changes in their brain scans.

Driver Charged in Texting-While-Driving Incident

Criminal charges have finally been lodged against Jack Dillon Young, the young man who allegedly caused an auto accident in Texas that killed 13 people back in March of this year (see this blog, Apr. 2, 2017). At the time, Mr. Young admitted that he had been using his cell phone at the time of the crash and that he had taken prescription drugs, according to a news report.

Among the multiple charges against Mr. Young are 13 indictments for intoxication manslaughter and manslaughter; one for each of the accident's victims. There are no charges directly related to his cell phone use because apparently it was not a specific crime at the time of the accident.

Perhaps prompted by widespread media coverage of the accident, in June of this year Texas passed a law banning texting while driving. The law will take effect on Sept. 1, making Texas one of the last states to adopt some form of texting while driving ban. The three current holdout states are Arizona, Montana, and Missouri.

Mr. Young is currently free on $380,000 bail pending his trial. 

Prevalence of CTE in NFL Football Players

Repetitive blows to the head can lead to a debilitating chronic brain disorder called chronic traumatic encephalopathy (CTE) later in life. CTE is of particular concern to football players, boxers, and even soccer players. How common is CTE among certain athlete groups, and should we be concerned?

Of course we should be concerned. We would be remiss if we didn't try to find out how and why it is happening to our athletes, and how to prevent it in the future. But we should not scare people needlessly with sensationalist headlines. Take, for example, a CNN news headline: "Study: CTE in 99% of dead NFL players". Or this one in the Daily Mail (a British journal): "Biggest Ever NFL brain study diagnoses CTE in 99% of deceased players' brains". Read more closely and you find that the correct statement is that CTE was found in 99% of deceased ex-NFL players whose brains were donated to scientific research by their relatives (my emphasis). It's not a group that is representative of all deceased NFL players, by any means. It is likely that many if not most of these relatives donated their loved-ones' brains because they already suspected the presence of CTE based on their loved ones' behavioral symptoms while still alive. A definitive diagnosis of CTE can only be made at autopsy. CTE generally isn't looked for in a deceased player without symptoms of CTE, so in fact we really don't know how prevalent it is.

Clearly, CTE is present in many ex-NFL players, and its time to do something about it. The numbers may turn out to be higher than initially thought, and that would be sad. But there's no evidence that it's as prevalent as 99%, at least at the moment. Let's approach this problem with level heads and open minds.

Some Sunscreens Slow the Progression of MS in Mice

Sometimes it's better to be lucky than good. Quite by accident, researchers have discovered that ingredients found in Coppertone and other sunscreens may be able to slow the progression of multiple sclerosis (MS). The researchers were studying the known protective effect of UV light on the development of multiple sclerosis (MS) in mice prone to the disease. In the course of these experiments they administered various sunscreens to mice without exposing them to UV light, as controls. Surprisingly, several of the sunscreens slowed the development of MS all on their own. Coppertone was particularly effective. Further tests revealed that two ingredients in particular, homosalate and octisalate, were responsible.

These findings sound like a real breakthrough, but realistically they just represent interesting observations that are worthy of being pursued. A lot more work will need to be done before we will know how these compounds exert their protective effect in mice, and whether they ultimately might be useful in treating MS in humans. The findings also highlight the importance of doing proper controls, because in this case the most interesting findings came from a group of animals receiving sunscreen alone, as controls for mice exposed to UV light and receiving sunscreen. No one expected sunscreen to have an effect on its own.

You can read the abstract of the research paper here. Unfortunately, you'll need access to the Proceedings of the National Academy of Sciences journal in your library to read the entire text. Or you can read a news article about the paper in The Economist.

Washington State's New "E-DUI" Distracted Driving Law

The State of Washington has had enough of distracted driving.

Under a new law that takes effect today, using a hand-held electronic device of any kind while operating a motor vehicle is a primary driving offense, meaning that the police may stop you for that offense alone. The law goes beyond prohibiting texting or talking on a handheld phone; it prohibits checking Facebook, reading texts and e-mails, and even just holding an electronic device while operating a motor vehicle, even while stopped at a stoplight. Violators will be issued a distracted-driving citation and fined $136 for the first offense. The violation will go on the driver's record and their insurance company will be notified, which might cause their insurance rates to go up. A second offense will lead to a larger fine.

Drivers can still talk on their hands-free phone, and they can use a hand-held phone to contact emergency services. And they are still allowed to employ the "minimal use of a finger" to change an electronic device's function (preferably to turn it OFF).

