Tom Maniatis, Ph.D. spent most of his career as a self-described “basic scientist” studying genes. However, when his sister was diagnosed with ALS 12 years ago, Dr. Maniatis became involved in ALS research. Initially he served as an advisor to the ALS Association, but after ALS took his sister’s life he initiated research in his own laboratory.

His current ALS research focus is to identify early events in the ALS disease process. “Scientists have been studying the causes of ALS in mouse models for many years, and these studies have provided many insights. However, it has been very difficult to pinpoint the earliest events that trigger the disease” said Dr. Maniatis.

Dr. Maniatis' laboratory is exploiting new stem cell technology that has made it possible to reprogram skin cells from ALS patients to produce motor neurons for studies in cell culture. He is applying powerful tools for genome research to these cells in an effort to detect molecular differences between normal and ALS motor neurons.

“We hope that studies of motor neurons produced in this manner will provide clues to the cause of ALS”, said Dr. Maniatis. "In fact, preliminary data has revealed significant differences between motor neurons from control and ALS patient samples."

Dr. Maniatis is particularly excited about the recent discovery in other labs that two genes that cause ALS when altered by mutations (familial ALS) also happen to function in gene expression - the focus of his basic research program. Thus, his lab has combined the approaches to study ALS described above with those of his basic research to search for new clues to the causes of ALS.

Dr. Maniatis is the Isidore S. Edelman Professor and Chairman of Biochemistry and Molecular Biophysics at the Columbia University Medical Center. He received his bachelor’s degree from the University of Colorado at Boulder, and a doctorate in molecular biology from Vanderbilt University. His postdoctoral studies were carried out at Harvard University and the Medical Research Council for Molecular Biology in Cambridge, England. He’s held research and academic positions at Harvard University, California Institute of Technology and the Cold Spring Harbor Laboratory. He also serves on the Board of Directors of the ALS Therapy Alliance.

Dr. Maniatis has won numerous awards, including the Eli Lilly Award in Microbiology and Immunology early in his career, and more recently the Scientific Achievement Award of the American Medical Association. He is a member of the U.S. National Academy of Science.

He’s best known for pioneering the development and application of recombinant DNA methods to the study of gene regulation. His research has impacted a broad spectrum of biomedical fields, from basic mechanisms of gene expression to advances in understanding human genetic and inflammatory diseases. He has also been a pioneer in biotechnology, founding companies that developed several FDA approved drugs. His leadership was recognized by the Jacob Heskel Gabbay Award in Biotechnology and Medicine.

For more information about Dr. Maniatis, visit or Directors & Staff page.

Lawrence Hayward, M.D., Ph.D. received his doctorate degrees from Baylor College of Medicine in Houston and completed a neurology residency in 1993 at Massachusetts General Hospital. He then started working in Dr. Robert Brown’s laboratory as a Neuromuscular research and clinical fellow. After following ALS patients in the clinic for several years, Dr. Hayward initiated biochemical studies to identify toxic properties of mutant SOD1 enzymes that cause a familial form of ALS.

Along the way, he also encountered a more personal connection to ALS, when it became clear that a few members of his own extended family had been diagnosed with the disease.

“Although the total number of individuals living with ALS may not be as high as those with more common neurodegenerative conditions, ALS nevertheless impacts families or friends with surprising frequency,” Dr. Hayward explained.

In 2000, he started his own ALS research laboratory at the University of Massachusetts Medical School, where he follows patients in the neuromuscular clinic and teaches medical and graduate students.

Dr. Hayward, an original member of the ATA Board of Directors, became interested first to define the consequences of SOD1 mutations upon the folding of the protein and its interactions in cells and mouse model tissues. Since 2008, his lab has focused on establishing new in vivo ALS models using both mouse and zebrafish systems.

“Genetically engineered mice are highly informative to ALS research because these mammals can mimic the conditions found in ALS patients on an accelerated time scale,” Dr. Hayward said.

He is currently studying newly identified ALS genes that may perturb RNA metabolism, which is normally critical for the health of the motor neurons. Initial studies suggest that mutant variants of an ALS gene called FUS, discovered by Dr. Brown’s group, may alter the ability of motor neurons to deal with environmental stresses. Early support from the ATA to establish mutant FUS transgenic mouse models in the Hayward lab led to an ongoing NIH-sponsored study to characterize the FUS mouse phenotypes.

In a promising new ATA-funded study, Dr. Hayward is examining the earliest consequences of mutant FUS in the motor neurons of zebrafish embryos.

“Because zebrafish eggs can be manipulated more easily than mouse embryos and develop very rapidly to form a functioning spinal cord within 2-3 days, these animals can be used to explore many hypotheses quickly, and the most promising results can further be validated in mouse models," Dr. Hayward explained. “Zebrafish embryos remain transparent as they develop, so scientists can express florescent proteins that allow visualization of the structure and physiological state of specific spinal cord cells in living animals.”

