Amyotrophic Lateral Sclerosis (ALS), colloquially known as Lou Gehrig’s disease, is a devastating neurodegenerative disorder. ALS has proved fatal in all cases, and so cures and effective treatment has been a major priority of scientists and researchers of the disease. I’d like to use this post to bring attention to some breakthrough work that has been conducted by Harvard stem cell scientists, as reported in the Harvard Gazette.
The research focuses on unconventional ways of treating the disease. The Harvard researchers have reportedly found a “meaningful treatment”, and are currently working with Massachustets General Hospital to drum up an intitial clinical trial to test not only the effectiveness of this new treatment, but the safety of it as well. The scientists were sure to add a cuationary note to their work, noting that much more work had to be done before physicians could offer it to patients with ALS.
By now, I’m sure you’re wondering exactly what this new treatment is, or what it entails. Basically, it’s helpful to know a couple of facts about ALS- principally, that it’s caused by a variety of mutations across dozens of genes. This in itself can be hugely problematic for researchers looking to develop a treatment because a clinical trials done on mice often focus on just one of these many mutations. Though these mutations are technically different, one commonality they all share is the fact that they increase activity in motor neurons to abnormally high levels. This overactivity proves detrimental to neuronic health. Put simply, the heightened activity causes production of defective proteins, and the strain from excitability of the proteins puts them in a vulnerable and weakened state. In order to further understand this, the researches used neurons developed from the skin cells of several ALS patients, and then conducted trials to see how the overactive neurons reacted to medication and treatment for epilepsy. It turns out the the anti-epilepsy medication effectively decrease the hyperactivity of the motor neurons.There’s still a long way to go, but clinical trials are now being conducted to see if there are any side-effects in patients being treated with this medication.
This does look pretty good, especially considering the time and financial resources that must go into researching diseases like this. And that brings us to even more good news.
This research out of Harvard is freshly on the heels of news of some great fundraising for the ALS Society. Only a week a ago, local news broadcasting station WAVE3 reported that Heaven Hill Distillers donated over $400,000 to the Louisville, Kentucky Chapter of the ALS. This is a record-setting corporate donation, and the funds have been raised via the sale of a bourbon called “Parker’s Heritage”. The spirit is named after founder Parker Beam, who has ALS. Oh, and the goal was $250,000. They raised over $200,000 in extra funds.
Another successful fundraising campaign comes from the organizers of LiveLikeLou.org. The site launched about two years ago, with a fundraising goal of $40,000. The Pittsburgh Gazette reports that the site recently held a fundraiser that brought in $140,000- bringing the total funds raised to over $800,000. Good work!