I came across the first while looking through lists of new medical patents. The Mayo Foundation for Medical Education and Research has been awarded rights to a stem cell-screening system. Specifically, the patent addresses a means of isolating substances that inhibit PAPP-A, a plasma protein. The reason for the search is that animals lacking PAPP-A have life spans up to a third longer than normal.

The patent is interesting for a couple of reasons. The most obvious is the prospect that a compound might extend human life spans significantly. It also, however, highlights the growing value of stem cells for scientific uses. A lot of money is going to be made selling stem cells before they deliver breakthrough therapies to the market. Then the dam will truly break. If PAPP-A inhibitors that lack major side effects can be found, life expectancies may increase by 20 years overnight. Even if such a drug were held by a Big Pharma company with diverse product lines, the profits would likely be transformational.

As the wealthiest generation in history ages, the demand for true longevity therapies will increase dramatically. As of now, however, the FDA doesn’t even recognize aging as a treatable problem. I suppose this is natural, since the ability to extend life spans therapeutically has become a reality only in the last few years. Still, the FDA needs to change its institutional prejudice against life extension therapies.

This prejudice is obvious in Glaxo’s efforts to bring its resveratrol compounds to market. Animal tests have shown significant life-extending abilities in resveratrol. Glaxo was convinced to the tune of $720 million, the price it paid for basic patents. Because slowing the aging process is not viewed as a legitimate therapeutic activity, approval for resveratrol as a diabetes treatment is being sought in the U.S. The company acknowledges tacitly, however, that the real market will be “off label” life extension.

The same issues are involved with myostatin inhibitors, the second of today’s breakthroughs. Myostatins are your body’s mechanism for preventing the growth of too much muscle. In the past, when malnutrition — instead of obesity — was the problem, they served a valuable function. When you put on muscle mass, your caloric needs go up. The ability to store fat is reduced, which increases the risk of starvation.

Myostatin inhibitors allow the body to build muscle easily. In animals, myostatin inhibitors increase the production of lean meat. The rare human with reduced myostatin activity has more muscle mass and strength. They also have very little body fat.

Obviously, a lot of people would like to bring myostatin inhibitors to market. The U.S. military is testing them now as a way to aid and accelerate the healing of wounded soldiers. Nobody is talking about it publicly, but they would also produce soldiers (and athletes) with significantly increased strength, speed, agility and endurance. Naturally, opposition to these drugs is growing.

This is what already happened with anabolic steroids. When Congress was in the process of lumping steroids in the same legal basket as heroin and cocaine, nearly all the experts protested, including, incidentally, the American Medial Association and the National Institute on Drug Abuse. AMA analysts forcefully argued that steroids could be an important tool to fight the symptoms of aging.

Almost 20 years later, I still talk to researchers who are angry over the Anabolic Steroid Control Act of 1990. The reason is that the benefits of increased muscle mass for older people are really quite remarkable. Lack of muscle strength leads to weakened bones and joints, which, in turn, work to make simple falls the leading cause of injury death in people 65 and older, according to the National Safety Council. Experts warned — accurately — during the hearings that making steroids Schedule III drugs would drastically reduce their medical use.

Beyond the mechanical benefits that come from increased strength and flexibility, there are also important physiological benefits. Many of these come from the decreased body fat content that is typical of people with more lean muscle mass.

The reason that Congress ignored the experts, by the way, is sports. Ben Johnson had just been stripped of his Seoul Olympics medals, and Congress seemed to think that it was more important to prevent “juicing” than to facilitate the treatment of age-related muscle loss and wasting.

Myostatin inhibitors could actually be more effective at building muscle than steroids. They would also be more useful. For one, steroids pose specific “masculinizing” problems for women that myostatin inhibitors do not. Even for men, they seem to have fewer side effects. Clearly, the sports industry is going to lobby to keep them off the market.

Incidentally, I watched Antonio Margarito beat the heck out of a far superior boxer, Miguel Cotto, on pay-per-view this Saturday. I don’t, however, put the so-called integrity of sports above the well-being of the aging and aged.

The company that holds the patents for myostatin inhibitors is MetaMorphix Inc. It’s privately owned, but somebody is going to sell these inhibitors eventually. I believe strongly that baby boomers, when they understand what is at stake, will prevent these drugs from being treated like anabolic steroids. I also believe that myostatin inhibitors are going to break pharmaceutical profit records. I’ll keep you in the loop as this story develops.

Until next time, here’s to transformational profits.

Patrick Cox
August 1, 2008

Profit from Myostatin Inhibitors was originally featured in the Penny Sleuth.