COALITIONS AND COLLABORATIONS IN 2011
On January 24, 2011, 63 participants gathered in New York City for the International Consortium of Stem Cell Network (ICSCN) Workshop Towards Clinical Trials Using Stem Cells for Amyotrophic Lateral Sclerosis (ALS) / Motor Neuron Disease (MND).
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The following extract is from the portion of the workshop moderated by Dr Rick Bedlack of Duke University and ALS Untangled. The panel was comprised of Stephen Byer OF ALS WORLDWIDE and Dr. Brian Dickie of Motor Neuron Disease Association.
Panel A: Unregulated ALS Treatments and Public Education
Dr. Rick Bedlack, a neurologist and founder of ALSUntangled, moderated the panel session comprised of Stephen Byer and Dr. Brian Dickie. ALSUntangled uses social networking via twitter and email, and discussions occur via a website called a NING. Once a critical mass of knowledge about a therapy or clinic comes to light, the organization will begin an investigation, the results of which are published (to date nine) in an open access journal called Amyotrophic Lateral Sclerosis. ALSUntangled has created a minimal dataset of questions, vetted among scientists, to help identify the suspect treatments. The organization would welcome input from the stem cell community. Dr. Bedlack posed the question of how to measure success. For example, the community currently relies on clinical trials, and although these are subject to rigorous scrutiny, the fact remains that that published work is not always correct. Another way to measure success would be to find as much anecdotal evidence as possible from those who have undergone a procedure. In the final analysis, nothing replaces an onsite visit and patient follow-up for understanding the context and discerning whether patients are being treated in a way that is likely to create a benefit.
Stephen Byer profiled ALS Worldwide, an organization that serves as a communication link between patients and physicians. On behalf of the scientific community, ALS Worldwide endeavors to express the goals, needs, requirements and willingness to treat patients, and to that end is open to feedback from the scientific community. The organization has helped 6000 families in over 70 countries, and has visited more than 60 labs and hospitals in five continents. There are plans to make additional site visits to 30 facilities. ALS Worldwide has published its recommendations to help the patient community judge the most important criteria for the validity of ALS therapies.
Criteria for Judging the Validity of ALS Therapy
1. Is the therapy part of a trial or procedure in a hospital?
2. Is the trial or procedure subject to rigid IRB?
3. Is trial or procedure closely regulated by countries FDA or other regulatory agencies?
4. Are there carefully designed and mediated clinical blind trials in place?
5. Does the facility in question enjoy international recognition?
6. Does the staff have proven credentials or have they been falsified?
7. Is there a proven collection of data and specifically scheduled collection of data pre and post therapy?
8. Are the claims relevant and supported?
9. Is there detailed information available to families and observers?
10. Does the protocol purport to treat a single disease or is does one process treat everything?
11. Is the informed consent a complete, thoughtful and intelligent document?
Brian Dickie from the MND Association professed that ‘snake oil’ salesmen have been around forever but the difference today is that stem cell clinics and the internet have taken the issues to new level. The MND Association is in touch with 75% of people with ALS in England, Wales and Northern Ireland, and has dealt with significant activity around clinics offering umbilical cord and oligodendrocyte cell therapy in the Ukraine and unconventional, he stressed that MND Association’s perspective is to provide patients with information that will allow them to make informed choices. In the past, the organization has not asked its membership about the use of alternative therapies, but he suggested that patient associations could integrate appropriate questions within their membership surveys.
Despite the obvious issues with emerging stem cell clinics, the scientific community needs to recognize that there is lack of consensus, even in this form, about what constitutes a good scientific approach. Education is important and measures of success might take the form of information on websites, articles in journals, and patient booklets. Collaborating with ISSCR, The ALS Association, MND Association and other ALS organizations worldwide is a good tactic.
Read the entire report at: International Consortium of Stem Cell Network.
The Euan MacDonald Centre for Motor Neurone Disease Research is a website well worth exploring.
This Centre, devoted to both clinical and research efforts for ALS, MS and other motor neuron disease, is a superb example of how inspiration, dedication and funding can come together with proper focus and direction to create a superb environment for the study and effective treatment of motor neuron disease.
Spanish Translations Now Available: Because we now serve the ALS community in over 70 countries, many of them Spanish speaking societies, the need for our written materials to be available in Spanish has never been more important. Persons with ALS in Spain, Colombia, Argentina, Peru, Ecuador, Mexico, Honduras, Costa Rica, Chile, Uruguay, Venezuela, Puerto Rico, United States and other locales regularly request information about our efforts in their own language. The Methylcobalamin materials have been translated into Spanish and are now available. Spanish Translation Introduction
The documentary film, "Indestructible" is also now available with Spanish subtitles. Email us with your mailing address and request for your copy of the film. We look forward to hearing from you.
Dexpramapexole Phase III Clinical Trial:
Phase III of the Biogen Idec Dexpramapexole Clinical Trial is scheduled to begin in March or April 2011. If you are interested in participating, which we wholeheartedly recommend information about this drug and why it is being touted can be found at the NIH website and also on ALSWW website.
