Treatment of diabetes mellitus

Drug – bio-affecting and body treating compositions – Designated organic active ingredient containing – Heterocyclic carbon compounds containing a hetero ring...

Reexamination Certificate

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C514S243000, C514S246000, C514S866000

Reexamination Certificate

active

06683080

ABSTRACT:

This invention relates to the use of particular potent and selective cGMP PDE5 inhibitor compounds and especially the compound vardenafil for the treatment of diabetes mellitus.
The revised classification of diabetes mellitus as detailed in Harrisons at Chapter 334: Diabetes Mellitus by J. Larry Jameson contains four major groups as listed below. In general, this classification is based upon the etiology of diabetes as it is currently understood. The term insulin dependant diabetes mellitus (IDDM) has been replaced by Type 1 diabetes. These patients have &bgr;-cell destruction, which is usually immune-mediated; most develop absolute insulin deficiency and are ketosis-prone. The term non-insulin dependant diabetes mellitus (NIDDM) has been replaced by Type 2 diabetes, which encompasses the most prevalent form of the disease. Most patients with Type 2 diabetes exhibit insulin resistance and ultimately develop a concomitant insulin secretory defect. The third class includes genetic defects in insulin secretion and action, diseases of the exocrine pancreas, endocrinopathies that induce hyperglycemia, drug-induced forms of diabetes, infectious causes of diabetes, and genetic syndromes that are associated with an increased incidence of diabetes. Gestational diabetes mellitus (GDM) remains a separate class.
Etiologic Classification of Diabetes Mellitus
I. Type 1 diabetes* &bgr;-cell destructions, usually leading to absolute insulin deficiency: A. Immune-mediated; B. Idiopathic
II. Type 2 diabetes* (may range from predominantly insulin resistance with relative insulin deficiency to a predominantly secretory defect with insulin resistance)
III. Other specific types:
A. Genetic defects of &bgr;-cell function such as: chromosome 12, HNF-1 &agr;-(formerly MODY3); chromosome 7, glucokinase (formerly MODY2); chromosome 20, HNF-4 &agr;-(formerly MODY1); mitochondrial DNA.
B. Genetic defects in insulin action such as: Type A insulin resistance; leprechaunism; Rabson-Mendenhall syndrome; lipoatrophic diabetes.
C. Diseases of the exocrine pancreas such as: pancreatitis; trauma/pancreatectomy; neoplasia; cystic fibrosis; hemochromatosis; fibrocalculous pancreatopathy.
D. Endocrinopathies such as: acromegaly; Cushing's syndrome; glucagonoma; pheochromocytoma; hyperthyroidism; somatostatinoma; aldosteronoma.
E. Drug- or chemical-induced such as: vacor; pentamdine; nicotinic acid; gluocorticoids; thyroid hormone; diazoxide; &bgr;-adrenergic agonists; thiazides; dilantin; &agr;-interferon.
F. Infections such as: congential rubella; cytomegalovirus.
G. Uncommon forms of immune-mediated diabetes such as: “stiff-man” syndrome; anti-insulin receptor antibodies.
H. Other genetic syndromes sometimes associated with diabetes such as: Down's syndrome; Klinefelter's syndrome; Turner's syndrome; Wolfram's syndrome; Friedrich's ataxia; Huntington's chorea; Lawrence Moon Beidel syndrome; myotonic dystrophy; porphyria; Prader Willi syndrome.
IV. Gestational diabetes mellitus (GDM)
* Patients with any form of diabetes may require insulin treatment at some stage of their disease. Such use of insulin does not, of itself, classify the patient.
Source: The expert Committee on the Diagnosis and Classification of Diabetes Mellitus, 1997.
It has been estimated that there will be 154,392,000 diabetics world-wide in the year 2000. Of these, 15,000,000 will be in the US and 934,000 in the UK. The burden of diabetes in both sexes in the WHO region estimated for 1998 was 11,668,000. Thus there exists a large medical need for an effective and safe oral therapy for the treatment of type 2 diabetes mellitus.
It is proposed herein that successful diagnosis and treatment of patients with type 2 diabetes mellitus may lead to a reduction in the risk of the development of cardiovascular disease in some of these patients. The primary pathology in diabetes mellitus is an inability to effectively control glucose. The underlying defect differs between the types of diabetes as outlined hereinbefore.
