Imagine replacing your failing kidney with a new one grown from your own skin cells.
Imagine your doctor knowing exactly which medicines will—and won’t—work with your genes.
Imagine curing chronic infections with a product that’s safer than antibiotics.
Imagine that some of these advances could happen within a few short years.
Those were among the predictions that held an audience spellbound at this spring’s “Women and Health” luncheon, sponsored by Sarasota Memorial Healthcare Foundation, at the Ritz-Carlton, Sarasota. A panel of experts, including two Sarasota physicians, pulmonologist Dr. Kirk Voelker and neurologist Dr. James Schumacher, described 21st-century medicine as racing towards new frontiers.
“We are about to reach a perfect storm of medical technology, information science and personal genomics in which innovation and discovery will grow exponentially,” Voelker told the luncheon-goers. Indeed, medical knowledge is growing as fast—or even faster—than technological knowledge has during the age of the Internet, the speakers said,and they stressed that patients in Sarasota will be among the first to benefit from the new information explosion.
Medical research is advancing especially rapidly in three key fields: stem cells, the human genome and the human microbiome. New discoveries in those fields are already transforming the treatment and prevention of a wide range of diseases. And experts say we can expect this 21st-century medical revolution to affect everything health-related: cancer screening, trauma recovery, Alzheimer’s prevention, cardiac surgery, psychiatric conditions, technology, medication, behavior—you name it.
The science is unfolding so rapidly that researchers are struggling to keep up, says Voelker: “The amount of data that we’re able to assimilate is increasing. But we don’t yet know exactly how to use the information correctly. Right now technology is outpacing our understanding.”
But medicine is about to catch up.
Turning Stem Cells Into Specialized Cells
They are the telltale signs of Parkinson’s disease: tremors, shuffled steps, hushed words. As the disease progressively destroys the brain’s dopamine-producing neurotransmitters, mobility worsens and dementia sets in.
Parkinson’s disease was identified in 1817. There is still no cure.
But if all goes well, a dopamine-replacement treatment for Parkinson’s patients will be available by 2020. And Sarasota will get it even sooner than that. It’s even possible that some of the 5,000 local patients diagnosed with Parkinson’s will have a chance to dance and sing within the next four years.
Schumacher, who serves as a research fellow for a neuroregeneration project at Harvard’s Stem Cell Institute, is one of those developing the treatment, which uses recent advances to grow the kind of dopamine that is destroyed by Parkinson’s disease and implant it into patient’s brains.
A key ingredient to the origin of life, stem cells have the unique ability to grow into almost any other kind of cell in the body. All other cells are specialized—cells from a kidney, for example, can create only other kidney cells. Until recently, scientists could harvest stem cells only from an embryo—a controversial practice. But now, scientists like those in Schumacher’s study can take specialized cells, like those from a patient’s skin, and actually transform them into what are called pluripotent stem cells. Like embryonic stem cells, these lab-created stem cells can then be differentiated into all manner of specialized cells—including dopamine. The Harvard researchers have already implanted dopamine they grew from stem cells into the brains of monkeys with Parkinson’s. “All the abnormal behavior stopped,” says Schumacher.
Since stem cell research no longer requires harvesting stem cells from fetuses, it’s no longer controversial. And because the stem cells used in Schumacher’s and other treatments are developed from the patient’s own skin, there is so far no indication of rejection; the body’s immune system recognizes the cells as “good guys.”
Phase I testing on humans will begin in Boston next year. “You can expect that we would include Sarasota in the Phase II part of this experiment,” says Schumacher. “We’ll be on the cutting edge of this.”
Schumacher moved to Sarasota from Boston because, as a friend told him, “This is where the Parkinson’s patients are.”
In addition to Schumacher’s Parkinson’s research, the Harvard Stem Cell Institute is performing stem cell studies regarding leukemia, cancer, heart disease, diabetes, kidney disease, Alzheimer’s, multiple sclerosis, hearing loss and more.
“The coolest thing with these stem cells is the rebuilding of organs,” says Schumacher. “If you need a new heart, grow one.” Or, in his case, grow enough dopamine to reverse Parkinson’s.
