Researchers at Beth Israel Deaconess Medical Center are diving into an area of genetic material previously considered “junk” to hunt for cures for cancer.
The hospital has launched a new Institute for RNA Medicine at its cancer center and hired a renowned biologist from Yale to run it. The institute will delve into what’s known as noncoding RNA, or RNA that does not make proteins, a different focus than other RNA research centers.
RNA typically is known as the molecules that provide the blueprints for making essential proteins. But these molecules represent only a tiny portion of genetic material — the other 98 percent of RNA, called noncoding RNA, has other functions.
Noncoding RNA, a relatively new area of research, is still not well understood but is being recognized as a vast untapped area with the potential to diagnose and treat many diseases. Some of these RNA can be used to regulate genes that cause disease. Other properties of RNA are still unexplored.
“We are facing a noncoding RNA revolution,” said Dr. Pier Paolo Pandolfi, director of the cancer center and cofounder of the RNA institute.
Pandolfi said the institute’s work will start with leukemia and cancers of the liver, prostate, breast, and lung. Research will span basic lab science to mouse and human studies.
Beth Israel’s focus on what was once considered “junk” comes as the hospital works to stay competitive with its bigger rivals in Boston, which receive more research dollars. Beth Israel received about $119 million from the National Institutes of Health in fiscal year 2013, while Massachusetts General Hospital received $339 million and Brigham and Women’s Hospital got $316 million.
‘It’s such a fresh area, it’s a little bit too new for companies to launch clinical trials.’
To launch the RNA Institute, Beth Israel has raised about $5 million. It is working to raise another $20 million so it can hire more scientists and bioinformaticians. Frank Slack, the institute’s new director, said he and his colleagues hope eventually to partner with drug companies to develop new cancer treatments. “It’s such a fresh area, it’s a little bit too new for companies to launch clinical trials,” Slack said. Over time, “we should be able to convince companies to take a chance on these molecules.”
Slack’s lab will work on identifying new RNA molecules, which could be used as targets for future drugs to slow or stop cancer cells. The Boston area is a hotbed of RNA research, but only in the last five to 10 years have researchers started shifting their focus to noncoding genetic material, after realizing that RNAs do more than provide the blueprint for proteins.
“Their real potential comes in understanding how genes are regulated, how genes are turned on and off in cells, which are key to controlling genes that contribute to disease,” said Phillip D. Zamore, codirector of the RNA Therapeutics Institute at UMass Medical School in Worcester.
Research in noncoding RNA, and treatments based on them, are poised to grow rapidly over the next several years as it attracts more interest from researchers and investors, said Jean-Francois Formela, a partner at the Cambridge venture capital firm Atlas Venture, and cofounder of RaNA Therapeutics Inc., a company developing medicines from noncoding RNA. But this field of research is probably many years from producing drugs that can treat cancer and other diseases, and much work remains.
“We know very little still,” Formela said. “We suspect we’re only scratching the surface.”