Worldwide HSCT Roadmap and Funding

[CarpeDiem2015]

My main question is who out there is thinking about how to bring this treatment to more people? And if there isn't a formal organization or research center, then perhaps the MS community of patients and caregivers who are interested in HSCT perhaps can begin to get this on the table.

In summary, at this time the success of HCST and related treatments has appeared to hit the mainstream; or at least the news of this treatment is reaching out to more folks with MS (such as with the recent press release amplified by social media (e.g. this forum, the HCST facebook page, and prior media reports such as 60 Minutes Australia). It's great that this has done well in clinical trials; and that similar treatments in Russia also appear to be helping folks with MS. This brings up a few questions and observations:

(1) what are the similarities and differences between the various treatments in the U.S., Russia, and elsewhere?

(2) can HSCT for MS be approved sooner? In the U.S. / FDA approval appears a few years off. I don't know for other countries.

(3) if/when this does become FDA approved, how easily can this treatment be administered by other MS and related centers, hospitals? Will the inventors / researchers hold a "patent" on the HSCT procedure and license it out? How is this done now for similar treatments?

(4) it seems traveling to Russia, or the like, will be the only way to get this type of treatment in the near couple years. I have read where there is a waitlist for Russia out to 2017 - is this true?. If there is increased demand, will the doctors/healthcare center in Russia expand? I realize there are a couple other places that can do this treatment; though it doesn't seem too many are making this available?

(5) will existing bone marrow facilities and/or new companies begin to offer HSCT for MSers (perhaps in countries that can approve this fast)? How hard would it be for the chemo / auto-immune reboot procedure to be replicated? Have the expert researchers published enough that it could be done? There appears to be many bone marrow transplant centers around the world. Or am I off the mark on bone marrow centers being relevant?

(6) The cost of this treatment is in $50K to $150K; and there would seem to be many folks who would like to have this treatment there are a couple questions. First, why is it $150K in the US and $50K in Russia? If the number of people interested in this treatment are hundreds, or even thousands, of people who want this treatment, then cumulatively that's a lot of money. It seems the R&D part is more or less done. What expenses are there to get a hospital up to speed? Is it mainly to train doctors? and/or to buy special equipment?

(7) MS sub-types -- some word out of Russia is that their HSCT procedure may help those who have been diagnosed as PPMS or SPMS; whereas in the US it seems the treatments are for RRMS only. How are these two treatments similar / different. As more folks are treated in Russia perhaps this will become better known as to the effectiveness for PPMS and SPMS.

I realize there are many unanswered questions, and that the above is very simplified. However, I figured to post this now to start a discussion as their appears more interest in HSCT. Lastly, given that we can connect better via forums, Facebook, perhaps at some point the community can pull together to hopefully speed up the availability of this treatment for everyone whom this could benefit.

#MSlivesmatter

[ElliotB]

There are valid reasons for clinical trials. And for not fast tracking most new drugs and procedure/protocols. This new procedure basically requires wiping out the immune system with powerful drugs through Chemotherapy. Dr. George Georges, a transplant expert involved with this "cautioned that stem-cell transplants may not be the solution for the vast majority of MS patients. There are real risks to the procedure and it can only be considered for people who have exhausted all other options"

There are currently protocols that are totally safe and seem to effective that the vast majority of MSers choose to ignore. This new therapy may prove to be effective and promising for many with MS and other illnesses. Only time will tell. I for one will wait for the testing to complete and until I have exhausted all other known safe options.

There are also many clinical trials going on for other promising drugs. Patience and diligence are necessary at this point in time, IMHO.

One final thought not related to the treatment: "Kearny is back to working 50 hours a week" I guess he didn't learn much about life from his MS - I am sure many of you know what I mean (hopefully).

[ElliotB]

Seeve also linked info about this procedure and that article states "the data so far does suggest that the treatment’s effects may not be sustained over time.".

MS is a complex illness and I think until the root cause is found we will just have to be patient.

[CarpeDiem2015]

I agree. Given that not everyone is seeking to have HSCT given DMT's, a question is how many MSers could currently benefit from HSCT? What is that number? A hundred, thousand, ten thousand, more? There are a reported 2.5M people with M.S. worldwide (400,000 in the U.S.).

- There are some folks who are RRMS that see HSCT as a way to stop things before things get worse since current drugs may not be effective. In addition to Dr. Nash's study, there are the efforts of Dr. Burt and his team at Northwestern; however I have yet to dig into the similarities / differences on their treatments. Also, their studies screen out people do to a variety of criteria - who is to say that HSCT wouldn't help more people beyond their current groups.

- For PPMS and SPMS there currently are no FDA approved options. There are a few drugs in clinical trials; and the recent statin clinical trial showed some promise of slowing brain atrophy.
- Also, if the approach developed by Dr. Fedorenko and his team in Russia is doing something different than other researchers; and Dr. F's treatment helps PPMSers, then this should be looked into since Dr. Burt has said his treatment is for RRMS.
- There are a few studies that show a couple different PPMS sub-types; some that might respond to immune-related treatment.