Baxter interested in funding PANDAS IVIG research

[momto2pandas]

So.... my immunologist is apparently quite connected at Baxter, maker of Gammagard IVIG, and he had a meeting with them a week or two ago where he approached them about supplying IVIG (for free) for a PANDAS clinical study with himself as Principal Investigator. He actually came up with this idea out of interest in studying me -- after I had that terrible Pneumovax reaction, he said that he would love treat me with IVIG first and then see if I still got the reaction - that it would be a great proof of concept for IVIG for PANDAS since I have the condition (and documented history) but not any significant background "noise" symptomatology to confuse things, so it could be kind of a "'clean" study. Not sure that makes sense (or that I would do it), but it got him talking to his Baxter buddies about PANDAS research, in any case.

 

Anyway, his Baxter connection told him that they were indeed very interested in pursuing this type of research. They suggested high doses (2 g or something like that) and asked him to submit a protocol describing the details of the PANDAS study he wanted to conduct. He told me that he is happy to be PI in the sense that he would deliver the treatment and would not charge a fee for being a research investigator, but he doesn't have the time to work up a protocol and all the other prep/paperwork stuff - and he is no expert in PANDAS, so he would want a neuropsychiatrist/specialist involved in clinical evaluation, etc.

 

When he first mentioned to me that he was planning to approach Baxter about a PANDAS study, I had offered to compile a protocol and do the admin for free if it would help to move things forward (this is my field so I've written and run loads of clinical trial protocols) and I gave him my CV to provide to Baxter in case he/they wanted to take me up on it. I was sure that they would not. I figured that Baxter must have been approached about PANDAS research before without success. But at my kids' appointment today he gave me the forms on which to submit a PANDAS IVIG protocol if I was still willing/interested.

 

Can it be true that no-one has approached Baxter in this way before? I don't mind blocking out time at some point to put a protocol together for my immunologist and Baxter (obviously nearly 100% gathering info from experts, since I am no IVIG expert whatsoever)...but can it really be true that no-one has previously done this and failed? My "real job" is already too busy and I don't want to waste my time if someone has already gone down this path and wound up at a dead end for reasons either scientific or political. I'm also wondering if my immunologist may just be well-connected enough to get this through, since obviously he wouldn't be the very best candidate out there for Investigator and yet still there is interest.

[MomWithOCDSon]

Wow! This is very exciting! I haven't heard of ANY clinical trials of any particular PANDAS treatment, only potential diagnostic tools, like the Cunningham study. I've often wondered why one of the pharmaceutical companies didn't get behind a trial, like GSK behind Augmentin XR or one of the immunoglobin providers behind IVIG.

 

I believe Dr. K. is a proponent of the higher dose IVIG (2 g), though I think I've heard that Latimer and some others prefer lower, more frequent IVIG treatments. Could Dr. K. add anything useful to the conversation, or would it create some conflict as another doctor will be leading the trial?

 

I admittedly know nothing about applying for or structuring a trial, but how fortunate for all of our families that this opportunity came your way, as you do! It is enough to make one believe in divine providence!

 

I'm sure you are beyond busy with all the demands of your day-to-day life, "real job," mothering PANDAS, etc. I, for one, would have to be directed very specifically and fundamentally, but if there is any assistance I might lend to any part of this process, please reach out! We might all have SO much to gain from the endeavor!

 

Thanks!

[matis_mom]

This is so encouraging! I don't have any answers to your questions but I do hope something works out. I makes sense for them to be interested. If IVIG is finally "proven" to work for PANDAS, then insurance companies will start covering (or one would hope!). All I can offer are my prayers that this goes through, and thank you so much for offering to put time into it

 

Isabel

 

PS: Sign us up as candidates, as our insurance does not cover IVIG for PANDAS! They do cover plasmapheresis, though... I wonder how much one costs compared to the other...

[momto2pandas]

Not to put a damper on, but I guess what I'm trying to say is that I'm not sure how encouraging it is. I just can't believe that this hasn't been tried before (going to Baxter for funding) and failed for some reason.

