Baxter interested in funding PANDAS IVIG research

[Worried_Dad]

Yeah, about 18 months ago or so, Dr. Leckman was working closely with other docs (Dr. K, Swedo, and Cunningham, I think) to try to initiate a PANDAS IVIG study with Talecris, the maker of Gamunex. Diana P was gathering case histories from about 20-30 of us to help identify common symptoms, progression, family autoimmune histories, etc.

 

I remember hearing that Talecris backed out and decided not to provide the Gamunex for free. As far as I know, Dr. Leckman's been struggling ever since to find a pharma partner for this.

 

Mom2pandas, if you think Baxter's serious about this, I'd imagine that Dr. Leckman would be overjoyed to participate. I believe he's already done a lot of the groundwork in identifying a protocol (based on Dr. K's), doing some statistical analyses of the case histories / survey results of that original patient group, etc. And Dr. Leckman is a really, really compassionate person: he graciously agreed to speak with me on the phone several times to try to provide guidance for our son's treatment when things were darkest.

 

If you contact Diana P (Diana on the forum), she can probably give you more details. She's kept in touch with Dr. Leckman regarding this.

 

Man, I hope this works out somehow. That would be awesome!

 

 

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!

[momto2pandas]

I've been in touch with Diana P, not on this specifically, and she actually gave me an introduction to Dr. Leckman. Thanks for the details on the fact that this was Talecris. Given that Leckman et al approached Talecris, I would be surprised if they hadn't also tried Baxter, but I will call him on Monday to try to find out. Having spent most of my career on "the inside", I know that often it is a matter of finding the medical director within the company that has the interest and going directly there - and company priorities change a lot from year to year and with different people in the various medical director positions. Also, sometimes it does boil down to the specific relationships between the company and the doctors that they have or want to have relationships with. I'll try to find out from my immunologist who he's been talking to that claimed strong interest, and then find out from Dr. Leckman what path he's already tread with Baxter.

 

Yeah, about 18 months ago or so, Dr. Leckman was working closely with other docs (Dr. K, Swedo, and Cunningham, I think) to try to initiate a PANDAS IVIG study with Talecris, the maker of Gamunex. Diana P was gathering case histories from about 20-30 of us to help identify common symptoms, progression, family autoimmune histories, etc.

 

I remember hearing that Talecris backed out and decided not to provide the Gamunex for free. As far as I know, Dr. Leckman's been struggling ever since to find a pharma partner for this.

 

Mom2pandas, if you think Baxter's serious about this, I'd imagine that Dr. Leckman would be overjoyed to participate. I believe he's already done a lot of the groundwork in identifying a protocol (based on Dr. K's), doing some statistical analyses of the case histories / survey results of that original patient group, etc. And Dr. Leckman is a really, really compassionate person: he graciously agreed to speak with me on the phone several times to try to provide guidance for our son's treatment when things were darkest.

 

If you contact Diana P (Diana on the forum), she can probably give you more details. She's kept in touch with Dr. Leckman regarding this.

 

Man, I hope this works out somehow. That would be awesome!

 

 

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!

[kim]

That sure seems like great info Worried Dad (and others).

 

I went back through and realized that I hadn't read all of the posts on this thread before I replied the first time. I too hope that something useful can come of the interest that has been indicated to Mom2two.

[Worried_Dad]

Maybe you can leverage the recent media attention, too? Specifically, I'm thinking of Dr. Geller's quote on the cover of "Saving Sammy" stating that "may well hold the key to curing the lives of millions." If millions of children have this, and IVIG is proven to be an effective treatment... that could be a pretty sweet payoff for Talecris.

 

Good luck!

 

 

I've been in touch with Diana P, not on this specifically, and she actually gave me an introduction to Dr. Leckman. Thanks for the details on the fact that this was Talecris. Given that Leckman et al approached Talecris, I would be surprised if they hadn't also tried Baxter, but I will call him on Monday to try to find out. Having spent most of my career on "the inside", I know that often it is a matter of finding the medical director within the company that has the interest and going directly there - and company priorities change a lot from year to year and with different people in the various medical director positions. Also, sometimes it does boil down to the specific relationships between the company and the doctors that they have or want to have relationships with. I'll try to find out from my immunologist who he's been talking to that claimed strong interest, and then find out from Dr. Leckman what path he's already tread with Baxter.

 

Yeah, about 18 months ago or so, Dr. Leckman was working closely with other docs (Dr. K, Swedo, and Cunningham, I think) to try to initiate a PANDAS IVIG study with Talecris, the maker of Gamunex. Diana P was gathering case histories from about 20-30 of us to help identify common symptoms, progression, family autoimmune histories, etc.

