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       A P u b L Ic AT I o n o f T he R e s ea Rc h I ns T I T u T e aT n aT I o nwI d e c hI l dRe n’s ho sp I Tal




A Day in the Life of
The ReseARch InsTITuTe
7:30 a.M.
daYBReaK

A crescent moon appears
above Research Building II
as dawn breaks on a beautiful
Ohio Tuesday morning.
fALL / WI nTeR 2008



                                                                              research is dedicated to the
                                                                              mission of informing and inspiring
Twenty-four hours from now, Nationwide Children’s Hospital                    readers by highlighting scientific
will not be the same hospital it is at this moment. Knowledge                 performance at The Research Institute
will grow. Possibilities will emerge. Enthusiasm will escalate.               at nationwide children’s hospital.

Lives will be changed through discovery.                                      This publication is produced
                                                                              biannually by the Marketing and
                                                                              Public Relations Department at
As the research engine of Nationwide Children’s, The Research
                                                                              nationwide children’s hospital.
Institute is a key contributor to this daily transformation. Each
day begins with a sense of excitement and mission. Innovative
experiments are conceived and carried out by our research teams,
data are collected and careful analyses are conducted. Data are
collaboratively shared among faculty, staff and students. We
strive for exciting conclusions, but sometimes more questions
arise than answers. We persevere, stubbornly emboldened by the
                                                                              l e a d e Rs hI p
potential that scientific inquiry will lead to better health for              The Research Institute at
                                                                              nationwide children’s hospital
children and their families. The beat goes on.
                                                                              John a. Barnard, Md
                                                                              President

In this issue of reSearch, we provide you with snapshots of a
                 reSearch                                                     lauren o. Bakaletz, phd
                                                                              Vice President, Basic Sciences Research
“typical Tuesday” in a day in the life of The Research Institute.
                                                                              Kelly Kelleher, Md, Mph
Through this photographic timeline, we hope you will appreciate               Vice President, Health Services Research
                                                                              william e. smoyer, Md
the heart and soul of one of the top pediatric research centers in            Vice President, Clinical and Translational Research
the United States.                                                            Grant Morrow III, Md
                                                                              Medical Director
                                                                              daniel R. Mann
                                                                              Vice President, Research Administration
                                                                              and Operations
                                                                              Katherine s. Milem
                                                                              Vice President, Research Business Services


                                                                              research
John Barnard, MD                                                              Writer and editor
                                                                              Melissa hamilton
President
The Research Institute at Nationwide Children’s Hospital                      Art Directors
                                                                              John ordaz
Professor of Pediatrics                                                       Tanya Burgess Bender
The Ohio State University College of Medicine
                                                                              Photographers
                                                                              Brad smith
                                                                              dan smith
                                                                              Manager, Research communications
                                                                              Jan arthur
                                                                              contact us at
                                                                              ResearchMagazine@nationwidechildrens.org




                                                                     The ReseaRch InsTITuTe at nationwide children’s hospital | 1
8:03 a.M.
                                                               FocusInG on FIRsT-RaTe FacIlITIes

                                                               Phil Bowers helps Mary Connell from
                                                               Children’s Hospital of Philadelphia pour
                                                               off excess liquid nitrogen from shipping
                                                               containers holding tissue samples that are
                                                               being sent to Boston.

                                                               As facilities manager at The Research
                                                               Institute, Bowers begins his work day
                                                               at 6 a.m. and spends his early hours
                                                               walking through and “waking up” the
                                                               research buildings. Bowers checks for alarms,
                                                               notes equipment problems and performs
                                                               safety checks. By the time he completes his
                                                               rounds he will have covered every floor in
                                                               the 300,000 square feet of research space,
                                                               trekking through more than 90 labs.




2 | The ReseaRch InsTITuTe at nationwide children’s hospital
9:18 a.M.
Fedex FRenzY

This Tuesday is like every other weekday in the Biopathology
Center. Staff members receive between 20 and 40 FedEx
packages containing pediatric and adult cancer samples
from hospitals all across North America. After the samples
are unpacked, they are entered into a detailed, secure
database. Tissue samples are prepared for digital microscopy
and shared with other national experts using a virtual
microscope. Some samples are carefully catalogued for storage
in a liquid nitrogen freezer, while others undergo clinical
testing to help make vital cancer treatment decisions.

