With a $58.5 million gift from an anonymous donor, the School of Medicine is launching an Institute for Cell Engineering, fostering research that not long ago would have been marked as science fiction.

The new institute's scientists will focus on selecting, modifying and reprogramming human cells, molding them into therapeutic transplants for everything from Parkinson's, ALS and diabetes to heart failure, stroke and spinal cord injury.

The institute, which will be multidisciplinary, is believed to be the first initiative of its kind at an academic center. Its mission is to develop core technologies enabling application of curative approaches to specific human diseases.

Elias Zerhouni, executive vice dean of the School of Medicine and a driving force in the institute's creation, said the use of cells as therapeutic agents is a revolutionary concept that has "enormous untapped potential to treat currently incurable diseases." To not investigate this potential, he added, would be unthinkable.

"Somebody has to do it. We as an institution cannot deny to our patients the investigation of the potential of these therapies for them. And who knows? You may have a breakthrough that cures diabetes or cancer. I know it sounds like pie in the sky, but why not? If the cure is there, let's make sure it's there. Or, for that matter, why it is not," Zerhouni said. "I just thought this was a theme of research that Hopkins could not not do. We wanted this field to progress as unimpeded as possible because it may have a huge potential for all mankind. We are trying to do the right thing."

In announcing the creation of ICE, university President William R. Brody said, "Every now and then a scientific discovery is powerful enough to change established thinking. The recent groundbreaking research in stem cells has opened our eyes to ways cells might be used to regenerate tissues and perhaps, ultimately, entire organs. This research forms the basis for a bold new cross-disciplinary endeavor in cell engineering at Johns Hopkins."

"Pioneering stem cell research of such Hopkins scientists as John D. Gearhart and Curt Civin brought this into the realm of the possible just within the past few years," said Edward D. Miller, dean of the medical faculty and CEO of Johns Hopkins Medicine. "Elias Zerhouni then had the vision that by creating an intense, multidisciplinary research incubator, we could build on this foundation and take the next great leap forward, deciphering some of the fundamental mysteries of how cells go awry in disease and behave in transplants. He was able to convey to the donor the exciting potential of such a venture."

The $58.5 million donation, the largest single gift to the Johns Hopkins Medical Institutions and the second-largest in the university's history, is considered a seed fund to get the institute up and running. Government funding and additional philanthropic support are seen as necessary to fully exploit a growing field, Zerhouni said.

The Institute for Cell Engineering will occupy 40,000 square feet, about one-third, of the new Broadway Research Building, scheduled to be completed in 2003. Until then, temporary laboratories will be made available. Miller said the state of Maryland's funding of $23.8 million toward the new research building was critical in attracting the private funding "because it allowed us to guarantee appropriate space for our investigators."

During the coming months, the School of Medicine will recruit a core faculty group to be dedicated to the center. Zerhouni said the plan is to attract world-class leaders in the field of cell engineering and pair them with Hopkins scientists.

"The intent of the institute is to assemble the very best minds in the world who work on cell engineering problems, and to have them do it right here at Hopkins. They will interact with each other in such a way as to advance the field on a fundamental basis, not just an applied basis."

An independent scientific advisory board will govern the institute and determine the most promising areas of research.

Zerhouni said ICE is not a department, nor an institute in the traditional sense.

"We don't want it to be a department. We want it to be the helm of multidisciplinary teams from whatever discipline is needed to solve the problem. That is why the investigators will have primary appointments in the most relevant department of interest," Zerhouni said. "The institute also will not be the instrument for clinical trials, application to patients or doing all cell engineering-based therapies at Hopkins. We want the institute focused on cracking mysteries, not applications. But obviously we want ICE to spawn those translational applications; that is going to be the key to success."

While the institute will advance Hopkins' already strong program of embryonic stem cell research, scientists will extend that work into adult stem cells as a source of tissue.

"In many devastating diseases and injuries, these cell replacement therapies offer the best hope for patients," said John D. Gearhart, whose team of scientists announced the discovery of the most basic human stem cells two years ago and who has, since then, been instrumental in defining the potential of these cells for international scientific and government audiences.

The institute will have a core section for basic research and will include scientists from various departments to conduct translational research. Scientists with the institute's program highlighting basic cell immunology, for instance, hope to discover how to remove the characteristics of stem or other cells that trigger transplanted tissue rejection.

A particular focus on regeneration and repair of nerve tissue via stem and other engineered cells should help researchers develop specific therapies for Parkinson's disease, spinal cord injury, stroke and other widespread neurological disorders, said Ted Dawson, who, with fellow neuroscientist Valina Dawson, heads the neuroregeneration program. "The institute is rare," he added, "in the extent of collaboration it will foster across disciplines. That in itself gives a landmark opportunity for fruitful research."

Zerhouni said that one of the institute's boldest areas of research will be cellular reprogramming experiments, in which parts of the DNA of several cells can be modified to create novel cells with highly specialized and controlled functions.

Zerhouni acknowledged the controversies associated with research that manipulates human cells, notably stem cells, "but it is in the best interest of the public to have a not-for-profit, science-based institution like Hopkins take a leading role," he stated.

University officials say the donor felt that Hopkins would be an ideal environment for a visionary and unique philanthropic contribution to a field of discovery with unprecedented potential.

"The donor deserves all the credit," Zerhouni said. "The donor is the one who challenged us to go and think outside of the box."