"Wave of the Future." The Field of Genomic Medicine Gains Steam—And Funders

Genomics is being hailed as the “wave of the future” in medicine. The growing field—just two decades old—is focusing attention on how the genetic code and sequencing unique to each person’s DNA becomes distorted during the development of cancer and other major diseases. By personalizing the genetic code sequencing, oncologists believe they can use genomics to design and implement far more precise and effective medical treatment plans, saving lives and also money.

In fact, therapeutic development has already been transformed by genomics. According to bio-geneticist Eric S. Lander, a pioneer in the field, there are 800 different anticancer drugs in clinical development today. “Cancer drugs used to be just cellular poisons, but almost all of these new ones are targeted at particular gene products that have been discovered,” he said recently.

The contribution of private philanthropies to genomics is fairly new—but mushrooming rapidly. Back in 2001, the federal government set up the Human Genome Project to create the first fully documented genetic code modeled on the DNA of a handful of individuals. It took 15 years and a $3 billion investment to achieve that result. Since then, other genomic projects like the Cancer Genome Atlas, the Human Microbiome Project, and the 1,000 Genomes Project have emerged, based largely on major grants from the National Institutes of Health and the National Cancer Institute.

But with growing cutbacks in federal funding for biomedical research—which are expected to increase under the new Trump administration—a number of private medical charities, often operating with a venture philanthropy model, have moved to fill the void.

Some of them, like the American Cystic Fibrosis Foundation, are large entities that used to advise their members on how to prevent or cope with a chronic disease. Now, they are integral parts of the search for a cure. “Medical and disease charities once focused almost entirely on raising awareness and encouraging prevention, leaving the search for cures to the imperfect and often serendipitous interplay among government agencies, university researchers and drug companies,” one source notes.

Numerous foundations are now funding in this space, each seeking a distinctive niche in genomic medicine. A good example is Nationwide Foundation, which last week approved its fourth consecutive $10 million annual grant to the Pediatric Innovation Foundation based at Nationwide Children’s Hospital. This year’s $10 million Nationwide grant focuses specifically on pediatric genomics, a fast-growing sub-field of its own. Researchers have found that the types of cancer occurring in the pediatric population are markedly different from those seen in adults. For example, the major brain and solid tumors that arise in children are exceedingly rare in adults. Similarly, the specific genetic subtypes of leukemia—the most common malignancy in children—are vastly different in children. Cancer, while highly curable in children, remains their highest source of mortality. Moreover, the drugs used to treat pediatric cancer, while highly effective, have severe side effects that can reduce the quality of life in survivors.

The grant to the Nationwide Children’s Hospital, like an earlier grant from St. Jude Children's Hospital from the Elizabeth H. and James S. McDonnell III Genome Institute at Washington University, is aimed at uncovering the unique spectrum of genetic mutations that lead to malignant cell transformations for a selected group of cancers in children. Once identified in newly diagnosed cancer patients, it becomes possible to analyze the specific genetic sequencing pattern that can determine the etiology of the disease.

A similar effort that began last fall at San Diego’s Rady Institute of Genomic Medicine—founded in 2014 with a $120 million grant from insurance billionaire Ernest Rady—allowed doctors to develop a three-day sequencing model that led them to radically alter their treatment plans for the affected children—in some cases, cancelling major surgeries and treatments that were deemed likely to be ineffective. The main problem with these genomic diagnostic efforts is the cost: about $20,000 per child, which is beyond the reach of many patients since it may not be covered by their insurance plans. Seeking approval from insurance carriers on a case-by-case basis is an option, but the process is prolonged and can unduly delay treatment, endangering patients in need.

Given the early impressive results from these privately funded efforts, follow-up funding requests to major foundations are expected to mushroom in the coming year. Rady, for example, has ambitions of transforming its three-day sequencing model—and the follow-up analysis required—into the industry gold standard. “We’ve sent in a proposal to the MacArthur Foundation for $100 million over the next five years to put this capability into every children’s hospital in the United States,” Dr. Stephen Kingsmore, the Institute’s director, said in an interview. (It didn't make the list of finalists in this competition.)

Dr. Lander likens the genomics field to the early days of HIV therapy. It took a couple of decades before treatments became available that made the disease non-life threatening. Private philanthropies could do the same for cancer and other ailments, he believes.

“In an objective sense, this is a unique moment to be investing. This is the first decade when we can actually look across diseases in this systematic way. The idea that we’re not investing to let a generation of young people try their riskiest, cleverest ideas is a tragedy, because we’ve got such an opportunity now.”

A growing number of philanthropists agree.