Working with tiny tropical fish, MGH Research Scholar Randall T. Peterson, PhD, is pursuing new treatments for Alzheimer’s, schizophrenia and other disease.
Decoding Diseases
Dr. Randy Peterson, PhD and Dr. Sylvie Breton, PhD in Dr. Breton's Simches Lab
Dr. Randy Peterson, PhD, and Dr. Sylvie Breton, PhD, in Dr. Breton’s Simches Lab

Randall T. Peterson, PhD, hopes his research with tiny, tropical fish and robots will improve the lives of people with Alzheimer’s disease, schizophrenia, heart conditions and leukemia.

Dr. Peterson investigates similarities between humans and creatures called zebrafish. He uses those characteristics to discover small molecules, which could play a role in the development of new drugs.

He hopes to help patients he’s never met. “There’s something really compelling about facing a true mystery and working hard to solve that problem,” Dr. Peterson says. “I’ve been involved a couple of times with discovery of something that develops potential to treat somebody and make a difference in that person’s life — and that is satisfying in a profound, almost inexplicable way.”

As a basic scientist at Massachusetts General Hospital, he collaborates with many researchers and physicians who share the goal of improving patient care.

Mass General has the largest hospital-based biomedical research program in the United States and attracts many of the world’s brightest medical researchers. But researchers venturing into new areas with creative, but unproven ideas, face many challenges in securing funding for their work.

That’s why the MGH Research Scholars Program is essential. With support from the Charles and Ann Sanders Mass General Research Scholar Award, Dr. Peterson can move forward and investigate diseases of the brain and nervous system. He can use the award to establish results that might later attract the attention of funding agencies like the National Institutes of Health.

Dr. Sanders, who had a distinguished career at Mass General as a cardiologist and general director, recognizes the critical importance of medical research. “There is simply no progress in medicine without research,” he says. “Ann and I are pleased to support the Research Scholars Program because Mass General’s collaborative research community is highly distinguished and recognized worldwide for the innovation and changes it has made possible in medicine.”

What Researchers Can Learn from the Minds of Fish

Diseases that affect the brain are devastating. One of the best known, Alzheimer’s disease, steals a person’s memory, personality and ability to live independently. Other diseases that damage the brain’s ability to function, like schizophrenia, also take a heavy toll on people, their families and society. Dr. Peterson will use his MGH Research Scholar Award to work with his colleagues to learn more about diseases of the brain from zebrafish.

Dr. Peterson’s innovative method of studying zebrafish is an example of how basic scientific research can touch so many different diseases.

Zebrafish have many of the same organs as people as well as complex nervous and cardiovascular systems. Genetically, they are similar, too. Because these tiny fish are transparent, researchers can peer into their brains with a microscope.

Dr. Peterson and his medical research team genetically engineer zebrafish to develop a human diseases, like leukemia or schizophrenia. Then, they use robotic microscopes to observe how the fish react to those genetic changes as well as to stress, pain and other stimuli. His lab has studied the fish’s reaction to antipsychotic drugs and medications used to treat attention deficit disorder and Parkinson’s disease. Their work has shown that zebrafish can be used to discover new chemical compounds that have the potential to become drugs for treating nervous system disorders.

Dr. Peterson’s approach is the opposite of the drug industry strategy of making incremental improvements on existing drugs to target a small number of well-studied proteins. In his lab, the robots can systematically test tens of thousands of chemical compounds to determine if any compound is improving the disease symptoms in the fish.

Dr. Peterson believes this approach to medical research opens up the possibility for finding transformative new drugs that work in unexpected ways and could help move new drugs to patients sooner.

A Critical Time to Support the Best of the Best

Competition for the awards is intense. During the program’s inaugural year, more than 115 researchers applied. Five researchers, the best of the best, including Dr. Peterson, were named MGH Research Scholars.

Nearly all of Dr. Peterson’s projects are collaborative. He currently works closely with J. Keith Joung, MD, PhD, of Mass General, who has developed man-made proteins called zinc-finger nucleases that Dr. Peterson uses to engineer the set of genes in zebrafish.

Dr. Peterson hopes his medical research with tiny, tropical fish and robots will improve the lives of people with Alzheimer’s disease, schizophrenia, heart conditions and leukemia.

Dr. Peterson came to Mass General in 2000 after finishing his doctoral degree and has been here ever since. “What’s amazing about being here at MGH is that you have the best clinical people mixing with really outstanding basic scientists. There are few places in the world that have the best of both worlds,” he says.

“Dr. Peterson’s innovative method of studying zebrafish is an example of how basic scientific research can touch so many different diseases,” says Bruce Walker, MD, acclaimed physician-researcher and co-chair of Mass General’s Research Scholars Award Committee.

Randall T. Peterson, PhD
Massachusetts General Hospital
Harvard Medical School

Associate professor of Medicine, Harvard Medical School

Assistant in Biology, Massachusetts General Hospital
Senior associate member, Broad Institute

Randy received his PhD from Harvard University after studying as a Howard Hughes Medical Institute predoctoral fellow in the laboratory of professor Stuart L. Schreiber. Following graduation, he completed a postdoctoral fellowship with professor Mark Fishman at Massachusetts General Hospital. Dr. Peterson is currently associate professor of Medicine at Harvard Medical School and assistant in Biology at Massachusetts General Hospital and senior associate member of the Broad Institute.