**Food and Drug Administration**

**Center for Biologics Evaluation and Research**

**BIOLOGICAL RESPONSE MODIFIERS ADVISORY COMMITTEE**

**Meeting #34, February 28, 2003**

**Holiday Inn, Silver Spring, Maryland**

**[Executive Secretary]{.underline} [Committee Management
Specialist]{.underline}**

Gail Dapolito Rosanna L. Harvey

Scientific Advisors & Consultants Staff Scientific Advisors &
Consultants Staff

Center for Biologics Evaluation and Research Center for Biologics
Evaluation and Research

Food and Drug Administration (HFM-71) Food and Drug Administration
(HFM-71)

1401 Rockville Pike 1401 Rockville Pike

Rockville, MD 20852-1448 Rockville, MD 20852-1448

Phone: (301) 827-1289 Phone: (301) 827-1297

Fax: (301) 827-0294 Fax: (301) 827-0294

E-mail: dapolito@cber.fda.gov E-mail: <harvey@cber.fda.gov>

**[MEMBERS]{.underline}**

**[CHAIR]{.underline}**

Daniel R. Salomon, M.D. 03/31/03 Jonathan S. Allan, D.V.M. 03/31/06

Associate Professor Scientist

Department of Molecular and Experimental Medicine Department of Virology
and Immunology

The Scripps Research Institute Southwest Foundation for Biomedical
Research

10550 N. Torrey Pines Road, MEM 55 7620 N.W. Loop 410 at Military Drive

La Jolla, CA 92037 San Antonio, TX 77030

Bruce R. Blazar, M.D. 03/31/05**^+^** David M. Harlan, M.D. 03/31/06

Professor Head

Division of Pediatric Bone Marrow Transplantation and Autoimmunity
Branch

Transplantation/Oncology National Institute of Diabetes and Digestive

University of Minnesota and Kidney Disease, NIH

420 Delaware Street, SE, Box 109 10 Center Drive, Building 10, Room
11S210

Minneapolis, MN 55455 Bethesda, MD 20892

Katherine A. High, M.D. 03/31/05 Joanne Kurtzberg, M.D. 03/31/04

William H. Bennett Professor of Pediatrics Professor of Pediatrics

University of Pennsylvania Pediatric Bone Marrow and Stem Cell Program

Children\'s Hospital of Philadelphia Duke University Medical Center

310 Abramson Research Center Box 3350

3615 Civic Center Boulevard 2400 Pratt Street

Philadelphia, PA 19104 Durham, NC 27710

**BIOLOGICAL RESPONSE MODIFIERS ADVISORY COMMITTEE**

## Meeting #34, February 28, 2003

**[MEMBERS]{.underline} (Cont\'d)**

Alison F. Lawton**^\*^** 03/31/05 Richard C. Mulligan, Ph.D. 03/31/04

Senior Vice President Mallinckrodt Professor of Genetics

Regulatory Affairs Harvard Medical School

Genzyme Corporation Children\'s Hospital

One Kendall Square Department of Molecular Medicine

Cambridge, MA 02139 320 Longwood Avenue

Boston, MA 02115

Mahendra S. Rao, M.D., Ph.D. 03/31/05 Anastasios A. Tsiatis, Ph.D.
03/31/06

Chief, Stem Cell Biology Section Professor

Laboratory of Neurosciences, 4B17 Department of Statistics

Gerontology Research Center North Carolina State University

National Institute on Aging, NIH Box 8203

5600 Nathan Shock Dr. 2501 Founders Drive

Baltimore, MD 21224 Raleigh, NC 27695

Alice J. Wolfson, J.D. **^\*\*^** 03/31/04 **\*** - Industry
Representative

Bourhis, Wolfson and Schlichtmann **\*\*** - Consumer Representative

1050 Battery Street ^**+**^ - Not Attending

San Francisco, CA 94111 **^++^** - Patient Representative

# TEMPORARY VOTING MEMBERS

Ms. Barbara Ballard**^++^** John Coffin, Ph.D.

President Professor of Molecular Biology

SCID Alliance Department of Molecular Biology and Microbiology

Immune Deficiency Foundation Tufts University School of Medicine

Clifton, VA 20124 136 Harrison Avenue

Boston, MA 02111

Kenneth Cornetta, M.D. John E. French, Ph.D.