The Biopathology Center is funded by the National Cancer
Institute and serves as the data repository for cancer samples
from more than 500 hospitals. Specimens from this Center are
used for cancer research in institutions worldwide. Because of
the Biopathology Center, The Research Institute at Nationwide
Children’s Hospital receives more grant funds from the
Children’s Oncology Group than any other national program.




               The ReseaRch InsTITuTe at nationwide children’s hospital | 3
9:24 a.M.
    FRoM BedsIde To Bench and BacK aGaIn

    Dr. W. Joshua Frazier is both a researcher in the Center for Perinatal Research and a critical care clinician at Nationwide
    Children’s. Shown here in the Pediatric Intensive Care Unit, (top), Dr. Frazier facilitates patient rounds and discusses clinical
    cases with colleagues.

    Under the mentorship of Dr. Yusen Liu, Dr. Frazier is studying septic shock, a condition he has seen affect many of his critical
    care patients. Using mouse models of sepsis and septic shock, Dr. Frazier measures markers and pathways of inflammation with
    hopes of identifying key molecular interactions that will lead to improved treatment of critically ill children.




4 | The ReseaRch InsTITuTe at nationwide children’s hospital
10:00 a.M.
 MIndInG The MedIa

 Dr. Lara McKenzie, a faculty member in the Center for Injury Research and Policy, prepares for an interview with ABC’s Good
 Morning America regarding her diving injury research. Later this same week, Dr. McKenzie would provide interviews to additional
 media outlets including USA Today and U.S.News & World Report.

 Collectively, the Center for Injury Research and Policy at Nationwide Children’s garners millions of media impressions annually.




10:16 a.M.                                                              10:35 a.M.
execuTIve exchanGe                                                      calIBRaTInG a cRITIcal coRe ResouRce

President of The Research Institute Dr. John Barnard,                   Dave Dunaway calibrates a flow cytometer, a highly sophisti-
(right), meets with Nationwide Children’s Chief Operating               cated instrument used for identifying and sorting cells. Dave
Officer Rick Miller and other hospital administrators during            places colored beads in the flow cytometer to mimic cells and
a facilities meeting. Dr. Barnard regularly meets with                  evaluates the accuracy of the machine’s four lasers by studying
hospital administration to discuss activities at The Research           color graphs produced on his computer monitor.
Institute, including details regarding Research Building III,
                                                                        The Flow Cytometry Core is one of 13 Shared Research Services
soon to break ground.
                                                                        at The Research Institute. Scientists are charged a users fee,
                                                                        creating a cost efficient way to keep state-of-the-art technology
                                                                        available to all the scientists at The Research Institute.

                                                                                       The ReseaRch InsTITuTe at nationwide children’s hospital | 5
10:45 a.M.                                                   11:00 a.M.
  FacIlITaTInG FInance and FundInG                             assIsTInG The assIsTanTs

  Director of Sponsored Projects, Aaron Ufferman,              Roy Goudy of Research Information Services helps Administrative
  works at his desk. Behind him appears a monthly list         Assistant Char Cameron sign onto a training program used to
  of upcoming grant applications for faculty under his         update faculty biographical sketches on The Research Institute’s
  purview. The Finance and Sponsored Projects                  Internet site. The Research Institute’s overhead provides
  Department supports grant and contract funding               outstanding administrative and information technology
  from the start of a search for funding through the           support for its scientists.
  closeout of a project.




    11:15 a.M.                                                 noon
    cRoss-caMpus collaBoRaTIon                                 shuTTlInG BeTween sITes

    Ohio State University residents, Drs. John Novak and W.    In 2007, Nationwide Children’s began a shuttle
    David Arnold, prepare to meet with Dr. Zarife Sahenk in    service to The Ohio State University Medical Center.
    the Center for Gene Therapy, to review muscle biopsies.    An average of 80 students, faculty and staff members
    Like most research faculty members, Dr. Sahenk works       ride the shuttle each day. The shuttle service is one of
    closely with colleagues at The Ohio State University       many services and programs designed to enhance
    Medical Center and serves as a professor in the OSU        interaction between the campuses.
    College of Medicine.



6 | The ReseaRch InsTITuTe at nationwide children’s hospital
12:14 p.M.
seMInaR seRIes

Dr. Kurt Albertine of the University of Utah Health Sciences Center, presents his research on bronchopulmonary dysplasia at a
seminar in the Research Building II amphitheater. The Research Institute holds such seminars twice a week, featuring visiting
scientists from all over the world.




