Chemotherapy is no longer the gold standard for treating a form of leukemia as a result of a national clinical study that was so successful, it was halted early.
That's good news from here on for patients 70 and younger who are treated for chronic lymphocytic leukemia (CLL), said Dr. Bret Friday, an oncologist-researcher for Essentia Health in Duluth.
"Ten years ago, the majority of cancer patients, whether it's CLL or other cancers, the primary treatment was chemotherapy," he said. "And the simple way I think of looking at chemotherapy is, these are poisons that are a little more selective to cancer cells, but they damage other parts of the body as well."
A new generation of cancer medications, known as targeted therapies, focus on the offending cells and minimize harm elsewhere, Friday said. One such drug, called ibrutinib, already was being used in later stages of treatment for CLL, he said. The question: Would it, in conjunction with a drug called rituximab, be more effective than chemo as a first-line treatment while also having fewer residual effects?
That's what designers of the trial in which Essentia participated wanted to determine, Friday said. Although CLL is the most common form of leukemia in adults - close to 21,000 new diagnoses and about 4,500 deaths were expected in 2018, according to the National Cancer Institute - no single site would have enough patients to carry out a meaningful study. Essentia would have only three.
"We have 60 to 80 trials open, and we really make efforts to have trials open for patients with less common diseases or situations," he said. "And so many of the trials we open, we may only enroll one or two patients to those trials."
One of the Essentia patients was Jerry Maurer, now 66, of Carlton who was diagnosed with CLL in 2013.
CLL is one of the few cancers that doesn't necessarily require treatment, said Friday, who was not Maurer's oncologist.
In Maurer's case, it was not until August 2015 that his white blood cell counts reached a point where treatment was advised. By then, Essentia was participating in the trial, and Maurer agreed to be part of it.
"It was an easy call," said Maurer, who has a master's degree in behavioral rehabilitation and runs a consulting firm for adult and family foster homes. "I've always tried to look at what are the best options."
Friday sees participating in clinical trials as a win-win. Depending on where the patient is randomly assigned in the study, he either gets a cutting-edge treatment for his illness that wouldn't otherwise be available, or he gets the treatment that is currently the gold standard, he said.
Maurer would have been satisfied either way, he said. "But I crossed my fingers and hoped that I would be in Arm A (the ibrutinib)."
He got his wish.
"From there I have gone in every three months, had blood tests, have met with the clinical nurse, Deb Ronding, who does an outstanding job," Maurer said. "She really is quite strict about the protocol. I always tease her that she keeps me in line, and that's good."
Each time so far, his white blood cell count has been stable.
Similar results were being found throughout the trial, which involved a total of 529 patients, according to a Dec. 4 news release from the National Institutes of Health. Overall, the risk of disease progression was 65 percent less for patients in the ibrutinib group than in the chemo group, the researchers told The American Society of Hematology's annual meeting on Dec. 4.
The trial was only at its original midpoint, but enough evidence was in.
"They did the interim analysis and found that the results were so good that it was unethical, really, to continue the trial," Friday said.
How common is it for a trial to be halted because of good results?
"Less common than we'd like," Friday said, with a hint of a chuckle. "It's probably one in 20 studies."
It's more common for studies to be halted because they aren't working as hoped, Friday said.
Maurer's diagnosis came out of a routine physical and not because he had been feeling unwell, he said.
Nonetheless, he realized as the treatments started taking effect that he was feeling better than he'd felt before, and his energy level is good, he said. The only side effect he has noticed is occasional blood blisters on his hands or inside his mouth, and those have been decreasing.
That's very different from chemotherapy, which can compromise immune systems and place patients at risk for severe and even life-threatening infections, Friday said.
But Maurer did have a concern: Now that the trial has been suspended, will the cost of his three daily doses of ibrutinib still be covered?
"It's prohibitive if not," Maurer said. (It is still covered by the trial, an Essentia Health spokeswoman said.)
The website Drugs.com places the price of ibrutinib's brand name, Imbruvica, at $12,612 for 28 70 mg capsules, or about $450 per capsule. There is no generic version.
If the price seems astonishing to you, it does to Friday, too.
"I can't believe they charge that much," Friday said. "It's amazing that we allow that."
But he's also enthused about how ibrutinib and other targeted therapies are changing the face of cancer treatment. It's possible for those treatments to advance because Maurer and others agree to participate in clinical trials, Friday said.
"It's nice to be able to go back to him and say, 'Look, you're part of this study and because you're a part of this study, we've now changed our treatment,'" Friday explained.
"And this will help everybody else diagnosed with cancer."