Something magical is going on here

[Dr_Rosario_Trifiletti]

Hi everyone,

 

 

It just dawned on me ...

 

Traditionally clinical research is done at some large institution, say the (John) Mayer Clinic (names changed to protect the innocent)

 

1. Dr. X, usually based on prior or often his own basic research formulates a hypothesis. Patients' ideas and observations don't figure much if at all in this step.

2. Dr. X attempts to "recruit" patients into his study, which must first pass an Institution Review Board, and many logistical hurdles. All sorts of safeguards are placed to ensure that the Mayer Clinic does not get sued.

3. Many months later, once those logistical hurdles are cleared, Dr. X opens his study

4. A year later, enough people have called Dr. X's office so that he has enough data to write a pilot grant to the NIH

5. Six months later, Dr. X finds out that his grant is just short of the threshold for funding

6. He submits another version, and prays - the Mayer Clinic is beginning to put pressure on Dr. X to bring in funding. After much white-knuckling, Dr. X finds his study is funded

7. Several years later Dr. X completes his study and finds out that his hypothesis is wrong

8. Dr. X is "non-renewed" (i.e. fired) by the Mayer clinic

 

Dr. Y, newly hired by the Mayer clinic to replace Dr. X, is inspired by Dr. X's work, and decides to embark on the same program as Dr. X

 

Dr. X, although disheartened, decides to try something new ....

 

There is something magical going on right here which might be a model for the way we do clinical research in the future:

 

1. Parents of patients (or with adults, patients themselves) tell their histories, make observations, discuss lab values

2. Interested doctors observe the proceedings

3. Doctors and parents formulate new hypotheses

4. Doctors set in motion ways to test the hypotheses by testing and treating patients (there's the rub)

5. Communal feedback on test results and treatments

6. Refine hypotheses and treatments to obtain improved diagnosis and treatment

 

What is the role of outside parties like the government and instiutions? really two-fold that I can see (but they are huge): ensure confidentiality and safety.

This has got to be incorporated in version 2.0 of the proces.

 

 

In the past 2 months alone, we've learned about mycoplasma and the p41 (anti-flagellin) connections by the new process. I'm sure there is more to come.

 

Can you appreciate how this rapid feedback iterative model VASTLY accelerates early clinical research? In the past, local doctors did this one patient at a time

 

Now, by using the "social networking" features of the internet, we can do it on large populations, i.e. statistically significant numbers.

 

The revolution will not be televised. But it will be broadcast on the internet and you are part of it!

 

Dr. T

[thereishope]

I do wonder how many docs look at these postings but are nervous to chime in. I'm so happy you put youself out there. It says a lot about you. You are a good man and a great doctor!

[momaine]

Hi everyone,

 

 

It just dawned on me ...

 

Traditionally clinical research is done at some large institution, say the (John) Mayer Clinic (names changed to protect the innocent)

 

1. Dr. X, usually based on prior or often his own basic research formulates a hypothesis. Patients' ideas and observations don't figure much if at all in this step.

2. Dr. X attempts to "recruit" patients into his study, which must first pass an Institution Review Board, and many logistical hurdles. All sorts of safeguards are placed to ensure that the Mayer Clinic does not get sued.

3. Many months later, once those logistical hurdles are cleared, Dr. X opens his study

4. A year later, enough people have called Dr. X's office so that he has enough data to write a pilot grant to the NIH

5. Six months later, Dr. X finds out that his grant is just short of the threshold for funding

6. He submits another version, and prays - the Mayer Clinic is beginning to put pressure on Dr. X to bring in funding. After much white-knuckling, Dr. X finds his study is funded

7. Several years later Dr. X completes his study and finds out that his hypothesis is wrong

8. Dr. X is "non-renewed" (i.e. fired) by the Mayer clinic

 

Dr. Y, newly hired by the Mayer clinic to replace Dr. X, is inspired by Dr. X's work, and decides to embark on the same program as Dr. X

 

Dr. X, although disheartened, decides to try something new ....

