The average person probably has not given too much thought about ALS, but if you’re looking at this specific of a topic you probably already have an above average amount of knowledge on the topic. For those that don’t know though, ALS compromises a person’s muscle control while keeping their mental capabilities completely intact. But as of recently, people have been more exposed to the disease even if they haven’t met someone with it in real life especially recently due to the ALS Ice Bucket Challenge and even just knowing of Stephen Hawking, who is a famous scientist living with the disease.
Unfortunately, even with today’s medical advances this is still an incurable disease. This is a problem because there is a near 100 percent fatality rate associated with the disease. There is however a drug that can prevent some of the symptoms of this disease currently, Rilutek.
Rilutek is one of the only approved drugs for treatment of ALS. It is shown to improve functioning and prevent nerve cell over excitation from glutamate. Phenytoin is used to ease cramps, and baclofen is used to relieve stiffness in the limbs and throat. Once again though, these just cure symptoms and don’t actually treat the disease.
There is though, a potential medicine that look’s extremely promising for those diagnosed with ALS, and that medicine is GM604. In animal trials with the drug it extended survival life spans by 500%. It also provided neuroprotection against soluble inflammatory factors in human ALS patients’ Cerebral Spinal Fluid by 175%
It does this through a wide variety of factors including insulin receptor binding, reducing the damages caused by free radicals to reduce ALS apoptosis, providing PIP3 Kinase Activation for neuroprotection and axonal transport stiumlation to delay ALS onset.
So what is the problem then? Well as with any new drug, the length of time it usually takes to get a drugs approval can be very long and it doesn’t look like it will get approval for at least another 10 years. This is a serious problem for someone looking to get treatment as this is a disease that usually kills or seriously disables you within just a few years of being diagnosed with the disease.
This could be alleviated through the FDA granting this drug a status of accelerated approval which is given to drugs that are deemed safe to help terminally ill people with. They are unlikely to do this soon though based on the fact that there still hasn’t been many studies done on the long term effects of the drug, even though someone suffering from this disease probably isn’t probably too concerned with these long term effects. It is my hope that the ability to use this drug for treatment of the disease happens as soon as possible, and hopefully if there is enough awareness of a need for a cure in the general community we might be able to get that done.
GM604 for Treatment of ALS – A New Hope for Treatment of the Disease?
