Medical experts push for precision medicine

Medical experts and researchers are urging for the use of precision medicine to help identify genetic causes of disease and to tailor therapies for individual patients.

Using precision medicine, physicians could pinpoint the biomarkers of a disease in a patient and apply treatments that work best based on that person’s genetic profile, instead of relying on a traditional “one-size-fits-all” approach, researchers said Tuesday in panel discussion.

“In an innovative way, it’s going to be able to provide more efficient, more focused care to patients and basically help revolutionize the practice of medicine in the future,” Joe Panetta, president and CEO of Biocom, an association representing the California life science industry, said during the discussion at the Longworth House Office Building.

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Mr. Panetta said precision medicine can help eliminate the inefficiency of prescribing drugs and treatments on a trial-and-error basis.

Precision medicine already has been applied in cancer diagnosis and treatment.

Leo Mascarenhas, deputy director of the Children’s Center for Cancer and Blood Diseases at the Children’s Hospital Los Angeles, shared a story about how precision medicine helped a cancer patient named Michelle Lowry.

Michelle was 2 years old when a pea-sized lump appeared on her neck. The tumor moved toward her spine and compressed her windpipe, obstructing her breathing, Dr. Mascarenhas said.

The doctors suspected she had sarcoma, a type of cancer that can develop in various areas of the body. Michelle could not receive traditional cancer treatments such as surgery, chemotherapy or radiation.

A test showed that her tumor was caused by a gene known as NTRK that had fused with another unrelated gene.

After the test, Michelle was enrolled in a clinical trial in which she was able to try an experimental drug called larotrectinib that targeted the genetic characteristics of her tumor. Her tumor shrank within days, and Michelle was discharged from the hospital a few weeks later.

Thanks to precision medicine, Dr. Mascarenhas said his patient was able to recover from a life-threatening cancer.

“We are very grateful to actually have this technology, which we can apply to our patients,” the oncologist said.

That technology includes next generation gene sequencing, which can be effective in finding mutations that cause cancer and guide precision medicine to target mutations, said Jeffrey Smith, global leader for Next-Generation Sequencing Precision Medicine Initiatives for Thermo Fisher Scientific, a biotechnology product development company.

With next generation sequencing, about 4,000 gene variations can be identified in a single test, which then can be delivered to a physician to help tailor a treatment. Much of current screening depends on sequential, single-gene testing, which consumes time and body tissues, said Mr. Smith.

While precision medicine may be more effective for treatment, it is not necessarily a viable, accessible option for everyone.

Joe Germino, vice president of U.S. Medical Affairs-Oncology for Bayer, a drug and life sciences company, noted that while health insurance might pay for a targeted treatment, it might not cover genetic screening, which he said can cost between $3,000 to $5,000.

He said he hopes doctors, payers, insurance companies, regulators and patients eventually reach a consensus about precision medicine and its value in treating diseases.

“I hope at some point that testing of the tumor becomes as common as sending off a slice of the tumor for pathology,” Mr. Germino said. “Because although right now, we have imperfect knowledge — and we will never have perfect knowledge in all probability — we’ll have better knowledge and we’ll have a better understanding of what drives the cancer and how to best treat it.”