Protein Therapeutics: a summary and pharmacological classification

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Protein Therapeutics a summary and
pharmacological classification

• Benjamin Leader, Quentin J. Baca and David E.
  Golan

Yiben Wang 11/16/11
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Protein Therapeutics -25,000-40,000 different
genes in the human genome -Alternative splicing
of genes -Post-translational modifications -Very
high number of functionally distinct
proteins Disease View -Immense
challenge -One protein is mutated or
abnormal -Present in high or low
concentrations Therapeutic View -Tremendous
opportunity 130 different proteins or peptides
FDA approval
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• Advantages of protein therapeutics
• Proteins highly specific and complex set of
  functions
• -Chemical compounds cant mimick
• Specificity
• -Less chance of interference reduce adverse
  effects
• Natural production
• -Well tolerated no immune response
• Diseases gene is deleted or mutated
• -Replacement treatment no gene therapy
  (Unavailable)
• Faster FDA approval time
• -2003 1980-2002 ? 33 therapeutic proteins, 294
  small-molecule drugs (gt1year)
• Unique form and function
• -Companies patent protection

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Functional classification of protein
therapeutics Group I -Protein therapeutics
with enzymatic or regulatory activity Group
II -Protein therapeutics with special targeting
activity Group III -Protein vaccines Group
IV -Protein diagnostics
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Group I Protein therapeutics with enzymatic or
regulatory activity Ia Replacing a protein that
is deficient or abnormal Ib Augmenting an
existing pathway Ic Providing a novel
function or activity
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Group II Protein therapeutics with special
targeting IIa Interfering with a molecule or
organism IIb Delivering other compounds or
proteins
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Group III Protein vaccines IIIa Protecting
against a deleterious foreign agent IIIb
Treating an autoimmune disease IIIc
Treating cancer
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Group IV Protein diagnostics
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• Challenges for protein therapeutics
• Protein solubility, route of administration,
  distribution, and stability.
• Immune response.
• Physiologically active post-translational
  modifications.
• Costs
• 5. Ethics

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Recombinant human proteins -FDA
biotechnology medicines -Monoclonal
antibodies -Natural interferons -Vaccines -Horm
ones -Modified natural enzymes -Cell therapies
More work needs to be done.