 

Unless someone tells me that it has failed, though, it's worth at least calling up his contact and assessing the situation more.

 

 

 

So.... my immunologist is apparently quite connected at Baxter, maker of Gammagard IVIG, and he had a meeting with them a week or two ago where he approached them about supplying IVIG (for free) for a PANDAS clinical study with himself as Principal Investigator. He actually came up with this idea out of interest in studying me -- after I had that terrible Pneumovax reaction, he said that he would love treat me with IVIG first and then see if I still got the reaction - that it would be a great proof of concept for IVIG for PANDAS since I have the condition (and documented history) but not any significant background "noise" symptomatology to confuse things, so it could be kind of a "'clean" study. Not sure that makes sense (or that I would do it), but it got him talking to his Baxter buddies about PANDAS research, in any case.

 

Anyway, his Baxter connection told him that they were indeed very interested in pursuing this type of research. They suggested high doses (2 g or something like that) and asked him to submit a protocol describing the details of the PANDAS study he wanted to conduct. He told me that he is happy to be PI in the sense that he would deliver the treatment and would not charge a fee for being a research investigator, but he doesn't have the time to work up a protocol and all the other prep/paperwork stuff - and he is no expert in PANDAS, so he would want a neuropsychiatrist/specialist involved in clinical evaluation, etc.

 

When he first mentioned to me that he was planning to approach Baxter about a PANDAS study, I had offered to compile a protocol and do the admin for free if it would help to move things forward (this is my field so I've written and run loads of clinical trial protocols) and I gave him my CV to provide to Baxter in case he/they wanted to take me up on it. I was sure that they would not. I figured that Baxter must have been approached about PANDAS research before without success. But at my kids' appointment today he gave me the forms on which to submit a PANDAS IVIG protocol if I was still willing/interested.

 

Can it be true that no-one has approached Baxter in this way before? I don't mind blocking out time at some point to put a protocol together for my immunologist and Baxter (obviously nearly 100% gathering info from experts, since I am no IVIG expert whatsoever)...but can it really be true that no-one has previously done this and failed? My "real job" is already too busy and I don't want to waste my time if someone has already gone down this path and wound up at a dead end for reasons either scientific or political. I'm also wondering if my immunologist may just be well-connected enough to get this through, since obviously he wouldn't be the very best candidate out there for Investigator and yet still there is interest.

[momto2pandas]

Thinking about this more and my immunologist and I are really not the ones who should be doing this. Neither one of us is a PANDAS expert by any means. If Baxter is really interested in funding this, presumably some of the experts (at least one?) would be interested in submitting a proposed protocol and running a study? Does anyone actually know? I'm not in that circle. There is a lot to running a clinical trial and maybe it's just too much for a busy practice focusing on patient care? Perhaps I could just get the name/contact info of the person who said that Baxter was very interested in this line of research and make an appropriate connection. I would rather pursue it than see the potential opportunity die if no-one else wants to pursue it, but my immunologist and I are definitely not the best ones to take advantage of this by a very long stretch.

 

 

 

So.... my immunologist is apparently quite connected at Baxter, maker of Gammagard IVIG, and he had a meeting with them a week or two ago where he approached them about supplying IVIG (for free) for a PANDAS clinical study with himself as Principal Investigator. He actually came up with this idea out of interest in studying me -- after I had that terrible Pneumovax reaction, he said that he would love treat me with IVIG first and then see if I still got the reaction - that it would be a great proof of concept for IVIG for PANDAS since I have the condition (and documented history) but not any significant background "noise" symptomatology to confuse things, so it could be kind of a "'clean" study. Not sure that makes sense (or that I would do it), but it got him talking to his Baxter buddies about PANDAS research, in any case.

