MDA RESEARCH AT YEAR 2000
Moving From Lab to Clinic

by Margaret Wahl

< < Story continued from previous screen

[illustration: mouse, stem cells, injection process]
MAKING NEW MUSCLE
Stem cells, removed from either the muscle or bone marrow of a healthy donor, are injected into the bloodstream of a mouse that lacks dystrophin. In response to injury signals (green arrow) from the muscle, the dystrophin-containing stem cells gradually migrate (blue arrows) out of the bloodstream and into all of the muscles. They may have to spend time in the bone marrow first. To make the process safer and more efficient, researchers hope to learn what signals from the muscle are required to attract the stem cells.

Stem cells. Stem cells are primitive cells that can develop into many differ-ent tissues, including muscle. Some years ago, muscle biologists were trying to get another form of immature muscle cell, known as the myoblast, to enter dystrophic muscles and repair the damage. After several years of trying to accomplish this, including clinical studies in children, the researchers concluded that myoblasts were, for the most part, unable to migrate into existing muscle tissue, survive there or repair the muscle.

Part of the reason may have been that the myoblasts the scientists were working with were too mature in their development.

Now, experts funded by MDA say they've isolated a population of stem cells from both bone marrow and muscle tissue that is far less mature and shows far more potential for migrating into muscle and doing the necessary repairs. Using such cells, either from donors with normal muscle genes, or from patients, after the cells have undergone gene transfer to correct the genetic defect, is a top priority for MDA at year 2000. .

Open Clinical Trials & Studies

Duchenne MD Glutamine and Creatine Trial
Age 5 to 9; ambulatory; no steroids
Diana M. Escolar
Washington, D.C.
(202) 884-6080

Duchenne MD Gentamicin Study
Trial Coordinator
Columbus, Ohio
(614) 293-9016

Duchenne MD Trial of Anabolic Steroid (Oxandrolone)
5 to 10 years old; no steroids
Shree Pandya
Rochester, N.Y.
(716) 275-1005

Charlie Harper
St. Louis
(314) 362-6981

Jerry Mendell
Columbus, Ohio
(614) 293-4962

Duchenne/Becker MD Learning Styles
6 to 16 years old
Veronica Hinton
New York
(212) 305-2512

Emery-Dreifuss MD Diagnostic Evaluation and Natural History
Alan Pestronk
St. Louis
(314) 362-6981

FSHMD Albuterol Trial
18 to 60 years old
Karen Downing
Columbus, Ohio
(614) 292-1234

Lynn Cos
Rochester, N.Y.
(716) 275-7680

FSHMD Tissue Bank
No history of bleeding; no blood thinners
Lynn Cos
Rochester, N.Y.
(716) 275-7680

Myotonic MD; FSHMD; CMT; Proximal Myotonic Myopathy Altered Metabolism and Muscle Wasting
21 to 60 years old, ambulatory
Cheryl Barbieri
Rochester, N.Y.
(716) 275-5409

Limb-Girdle MD & Paget Disease of Bone Characterization, Gene Identification & Treatment
Virginia Kimonis
Springfield, Ill.
(217) 782-4839

Myotonic MD Cardiology Study
Adults and families
William J. Groh
Indianapolis
(317) 630-6629

ALS Biological Markers in Cerebrospinal Fluid
Merit E. Cudkowicz
Boston
(617) 724-1873

ALS BDNF Safety and Efficacy Study
Regeneron Pharmaceuticals Inc.
Tarrytown, N.Y.
(914) 345-7400

Jason Mass
San Francisco
(415) 476-7581

Lisa Marello
San Francisco
(415) 923-3963

Linda Sepulveda
Los Angeles
(310) 825-9816

Massoud Shamel
La Jolla, Calif.
(619) 455-5463

Kristin Howell
Denver
(303) 315-7046

Joseph Choi
Washington, D.C.
(202) 994-5359

Julie Steele
Miami
(305) 243-7526

Meraida Polak
Atlanta
(404) 778-3754

Lauren Bradbury
Boston
(617) 726-8741

Charlie Harper
St. Louis
(314) 362-6981

Maura Del Bene
New York
(212) 305-1319

Carrie Luteran Bagley
Syracuse, N.Y.
(315) 464-5301

Jennifer Kuyl
Portland, Ore.
(503) 494-4987

Terry Paylor
Philadelphia
(215) 762-5186

Suzanne M. Holbach
Pittsburgh, Pa.
(412) 692-4441

Kimberly Hardin
Dallas
(214) 648-8668

Donna Baum
Salt Lake City
(801) 581-4944

Bryn Floyd
Seattle
(206) 598-3797

Janine Diana
Madison, Wis.
(608) 265-2451

 Details about all the studies listed here are available in the Research: Active Clinical Trials section of this Web site.

ALS Trial of Creatine
Had disease less than five years
Jeffrey D. Rothstein
Baltimore
(410) 614-5972

Merit Cudkowicz
Boston
(617) 724-1873

Carrie Luteran Bagley
Syracuse, N.Y.
(315) 464-5301

ALS Study of CoQ10
Maura Del Bene
New York
(212) 305-1319

ALS Topiramate Trial
Paula Sexton
Boston
(617) 724-1872

ALS Sanofi Drug Trial
No Myotrophin, BDNF, GDNF
E. Peter Bosch
Scottsdale, Ariz.
(602) 301-7583

Richard Smith
San Diego
(619) 455-5463

Robert G. Miller
San Francisco
(415) 923-3604

Michael C. Graves
Los Angeles
(310) 825-9816

Hans Neville
Denver
(303) 315-7046

Kevin Felice
Farmington, Conn.
(860) 679-4837

Walter Bradley
Miami
(305) 243-7526

Jonathan Glass
Atlanta
(404) 727-3507

Robert L. Sufit
Chicago
(312) 908-0774

Jeffrey D. Rothstein
Baltimore
(410) 955-6435

Alan Pestronk
St. Louis
(314) 362-6981

Raul N. Mandler
Albuquerque, N.M.
(505) 272-3342

Maura Del Bene
New York
(212) 305-1319

Jeremy Shefner
Syracuse, N.Y.
(315) 464-5358

Peter D. Donofrio
Winston-Salem, N.C.
(910) 716-9056

Jerry R. Mendell
Columbus, Ohio
(614) 292-1234

Terry Heiman-Patterson
Philadelphia
(215) 762-7635

Yadollah Harati
Houston
(713) 798-5993

Rup Tandan
Burlington, Vt.
(802) 656-4177

Benjamin R. Brooks
Madison, Wis.
(608) 263-0170

Identification of Predisposing Genetic Factors in ALS
Nailah Siddique
Chicago
(312) 503-2712

Diane McKenna-Yasek
Boston
(617) 726-5750

Juvenile Dermatomyositis and Polymyositis—Immunologic Pathogenesis
Muscle or skin biopsies and calcinosis specimens; 18 and younger
Lisa Rider
Bethesda, Md.
(301) 827-0460

Etanercept (Enbrel) in Dermatomyositis
18 to 60 years old
Mary Harrigan
Phoenix
(602) 406-6651

Calcinosis Lesions (Calcium Deposits) in Myositis
Samples of calcinosis lesions
Lisa G. Rider
Bethesda, Md.
(301) 827-0679

Gene Mutations in Families With CMT and Deafness
Virginia Kimonis
Springfield, Ill.
(217) 782-4839

Enzyme Replacement Therapy for Pompe's Disease
Less than age 1
Jennifer Sullivan
Durham, N.C.
(919) 684-2036

Dichloroacetate Treatment in MELAS (a Mitochondrial Myopathy)
Jean Stewart
San Diego
(888) 367-6476