The ALSETF

As my readers may know, my posts this year have been a little farther and further between. I have been working on some projects. Hopefully one or more can make a real difference for PALS. It's time to talk about one of these projects: The ALS Emergency Treatment Fund.

The ALSETF is about bringing treatments in late development (post-Phase 2) to ALS patients. Right now there is no hope for the majority of living patients because over 50% do not qualify for clinical trials. For a newly diagnosed patient, the odds of living to see a drug approved which is starting trials at the same time is about 10% (actually much less considering the historical approval rate of ALS treatments). However, with recent advances into the nature of ALS, certain drugs have been developed which show real promise for treating at least a subset of PALS. More are planned to enter trials in the United States very soon. We at ALSETF mean to get that hope ASAP to PALS who are currently living.

ALSETF is a 501C3 non-profit organization with the mission to partner with government and industry agencies to enable Expanded Access Programs. Expanded Access Programs (EAPs) are FDA authorized programs that permit the use of yet-unapproved drugs under medical supervision, in specific cases where those drugs are in late stages of development and have shown preliminary evidence of safety and efficacy. EAPs are only for immediately life threatening conditions for which no effective approved therapies exist. We have open communication with the FDA's Office of Neurology products for guidance on EAPs involving investigational drugs for ALS. We also maintain open discussion with clinical leaders on the best practices for EAPs, as well as with certain pharmaceutical companies with drugs in trial and in the pipeline.

Our focus right now is to raise up to $5M to help fund certain costs of an EAP such as upgrading manufacturing to clinical-grade, production quantity necessary for fulfilling EAP demand, etc. These are all issues which can currently prevent a pharmaceutical company (especially the small start-ups likely to take a chance on ALS) from accommodating a large EAP. We believe the financial issues can be solved and that the drugs being talked about with excitement in the ALS community can be brought to those who don't qualify for trials now, while they are still living.

Wide Open

Tonight I found a double-helping of tasty news: "Regulatory T-lymphocytes mediate amyotrophic lateral sclerosis progression and survival" (PDF). So what's the big deal? Let me explain in two parts - the scientific then the soapbox.

Open Book - Pt. 2

(sorry for the long delay between parts but I was engaged in other projects, and I had to find the top of my head)

As I said in Part 1, Elsevier, a multibillion-dollar scientific publishing company, is trying to present itself as a friend of Open Access.

Is this a joke?

Open Book - Pt. 1

Earlier this year I wrote about publishers who want to hide public research information behind paywalls. Well I walk my talk. On October 20th, I personally delivered a speech to the Open Science Summit 2012. You can see my full presentation here (at 47:40.00). You can also view the video portion of my presentation on Youtube.

I have a little more to say about open publishing coming up in my next post as soon as I find where the top of my head landed...
(those who follow my tweets might already know what's coming)

Dope O' Mine

Something very interesting popped up in my searchbot results the other day. Most of us know dopamine as a neurotransmitter, the lack of which is the reason for the symptoms of Parkinson's Disease. Dopamine also exerts a potent immunoregulatory effect. Two new studies provide a new look at dopamine, and suggest that immune cells can create and secrete their own dopamine which can then interact with the immune cells' own dopamine receptors to influence those same cells' behavior.

Cross The Blogosphere - Part 2

Last week I wrote about Part 1 of a 2-part series at forbes.com regarding something called "biohacking". Today, Part 2, written by me, is now available. Hopefully I make a smidgeon of sense to you, dear readers, and that the team is successful.

Cross The Blogosphere

I would like to direct my readers to the blog of my friend Dan Munro. Dan writes, quite intelligently, about the healthcare space with a focus toward Information Technology (IT). As healthcare goes more and more digital in terms of research, treatment, and record-keeping, IT is critical to that development. As with the Internet boom, many gizmos and techniques will rise and fall away. Dan does a great job of sorting through it and his blog is worth a bookmark.

You might remember Dan from an earlier post featuring me. The earlier post was about the oral sodium chlorite project I created. Today Dan posted Part 1 of a 2-part series on "Biohacking". This is the result of his own research and some discussion we had about a project in which I am involved. Part 2 comes out next week, authored by yours truly, so stay tuned!