Autism and related disorders-a constellation of neurodevelopmental conditions affecting one in 59 children in the United States alone-;have become one of modern medicine's most confounding mysteries.
The condition is believed to arise from the complex interplay between genes and environment, yet its basic biology remains largely a black box.
Now, a new research effort at Harvard University led by Harvard Medical School is poised to identify the biologic roots and molecular changes that give rise to autism and related disorders with the goal of informing the development of better diagnostic tools and new therapies.
Harvard University has received a $20 million gift from philanthropists Lisa Yang and Hock Tan, an alumnus of Harvard Business School, to establish The Hock E. Tan and K. Lisa Yang Center for Autism Research at Harvard Medical School. The latest gift brings the total autism-related research funding provided by Yang and Tan to nearly $70 million.
The center will serve as a hub that brings together the diverse expertise of scientists and clinicians working throughout Harvard University, Harvard Medical School and its affiliated hospitals.
There is an urgent need to understand the fundamental biology of autism. I strongly believe that the multidisciplinary expertise convened by this center will propel us into a new era of autism research, enhancing our understanding of the condition and yielding critical new insights into its causes. This generous gift will be transformative for the field."
Greenberg is chair of the Department of Neurobiology at Harvard Medical School and the center's inaugural faculty leader.
Working under the premise that autism's complexity demands the cross-pollination of diverse expertise across different modes of scientific inquiry, the center will encompass the efforts of basic, translational and clinical scientists from the entire Harvard ecosystem.
The center will have its administrative home within the Harvard Brain Science Initiative, which brings together researchers from Harvard Medical School and its affiliated hospitals as well as from the Harvard Faculty of Arts and Sciences, the Harvard T.H. Chan School of Public Health and the Harvard John A. Paulson School of Engineering and Applied Sciences.
"Neuroscience has reached a unique inflection point. Advances such as single-cell analysis and optogenetics, coupled with an unprecedented ability to visualize molecular shifts down to the minutest level, will enable today's researchers to tackle a disorder as dauntingly complex as autism," said Harvard Medical School Dean George Q. Daley.
"Medical history has taught us that truly transformative therapies flow only from a clear understanding of the fundamental biology that underlies a condition," Daley added. "This gift will allow our researchers to generate critical insights about autism and related disorders."
Investigators at the new Harvard University center will collaborate with peer researchers at MIT and complement efforts already underway at The Hock E. Tan and K. Lisa Yang Center for Autism Research at the McGovern Institute for Brain Research at MIT, with the unique strengths of each institution converging toward a shared goal: understanding the roots of autism, explaining the condition's behavior and evolution and translating those insights into novel approaches to treat its symptoms.
"In a short time, the Tan-Yang Center at the McGovern Institute has supported groundbreaking research we believe will change our understanding of autism," said Robert Desimone, the director of the sibling center at MIT. "We look forward to joining forces with the new center at Harvard, to greatly accelerate the pace of autism-related research."
"We are excited and hopeful that these sibling centers at Harvard and MIT-;two powerhouses of biomedical research-;will continue to collaborate in a synergistic way and bring about critical new insights to our understanding of autism," Yang said.
Yang is a former investment banker who has devoted much of her time to mental health advocacy. Tan is president and CEO of Broadcom, a global infrastructure technology company.
Autism-spectrum disorders-;neurodevelopmental conditions that typically emerge in the first few years of life-;are marked by a cluster of symptoms, impaired social interactions and compromised communication skills.
Yet exactly what portion of these cases is rooted in genetic mutations and how they are influenced by environmental factors is an area of lingering uncertainty. Another key area of uncertainty is how much of autism's fundamental features arise in the brain and what influence organs and systems outside of the brain might have.
Two of the new center's initial areas of inquiry will address these critical gaps in knowledge.
One group of researchers will focus on understanding precisely what goes awry during critical windows in the first two years of life-;a period marked by rapid brain development, great neuroplasticity and intense wiring of the brain's circuits.
This is also the typical window of autism diagnosis. The scientists will try to understand what molecular, cellular or neural-circuitry changes underlie autism-fueling processes during this stage. Identifying such critical changes can help illuminate how experiences modulate brain development in individuals with autism.
Another group of researchers will examine the role of factors arising from organs and organ systems outside the brain that may drive autism risk. For example, the peripheral nervous system-;made up of nerve cells throughout the body that act as nodes to collect and transmit signals to the brain-;has emerged as a central player in the development of autism.
Heightened sensitivity to even light touch is a common feature in autism and one of the disorder's many perplexing symptoms. Recent research from neurobiologists and geneticists at Harvard Medical School has not only identified the molecular changes that give rise to heightened touch sensitivity in autism-spectrum disorders but also points to a possible treatment for the condition.