This could be detrimental to the blind faith most consumers currently place in FDA approved drugs and undermine the progress that has been made over decades. But if regulated differently with an increased focus on potential intervention in cases where the drug is showing negative prospects, could an ability to skip the regular process and gain access to unapproved drugs be in the public's interest? The story of Gleevec is a great example that illustrates why a deeper look at the process is necessary.
One of the four main types of leukemia, chronic myeloid leukemia (CML) is relatively rare. It affects around 5,000 people every year in the United States. While that seems like a worrying statistic at first, the chances of an average person getting it are close to 0 when you consider the population of the United States is above 300 million people. CML has three phases: chronic, accelerated and blast. The disease is most commonly found in adults, but it can occur at any age. The way to diagnose CML is by detecting an elevated white blood cell count; a normal person's white blood cell count is under 10,000, but patients suffering from CML have counts in the hundreds of thousands. The most common way of detecting the disease is through a routine blood test where the white blood cell count should stand out to any doctor. Treatment of Gleevec is given in daily doses of oral therapy with very minimal side effects.
Doctors like Druker, Lydon, Zimmerman found that Gleevec caused bcr-abl to take on a closed form without inhibiting most protein tyrosine kinases or serine-threonine protein kinases. Instead, it targeted specific protein tyrosine kinases such as the bcr-abl, c-abl and v-abl, among others. It represented a groundbreaking scientific breakthrough, which proved that a thorough understanding the cause of cancer at a molecular level could result in a targeted and non-toxic treatment. When people originally considered treating cancer, specifically a cancer of the blood like leukemia, non-toxic treatments did not seem realistic.
