Man Survives Rare Cancer Thanks to New 'Targeted' Therapy

"Genomic" cancer treatment uses patient's DNA to fight cancer.

ByABC News
September 16, 2014, 10:00 AM

— -- James “Rocky” Lagno was so sick that doctors only gave him about a year to live. Having been diagnosed with late-stage lung cancer, even aggressive chemotherapy and radiation didn’t prevent the New Hampshire native’s tumors from growing larger.

To top it off, he was also diagnosed with thyroid cancer and then, several months later, an MRI revealed a dozen brain lesions.

“The oncologist told me I should probably think about getting my bucket list together” Lagno, 53, recalled of the 2011 conversation he had with his doctor.

Fortunately for Lagno, his wife, Geralynn, lobbied for a biopsy that uncovered a rare genetic mutation linked to lung cancer. Once discovered, Lagno was entered into a clinical trial to test out a relatively new approach to cancer treatment known as molecular targeted therapy.

Traditional cancer drugs are indiscriminant, attacking not just cancer cells but every living cell in the body. Molecular targeting agents like the one Lagno received - which are no longer experimental and are being used with increasing frequency - are designed to target specific cancer mutations, explained Washington University in St. Louis cancer researcher, Elaine Mardis.

“Many cancers revolve around novel proteins that are highly active and constantly stimulated so that the growth of cancer is stimulated,” Mardis said. “These new targeted drugs seek out these novel proteins and shut them down.”

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In Lagno’s case, the therapy seems to have worked. The real estate agent has been taking two pills of the drug Ceritinib daily for the past three years and, while his tumor isn’t entirely gone, it hasn’t grown or spread either, he said.

Lagno’s remarkable turnaround is no longer unique, researcher Mardis said. Ceritinib has since been approved by the Food and Drug Administration earlier this year along with four other similar drugs, she added. It’s now widely used to treat lung cancer patients in hospitals all across the country.

“Many patients will now get this sort of drug in the first round of treatment even before chemo or radiation,” Mardis said.

Because gene sequencing has become so much simpler and commonplace, more and more tumor varieties are identified every year, Mardis said. This allows pharmaceutical companies to create drugs with more precision.

As a result, the FDA has trimmed back many of the longer, more expensive trials so drugs are reaching the public faster than ever before, she said.

“For some type of cancers, you’re seeing these new therapies replacing traditional cancer treatment like chemotherapy and radiation,” Mardis said, adding that the new drugs are more effective in many cases than older treatments and carry far fewer side effects.

Targeted molecular therapy (also known as genomic medicine) has led to significant breakthroughs for many cancers. As Mardis pointed out, some kinds of lung cancers and melanomas that would have been considered deadly less than a decade ago now have a better than 75 percent response rate with this treatment.

In the near future, perhaps five years, medicine may be truly personalized, Mardis said.

“There may be a time when you will be given a drug or a combination of drugs designed to treat your illness based on your unique genetics,” she said.

Even though personalized medicine isn’t quite a reality, Lagno said he thought this was a hopeful moment for anyone who is a cancer patient like him.