FightMND is an Australian nonprofit organization dedicated to improving the lives of motor neurone disease patients through funding research for cures. They’ve successfully provided millions toward research initiatives and clinical trials. One of their most recent ventures was funding a substantial portion of the new Phase 2 RESCUE-ALS trial.
Clene Nanomedicine has just announced that enrollment is complete for this investigation and the very first participant has received the therapy. This trial is investigating CNM-Au8 and researchers are extremely excited about the promise of this treatment for amyotrophic lateral sclerosis (a more widespread name for motor neurone disease), a lethal disease that currently has no approved therapeutic option.
RESCUE-ALS is a randomized, placebo-controlled, double-blind trial which will occur at multiple trial sites. Specifically, it will investigate the safety, efficacy, pharmacodynamics, and pharmacokinetics of an oral CNM-Au8 formulation in newly symptomatic patients.
All patients in this trial will continue to receive their normal care in addition to either the treatment or placebo. The treatment period will be 36 weeks during which the therapy will be taken once each morning.
The primary endpoint of this trial is the difference in motor unit index score. This score measures the loss of motor neurons which is the primary cause of the progression of disease. The main objective of the trial is to understand the effect this therapy could have on preserving the survival of motor neurons for patients who have just recently begun experiencing symptoms.
So what exactly is CNM-Au8? It’s a concentrated version of nanocrystalline gold. It has already been tested in a Phase 1 study where safety was demonstrated in a group of healthy volunteers.
Preclinical studies have shown remyelination effects and neuroprotection effects. Improved neuron survival and mitochondrial capacity during stress, the protection of neurite networks, and a decrease of reactive oxygen species have all been positive effects demonstrated by this drug.
This investigative therapy has received approval to be studied in clinical trials for ALS, multiple sclerosis (MS) as well as Parkinson’s disease. In animal models of all of these conditions, improvement was found in functional behaviors.
The ultimate hope is of course that CNM-Au8 will become the very first approved therapy which can effectively modify disease for ALS patients.
You can read more about this trial here.