Raili Kerppola had a thriving career in the pharmaceutical industry when she was diagnosed with stage four adrenal cancer in 2011.
While seeking treatment at the University of Michigan, she began working with an endocrinologist named Dr. Gary Hammer and her husband, a professor of biological chemistry at UM, on researching and discovering new treatments for the rare disease.
Kerppola died this June. But her legacy lives on through a VC-backed company called Atterocor, for which she is credited as a co-founder. The University of Michigan spinoff is conducting a Phase 1 clinical study of a drug that it hopes could prolong the lives of patients with adrenocortical carcinoma, and cause fewer toxic side effects, than the current treatment option.
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It’s a rare form of cancer that carries a short life expectancy after diagnosis. That’s because it’s usually caught late-stage, when a patient presents with symptoms like unexplainable weight gain, hypertension, abdominal pain or other metabolic complications caused by excessive production of hormones by the adrenal glands.
Treatment starts with surgery, if the cancer is caught early enough. Then, doctors usually try to extend life with a cocktail of chemotherapy drugs including mitotane, a drug that reduces the amount of hormone produced by the adrenal cortex.
The problem? Mitotane, a derivative of the pesticide DDT, is terribly toxic. And its efficacy is questionable, too. Atterocor co-founder Julia Owens said the FDA approved the drug decades ago, before the agency required drugmakers to demonstrate that it works before approval. She said that although it likely does have some benefits for patients, they usually have to take multiple doses a day for three to four months before their mitotane levels reach a therapeutic range. Meanwhile, they’re usually also taking chemotherapy drugs.
“One endocrinologist recently told me that the way you can tell if a patient is getting close to efficacious mitotane levels is when they can’t walk down the hallway without assistance,” she said.
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Atterocor says its compound is highly specific to the cells of the adrenal cortex, which is where the cancer occurs. “We haven’t done human trials yet so we can’t make any claims, but based on toxicity studies, our effect seems much more specific, much more rapid, with a better (pharmacokinetic) profile,” she explained.
It’s received orphan drug designation in the U.S. and in Europe. The company started a Phase 1 trial over the summer at MD Anderson Cancer Center and the University of Michigan Comprehensive Cancer Center. Owens said the company should have some data on tumor responses and steroid markers in the next six to 12 months.
With six employees, Atterocor is a small company, but it’s attracted some big-name investors. Last year the Ann Arbor startup reined in a $16 million Series A from Frazier Healthcare, Osage University Partners and 5AM Ventures.
Owens said that, for a company developing an orphan drug, that capital makes all the difference. “We can move this compound forward ourselves without being at the mercy of a big pharma company (partner),” she said. “We haven’t been around for two years yet and have taken this idea from the University of Michigan and run our toxicology studies, synthesize the drug and now are running the study. If we see encouraging results, we could go into a Phase 2 study and seek approval ourselves.”
You have to think that’s something Raili Kerppola would be proud of.