Global Adrenoleukodystrophy Treatment Trends and Highlights – Bulletin Line

Posted: August 4, 2020 at 8:56 pm

Adrenoleukodystrophy (ALD) is a rare genetic condition that causes the buildup of very-long-chain fatty acids (VLCFAs) in the brain. The defective gene in ALD, commonly referred to as a genetic mutation, can cause several different but related conditions: adrenomyelopathy (AMN), Addisons disease, and the most common and most devastating form cerebral ALD. Cerebral ALD strikes boys between ages 4 and 10, leading to permanent disability and death usually within four to eight years.

Signs and symptoms of the adrenomyeloneuropathy type appear between early adulthood and middle age. Affected individuals develop progressive stiffness and weakness in their legs (paraparesis), experience urinary and genital tract disorders, and often show changes in behavior and thinking ability. Most people with the adrenomyeloneuropathy type also have adrenocortical insufficiency. In some severely affected individuals, damage to the brain and nervous system can lead to an early death.

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The diagnosis of the disease includes genetic counseling, differential diagnosis, biochemical and molecular diagnosis. Genetic counseling must be offered to the parents of affected boys, adult males, and women with X-ALD and their family to detect: carriers who can be offered prenatal diagnosis, and asymptomatic or pre-symptomatic men or women who can benefit from therapeutic interventions. Regular follow-up in presymptomatic males can prevent serious morbidity and mortality.

Despite significant mortality risk, allogeneic HCT remains the only therapeutic intervention that can arrest the progression of cerebral demyelination in X-ALD, provided the procedure is performed very early, i.e., when affected boys or men have no or minor symptoms due to cerebral demyelinating disease. In the future, transplantation of autologous hematopoietic stem cells that have been genetically corrected with a lentiviral vector before re-infusion might become an alternative to autologous HCT, once the very encouraging results obtained in the first two treated patients would have been extended to a larger number of patients with cerebral X-ALD.

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There are currently only two available treatments for childhood cerebral ALD: Lorenzos oil and stem cell transplantation, using either umbilical cord stem cells or bone marrow stem cells. Both treatment approaches have shown promise and been effective for some boys with ALD, but they also both have drawbacks. The therapeutic pipeline of Adrenoleukodystrophy consists of approximately 9+ products in different stages of development. Currently, 3+ drugs are in Phase III development and major drugs are in the late stage.

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Some of the key players include Applied Genetic Technologies Corporation; Bluebird bio; Magenta Therapeutics; MedDay Pharmaceuticals; Minoryx Therapeutics; NeuroVia; Orpheris; ReceptoPharm; SOM Biotech; and Viking Therapeutics. Several M&As along with partnerships have been undertaken by these players to facilitate costumers with hi-tech and innovative products.

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