Adrenoleukodystrophy is also known as Adrenomyeloneuropathy or Schilder-Addison Complex, it is a hereditary condition that damages the myelin sheath (membrane surrounding nerve cells in your brain) and disrupts the breakdown process of long-chain fatty acids (VLCFA). Adrenoleukodystrophy is passed down from parents to their children in a form of X-linked genetic trait.
The genetic trait causes deposition of very-long chain fatty acids in the body tissues due to impaired beta oxidation. Myelin sheath in central nervous system, the adrenal cortex and Ledydig cells in the testes are the most severely affected tissues. Adrenoleukodystrophy give rise to three major disease categories such as childhood cerebral form (observed between 4 to 8 years of age), adrenomyelopathy and impaired adrenal gland function (also known as Addison disease).
The major symptoms observed in a childhood cerebral form adrenoleukodystrophy patient are muscle spasms, crossed eyes (strabismus), hearing loss, seizures and other disorders related with the nervous system. In adrenomyelopathy the patients are observed with difficulty in controlling urination, muscle weakness or leg stiffness, difficulties in thinking speed and lack of visual memory. In Addison disease or adrenal gland failure the major symptoms observed are coma, decreased apetite, skin pigmentation, loss of weight, muscle weakness and vomiting.
According to Centers for Disease Control and Prevention (CDC), approximately 1 in 20,000 people suffer from X-linked adrenoleukodystrophy. The Office of Rare Diseases (ORD) of the National Institutes of Health (NIH) has listed Adrenoleukodystrophy as a “rare disease”. In addition to this, CDC also reported that adrenoleukodystrophy, is a subtype of adrenoleukodystrophy, affects less than 200,000 people in the U.S. population annually.
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The adrenoleukodystrophy is diagnosed primarily with plasma very long chain fatty acid (VLCFA) examination by application of gas chromatography and/or mass spectrometery. The other diagnostics methods include chromosome studies that are carried out to understand the mutation in ABCD 1 gene and magnetic resonance imaging (MRI) scan of head. Adrenoleukodystrophy is treated with dietary therapy, transplant, adrenal insufficiency and gene therapy.
The dietary therapy consists of prohibiting the patient for the intake of very-long chain fatty acids (VLCFA) and this is a supportive therapy to normalize the disease conditions of the patient. The transplants are performed with allogeneic hematopoietic stem cells that assist in the demyelination process where myelin sheath is restored and its deterioration is inhibited. In gene therapy appropriate vectors are selected and modified according to the normal ABCD 1 and later these are transplanted into patient’s bone marrow or stem cell transplant.
Adrenal insufficiency is the treatment still under research and trials as this process is ineffective and needs assistance form hormonal replacement therapy. In some cases genetic counseling is recommended for prospective parents with a family history of X-linked adrenoleukodystrophy.
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The product pipeline of adrenoleukodystrophy undergoing phase III trials is as follows:
- Campath-1H + Clofarabine + Melphalan + Hematopoietic Stem Cell Transplantation
- Campath-1H + Cyclophosphamide + Busulfan + Allogeneic Stem Cell Transplantation
- Busulfan + Cyclophosphamide + Antithymocyte Globulin + Hematopoietic Stem Cell Transplantation
North America was observed to be the leading geography followed by Europe due to high prevalence rate, increasing social awareness and key players based in the same geography. Asia-Pacific and Rest of the World lack due to unavailability and inaccessibility of the diagnostic techniques, counseling bodies and modern treatments.
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The key players involved in the adrenoleukodystrophy therapeutics market are :
- Nutra Pharma Corporation ,
- GENETIX Pharmaceuticals, Inc.