By Kate Connors
MMS Director of Communications and Media Relations
Although personalized medicine, including RNA therapeutics, is often associated with oncology, this innovative approach to drug development has the potential to shape the future of health care in a variety of fields, says Phillip A. Sharp, Ph.D., institute professor at the
Massachusetts Institute of Technology (MIT) and recipient of the 1993 Nobel Prize in Physiology or Medicine.
Dr. Sharp will present the 127th annual Shattuck Lecture at the MMS Annual Meeting on April 28 at 12:30 p.m., at the Seaport Hotel in Boston. The Shattuck Lecture and Luncheon, organized by the New England Journal of Medicine, is named after MMS Past President Dr. George Cheyne Shattuck (1839–1840).
Dr. Sharp spoke with Vital Signs about the progress made in RNA therapeutics and in the future of personalized medicine in physician practice.
“RNA very generally is becoming a much more exciting therapeutic target and therapeutic agent,” he says. “RNA is being used now for vaccines. It’s being used to supplement for genetic diseases. It’s even being used as an agent to complement or stimulate the immune system in one way or
As with most medical developments, the process has been painstaking, Dr. Sharp acknowledges. But the promise is profound. “It’s taken 18 years and well over a billion dollars to get the science to where we are in terms of its use in humans. But once we solve that problem, we will have
solved it forever.”
Compared with conventional small molecule drugs, RNA therapeutics have the potential for much higher levels of efficacy, Dr. Sharp explains. Researchers in the field are able to target the specific sequence of a gene that is involved in a disease pathway. Although personalized
medicines — in which a genetic profile of a patient is used to guide the course of treatment — have been on the market for more than a decade, RNA therapeutics are new to a physician’s arsenal, with one approved in December for spinal muscular atrophy and with several Phase III clinical
It is the specificity that sets RNA therapeutics apart, says Jeffrey M. Drazen, M.D., editor-in-chief of the New England Journal of Medicine. “The future of nucleic acid-based therapeutics matters to physicians because of the potential to provide treatment for conditions that have
been hard or difficult to treat with standard drugs,” he says.
New Frontier of Science
In discussing his work, Dr. Sharp opines that RNA research is a new frontier of science, explaining that the Human Genome Project, rather than a culmination of understanding, was just the beginning: “We have a tendency to say that when we got the sequence of the human genome, we
understood man. We didn’t. We don’t. Our understanding of how disease processes occur, what the role of genetic variation between us is in that process, environmental impact, lifestyle impact: it is really quite incomplete. And so we still have fundamental discoveries to make.”
Dr. Sharp, who has been at MIT since 1974 and who has co-founded and advised several local biotechnology companies, praises the unique medical ecosystem in the Boston area: “The ability to conduct science at the forefront of understanding how biological systems work and then
walking across the street and working with venture capital and companies and all of the other parts that are necessary to translate that basic science into new therapeutic agents is fascinating. And then you come back and do that basic science all over again.”