 

I remember hearing that Talecris backed out and decided not to provide the Gamunex for free. As far as I know, Dr. Leckman's been struggling ever since to find a pharma partner for this.

 

Mom2pandas, if you think Baxter's serious about this, I'd imagine that Dr. Leckman would be overjoyed to participate. I believe he's already done a lot of the groundwork in identifying a protocol (based on Dr. K's), doing some statistical analyses of the case histories / survey results of that original patient group, etc. And Dr. Leckman is a really, really compassionate person: he graciously agreed to speak with me on the phone several times to try to provide guidance for our son's treatment when things were darkest.

 

If you contact Diana P (Diana on the forum), she can probably give you more details. She's kept in touch with Dr. Leckman regarding this.

 

Man, I hope this works out somehow. That would be awesome!

 

 

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!

[momto2pandas]

This is a really good idea. I don't know, but I'm guessing maybe. When I got into the issue with the immunologist yesterday where he said that insurance would not cover IVIG for my kids without revax, and I then refused to revax, he suggested that we could look at the kids' response to the vaccine in vitro at a research lab and assuming that it was awry, use that information to go forward with the insurance companies to justify coverage without revax. I think he said that there is a lab at UCLA that could do that? Not sure about that part. Anyway, I didn't pursue it because it sounded like a long and complicated and expensive path and I wasn't sure the insurance company would buy it in any case (what would be their motivation to do so?), and I figured that there must be some other way to approach the insurance that someone had already figured out. But obviously there are labs that can look at responses to vaccines (or, for that matter, bacterial challenges directly) and it would be fascinating to see results to such a clear-cut challenge in pre- and post-IVIG blood. If a lab could be found to do it, this would be a fascinating study to do as a piggyback study onto an IVIG study. For reasons you mentioned, though, I think funding that part would be tough.

 

I wonder how much it would cost. Maybe I am getting carried away here, but if we were able to get IVIG supplied for free as part of a clinical study, perhaps parents who could wouldn't mind chipping in for a piggyback study or two.

 

 

 

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.

[momto2pandas]

One of my concerns about IVIG is that it's a blood product and there can be supply issues... if millions of children (not to mention probably adults) need it, where are we going to get it? My understanding is that there have been IVIG supply issues in the past just trying to get enough to treat deficiency patients.

 

That's why I think that other, perhaps anti-inflammatory, approaches will need to be investigated eventually as well and we will need to really figure out if IVIG is actually "fixing" things by retraining the immune system or just providing an effect (helping with the BBB permeability) that could perhaps be gotten with other products that don't rely on the blood supply. Kim has really gotten me thinking about in vitro studies that could be done... though it's all kind of dreaming since I don't know that much about the limitations of in vitro studies of these kinds of things.

 

 

Maybe you can leverage the recent media attention, too? Specifically, I'm thinking of Dr. Geller's quote on the cover of "Saving Sammy" stating that "may well hold the key to curing the lives of millions." If millions of children have this, and IVIG is proven to be an effective treatment... that could be a pretty sweet payoff for Talecris.

 

Good luck!

 

 

I've been in touch with Diana P, not on this specifically, and she actually gave me an introduction to Dr. Leckman. Thanks for the details on the fact that this was Talecris. Given that Leckman et al approached Talecris, I would be surprised if they hadn't also tried Baxter, but I will call him on Monday to try to find out. Having spent most of my career on "the inside", I know that often it is a matter of finding the medical director within the company that has the interest and going directly there - and company priorities change a lot from year to year and with different people in the various medical director positions. Also, sometimes it does boil down to the specific relationships between the company and the doctors that they have or want to have relationships with. I'll try to find out from my immunologist who he's been talking to that claimed strong interest, and then find out from Dr. Leckman what path he's already tread with Baxter.

 

Yeah, about 18 months ago or so, Dr. Leckman was working closely with other docs (Dr. K, Swedo, and Cunningham, I think) to try to initiate a PANDAS IVIG study with Talecris, the maker of Gamunex. Diana P was gathering case histories from about 20-30 of us to help identify common symptoms, progression, family autoimmune histories, etc.

 

I remember hearing that Talecris backed out and decided not to provide the Gamunex for free. As far as I know, Dr. Leckman's been struggling ever since to find a pharma partner for this.

 

Mom2pandas, if you think Baxter's serious about this, I'd imagine that Dr. Leckman would be overjoyed to participate. I believe he's already done a lot of the groundwork in identifying a protocol (based on Dr. K's), doing some statistical analyses of the case histories / survey results of that original patient group, etc. And Dr. Leckman is a really, really compassionate person: he graciously agreed to speak with me on the phone several times to try to provide guidance for our son's treatment when things were darkest.