                                                                        12:40 p.M.
                                                                        couch-sIde collaBoRaTIve

                                                                        Research associates Adam Nation and Michele
                                                                        Patak discuss plans for a research project exploring
                                                                        the relationship between safety practices and
                                                                        impulsivity, a collaborative effort between the Center
                                                                        for Biobehavioral Health and the Center for Injury
                                                                        Research and Policy. Adam and Michele work under
                                                                        the supervision of Dr. Lara McKenzie, a safety expert,
                                                                        and Dr. Brady Reynolds, a psychologist with expertise
                                                                        in impulsive behavior. The project is funded by the
                                                                        Centers for Disease Control and Prevention. At The
                                                                        Research Institute, collaboration between research
                                                                        centers is commonplace.




                                                                                    The ReseaRch InsTITuTe at nationwide children’s hospital | 7
1:10 p.M.
    a sTudenT’s schedule

    Chelsea Bolyard injects a drug into cultured cancer cells to test an alternative gene therapy treatment for ovarian cancer. As a
    third-year graduate student at The Ohio State University and a member of the Center for Gene Therapy, Chelsea divides her time
    between Dr. Jeff Bartlett’s lab at Nationwide Children’s and classes on the main OSU campus. Annually, more than fifty graduate
    students from OSU receive training at The Research Institute.




8 | The ReseaRch InsTITuTe at nationwide children’s hospital
2:06 p.M.
                    coMMuTInG FoR
                  clInIcal ReseaRch

  12-year-old Caroline Brendsel begins
    a study visit with Clinical Research
   Coordinator Kassi Speakman in the
  Clinical Studies Center. Caroline has
         been participating in a research
       trial for hereditary angiodema, a
        rare genetic disorder that causes
    intermittent severe tissue swelling,
  sometimes in vital areas of the body.
As part of the trial, the test medication
 is delivered via a central venous line.

         Although the initial portion of
 the phase III clinical trial is complete,
Caroline receives the medication under
  an open label protocol. Caroline and
         her mother faithfully make the
     100-mile round trip from south of
         Lancaster, Ohio to Nationwide
  Children’s two to three times a week
      as they have done for three years.

   Hundreds of other children visit the
    Clinical Studies Center annually as
    participants in dozens of local and
        national clinical research trials.




                                             The ReseaRch InsTITuTe at nationwide children’s hospital | 9
3:00 p.M.
 scRuTInIzInG huMan ReseaRch sTudIes

 Members of the Institutional Review Board (IRB) at Nationwide Children’s meet twice a month on Tuesday afternoon. This 19-member
 board provides critical oversight of all research involving human subjects at Nationwide Children’s and its affiliated institutions. The IRB
 has the authority to approve, require modifications in, or disapprove all research activities that fall within its jurisdiction. This function is
 federally-mandated and regulated by the Department of Health and Human Services and the Food and Drug Administration. The
 Nationwide Children’s IRB was among the first of such committees in the United States to be fully electronic.




                                                                                                                   3:32 p.M.
                                                                                                                   a pReMIeR pRoGRaM
                                                                                                                   FoR oTITIs MedIa
                                                                                                                   pRevenTIon

                                                                                                                   Laura Novotny, senior
                                                                                                                   research associate in the
                                                                                                                   laboratory of Dr. Lauren
                                                                                                                   Bakaletz, checks a bacterial
                                                                                                                   culture to make sure it is
                                                                                                                   sufficient for her experi-
                                                                                                                   ment, which will test how
                                                                                                                   well vaccine candidates
                                                                                                                   block bacterial adherence.

                                                                                                                   As part of the Center for
                                                                                                                   Microbial Pathogenesis,
                                                                                                                   the team led by Dr.
                                                                                                                   Bakaletz is an international
                                                                                                                   leader in understanding
                                                                                                                   immune mechanisms in
                                                                                                                   the middle ear. The group
                                                                                                                   is working on a promis-
                                                                                                                   ing vaccine to prevent
                                                                                                                   ear infections in children,
                                                                                                                   one of the most common
                                                                                                                   childhood illnesses.




10 | The ReseaRch InsTITuTe at nationwide children’s hospital
4:07 p.M.
        InTeRnaTIonal InvesTIGaToR

        Postdoctoral fellow Mandar Joshi in
        the Center for Cardiovascular and
        Pulmonary Research processes human
        and animal cardiac tissue samples by
        “sealing” them in paraffin wax and
        placing them in cassettes for use in later
        research. Clinical cardiac tissue samples
        are obtained through partnerships with
        The Heart Center at Nationwide Children’s
        and The Ohio State University.

