Possible Hope in Toronto, Canada

For those of you who do not know, we have been in contact with a doctor and scientist in Toronto, Canada since August of 2009, whom are testing Gavin’s cells (Fibroblast Cultures) to see if they respond to an experimental drug known as Pyrimethamine. This drug was used originally to treat Malaria, but it has been researched and tested among adults with late-onset Tay-Sachs and so far based on that study, it has revealed to increase the Hex-A residual activity among such patients. Hex-A again, is the enzyme that Gavin desperately needs, but he has two gene mutations that is not allowing his body to produce enough Hex-A. So now, 2 doctors are testing to see if his cells respond to Pyrimethamine which will be used as Pharmacological Chaperones, in other words, as helpers to unfold the genes so that it could increase the Hex-A residual in Gavin’s body. The nature of the testing is very extensive and can take up a lot of time. So far, there has been no data on successful responses on this drug among babies or children with Infantile or Juvenile Tay-Sachs, but it is worth a try to see if this drug responds with Gavin’s cells. Please join me and Ferd to continue praying for Gavin and for a positive outcome and report from the doctors and scientists in Canada. I will be getting an update on how things are going with Gavin’s cells sometime this week.

November 11, 2009 at 7:21 pm

jennifer mcguire

i am praying for Gavin!

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November 12, 2009 at 12:45 pm

Mich

we will continue to have that HOPE!!!