My primary kidney disease is atypical Hemolytic Uremic Syndrome (aHUS). This is a very rare disease in adults. It is more common in children. The problem with rare diseases is that they do not get too much attention. Researchers prefer to work on things that have more far-reaching benefits. So, a disease that affects a few hundred the world over does not have too many people working on it.
The chances of recurrence of my primary disease is fairly high after a transplant. This is what was suspected to have happened in my first transplant in November 1998. Recently, however, a drug called Eculizumab (manufactured under the trade name Soliris by Alexion Pharmaceuticals in the US) has been found to help in this disease. It has not only helped children with an initial primary diagnosis of aHUS by helping to arrest the progression of the disease and the concomitant kidney damage, it has also helped prevent kidney failure after a transplant!
The drug however is still undergoing clinical trials for use in aHUS. It is approved for use in the US for another disease called Paroxysmal Nocturnal Hemoglobinuria. I don't think it is available yet in India.
The problem is the amount of time these clinical trials take and the amount of time these drugs take to become available for general use. They need to make sure the drug does not cause any other harm. For this, they need to try the drug on a large number of people with the problem. I would have gladly registered for a trial but it was restricted only to people in the US. Understandably so.
While I totally understand the need for such trials before beginning to use these on a wider scale, I feel frustrated that the process takes so long. There is something that can be done to change my life. Its just that I cannot use it yet.
I wish there was a way by which people could be allowed to choose whether they should use the drug or not. They should be allowed to sign a document that states that they assume full responsibility for any adverse effects of the drug and then they should be allowed to take the drug once the drug is preliminarily proven to be safe. Soliris has been used successfully in many cases where people have been given a new lease of life. There have been no cases of adverse effects at all. In this case, why should I wait for years before getting a shot at trying it?