FDA Pressed to Allow Access GM604

ALS patients are now pushing the FDA to allow GM604 to be used. Patients are pushing for “accelerated approval” which will allow the drug to bypass large-scale trials.

A small Californian company named Genevron made this new drug. GM604 controls over 4,000 genes and modifies ALS progression. The drug was tested on mice with the SOD1 strain of ALS. Genevron said that  GM604 was able to delay onset of symptoms by 27% and extend life for up to 30%.

The preclinical and Phase 1 trials also have good news. The results show statistically significant results with no side effects. The campaign for accelerated approval would make the drug available to all patients immediately.

One particular patient and his mother have made a very strong case for the approval. Anthony Carbajal and his mother made a video that went viral and caught thousands of attention. This particular video has over 530,000 supporters. In this video, Carbajal talks about how his grandmother and thousands of others died.

Although there is a solid case for accelerated approval, some are skeptical. Steve Perrin, a chief scientific officer of the ALS Therapy Development Institute, is such a skeptic. “The bottom line with the Genervon drug is there is absolutely no data.” He said. “There is no mathematical way or statistical way that they could measure a drug effect.”

Even with a few critics, this is surely a step forward to a cure for ALS.

To see Carbajal’s video click here

What May Slow ALS Degeneration?

In a recent article in ALS News Today, researchers at Penn Medicine found that hypermethylation can slow the development of ALS. The normal methylation process impacts genes that affect DNA structures.

A research team at Penn Medicine recently found that hypermethylation is able to inhibit the development of Amyotrophic Lateral Sclerosis(ALS) and Frontotemporal Degeneration (FTD) in a study titled,

“This is the first epigenetic modification of a gene that seems to be protective against neuronal disease,” said lead author Corey McMillan, PhD, research assistant professor of Neurology in the Frontotemporal Degeneration Center in the Perelman School of Medicine at the University of Pennsylvania in a recent news release.

A gene called C9orf72 is linked to a DNA binding protein which is related to ALS and FTD.

“Understanding the role of C9orf72 has the possibility to be truly translational and improve the lives of patients suffering from these devastating diseases,” said senior author, Edward Lee, MD, PhD, assistant professor of Neuropathology in Pathology and Laboratory Medicine at Penn.

The study included 20 patients who had a gene mutation of the C9orf72 gene and a diagnosis of FTD or ALS. These patients were compared to 25 healthy individuals.

According to the report:

Results from the MRI showed a reduction in grey matter in many brain areas in the patient groups compared to the controls. Those patients that had hypermethylation of C9orf72 were found to have in the hippocampus thalamus and frontal cortex, more grey matter volume in areas that are affected in ALS and FTD. In order to validate the results the researchers evaluated autopsies of 35 patiets that had C9orf72 expansions and observed that an increased methylation was related with less neuronal loss in the hippocampus and the frontal cortex.

This implies that in patients with C9orf72 hypermethylation, the disease progresses slower. The researchers also found a relationship between hypermethylation and predicted memory decline.

The work identifies a gene that can be targeted to precision medicine treatments and for new drug development in motor neuron diseases. C90rf72 may be a “neuroprotective agent”.

References: “C9orf72 promoter hypermethylation is neuroprotective: Neuroimaging and neuropathologic evidence.” The results are published in the journal Neurology.


The “Right to Try”

When presented with life or death options, people will try just about anything. That is the basic idea behind the “Right To Try” Act, a movement that is now becoming law in states all over the country. This law gives patients with terminal diseases the option to use medication or treatments that aren’t passed by the FDA. This essentially bypasses the FDA’s trial program which can cost around $1 billion and take up to 10 years.

Advocates for the “Right to Try” argue that if we don’t give these patients the option to try experimental treatments, then we are taking away their chance of extending life span. Advocates also argue that patients should be able to do what they wish with their own bodies and lives. Furthermore, giving treatments to patients could provide a valuable research opportunity for clinics and companies.

David Huntley, a former collegiate geology professor, is a supporter of the “Right to Try” Act. Diagnosed with ALS in June of 2013, he has lost use of his arms, and has difficulty speaking due to the loss of use in his lips. Huntley and other ALS patients are frustrated with the absence of choice in determining his future. Huntley said, “It’s frustrating to deny access of a drug to a population afflicted with a rapidly progressing disease that is 100 percent fatal.”

In contrast, there are others who believe that the “Right to Try” is a step in the wrong direction. These critics argue that the experimental medication could be doing much more harm than good. These critics also worry that these laws would undermine the authority of the FDA. They also realize that the companies don’t have to supply these medications to patients. Producing, creating and manufacturing drugs and treatments is as very expensive process.  As a result, drug companies may be unlikely to provide experimental treatments to individuals with a progressive form of ALS.

The “Right to Try” Act will allow more patients and companies access to potentially lifesaving medicine and could be a giant leap forward in the ALS community. However, these new drugs could be taking away precious time from ALS patients and their families. This controversial law is in effect in a few states across the country, and is being advocated in many more.

To get more information about the “Right to Try” visit http://www.yourrighttotry.com/