Two initial ideas for crowdfunding. #CrowdSpeak #MSBlog #MSResearch
"As a follow-up to my two posts this week on crowdfunding I have been thinking about the kind of projects that would fulfill our basic crowdfunding criteria:
Two topics based on interest from you the reader include the hematopoietic stem cell therapy (HSCT) trial and the Charcot project.
- Research done using crowd funding must come from the crowd.
- The research can't be funded by the usual routes of funding.
- Initial projects can’t be massive large scale projects; they should be small milestone driven projects; i.e. enabling projects.
The proposed ZEUS trial below has had a lot of interest from you the readers. To get this project off the ground, and funded, we would need to explore the community's attitude to the trial and whether it is addressing an unmet need. Exchanges and surveys done via this blog are not necessarily representative of the wider community. We as a group have no expertise in HSCT and we would therefore need to include groups and centres that do have expertise. Paolo Muraro's group at Imperial College would be a natural fit us. In fact Paolo has expressed an interest in leading this trial.
What we would need to answer are the following list of questions:
- Do MSers understand the immediate risks associated with HSCT?
- Do MSers understand the undefined risks associated with HSCT?
- Would MSers be prepared to be randomised to receive alemtuzumab or HSCT?
- Would British Neurologists be prepared to refer patients for a HSCT trial?
- Do haematology units in the UK have enough capacity to handle a large national MS-HSCT trial?
- What are the economics of HSCT? How do they compare to alemtuzumab treatment?
- Would a non-myeloablative or a myeloablative protocol be best?
- What is the optimal trial protocol?
- Would we only target DMT failures or MSers naive to DMTs?
- Would eligible MSers have to have highly active MS or just active MS?
- Would the NIHR or MRC be prepared to discuss funding a trial of this nature?
We as a group would want to focus on targeting both EBV and HERVs (human endogenous retroviruses) with antivirals in MS. Before doing a formal clinical trial we would need to do a small exploratory study using a combination of anti-EBV and anti-retroviral drugs. The primary outcome of the study would be to makes sure our anti-EBV drug is safe in combination with HAART (highly-active anti-retrovirals) and whether the combination were suppressing both EBV and HERV activity within the body. We would propose doing a small open-label study of approximately 20 MSers looking at EBV shedding, and EBV viral loads, and HERV activity in the peripheral blood before and after treatment with the combination of anti-virals. If positive data from this enabling study would be used as part of a grant application to fund a phase 2 trial assessing the efficacy of combination antivirals on MS disease activity. Ideally we would want the phase 2 study to be a parallel design placebo-controlled double-blind study, rather than the cross-over study we have just completed for raltegravir (INSPIRE TRIAL).
In the spirit of crowdsourcing we would appreciate your input on these initial ideas. This is a community project. Thank you."
Labels: Charcot Project, crowdfunding, CrowdSpeak, HSCT, ZEUS Trial