Andy ran a 10K race in Sacramento on April 15. We drove down the day before and back on the 15th. The drive to and from Sacramento takes us over Donner Summit, a pass in the Sierra Nevada mountains.Stan and I went over the mountains again on Tuesday for ALS Unfiltered, a discussion of the current state of medication research for ALS on Berkeley. It had been snowing on the summit in the morning, but we timed it right: leaving early afternoon we hit the tiniest amount of rain and no traffic problems all the way to Berkeley. We even had time to check into our hotel before the presentation.
ALS Unfiltered was put on by ALS TDI, the only nonprofit biotech pharmaceutical company dedicated solely to ALS. It was presented by Rob Goldstein, Vice President of Marketing, Communications, and Development and ALS.net Research News Reporter and Editor. He first discussed why ALS progresses differently in different people.
There are so many variables that can contribute to the disease and not all the variables contribute to each case.
He then talked about the gene mutation that my mother was confirmed to have. (I did finally get my blood work done and sent off to Washington University in St. Louis. I should have confirmation in about a month).
- C9orf72 is often called the most common genetic link to ALS known today (about 20% of those with genetically caused ALS have this repeat expansion mutation.)
- There are likely to be some up coming clinical trials in 2018 using antisense technology specifically for the C9orf72 gene expansion.
He then discussed some ALS drugs in the pipeline.
Edaravone is a drug approved for ALS in Japan and Korea. The FDA is supposed to rule on the U.S. approval in the next couple of months.
Tirasemtiv, developed by Cytokinetics in the San Francisco Bay Area, is well down the clinical trial pathline.
And then AT-1501, developed by ALS TDI specifically for ALS. It delayed disease onset and significantly extended life span in mice. It is currently being tested in primates before human trials can start. So it is exciting. But it is very expensive, as in $30 million, to bring a new drug to clinical trial. That is what ALS TDI is raising funds for now.
Stan and Andy are a team for the Corntoss Challenge on June 4 in Piedmont, California. There was a young man at the ALS Unfiltered presentation from the University of Oregon who will be married in May and he was just diagnosed. My friend Cory Reich was also diagnosed in college.
Please consider a donation to support Young Faces of ALS and ALS TDI. It is truly an unfair disease. You can donate by clicking this link; http://yfals.als.net/page.aspx?t=2413
After the presentation, Stan and I went with Jen McErlain of ALS TDI and Dallas Forshew of The Forbes Norris MDA/ALS Research Center (in SF) to an Irish pub a few blocks away where there was an open mike night.
There was some good music, some funny stand up comics, and it was wonderful to get know Dallas. She will be able to keep me informed of when the C9 antisense trials will start. She is a nurse who has been working with ALS patients for 30 years. Her center does clinical trials and she anticipates they will be doing that one.
We stayed at the Rose Garden Inn in Berkeley. At breakfast the next morning I had time to practice with my new camera.
Back home, on Thursday night I had an overnight pulse ox test ordered by UCSF.
On Saturday we were invited to a pre birthday celebration with Stan’s second family, the Hines, in Carson City. It was a love filled evening with lots of funny memories shared.
And then on Sunday we headed over the mountains again to go to a funeral in Palo Alto for our good friend Joannie’s uncle.
It has been a couple of weeks of lots of driving over the mountains but lots of love, connection, and hope too.