Biotechnology Company Neuralgene Provides Hope to Children With Genetic Disorder ALD, Announces First Human Patient of New Genetic Therapy

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April 29, 2014

If successful, Neuralgene’s PRCN-323 treatment could significantly change the prognosis for everyone with the rare genetic disorder known as adrenoleukodystrophy, or ALD. The biotech company has recently celebrated a series of early successes in the treatment of other neurological conditions like ALS (Lou Gehrig’s disease). Founder and CEO Jason Williams, M.D., believes his team is on the path toward a cure for these currently incurable maladies.

“We are very excited to closely monitor the results of this first patient,” added Dr. Williams. “We plan to expand our trial to more patients right away. This phase of the study will be performed in Playa del Carmen, Mexico.”

“In the meantime, we continue to study gene therapy options for other neurodegenerative disorders. We feel that we are close to having treatment options for several of these, including ALS and Parkinson’s. At this point, the biggest hurdle is building the infrastructure to manufacture enough gene therapy for these patients.”

ALD is an inherited genetic disease linked to the X chromosome, specifically a mutation of the ABCD-1 gene. Because males have but a single X chromosome, they experience much more severe effects from the disease. ALD causes a buildup of very long-chain fatty acids in the brain and adrenal cortex, disrupting the normal functioning of these organs.

A marked deficiency in adrenalin, also known as Addison’s disease, is among the first noticeable symptoms of ALD. Later, mood changes may occur, followed by cognitive decline and dementia. When the disease manifests during adulthood, this downward progression may take decades. Childhood onset of ALD, on the other hand, usually evolves rapidly to a vegetative state and then death. The life expectancy of a child diagnosed with ALD is typically 1 to 10 years.

The current best treatments for ALD primarily address symptoms rather than the cause. Hormone therapy can make up for the lack of adrenal function, but there’s little that can be done once cognitive impairments become obvious. Chemotherapy and bone marrow transplants have sometimes been useful, but these are both dangerous and exhausting options, especially for young people already weakened by ALD. Physical and psychological therapy can help children and adults deal with the day-to-day challenges of living with the disease, but a bona fide cure is out of reach at this time.

Neuralgene believes that a cure for ALD and similar diseases is perhaps not so far out of reach after all. The company’s approach to gene therapy for neurodegenerative conditions is entirely new. The process starts with an adeno-associated virus, or AAV. The AAV can interface with human and primate cells but is not known to cause any diseases or trigger an immune response, making it an ideal tool for the delivery of genetic material. Also unique to PRCN-323 is that the viral payload is injected directly into the spinal fluid, and the AAV preferentially targets neural tissue. It’s believed that expression of the “corrected” genes might begin in a matter of a few weeks.

PRCN-323 is the fourth novel technique developed by Neuralgene. Previous therapies have addressed Parkinson’s disease, ALS, and macular degeneration. Human testing is ongoing for the ALS and ALD treatments, with more extensive trials on the horizon. The company has partnered with Helica Labs in Playa del Carmen to produce its gene therapies.

About Neuralgene

Neuralgene is a biotechnology company with the main focus of developing neural-based gene therapy to treat neurodegenerative disorders. The company’s main product is a neural-based gene therapy for the treatment of ALS.

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