More PANDAS Research Desperately Needed!

[MomWithOCDSon]

I realize it is controversial and we wouldn't want to compromise the perceived integrity of any findings or results, but what do you all think? Should we look to enlist the help/funding of some of the pharmaceutical companies like GlaxoSmithKline who makes Augmentin XR, or the maker of Azith, for more PANDAS research? I know these companies frequently fund studies, so why not ones on this "daring" and "pioneering" field of PANDAS?

 

A lot of what I read indicates that so much more research is needed, and some like Cunningham have to turn to parents to pay high prices for testing because they don't have the necessary funding to cover costs. Don't you think Big Pharma would jump at the chance if the results were likely to be that their products are viable responses to PANDAS? And isn't that what many of our posts here are recognizing, that abx DOES help?!?! Think of the sales volumes!

 

Am I nuts? Or maybe a day late and a dollar short and somebody with Big Pharma already has their hand in on the PANDAS research front? If so, somebody school me here! I'd love to know so I could talk 'em up instead of beating them up like I usually do (sick of all those TV commercials for prescription meds, for one thing!)!

[momto2pandas]

I'm a consultant in the pharma industry who has been doing some research on this topic (I consult or have worked full time as medical director in charge of immunology programs for many of the big players), and so far I don't know of any company that is actually working in this indication. I might be missing something, though, since patents have been submitted for immunological approaches to PANDAS-like syndromes. I am actually supposed to have a meeting next week to talk to a pharma executive in charge of a product that I think would be promising for PANDAS (and who is interested in hearing me out), but I'm going to have to reschedule since I haven't had enough time to prepare slides, etc. (my paid work and 2 [PANDAS] kids, unfortunately, have to take precedence...). I firmly believe that we could get pharma backing for this, and that it would be a great advance. The FDA even provides money and special incentives for companies to do research on "orphan indications," of which this is certainly one. I've developed products for orphan immunology and other indications before, so know how to navigate this process. Unfortunately again, though, the grant deadline is early Feb. I was going to try to submit a proposal on behalf of one of my clients, but simply won't have the time this year....

 

I've been wanting to try to get the main doctors who treat PANDAS together to go over research ideas and draft up some real proposals for pharma, but again, time has been limiting...

 

 

I realize it is controversial and we wouldn't want to compromise the perceived integrity of any findings or results, but what do you all think? Should we look to enlist the help/funding of some of the pharmaceutical companies like GlaxoSmithKline who makes Augmentin XR, or the maker of Azith, for more PANDAS research? I know these companies frequently fund studies, so why not ones on this "daring" and "pioneering" field of PANDAS?

 

A lot of what I read indicates that so much more research is needed, and some like Cunningham have to turn to parents to pay high prices for testing because they don't have the necessary funding to cover costs. Don't you think Big Pharma would jump at the chance if the results were likely to be that their products are viable responses to PANDAS? And isn't that what many of our posts here are recognizing, that abx DOES help?!?! Think of the sales volumes!

 

Am I nuts? Or maybe a day late and a dollar short and somebody with Big Pharma already has their hand in on the PANDAS research front? If so, somebody school me here! I'd love to know so I could talk 'em up instead of beating them up like I usually do (sick of all those TV commercials for prescription meds, for one thing!)!

[momto2pandas]

By the way, I don't think that there is any concern about the integrity of the results if they come out of, or are funded by, pharma. I know that's the popular perception and it was my perception before I joined industry as well, but at this point in my experience, having worked for more than a decade in each of academia and industry, I would say that the research done by industry is far and away more reliable than the research done in academia. I will spare you my rant on this topic. :-)

 

 

I realize it is controversial and we wouldn't want to compromise the perceived integrity of any findings or results, but what do you all think? Should we look to enlist the help/funding of some of the pharmaceutical companies like GlaxoSmithKline who makes Augmentin XR, or the maker of Azith, for more PANDAS research? I know these companies frequently fund studies, so why not ones on this "daring" and "pioneering" field of PANDAS?

 

A lot of what I read indicates that so much more research is needed, and some like Cunningham have to turn to parents to pay high prices for testing because they don't have the necessary funding to cover costs. Don't you think Big Pharma would jump at the chance if the results were likely to be that their products are viable responses to PANDAS? And isn't that what many of our posts here are recognizing, that abx DOES help?!?! Think of the sales volumes!

 

Am I nuts? Or maybe a day late and a dollar short and somebody with Big Pharma already has their hand in on the PANDAS research front? If so, somebody school me here! I'd love to know so I could talk 'em up instead of beating them up like I usually do (sick of all those TV commercials for prescription meds, for one thing!)!

[dcmom]

Somebody chime in, but I am thinking Tanya Murphy is doing a study in FLA involving antibiotics, and I also think Saving Sammy's doc from Mass is also doing a study.