 

There is something magical going on right here which might be a model for the way we do clinical research in the future:

 

1. Parents of patients (or with adults, patients themselves) tell their histories, make observations, discuss lab values

2. Interested doctors observe the proceedings

3. Doctors and parents formulate new hypotheses

4. Doctors set in motion ways to test the hypotheses by testing and treating patients (there's the rub)

5. Communal feedback on test results and treatments

6. Refine hypotheses and treatments to obtain improved diagnosis and treatment

 

What is the role of outside parties like the government and instiutions? really two-fold that I can see (but they are huge): ensure confidentiality and safety.

This has got to be incorporated in version 2.0 of the proces.

 

 

In the past 2 months alone, we've learned about mycoplasma and the p41 (anti-flagellin) connections by the new process. I'm sure there is more to come.

 

Can you appreciate how this rapid feedback iterative model VASTLY accelerates early clinical research? In the past, local doctors did this one patient at a time

 

Now, by using the "social networking" features of the internet, we can do it on large populations, i.e. statistically significant numbers.

 

The revolution will not be televised. But it will be broadcast on the internet and you are part of it!

 

Dr. T

That would be fantastic if other doctors learned from what you are doing. My concern is that most won't put themselves out there like you have. I thank God every day for doctors like you.

[Chemar]

another aspect to this thought....parents and patients have been using the Internet for years to share health experiences and information and support, especially when they are reaching roadblocks in their healing because so many conventional doctors have refused to acknowledge the value of anecdotal evidence as clinical evidence.

 

thankfully, when my son was in crisis, I found a team of physicians who were willing to read the info I was bringing them from discussions with other parents, and then applied their medical knowledge to that anecdotal evidence to help me help my son

[Worried_Dad]

Dr. T, I think this is a brilliant observation!

 

I'm a computer geek by trade, so this resonates with my own experience. The web has revolutionized many traditional business models, social interaction, research processes, etc. I think for software engineers, the emergence of the "open-source software" model is very analogous to what you're proposing. Open-source software now often outpaces the traditional commercial stuff - even the packages backed by mega-corporations - because the worldwide community of "volunteer contributors" improves software quality and accelerates development of new features.

 

I think this is a great idea for at minimum collecting data for rare, understudied disorders from a broad, geographically dispersed "virtual community"; searching for patterns that would not be visible with smaller, localized patient populations; and just comparing notes and experiences among members of a "cyber-support group." As you said, this web site and forum are proof positive that this works well for parents searching for help for their suffering kids that is just not available locally.

 

If there is anything we on this forum can do to facilitate your efforts - e.g., responding to a detailed questionnaire, crunching data, sharing case histories, or even working with local docs to order additional tests - please let us know. You've shared so much valuable info with us. We want to return the favor!

[colleenrn]

Dr T,

 

We would also be willing participate in anything that would facilitate your efforts!

Colleen

[Kayanne]

If there is anything we on this forum can do to facilitate your efforts - e.g., responding to a detailed questionnaire, crunching data, sharing case histories, or even working with local docs to order additional tests - please let us know. You've shared so much valuable info with us. We want to return the favor!

 

TOTALLY AGREE!

[nomoz]

Dr T,

 

We would also be willing participate in anything that would facilitate your efforts!

Colleen

 

 

I agree to that, Colleen.

Also, it is so true - this forum allows for accelerated research and provides a learning lab for doctor and parent (and patient). I've just joined recently, and have learned a vast amount. Hope it accelerates finding the wonder drug that stops the whole process from compromising the immune system, too!

[bubbasmom]

Another concern to address however, is that in parents' desperation to solve their child's medical issues they tend to trust whoever they see on these sites so there needs to be a way for parents to confirm a doctor's credentials.

However, I have taken many times what I have found on this forum to my pediatrician as a way of 'triggering thoughts' for him then I let him make the medical decisions.

