There is a lot of effort going into research on ALS around the world, despite it being a relatively rare condition. These efforts were without doubt boosted over the last decade or so by fundraising initiatives such as the ALS Ice Bucket Challenge.
Whilst there is still no cure, there are a few FDA-approved treatments for ALS that have been shown to slow the progression of the disease. The first approved drug was Rilutek (riluzole). Thanks to ongoing research, however, some other treatments have been approved more recently.
One of these is Tiglutik, which is an oral liquid formulation of Rilutek. The importance of oral preparations for individuals with ALS, especially during the later stages of the disease, is related to the difficulty patients can experience when trying to swallow pills.
Here we take a closer look at this FDA-approved ALS treatment, how it works, what possible side-effects there are and the research behind the drug.
Produced by ITF Pharma in the USA, Tiglutik is the latest FDA-approved ALS medication that is being used to help slow down the progression of the disease. Riluzole has only been available as a tablet in the past and this has caused some issues for ALS patients who experience swallowing difficulties.
The drug is in liquid form and can be administered easily using a dosing syringe. Tiglutik is defined as an orphan drug. These are simply medications that have been developed for rare conditions and depend on government assistance to finance their production. Without this support, they would not be profitable for drug companies to produce at all.
As you might expect, Tiglutik works in a similar way to riluzole on which it is based. An overabundance of the neurotransmitter glutamate seems to have a role in damaging nerve cells and this is important in understanding how the drug works.
When nerve cells are transmitting messages between each other, a neurotransmitter is essential to make sure this happens seamlessly. In a healthy nerve gap, the glutamate is produced, aids transmission and then quickly dissipates once its job is done. If this neurotransmitter hangs around, it can mean a build-up occurs and the nerve keeps firing, eventually causing damage.
Whilst the way that riluzole and Tiglutik work is still not that well understood, it’s thought the drugs act by inhibiting this glutamate release, preventing a build-up and therefore slowing down any potential damage.
However, it’s important to remember that it is not a cure for ALS and its effectiveness can vary from individual to individual.
As with most drugs, there are some side effects related to Tiglutik. These include feelings of numbness in the mouth, nausea and weakness. Some patients may experience an increase in blood pressure and there is also the potential for decreased lung function.
Minor side effects such as a dry mouth, dizziness, poor sleep, joint pain and itchy skin have also been experienced in some cases by those who have taken Tiglutik. Because of its ability to cause dizziness, individuals that are taking the drug are advised not to drive or handle heavy machinery.
There have already been a number of different studies looking at the effectiveness of Rilutek which show that it helps to slow the progression of the disease. One of the more important of these found that a dose of the drug between 100 and 200 mg produced the most efficacious effect for patients.
Another initial piece of research compared taking 50 mg of Rilutek with administering a placebo. This study found that life expectancy was significantly increased in clinical terms in Rilutek group and the time before a tracheotomy had to be performed to aid breathing and death was increased on average by 90 days.
The research related to Tiglutik largely looked at how it compared to the pill version, Rilutek. The main study was carried out on healthy male volunteers, in other words, individuals who had not been diagnosed with ALS. One set of subjects were given various doses of Rilutek and the other Tiglutik.
The research found that the liquid form of the drug was absorbed quicker and produced higher concentrations in the blood than Rilutek.
As a liquid form of the Rilutek pill, Tiglutik has proved useful for individuals with ALS, particularly once difficulties with swallowing become apparent. It’s easy to administer both by the individual themselves and caregivers simply using a syringe.
Whilst there are certain side-effects associated with the drug, it means that treatment can still be administered easily after the swallowing reflex begins to decline.