On the ALS treatment trail

The condition is still incurable, although there are hints of progress. For decades, the neurodegenerative disease amyotrophic lateral sclerosis (ALS) was commonly referred to as "grim." Not only were the consequences terrible, but there was no viable treatment. Although ALS remains a dismal diagnosis, rays of optimism have begun to emerge.

There is presently no treatment for ALS or even a means to slow the disease's course. However, medications with the ability to halt it are on the way, and more daring treatments that might lead to a cure are showing promise. The medicine edaravone, which was developed and licensed for treating ALS in Japan in 2015, is the most promising and highly recognized advancement.

The US Food and Treatment Administration authorized edaravone in May 2017, making it the first new drug for ALS in more than 20 years (FDA). Edaravone (also known as Radicava or Radicut) is a tiny antioxidant molecule that has been used to treat stroke for many years. Researchers led by Joseph Palumbo, chief of translational research and medical science in Mitsubishi Tanabe Pharma Development America in Jersey City, New Jersey, demonstrated in 2015 that the medicine might considerably delay the course of symptoms in the early stages of ALS.

Edaravone isn't the only new medication on the market. Masitinib, for example, suppresses inflammatory enzymes, which is a promising target because nerve inflammation is a common symptom of ALS. Masitinib, developed by AB Science in Paris, has been shown to halt ALS progression when used in conjunction with riluzole, a medicine that inhibits particular kinds of neurotransmission and has long been the sole mainstream choice for most patients with ALS. ALS Worldwide, a non-profit group, is looking into the 'off-label' usage of medications that may help with symptoms.

Molecular genetics offer hope

ALS cannot be cured or even treated since its origins are unclear. Rapid advances in molecular genetics are already helping to understand and cure cancer. Scientists worldwide are increasingly using DNA and RNA sequencing to identify disease-causing genes and mutations. Techniques to switch genes on or off or modify their nucleotide sequences are being investigated as disease therapies. Several Northeast ALS Consortium sites are testing this approach in early stages.

We don't know where ALS comes from, hence it can't be cured Rapid molecular genetic advancements are already helping to treat cancer. Globally, scientists are employing DNA and RNA sequencing to find disease-causing genes and mutations. Disrupting genes or altering their nucleotide sequences is being studied as a disease therapy. In the early phases of the Northeast ALS Consortium.

Genes and cells are being repaired.

Injecting fresh gene copies into diseased cells is a bolder, and perhaps more long-lasting, way to combat ALS' genetic underpinnings. Adding more genes and getting them to work correctly is tough, not least because choosing which genes to add is complex. SMA-linked genes are not the same as ALS-linked genes, yet both destroy motor neuron cells.

Other cells beyond motor neurons are being explored for gene therapy. To help restore motor neuron function, glial cells may be able to change their genes. This notion is based on studies that will imply glial cell troubles may be partially to blame for motor neuron death in ALS.

A second long-term solution is stem-cell therapy, which may lead to a cure. The prospect of giving stem cells that can multiply and expand into the different cells necessary to keep motor neurons alive is being investigated globally.