[Dr_Rosario_Trifiletti]

Somebody made a "2026" comment which got me to thinking how can we speed this up - and so my post followed

 

I think the high quality of everybody's contributions on this forum (undoubtedly in part because of the fact that you've been forced to become experts in order to advocate for your child) is a proof of concept that this idea might work. I can think of a thousand pitfalls - as a father of two teenage girls, I always think of all the child molesters that thrive on social networking. So, with any free societal structure, it's easily abused. As long as there is a relationship built on trust and "self-police-ing", it should work - as it has in many other places.

 

 

But this is medicine and people's lives that we're talking about here, not a fan club or dating service.

 

How do we incorporate many doctors, with their differing opinions, personalities and (yes) egos into this process?

 

How do we convince doctors that they are not going to get sued by thinking out of the box. Some way of indemnifying doctors that participate.

 

This might be the hardest problem of all!

 

I have some ideas. One is: the patient community elects a panel of say 6 doctors to participate to begin with, a sort of think tank . Each such doctor is offered an opportunity to participate, and the participating doctors decide amongst themselves how best to divide labor: one work say on basic science, another on epidemiology, another on clinical definition, another on treatment, etc.

 

The think tank meets quarterly in cyberspace (i.e. Webinar) to discuss findings. Parents of patients are invited to attend.

 

The think tank meets in physical space (rather than cyberspace) once annually at a conference

 

Every so often, the patient community holds elections to renew/enlarge the think tank.

 

 

Ubuntu. The data belongs to no one and belongs to everyone.

 

 

Dr. T

 

Do you think anyone (I'm thinking Google, Microsoft) might fund development of this process?

[thereishope]

I agree with the concern about trusting any doctor that is listed on here. Even though I know that the parents who have listed a name have had a good experience, it doesn't always mean a few others haven't had bad experiences. If someone has a bad experience, sometimes they share it (along with the name) and sometimes they do not provide the name and I understand hesitation in naming names. It would be nice if one trustworthy person could be in charge of compiling office experiences and maybe give a parent the "lowdown" of the good and the bad, off public forum.Maybe one doctor is great if the patient was sudden onset, but not chronic. Maybe one is good for young adults, another only kids etc.

 

Another concern to address however, is that in parents' desperation to solve their child's medical issues they tend to trust whoever they see on these sites so there needs to be a way for parents to confirm a doctor's credentials.

However, I have taken many times what I have found on this forum to my pediatrician as a way of 'triggering thoughts' for him then I let him make the medical decisions.

[thereishope]

Google is odd in what they support and what the don't. It brings me back to when the March of Dimes treid to get Google to do that artsy thing to their google name for the day in honor of March of Dimes. Well, March of Dimes didn't get enough votes so google didn't do it. That amazed me. I mean...it the March of Dimes... for premature babies!

[faith]

I agree, Vickie, its really all political I think, in what certain organizations will support. Afterall, I guess it all comes down to who it will really make money for. Sometimes its really not the 'cause' at all, but the money that some will make by 'promoting' it. .. sad, but true.

 

Speaking of television,... Dr. T., do you know of any upcoming segments re PANDAS? I thought someone recently posted about something being on tv next week, but I can't find it now. thought it mentioned a doctor, would you know what its about?

 

Also, what will you be speaking about at the Autism Conference? Will you be talking about some of the things and interesting findings you've mentioned above?

 

Thanks!

Faith

[Fixit]

Dr. T

 

Do you think anyone (I'm thinking Google, Microsoft) might fund development of this process?

 

I'm pretty sure they do this every year at google

 

http://www.cnn.com/2008/TECH/biztech/09/24...ject/index.html

 

I was saving this on my favorites because i thought maybe i'd come up with something...but the hamster in the wheel is tired

If you get this doc T....i need a job, maybe you could hire me...maybe i could clean the bathrooms or something...cause i've got to get out of here!!!!

[momto2pandas]

Much of what you describe in the beginning is why I left academia! It's a painfully slow process, and you didn't even mention the parts about politics or the fact that what's vogue in any particular era is what gets funded....or the fact that research findings can be suspect due to publication biases, etc. Pharma moves more quickly and easily (and with more regulation), but of course mostly purues profit-oriented questions.

