Forward progress......

As a board member going in to the 2012 Little People of America Conference in Dallas, I knew one of the most significant issues at the board meetings and at the conference would be that of medical research.  The stage was set at the conference in 2011.  In Anaheim in 2011 we heard for the first time about groups like Biomarin, a pharma company developing what is called a treatment for achondroplasia, and we heard about groups that are raising money that will be directed toward research.  In this case the term treatment means drugs that address certain impairments found in people with achondraplasia, such as sleep apnea, spinal stenosis, fluid in the brain and bone growth.  Little People of America has never been directly connected to research.  However, as the major national organization for people of short stature, LPA would need to be in the foreground on the issue, providing information about updates in medical research to the membership and to the dwarfism community.

In the months just prior to the conference, Little People of America did two things.  First, it created a Frequently Asked Question document that aimed to answer some of the basic questions about medical research related to dwarfism. The document, titled, Emerging research and potential treatments in achodroplasia and other skeletal dysplasias is on the LPA website. Second, just prior to the conference, LPA drafted a position statement.  The statement was approved by the board of directors at the Dallas conference.  It is not yet published on the website.  Basically, in the words of the authors of the statement, it is "strongly neutral."  It stresses the importance of dwarf culture and of the community that is nourished and supported by a group such as Little People of America.  It does not support or endorse medical research but indicates that no matter what choices dwarfs and parents of dwarfs make in the context of medical research, they will continue to find support within Little People of America.

Though I was pleased with the outcome of the two documents, they didn't prevent the membership from getting more involved with the issue at the conference in Dallas.  We have a position statement, but there is still the question of whether or not LPA gets involved in research.  That question comes with a lot of complexity.  The complexity rose to the surface at the tail end of a medical presentation on Sunday morning of the conference.  At the end of the presentation, after members of LPA's Medical Advisory Board opened the floor to questions, a few members from the audience delivered passionate statements about the implications of "treating" dwarfism.  Though treatments are intended to improve quality of life from a medical standpoint, it is hard for some to navigate around the idea that the treatments are changing who we are as a community of people with dwarfism.  With this in mind, the treatments, and discussions around the treatments, can be interpreted as challenges to who we are. 

Also, some people had difficulty with the emphasis on the medical model of the research.  Little People of America is a social support organization, and any venture into the area of medicine should also have a social component.  Yet, there has not been any social research done in tandem with what the community is hearing about Biomarin and medical treatments. This made it very difficult to listen to the medical presentation, because there was little to suggest the social standing of the people who would be "treated" and of the social implications of the treatment on the entire community.

As the conference progressed, it became clear what would be expected of Little People of America.  Again, though LPA is not involved in research, it was agreed that the Medical Advisory Board and that at least one of the groups raising money for medical research would look to LPA for guidance when it came to research.  Though this is new to the organization, it's probably a good thing.  It's better that LPA try to figure out what is going on, then make recommendations on research that would potentially benefit our membership both medically and socially, rather than stay silent and allow things to proceed without our input.  It won't be easy.  This is new territory.  But it's territory about which we should be aware, and perhaps even into which we should venture.

don't get in the way of progress

About a month ago, maybe less, I was interviewed by a reporter with Bloomberg News. The reporter was writing a story about research conducted by Biomarin on what are being referred to the science industry as treatments for dwarfism. Biomarin is a pharmaceutical company based in California that develops medicine for "rare diseases." Evidently, in the world of science, or at least within this particular realm of science, dwarfism is a rare disease. According to the article, Biomarin has a new drug for dwarfism that is ready for human trial. Mice trials were successful. (I wonder if the mice were transferred to the rat lab after the trials.)

While short stature is the most obvious visible affect of dwarfism, the new biomarin drug is not actually intended to affect a person's height. Rather, it is supposed to address other health issues common in people with achondroplasia (the most common type of dwarfism), such as sleep apnea, bowing of the legs, and compression of the spine.

The story ran a little more than a week ago, on January 23. Little People of America knew that phone calls from reporters writing about this type of treatment would be coming at some point. Back in July of 2011, at the LPA National Conference, a few individuals with connections to companies such as Biomarin attended the event. They were excited to share news about emerging treatments.

While Little People of America is primarily and a support and resource organization, and has nothing to do with research, individuals and organizations may continue to approach us, perhaps for guidance, perhaps for human trial participants, perhaps for public support. In mid-February, the Little People of America Board of Directors is scheduled to talk about the issue of so-called "dwarfism cures" at a meeting. Though the meeting probably won't allow enough time for LPA to develop a position statement on the issue, the meeting will at least afford some time for individual members to share their thoughts. Hopefully, those thoughts will lead to some organizational clarity down the road.

As an individual with dwarfism, and as an officer with LPA, it's hard to figure out a good approach. On one hand, would I, as a little person, be better off today, or would I have been better off when I was a child, if I have to endure the leg surgeries and the back treatment that were required as a result of my dwarfism. Would others in my situation be better off?

On the other hand, why should an individual with dwarfism have anything to do with an industry that treats dwarfism as a disease and people with dwarfism as a commodity. Plus, is it ethically correct to pursue drug treatments that cost hundreds of thousands of dollars. Treatments that, even if they did work, would only impact a tiny fraction of the community. So many people with dwarfism will tell you that the real obstacles they have to face in life have very little to do with medical complications. Rather, they have to do with social prejudice, and to some extent physical barriers in the environment. With this in mind, in the spirit of Ed Roberts -- a pioneer of the disability movement and of the social model of disability -- rather than changing individuals with dwarfism to make them supposedly more healthy and less diseased, wouldn't it be more productive, and more healthy, to invest our resources in creating a better, more accepting, and more accessible community.

Who knows. But as technology moves forward, it will be interesting to observe how the dwarfism community responds.