Dear Governor Palin...

October 25, 2008

Dear Governor Palin,

I applaud your interest and stated commitment to improving the quality and availability of care for children with special needs. As the mother of a now grown special needs child and the founder and director of an organization dedicated to research on her disorder, I know firsthand the challenges families of these children face. Unlike trisomy 21 (Down syndrome), my daughter’s disorder, a rare genetic disease called primary ciliary dyskinesia (PCD), does not enjoy broad public awareness, which makes finding health services and funding for research even more difficult.

Last week in a policy speech you indicated that certain research expenditures were wasteful and foolish. The specific example you used was fruit fly research. Because I believe you are sincere in your desire to help those with special needs, I was dismayed to hear your perspective on basic science research. When my daughter was diagnosed in 1991, we were told that her disorder was so rare there would never be genetic research done on it.  Finding a patient group large enough for human genetic studies would most likely be an insurmountable challenge.  But people were working on PCD genetics--albeit indirectly. Basic scientists were unraveling the genome of a single-celled algae called Chlamydomonos. This organism was simple and plentiful and scientists could grow pools full of it for research.  In 2002, the first PCD gene was discovered in an ortholog of Chlamydomonas. Since then, several more have been identified, resulting in the first-ever genetic test for PCD and the promise of genetic therapies for this incurable condition in the future. It can not be overstated that these advances are the direct result of research on, quite literally, pond scum. It was heartbreaking to me to hear a candidate for national office make a joke of these efforts simply to score political points and I was saddened beyond measure for the families relying on basic science research in fruit flies that you chose to single them out for this unwarranted ridicule.

Translational research, applying the discoveries of basic science to human populations, is a critical element of medical science advancement.  In this country, we have suffered through the William Proxmire “Golden Fleece” awards and other attempts by politicians to gain political points by criticizing science they simply don’t understand. Because I was hopeful that you would be a true ally to special needs community, I was particularly dismayed to see you participate in this activity and ask you to spend some time speaking with the individuals involved in basic science research and educating yourself on the promise these activities offer to people with rare, genetic and incurable diseases.

A recent article by the Massachusetts General Cancer Center published in the peer-reviewed journal Current Opinion in Genetic Development states that “more than 60% of genes implicated in human disease have Drosophila (fruit fly) orthologues.” A very short list of human diseases with fruit fly genetic correlates includes neurological and neurodegenerative disorders (including Parkinson’s, Alzheimer’s and spinal muscular atrophy), birth defects, development delays (including autism), tumor suppressor genes (ironically in melanoma, your running mate’s genetic curse), infertility, polycystic kidney disease, deadly and catastrophic lipid metabolism disorders, and Huntington’s chorea.  Again, this is a short list.

I am not an expert at fruit fly research, but was able to compile this list in about five minutes by doing a simple Internet search of the government-maintained National Library of Medicine database of scientific publications (PubMed:http://www.ncbi.nlm.nih.gov/sites/entrez?db=PubMed&itool=toolbar). I do not think it is too much to ask our national leaders who will be responsible for establishing crucial health and science policies to do at least that much.

Special needs communities have never had adequate representation in this country. Attempting to manage the extraordinary challenges of caring for chronically or catastrophically ill family members leaves us short of the financial resources and time required to mount effective lobbying efforts. As a result, we do not wield political clout and end up being an invisible minority group with few supporters. Your high-profile status gives you the opportunity to change that for us and I ask you to consider working with the groups who are engaged in daily efforts to improve the lives of special needs individuals through important research initiatives. We truly welcome your support. 

Michele Manion, Executive Director
Primary Ciliary Dyskinesia Foundation
Phoenix, Arizona

For more information on PCD: www.pcdfoundation.org