According to the present invention there is provided a method of treating type 2 diabetes mellitus in a mammal comprising administering to said mammal an effective amount of vardenafil or a pharmaceutically acceptable salt, solvate or composition thereof.
It is proposed herein that successful diagnosis and treatment of patients with type 2 diabetes mellitus may lead to a reduction in the risk of the development of cardiovascular disease in some of these patients.
The primary pathology in diabetes mellitus is an inability to effectively control glucose. The underlying defect differs between the types of diabetes as outlined hereinbefore.
In type 2 diabetes mellitus, resistances to the effects of insulin (insulin resistance) are manifest in the metabolic target organs such as the liver, adipose tissue and skeletal muscle. Metabolic pathways affected by insulin resistance include: glycogenesis, glycolysis, gluconeogenesis and the GLUT-4 transport mechanism as well as lipogenesis, lipolysis and protein synthesis within liver and muscle cells.
Resistance to the effects of insulin can also be observed in the diminished biological response of the endothelium to the vascular effects of insulin. That is, insulin promotes relaxation of blood vessel(s) at least in part through the action of nitric oxide. Nitric oxide generated in the endothelium then stimulates cGMP production in blood vessels and causes them to relax or dilate. This opening of the blood vessel allows more blood to flow, which is particularly important when more blood flow is needed to critical organs, like the heart. It has been demonstrated that there is a decreased release of nitric oxide (NO) from the endothelium of patients with insulin resistance. This decreased release of nitric oxide is not only from insulin, but also from other important vasodilators like acetylcholine. It is thought that the vascular effect of insulin contributes to the effect of insulin to regulate metabolism, particularly, but not necessarily limited to, glucose metabolism.
In addition to the vascular actions of nitric oxide, NO also has direct effects on glucose uptake by skeletal muscle. That is, treatment with a NO-donor substance (nitroprusside) or an analogue of cGMP treatment in vitro increases glucose uptake (transport by GLUT4 glucose transporters). This vasodilation-independent pathway is described in G. J. Etgen, D. A. Fryburg and E. M. Gibbs in
Diabetes,
46, 1997 pp. 1915-1919 the contents of which are incorporated herein by reference. It is proposed herein that, taken together, nitric oxide and cGMP likely have direct tissue level and vascular actions that influence, mediate, or mimic insulin's actions.
In a “normal” physiology NO diffuses through the endothelial layer and produces vascular smooth muscle cell (VSMC) relaxation and through activation of guanylate cyclase and cyclic guanosine monophosphate production, brings about a vascular dilatation (vasodilation). It is believed that on a chronic basis impaired response to insulin and associated reduced NO release and related vasodilation can lead to migration and multiplication of VSMC cells under the endothelial layer which can cause atherosclerosis.
Individuals with type 2 diabetes mellitus have varying degrees of insulin sensitivity (insulin resistance), by virtue of their type 2 diabetes mellitus/glucose intolerance and hence have an inefficient insulin dependant pathway.
Whilst type 2 diabetes mellitus has many manifestations it is proposed herein that an important underlying mechanistic basis for the condition resides in a resistance to both the vascular and metabolic effects of insulin. It is also understood that the underlying pathology of vascular resistance in type 2 diabetes mellitus, is a diminished amount of NO produced by the endothelial cells in response to insulin. In the insulin pathway in insulin resistant individuals, there may be impaired signalling of insulin for glucose uptake (via the phosphatidylinositol 3-kinase, PI3-K, pathway) which may lead to an inefficient GLUT-4 transpor

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