Ask The Experts and fALS Meeting at UCLA

Happy to have helped organized and initiated a fALS Meeting prior to the Ask The Experts Meeting Saturday at UCLA.

Approached both ALSA and the MDA six months ago and asked them to work together and get as many patients and their families to this as possible. Glad they worked together closely and pulled it off. Will continue to encourage more of these meetings be recorded and streamed live in the future so PALS unable to attend will be able to view.

My fellow PALS Jim Barber has worked tirelessly to promote the California ALS Research Summit, and the list of researchers involved is impressive. Getting them to stay around late on a Saturday night is proof of their commitment to engage with the PALS community.

Many of us are pleasantly surprised at the amount of activity regarding active clinical trials, link here, and those in the works shortly. Dexpramipexole, NP001, Isis - SOD1 and the Neuralstem/ Emory trials in particular look very promising to me.

Promising Developments in ALS Research Discussed at Special Event Co-Hosted by MDA and The ALS Association Golden West Chapter

By Jenica Lancy (October 12, 2011- Los Angeles, California)

Following the second annual California ALS Research Summit, The ALS Association Golden West Chapter and the Muscular Dystrophy Association held a special event open to the public with ALS experts discussing the most promising developments in familial and sporadic ALS research. The evening opened with a facilitated conversation to increase awareness of key issues facing people with familial ALS (fALS) and their families, led by Steve Gibson, Chief Public Policy Officer for The ALS Association and by Greg Merfeld, advocate and person with fALS. Link to Video “This was the first of hopefully many meetings of this type,” said Merfeld. “While the fALS population is relatively small in size, we have an enormous role to play in the ALS community.” “It was a great opportunity to come together and learn about the exciting developments in research and public policy,” shared Gibson. “Our goal for our presentation was to encourage volunteerism, advocacy, clinical trial participation by both the patient and family members, and provide another layer of support and resources," said Merfeld. Steve Gibson and Greg Merfeld, leading the fALS session The evening continued with a program titled, “Ask the Experts -Commitment to a Cure.”  The event was moderated by Dr. Lucie Brujin, Chief Scientist for The ALS Association, with opening comments by Fred Fisher, President and CEO of The ALS Association Golden West Chapter, both of whom are members of the California ALS Research Network. “It is an incredibly hopeful time for ALS,” Brujin said. “There is so much going on in the research arena and such brilliant minds working on it. " Dr. Robert Miller and Dr. Lucie Brujin, listing to questions

Dr. Seward Rutkove  during his presentation "What you will hear today will reflect some of that excitement and give you an opportunity to ask your questions directly of the researchers," said Brujin. The program the following featured presentations: Dr. Robert Miller, Medical Director, Forbes Norris MDA/ALS Research Center, “Clinical Trials for ALS”Link to Video Dr. Clive Svendsen Director of Regenerative Medicine Institute, Cedars-Sinai, “Overview of the ALS California Research Summit"Link to Video Dr. Richard Smith reviewing the ISIS Study Dr. Bob Baloh, Assistant Professor of Neurology, Washington University, “The Role of the Protein TDP43 in ALS” Link to Video  Dr. Richard Smith, Director, Center for Neurologic Study, “ISIS Study Update”Link to Video  Dr. Seward Rutkove, Chief of the Division of Neuromuscular Disease, Beth Israel Deaconess Medical Center Winner of the Prize4Life ALS Biomarker Challenge. Link to Video

“The ‘Ask The Experts’ was a very good conclusion of the Research Summit, as several excellent speakers could stay on and inform the ALS community,” said Dr. Martina Wiedau-Pazos, Assistant Professor of Neurology, UCLA School of Medicine and member of the Network. “I would like to continue this model for future California ALS Research Summits,” said Svendsen, who also serves as the Chair of the California ALS Research Network. “Knowing that we will be rotating the Summit location each year will enable us to access different patient groups in different geographic areas. I am very committed to presenting this emerging science directly to patients and families. It is essential to those who are holding on through this disease and relating their clinical experience to us.  The scientists are very indebted to the ALS community and I think many would really like to talk one-on-one with them,” he said.

More than 150 people with ALS and their families were in attendance, through the invitation of The ALS Association and The Muscular Dystrophy Association.  Many traveled long distances from areas like Bakersfield, Fresno, San Diego and San Francisco to be at the UCLA campus in person for this event. The event was also the Chapter's first live web cast, and to date, more than 1760 streams, of which 245 unique viewers from 9 countries have been able to view the presentations. The videos are all now on the Chapter's YouTube Channel.

“I was very excited that both The ALS Association and Muscular Dystrophy Association co-promoted this event,” Merfeld shared.  “It is truly all about the patients and to have both national organizations reach out to the patient community was outstanding. "The ALS Association's efforts to live stream this event via the Internet enabled questions to be asked and answered from both those in attendance and from the audience watching.  It created a great atmosphere for all.  Many people with ALS and their families have great difficulty in attending these key meetings in person and the ability to participate live, whenever and wherever these events take place, was a godsend." Participants from the webcast chatted in their words of appreciation. “We want to express our thanks for the opportunity to listen, to learn, and to participate,” said one. "We appreciate the efforts on the part of the scientists, sharing their time and expertise,” said another. "I was encouraged to see so many recent advancements in somewhat rapid succession," said Linda Della who's husband's family has experienced three generations of ALS. "It is also so helpful to be able to attend these meetings via webcast and stay connected to the latest breakthroughs that may help us understand the many causes of familial ALS."  Event organizers and speakers agreed about the importance of broadening the audience for these educational events. “Through the continued involvement of people with ALS and their families, we can make even greater progress in the fight to find a treatment as soon as possible,” said Gibson. At the evening’s conclusion, Jim Barber, Co Chair of the California ALS Advocacy Committee and a person with ALS, offered his personal insights. “I would like to extend my thanks to my wife Cheryl and to extend that thanks to all of the other families and caregivers who helped their loved ones attend this event tonight.  There have been more breakthroughs in research since I was diagnosed four years, than have been in the history of the disease.  The members of California ALS Research Network are absolutely first rate and worthy of our support.”

“There are many people with ALS and their families who, like Jim Barber and Greg Merfeld, are engaged in the fight against this disease,” said Fred Fisher, President and CEO, The ALS Association Golden West Chapter.  “We invite you to join with them in their efforts and others across the country as we continue to fight this disease.  We are here for you in this fight.  It is very exciting to be a part of this time in ALS research.” To view more photos, please visit our flickr site.