 

I have to be honest here, I would NEVER put my kids in an antibiotic, or other medication study at this point. I wouldn't risk the possibility of placebo, or of something that might aggravate their condition. This may be selfish- but this is such a tricky disorder, I couldn't bear to rock the boat. In good concience I couldn't recommend another parent doing this either. Maybe there are some types of studies I would be comfortable with, but not the double- blind placebo type studies. Sorry.

 

I, of course, would like more research, though. Heck if someone just followed all of our kids...

[kim]

Recent study by norvartis

 

I think most of the research has been done in the hopes of developing a vaccine for strep, which would be a blockbuster... not RF, PANDAS etc. Seems this type of disease, with an autoimmune component, are the ones that have stood in the way of vaccine develop. ( a no thank you on that front for this family).

 

 

Protein array profiling of tic patient sera reveals a broad range and enhanced immune response against Group A Streptococcus antigens.

 

Novartis Vaccines and Diagnostics, Siena, Italy.

 

http://www.ncbi.nlm.nih.gov/pubmed/1962325...ogdbfrom=pubmed

 

QUOTE

This comparative analysis identified 25 antigens recognized by sera of the three patient groups and 21 antigens recognized by tic and pharyngitis sera, but poorly or not recognized by sera from children without tic. Interestingly, these antigens appeared to be, in quantitative terms, more immunogenic in tic than in pharyngitis patients. Additionally, a third group of antigens appeared to be preferentially and specifically recognized by tic sera. These findings provide the first evidence that tic patient sera exhibit immunological profiles typical of individuals who elicited a broad, specific and strong immune response against GAS. This may be relevant in the context of one of the hypothesis proposing that GAS antigen-dependent induction of autoantibodies in susceptible individuals may be involved the occurrence of tic disorders.

[thereishope]

Murphy is doing an antibiotic study. Geller is starting one in Feb.

[momto2pandas]

There are lots of ways of doing studies without placebo controls. These days, ethics guidelines actually prohibit doing placebo-controlled studies where there are existing/alternative treatment options available. It forces researchers to use more clever clinical trial designs, but it is routinely done.

 

 

Somebody chime in, but I am thinking Tanya Murphy is doing a study in FLA involving antibiotics, and I also think Saving Sammy's doc from Mass is also doing a study.

 

I have to be honest here, I would NEVER put my kids in an antibiotic, or other medication study at this point. I wouldn't risk the possibility of placebo, or of something that might aggravate their condition. This may be selfish- but this is such a tricky disorder, I couldn't bear to rock the boat. In good concience I couldn't recommend another parent doing this either. Maybe there are some types of studies I would be comfortable with, but not the double- blind placebo type studies. Sorry.

 

I, of course, would like more research, though. Heck if someone just followed all of our kids...

[MomWithOCDSon]

Somebody chime in, but I am thinking Tanya Murphy is doing a study in FLA involving antibiotics, and I also think Saving Sammy's doc from Mass is also doing a study.

 

I have to be honest here, I would NEVER put my kids in an antibiotic, or other medication study at this point. I wouldn't risk the possibility of placebo, or of something that might aggravate their condition. This may be selfish- but this is such a tricky disorder, I couldn't bear to rock the boat. In good concience I couldn't recommend another parent doing this either. Maybe there are some types of studies I would be comfortable with, but not the double- blind placebo type studies. Sorry.

 

I, of course, would like more research, though. Heck if someone just followed all of our kids...

 

I have to admit, I hadn't given the double-blind side of things much thought and I, like you, would not want my kid to suffer any more than he already has either. But we desperately need more research.

 

I'm pretty far removed from the process, to be honest. Does it take double-blinds for the scientific or medical community to accept results as valid? Or could freshly-diagnosed PANDAS kids not be given abx courses and studied and tracked for changes in their behavioral manifestations and bloodwork before, during and after, without potentially harming someone in the process by giving them a placebo or withholding the intervention? Would such results be considered valid? Or would they, as my psych likes to label much of the stuff I tell him, be considered only "anecdotal evidence."

 

And how does the process work to begin with, do you know? Does a doctor or team of researchers determine a project they'd like to address and then go after funding for it? Or could/would a pharm company itself take an interest in research of a certain kind and commence the research project, from A to Z, on its own? And what about human analogues for the studies, if they must be double-blind: mice or swine or something?

 

Mostly, the gist of my initial query was, would contacting one of the primary abx manufacturers along this vein yield any results?

[momto2pandas]

Studies do not need to be placebo-controlled to have an impact in the medical community, particularly for children. It helps, but it's not necessary.

 

The process depends on the goal. Both of the avenues you mentioned are used. If the product is already on the market (presuming it's a pharmacological treatment trial), then researchers can just decide what they want to do, design the study, apply for and hopefully get the funding (usually from NIH or some foundation), get approval from an ethics board (IRB), run the study, and publish the results. This process informs the medical community and may influence treatment, but does not lead to the drug being approved by the FDA for treatment of the disease, which in some cases means that insurance companies will call it "experimental" and won't cover it.

