Ten years ago, in 2002, I read an article in WIRED magazine about an experimental drug called Melanotan. Early test results indicated that the drug could make a pale person tan, without sun exposure. Side effects included reduce appetite and weight loss, and a high frequency of spontaneous erections.
Thin, tan, and horny … WIRED dubbed Melanotan “the Barbie drug.”
Within a week of reading the article, I hunted down the Australian company developing the drug and purchased a thousand shares at eight cents a share. Within a year the stock price had skyrocketed to tenfold my purchase price. Press releases from Epitan (the Australian pharma company developing the drug) continued to say that Melanotan would be ready for market “within a few years.”
Ten years later, Epitan has changed its name to Clinuvel, and has a new CEO. The company is on track to get European approval for afamelanotide (the technical name for Melanotan, now marketed as SCENESSE) to prevent erythropoietic protoporphyria (EPP) in adults. EPP is a rare disease which causes “absolute intolerance of patient’s skin to light.” (I think the kids in that Nicole Kidman movie The Others had EPP.)
After a reverse split, the stock price has settled at around twice my purchase price (I still own most of it).
Watching the molasses-like progress of afamelanotide to market has taught me something about the pharmaceutical industry. It’s slow and risky. While afamelanotide seems poised for moderate success (both medical and commercial), most drugs fail after a few years of development. Maybe they work in test tubes, but not in animals. Maybe they work in mice, but not in people. Maybe early test results that showed that the drug “worked” were due to random data fluctuations. Maybe the drug does what it’s supposed to do, but causes toxicity or intolerable side effects.
I got lucky with my investment in Epitan (now Clinuvel). So far their flagship product has proved to be at least moderately effective, not obviously toxic, and helpful to at least some people. Afamelanotide won’t be a “blockbuster” drug (one reason is that it’s only injectable — there’s no effective oral version), but it’s not a total failure either. Most new drugs are.
The last few weeks have given us some amazing headlines regarding potential medical advances. I’d like to review a few of them, but I’ll do it with my Melanotan goggles on. Add at least a decade to any estimates regarding when the drug or treatment will be available for widespread use. Consider that human trials will probably reveal toxicity of some sort, undesirable side effects, and/or lack of effectiveness. Even if a drug proves to be effective and non-toxic, getting FDA approval can be derailed for any number of reasons.
Skepticism firmly in place, prepare to be amazed. Basic medical research appears to be paying off in a number of areas.
CD47 Antibody Cancer “Cure”
I put cure in quotes because we’ve seen cancer cures come and go many times. There are many types of cancer and it’s unlikely that any single treatment will successfully treat all types of cancer. However the CD47 antibody has been shown to shrink “human breast, ovary, colon, bladder, brain, liver, and prostate tumors that have been transplanted into mice.”
CD47 is a protein that signals the human (and apparently mouse) immune system not to eat healthy cells (via autophagic processes, explained technically in this paper). Cancer cells can use CD47 to trick the immune system into not destroying them. The CD47 antibody blocks the action of CD47, and stimulates the immune system to destroy the cancer cells.
Does blocking CD47 also trick the immune system into destroying more healthy cells? According to this article in the SF Chronicle, it does, but only older red blood cells, resulting in a side effect of mild anemia.
Dr. Irving Weissman of Stanford University is developing the CD47 antibody treatment, and hopes to move forward to Phase I human trials soon. The research is being funded by a grant from the California Institute for Regenerative Medicine, an organization created by Proposition 71 (stem-cell research).
Given that most current cancer treatments are highly toxic (chemotherapy, radiation, etc.), CD47 is promising direction for continued research. It’s too soon to say if the treatment will be effective in humans, but it’s worth keeping an eye on.
In the meantime, we can reduce cancer risk by:
- not smoking
- drinking less alcohol (though red wine may be protective against some cancers)
- staying lean/maintaining insulin sensitivity (for most people reducing grain and sugar intake is the easiest to lose fat and become more insulin sensitive)
- reducing intake of foods cooked at high heat (grilled meats, fried foods, baked foods)
- eating broad beans
- consuming more cruciferous vegetables (cauliflower, broccoli, cabbage, kale, etc.).
