Most of you know my history. I was diagnosed with a very rare form of familial amyloidosis in 1996. There's no treatment or cure because there are so few of us worldwide, and I'm at the bottom of the list when it comes to research. It took many years for me to accept this fate.

I saw an article recently about some scientists at Scripps working on the gelsolin protein, the amyloid protein causing the damage/dysfunction in my type. I wrote them a thank you note just to let them know they weren't just playing around with cells in a dish - there was a funny looking lady in Chicago cheering them on.

I got a reply, and I'm blown away because it sounds VERY promising. To me this is a miracle because I was NOWHERE on the radar for scientific research. I'm a little torn though, because I've seen some disastrous initial human trials. If they'll require heart and kidney biopsies, it could prove fatal. I've never been much of a gambler. Geesh, I thought gambling with money was hard, lol.

Here's what he had to say:

Dear Kathy;

Thank you for your thoughtful note. We have reason to believe that Fibrillex, a neurochem Inc (Canadian company) drug that is about to be approved by the FDA, may offer some relief to gelsolin amyloidosis patients like yourself. I would encourage you to have your physician call me about this possibility XXX-XXX-XXXX and I can explain the highly technical reasons why we think this could ameliorate your disease. This drug appears to be quite safe.

We need patient samples to better understand this disease and if you would be willing to help us through your local physician that could be terrific. Is there a patient organization for gelsolin amyloidosis patients in the US ?

Regarding your son, I am not a genetics expert. What I do know is we and others are trying to develop therapies for these diseases and we are starting to be successful. If he is a mutation carrier, knowing what to look for and treating the disease early in its course will be critical tp have the best chance at successful treatment.

We are currently testing several strategies against gelsolin amyloid disease in a mouse model of this disease we just developed, Hopefully we will learn enough to help your son in the future in the unfortunate event that he is a carrier.

With best regards

Jeff Kelly