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→ Main indications II
Main indications I
Sarcoidosis, also known as Besnier-Boeck-Schaumann disease, is an auto-aggressive systemic granulomatous disease that primarily affects the lungs, but can affect almost all organs of the body. The prevalence of sarcoidosis is between 1 and 60 people affected every 100’000 individuals, and presents a clear south-north gradient and racial variations. It is usually considered as a fairly mild disease as most cases of sarcoidosis disappear spontaneously. However 10 to 20% of sarcoidosis patients develop a chronic form that could even be life threatening.
Sarcoidosis commonly develops in younger and middle-aged adults, most probably following exposures to poorly degradable antigens of infectious or environmental origin, which trigger an exaggerated immune reaction. It is a globally occurring disease with potential severe manifestations in the skin (16-35%), in the eyes (12%), in the lymph glands (5-15%), in the liver (12%), heart, kidney, bones or the central nervous system of the patients in addition to the lungs, affected in more than 90% of the patients.
In most cases, sarcoidosis leads to constitutional clinical manifestations like dry unproductive cough, severe chronic fatigue and dyspnea on exertion, and may lead to terminal stages of lung fibrosis. The cellular and molecular hallmark of sarcoidosis is the formation of noncaseating granuloma, which is characterized by the accumulation, proliferation and exaggerated spontaneous activity of macrophages and T-lymphocytes at sites of inflammation.
The goals for treatment of sarcoidosis are clear: suppressing the initiation of granuloma formation by inhibition of antigen processing and presentation, limiting inflammatory lesions that are interfering with organ function, preventing fibrosing processes, and inhibiting constitutional manifestations like cough, severe chronic fatigue and exertional dyspnea.
Diabetic neuropathy (DN) is progressive and develops as diabetes persists; it is characterized by the degeneration of peripheral nerves starting at the arms’ and legs’ extremities, and progressing to internal organs. This condition remains extremely serious as patients experience pain, as well as non-pain-related signs such as loss of balance, lack of sensation, autonomic dysfunctions (heart, bladder, sexual and digestive function). These deficits contribute to the degradation of the patients’ quality of life and reduce their life expectancy.
Distal Symmetrical Polyneuropathy (DSPN), the most common form of diabetic neuropathy affecting sensory fibres, often coexists with autonomic neuropathy, which concerns internal organs innervation. Both pathologies display the hallmark of nerves deterioration and abnormal innervation with segmental demyelination and/or axonal degeneration. Besides, once initiated, nerve degeneration and deficient microvascularization, an intertwined pathology, only worsen and lead to additional disabilities (foot ulcer, amputations etc.).
By slowing or stopping the course of the disease, re-establishing adequate sensations and stimulating vascular function, a disease modifier treatment has the potential to prevent the occurrence of painful and non-painful symptoms, foot sores and ulcers and hence to greatly reduce the comorbidities’ burden of diabetes-induced neuropathic complications.
Main indications II
Acute Lung Injury
Acute lung injury (ALI) and its most severe form, acute respiratory distress syndrome (ARDS), represent important clinical entities with mortality rates of 30-50%. ALI has a high incidence (200,000 per year in the US) and remains a major worry of intensive care units. According to the American-European Consensus Conference, ALI/ARDS is defined as a syndrome of reduced pulmonary gas exchange caused by diffuse inflammatory processes with increased vascular permeability and diverse pathophysiological mechanisms lead to ALI/ARDS development. There is currently no pharmacological therapy approved for ALI/ARDS and the only intervention consists in mechanical ventilation. Based on its proven biological properties aviptadil offers the possibility to be a first line treatment in intensive care units to reduce mortality.
Chemotherapy-induced peripheral neuropathy
Chemotherapy-induced peripheral neuropathy (CIPN) is a common treatment-related adverse effect. Not only has it the risk to impose dose reductions of chemotherapy and/or early discontinuation, thereby compromising the treatment of cancer, but it also affects long-term quality of life in cancer survivors. The overall incidence of CIPN is estimated to be approximately 40% in patients. With almost 30 million cancer survivors accumulated between US and Europe, treating CIPN becomes a major need to improve quality of life and associated co-morbidities. Unfortunately, this condition has currently no effective treatments and practitioners tend to under-diagnose or ignore CIPN. Atexakin alfa offers a unique opportunity as the first potential pharmacological therapy for CIPN.