 

Anyway, his Baxter connection told him that they were indeed very interested in pursuing this type of research. They suggested high doses (2 g or something like that) and asked him to submit a protocol describing the details of the PANDAS study he wanted to conduct. He told me that he is happy to be PI in the sense that he would deliver the treatment and would not charge a fee for being a research investigator, but he doesn't have the time to work up a protocol and all the other prep/paperwork stuff - and he is no expert in PANDAS, so he would want a neuropsychiatrist/specialist involved in clinical evaluation, etc.

 

When he first mentioned to me that he was planning to approach Baxter about a PANDAS study, I had offered to compile a protocol and do the admin for free if it would help to move things forward (this is my field so I've written and run loads of clinical trial protocols) and I gave him my CV to provide to Baxter in case he/they wanted to take me up on it. I was sure that they would not. I figured that Baxter must have been approached about PANDAS research before without success. But at my kids' appointment today he gave me the forms on which to submit a PANDAS IVIG protocol if I was still willing/interested.

 

Can it be true that no-one has approached Baxter in this way before? I don't mind blocking out time at some point to put a protocol together for my immunologist and Baxter (obviously nearly 100% gathering info from experts, since I am no IVIG expert whatsoever)...but can it really be true that no-one has previously done this and failed? My "real job" is already too busy and I don't want to waste my time if someone has already gone down this path and wound up at a dead end for reasons either scientific or political. I'm also wondering if my immunologist may just be well-connected enough to get this through, since obviously he wouldn't be the very best candidate out there for Investigator and yet still there is interest.

[Allison]

This is really interesting. I have long thought that it would be in Pharma's interest to "prove" IVIG as standard of care for PANDAS/PITAND so that the insurance companies will have no excuses and will have to pay for it. Basically, it's a "growth market" right now, and the insurance issue is a big barrier to entry.

 

I even called Baxter recently, especially as I saw posts on this board that they have a financial support service for anyone wanting to self-pay for IVIGs. I called our local hospital and found out the brands they use for IVIG, one was Baxter's and one was Gamunex. I left a message and although Baxter called me back, I didn't follow through with them. Kinda busy with pandas stuff...

 

But I did have success with Gamunex. They've offered to help talk with my insurance company, and also referred me to an insurance advocacy company called Paraplex. I haven't had time to follow up on these leads either. Anyone hear of them?

 

But they never said they would fund research -- that's what's really needed, so I'm really excited about this news from Baxter. Please keep us updated on how it goes, and don't be afraid to ask for help submitting the protocol, application, or forms or whatever you need.

 

I'm sure you'll find a lot of us willing to participate in any study too! Sign me up for sure.

 

Allison

[LNN]

I would contact Dr Leckman at Yale - he's discussing an IVIG study but I'm not sure where he is in the planning/finding process. I'd also contact Dr K for his input, as he's obviously the expert on IVIG protocol and Dr B, who also does IVIG for Pandas. Between the three of them, they'd have the most insight. Dr Leckman might also have research assistants available to help with the administration/data collection. I'll get you their contact info.

 

Great job networking! I really hope this goes somewhere!

[thereishope]

This is an exciting post to wake up to! The fact that a big company now has officially heard the word PANDAS and IVIG is great!

 

Awesome work!

[Dr_Rosario_Trifiletti]

This is an exciting post to wake up to! The fact that a big company now has officially heard the word PANDAS and IVIG is great!

 

Awesome work!

 

I can tell you right now that undoubtedly PANDAS will be the #1 use for IVIG in all of child neurology, and maybe high on the list of all pediatric diseases

 

There are quite a few other diseases in child neurology where IVIG is indicated, but they are rare compared to PANDAS

 

Why would Baxter not want to fund this, at least on a pilot scale ? ... the potential benefits to them could be enormous.

 

By the way, it's hard to get FDA approval and insurance coverage for treatment of a disease that officially "doesn't exist"! That's the first mountain to climb.

 

But I'm very excited by this.

 

 

Dr. T

[thereishope]

Can someone tell me how it is finally determined that a disorder warrants a diagnostic code? Who makes that decision?