The hope is that zebrafish embryos will allow the characterization of early perturbations caused by the mutant ALS genes that may be driving the subsequent neuronal loss. Scientists can turn on or off genes of interest in hundreds of zebrafish eggs and – a few days later – have many animals ready for analysis or even for screening of drugs that might counteract the effect of the mutants. Findings from the zebrafish can be transferred to the mouse models for further study and the possible discovery of new therapeutic targets in ALS.

For more information about Dr. Hayward, visit or Directors & Staff page.

Jonathan Roberts, Executive Vice President and COO for CVS Caremark Pharmacy Services, has been touched by ALS in a number of ways. First, a friend and colleague at CVS, John Finneran, was diagnosed with the disease. Soon after, Roberts’ wife, Resi, was diagnosed with ALS. Her disease progressed rapidly and she passed away in 2003. Since then, other CVS colleagues and a family friend have also been diagnosed.

“I have lots of connections to ALS. Before John Finneran was diagnosed, I knew what ALS was, but didn’t know anyone living with the disease. Now, I wonder if I’m just more aware of it, or if its prevalence is increasing,” said Roberts.

As the father of three children – two boys and a girl – he notes that ALS doesn’t just afflict the patient. It impacts the entire family. Therefore, Roberts, an active member of the ALS Therapy Alliance’s (ATA) board of directors, is working to find a cure.

As Co-Chair of CVS/pharmacy’s annual ALS in-store fundraising initiative, Roberts is proud that the campaign has raised nearly $24 million over the last nine years. The 2011 campaign will run from June 5-25.

“The success of our campaign is a testament to our store employees who support it, and our customers who have generously donated to the cause every year,” Roberts explained. “For every customer and CVS colleague that makes a donation, we’re one dollar closer to curing this devastating disease.”

As part of his role on the ATA board, Roberts reviews research grant proposals, which he calls “a very interesting process.” In addition, he serves on the ALS Association’s board of directors and is a trustee. He is also involved with their Rhode Island chapter, regularly supporting their annual "Evening of Hope" dinner.

Roberts first learned about the great work Dr. Robert Brown was doing when his wife got sick. The Roberts family was living in Atlanta, and local physicians were having difficulty diagnosing Resi. Roberts found Dr. Brown, a leading ALS clinician, and brought Resi to his practice.

In 2002, CVS/pharmacy and the ALS Therapy Alliance collaborated on a small pilot program in New England, raising funds and awareness for ALS. From there, the campaign evolved into an annual, national effort, which continues today, nearly a decade later.

Roberts is a seasoned pharmacy executive with more than 30 years of experience in the field, 21 of those with CVS Caremark. Roberts oversees CVS Caremark’s operations, as well as pharmaceutical purchasing and marketing.

He earned his degree in pharmacy from the Virginia Commonwealth University School of Pharmacy, and is a graduate of the Wharton Executive Management Program.

For more information about Mr. Roberts, visit or Directors & Staff page.

In 1986, Dr. H. Robert Horvitz’s father was diagnosed with amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease). He came to Boston and was seen by Dr. Robert Brown, a supportive, sympathetic neurologist from Mass General Hospital. After ongoing talks about ALS, Dr. Horvitz and Dr. Brown decided to collaborate to study the genetics on ALS.

Since then, a small team engaged by Dr. Horvitz has performed research in Dr. Brown's laboratory. This effort, in conjunction with that of other scientists globally, led to the discovery that one gene is responsible for familial ALS encodes - the enzyme copper-zinc superoxide dismutase.

In 1989, Dr. Horvitz’s father died from ALS. Seeing his father suffer from – and succumb to – this horrific neurodegenerative disease, reinforced Dr. Horvitz’s strong desire to find a cure.

Dr. Horvitz received the Nobel Prize in Physiology or Medicine in 2002 for his discoveries concerning genetic regulation of organ development and programmed cell death.

Today, his valuable work continues. He is the David H. Koch Professor of Biology at the Massachusetts Institute of Technology (MIT); an Investigator of the Howard Hughes Medical Institute; Neurobiologist (Neurology) at the Massachusetts General Hospital; and a Member of the MIT McGovern Institute for Brain Research and the MIT Koch Institute for Integrative Cancer Research.

Dr. Horvitz graduated from MIT, performed his graduate studies at Harvard University and received his Ph.D. in Biology for biochemical and genetic studies of mechanisms of regulating gene expression. Since then he has studied the development and behavior of the microscopic roundworm Caenorhabditis elegans, and this research has helped define evolutionarily conserved molecular genetic pathways important in human biology and human disease, including the pathway responsible for programmed cell death (apoptosis).

Dr. Horvitz has served on many editorial boards, visiting committees and advisory committees, and has received many honors, including the U.S. National Academies of Science Award in Molecular Biology; the Charles A. Dana Award for Pioneering Achievements in Health; the Gairdner Foundation International Award; the March of Dimes Prize in Developmental Biology; and the Bristol-Myers Squibb Award for Distinguished Achievement in Neuroscience.

For more information about Dr. Horvitz, visit or Directors & Staff page.