What is AMX0035, and Why Is It Important for All of Us?

In 2014, the Ice Bucket Challenge was created to raise awareness about ALS (Amyotrophic Lateral Sclerosis or Lou Gehrig’s Disease.) Chris Kennedy, a pro golfer, challenged a relative on behalf of a relative with ALS. Pat Quinn, who has ALS, continued it. Peter Frates’ family extended it even further. The Frates’ family also has a foundation to help with financial needs for individuals with ALS.

The ice bucket challenge went viral over social media, and a worldwide phenomenon was started. Everyone was pouring or having a large amount of ice water poured over their heads. Donations went to research for ALS. The purpose of the Ice Bucket ALS research is to get researchers interested in studying ALS, develop treatments, and to find a cure.

Now imagine, about six years later, after over 200 million dollars was raised. The results are outstanding. One of the most significant findings is the development of a new medication, AMX0035. This drug is a combination of sodium phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA). The drug minimizes the process of cell death by blocking the pathways in ALS. It works by regulating the mitochondrial (cell power supply) and endoplasmic reticulum (quality control centers). To put it simply, both processes should work in unison, but when they don’t, ALS can develop. Cells in the body die, and internal, neurological inflammation develops.

AMX0035 regulates cells in the body to prevent the stress that kills those cells and prevents neurological inflammation from developing. This might sound familiar to you with any neurological disease or trauma. You can read more details about this here: https://alsnewstoday.com/amx0035/

Drugs are developed in a three-phase process. Phase I is a study of safety. Laboratory information has been completed with enough evidence to think that a drug is safe for humans, but the Phase I trial, performed in a small number of people, ensures this.

Phase II is done in a larger number of individuals specifically to test dosing sizes. This is to establish if a little of the medicine works well or if you need large doses. Also, some people will get the medication, and others will receive a fake medicine (placebo) to make sure the effects of the medicine are real. No one knows who is getting the real dose or who is not. The study participant, physician, nurse, no one who is working with study volunteers know who is getting the drug and who is getting the placebo.

The next step is Phase III, which is done with many people. This is the last step before the drug becomes available on the market. Sometimes, there are some subphases, but basically, these are the main three.

Interestingly, Phase II of the study of AMX0035 has produced some excellent results. Because ALS has no known cure, the drug is going to be released for people to use now. This is sort of a loophole in drug testing where humanitarian issues take precedent due to the severity and negative outcomes of some diseases.

The use of AMX0035 has resulted in the improvement of quality of life and even, in some cases, extended life for those with ALS. It is not a cure but a treatment that makes individuals’ quality of life significantly better. The study of AMX0035 will continue with Phase III trials, but in the meantime, individuals with ALS should be able to take advantage of the drug now, if they choose. If you have ALS and you think this drug might help you, talk to your healthcare provider who can make the contacts to secure the medication possible. The pharmaceutical company that is developing the drug is Amylyx. There are two trials currently in process.scientist in a lab

Taking a new drug requires serious consideration. Since the drug has not gone through all phases of the study, there could be some non-favorable consequences that are yet to be discovered. Also, there could be something unique to the body function that could cause the drug to act differently in you. Be sure to discuss this with your healthcare provider and become well educated about any medication before taking any drug, prescription, over the counter, or recreational.

Why this drug is important to everyone with neurological disease and trauma

You might be thinking that you don’t have ALS, so how could this discovery be important to you. Some of the terminologies in the mechanism of action should be familiar to your cell death and neurological inflammation, which occurs in many neurological events, both disease and trauma.

AMX0035 is being studied in individuals with other neurological issues. These include:

  • Amyotrophic Lateral Sclerosis
  • Motor Neuron Disease
  • Neuromuscular Diseases
  • Neurodegenerative Diseases
  • Spinal Cord Diseases
  • TDP-43 Proteinopathies
  • Nervous System Diseases
  • Central Nervous System Diseases

There are some that are considering AMX0035 in the treatment of Alzheimer’s Disease and Parkinson’s Disease.

In the study of neurological disease and trauma, what works in one area of study often is translated into other areas. As more information becomes available, individuals with SCI could benefit. AMX0035 could be studied for use as a treatment at the time of injury or those with chronic spinal cord injury or both.

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Apoptosis is a process the body uses when nervous cells become overcrowded. This can be from increased swelling from injury or disease. Some nerve cells die so others can live. AMX0035 could possibly decrease this activity.

We know that any source of SCI sets off a cascade of internal body neurological inflammation. This drug helps with control of that neurologically toxic inflammation, so it would make sense that there might be a link that would help improve the lives of those with SCI. That theory is yet to be demonstrated, but it is interesting to consider.

For now, we need to keep following the progress of AMX0035 and other drugs that work internally to improve the neurologic system. If this drug prevents cell death and neurological inflammation for those with SCI is just starting to be studied. More exploration of research is needed.

There are many aspects of therapies that are being studied for the improvement of SCI and cure. From the research that is currently approved, we know the treatment probably will not be one therapy alone, maybe in the future, but not with the information that is currently available. Treatments to improve function include therapies, both traditional and advanced, to keep the body healthy and ready for function again. Surgical interventions have made improvements in early stabilization. There is knowledge about reducing edema and pain control. Implants are improving movement and function. Some implants assist with some body functions such as hand and arm movement, bowel, bladder, and sexual function and mobility. Medications are being explored for the reduction of edema, improvement in function, and decreasing internal inflammation. Dietary treatments are being studied to do the same. Also, the equipment has become much more advanced. Secondary complications of SCI are being reduced. Quality of life is enhanced.

Is this an over-rosy picture? Perhaps for some, but these therapies are real. When you or your loved one is waiting, the time is painfully long. However, we can take some pleasure in the success of a new possibility. 

Pediatric Consideration:

Treatments for children generally follow discoveries in the adult population. As parents or caregivers, keep following what is going on in adult research and pediatric SCI research. Sometimes, modification of studies is made to aid in the rehabilitation of children.

Success for adult treatment typically follows with pediatric advances. We need to encourage our healthcare professionals to consider adult treatments for study in the pediatric population. Explanations of what is happening in age-related research for adults and children is beneficial to understanding progress. 

About the Author - Nurse Linda

Linda Schultz, Ph.D., CRRN is a leader, teacher, and provider of rehabilitation nursing for over 30 years. In fact, Nurse Linda worked closely with Christopher Reeve on his recovery and has been advocating for the Reeve Foundation ever since.

Nurse Linda

The opinions expressed in these blogs are the author's own and do not necessarily reflect the views of the Christopher & Dana Reeve Foundation.