        As an international employee, Dr. Joshi
        is on a visa from India. International
        employees on visas account for about 10
        percent of The Research Institute’s total
        employees and come from 24 countries.




The ReseaRch InsTITuTe at nationwide children’s hospital | 11
4:28 p.M.
 leadInG MusculaR dYsTRophY ReseaRch

 Research Assistant Danielle Tucker prepares a muscle biopsy extracted from the left deltoid of a patient with limb-girdle muscular
 dystrophy being evaluated at the Nationwide Children’s Hospital Close To HomeSM Center in Westerville, Ohio.

 After the sample is frozen and stained, researchers will examine it to determine whether or not the patient could be a candidate
 for gene therapy trials to treat limb girdle muscular dystrophy type 2D.

 The Mendell Lab is part of the Center for Gene Therapy and is an international leader in muscular dystrophy research. Dr. Jerry
 Mendell’s group initiated the first U.S. human gene therapy trial directed at Duchenne muscular dystrophy in children and the
 first national muscular newborn screening study.



                                                                            4:44 p.M.
                                                                            unITInG scIence and TechnoloGY

                                                                            Dr. Richard Ransom, principal investigator in the Center
                                                                            for Clinical and Translational Research, inspects an image
                                                                            of a specialized kidney cell known as a podocyte.

                                                                            Using the graphics editing program Adobe® Photoshop®,
                                                                            Dr. Ransom is able to stack images of the same cell using
                                                                            different fluorescence filters. This allows him to superim-
                                                                            pose images for comparison, in this case, images labeling
                                                                            the actin filaments that make up the cell’s “skeleton”
                                                                            (shown in green), and the heat shock proteins (shown in
                                                                            red) that are expressed when cells are exposed to stress.

                                                                            Dr. Ransom uses this method to determine whether actin
                                                                            filaments and heat shock proteins are found in the same
                                                                            structures in the cell, since the areas where they co-localize
                                                                            are yellow in the superimposed image. This examination is
                                                                            a first step in studies aimed at treating nephrotic syndrome,
                                                                            one of the leading reasons for kidney disease in children.

12 | The ReseaRch InsTITuTe at nationwide children’s hospital
8:54 p.M.
ReseaRch aFTeR daRK

As darkness falls, Postdoctoral Fellow Benoit Callendret continues his work. At The Research Institute, scientific discovery often
continues late into the evening.

This evening, Dr. Callendret uses the flow cytometer to evaluate a liver biopsy that arrived at 8:30 a.m. He is quantifying the
frequency of hepatitis C virus-specific T cells in order to develop a baseline for an upcoming study. The study aims to assess
whether or not identified hepatitis C virus-specific T cells are functional in the context of chronic infection. This research will
be the first of its kind to be performed directly in vivo.

Dr. Callendret is part of Dr. Chris Walker’s team in the Center for Vaccines and Immunity. Dr. Walker is an international leader
in hepatitis C virus research.
                                                                                         The ReseaRch InsTITuTe at nationwide children’s hospital | 13
2221




                                   nonPRofIT oRG.