[momto2pandas]

Yes, but if we can get PANDAS to "exist," then things move from hard to expedited, given that it is a pediatric, "orphan" (probably not truly - but at least for now the numbers would say so) disease. This is why I think that a very systematic database (perhaps filled at least partly by survey for reasons of practicality) on which one can do cluster analyses etc. to figure out appropriate criteria (and probably wind up removing the P and the S from PANDAS), would be a big step in the right direction.

 

Honestly, I predict that there will be a problem in trying to distinguish PANDAS from "regular" OCD, tic disorders, etc., on some fronts, since I'm guessing that the latter sample is liberally littered with people who belong in the former sample, making differentiation difficult.

 

I wonder if the way to approach a protocol is not to have it be on PANDAS per se but on autoimmune neuropsychiatric conditions of childhood meeting certain criteria (CAM Kinase/autoantibodies, DSM symptomatology, maybe immune irregularities, etc.). I still think getting hung up on the "S" in PANDAS makes the applicability of PANDAS findings more restricted than I think they should be. Mustn't there already also be a code somewhere for autoimmune neurological condition NOS, or something like that, and can't one get treated for it in a way that's appropriate to the clinical findings and available, replicated, peer-reviewed research, and get coverage? Maybe I'm being naive about the insurance situation in the US. I know that in places with more socialized medicine they rely more on research findings and expert opinions. IVIG is already covered for PANDAS in Canada, for example.

 

I also still think it would be important, in advance of putting together a protocol, to get some idea from existing cases of what traits separate those who get the best benefit from IVIG from those who don't, at least with respect to whether there are pre-existing deficiencies. If you get a chance to do a pilot you have to make sure that your inclusion criteria give it the best possible chance of success.

 

 

 

This is an exciting post to wake up to! The fact that a big company now has officially heard the word PANDAS and IVIG is great!

 

Awesome work!

 

I can tell you right now that undoubtedly PANDAS will be the #1 use for IVIG in all of child neurology, and maybe high on the list of all pediatric diseases

 

There are quite a few other diseases in child neurology where IVIG is indicated, but they are rare compared to PANDAS

 

Why would Baxter not want to fund this, at least on a pilot scale ? ... the potential benefits to them could be enormous.

 

By the way, it's hard to get FDA approval and insurance coverage for treatment of a disease that officially "doesn't exist"! That's the first mountain to climb.

 

But I'm very excited by this.

 

 

Dr. T

[kim]

Given Baxters involvement in vaccine distribution....might this part be a problem? Might your case be an argument for a different proff of concept? I truly ask this with all due respect. The prospect is very exciting in regards to knowledge gained in PANDAS research, but I can see an area here that might not be quite as exciting from Baxters perspective?

 

He actually came up with this idea out of interest in studying me -- after I had that terrible Pneumovax reaction, he said that he would love treat me with IVIG first and then see if I still got the reaction - that it would be a great proof of concept for IVIG for PANDAS since I have the condition (and documented history) but not any significant background "noise" symptomatology to confuse things, so it could be kind of a "'clean" study.

[Fixit]

Yes, but if we can get PANDAS to "exist," then things move from hard to expedited, given that it is a pediatric, "orphan" (probably not truly - but at least for now the numbers would say so) disease. This is why I think that a very systematic database (perhaps filled at least partly by survey for reasons of practicality) on which one can do cluster analyses etc. to figure out appropriate criteria (and probably wind up removing the P and the S from PANDAS), would be a big step in the right direction.

 

Honestly, I predict that there will be a problem in trying to distinguish PANDAS from "regular" OCD, tic disorders, etc., on some fronts, since I'm guessing that the latter sample is liberally littered with people who belong in the former sample, making differentiation difficult.

