‘Precision Medicine’ Needs Rev Up as Genetic Diversity Grows

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By Jacquie Lee

Drugmakers are feverishly investing in precision medicine as Americans develop increasingly complicated genetics thanks in part to the rising birth rates of children with multi-ethnic backgrounds.

Precision medicine—sometimes referred to as personalized medicine—is an emerging approach for disease treatment and prevention that seeks to maximize effectiveness by taking into account individual differences in lifestyle, environment, and genes. Although relatively new, it is becoming an increasingly needed approach to treatment, doctors told Bloomberg Law.

Drug companies are heeding the industry’s call for more effective treatments and have poured money into research for personalized medicine, and they’re having some success. But they’re far from getting a wide range of exact, gene-specific treatments some of these multiracial patients might need. And as drug companies race against each other to create the next blockbuster treatment, the rate of multi-ethnic children will continue to grow.

“We are not a nation of white men,” biologist David Meyer said while noting the importance of increased diversity within clinical trials.

Fourteen percent of children born in the U.S. in 2015 were multiracial or multi-ethnic, which is three times the rate of such births in 1980, according to the most recent data from the Pew Research Center. That number is expected to keep growing, which adds “an entirely new dimension of genomics” to research and treatment, Meyer, also the chief executive officer of LA BioMed, a nonprofit scientific research organization, told Bloomberg Law.

Some companies have already gotten a jump on precision treatments. Amgen’s Repatha and Sanofi’s Praluent take into account genetic predispositions of patients who are difficult to treat for high cholesterol, Bob Kirby, the head pharmaceutical analyst at Fitch Ratings Inc., told Bloomberg Law. “The industry is moving forward,” Kirby said. Some cancer drugs are now matched to an individual’s specific genes, like Novartis’s Kymriah.

The Novartis drug was approved in August 2017 and trains the immune system to attack cancer cells. Eventually all drugmakers will move toward those types of highly-specialized treatments, Kirby said, but “it’s still relatively early in the process.”

“Advances in molecular biology and genomics have opened up a panoply of potential drug targets and potential markers that can help identify patients who may benefit from a given therapy,” Kelley Davenport, a spokeswoman for Amgen, told Bloomberg Law. Biologic markers are medical signs that can be measured accurately and reproducibly. Now there’s a “push to expand the ability of global regulators and industry to use biomarkers for patient selection or exclusion in clinical trials, and as surrogate endpoints to support approval pending confirmation of clinical benefit,” Davenport said. A formal biomarker qualification process is underway, Davenport said, and Amgen is “dedicated to the development of precision and personalized medicines,” she added.

The Food and Drug Administration is interested in personalized medicine as well.

The agency approved 19 personalized medicines in 2017--a record number--and wants to speed up the approval rates even more. The next step will be to ensure patients can afford these medicines. The one-time treatment cost of $475,000 for Kymriah (tisagenlecleucel) is one type of barrier patients will face as precision treatments become more common.

Race and Drugs

Scientific discussions surrounding race and whether it’s a biologic category, or simply a societal identifier, are becoming more common. Some scientists are asking for race to be removed as a variable in genetic research altogether, saying it is typically poorly defined and misunderstood.

Race as it’s related to medical treatment becomes even more complicated when medical professionals rely primarily on skin color or physical characteristics to identify someone. It happens a lot with patients like Athena Asklipiadis, who is Japanese, Greek, Italian, Armenian, and Egyptian, but is typically just categorized as “Asian,” she said.

“Some places instruct their nurses or ER physicians to go visually off of what they think you are,” Asklipiadis told Bloomberg Law. “They may not even ask you,” she said. Asklipiadis founded Mixed Marrow, a nonprofit that specializes in finding bone-marrow donors for mixed-race patients.

Ryotaro Nakamura, a doctor at City of Hope, a cancer treatment and research center in California, says there’s a nuanced line to walk when it comes to linking genes to race.

“Genetic backgrounds aren’t necessarily directly linked to the genes that determine race, but they can be linked,” Nakamura told Bloomberg Law. He noted some drugs used in organ transplants generally metabolize differently in different patient populations as well.

Cardiovascular and kidney diseases are “the most common instances of when you might see these genetically-based, race-related differences play out and how they’re treated,” said Sally Satel, a research scholar at the American Enterprise Institute and a lecturer at Yale University.

How quickly the liver metabolizes a drug varies among black and white, and Asian patients, which affects dosing and which kinds of medicine a patient should take, Nakamura and Satel said.

Race plays a particularly big role in bone-marrow transplants, which treat blood cancers, bone marrow diseases, immune system disease, or genetic diseases like sickle cell disease. Biracial patients have an especially hard time finding the right matches for bone-marrow transplants, Nakamura told Bloomberg Law. He specializes in bone-marrow transplants, blood diseases, and cancer vaccines.

“The more diverse the person is, the more challenges they find to find a well-matched donor from the existing donor pool,” Nakamura said. That pool is made up of about 20 million people worldwide, but because mostly European Caucasians are included in the database, minorities and multiracial patients get the short end of the stick, he said.

In 2017, multiracial patients had a 3 percent chance of finding a bone-marrow donor, according to statistics from Mixed Marrow.

A crucial aspect of treating diverse patients is including them in clinical trials—a need the FDA highlighted in 2016.

“If you go back and look at all of these longitudinal studies like the Framingham study, you can draw all kinds of conclusions but it’s basically white men you’ve been studying all these years,” Meyer said. He’s referring to a heart study that began in 1948 that sought to study the causes of heart disease and included mostly white men. That study has since shed light on the need for more diversity in medical research.

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