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2. SMALL MOLECULES: THE PAST OR THE FUTURE IN DRUG INNOVATION?

  • Anne Robert , Françoise Benoit-Vical , Yan Liu and Bernard Meunier
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Essential Metals in Medicine: Therapeutic Use and Toxicity of Metal Ions in the Clinic
This chapter is in the book Essential Metals in Medicine: Therapeutic Use and Toxicity of Metal Ions in the Clinic

Abstract

With the impressive development of molecular life sciences, one may have the feeling that biopharmaceuticals will dominate the world of drug design and production. This is partly due to the evolution of pharmaceutical industry, especially since the 1980s. As a matter of fact, small molecules are still dominating the field of drug innovation, in contradiction with claims predicting their downfall and the exponential raise of biopharmaceuticals. The strong association of chemistry with biochemistry and pharmacology has been the scientific base of the establishment and the success of strong powerful pharmaceutical companies throughout the twentieth century. To meet the needs of new therapeutic agents, it is necessary to assess the role and future position of medicinal chemistry. In fact, the reasonable balance between small molecules and biopharmaceuticals will depend on scientific and economic factors, including the goal of having highly efficient drugs to cure the largest possible number of patients, at a cost that is compatible with the limits of national health budgets. In the present chapter, we would like to emphasize the future important role of small molecules based on new chemicals, to build a new portfolio of efficient, safe and affordable drugs to solve major therapeutic challenges. Two examples are then given. In the blood parasitic diseases such as malaria and schistosomiasis, the iron of heme is an “old” and relevant therapeutic target to kill the parasite. Investigations on the mechanism of action of the antimalarial endoperoxide sesquiterpene artemisinin, have paved the way to the design of new efficient synthetic endoperoxide drugs. In the case of Alzheimer’s disease, the loss of copper homeostasis in patient brain is one of the key features of neurodegeneration. The development of small copper specific ligands able to retrieve copper from its pathological sinks to reintroduce it into physiological circulation is a challenging but promising approach to effective therapy.

Abstract

With the impressive development of molecular life sciences, one may have the feeling that biopharmaceuticals will dominate the world of drug design and production. This is partly due to the evolution of pharmaceutical industry, especially since the 1980s. As a matter of fact, small molecules are still dominating the field of drug innovation, in contradiction with claims predicting their downfall and the exponential raise of biopharmaceuticals. The strong association of chemistry with biochemistry and pharmacology has been the scientific base of the establishment and the success of strong powerful pharmaceutical companies throughout the twentieth century. To meet the needs of new therapeutic agents, it is necessary to assess the role and future position of medicinal chemistry. In fact, the reasonable balance between small molecules and biopharmaceuticals will depend on scientific and economic factors, including the goal of having highly efficient drugs to cure the largest possible number of patients, at a cost that is compatible with the limits of national health budgets. In the present chapter, we would like to emphasize the future important role of small molecules based on new chemicals, to build a new portfolio of efficient, safe and affordable drugs to solve major therapeutic challenges. Two examples are then given. In the blood parasitic diseases such as malaria and schistosomiasis, the iron of heme is an “old” and relevant therapeutic target to kill the parasite. Investigations on the mechanism of action of the antimalarial endoperoxide sesquiterpene artemisinin, have paved the way to the design of new efficient synthetic endoperoxide drugs. In the case of Alzheimer’s disease, the loss of copper homeostasis in patient brain is one of the key features of neurodegeneration. The development of small copper specific ligands able to retrieve copper from its pathological sinks to reintroduce it into physiological circulation is a challenging but promising approach to effective therapy.