 

I would like to expand further on the "new" model. I think it would be great to take a formal survey approach - allowed any patients with verfied diagnoses to "opt in" to email mailing lists, and then each time a new question arises (by the scientific managers) or new data is created (by the patients), a survey expert formulats appropriate questions and patients can follow a link to enter their information into a well-designed survey/data collection instrument. Someone expert in data analysis then mines the data at appropriate intervals to look for patterns and generate/test hypotheses and shares this information -- of course with lots of caveats about biases, not "scientifically collected", etc., with interested parties. They can then make what they wish out of it or use it as supportive information to get pilot funding for confirmatory research.

 

There are sources of bias and it's not perfect (and not interventional), but I think it would be an observational model from which we could learn a TON particularly about orphan diseases with highly motivated patients. Like having everyone's stories organized as data to be able to search patterns, etc.

 

I have actually already approached a well known clinical research survey company about setting this up as a model for orphan diseases, and have very strong interest. I think I also have funding through personal connections (royal family - not kidding), at least for a pilot, maybe more. But need to choose the pilot disease, finalize the initial data collection instrument with help of experts (have a general one drafted already), organize the medical and patient community around it, and figure out the ethics parts re. consultation with an IRB (e.g. code assignment for surveys to ensure anonymity). I actually think this could be such a promising approach to learning and sharing information about orphan diseases (and maybe ultimately other diseases) that I'm thinking about cutting back my consulting hours to pursue it more freely.

 

We should really talk.

 

 

 

Hi everyone,

 

 

It just dawned on me ...

 

Traditionally clinical research is done at some large institution, say the (John) Mayer Clinic (names changed to protect the innocent)

 

1. Dr. X, usually based on prior or often his own basic research formulates a hypothesis. Patients' ideas and observations don't figure much if at all in this step.

2. Dr. X attempts to "recruit" patients into his study, which must first pass an Institution Review Board, and many logistical hurdles. All sorts of safeguards are placed to ensure that the Mayer Clinic does not get sued.

3. Many months later, once those logistical hurdles are cleared, Dr. X opens his study

4. A year later, enough people have called Dr. X's office so that he has enough data to write a pilot grant to the NIH

5. Six months later, Dr. X finds out that his grant is just short of the threshold for funding

6. He submits another version, and prays - the Mayer Clinic is beginning to put pressure on Dr. X to bring in funding. After much white-knuckling, Dr. X finds his study is funded

7. Several years later Dr. X completes his study and finds out that his hypothesis is wrong

8. Dr. X is "non-renewed" (i.e. fired) by the Mayer clinic

 

Dr. Y, newly hired by the Mayer clinic to replace Dr. X, is inspired by Dr. X's work, and decides to embark on the same program as Dr. X

 

Dr. X, although disheartened, decides to try something new ....

 

There is something magical going on right here which might be a model for the way we do clinical research in the future:

 

1. Parents of patients (or with adults, patients themselves) tell their histories, make observations, discuss lab values

2. Interested doctors observe the proceedings

3. Doctors and parents formulate new hypotheses

4. Doctors set in motion ways to test the hypotheses by testing and treating patients (there's the rub)

5. Communal feedback on test results and treatments

6. Refine hypotheses and treatments to obtain improved diagnosis and treatment

 

What is the role of outside parties like the government and instiutions? really two-fold that I can see (but they are huge): ensure confidentiality and safety.

This has got to be incorporated in version 2.0 of the proces.

 

 

In the past 2 months alone, we've learned about mycoplasma and the p41 (anti-flagellin) connections by the new process. I'm sure there is more to come.

 

Can you appreciate how this rapid feedback iterative model VASTLY accelerates early clinical research? In the past, local doctors did this one patient at a time

 

Now, by using the "social networking" features of the internet, we can do it on large populations, i.e. statistically significant numbers.

 

The revolution will not be televised. But it will be broadcast on the internet and you are part of it!

 

Dr. T