Tackling cancer treatment reality in "rare” America: An Op-Ed by SHEPHERD Therapeutics CEO David Hysong

Have you ever held the hand of a child dying of cancer? I begin my week volunteering at Vanderbilt Children's Hospital in the Inpatient Acute Care Wing. I spend a few saddening, yet inspiring, humbling hours holding infants, comforting toddlers, and trying to brighten a severely ill child's day. Some have rare disorders or severe injuries and developmental delays; some are 7 days old, some 7 years; and some are dying of various rare forms of cancer that exclusively or predominantly affect children.

For the more than 50 distinct forms of cancer that primarily affect children, currently only 1 has an FDA-approved targeted therapeutic. As a recent Newsweek feature noted, "Children's Cancer is Unprofitable and Ignored." Once you've seen a child dying of cancer, you can't unsee. And once you've been diagnosed as a formerly robust and healthy 27-year-old, you can't ever go back to normal. And once you've been told that modern medicines for you and that child were deemed too expensive to make - that you have both been left to die - a fire is lit inside of you that never goes out. That became my life. I am a volunteer and I am a patient. I am also the Chief Executive Officer of a pharmaceutical company focused exclusively on rare forms of cancer for which there are no modern medicines. It's called SHEPHERD Therapeutics - and exists to treat the most devastating cancers - those deemed too rare, too costly, too hopeless. But not for us.

Based on the strictest definition of "rare disease" - affecting 6 out of every 100,00 people - 428,712 Americans were diagnosed with one of at least 340 rare cancers in 2017. And yet the 91 FDA approved cancer therapeutics treat only a handful of diseases - 20 treat leukemia, 17 treat lymphoma, 16 lung cancer, 14 breast. The vast majority of cancers have nothing.

Shortly after my own diagnosis, I met a pair of blonde-haired, blue-eyed little girls. They were 6 years old. And they had the same form of "untreatable" cancer - Adenoid Cystic Carcinoma - that I did. I decided then that I wanted to be the one to give them a fighting chance.

I consider myself a steward - one given an opportunity to turn my own suffering into a place to stand and a chance to save the lives of the patients I interact with every Monday. We do things differently at SHEPHERD. I'm a patient, not a scientist or businessman. Most of my C-level team are women - unheard of in biotech. Instead of the biggest annual biotech conference where deals are done in terms of millions made, not patients saved, all of us at SHEPHERD will be volunteering at St. Jude's Hospital - to remind us of why we do what we do.

I've been humbled to watch a diverse and brilliant team of people give up jobs, turn down more lucrative offers, and come together in a unique way to tackle a devastating epidemic's more devastating treatment reality. At SHEPHERD we try to remind ourselves daily that patients are dying - and that it is our calling to fight for those who cannot fight for themselves. That the Neglected May LIVE.