Stamulumab
Encyclopedia
Stamulumab is an experimental myostatin
inhibiting drug developed by Wyeth Pharmaceuticals
for the treatment of muscular dystrophy
(MD). Stamulumab was formulated and tested by Wyeth in Collegeville, Pennsylvania
. Myostatin is a protein
that inhibits the growth of muscle tissue, stamulumab is a recombinant
human antibody
designed to bind to and inhibit the activity of myostatin.
Stamulumab is a G1
immunoglobulin antibody which binds to myostatin and prevents it from binding to its target site, thus inhibiting the growth-limiting action of myostatin on muscle tissue. Research completed in 2002 found that Stamulumab might one day prove to be an effective treatment for Duchenne muscular dystrophy
in 2005 and 2006 of stamulumab. The multiple ascending dose trial (36 patients per cohort
) contained some measures of efficacy. The trial's participants included people afflicted with Facioscapulohumeral muscular dystrophy
, Becker's muscular dystrophy
, and Limb-girdle muscular dystrophy
. Through 2007 Wyeth had been analyzing the results but the hoped-for news and/or a publication in 2007 did not occur. In January 24, 2008, Wyeth announced that the study had been accepted by a peer-reviewed journal and publication was expected "in the next few months". The publication appeared in Annals of Neurology
in May 2008.
On 11 March 2008 it was announced that Wyeth would not develop the drug further for MD, but would continue to explore myostatin inhibition along with other strategies.
Myostatin
Myostatin is a secreted TGF beta protein family member that inhibits muscle differentiation and growth. Myostatin is produced primarily in skeletal muscle cells, circulates in the blood and acts on muscle tissue, by binding a cell-bound receptor called the activin type II receptor...
inhibiting drug developed by Wyeth Pharmaceuticals
Wyeth
Wyeth, formerly one of the companies owned by American Home Products Corporation , was a pharmaceutical company. The company was based in Madison, New Jersey, USA...
for the treatment of muscular dystrophy
Muscular dystrophy
Muscular dystrophy is a group of muscle diseases that weaken the musculoskeletal system and hamper locomotion. Muscular dystrophies are characterized by progressive skeletal muscle weakness, defects in muscle proteins, and the death of muscle cells and tissue.In the 1860s, descriptions of boys who...
(MD). Stamulumab was formulated and tested by Wyeth in Collegeville, Pennsylvania
Collegeville, Pennsylvania
Collegeville is a borough in Montgomery County, Pennsylvania, 30 miles northwest of Philadelphia on the Perkiomen Creek. Collegeville was incorporated in 1896. It is the seat of Ursinus College, opened in 1869...
. Myostatin is a protein
Protein
Proteins are biochemical compounds consisting of one or more polypeptides typically folded into a globular or fibrous form, facilitating a biological function. A polypeptide is a single linear polymer chain of amino acids bonded together by peptide bonds between the carboxyl and amino groups of...
that inhibits the growth of muscle tissue, stamulumab is a recombinant
Recombinant DNA
Recombinant DNA molecules are DNA sequences that result from the use of laboratory methods to bring together genetic material from multiple sources, creating sequences that would not otherwise be found in biological organisms...
human antibody
Antibody
An antibody, also known as an immunoglobulin, is a large Y-shaped protein used by the immune system to identify and neutralize foreign objects such as bacteria and viruses. The antibody recognizes a unique part of the foreign target, termed an antigen...
designed to bind to and inhibit the activity of myostatin.
Stamulumab is a G1
G1 phase
The G1 phase is a period in the cell cycle during interphase, before the S phase. For many cells, this phase is the major period of cell growth during its lifespan. During this stage new organelles are being synthesized, so the cell requires both structural proteins and enzymes, resulting in great...
immunoglobulin antibody which binds to myostatin and prevents it from binding to its target site, thus inhibiting the growth-limiting action of myostatin on muscle tissue. Research completed in 2002 found that Stamulumab might one day prove to be an effective treatment for Duchenne muscular dystrophy
Duchenne muscular dystrophy
Duchenne muscular dystrophy is a recessive X-linked form of muscular dystrophy, which results in muscle degeneration, difficulty walking, breathing, and death. The incidence is 1 in 3,000 boys. Females and males are affected, though females are rarely affected and are more often carriers...
Phase 1 and 2 trials
Wyeth undertook a Phase 1 and 2 clinical trialClinical trial
Clinical trials are a set of procedures in medical research and drug development that are conducted to allow safety and efficacy data to be collected for health interventions...
in 2005 and 2006 of stamulumab. The multiple ascending dose trial (36 patients per cohort
Cohort study
A cohort study or panel study is a form of longitudinal study used in medicine, social science, actuarial science, and ecology. It is an analysis of risk factors and follows a group of people who do not have the disease, and uses correlations to determine the absolute risk of subject contraction...
) contained some measures of efficacy. The trial's participants included people afflicted with Facioscapulohumeral muscular dystrophy
Facioscapulohumeral muscular dystrophy
Facioscapulohumeral muscular dystrophy , which is also known as Landouzy-Dejerine, is a usually autosomal dominant inherited form of muscular dystrophy that initially affects the skeletal muscles of the face , scapula and upper arms...
, Becker's muscular dystrophy
Becker's muscular dystrophy
Becker muscular dystrophy is an X-linked recessive inherited disorder characterized by slowly progressive muscle weakness of the legs and pelvis....
, and Limb-girdle muscular dystrophy
Limb-girdle muscular dystrophy
Limb-girdle muscular dystrophy or Erb's muscular dystrophy is an autosomal class of muscular dystrophy that is similar but distinct from Duchenne muscular dystrophy and Becker's muscular dystrophy...
. Through 2007 Wyeth had been analyzing the results but the hoped-for news and/or a publication in 2007 did not occur. In January 24, 2008, Wyeth announced that the study had been accepted by a peer-reviewed journal and publication was expected "in the next few months". The publication appeared in Annals of Neurology
Annals of Neurology
Annals of Neurology is a peer-reviewed medical journal covering neurology....
in May 2008.
On 11 March 2008 it was announced that Wyeth would not develop the drug further for MD, but would continue to explore myostatin inhibition along with other strategies.
See also
- ACVR2BACVR2BActivin receptor type-2B is a protein that in humans is encoded by the ACVR2B gene. ACVR2B is an activin type 2 receptor.-Interactions:ACVR2B has been shown to interact with SYNJ2BP and ACVR1B.-Further reading:...
is similar to stamulumab but is not an antibody; rather, it provides a portion of the molecule to which myostatin would normally bind thus preventing the myostatin from binding with the actual molecule.