The new law makes all forms of distracted driving secondary offenses, meaning that motorists who are stopped for a primary offense (such as running a red light) can be fined another $99 for "any activity not related to the actual operation of a motor vehicle". That might include reading a book or newspaper, eating, drinking, applying makeup, or reaching for something on the floor while driving.

So what are you supposed to do while driving? Concentrate. On driving.

Experience Matters When It Comes to Driving

According to a study by the AAA Foundation for Traffic Safety, people become better drivers with age and experience, at least until old age sets in. For the same number of miles driven, rates of both crashes and deaths are relatively high among 16-17-yr-olds, decline steadily with age until 70, and then rise again.

None of this should be a surprise; new drivers lack the experience that comes with a long history of driving, and older drivers are beginning to lose their sensory acuity and reaction times. But the numbers are startling. For the same number of miles driven, 16-17-yr-old drivers are about 6 times as likely to crash (and to die in a crash) as the safest group of drivers, 60-69-yr-olds. So, too, are drivers over the age of 80.

Traffic safety experts have been aware of these facts for some time. That is why some states now limit the conditions under which younger drivers can drive (forbidding driving with other teenagers or driving at night, for example). Most states require older drivers to have a vision test to renew their licenses, and a few states require older drivers to take a written test or a road test as well. All this seems to be working, as the number of people killed in automobile crashes declined more than 25% between 1995 and 2010.

Incidentally, teen crashes and deaths go up about 15% in the summer, when teens are out of school and may be driving more than during the school year.

Compounding Pharmacy Executive Goes to Prison

The co-founder and head pharmacist of a now-bankrupt New England compounding pharmacy has finally been sentenced to prison. Under his direction the pharmacy failed to ensure the sterility and safety of its products, which ultimately killed 76 people (see this blog, Jan 2, 2015).

Although the executive's conviction and sentencing finally closes this unfortunate case, not everyone is satisfied with the verdict. The executive was cleared of charges of second-degree murder that might have resulted in a 35-year sentence. Instead, he was convicted of racketeering and fraud, for which he will spend only 9 years in prison.

A Second Drug is Approved to Treat ALS

The FDA has approved a second drug to treat amyotropic lateral sclerosis (ALS), also known as Lou Gehrig's disease. ALS is a progressive, debilitating, neurodegenerative disease that is usually fatal within 3-5 years. The new drug, called Radicava, slows the progression of the disease somewhat after six months of use; apparently that was enough for the FDA to approve the drug.

Before you get too excited, though, here are some facts about the new drug; 1) It must be given intravenously. That means that the patient must have a permanent IV port installed, which poses an infection risk. 2) The drug is only partially effective. Patients using the drug won't feel better; their health will just decline less rapidly. 3) It's expensive - $146,000 a year, according to its manufacturer.

The first drug to treat ALS, called riluzole, costs only about a tenth as much as Radicava.  Riluzole extends the life of ALS patients by 2 or 3 months; whether Radicava does the same isn't yet known.

If you had a fatal disease and were expected to live only 5 more years, would you pay $146,000 per year to slow the rate of disease progression just a little, or to live an extra 2-3 months? That's a judgment call only you can make. But your insurance company might balk at the price of this drug, considering its minimal effectiveness.

A Texting-While-Driving Accident Claims 13 Lives

Here we go again. A lot has been written about the dangers of texting while driving, yet many of us just can't seem to resist the temptation. The death toll continues to climb.

Consider the most recent incident. Local sheriff's offices received several calls that a pickup truck was driving erratically on a two-lane highway west of San Antonio, Texas. One of the callers even recorded video footage of the truck. Although police responded to the calls quickly, they reached the pickup only after it had plowed head-on into a church van, killing 13 people. According to a Fox News report, the 20-year-old driver of the pickup truck (who survived the crash) said "I'm sorry, I'm sorry, I was texting."

Thirteen deaths is a lot to feel sorry for, especially when they could have been avoided so easily. And a lifetime of remorse is only part of what this young man will have to endure; what's the law going to do to him? I do truly feel sorry for him, because I'm sure he had no intention of harming anyone.

What's it going to take to get you to put down your phone while driving?

Microdosing With LSD to Treat Depression?

There seems to be a lot interest these days in the possible use of hallucinogens for medical purposes. Last month I reported that in a clinical study, the hallucinogen found in "magic mushrooms" seemed to reduce the symptoms of depression and anxiety, specifically in cancer patients (see this blog, Jan. 9, 2017).  Now it appears that very small doses of another potent hallucinogen, lysergic acid (LSD), are being used by some people as a self-cure for more common forms of anxiety and depression.

