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.
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:
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.