You want to be filled with emotion?

By George Simpson

Let’s say your daughter has a progressive, degenerative, neurological disease — like mine does. That means today is the best day of her young life because tomorrow she will only get worse. Eventually, she may not be able to walk or be so wobbly that she chooses to have her leg amputated from the knee down to regain stability (it happens more often than you think). Her hands may become so weak that she won’t be able to use her phone, cook, or even scratch her cat. It probably won’t kill her (although it has killed others) but slowly take away many of the pleasures of life as her muscles degenerate and her independence ebbs away.

Here is what her hand can become according to French sculptor Auguste Rodin.

Her condition — Charcot-Marie-Tooth disease — has been around for hundreds of years (if not longer) and so far, nobody has found a cure. Or even a treatment to lessen the pain and suffering CMT causes. But a bunch of us are trying.

I volunteer at the CMT Research Foundation which invests in smart research projects at universities, biotechs, and pharma that have potential treatments or a cure for CMT in the works. We don’t just wait for the phone to ring; we go out and find new science and bring CMT into their labs. We did that with one biotech — shifting their whole mission towards CMT — and they will be in human trials next year!

A study published in 2020 in the JAMA Network (run by the American Medical Association) notes that “…after accounting for the costs of failed trials, the median capitalized research and development investment to bring a new drug to market was estimated at $985 million.” So, a billion bucks.

To be clear, we are not writing checks for millions of dollars to our research partners. We get them going (or farther along) and they must figure where the $ billion will come from. Our projects are designed to provide the evidence needed to attract the investors that it will take to fund human trials. All of our investments have some sort of ROI so that if one of the treatments we support gets to market, we will get back money to reinvest in yet more shots on goal. Remember, in the 135 or so years since CMT was named, nobody has put one in the net — yet.

Where does all that money go? Here are some examples:

Studies designed to show that the treatment is interacting with its theoretical target, cost about $500,000. Also, at 500k, a study in which multiple nonhuman primates are each given a single dose — but at different strengths — to find the optimal dose. Then you have to do it again, but dosing the animals over time (rather than just given a single dose) and that runs $1.2 million.

As the late Everett Dirksen once famously said, “A billion here, a billion there, and pretty soon you’re talking real money.”

It breaks a father’s heart to read about:

Axiom Space, a Texas startup marketing a 10-day trip to the International Space Station for $55 million a seat. Or Blue Origin auctioning off a seat on its maiden flight for $28 million. Or for the destitute out there, Virgin Galactic selling a 90-minute ride to suborbital space for $450,000 per seat.

I am not against science or space exploration, but this is just glorified joyriding. Buying bragging rights so you can say stuff like “To see the blue color whip by you, and now you’re staring into blackness…I’m so filled with emotion…”

You want to be filled with emotion? Try sitting down with your daughter and saying, “It’s over. Your body is safe. Your life can be like everyone else’s.”

And she gets to tell the other 3 million folks with CMT.