 

If you contact Diana P (Diana on the forum), she can probably give you more details. She's kept in touch with Dr. Leckman regarding this.

 

Man, I hope this works out somehow. That would be awesome!

 

 

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!

[Worried_Dad]

Good, pragmatic point. Bummer!

 

I see what you mean about finding other (hopefully cheaper) alternatives for treatment. The IVIG has put us pretty deep in the hole financially. FYI, was talking with Diana P yesterday: she recently met with some of the "PANDAS brain trust" (Leckman, Swedo and Grant from NIMH, Cunningham, not sure who else). They were hypothesizing that the kids who show dramatic improvement on high-dose abx may be benefitting more from an anti-inflammatory effect that an anti-bacterial effect, at least after a time. Guess they started brainstorming about using drugs like infliximab for PANDAS kids. Does that make sense to you?

 

So many questions... so few definitive answers. Makes my head hurt!

 

 

One of my concerns about IVIG is that it's a blood product and there can be supply issues... if millions of children (not to mention probably adults) need it, where are we going to get it? My understanding is that there have been IVIG supply issues in the past just trying to get enough to treat deficiency patients.

 

That's why I think that other, perhaps anti-inflammatory, approaches will need to be investigated eventually as well and we will need to really figure out if IVIG is actually "fixing" things by retraining the immune system or just providing an effect (helping with the BBB permeability) that could perhaps be gotten with other products that don't rely on the blood supply. Kim has really gotten me thinking about in vitro studies that could be done... though it's all kind of dreaming since I don't know that much about the limitations of in vitro studies of these kinds of things.

[T_Mom]

Good, pragmatic point. Bummer!

 

I see what you mean about finding other (hopefully cheaper) alternatives for treatment. The IVIG has put us pretty deep in the hole financially. FYI, was talking with Diana P yesterday: she recently met with some of the "PANDAS brain trust" (Leckman, Swedo and Grant from NIMH, Cunningham, not sure who else). They were hypothesizing that the kids who show dramatic improvement on high-dose abx may be benefitting more from an anti-inflammatory effect that an anti-bacterial effect, at least after a time. Guess they started brainstorming about using drugs like infliximab for PANDAS kids. Does that make sense to you?

 

So many questions... so few definitive answers. Makes my head hurt!

 

 

One of my concerns about IVIG is that it's a blood product and there can be supply issues... if millions of children (not to mention probably adults) need it, where are we going to get it? My understanding is that there have been IVIG supply issues in the past just trying to get enough to treat deficiency patients.

 

That's why I think that other, perhaps anti-inflammatory, approaches will need to be investigated eventually as well and we will need to really figure out if IVIG is actually "fixing" things by retraining the immune system or just providing an effect (helping with the BBB permeability) that could perhaps be gotten with other products that don't rely on the blood supply. Kim has really gotten me thinking about in vitro studies that could be done... though it's all kind of dreaming since I don't know that much about the limitations of in vitro studies of these kinds of things.

 

 

My own sense is that the antibiotics are having an anti-inflammatory effect and thus the results that we see -- (decrease in Ps symptoms, etc.)

 

Seems that Leckman would be an ideal candidate for helping--What an opportunity!

[nevergiveup]

Supply of ivig is not a concern here at least not for now, it takes years for medical practices to go mainstream. As we all already know. Anyway, talking with ivig specialist, he wasn't even concerned about the new data on Alzheimer and ivig and its supply. He felt the production will match demand. It is blowing docs minds, they only wish they knew why is helps alzheimer. So invest in Talecris. Anyway, for autoimmune disease its the globins that inactivate the production of antibodies that work for autoimmune disease, that randomly are found in about 5 percent of the globins in each dose. Hence the higher the dose the more inactivation globins you receive turning off your autoantibodies. Currently some of the companies are looking into separating out these inactivation globins from the rest, (by molecular weight?) therfore having an ivig product( or monoclonal antibody product) that shuts off the production of antibodies without all the other junk not necessary for autoimmune disease. Anyway, the opportunities are endless. I wonder if Talecris stock is going to rise any higher ?

 

Momto2pandas,

Pleae contact Leckman with your contact info they want another study. Also call Tanya Murphy in USF and see if they are interested in talking with your contact. At least its gets the right parties talking.

[T_Mom]

This is good news--

and the results may provide additional insights. Leckman/Swedo--

[momto2pandas]

Yes, I agree about the anti-inflammatory effect. I think that's why my kids seem to feel and look dramatically better on Azith even though it doesn't seem to be killing whatever infection this is that they can't get rid of, and why they start to look bad (circles under eyes, etc.) within a couple/few days off.