                                    u.s. PosTAGe

                                       pa I d
                                   coLuMbus, oh

       700 children’s Drive        PeRMIT no. 777
       columbus, ohio 43205-2696

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reSearch Magazine Issue 3

  • 1. research A P u b L Ic AT I o n o f T he R e s ea Rc h I ns T I T u T e aT n aT I o nwI d e c hI l dRe n’s ho sp I Tal A Day in the Life of The ReseARch InsTITuTe
  • 2. 7:30 a.M. daYBReaK A crescent moon appears above Research Building II as dawn breaks on a beautiful Ohio Tuesday morning.
  • 3. fALL / WI nTeR 2008 research is dedicated to the mission of informing and inspiring Twenty-four hours from now, Nationwide Children’s Hospital readers by highlighting scientific will not be the same hospital it is at this moment. Knowledge performance at The Research Institute will grow. Possibilities will emerge. Enthusiasm will escalate. at nationwide children’s hospital. Lives will be changed through discovery. This publication is produced biannually by the Marketing and Public Relations Department at As the research engine of Nationwide Children’s, The Research nationwide children’s hospital. Institute is a key contributor to this daily transformation. Each day begins with a sense of excitement and mission. Innovative experiments are conceived and carried out by our research teams, data are collected and careful analyses are conducted. Data are collaboratively shared among faculty, staff and students. We strive for exciting conclusions, but sometimes more questions arise than answers. We persevere, stubbornly emboldened by the l e a d e Rs hI p potential that scientific inquiry will lead to better health for The Research Institute at nationwide children’s hospital children and their families. The beat goes on. John a. Barnard, Md President In this issue of reSearch, we provide you with snapshots of a reSearch lauren o. Bakaletz, phd Vice President, Basic Sciences Research “typical Tuesday” in a day in the life of The Research Institute. Kelly Kelleher, Md, Mph Through this photographic timeline, we hope you will appreciate Vice President, Health Services Research william e. smoyer, Md the heart and soul of one of the top pediatric research centers in Vice President, Clinical and Translational Research the United States. Grant Morrow III, Md Medical Director daniel R. Mann Vice President, Research Administration and Operations Katherine s. Milem Vice President, Research Business Services research John Barnard, MD Writer and editor Melissa hamilton President The Research Institute at Nationwide Children’s Hospital Art Directors John ordaz Professor of Pediatrics Tanya Burgess Bender The Ohio State University College of Medicine Photographers Brad smith dan smith Manager, Research communications Jan arthur contact us at ResearchMagazine@nationwidechildrens.org The ReseaRch InsTITuTe at nationwide children’s hospital | 1
  • 4. 8:03 a.M. FocusInG on FIRsT-RaTe FacIlITIes Phil Bowers helps Mary Connell from Children’s Hospital of Philadelphia pour off excess liquid nitrogen from shipping containers holding tissue samples that are being sent to Boston. As facilities manager at The Research Institute, Bowers begins his work day at 6 a.m. and spends his early hours walking through and “waking up” the research buildings. Bowers checks for alarms, notes equipment problems and performs safety checks. By the time he completes his rounds he will have covered every floor in the 300,000 square feet of research space, trekking through more than 90 labs. 2 | The ReseaRch InsTITuTe at nationwide children’s hospital
  • 5. 9:18 a.M. Fedex FRenzY This Tuesday is like every other weekday in the Biopathology Center. Staff members receive between 20 and 40 FedEx packages containing pediatric and adult cancer samples from hospitals all across North America. After the samples are unpacked, they are entered into a detailed, secure database. Tissue samples are prepared for digital microscopy and shared with other national experts using a virtual microscope. Some samples are carefully catalogued for storage in a liquid nitrogen freezer, while others undergo clinical testing to help make vital cancer treatment decisions. The Biopathology Center is funded by the National Cancer Institute and serves as the data repository for cancer samples from more than 500 hospitals. Specimens from this Center are used for cancer research in institutions worldwide. Because of the Biopathology Center, The Research Institute at Nationwide Children’s Hospital receives more grant funds from the Children’s Oncology Group than any other national program. The ReseaRch InsTITuTe at nationwide children’s hospital | 3
  • 6. 9:24 a.M. FRoM BedsIde To Bench and BacK aGaIn Dr. W. Joshua Frazier is both a researcher in the Center for Perinatal Research and a critical care clinician at Nationwide Children’s. Shown here in the Pediatric Intensive Care Unit, (top), Dr. Frazier facilitates patient rounds and discusses clinical cases with colleagues. Under the mentorship of Dr. Yusen Liu, Dr. Frazier is studying septic shock, a condition he has seen affect many of his critical care patients. Using mouse models of sepsis and septic shock, Dr. Frazier measures markers and pathways of inflammation with hopes of identifying key molecular interactions that will lead to improved treatment of critically ill children. 4 | The ReseaRch InsTITuTe at nationwide children’s hospital