It's called "microdosing".  The trend started with the publication of the 2011 book, The Psychedelic Explorer's guide: Safe, Therapeutic, and Sacred Journeys.  More recently, microdosing moved into the mainstream, more or less, with the publication of one woman's memoir, entitled A Really Good Day: How Microdosing Made a Mega Difference in My Mood, My Marriage, and My Life.

Wow, who wouldn't want a better mood, marriage, and life!?  To be fair, microdosing with halucinogens might actually have some positive effects.  But skeptics such as Richard Friedman would say that it's not a good idea to self-treat depression with psychogenic drugs just because someone wrote a self-help book about it.  Microdosing with hallucinogens is untested; it's not even in the preliminary clinical research stage.  Anyone trying it is taking unknown risks.

An Experimental Alzheimer's Drug Fails in a Clinical Trial

A potentially promising drug for the prevention of Alzheimer's disease has failed in a key clinical trial, according to Eli Lilly, the drug's developer. In a study involving more than 2,000 patients with early signs of dementia, the drug, known as solanezumab, failed to prevent the advancement of the cognitive impairment that accompanies dementia and that leads ultimately to Alzheimer's disease.

The results were disappointing, but not all that surprising. So far, no drug has been able to prevent the progression of dementia once it has started. Researchers are reluctantly coming to the conclusion that by the time dementia first becomes apparent, it is already too late to affect the future course of Alzheimer's disease.

Whether solanezumab could prevent the development of dementia if it were given before the first signs of dementia is not known. Sadly, it may never be known. That's because it would be prohibitively expensive to conduct a drug study involving tens of thousands of seemingly normal people, in the hope of observing an effect on the few people among them that will ultimately develop dementia.

So we're at an impasse at the moment. There are several drugs on the market that can reduce (at least temporarily) the symptoms of the dementia associated with Alzheimer's disease, but there are no drugs that can prevent the slow progression of the disease. There aren't even any on the horizon, apparently. Don't expect progress any time soon.

Does Prevagen Improve Memory?

You've seen the commercials for Prevagen, right? It's a dietary supplement containing a protein derived from jellyfish that (according to the manufacturer) "improves memory and reduces common cognitive problems associated with aging." Trouble is, those claims are bogus, according to both the Federal Trade Commission (FTC) and the New York Attorney General's office. This week the two agencies sued Prevagen's manufacturer, Quincy Bioscience for making false and misleading claims, most notably the claim that Prevagen has been "clinically proven" to work. According to the complaint, the experiments undertaken to prove Prevagen's effectiveness were scientifically invalid because they did not show that Prevagen worked any better than a placebo.

No big deal, you say? Well, if you give people memory tests over time, you'd expect some improvement just from test familiarity alone. That's why it's so important to compare the experimental (Prevagen) group to a control group (a group given a fake pill, or placebo), using valid statistical methods.

Prevagen can cost upwards of $60 for a 30-pill bottle. According to the FTC, about $165 million a year is spent on the supplement. That's a lot for an untested cure.

Quincy Bioscience has responded to the lawsuit with a statement that says, in part, that Prevagen hasn't been shown to harm anyone (which is beside the point), and that "hundreds of thousands" of people tell them that Prevagen works and improves their lives. The latter statement, of course, is unscientific and unverifiable.

A Hallucinogen Reduces Anxiety and Depression in Cancer Patients

Two well designed studies published simultaneously by research teams at New York University and Johns Hopkins University have both come to the same conclusion: that a single dose of a powerful hallucinogen reduces anxiety and depression in patients with life-threatening cancers. The hallucinogen, known as psilocybin, is the active ingredient in "magic mushrooms". Nearly 80% of the patients reported feeling better following their hallucinogenic experience. The most striking finding was that they felt better for a long time - more than six months, in fact.

The federal government doesn't allow the use of public funds for research into the possible therapeutic uses of criminalized drugs. However, the research itself isn't banned. The present studies were funded largely from private sources after careful review by university research panels and federal regulators.

No one is suggesting that psilocybin should be made widely available to cancer patients. Nor is anyone suggesting (yet) that psilocybin should be used to treat general anxiety and depression. Still, the very long-lasting effect on anxiety and depression of a single hallucinogenic dose of psilocybin is intriguing. We need to know more about how this hallucinogen works. What if, for example a closely related drug could be found that reduces anxiety and depression, without the initial hallucinogenic effect?  Now, that would be a blockbuster drug.