 

I wonder if the way to approach a protocol is not to have it be on PANDAS per se but on autoimmune neuropsychiatric conditions of childhood meeting certain criteria (CAM Kinase/autoantibodies, DSM symptomatology, maybe immune irregularities, etc.). I still think getting hung up on the "S" in PANDAS makes the applicability of PANDAS findings more restricted than I think they should be. Mustn't there already also be a code somewhere for autoimmune neurological condition NOS, or something like that, and can't one get treated for it in a way that's appropriate to the clinical findings and available, replicated, peer-reviewed research, and get coverage? Maybe I'm being naive about the insurance situation in the US. I know that in places with more socialized medicine they rely more on research findings and expert opinions. IVIG is already covered for PANDAS in Canada, for example.

 

I also still think it would be important, in advance of putting together a protocol, to get some idea from existing cases of what traits separate those who get the best benefit from IVIG from those who don't, at least with respect to whether there are pre-existing deficiencies. If you get a chance to do a pilot you have to make sure that your inclusion criteria give it the best possible chance of success.

 

I understand that studies would have to start with a certain set of perameters...

But as a paranoid mom..afraid of being left behind..

My DS would have been Your poster child for Pandas...tic, pos strep , abx, cured... repeat

now the last couple of times quick and long streps came back neg but i was able to get abx do to strep history and his presentation in office of some illness going on...now i didn't get his aso's checked or myco p....but none the less, abx, cured things again

Till this last onset..nothing on anystreps including aso and camK 105(sitting on the foul line)..but presetation with a doctor the day before onset with illness..

my concern would be he always presents with illness at time of onset....and we just found myco p, 11 months later...what if there were some other thing going on (maybe 2 things)that we coudln't find, yet....

Maybe it's the prior streps knocked his t-cells out of wack for ANY future illnesses????

 

just trying to keep things open, that's all

 

Kudos to you for all you're doing and keeping us informed

i'd gladly volunteer to you or Dr T if there is anything i can do....

other than feeding my kids and doing my minimal to get through each day...this is all i do anyway...

just let me know if i can help

[momto2pandas]

Good point! Though I guess that if the idea is that you could treat with IVIG before vaccination in a select group of vulnerable individuals for optimal benefit/risk profile, they get two hits in one there. Still, it calls to light the fact that the vaccine can set off a bad reaction.

 

Point was that Baxter stated that they were interested in this area. If there is an opportunity with Baxter, I'd actually rather see if we can get in a real (small) clinical trial, rather than a vaccine pre-treatment thing for me, in any case. I was just saying that this is where the connection started. To be honest, as I mentioned before, I'm not sure that I would do it - I would need to know a lot more than I currently know. For example, what if the IVIG did NOT prevent a reaction to the vaccine? And what if the reaction was worse this time around or harder to stop? Don't think I could take that risk. The last time around was really bad.

 

 

Given Baxters involvement in vaccine distribution....might this part be a problem? Might your case be an argument for a different proff of concept? I truly ask this with all due respect. The prospect is very exciting in regards to knowledge gained in PANDAS research, but I can see an area here that might not be quite as exciting from Baxters perspective?

 

He actually came up with this idea out of interest in studying me -- after I had that terrible Pneumovax reaction, he said that he would love treat me with IVIG first and then see if I still got the reaction - that it would be a great proof of concept for IVIG for PANDAS since I have the condition (and documented history) but not any significant background "noise" symptomatology to confuse things, so it could be kind of a "'clean" study.

[kim]

Can you prove it in a dish? Would one of Cunninghams ganglioside "everybody holding hands and getting along nicely" until the PANDAS sera was added, then they started "boxing each other," work (or something similar using your blood prior and post IVIG)?

 

As much as it pains me to say it, I'm just afraid if a vax of any kind were to be involved here, you will probably have a much harder time getting funding.

 

Though I guess that if the idea is that you could treat with IVIG before vaccination in a select group of vulnerable individuals

 

That is exactly what parents are clamoring for!!!!!! If you can get Baxter to acknowledge that there is a substantial group of individuals that are predisposed to adverse reactions, develope a test for it, well, I will host that party that others have talked about! The implications of acknowledging that scenerio tho are huge and none of the Pharma people have wanted to persue it, that I'm aware of. Seems it's much easier to stick with the rare rare event mantra.