ALS Advocacy

ALS Advocacy
Lou Gehrig's Disease - Motor Neuron Disease - Amyotrophic Lateral Sclerosis
Thought it had been cured by now? Still no known cause. Still no cure. Still quickly fatal. Still outrageous.

Sunday, November 30, 2014

What Should I Talk About?

That's every speaker's quandry.

There is a wonderful old story about the young, new preacher who arrives in a small town in Indiana. He is replacing a long-time fixture and is worried about his first sermon. He goes around to the barber and the banker and the grocer and asks, "What should I talk about?" He gets much guidance. Then he goes to the little old lady who is known to be the most opinionated person in town. "What should I talk about?" She gives him the wisest advice of all, "You should talk about God, and you should talk about five minutes."

So often at ALS MND meetings speakers prepare all kinds of intricate material when there are hundreds of questions that those with ALS and their caregivers would like to ask and have discussed.  There is never enough time for Q and A at the end of presentations, and many questions go unanswered.  Maybe they should just talk about ALS and talk about five minutes and let the questions lead the way.

This week prior to the big ALS MND Symposium in Brussels, there will be a two-hour "Ask the Experts" session for patients and caregivers to ask questions.  We asked, and they are accepting submitted questions via ALSMNDquestions@gmail.com .  Some patients and caregivers who participate in the research forum at als.net asked me to submit the following list of varied questions:

  1. Do you have any ideas why the developers of Generveron are not participating in the Symposium? As a most exciting announcement this year of halting or slowing progression in 7 of 8 PALS in a trial, it seems incredible that the subject does not appear to be anywhere on the agenda.
  2. The idea of a GUID for de-identified patient-level data in ALS data repositories seems like the ultimate common sense to me, especially with the proliferation of new, similar iPS projects. Please share your thoughts on what it will take to implement a Global Unique Identifier in ALS project databases.
  3. What can we learn about speeding up drug development from the Ebola crisis? Bill Gates recently made some comments about needing better global disease surveillance. He and others have suggested that there are ways to gather powerful clinical evidence without placebo arms. It seems to bother people a lot to suggest a placebo for Ebola patients but yet placebo is just fine for someone with ALS. Your thoughts, please?
  4. Has it become customary practice in clinical trials to sequence the DNA of the participants? If so, whole genome or just for specific known and suspected genetic markers? And if so, what is the customary practice regarding providing participants with those results? And what is the customary practice with respect to releasing the data to ALS genetic databases?
  5. Regarding the Cytokinetics… It is frustrating to me when a trial fails and then gets redesigned for a costly and time-consuming do-over. Have any other trials used SVC as a primary endpoint? Are you allowed to go into a trial with a premise that a drug could positively A or B (alternative primary endpoints)?
  6. If standard-of-care were not an issue, should every clinical trial have a branch without riluzole? Is it possible or likely that riluzole adversely effects efficacy of other treatments?
  7. If you had ALS, what would you be trying?
  8. For years ALSA has cited multiple studies of a much higher incidence of ALS among those who have served in the US military.  Have there been such studies of those who have served in other countries’ military?
  9. What ideas do you have for the next year’s equivalent of this year’s IBC?
  10. If an MND patient is getting a balanced diet, are vitamin and mineral supplements a waste of time and money?
  11. How important are large-animal MND studies. Have any large animals with ALS-related conditions ever been successfully treated?
  12. How important will bioinformatics become? Will Terry Speed be helping with MND-related research in the near future?
  13. Why is it so difficult for any research lab to win the $1 million Prize4Life prize for extending the lives of SOD1mice by 25%
  14. A $1 million Prize4Life prize was awarded for a Biomarker Tool that does not seem to be very useful. Do you have any updates on this tool or other biomarkers in the pipeline?
  15. Do you think that there’s any future in exoskeletons (and 3D printers to make them cheaply) for MND patients?
  16. In the panel of experts suddenly developed MND, what would be the one main question that they would ask? And how would the others respond?

We look forward to the discussion of the answers.



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