Chairman and Professor Group and Discipline Leader

Department of Medical and Molecular Genetics Laboratory of Experimental
Carcinogenesis and

Indiana University Mutagenesis

975 W. Walnut Street, Room 130 National Institute of Environmental
Health Sciences

Indianapolis, IN 46202 National Institutes of Health

> 4401 T.W. Alexander Drive

Research Triangle Park, NC 27709

**BIOLOGICAL RESPONSE MODIFIERS ADVISORY COMMITTEE**

## Meeting #34, February 28, 2003

**[TEMPORARY VOTING MEMBERS]{.underline} (Cont'd)**

Warren Leonard, M.D. Crystal Mackall, M.D.

Chief Head, Immunology Section

Laboratory of Molecular Immunology Pediatric Oncology Branch

National Heart, Lung and Blood Institute National Cancer Institute

National Institutes of Health National Institutes of Health

10 Center Drive, Room 7N252 10 Center Drive, Room 13N240

Bethesda, MD 20892 Bethesda, MD 20892

Abbey S. Meyers Thomas Murray, Ph.D.

President and Executive Director President

National Organization for Rare Disorders The Hastings Center

Fairwood Professional Building 21 Malcolm Gordon Road

P.O. Box 8923 Garrison, NY 10524

New Fairfield, CT 06812

Stuart H. Orkin, M.D. Bruce E. Torbett, Ph.D.

David G. Nathan Professor of Pediatrics Associate Professor

Department of Pediatric Oncology Department of Molecular and

Harvard Medical School Experimental Medicine, MEM 55

Dana Farber Cancer Institute The Scripps Research Institute

44 Binney Street 10550 North Torrey Pines Road

Boston, MA 02115 La Jolla, CA 92037

Linda Wolff, Ph.D.

Chief, Leukemogenesis Section

Laboratory of Cellular Oncology

National Cancer Institute

National Institutes of Health

Building 37, Room 4124A

37 Convent Drive, MSC 4255

Bethesda, MD 20892

**BIOLOGICAL RESPONSE MODIFIERS ADVISORY COMMITTEE**

## Meeting #34, February 28, 2003

**[GUEST SPEAKERS]{.underline}**

Claudio Bordignon, M.D. Marina Cavazzana-Calvo, M.D., Ph.D.

Professor of Hematology Professor in Hematology

Scientific Director Director, Biotherapy Department

Istituto Scientifico HS Raffaele INSERM U429

Via Olgettina 58 Hopital Necker-Enfants Malades

20132 Milan ITALY 149 rue de Sevres

75015 Paris FRANCE

Adrian Thrasher, M.D., Ph.D.

Professor of Paediatric Immunology

Centre for Gene Therapy of Childhood Disease

Molecular Immunology Unit

Institute of Child Health

30 Guilford Street

London WC1N EH

**[NIH REPRESENTATIVES]{.underline}**

Amy Patterson, M.D. Stephen M. Rose, Ph.D.

Director Deputy Director , Recombinant DNA Program

Office of Biotechnology Activities Office of Biotechnology Activities

National Institutes of Health National Institutes of Health

6705 Rockledge Drive 6705 Rockledge Drive

Suite 750, MSC 7985 Suite 750, MSC 7985

Bethesda, MD 20892 Bethesda, MD 20892

# FDA PARTICIPANTS

Philip D. Noguchi, M.D. Raj K. Puri, M.D., Ph.D.

Acting Director Acting Director

Office of Cellular, Tissue and Gene Therapies Division of Cellular and
Gene Therapies, OCTGT

Center for Biologics Evaluation and Research Center for Biologics
Evaluation and Research

Food and Drug Administration Food and Drug Administration

1401 Rockville Pike, HFM-515 1401 Rockville Pike, HFM-500

Rockville, MD 20852 Rockville, MD 20852

Carolyn A.Wilson, Ph.D. Cynthia A. Rask

Senior Investigator Acting Chief

Laboratory of Molecular Tumor Biology Clinical Review Branch

DCGT/OCTGT/CBER Division of Clinical Evaluation and

Food and Drug Administration Pharmacology/Toxicology, OCTGT, CBER

1401 Rockville Pike, HFM-518 1401 Rockville Pike, HFM-576

Rockville, MD 20852 Rockville, MD 20852

**[BRMAC #33 CBER Working Group]{.underline}**

**OCTGT**

Dr. Philip Noguchi

Dr. Joyce Frey-Vasconcells

Dr. Raj Puri

Dr. Carolyn Wilson

Dr. Cynthia Rask

Ms. Mercedes Serabian

Dr. Stephanie Simek

Ms. Tina Moulton

Dr. Daniel Takefman

Dr. Anne Pilaro