 

Yes, the "biologics" (like infliximab, etc.) and other RA drugs were my babies when I worked in rheumatology clinical development. I've headed up clinical development programs on a few of them (not including infliximab), some of which are now on the market. I've been thinking about these for PANDAS for a long time and wanting to get feedback on their use from some experts. I still consult for some of the companies that make them. I actually had a meeting arranged to talk with a VP at one of the companies about it, but had to cancel when I got too swamped with other work for that same company. One issue is - they are generally contraindicated in people with known serious infections or immunodeficiencies, since the theory is that inhibiting inflammatory cytokines inhibits the immune response -- and serious infections have been seen in people taking these drugs. Given that I think a lot of PANDAS patients already have infections that they're having a hard time with and/or have immunocompetence issues, they probably wouldn't be a good choice for that group. Probably not too different from steroids in that regard. However, I think there may still be a subset of patients for which they could produce relief. Dr. T's theory about a "hyperimmune" subset comes to mind. These drugs are expensive, too.

 

And I have wondered about COX-2 inhibitors, which in theory should also work and which are much less expensive, since they aren't biologics. The fact that Advil (a COX-1 and COX-2 inhibitor) helps so many suggests that the COX-2 inhibitors could help more. Don't have any direct contacts to talk to in that area, though.

 

 

Good, pragmatic point. Bummer!

 

I see what you mean about finding other (hopefully cheaper) alternatives for treatment. The IVIG has put us pretty deep in the hole financially. FYI, was talking with Diana P yesterday: she recently met with some of the "PANDAS brain trust" (Leckman, Swedo and Grant from NIMH, Cunningham, not sure who else). They were hypothesizing that the kids who show dramatic improvement on high-dose abx may be benefitting more from an anti-inflammatory effect that an anti-bacterial effect, at least after a time. Guess they started brainstorming about using drugs like infliximab for PANDAS kids. Does that make sense to you?

 

So many questions... so few definitive answers. Makes my head hurt!

 

 

One of my concerns about IVIG is that it's a blood product and there can be supply issues... if millions of children (not to mention probably adults) need it, where are we going to get it? My understanding is that there have been IVIG supply issues in the past just trying to get enough to treat deficiency patients.

 

That's why I think that other, perhaps anti-inflammatory, approaches will need to be investigated eventually as well and we will need to really figure out if IVIG is actually "fixing" things by retraining the immune system or just providing an effect (helping with the BBB permeability) that could perhaps be gotten with other products that don't rely on the blood supply. Kim has really gotten me thinking about in vitro studies that could be done... though it's all kind of dreaming since I don't know that much about the limitations of in vitro studies of these kinds of things.

[peglem]

Any updates on this?

[7upMom]

Also- this is prob a dumb ? but since PANDAS isn't an "on the books" disorder how is it Canada has it as something they pay for then? Does Canada have a protocol for what they consider PANDAS and we just don't here- how confusing and strange

[momto2pandas]

No updates. I got overwhelmed with work in the spring but am getting back to PANDAS now - have reserved a bunch of time this summer to try to make progress on such leads.

 

Re. Canada, I also thought it was interesting that it's covered there. Countries with nationalized health care typically follow "treatment guidelines" put together by experts based on literature review, and they don't have to follow what's e.g. FDA approved or "on the books" or whatnot. Obviously there must be experts that Canada drew upon who are believers and who considered the research that was out there and the fact that antibiotics and psych meds don't work for everyone. I recently heard that Denmark also pays for even CamKinase etc. testing for citizens with suspected PANDAS, and that's also considered experimental at this point.

[PKM]

Also- this is prob a dumb ? but since PANDAS isn't an "on the books" disorder how is it Canada has it as something they pay for then? Does Canada have a protocol for what they consider PANDAS and we just don't here- how confusing and strange

 

As a Canadian I don't get it either. I don't get why it is even listed as treatment for PANDAS here in Canada because my son's neurologist has been trying for a very long time to find someone in Canada with some experience using IVIG for PANDAS or post-strep problems (Sydenham's etc.). VERY FRUSTRATING!!!!!!!! So, it is listed here in Canada as a treatment option for PANDAS but apparently there are no Canadian doctors actually using it!!!!!!!!!!!!!!!!

 

(Sorry - just venting a little!) But if anyone in the USA thinks Canadian kids are actually getting IVIG treatment for PANDAS - it seems that this is not the case. In fact we just got back from a medical trip to Calgary (that Manitoba Health sent us on) only to be told that no one in Canada has PANDAS etc as an area of expertise - that my son would have to go to the USA or to London, England to be seen and treated.

 

PKM

Edited July 3, 2010 by PKM