Mapping the Human Genome
The human genome is the collection of a person’s DNA, including billions of genetic and chromosomal characteristics. Every human cell with a nucleus contains a copy of the entire genome, an instruction book that tells cells how to grow and behave.
“Ten years ago it took $2 billion and a decade to come up with the first human genome,” says Voelker. “Now we can [map a person's genome] in a couple weeks and for less than $5,000.”
While stem cells are being used to fight existing conditions, the genome is the key to unlocking the new field of predictive medicine. Scientists can now pinpoint which genes cause many medical conditions, from cystic fibrosis to certain types of breast cancer. They’re also working to isolate genes that cause different people to respond to treatments and medications in different ways.
For example, if your CYP3A4 gene is not producing enough of the particular protein it’s responsible for, then the codeine you were prescribed to reduce pain can be metabolized into morphine—to a potentially toxic degree. On the flip side, if that same gene is a protein-producing fool, your body might not experience the codeine’s full effects.
Sarasota is currently one of 59 sites in a clinical trial focusing on the varied genomic responses to the blood thinner Warfarin. Researchers are studying how sensitivity to Warfarin varies with the genetic make-up of people 65 and older, with the goal of learning how to adjust the dose to the individual, resulting in fewer dangerous complications.
“Now we can match up the different kinds of patients with their genetic profile and someday provide a specific treatment plan for them,” says Schumacher. Already, some prescription medications are being paired with genetic testing to ensure their efficacy.
Excitement abounds about genome research, but skeptics question whether we will ever be able to match every disease to specific genes, especially in conditions as complex as cancer. But as the science progresses, more implications and applications will doubtlessly emerge.
Manipulating the Human Microbiome
At Sarasota’s sunlit Hawthorne Clinic and Quorum Innovations, founded in 2010 by the wife-and-husband team of Drs. Eva Berkes, a clinical immunologist, and Nicholas Monsul, an oculofacial surgeon, patients heading upstairs for their allergy shots walk past a vast lab. Floor-to-ceiling glass walls offer a full view of white boards bearing chemical equations and Ph.D. researchers growing experimental cultures. The scientists are engaged in studying the human microbiome, and they are among the many who believe tomorrow’s medical breakthroughs won’t be entirely about the body, but also about the organisms that inhabit and surround it.
Those organisms form the human microbiome, a collection of hundreds of species of microscopic organisms. And don’t let the “micro” part fool you: Those little creatures make up (believe it or not) 90 percent of your cellular mass—on your skin, in your digestive track and pretty much everywhere else.
What you may already know of the microbiome is probably related to digestive-aiding probiotics, made up of microbiotic organisms that help maintain a healthy “gut culture,” and the negative effects of broad-spectrum antibiotics on the body’s “good” bugs. Dr. Martin Glaser, director of NYU’s Human Microbiome Program and the author of a new book on the subject, argues that in addition to widespread overuse of antibiotics—in the animals we consume as well as in our own bodies—other aspects of modern medicine, such as the growing rate of Caesarean births in America, could also be damaging our microbiomes and causing a host of disorders, from asthma and allergies to childhood obesity.
“The genome is kind of like a computer hard drive—it’s difficult to change,” says the Hawthorne Clinic’s Monsul. “The microbiome is like the software—very malleable, always in flux, and in continual interaction with the genome.”
Some experts have compared the study of the microbiome to the invention of the microscope for its potential to reveal volumes of information about the human body. Chronic infection and inflammation, which are now likely to be treated with long-term doses of antibiotics, are some of the first conditions Berkes and Monsul believe can be addressed. By manipulating that ever-fluctuating collection of organisms—or by preventing them from being overly manipulated—scientists may one day be able to eliminate many diseases and disorders plaguing humanity.
Monsul and Berkes point to the potential for a disease like cystic fibrosis. The genetic marker for cystic fibrosis was identified 20 years ago, but the disease continues to ravage those afflicted with it. The leading cause of death? Chronic infection.
In addition to performing clinical and laboratory research, the couple’s company also develops products, and they recently received their first patent, for anti-inflammatory and anti-infective products derived from the human microbiome. Their first product, used for skincare, should debut by the end of the year. (Products to support health, rather than treat a disease, do not have to undergo FDA testing.)
