What can we Learn from Patients who Self-Prescribe?

It is time to support patients who choose to prescribe their own treatments and manage their own healthcare outcomes.

Clinical trials are ALS patients’ only hope towards achieving an effective treatment in their lifetime. Yet, thousands of them will never enroll. That may be due to economic or geographical constraints. However it is more likely because they are excluded from participation due to specific enrollment criteria designed to facilitate the interpretation of a treatment benefit in the clinical trial.

So where do patients turn when they are no longer eligible to participate in the regulatory trial process? They turn to “experimental” options. Options where there are often no clinical data to support the treatment. Or even data to show it is safe.
But options they can self-prescribe and take “off-label”.

Continue reading What can we Learn from Patients who Self-Prescribe?

Welcome to TranslateALS!

I know. People who know me well are wondering…

A blog? Why? Why now?

As I have thought about it a little bit more, I’ve realized that my motivation to create a blog is driven by recent events that have galvanized opposing opinions in the world of ALS. A discussion of which will be be the topic of my first post!

My blog will focus on topics relevant to ALS. Based on the last fifteen years of my life there is plenty here to discuss. I would surmise that I may post some discussion on critical hurdles and advances in drug development and clinical trials as well; even if they are currently outside the realm of ALS with hopes of future application.

Continue reading Welcome to TranslateALS!

GM604: Is it Ready For FDA Approval?

It is challenging for any scientist, clinician, or stakeholder to keep abreast of the peer-reviewed published observations in their respective field. ALS is no different. It is even more challenging and critical to filter through all the available daily content from additional sources and focus on the pivotal advances. The breakthroughs at any point in the drug development value chain that could lead to the “Ah ha” moment and eventually a treatment for ALS.

Looking back, it’s not very surprising how little mental energy I put into the announcement of Genervon successfully completing a phase IIA trial of GM604 in ALS back in October of 2014. After all it was only 12 patients and 12 weeks of exposure to GM604. Typically that would make it just the first check box in the long and arduous process of testing if a drug is safe and provides any measureable benefit to ALS patients or any other disease.

Continue reading GM604: Is it Ready For FDA Approval?