Report: Pharma Can Resume Testing Drug Efficacy for Neurological and Respiratory Conditions

RespireRx Pharmaceuticals announced last month it was cleared to resume its ampakine CX717 clinical trials to evaluate the drug’s efficacy in several central nervous system and respiratory disorders, including Pompe disease.

RespireRx was in the middle of very promising clinical development of the drug, when it hit a snag in the road. Vacuoles were discovered in brain tissue samples of the animal subjects, and it was feared CX717 carried neurotoxic agents that would have rendered the trials – and hope for the treatment – null.

But a new study addressing this adverse discovery clears it all up!

The study presented clear scientific evidence these vacuoles (found in cells) were actually an artifact of tissue processing rather than a toxic drug effect.

They were not an effect of CX717  as previously thought. And according to the study:
“CX717-associated vacuoles do not represent a risk for human patients being treated with agents of this molecular class.  Accordingly, the current data should clear the path for continuing clinical research on the use of this promising class of compounds to treat a broad range of currently underserved neurological diseases.”

According to Gary Lynch, Ph.D., Professor at the Center for the Neurobiology of Learning and Memory at the University of California, Irvine, and a co-author on the study:

“The compelling results reported in this paper are tremendously exciting because they resolve a nagging issue about an ampakine that had intriguing, positive effects in clinical trials.  This is a major step in the development of the entire family of compounds.”
This is heartening for several reasons:
  1. It’s always great and important to see that indeed safety matters. Something was flagged during the clinical trial, and in an abundance of caution, the research scientists had to re-examine these vacuoles to verify if indeed it was toxic.
  2. Research science is a communal sport. When one branch hits a breakthrough, another branch can flourish. Every day that there are new developments means one day closer to more treatments and cures for any and all ailments.
  3. And of course, this is great news for those with rare CNS and respiratory disorders (of which CX717 is being tested for) – including Pompe disease.
Pompe disease is a rare genetic disorder that occurs as a result of certain mutations found in the GAA gene; it causes high levels of glycogen, a complex sugar found at the cellular level, to accumulate. Because the body is unable to process the buildup of glycogen in people with Pompe disease, it impairs the functioning of various muscles, tissues, and organs. To read more about Pompe disease, click here.

Click here to read the full abstract.

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