Mark Batshaw
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Mark Levitt Batshaw (born 19 September 1945) is a Canadian-born physician, medical researcher and academic administrator. He is a professor in the department of pediatrics and an associate dean at the
George Washington University School of Medicine & Health Sciences The George Washington University School of Medicine and Health Sciences (abbreviated as GW Medical School or SMHS) is the professional medical school of the George Washington University, in Washington, D.C. SMHS is one of the most selective medi ...
and is the physician-in-chief and chief academic officer at the Children’s National Medical Center in Washington, D.C. He is known for his research into
urea cycle The urea cycle (also known as the ornithine cycle) is a cycle of biochemical reactions that produces urea (NH2)2CO from ammonia (NH3). Animals that use this cycle, mainly amphibians and mammals, are called ureotelic. The urea cycle converts highl ...
disorders and
gene therapy Gene therapy is a medical field which focuses on the genetic modification of cells to produce a therapeutic effect or the treatment of disease by repairing or reconstructing defective genetic material. The first attempt at modifying human DN ...
, and is the author of the classic textbook "Children with Disabilities".


Biography

Batshaw was born in
Montreal Montreal ( ; officially Montréal, ) is the List of the largest municipalities in Canada by population, second-most populous city in Canada and List of towns in Quebec, most populous city in the Provinces and territories of Canada, Canadian ...
, Canada, in 1945 and is a United States citizen. His father, Manuel G. Batshaw, was a social worker. Batshaw manifested
dyslexia Dyslexia, also known until the 1960s as word blindness, is a disorder characterized by reading below the expected level for one's age. Different people are affected to different degrees. Problems may include difficulties in spelling words, r ...
and
attention deficit hyperactivity disorder Attention deficit hyperactivity disorder (ADHD) is a neurodevelopmental disorder characterised by excessive amounts of inattention, hyperactivity, and impulsivity that are pervasive, impairing in multiple contexts, and otherwise age-inap ...
as a child at a time where there were limited special education services or medication for treatment. It was this experience that led him to the fields of developmental pediatrics and genetics. Batshaw received his M.D. from the
University of Chicago The University of Chicago (UChicago, Chicago, U of C, or UChi) is a private research university in Chicago, Illinois. Its main campus is located in Chicago's Hyde Park neighborhood. The University of Chicago is consistently ranked among the b ...
Pritzker School of Medicine The Pritzker School of Medicine is the Doctor of Medicine, M.D.-granting unit of the Biological Sciences Division of the University of Chicago. It is located on the university's main campus in the historic Hyde Park, Chicago, Hyde Park neighbo ...
in 1971, performed his pediatric residency at The Hospital for Sick Children,
University of Toronto The University of Toronto (UToronto or U of T) is a public research university in Toronto, Ontario, Canada, located on the grounds that surround Queen's Park. It was founded by royal charter in 1827 as King's College, the first institution ...
, and his clinical fellowship at the
Kennedy Krieger Institute The Kennedy Krieger Institute () is a nonprofit, 501(c)(3) tax-exempt, Johns Hopkins affiliate located in Baltimore, Maryland, that provides in-patient and out-patient medical care, community services, and school based programs for children and ...
,
Johns Hopkins Hospital The Johns Hopkins Hospital (JHH) is the teaching hospital and biomedical research facility of the Johns Hopkins School of Medicine, located in Baltimore, Maryland, U.S. It was founded in 1889 using money from a bequest of over $7 million (1873 mo ...
. From 1988-1998, Batshaw was Physician-in-Chief of the Children’s Seashore House at the
Children’s Hospital of Philadelphia The Children's Hospital of Philadelphia (CHOP) is a children's hospital in Philadelphia, Pennsylvania, with its primary campus located in the University City, Philadelphia, University City neighborhood of West Philadelphia in the campus of the Uni ...
. He was Chairman of Pediatrics at the
George Washington University School of Medicine & Health Sciences The George Washington University School of Medicine and Health Sciences (abbreviated as GW Medical School or SMHS) is the professional medical school of the George Washington University, in Washington, D.C. SMHS is one of the most selective medi ...
from 1998-2014 and as of 2017 is the Associate Dean for Academic Affairs at the university, and Chief Academic Officer and Physician-in-Chief at Children's National Medical Center. He has served as president of both the Society for Inherited Metabolic Disorders and the
American Pediatric Society The American Pediatric Society (APS) is the first pediatric society established in North America. Created in 1888, the APS pursues a vision of an engaged, inclusive, and impactful community of pediatric thought leaders. The APS mission is to sha ...
. He and his wife Karen are the parents of three children.


Research

During his clinical fellowship at the
Kennedy Krieger Institute The Kennedy Krieger Institute () is a nonprofit, 501(c)(3) tax-exempt, Johns Hopkins affiliate located in Baltimore, Maryland, that provides in-patient and out-patient medical care, community services, and school based programs for children and ...
, Batshaw and his colleague Saul Brusilow developed a successful treatment for a fatal
urea cycle The urea cycle (also known as the ornithine cycle) is a cycle of biochemical reactions that produces urea (NH2)2CO from ammonia (NH3). Animals that use this cycle, mainly amphibians and mammals, are called ureotelic. The urea cycle converts highl ...
disorder. The treatment, involving
sodium phenylbutyrate Sodium phenylbutyrate, sold under the brand name Buphenyl among others, is a salt of an aromatic fatty acid, 4-phenylbutyrate (4-PBA) or 4-phenylbutyric acid. The compound is used to treat urea cycle disorders, because its metabolites offer an ...
and
glycerol phenylbutyrate Glycerol phenylbutyrate (USAN), trade name Ravicti, is a medication used in the treatment of certain inborn urea cycle disorders. The medication works by preventing the harmful buildup of ammonia in the body. It is an FDA-approved prescription dr ...
, is still in use. In 1988, Batshaw moved from Hopkins to the
University of Pennsylvania The University of Pennsylvania (also known as Penn or UPenn) is a private research university in Philadelphia. It is the fourth-oldest institution of higher education in the United States and is ranked among the highest-regarded universitie ...
where he began a collaboration with
James Wilson James Wilson may refer to: Politicians and government officials Canada *James Wilson (Upper Canada politician) (1770–1847), English-born farmer and political figure in Upper Canada * James Crocket Wilson (1841–1899), Canadian MP from Quebe ...
. They developed an adenovirus vector and started a
gene therapy Gene therapy is a medical field which focuses on the genetic modification of cells to produce a therapeutic effect or the treatment of disease by repairing or reconstructing defective genetic material. The first attempt at modifying human DN ...
clinical trial that resulted in the death of a patient,
Jesse Gelsinger Jesse Gelsinger (June 18, 1981 – September 17, 1999) was the first person publicly identified as having died in a clinical trial for gene therapy. Gelsinger suffered from ornithine transcarbamylase deficiency, an X-linked genetic disease of the ...
, in 1999 and a subsequent slowdown of the development of gene therapy. In the past decade, the field has recoveredGene Therapy Emerges From Disgrace to Be the Next Big Thing, Again
by Carl Zimmer August 13, 2013 Wired
and Wilson and Batshaw’s teams have developed an adeno-associated virus vector that has been successful in preclinical studies of an animal model of urea cycle disorders.


References

{{DEFAULTSORT:Batshaw, Mark Living people 1945 births George Washington University faculty American geneticists Canadian emigrants to the United States People from Montreal Pritzker School of Medicine alumni University of Pennsylvania alumni