Adrenoleukodystrophy Drugs in Ayurveda Focus on Preventing or Delaying the Progression of Adrenoleukodystrophy
Adrenoleukodystrophy (ADR) is a fatal genetic disorder that affects 1 in 13,500 individuals worldwide. It is an X-linked progressive non-insulin-dependent neuropathy. ADR involves multiple body organs in the human body so it predominantly effects the nervous system and spinal cord in most patients. This neuropathy results from damage to myelin in the myelin sheath of the brain. When the myelin sheath is damaged, the messages sent by the nervous system are not properly relayed or interpreted by the body.
It
is important to note that patients suffering from adrenoleukodystrophy should
be under the regular care and supervision of a hematologist. Such patients are
required to be under the regular therapy in order to prevent the occurrence of
adverse events and to improve the overall efficiency of the treatment.
The
Ayurveda treatment of adrenoleukodystrophy is aimed at improving the physical condition
of the affected individuals, preventing or delaying the progression of the
disease, preventing complications and treating the symptoms of the disease. Adrenoleukodystrophydrugs in this treatment include: Triphala Guggulu, Medohar Guggulu, and others.
Medicines that act on the 'Mansa' data (tissue) of the body are useful in this
condition, such as Charaka-Krishna-Upanishad, Shatavari-Churna, Brahmi (Bacopa
monnieri), and Tulsi (Ocimum sanctum).
Some
specific disorders of adrenoleukodystrophy may present with symptoms such as
severe loss of bone density, wasting, anemia, kidney or liver function, high
levels of blood cholesterol, increased susceptibility to infection, lethargy,
hypothermia, poor appetite, fluid retention, polydipsia, polyuria and weight
reduction. Patients diagnosed with a form of this disorder are advised to take
additional adrenoleukodystrophy drugs and to reduce their activities so that
their symptoms can be ameliorated. Corticosteroid drugs are usually the first
line of therapy in such cases. However, prolonged use of glucocorticoids could
have some long term side effects, such as osteoporosis, kidney disease and
hypertension.
Gene
therapy has demonstrated potential for the treatment of patients with cerebral
adrenoleukodystrophy. In October 2020, European Medicines Agency accepted
Bluebird Bio’s marketing authorization application for the company’s
investigational elivaldogene autotemcel (eli-cel, Lenti-D) gene therapy for the
treatment of patients with cerebral adrenoleukodystrophy. The U.S Food and Drug
Administration also granted eli-cel Orphan Drug status, Rare Pediatric Disease
designation, and Breakthrough Therapy designation for the treatment of CALD.
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