Chapters in this book

  1. Frontmatter i
  2. About the Editors v
  3. Historical Development and Perspectives of the Series vii
  4. Preface to Volume 19 ix
  5. Contents xiii
  6. Contributors to Volume 19 xix
  7. Titles of Volumes 1–44 in the Metal Ions in Biological Systems Series xxiii
  8. Contents of Volumes in the Metal Ions in Life Sciences Series xxv
  9. 1. METALS IN MEDICINE: THE THERAPEUTIC USE OF METAL IONS IN THE CLINIC 1
  10. 2. SMALL MOLECULES: THE PAST OR THE FUTURE IN DRUG INNOVATION? 17
  11. 3. IRON CHELATION FOR IRON OVERLOAD IN THALASSEMIA 49
  12. 4. IRONING OUT THE BRAIN 87
  13. 5. INFECTIONS ASSOCIATED WITH IRON ADMINISTRATION 123
  14. 6. IRON OXIDE NANOPARTICLE FORMULATIONS FOR SUPPLEMENTATION 157
  15. 7. BUILDING A TROJAN HORSE: SIDEROPHORE-DRUG CONJUGATES FOR THE TREATMENT OF INFECTIOUS DISEASES 181
  16. 8. DEVELOPING VANADIUM AS AN ANTIDIABETIC OR ANTICANCER DRUG: A CLINICAL AND HISTORICAL PERSPECTIVE 203
  17. 9. CHROMIUM SUPPLEMENTATION IN HUMAN HEALTH, METABOLIC SYNDROME, AND DIABETES 231
  18. 10. MANGANESE: ITS ROLE IN DISEASE AND HEALTH 253
  19. 11. COBALT-SCHIFF BASE COMPLEXES: PRECLINICAL RESEARCH AND POTENTIAL THERAPEUTIC USES 267
  20. 12. COPPER DEPLETION AS A THERAPEUTIC STRATEGY IN CANCER 303
  21. 13. METAL COMPOUNDS IN THE DEVELOPMENT OF ANTIPARASITIC AGENTS: RATIONAL DESIGN FROM BASIC CHEMISTRY TO THE CLINIC 331
  22. 14. CHEMICAL AND CLINICAL ASPECTS OF METAL-CONTAINING ANTIDOTES FOR POISONING BY CYANIDE 359
  23. SUBJECT INDEX 393
https://doi.org/10.1515/9783110527872-002

Chapters in this book

  1. Frontmatter i
  2. About the Editors v
  3. Historical Development and Perspectives of the Series vii
  4. Preface to Volume 19 ix
  5. Contents xiii
  6. Contributors to Volume 19 xix
  7. Titles of Volumes 1–44 in the Metal Ions in Biological Systems Series xxiii
  8. Contents of Volumes in the Metal Ions in Life Sciences Series xxv
  9. 1. METALS IN MEDICINE: THE THERAPEUTIC USE OF METAL IONS IN THE CLINIC 1
  10. 2. SMALL MOLECULES: THE PAST OR THE FUTURE IN DRUG INNOVATION? 17
  11. 3. IRON CHELATION FOR IRON OVERLOAD IN THALASSEMIA 49
  12. 4. IRONING OUT THE BRAIN 87
  13. 5. INFECTIONS ASSOCIATED WITH IRON ADMINISTRATION 123
  14. 6. IRON OXIDE NANOPARTICLE FORMULATIONS FOR SUPPLEMENTATION 157
  15. 7. BUILDING A TROJAN HORSE: SIDEROPHORE-DRUG CONJUGATES FOR THE TREATMENT OF INFECTIOUS DISEASES 181
  16. 8. DEVELOPING VANADIUM AS AN ANTIDIABETIC OR ANTICANCER DRUG: A CLINICAL AND HISTORICAL PERSPECTIVE 203
  17. 9. CHROMIUM SUPPLEMENTATION IN HUMAN HEALTH, METABOLIC SYNDROME, AND DIABETES 231
  18. 10. MANGANESE: ITS ROLE IN DISEASE AND HEALTH 253
  19. 11. COBALT-SCHIFF BASE COMPLEXES: PRECLINICAL RESEARCH AND POTENTIAL THERAPEUTIC USES 267
  20. 12. COPPER DEPLETION AS A THERAPEUTIC STRATEGY IN CANCER 303
  21. 13. METAL COMPOUNDS IN THE DEVELOPMENT OF ANTIPARASITIC AGENTS: RATIONAL DESIGN FROM BASIC CHEMISTRY TO THE CLINIC 331
  22. 14. CHEMICAL AND CLINICAL ASPECTS OF METAL-CONTAINING ANTIDOTES FOR POISONING BY CYANIDE 359
  23. SUBJECT INDEX 393
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