DRACO (broad spectrum antiviral)
An MIT team including Dr. Todd Rider is developing a drug called DRACO (Double-stranded RNA Activated Caspase Oligomerizer) that selectively induces apoptosis, or cell suicide, in cells containing any viral dsRNA, rapidly killing infected cells without harming uninfected cells.
In other words, this drug cures all viruses. This would include deadly viruses like Ebola, smallpox, and HIV/AIDS, harmful viruses like flu, herpes, hepatitis, and HPV, and merely irritating viruses like CMV and the common cold.
Here’s an interview with Dr. Rider that gets into the details.
Modern medicine is largely divided into pre-antibiotic days (when a deep scratch or puncture might end up killing you) and post-antibiotic days (in which bacteria can still kill you, but you’re likely to survive an infection if you can tolerate the side-effects of powerful antibiotics). With DRACO, we might eventually see the same kind of line in relation to viruses.
This article in WIRED brings up a good point — we have co-evolved with many viruses, and we may be better off with them than without them. We now know that our bodies play host to billions of helpful bacteria, our biotic community. These helpful bacteria help digest food, synthesize vitamins, and protect us from pathogenic bacteria. Just as taking an antibiotic can destroy your biotic community (for up to a year in some cases), taking DRACO might end up destroying viruses that we need to keep us healthy.
Viral “communities” may support our health and protect us from other diseases (parasites, cancer, even diabetes). Read the article in WIRED linked above for some examples.
It would be nice to have a broad-spectrum anti-viral (it could usher in a new sexual revolution, like the Pill did, which could be fun). On the other hand, if DRACO works in humans, we should proceed ultra-cautiously until we learn more about human-viral symbiotic relationships.
REV-ERB for Circadian Control (cure obesity, sleep disorders, and jet lag)
A research team from the The Scripps Research Institute (Florida campus) has synthesized a pair of molecules that can modulate proteins called REV-ERBα and REV-ERBβ, which control the “biological clock” and influence metabolism and activity.
When mice were given the synthetic molecules, they became leaner, had higher resting metabolisms, lower blood sugar levels, and improved lipid profiles (lower cholesterol and triglycerides).
Furthermore, there were no side effects. “As it turns out, the effect of that modulation is surprisingly positive — everything has been beneficial so far,” said Thomas Burris, who led the study.
Here’s the original paper.This is really interesting. I’d love to be able to take a pill and instantly adjust to a new timezone, which control of the REV-ERB proteins might facilitate. I’d also like to avoid going into a pre-diabetic state if I lose a few hours sleep for multiple nights (this has already happened to you).
Part II, and a Parting Thought
I’ve run out of time, but I have a few more of these advances to share (including a vaccine that can prevent heart attacks). I’ll eventually revisit this topic and write Part II.
I suppose there is a potential objection to research like this, on the grounds of living “naturally.” If we all ate as our paleolithic ancestors ate, and avoided artificial light, and didn’t smoke or drink whiskey or sleep around, then we wouldn’t need drugs that destroyed or viruses or cured cancer or fixed our circadian rhythms. Right?
No — we’d still need them. Some people lose the genetic lottery and will get cancer no matter how well they eat or how strictly they avoid carcinogens. Some people don’t want to eat paleo because they can’t stand the thought of animals being raised/held captive/slaughtered for the purpose of being eaten by humans.
It’s impossible to completely optimize your life for health (physical and mental), if you partake of modern civilization. Even if you are disciplined and vigilant, noise, light, sustained stress, processed food, too much sitting, and glowing screens will worm their way into your life. We should consider medical/pharmaceutical/nutritional advances (including vaccination, supplements, drugs, medical procedures, etc.) with measured skepticism (learn the risks) but also without moral judgement (embrace the benefits). What enhances quality of life? What gives humans more flexibility and power? (And by power I mean non-coercive, non-zero sum, creative/progressive power.)