First John W. Walsh Translational Research Award Granted
The Alpha-1 Foundation (A1F) celebrates the first granting of the John W. Walsh Translational Research Award in Alpha-1 Antitrypsin Deficiency (Alpha-1), an honorable award named after John W. Walsh, co-founder of the A1F, a visionary man who dedicated his life’s work to finding a cure for Alpha-1 and helping those affected by it.
The peer reviewing Committee received four very strong submissions, and the award was granted to Joseph Kaserman, MD, instructor of medicine at Boston University School of Medicine (BUSM) and a physician in Pulmonary, Allergy, Sleep and Critical Care Medicine at Boston Medical Center.
The inaugural John W. Walsh Translational Research Award will be used by Kaserman to address a long outstanding question in the field of whether Alpha-1 carriers (these are individuals who have one normal Alpha1 gene (M) and one mutated gene (Z) have an increased risk of developing liver disease. As up to 2% of the U.S. population could be an Alpha-1 carrier there remains a critical need to address this important question.
For his research Kaserman will be working with specialized adult stem cells named induced pluripotent stem cells (iPSCs), which are stem cells that have been created from patients with Alpha-1. Importantly, iPSCs contain all the genetic information of the original patient, and provide the ability to compare cells from patients that have the same mutation but different disease severity, i.e. patients who have the Z-mutation with or without liver damage.
To understand the risk associated with having either one or two Z genes, Kaserman will take advantage of a technology known as CRISPR that allows for altering or “editing” the DNA of cells including iPSCs that he has made from Alpha-1 patients. Using this approach, Kaserman will be able to conduct experiments comparing “ZZ”, “MZ”, and “MM” cells that all come from the same individual and thus are genetically identical, varying only at the site of the Alpha-1 gene that has been edited.
Kaserman will then create liver cells from these iPSCs and examine whether MZ cells accumulate more misfolded AAT protein or are more susceptible to injury than MM cells. As there remain no approved therapies for Alpha-1 liver disease this study will also test new therapeutic agents targeting the mutated Z-protein within cells to see if this will protect the susceptible liver cells from injury.
Overall this study has the potential to not only address the concerns of many Alpha-1 carriers about their risk of developing liver disease, but also to find new treatments for Alpha-1 related liver disease helping both Alpha-1 carriers as well as Alpha-1 patients.
The award consists of up to a total of $225,000 over three years. In October 2016, in recognition of John Walsh’s many contributions, Mark Brantly, MD, announced a $225,000 gift from himself and his wife Judy Lew, MD, to the A1F, which was intended to establish the Research Award. Brantly is a professor of medicine, chief of molecular genetics and microbiology and A1F research professor at the University of Florida.
The Walsh Family is incredibly grateful to Dr. Mark Brantly and Dr. Judy Lew for their generous contribution to establish the Research Award, and expressed that “it is only through supporting research that we can find a cure for Alpha-1. We congratulate Dr. Kaserman for his work and dedication to moving John’s legacy forward. Keep the Faith”.
“This is a tangible, concrete way to continue the work that John was so committed to do. The Alpha-1 Foundation is honored that AlphaNet was able to make a matching contribution to this grant, enhancing even further the value we give to John’s legacy and his commitment to research”, said Henry Moehring, President and CEO of the A1F.
“We are extremely gratified that Kaserman has received this award because of the significance this kind of award has in sustaining the career of a young researcher,” said Andrew Wilson, MD, Associate Professor of Medicine at BUSM, and Director of The Alpha-1 Center at Boston University and Boston Medical Center.
“John Walsh had a major impact on my career and on the Alpha-1 research that we carry out at the Center for Regenerative Medicine of Boston University (CReM) and Boston Medical Center. He served as a mentor both to me and to Dr. Darrell Kotton and was instrumental in bringing us into the Alpha-1 community,” added Wilson.
“I am both excited and extremely humbled to have been selected as the inaugural recipient for this award. I never met John personally, but I know how relentless he was about finding a cure for Alpha-1. As a pulmonologist caring for patients suffering from the complications of Alpha-1, I am continually reminded that our current therapies remain grossly inadequate, but this only reinforces the clinical relevance of our research especially if we are going to one day realize John’s goal of finding a cure for Alpha-1,” said Kaserman.
Boston Medical Center and the Boston University Pulmonary Center have long-standing ties to the Alpha-1 community through Gordon Snider, MD, the first chief of pulmonary medicine at Boston University, and a seminal clinician, teacher and researcher in the field. Snider’s work demonstrating the role of neutrophil elastase in the pathogenesis of emphysema, and interest in the role of Alpha-1 Antitrypsin (AAT) in protecting the lung against it, created a legacy that lives on today in the form of the research that Kaserman and Wilson carry out in their lab.
Translational research in the biomedical field is understood as the application of basic knowledge acquired in the research laboratory to clinical practice, to improve medical care. This type of research is a necessary element for the success of any strategy that aims to improve the health of citizens through specific programs.
Press Release: PR Newswire
Original Source: Alpha-1 Foundation