Clene Nanomedicine Develops New Therapy for ALS Using Gold Nanocrystals
Clene Nanotechnologies, a Utah based company, has developed a nano-therapetic for neurodegenerative disorders including ALS. To date, Clene's preclinical trials have shown that the drug, CNM-Au8, improves neurological function in those with these neurological disorders.
Clene’s Chief Medical Officer, Robert Glanzman, MD, explained the theory for how CNM-Au8 works:
- The brain is only 2% of a human’s body weight but it consumes 25% of the body’s energy. The reason is that neurons are highly bio-energetically dependent. Our bodies require them to do a lot of things: transmit and process information and produce output thousands of times a second, 24 hours per day.
- These cells are supported by glial cells, a type of cell that produces myelin, the sheath around nerves. However, Glanzman cautions against underestimating the importance of myelin. It’s like insulation on a wire, but much more complex.
- The axons, or parts of the nerves that connect to each other and communicate, can’t live without their myelin sheath for very long because the myelin provides trophic support to the axons. Thus, myelin feeds axons. If axons demyelinate, they die and disease sets in.
- Unfortunately, the human brain becomes less bio energetically efficient as we age. Aging brain cells have less mitochondria and the remaining mitochondria don’t work as well. Worse, many people have a genetic predisposition for neurodegenerative diseases. When the neurons and glial support cells can’t keep up with these underlying metabolic problems caused by genetics (which they are normally quite good at dealing with), then these cells begin to die. This die-off causes neurodegenerative diseases such as Multiple Sclerosis, Parkinson’s, and ALS.
- The hallmark of neurodegenerative diseases is the build up of proteins within the cytoplasm--the jellylike material that makes up much of a cell inside the cell membrane.
Clene uses a non-chemical process to manufacture tiny gold nanocrystals. These crystals are then exposed to a large amount of electricity and a very small amount of sodium bicarbonate (baking soda) which causes them to stably aggregate and align in a pure, pharmaceutical-grade water suspension that a person with a neurodegenerative disorder drinks each day--an amount of about 60 ml, or a quarter cup. The gold crystals move through the person’s body, reach neural cells, including the demyelinated axons, which are given a shot of pure energy or “free chi”, as Glanzman puts it -- millions of donated electrons from the gold nanocrystals, thereby giving the depleted nerve cells the energy to remyelinate or to create new myelin sheaths on demyelinated axons.
Clene has observed positive results from preclinical data. Early blinded clinical data involving MS patients under standard of care are showing clinically relevant improvement of function, including vision, gait, and fine motor movement.
The proprietary and novel approach is in its clean-surface manufacturing process of the nano-crystals that eliminates contamination of the nano-particles’ surfaces. Clene believes that its unique process of creating the nanocrystals--a super clean process that utilizes large quantities of electricity rather than lasers or chemicals that other approaches have taken and which contaminate the crystals with other substances--allows for the human body to effectively use the gold nanocrystals as bio- energetic support. Clene claims its process results in the gold nanocrystals remaining stable in the liquid suspension for years.
The development of CNM-Au8 is continuing through its ongoing Phase 3 study for the treatment of ALS, and multiple Phase 2 clinical trials for the treatment of progressive neurologic impairment seen in people suffering from MS, Parkinson's disease, and ALS.
The CNM-Au8 product is included in the multi-drug HEALEY ALS Platform trial clinical trial led by Massachusetts General Hospital which divides patients into three treatment cohorts: Zilucoplan, Verdiperstat or CNM-Au8.
An additional Expanded Access Program clinical trial for up to 20 participants is also in progress at Mass Gen Hospital.