Too good to be true?

On the Internet, sometimes something can look like a medical breakthrough, but may not be as it appears. For instance, if you read this article written by a biotech startup company, you might think that a cure for ALD is right around the corner.

However, we must consider that the article was created by the company, and therefore the source is biased. ALD Connect, which is looking out for ALD patients, offers the following commentary on the new gene therapy:

A provisional assessment of the Neuralgene treatment in Mexico (as of May 1, 2014):

Neuralgene issued a press release stating that they are using their gene therapy platform to treat ALD. They claim to have an Adeno Associated Virus (AAV) ...targeted at delivering corrected gene copies to neural tissue. They injected their product, PRCN-323, into the spinal fluid of an ALD patient with the intention... of "infecting" neural tissue with a correct copy of a gene. They believe that expression of the "corrected" genes might begin in a matter of a few weeks. The press release states that Neuralgene intends to monitor the first patient and treat additional patients in Mexico. The technology pipeline page of their website says that the AAV vector is delivering the ALD gene.

What is known about this treatment?

· An email sent to the company to ask about the treatment has not yet been answered.
· Leading ALD physicians were not familiar with the treatment.
· ALD Connect does not have a relationship with Neuralgene.
· The trial was not listed in (see explanation below).
· There is no article published or data generally available to explain what studies have been done in animals to warrant trying it in patients.

While delivering a corrected version of the ALD gene sounds like a good idea, one must be cautious:

· Specific details of the procedure need to be evaluated to learn how safety is being ensured.
· It is not known where the AAV vector was manufactured and whether it was done to a standard that would support safety.
· It isn't clear what is being measured to demonstrate whether treatment is efficacious.

Should anyone be able to make contact with the company, please help us answer the following questions:

· Can Neuralgene confirm that ALD gene is being delivered?
· Can you specify how safety of the procedure is ensured?
· What kind of patients are you treating? children? adults? with cerebral demyelination? AMN? adrenal insufficiency?
· What kind of effect are you looking for?
· What makes Neuralgene think this treatment will work?
· Is there pre-clinical data you can share with us? animal-model data?
· What would it take for this treatment to be available in the US?
· When will you know if the treatment was helpful to the first patient? how will you share the information?
· While Neuralgene’s founder is listed as a radiologist, does the company employ a consulting neurologist with expertise in ALD?
· Is there pre-clinical data in non-human models? is a registry and results database of publicly and privately supported clinical studies of human participants conducted around the world


This discussion serves as a reminder that if something sounds too good to be true, it probably is. If you run into promises of miracle cures or magical treatments written by the company who stands to profit from the treatment, please maintain a healthy degree of skepticism. There is research taking place, and hopefully it will bear fruit, but all the glitters is not gold.