The ALS Research Lecture

The morning lecture consists of four speakers.

Petra Kaufamann, MD, MSc, Director of the Office of Clinical Reseach (OCR) at the National Institute of Neurological  Disorders and  Strokes, which is part of the National Institute of Health.

Kevin Horoton, DrPH, MSPH, Cheif of the Surveillance and Registries Branch, with the Division of Health Studies, at the agency for Toxic Substances and Disease Registry (ATSDR) in Atlanta, GA.

Dr. William (Bill) Mathew is the Director of the Office of Translational Reseach at the National Institute of Neurological  Disorders and Stoke (NINDS) which is part of the National Institute of Health.

Lee Rubin, PHD is Director of the Translational Medicine and the Harvard  Stem  Cell Institute and a member of the Department of Stem Cell & Regenerative Biology.

The stem cell lecture was the best.

There was someone else after him, a woman from Columbia University, who talked about a program where scientists are taking cues from ALS patients, listening in on their homegrown treatments (so to speak) on the web, and then doing their mini clinical trials based on these hearsay treatments.

This startled me. I realized, when I heard this, that you cannot count out a single possible treatment because research is so still so nascent. The program is called something like,

 "Tweets and Twitters, ALS Untangled"

This woman also talked about the importance of clinical trials.
 Treatments without controlled trials is lost time.

All of the lectures were good. Still, the phlegmatic approach to the lectures made me angry.
This country wasn't founded by cool and collected revolutionaries enjoying lavish dinners and lovely table settings. AIDS wasn't controlled by the calm and collected. After listening to one of the speakers I had this impulse to yell ... "FIRE! There's a f-cking fire going on and your cooly talking about setting up programs to discuss the best fire escape protocol." It's hard to swallow.

As one patient said, through a computerized voice, "it feels like Ground's Hog Day. Every year we hear promises of great break throughs but nothing ever changes. You talk about the importance of Advocacy. Where are the PSAs for ALS?"

ALSA's website, proclaiming amazing things about to happen, was written twenty years ago. It hasn't been changed, nothing has changed. Maybe, better wheelchairs and more money for Vets?

__________

I asked CAR to send me her notes on Rubin.
Here is what she emailed back.

I will write from memory for 15 minutes before I pack the lunches -- and meanwhile here is a link to an article about Lee Rubin.

 http://www.hsci.harvard.edu/newsroom/foundations-support-hscis-search-potential-motor-neuron-diseases-therapies 

He said that they have been able to create motor neurons from stem cells and do experimemts with those new motor neurons to find out what kills and what helps build them. They've discovered three compounds which affect motorneurons (1) receptor inhibitors, 2) ___________, 3) ________________)) and are trying to discover which environmental chemicals contain those compounds, and they are also working with pharmaceutical companies to develop medicines which, for now, might help defend the remaining motorneurons and slow or stop the disease. Hopefully then in the future they can also find ways to rebuild motor neurons -- I think that's what he said.

Also good was the Columbia U researcher who explained why studies are important, and that rumors and blogged and tweeted treatments of things that MIGHT work can create confusion and not knowledge. Examples of things that turned out to be ineffective: the medicine for bipolar, and something else. She said that now scientists are trying to capture the "buzz" from the internet on what works and they are trying to test them in small Phase I and Phase II trials to see if any potential effectiveness is suspected before moving on to the more time-consuming Phase III trials.

Okay -- more later.....