ISPP 2015

Career Opportunities in Pharmacy
Finding Hope for the Smallest Patients | Dr. Virginia Pascual | Weill Cornell Medicine

My goal here at the Drukier Institute
for children’s health is to create a research environment both basic and
patient oriented that will lead to discoveries especially new treatments to
improve the life of sick children systemic onset when I’ll arthritis has
been the main focus of our research because no therapy was working when I
first started these were the hardest children to treat they really didn’t
respond to any medications and were treated largely with high-dose steroids
and the problem with this was that they suffered from tremendous side effects
including gaining of weight changing the in their face they would be severely
immunosuppressed and specifically they got a lot of arthritis of their hips and
many of them needed to have hip replacements there was really nothing to
treat these children with we did some basic research and we
discovered one molecule that we thought could be responsible for these disease
we developed a pilot clinical trial with the drug that had been developed to
treat adult rheumatoid arthritis that drug didn’t work very well at all in
adult arthritis but we brought it to our kids and it worked very well you have
children in the hospital who are desperately ill with daily fevers and
you give them the medication and it just stops instantaneously it is one of the
most remarkable things I have ever seen one of the cases we are collaborating
with Dr. Donnell he is a two year old precious Eva Qi who has benefited
tremendously from the therapies that we found we met her when she was really
tiny and very sick she came to us from another hospital with high spiking
fevers she was so ill she was unbelievably anemic she had terrible
arthritis really all she did was cry and then we started to use the il-1
inhibitors and the most amazing thing was this truly miserable child became
happy here is a great example of how this therapy worked how she could come
out of it and go back to be a normal girl so it is a very exciting time
because we can really apply technology that we didn’t have a few years ago that
allow us to look at small volumes of blood a little piece of tissue and we
can really bring tremendous discoveries so I am convinced that the future is
very bright because if we can bring a child to good health we can give them
good health for the rest of their lives

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