Boettcher Investigator’s clinical trial is saving lives

09 Mar Boettcher Investigator’s clinical trial is saving lives

For 2011 Boettcher Investigator Dr. Robert Doebele, the satisfying part of medicine is researching an idea in his lab, and then seeing that idea come to fruition in treating his patients. And that’s exactly what is happening in the first-of-its-kind clinical trial that he is currently leading.

Dr. Doebele holds both an M.D. and a Ph.D., which allows him to spend part of his time in the lab and part with his patients, which is also what guided him to specialize in lung cancer. 

“I like the patient relationship. Some doctors like healthier patients, and while I don’t like having sick patients, I don’t mind it,” Dr. Doebele explained. “I get to see my patients often, from diagnosis through treatment.”

It’s that same patient interaction that helped Dr. Doebele to make a huge breakthrough in his lab.

Dr. Doebele researches oncogene targeted therapy, or precision medicine, where he identifies the dominant genes that are critical for developing cancer. When those genes are specifically targeted in treatment, doctors are able to shrink tumors.

“When I started working in lung cancer in 2005, the treatment for lung cancer was pretty much all the same,” Dr. Doebele said. “The treatment is now very different based on the genetic abnormalities present in each patient.”

Because of this genetic research, lung cancer now has identifiable mutations that make gene-targeted therapy possible.

But what happens when a patient has none of the identified mutations, is a non-smoker in her forties, and presents with stage-four lung cancer?

Around that same time, Dr. Doebele realized there must be other targetable genes that can become cancer-driving cells. Thus, his most-recent research was born.

His lab did not discover a new gene, but using modern techniques, they were able to identify that this specific gene was truly cancer-driving and, beyond that, they developed a method for targeting that gene in treatment. Working with Array Biopharma out of Boulder, Dr. Doebele helped develop a drug to test in these cancers.

Unfortunately, Dr. Doebele’s inspiring patient passed away, but thanks to the tissue that she donated, Dr. Doebele was able to directly advance his research, initiate a clinical trial, and literally save lives.

The first patient to participate in his clinical trial was much like Dr. Doebele’s patient who donated tissue. She was incredibly ill with widely metastatic sarcoma in her lungs, and even moved to Denver with her family for a month to participate in phase one of the study.

“I was nervous because this was the first test of my research to see if it actually does anything,” remembers Dr. Doebele. “You feel like you are laying your chips on the table, and it was my idea combined with a real patient.”

Week by week during the clinical trial, his new patient improved. Now, almost two years later, she is still taking the drug he developed and has no measurable tumor in her lungs. And she’s not alone. All of the trial patients have had tumor shrinkage and most have had a highly measurable response.

“This is not going to cure millions of cancer patients, but if we test patients and identify these mutations, it’s a very good start. It’s a bedside-to-bench-to-bedside approach,” said Dr. Doebele.

Two companies are now developing drugs to specifically treat what Dr. Doebele identified, and are awaiting FDA approval.

“It’s rare but satisfying when you get to come full-circle and see your research directly affect patients.” Dr. Doebele said.

Looking back, he credits his scientific success with both luck and preparedness, plus a boost from the Boettcher Foundation.

“The luck was finding a patient that had this mutation, but the Webb-Waring Biomedical research funding allowed me to prepare my lab to take on this project very quickly,” said Dr. Doebele.

This combination allowed Dr. Doebele to publish his findings in approximately a year, which meant getting this treatment to clinical trial, and ultimately, to patients sooner.

Since then, he has received more funding, including an R01 grant from the National Institute of Health.

Looking ahead, Dr. Doebele is very interested in bringing increasingly better drugs to patients, and plans to continue testing new drugs and new ideas with the ultimate goal of saving lives.