When I was first diagnosed with dermatomyositis, I felt hopeless about the prospects of humankind ever developing a drug specifically for my orphan disease. What profit-seeking entity would throw money at a disease so rare it makes lupus look like a pandemic? What congressional representative could justify spending taxes researching an autoimmune condition that may not even exist in the district they represent?

Could I count on the benevolence of strangers? Should I expect self-interested organisms hellbent on survival to be interested in preserving, or at least, improving, a genetically malformed member of their species?

In fall 2019, I received a call from Mayo Clinic in Scottsdale, Arizona. A dermatologist there wanted to enroll me in a phase 3 clinical trial for a drug developed for dermatomyositis and other rare inflammatory conditions.

I jumped at the opportunity. Any new drug had a strong chance of being an improvement over the azathioprine I had been taking.

Becoming a guinea pig

When the trial started, many asked if I was nervous about ingesting a substance not yet approved by the Food and Drug Administration. The answer was a resounding no.

For one thing, dermatomyositis is life-altering and irritating enough that I would prefer side effects from any drug–including the azathioprine I already take.

Azathioprine is classified by the U.S. Department of Health and Human Services as a carcinogen. According to the Mayo Clinic dermatologist, spending 35 years on the medicine I have been on, azathioprine, virtually guarantees I will end up with skin cancer before I turn 70. Other studies confirm risks of skin cancer and lymphoma. The sooner I can rid my body of azathioprine, the better.

Even then, as an anarchist, an agency stamp on a drug means little to me. Many of the FDA-approved autoimmune treatments carry risks of depression, insomnia, even death. A woman with arthritis I used to work with on rituximab was hospitalized after the medicine caused Guillain-Barre syndrome. And there is not one autoimmune treatment on the planet that does not put the patient at risk for humanity’s latest afflictions, COVID-19 and monkeypox.

Even if all pharmaceutical companies cared about was money, they have no incentive to kill or harm their customers. And even if a company were okay rushing drugs to market in search of short-term gains, a single nasty side effect from a single drug could ruin a billion-dollar corporation. Profits would be spent settling lawsuits. Few doctors or patients would ever trust the company again.

Finally, the drug I tested, lenabasum, had entered phase 3 clinical trials. By the time a drug reaches this late phase, it has already undergone years of safety and efficacy testing.

Schedule I substances

Lenabasum is ajulemic acid, synthetic cannabinoid designed to attack the severe inflammation and muscle degeneration associated with dermatomyositis. Yes, it is derived from cannabis, making it a Schedule I substance. In short, that means the federal government recognizes no acceptable medical use for it. Merely possessing the drug can land you 10 years in prison.

I have no idea how exceptions for this work, especially in the era of marijuana legalization, but the trial drug was definitely treated like contraband. The pharmacist had to hand deliver it to the patient, me, inside the study coordinator’s office. I was required to sign that I received and accepted the drug. The pharmacist had to affirm she gave it to me.

Lost in the insanity of this procedure was that the manufacturer had removed the pscyhoactive component of cannabis, tetrahydrocannabinol (THC). And even if they had not, I can buy recreational or medicinal cannabis containing ample THC at a strip-mall shop 3 miles from my house.

Perhaps even more hilariously, during the height of COVID-19, Mayo Clinic was shut down to any non-emergency use, and the pharmacist started shipping the scheduled substance to my house. Adhering to the strictest DEA definitions, this meant the pharmacist, the mailman, Mayo Clinic, and the U.S. Postal Service, were trafficking drugs.

Less guinea pig, more zoo exhibit

When I started the trial in February 2020, the violet-red Gottron papules adorning my knuckles were severely inflamed. They alone qualified me for the trial and turned me into a traveling zoo exhibit for medical students. Little in patient life underscores how different you are from everyone else like being introduced to a parade of twenty- and thirty-somethings for the first time as a textbook example.

Little in patient life underscores how different you are from everyone else like being introduced to a parade of twenty- and thirty-somethings for the first time as a textbook example.

To track adverse reactions, every visit, I filled out a 150-question survey, most of which were about itching and mental health. They also drew about six tubes of blood for every lab visit. Unlike labs ordered by me or my doctor, I was never able to see any of my results.

Apart from these procedures, the bimonthly study visits varied little from any other medical appointments. Admittedly, I looked forward to my visits. I enjoyed talking to someone who knew more about dermatomyositis than me for once.

Answering the $30,000-per-year question

Three months into the double-blind drug trial, the papules had all but disappeared. Hair loss slowed down. My scalp barely ever itched. I was even able to decrease my daily dose of azathioprine. I thought for sure I was on the lenabasum and that it had worked.

I was wrong.

In June 2021, the study coordinator phoned to inform me Corbus Pharmaceuticals, the drug manufacturer, canceled the trial. They had not seen the results needed to justify spending millions more to develop and market a drug that would cost patients or insurance companies tens of thousands of dollars per year.

The study coordinator also told me I had been on a placebo. Any improvements the dermatologist, my rheumatologist, and I observed had nothing to do with the lenabasum.

Though I was mildly disappointed, this was actually good news. Not only had my immune system backed off, but it did so while significantly decreasing my dose of azathioprine.

Further, despite lenabasum containing no THC, throughout the drug trial, I felt drained and unmotivated. Because the study overlapped the social withdrawal from worldwide quarantines and one-size-fits-all policies to prevent the spread of COVID-19, no one can say for sure if the lenabasum caused those feelings. That said, other patients reported similar side effects. Regardless, I was happy to rid my body and mind of anything that had contributed to 14 months of lethargy.

It would have been nice if lenabasum truly worked, but even if it had, insurance companies would have been reluctant to cover such an expensive drug.

Would it have been nice if the new drug worked? Yes. But even if it had, I am not certain it stood a future. Insurance companies would have used any excuse to avoid paying the $30,000 per year lenabasum would no doubt fetch following FDA approval. That would include remaining on the azathioprine that had worked all along and could be bought at prices even developing nations could afford. Cancer risks be damned.