New ALS Therapy in Clinical Trials

A new drug is being tested and doctors say it has the ability to extend survival as well as reverse neuromuscular damage in people battling with ALS.

Records say close to 20,000 people in the U.S. surfer from Amyotrophic Lateral Sclerosis and that it is an invariably fatal disease.

Although slowly, ALS kills nerve cells that are responsible for mobility. These nerves control walking, breathing and eating, and statistics have it that most people succumb to the condition three years after diagnosis.

ALS treatment

Source: medicalnewstoday

10% of ALS cases are inherited and a fraction of those are caused by a mutation in SOD1. Now, what happens is that these mutations make SOD1 super active. Hence suggesting that reducing the number of proteins might help ALS patients in controlling SOD1 mutations.

People battling ALS have limited options for treatment because so far, FDA, the body that approves drugs for human use has okayed only two drugs and both of them function to only slow the symptom course of the disease.

Hopeful investigations

The thought has been, once the patient is diagnosed with the disease, very little to nothing can be done. But from a recent research, pioneered by Washington University’s School of Medicine, results indicate that a new therapy does indeed lengthen the lifespan of rats and mice in the inherited form of ALS.

In addition to that, the animals exhibited some recovery in their previously damaged neuromuscular tissues. The findings also appear in Monday’s issue of the Journal of Clinical Investigations. This has sparked hope and the researchers speculate that the drug could benefit people with the ALS that resulted from mutations in a gene called SOD1.

Although still in the confines of clinical investigations, the new drug has shown an impressive effect in rats and mice with as little as just two doses,” explained prof. Timothy Miller Ph.D. a neurologist at Washington University. “We have already done a safety test and was successful, what we are yet to establish is the right dosage and how effective this would be on people.”

Details of the test


Among others, the goal was to test DNA-based compounds, essentially those that hinder the body from processing SOD1 protein. The researchers at Ionis tested two of these – antisense oligonucleotide (oligos) –in rats and mice.

The specimens received some genetic modifications to enable them to carry a mutated form similar to the human SODI gene. The experts then waited for a month until the animals begun having trouble eating and walking.

After which, the first group was given anti-SODI oligo and the second a placebo at day 50. The second dose on both groups was administered six weeks later.

Under the active medication, the mice maintained their weight for 26 days longer and survived 37 days longer than those given the placebo, which in summary indicates an additional lifespan of over 20%.


The researchers then tried the treatment in rats, which received an active aligo and were seen to fare on better than those that received the placebo. Their weight remained intact for nine weeks longer and they lived for another eight weeks longer.

There was also clear evidence of neuromuscular recovery in the animals. Basically, after nine weeks animals that received the mutant SOD1 would show molecular signs of declining neuromuscular function. So to confirm the effect of the drug on this deterioration, the researchers put 9-week-old mice under anti-SOD1 oligo and another bunch under placebo.

Those that received the active drug recorded a steady improvement in muscle function while those in the placebo group become worse and worse as time passed.

Wrapping it up

So far, the drug has passed the crucial safety test at the clinical level, after which the researchers will need volunteers to see how it fares on humans.

However, challenges in getting enough people with this form of ALS might be an uphill task because there is only a fifth of them in the 10 percent population of people that suffer from the inheritable ALS form. Nonetheless, the researchers are very hopeful that the drug will be a good success.