A wonderful story of participatory medicine

Amy Marcus, in today’s WSJ,  wrote a powerful article about a mom moving medical mountains to help her twin daughters survive a rare and deadly disease. Entitled “A Mom Brokers Treatment for Her Twins’ Fatal Illness. Bucking Scientific Convention, Ms. Hempel Gets Researchers From Different Fields to Share Data on Potential Therapy“, it is a beautiful story about the value of participatory medicine when it is at its best because:

  • the mother is a prime example of the power of informed patients and caregivers,
  • the researcher(s), Dr. Hildreth and others, are willing to work with this empowered caregiver in uncharted waters

The story is the demonstration, once again, of the value of freely available scientific information:

She (Ms. Hempel) found a short report in the medical literature about a doctor who had treated a child with a different disease using cyclodextrin and tracked him down”.

This is typical of the way innovative treatments are discovered in communities of people dealing with rare diseases. There is a fast growing number of stories of patients/caregivers capturing scientific news and infering that it may work for teh condition that concerns them. Because it is most often happening with rare diseases, the public at large is unaware of the sea change that is taking place right under our nose. It is truly like watching in real time a paradigm shift in action!

Ms. Hempel’s long journey through the health-care research community — a distributed and labyrinthine collection of researchers who, for all their expertise, often remain unaware of advances made elsewhere. The problem is even more acute among researchers working on different diseases. But for some serendipity, curiosity — or, in this case, a willful Ms. Hempel — some knowledge in one lab may never make its way to another that could be on the verge of a new therapy.

This is why the old method of using only proven, demonstrated and utterly static peer-reviewed authoritative answers from within the disease-specific silo is no longer fitting the model of patient-driven medical innovation. We may still be nobodies but we are moving the innovation faster and further everyday!

Dr. Hildreth says that Ms. Hempel’s involvement got his research “the attention of individuals higher up in the organization than I might have been able to get on my own.”

Unbelievable but true! Well in fact it is not so unbelievable! One of the greatest moments in my life was the presentation made by Norman Scherzer of the budding Life Raft Group to the scientific team at Novartis in charge of the clinical trials of Gleevec for GIST. Anyone present that day will never forget the discernable shock of all the scientists and MDs at the table, when this unknown entity, made up of patients and caregivers, presented data about multiple aspects of the trial results, with numbers that confirmed what was just starting to trickle down from the Principal Investigator Offices. In some ways, the patient group knew more about the trial result than the senior scientists at Novartis. From that moment on, it was clear that we no longer have the time to wait for the old-style publications. As Norman said in the e-Patients White Paper section entitled “Bypassing The Lethal Lag Time”:

One of the great benefits of patient-initiated research is its speed. We can get lifesaving information out to the people who need it right away, much faster than professional researchers, who must go through many time-consuming steps.


Posted in: e-patient stories | general | positive patterns | pts as teachers | trends & principles




10 Responses to “A wonderful story of participatory medicine”

  1. Gilles, thank you so much for your additions to this inspiring story.

    Readers out there: the Journal of Participatory Medicine wants to collect stories like this. In business lingo I call them “existence proofs,” a logical method that says “If something exists, that proves isn’t not impossible.” A story like this, as Gilles says, disproves those who would say “Hey, who’s the doctor here? Stop asking questions.”

    Clearly, this story proves the existence of value from empowered caregivers. Well done, G-man.

  2. Wendy says:

    Great illustration of the changing dynamics in healthcare. As you pointed out, for rare disorders the change where empowered patients/caregivers are making a difference is happening even faster than it may be in other areas.

    Just as in this case, most rare disorder associations are staffed by patients and families of patients. Information, although available, may be disjointed and difficult to understand. These organizations also tend to be small and may not have a Chris Hempel as a member; but many share similar issues and could benefit from “cross pollination” under an umbrella group like NORD (the National Organization of Rare Disorders).

  3. […] was catching up on my reading today, and Gilles Frydman’s post, “A wonderful story of participatory medicine” pointed me to a powerful article from last week’s WSJ.  The WSJ article, written by Amy […]

  4. Gilles, I’m just catching up with this post. You’re right, it’s a powerful teaching example. We who grew up thinking docs must know all the info worth knowing… well, we’ve got another think coming.

    I traced over to the original WSJ article, and ha ha ha, we got one on them. They say “Researchers at Pfizer were developing Viagra to treat high blood pressure when they noticed during early tests that it treated impotence.” They noticed? Tom Ferguson reported in 2002 (cited in the white paper Chapter 5) that it was patients who reported the unexpected side effect that made the drug so popular – and not for BP (or angina, as Tom reported it).

    My takeaway, as the white paper and the WSJ article make clear, there are enormous amounts of clinical research results that are unpublished, some of which never will be published, and others that are published and unknown, that could save lives.

    And: nobody could possibly build a business model to justify hunting down that information – but patients sure can.

    Knowledgeable patients can make a phenomenal contribution in such cases. Thanks.

  5. Dave,

    part of the presentation that Norman did to Novartis regarding his new scale of the severity of side effects: “It introduced a new scale for rating the severity of side effects from the patient’s point of view, as opposed to the clinical trial toxicity standards established by the National Cancer Institute.

    This last point requires some explanation. Scherzer, trained to conduct population studies, looked for the existing instruments used to report the severity of side effects. They were available from the National Cancer Institute, but Scherzer was surprised to find that side effect measurements were focused exclusively upon toxicity as opposed to quality of life. For example, a patient who developed diarrhea several times a day, every day, on an indefinite basis would be rated a one on the NCI Toxicity Scale of one through five, with five being death and one being the mildest rating. As an alternative Scherzer adapted a common pain-management rating scale of one to ten, with one being the mildest and ten the most intolerable and then asked Gleevec patients to rate a number of possible side effects using this scale. A situation like the one described above would rank as an eight or higher using this scale.”

    Clearly the scale he invented was much more in tune with the experience of patients who were now living the unexpected life of patients suffering from a chronic condition instead of having to deal in an accute way with the side effects of a chemotherapy treatment.

    There are many examples of great innvovation in patients communities. It;s just that no one has really payed systematic attention to it. Hopefully this will change now.

  6. CiscoGIII says:

    Gilles and e-P. Dave,

    What a wonderful story and I very much look forward to reading the full version in the Journal of Participatory Medicine.

    However, the physician buyout will be the difficult route, particularly when looking for measurable results that can be justifiably used on our patients. Even then, what composition of the patient population is empowered and capable of pursuing this route?


  7. A wonderful story of participatory medicine http://bit.ly/3Kwq

  8. […] A wonderful story of participatory medicine (E-patients.net) […]

  9. […] A wonderful story of participatory medicine | e-Patients.net: “One of the great benefits of patient-initiated research is its speed. We can get lifesaving information out to the people who need it right away, much faster than professional researchers, who must go through many time-consuming steps.” […]

  10. […] is its speed.” It’s spoken by Norman Scherzer, who was mentioned in our April 3 post A wonderful story of participatory medicine, and describes how when the medical industry falls shorts, e-patients step in and gather vitally […]

Leave a Reply