  • 7. 10:00 a.M. MIndInG The MedIa Dr. Lara McKenzie, a faculty member in the Center for Injury Research and Policy, prepares for an interview with ABC’s Good Morning America regarding her diving injury research. Later this same week, Dr. McKenzie would provide interviews to additional media outlets including USA Today and U.S.News & World Report. Collectively, the Center for Injury Research and Policy at Nationwide Children’s garners millions of media impressions annually. 10:16 a.M. 10:35 a.M. execuTIve exchanGe calIBRaTInG a cRITIcal coRe ResouRce President of The Research Institute Dr. John Barnard, Dave Dunaway calibrates a flow cytometer, a highly sophisti- (right), meets with Nationwide Children’s Chief Operating cated instrument used for identifying and sorting cells. Dave Officer Rick Miller and other hospital administrators during places colored beads in the flow cytometer to mimic cells and a facilities meeting. Dr. Barnard regularly meets with evaluates the accuracy of the machine’s four lasers by studying hospital administration to discuss activities at The Research color graphs produced on his computer monitor. Institute, including details regarding Research Building III, The Flow Cytometry Core is one of 13 Shared Research Services soon to break ground. at The Research Institute. Scientists are charged a users fee, creating a cost efficient way to keep state-of-the-art technology available to all the scientists at The Research Institute. The ReseaRch InsTITuTe at nationwide children’s hospital | 5
  • 8. 10:45 a.M. 11:00 a.M. FacIlITaTInG FInance and FundInG assIsTInG The assIsTanTs Director of Sponsored Projects, Aaron Ufferman, Roy Goudy of Research Information Services helps Administrative works at his desk. Behind him appears a monthly list Assistant Char Cameron sign onto a training program used to of upcoming grant applications for faculty under his update faculty biographical sketches on The Research Institute’s purview. The Finance and Sponsored Projects Internet site. The Research Institute’s overhead provides Department supports grant and contract funding outstanding administrative and information technology from the start of a search for funding through the support for its scientists. closeout of a project. 11:15 a.M. noon cRoss-caMpus collaBoRaTIon shuTTlInG BeTween sITes Ohio State University residents, Drs. John Novak and W. In 2007, Nationwide Children’s began a shuttle David Arnold, prepare to meet with Dr. Zarife Sahenk in service to The Ohio State University Medical Center. the Center for Gene Therapy, to review muscle biopsies. An average of 80 students, faculty and staff members Like most research faculty members, Dr. Sahenk works ride the shuttle each day. The shuttle service is one of closely with colleagues at The Ohio State University many services and programs designed to enhance Medical Center and serves as a professor in the OSU interaction between the campuses. College of Medicine. 6 | The ReseaRch InsTITuTe at nationwide children’s hospital
  • 9. 12:14 p.M. seMInaR seRIes Dr. Kurt Albertine of the University of Utah Health Sciences Center, presents his research on bronchopulmonary dysplasia at a seminar in the Research Building II amphitheater. The Research Institute holds such seminars twice a week, featuring visiting scientists from all over the world. 12:40 p.M. couch-sIde collaBoRaTIve Research associates Adam Nation and Michele Patak discuss plans for a research project exploring the relationship between safety practices and impulsivity, a collaborative effort between the Center for Biobehavioral Health and the Center for Injury Research and Policy. Adam and Michele work under the supervision of Dr. Lara McKenzie, a safety expert, and Dr. Brady Reynolds, a psychologist with expertise in impulsive behavior. The project is funded by the Centers for Disease Control and Prevention. At The Research Institute, collaboration between research centers is commonplace. The ReseaRch InsTITuTe at nationwide children’s hospital | 7
  • 10. 1:10 p.M. a sTudenT’s schedule Chelsea Bolyard injects a drug into cultured cancer cells to test an alternative gene therapy treatment for ovarian cancer. As a third-year graduate student at The Ohio State University and a member of the Center for Gene Therapy, Chelsea divides her time between Dr. Jeff Bartlett’s lab at Nationwide Children’s and classes on the main OSU campus. Annually, more than fifty graduate students from OSU receive training at The Research Institute. 8 | The ReseaRch InsTITuTe at nationwide children’s hospital
  • 11. 2:06 p.M. coMMuTInG FoR clInIcal ReseaRch 12-year-old Caroline Brendsel begins a study visit with Clinical Research Coordinator Kassi Speakman in the Clinical Studies Center. Caroline has been participating in a research trial for hereditary angiodema, a rare genetic disorder that causes intermittent severe tissue swelling, sometimes in vital areas of the body. As part of the trial, the test medication is delivered via a central venous line. Although the initial portion of the phase III clinical trial is complete, Caroline receives the medication under an open label protocol. Caroline and her mother faithfully make the 100-mile round trip from south of Lancaster, Ohio to Nationwide Children’s two to three times a week as they have done for three years. Hundreds of other children visit the Clinical Studies Center annually as participants in dozens of local and national clinical research trials. The ReseaRch InsTITuTe at nationwide children’s hospital | 9
