Zaini asked about why L1 is not available in the US. This topic has been raised many times, although with the other oral drug, Exjade, now available to those with medical insurance, we don't hear many people in the US asking anymore.
Perhaps the most ironic thing, is that Ferriprox (brand name for L1), made by the Canadian company, Apotex, is not available even in Canada. Why this is can be traced back to the research done by Dr Nancy Olivieri in Toronto, during the 90's. Apotex commissioned Olivieri to do a study on patients using the drug, and she claimed that her research showed that L1 caused liver fibrosis in thal patients. However, she refused to turn her data over to Apotex and a huge controversy developed which led to Olivieri being portrayed as the champion of the little guy, fighting the big bad drug company. This made for a lot of good news stories in Canada and the US, but unfortunately, did nothing to help thal patients. My own feeling about this is the patients were the LAST thing on the mind of Dr Olivieri. Defending her research became the ONLY thing. Filing lawsuits against detractors became far more important than the fate of the patients who were prohibited from using this life-saving chelating drug. Perhaps the hardest thing to understand about all this is why Dr Olivieri, to this day, refuses to renounce the results of her research. I say this because every single study conducted worldwide has been unable to duplicate her results. Instead, researchers have found a drug that is safe for the vast majority of patients and has been proven to be a superior chelator to desferal in removing iron from the heart and also preventing the buildup of iron in the heart in the first place. At the Pune thal conference in October, 2005, the research that Olivieri did was portrayed as fatally flawed. It is believed that the patients in the trial who had liver fibrosis already had liver damage and it had no relation to the trial of L1. Our own Ashish has recently observed the 20th anniversary of his use of L1 in India. I say with much confidence that Ashish, Shilpa, Poirot and many other thals are alive today because they have been able to use L1 these many years. The story of Olivieri and Apotex has been very well chronicled by Dr Miriam Shuchman in her award winning book, The Drug Trial: Nancy Olivieri and the Science Scandal that Rocked the Hospital for Sick Children. My own response to a review of this book along with the response from Shilpa can be read at
http://www.bmj.com/cgi/eletters/331/7508/115This book was the winner of the Writers' Trust of Canada's Shaughnessy Cohen Prize for Political Writing and the Canadian Science Writers' Association's Science in Society Book Award.
There is another major obstacle to L1 being available in the US. It is available to some patients under a program that allows patients who cannot take a drug, to purchase a replacement drug even if it is not legal in the US. However, for the vast majority of thal patients in the US, L1 is not available. Novartis is a huge international company. They do not make L1. They did make desferal for many years and also now manufacture Exjade. Novartis is also a major contributor to political campaigns in the US, and have coincidentally pretty much had their way when it comes to iron chelators available to patients in North America. For 20 years there were rumors about an oral chelator being developed by Novartis. Lisa told me about this. Her mom has told me about this. One very in the know member of this group told me that this oral drug sat on the shelf for many years until the patent on desferal expired, allowing other manufacturers to get a piece of the market selling desferal. Very soon after the patent had expired for desferal, there was Novartis presenting their "new" oral drug, Exjade, to the FDA for approval. In what seems like lightning quickness, Exjade was approved by the FDA for use by patients in the US. Is there a connection? I doubt we will ever be able to prove it but the coincidences are both amazing and alarming.
My questions are, did Novartis exert influence on politicians in the US with their hefty donations so that L1 has never found approval? Did Novartis put Exjade on the shelf until their patent for desferal expired? How many patients have died because they did not fit into the profit picture of a drug manufacturer? One question I can answer. Does this whole circus piss me off?
Yes it does and it really pissed Lisa off and it also really pissed off her mom, who saw her own daughter die because no oral chelator was there for a young woman who could not tolerate desferal enough to do her any good late in her life. Lisa flat out told me that she would die because L1 was not available to her. Is this a sore subject for me? Yes, and no matter what happens in the future, it always will be.
One more point I wish to make is that by all accounts, Dr Olivieri is an excellent and dedicated doctor who has done much good for thals around the world. But I can never forgive her stubbornness when it comes to L1. Lisa would boil when she mentioned Oliveri and I will always believe that Lisa would still be here today had she been allowed an oral chelator.