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Beth Israel Deaconess Medical Center Launches Institute for RNA Medicine

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NEW YORK (GenomeWeb) – The Cancer Center at Beth Israel Deaconess Medical Center (BIDMC) has just launched the Institute for RNA Medicine (IRM), an initiative that aims to bring together scientists from across the Harvard Medical School community to study and develop RNA-based therapeutics for cancer and other diseases.

Currently, the institute is composed of a core group of BIDMC investigators with expertise in the non-coding RNA field. These include Pier Paolo Pandolfi, director of the BIDMC Cancer Center; Harvard's John Rinn, who studies the epigenetics and disease associations of long, non-coding RNAs; and BIDMC's Daniel Tenen, who is investigating the relationships of transcription factors and gene regulation to cellular differentiation.

However, Frank Slack, who has come aboard as IRM's director after 14 years at Yale University conducting pioneering research on microRNAs, said that one of his first priorities is to recruit new faculty for the institute while helping to establish ties between other BIDMC/Harvard researchers.

"At the moment, [IRM] is a matrix institute, if you will," he said. "So the individual faculty [members] are staying where they are." But IRM anticipates arranging retreats, joint research and progress meetings, and symposia to foster collaboration.

IRM's first symposium, which will be focused on ncRNA research, is slated for the spring of 2015.

At the same time, Slack said BIDMC's Cancer Center has earmarked funds he can use to bring on a number of junior faculty members, the first of which is expected to be hired in the fall. He added that he hopes to secure additional funding in the near future to find senior faculty, as well.

According to Slack, the vision for IRM is to focus on basic science, translational science, and clinical drug development. In terms of the first area, research will be directed to the discovery and exploration of new classes of RNA such as lncRNAs and competing endogenous RNAs (ceRNAs) — RNAs that communicate and regulate each other by competing to bind to shared miRNAs, and which were discovered by Pandolfi and Rinn.

In general, Slack expects the IRM will focus on naturally occurring ncRNAs rather than, for instance, the synthetic siRNAs that are used in RNAi-based drugs. "My feeling is that the natural RNA molecules have a benefit in that the body's use of them has had millions of years to evolve around them and against off-target effects and the like," he explained.

However, Slack said that he is keeping an eye on the burgeoning CRISPR/Cas9 space, even though the molecules that comprise the gene-editing technology aren't naturally occurring in human cells. Further, therapeutic approaches based on protein-coding messenger RNA will also be considered.

Translational research at IRM will center around elucidating the roles of such RNAs in both healthy and disease states, finding ways in which they can be used therapeutically and developing technologies to enable their delivery, Slack said. "In fact, that's already beginning with some of the researchers in the institute."

In addition to drawing from the pool of scientific expertise that exists within Harvard and its affiliate organizations, the translational component of IRM's mission is also expected to benefit from the BIDMC Cancer Center's so-called "mouse hospital" — a facility for the generation and study of transgenic mouse models of disease.

"By bringing together systems biologists, mouse modelers, bioinformaticians, and molecular biologists within a clinically based platform, we will provide a unique opportunity to not only identify new non-coding RNA pathways and determine if they are targets for therapeutic intervention, but also to test candidate drugs," Pandolfi said of the mouse hospital in a statement.

Longer term, Slack hopes that IRM will be able to carry the most promising of its discoveries into human testing on its own.

"We are emboldened by the fact that there are already some [RNA drug] clinical trials underway that are sponsored by industry, but I would like to see a situation where academic centers are not beholden to industry in order to start these clinical trials," he told Gene Silencing News.

To Slack, there is often reluctance on the part of the pharmaceutical industry to take therapies based on novel technologies into human testing. By taking the lead on moving RNA therapies into initial clinical studies, IRM would not need to delay starting trials while "a board of directors [makes] a decision about whether [the drug] is going to be lucrative down the road."

Should IRM be able to demonstrate the safety and pharmacokinetics of an innovative RNA drug in its own Phase I study, he added, it will likely be easier to get the industry support necessary to advance such an agent into later-stage trials.

Given its roots in BIDMC's Cancer Center, IRM will initially focus on cancer, particularly blood cancers, prostate cancer, lung cancer, and breast cancer. But given the overall breath of RNA drug research, IRM expects to expand into other areas including cardiovascular disease, metabolic conditions, and central nervous system disorders.

With the creation of IRM, BIDMC joins a growing list of academic centers setting up institutes tasked specifically with studying the therapeutic potential RNA.

In late 2009, the University of Massachusetts Medical School began setting up its RNA Therapeutics Institute, which facilitates collaboration between top researchers in the field while providing the resources required to advance their work.

And in 2010, the University at Albany established the RNA Institute to conduct research and develop tools for the development of RNA-based medicines and diagnostics.

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