FDA Grants Orphan Drug Status to Promising Treatment for ALS Called SLS-005
Seelos Therapeutics is working on a treatment called SLS-005 or trehalose to treat Amyotrophic Laterals Sclerosis. The drug proves to be effective in slowing motor neuron decay in lab models. Last week Seelos Therapeutics announced that they were granted Orphan Drug Status by the FDA (Food and Drug Administration). Orphan Drug Status provides special incentives to sponsors to develop treatments for rare disease such as ALS.
Currently, the ALS community has a short list of promising treatments under development and an even shorter list of available treatments. Because treatment options are so limited, every new drug granted Orphan Drug Status is a new door opened for ALS patients. Orphan Drug Status can help speed up the drug development process by eliminating FDA fees and waving restrictions.
Even so, the period for new drug development may equate to multiple life spans for someone with ALS. This is not going to be the case with SLS-005 because it has been under development for the last several years. It has complete phase 1 and 2 for clinical trials while under investigation for other motor neuron diseases and is now on its way to be approved for ALS as well.
Having undergone phase 1 and 2 clinical trials, Seelos now plans to initiate a phase 2b/3 clinical trial to investigate ALS treatment. This trial will enroll 160 patients with either familial or sporadic ALS in a double-blind placebo-controlled trial. This means that it will be a randomized study with 3 patients given the drug for every 1 placebo patient. The clinical trial will last 24 weeks but there has been no announcement as to when the clinical trials will begin.
The active ingredient in SLS-005 is a small sugar molecule called trehalose. It is not found in human cells but is common among many other organisms. Trehalose is an especially useful tool in treating ALS because it can penetrate the brain-blood barrier, a very selective barrier that prevents viruses and infections from getting into the brain and spinal cord. Because the blood brain barrier is highly effective, it is extremely difficult to get treatments to pass it. Therefore, active and developmental treatments for ALS often require regular spinal infusions. Trehalose’s ability to bypass the blood-brain barrier is a big step for drug development and future treatments.
How SLS-005 Helps ALS
One of the identified problems in most cases with ALS is the accumulation of mutated proteins that build up and overwhelm the cell, often leading to cell death. Motor cells do not regenerate the same way the other cells in our body do, so people with ALS slowly lose control of motor function. It is hypothesized that preventing this protein buildup could prevent or at least delay the decay of motor neurons in ALS patients.
This is why trehalose is exciting to researchers. It prevents protein build up, thereby allowing the cell to breakdown excess proteins. This leads to healthier cells and less cell decay.
Trehalose was originally under investigation for other diseases that involve neuron decay. In previous phase 2 studies, including over 70 patients, trehalose has proven highly effective in reducing protein excess. This makes it a promising treatment for ALS as well.
Recently Seelos Therapeutics has started investigating the effects of this drug on ALS. These Lab studies on ALS have proven to show exactly what we would hope for. Trehalose reduces the number of excess toxic proteins within motor neurons. The study results also show a preservation of motor neurons and an increase in the size of muscle fibers.