On Our Minds: Musings about Collaboration, Continued

Our Community Outreach & Ethics Core works to ensure that collaborations between researchers and community groups are mutually beneficial and transparent.  Photo © 2014 Clipart.com.

My recent leadership responsibility with the IFOPA has me thinking more directly about the role collaboration plays within our Center. Collaboration is a bit different in the Center for Ecogenetics and Environmental Health than for the rare disease community I described earlier. There are no pharmaceutical companies. There is no single disease population or organized patient group. Still, scientists engaged in basic research need participants for their studies. Basic scientists need clinicians to describe the disease phenotype. Researchers need informaticians to help ask the right questions. And findings need to be translated to the next phase of research to be included in policy and practice.

Let’s take one of those stakeholders: the general public. Why should they care about environmental health research? 

  • People get sick from environmental exposures. Anybody who has asthma, or whose children have asthma, has a stake in research about air pollution. People with a family history of cancer or heart disease have a stake in research related to those diseases. 
  • An emerging hazard may alarm the public, for example the meltdown of Japan’s Fukishima nuclear reactor in 2013. People on the west coast want to know if they are being exposed to radiation. Thankfully, tests indicate low radiation levels, but if results were otherwise, concerned citizens might ask to participate in research studies. In cases of industrial pollution or exposures from factories, citizens come to scientists asking for research. 
  • Potential participants might be interested in science, or simply be curious. Others are activists who like to be involved. Some sign up to get the monetary incentives. 

Public engagement professionals like us in the COEC can help educate the public about the importance of research and the need for participants. 

Once identified and recruited, researchers need to retain their participants. A few simple strategies can maintain participant interest. Hearing back about the study lets participants know their role is important and appreciated. Researchers can send results via newsletter updates or a summary of publications written for a general audience. 

In January, the Center sponsored the first meeting of our new CEEH Breakfast Club, a monthly gathering of Center members for an informal presentation and discussion. The topic was crowdfunding for science. Dick Beyer spoke about participating in the American Gut Project to have his microbiome sequenced. Dick displayed a placemat-size printout with his name in large letters and pie charts of the relative abundance of his microbes and how he compared to others. Not only did he participate, he paid $100 for the privilege, although scientists don’t claim to understand how the composition of our microbiome affects our health. 

Someone asked whether Dick would participate again. “Oh, yeah,” he responded.  “I'm really glad American Gut is doing this. But I mainly wanted to learn something about me.” Getting his results, even results that aren't closely tied to health, was worth not only participating but paying for it.  

It could be we need to think a bit more – and ask – about what we can get from and give back to participants. The answers might surprise us!

On Our Minds: Musings about Collaboration, A Case Study of Science in Action

In my outside-of work life, I am the mother of a child with a rare disease. I recently volunteered to chair the Board of Directors of the international patient group. My involvement with leading this group as it approaches a clinical trial has taught me a considerable amount about research-in-action and the importance of collaborative partnerships – two themes of high relevance to our Center.

The disease is an ultra-rare, catastrophic genetic condition called Fibrodysplasia Ossificans Progressiva (FOP). FOP causes bone to grow in skeletal muscles, creating a second skeleton that freezes joints and prevents movement. There are fewer than 900 known cases of FOP in the world.

Marilyn at a recent IFOPA meeting.
The patient group is the International FOP Association or IFOPA, founded in 1988. Medical research in FOP has increased dramatically since the patients organized, and what is understood about the condition has progressed greatly. The most important discovery so far happened in 2006 when researchers at the University of Pennsylvania identified the FOP gene. Since the gene discovery, attention from researchers and, recently, from pharmaceutical companies, has grown exponentially, and along with it, our need to navigate across stakeholder groups.

To collaborate is to work jointly on an activity to produce something or work towards a shared goal. The collaboration of patients, researchers, and pharmaceutical companies fits that definition, but it is not exactly a group project where everyone has the same goal. These stakeholders have different perspectives, and although they are all working on a treatment for FOP, their goals and motivations differ:

  • FOP patients want a treatment for their disease, and something to allow rehabilitation from damage already done. Patients are in a race against time because FOP is progressive. 
  • The patient group, the IFOPA, wants to encourage drug development by contributing natural history data to document the natural course of FOP, collecting patient data, and encouraging its members to participate in clinical trials. Data collected in one international registry and owned by the IFOPA would let the patient group control the data and share it with research and pharmaceutical stakeholders. 
  • Researchers want their work to lead to discovery: A gene, a disease mechanism, a drug candidate. Researchers need publications and grants. Since they compete with other researchers, they also want to protect their data, which can be an unfortunate barrier to collaboration. 
  • Pharmaceutical companies want a product. They want successful clinical trials, regulator approval, and a useful drug. In rare disease research, pharmaceuticals want a wider audience to buy the drug, to meet their ultimate goal of making a profit. The pharmaceutical companies working on FOP hope their drugs will also help non-genetic conditions of bone growth that comes up after hip and knee replacements, spinal cord injuries, stroke, atherosclerosis, closed head injuries, and heart valve replacement surgeries. 

Despite their different perspectives, we stakeholders need each other. Only through collaboration will we be able to reach the goal of finding a treatment for FOP.