 

If, on the other hand, a pharmaceutical company decides to go after marketing approval for a drug - either a drug that is already on the market for other diseases, or one that is still being developed - then there is a specific process they need to follow, with dose-finding trials (what dose do you need to treat that particular disease?), efficacy and safety trials, etc., all with a number and variety (gender, age, race, etc.) of patients that the FDA deems adequate to prove that the drug is safe and effective. If the product is already on the market for something else, the requirements may be less than if it's a brand new product (since a lot of safety issues will already have been explored.) The study designs need to be approved by the FDA and ethics boards, and the pharma company then works with doctors at universities or in the communitty to treat their patients according to the study protocol. The data from all of the studies is then assembled and submitted to the FDA for a determination of whether the drug can be "approved" for marketing for that disease. This smoothes the way to insurance coverage. This process is generally much more definitive, but much more expensive, than drug trials typically initiated by single investigators.

 

If a drug is developed for an "orphan" disease, essentially one that meets criteria for being rare and under-treated, then the process for getting FDA approval is abbreviated and much faster and less expensive. FDA also provides funding to encourage companies to study these diseases, since they are rarely profitable once all of the research costs are taken into account.

 

That's an very abbreviated summary, but that's it in a nutshell.

 

-----

I have to admit, I hadn't given the double-blind side of things much thought and I, like you, would not want my kid to suffer any more than he already has either. But we desperately need more research.

 

I'm pretty far removed from the process, to be honest. Does it take double-blinds for the scientific or medical community to accept results as valid? Or could freshly-diagnosed PANDAS kids not be given abx courses and studied and tracked for changes in their behavioral manifestations and bloodwork before, during and after, without potentially harming someone in the process by giving them a placebo or withholding the intervention? Would such results be considered valid? Or would they, as my psych likes to label much of the stuff I tell him, be considered only "anecdotal evidence."

 

And how does the process work to begin with, do you know? Does a doctor or team of researchers determine a project they'd like to address and then go after funding for it? Or could/would a pharm company itself take an interest in research of a certain kind and commence the research project, from A to Z, on its own? And what about human analogues for the studies, if they must be double-blind: mice or swine or something?

 

Mostly, the gist of my initial query was, would contacting one of the primary abx manufacturers along this vein yield any results?

[momofgirls]

I have no idea where we would begin but how about an ivig study. Maybe the supplier of Gammaguard or Gammunex could help.

 

I realize it is controversial and we wouldn't want to compromise the perceived integrity of any findings or results, but what do you all think? Should we look to enlist the help/funding of some of the pharmaceutical companies like GlaxoSmithKline who makes Augmentin XR, or the maker of Azith, for more PANDAS research? I know these companies frequently fund studies, so why not ones on this "daring" and "pioneering" field of PANDAS?

 

A lot of what I read indicates that so much more research is needed, and some like Cunningham have to turn to parents to pay high prices for testing because they don't have the necessary funding to cover costs. Don't you think Big Pharma would jump at the chance if the results were likely to be that their products are viable responses to PANDAS? And isn't that what many of our posts here are recognizing, that abx DOES help?!?! Think of the sales volumes!

 

Am I nuts? Or maybe a day late and a dollar short and somebody with Big Pharma already has their hand in on the PANDAS research front? If so, somebody school me here! I'd love to know so I could talk 'em up instead of beating them up like I usually do (sick of all those TV commercials for prescription meds, for one thing!)!

[thereishope]

I may totally off here but does anyone remember reading that Swedo was interested in an IVIG study?

[thereishope]

I have no idea what the status of this is. I don't know if it's still a possibility or if it is now defunct.

 

http://www.latitudes.org/forums/index.php?...ic=5918&hl=

 

 

She (Swedo) and Leckman and Cunningham are fighting to get the IVIG study resurrected by this summer. Swedo says she understands now that we parents are loosely banded together and not under the HIPPA regulations of her past studies -- that with our help she can help right up clinical diagnostic guidelines for pandas treatment.

[Worried_Dad]

As others have mentioned, Dr. Geller at Mass General (thru association with PANDAS Foundation) and Dr. Murphy in FL are starting an augmentin study for PANDAS in the Spring. Beth Maloney told me this via e-mail a few days ago. Don't know if Glaxo is going to contribute the meds - is that customary for this type of study?

 

And Dr. Leckman at Yale was trying to get an IVIG study for PANDAS funded last year, but Talecris (maker of Gamunex) pulled out and the effort lost momentum. I think that study is dead (or at least comatose) at this point. Diana P could answer this better.

 

Man, I hope and pray that the burst of recent publicity will stimulate more interest from researchers and pharma. If Geller and others are right, there might be millions of potential patients who could benefit from proven treatments!

[thereishope]

Do you think that one the PANDAS "heavy weights" speak at the Autism One Conference, more funding will come the PANDAS way?

 

So, Murphy is doing Cefdinir now.

Geller is doing Cefdinir/Augmentin in Feb.

Then what is the spring one entailing? Is that the Cefdinir/Augmentin or a different one?