“We’re trying to affect the software—first, the body’s surface,” says Monsul. “Sarasota is going to be the epicenter for a sneak peek of products for and derived from the human microbiome.”
The couple believes public demand for proactive, preventive health products will drive the microbiome field forward. And other research and development companies like theirs, including Colorado-based Microbiome Therapeutics, founded by Whole Foods board chair Dr. John Estrott, are springing up.
“It’s a topic whose time has come, and Sarasota is ready for it,” says Monsul. “It’s not trending that way. It is that way.”
Coming Medical Miracles
This spring in Geneva, the World Economic Forum identified 10 emerging technologies shaping society’s future. Five of them were medical:
Brain-computer interfaces: There are already ways to monitor electrical brain activity so that people with disabilities can type by controlling their brain waves. But as the technology advances, those people may also be able to operate wheelchairs using only their thoughts.
Body-adapted wearable electronics: Whether worn on the body, embedded in clothes or even under the skin, these devices can track information, such as heart rate and stress levels, giving people real-time feedback about their health.
Human microbiome therapeutics: Human microbiome technology is increasingly seen as an important source of treatment for serious diseases as well as for improving health. (Sarasota’s Quorum Innovations, see page 72, is at the forefront of this field.)
RNA-based therapeutics: A new generation of targeted, RNA-based drugs could help find new treatments for cancer and infectious diseases.
Predictive analytics: Using data and specialized machine-learning algorithms, scientists can now build detailed predictive models about human behavior, which can help in medical diagnosis.
Local Clinical Trials
In contrast to many other small cities, Sarasota hosts a variety of FDA clinical trials, which make the latest medications and technology available to local doctors and consumers even before they’re approved for widespread use.
“We have a medically sophisticated population that demands cutting-edge technology and doesn’t want to wait,” says Dr. Kirk Voelker, who directs clinical research for Sarasota Memorial Healthcare System. “Sarasota has a superb medical environment. By doing clinical research we attract world-class physicians interested in continuing to do clinical research while doing private practice. They don’t want the encumbrance of a university system.” Doctors are enticed by the trials, and, in turn, Sarasota is able to host more trials because of the influx of doctors, he explains.
In addition to local hospitals, medical centers and medical groups that participate in national clinical trials, the Sarasota-Manatee area has scientific research centers such as Roskamp Institute conducting research on everything from Alzheimer’s disease and orthopedic treatments to superbug vaccines. Sarasota Memorial Hospital is host to 15 current and impending cancer-related trials alone, plus others involving cardiology, pulmonology and surgery. One Sarasota Memorial trial uses a plasma proteomic classifier for diagnosing pulmonary nodules; another tests the new MitraClip valve for treating mitral regurgitation.
Voelker prefers to recruit later-phase trials. For example, he says, “Phase III is determining whether or not [a new drug] really works. We like that it’s been vetted for toxicity and proof of concept before bringing it here.”
Of course, medical innovation itself is subject to—and some would say hindered by—the FDA’s approval process, often criticized for cumbersome regulations that may delay widespread access to lifesaving measures—although no one wants distribution of unsafe procedures, either. “If we’re too quick to adapt things before fully vetting them in clinical trials, we risk putting people in undue harm,” says Voelker. “But if we don’t approve things, we risk putting people in undue harm, too.”
Voelker’s SMH predecessor, Dr. Pamela Tenaerts, is now working with Duke University and the FDA in trying to streamline the clinical trial process, especially by improving data-gathering techniques and technology.
A sampling of local companies creating breakthrough medical products, services and technologies.
Develops and markets software and smartphone communication apps for health care providers.
CAE Healthcare USA
Develops and manufactures medical simulation products for educating health care professionals.
Neurological research and treatment development, especially regarding Alzheimer’s disease.
Manufactures the O-Ace-Sys wound-care system developed by Sarasota’s Dr. Ed Lin.
Rapid Pathogen Screening
Develops and manufactures point-of-care diagnostic tests.
Gatorade Sports Science Institute at IMG Academy
Athletic performance research center.
Florida Clinical Research Center
Hosts pharmaceutical trials for treatments of psychiatric and other disorders.
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This article appears in the June 2014 issue of Sarasota Magazine. Like what you read? Click here to subscribe. >>