Rare disease research

In the past week or so I have watched two interesting shows that raise questions about the process by which drugs are approved for marketing in this country.  The first program was an Outside The Lines program on ESPN that addresses the use of stem cell therapy by elite athletes to recover from injuries.  The second was a documentary, Burzynski, that follows a researcher’s 15 year battle with the government to use gene-based therapies to treat incurable cancer.  There are groups and individuals on both sides of these controversies, but what is so striking is how in both cases the success of individuals using the therapies are ignored and written off as random, despite overwhelming evidence.  Additionally, both shows speak to how the game is rigged to benefit large pharmaceutical companies, and in this era of crony capitalism it makes one wonder about the prospects for really advancing research into rare diseases such as rasmussen’s encephalitis.

Towards the end of last year the following bill was introduced in the House of Representatives.

H.R. 3699 – On December 16, 2011, Representatives Darrell Issa (R-CA) and Carolyn Maloney (D-NY) introduced H.R. 3699, the Research Works Act, which would prohibit any Federal agency, including NIH, from requiring that investigators make any research paper arising from research funds publicly accessible via the Internet without the prior consent of the publisher. The bill would also prevent government agencies from including in its grant and contract agreements a prospective requirement that the results of the research be made publicly available on the Internet. The bill would effectively prevent NIH from posting peer-reviewed papers arising from NIH funds to PubMed Central as required by Division G, Title II, Section 218 of P.L. 110-161.

The last line states that the results of government funded research would no longer be allowed to be published on the internet.  Whatever the issues are behind this bill, it’s result is anti-rare disease, anti-research, and creates poor transparency in the research process.  We need to pay attention more attention to these issues as path-breaking research will migrate off-shore.  Already today, individuals with resources are importing drugs not available in the US to treat epileptic conditions.  This is one reason why one of our first research fundings was to a group outside of the US in Germany, and two of our scientific advisors are located overseas.

I came across a really cool website this week sponsored by the Khan Academy.  I mentioned it on our facebook page. The educational videos on this site are addicting and there is something for everybody.  Watch this one and learn about B lymphocytes!  This site is perfect for Grace as it addresses math problems.  Staying on the educational front I watched another interesting video posted by friends at the Brain Recovery Project that addresses teaching math without words.  This is just plain brilliant.  The rewiring of the brain post-hemispherectomy is absolutely critical and digital versus analog teaching methods facilitate this process.

Our fundraiser is two weeks away.  It is the only fundraiser we do all year and last year it raised the bulk of our research expenses.  Please help us in our mission to advance research into rasmussen’s encephalitis and let us know you share our passion to help families avoid the despair we went through.  Please hit the donate button, every dollar counts.  Thank you.


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