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A wonderful story of participatory medicine - SPM Blog
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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.


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