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Extraordinary measures of an (extra)ordinary man

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It is always said that ‘Victory has many claimants but failure is an orphan!’ However, this failure is not an orphan. This failure to recognize, probe and find solutions for rare diseases is not uncommon in the typical industrial set-ups. There is an urgent need to accept the situation through which few people suffering from rare diseases faces. These rare diseases are very rare but it brings turmoil to some person’s entire life and these conditions need to be checked as early as possible to prevent the coming generation from these unpleasant diseases. The need to tackle these are as much important as any other disease or disorder irrespective of its level of prevalence.

Our ongoing featured series mark and celebrate the Rare Disease Day, this week get inspired by the extraordinary spirit and story of a man John Francis Crowley, filled with the courage to overcome the odds in life by his journey. Mr. Crowley is already a recognized name in rare disease drug discovery industry. I thought to share this with you because his story inspired me personally. Hope you will like this too!

One of the well known remarkable fights against rare disease has been put forth by John F. Crowley, the biotech executive, who had been the CEO of organizations like Amicus Therapeutics, Orexigen Therapeutics and Novazyme Pharmaceuticals came ahead with the determination to battle and win against the Lysosomal Storage Disease Type 2. When Mr. Crowley discovered that two of his children suffer from Pompe’s Disease, his battle took a much sharper turn. His organization came out with options of Enzyme replacement therapy as it had been pioneering in this field. Mr. Crowley, being the then CEO and President of Novozyme Pharmaceuticals, had to choose between a partnership and a buy-out to garner support and funds and to actively push the medication options into the market in full throttle. Subsequently, Novozyme was acquired by Genzyme Corporation.

Pompe disease is associated with the inefficiency in lysosomal metabolism. The glucose moiety metabolized by acid alpha-glucosidase enzyme in lysosome, is deficient in children with Pompe disease. This leads to the accumulation of glucose and its further conversion to glycogen in the  borne fruit in the form of alglucosidase alpha. This enzyme replacement therapy with alglucosidase alpha, takes up the role of the deficient acid alpha glucosidase in the body preventing the accumulation and building of glycogen in the lysosomes. Coming victorious with this orphan drug, providing treatment for his children through Enzyme Replacement Therapy, Mr. Crowley has shown a path to many pharmaceuticals for which it is a failure to venture into rare diseases, to consider and move aggressively to help those aggrieved with a rare disease. The medicine is marketed under the name Lumizyme and Myozyme. The medication falls under the ‘orphan drugs’ category of FDA. Lumizyme and Monozyme are considered as different products as their manufacturing takes place in different kinds of bioreactors exclusive for each. Myozyme is approved for infantile-onset Pompe disease and Lumizyme for the late-onset Pompe disease by the FDA. This orphan is certainly not a failure!

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P.S.Extraordinary Measures– film is based on the true story of Mr. Crowley and you can also read a book-The Cure: How a Father Raised $100 Million – And Bucked the Medical Establishment – In a Quest to Save His Children by Pulitzer Prize-winning journalist Geeta Anand.

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