  • 12. 3:00 p.M. scRuTInIzInG huMan ReseaRch sTudIes Members of the Institutional Review Board (IRB) at Nationwide Children’s meet twice a month on Tuesday afternoon. This 19-member board provides critical oversight of all research involving human subjects at Nationwide Children’s and its affiliated institutions. The IRB has the authority to approve, require modifications in, or disapprove all research activities that fall within its jurisdiction. This function is federally-mandated and regulated by the Department of Health and Human Services and the Food and Drug Administration. The Nationwide Children’s IRB was among the first of such committees in the United States to be fully electronic. 3:32 p.M. a pReMIeR pRoGRaM FoR oTITIs MedIa pRevenTIon Laura Novotny, senior research associate in the laboratory of Dr. Lauren Bakaletz, checks a bacterial culture to make sure it is sufficient for her experi- ment, which will test how well vaccine candidates block bacterial adherence. As part of the Center for Microbial Pathogenesis, the team led by Dr. Bakaletz is an international leader in understanding immune mechanisms in the middle ear. The group is working on a promis- ing vaccine to prevent ear infections in children, one of the most common childhood illnesses. 10 | The ReseaRch InsTITuTe at nationwide children’s hospital
  • 13. 4:07 p.M. InTeRnaTIonal InvesTIGaToR Postdoctoral fellow Mandar Joshi in the Center for Cardiovascular and Pulmonary Research processes human and animal cardiac tissue samples by “sealing” them in paraffin wax and placing them in cassettes for use in later research. Clinical cardiac tissue samples are obtained through partnerships with The Heart Center at Nationwide Children’s and The Ohio State University. As an international employee, Dr. Joshi is on a visa from India. International employees on visas account for about 10 percent of The Research Institute’s total employees and come from 24 countries. The ReseaRch InsTITuTe at nationwide children’s hospital | 11
  • 14. 4:28 p.M. leadInG MusculaR dYsTRophY ReseaRch Research Assistant Danielle Tucker prepares a muscle biopsy extracted from the left deltoid of a patient with limb-girdle muscular dystrophy being evaluated at the Nationwide Children’s Hospital Close To HomeSM Center in Westerville, Ohio. After the sample is frozen and stained, researchers will examine it to determine whether or not the patient could be a candidate for gene therapy trials to treat limb girdle muscular dystrophy type 2D. The Mendell Lab is part of the Center for Gene Therapy and is an international leader in muscular dystrophy research. Dr. Jerry Mendell’s group initiated the first U.S. human gene therapy trial directed at Duchenne muscular dystrophy in children and the first national muscular newborn screening study. 4:44 p.M. unITInG scIence and TechnoloGY Dr. Richard Ransom, principal investigator in the Center for Clinical and Translational Research, inspects an image of a specialized kidney cell known as a podocyte. Using the graphics editing program Adobe® Photoshop®, Dr. Ransom is able to stack images of the same cell using different fluorescence filters. This allows him to superim- pose images for comparison, in this case, images labeling the actin filaments that make up the cell’s “skeleton” (shown in green), and the heat shock proteins (shown in red) that are expressed when cells are exposed to stress. Dr. Ransom uses this method to determine whether actin filaments and heat shock proteins are found in the same structures in the cell, since the areas where they co-localize are yellow in the superimposed image. This examination is a first step in studies aimed at treating nephrotic syndrome, one of the leading reasons for kidney disease in children. 12 | The ReseaRch InsTITuTe at nationwide children’s hospital
  • 15. 8:54 p.M. ReseaRch aFTeR daRK As darkness falls, Postdoctoral Fellow Benoit Callendret continues his work. At The Research Institute, scientific discovery often continues late into the evening. This evening, Dr. Callendret uses the flow cytometer to evaluate a liver biopsy that arrived at 8:30 a.m. He is quantifying the frequency of hepatitis C virus-specific T cells in order to develop a baseline for an upcoming study. The study aims to assess whether or not identified hepatitis C virus-specific T cells are functional in the context of chronic infection. This research will be the first of its kind to be performed directly in vivo. Dr. Callendret is part of Dr. Chris Walker’s team in the Center for Vaccines and Immunity. Dr. Walker is an international leader in hepatitis C virus research. The ReseaRch InsTITuTe at nationwide children’s hospital | 13
  • 16. 2221 nonPRofIT oRG. u.s. PosTAGe pa I d coLuMbus, oh 700 children’s Drive PeRMIT no. 777 columbus, ohio 43205-2696