Chapter 5 - Bone Biopsy, Stem Cell Harvesting, and Prayer

Summary

Multiple myeloma (MM) is an incurable blood cancer that occurs when malignant plasma cells grow uncontrollably in bone marrow. It is not curable, but doctors have made remarkable strides in treating it. As much as I wish that this never happened to me, I feel very fortunate that it did in a time when these pharmaceutical advances were possible. Whereas MM was a death sentence as recently as 10 years ago, it can be treated with a far greater life expectancy than was previously possible. AL amyloidosis is a rare disease that occurs when an abnormal protein, called amyloid, builds up in your organs and interferes with their normal function. MM and AL amyloidosis are similar diseases resulting from clonal proliferation and dysfunction of plasma cells and have likely outcomes in the kidneys because of protein deposits.

The advances in the treatment of MM have been valuable for patients who suffer from AL amyloidosis.

In an earlier section of this book, I described the challenges of illness associated with a rare disease. Because fewer people are afflicted by rare diseases, they garner less research and investment from the pharmaceutical industry, which is driven by potential profits. Developing safe, disease-curing and life-saving medicines requires considerable time and money. According to the Tufts Center for the Study of Drug Development, the cost of developing medicine from invention to pharmacy shelves is $2.7 billion over an estimated 10 years.

The primary expense of developing any drug into something that consumers can safely use is the cost of conducting the research, including clinical trials, that will show it is safe and effective and ultimately able to secure regulatory approval. These expenses can range from $10 million to $2 billion, depending on what the drug is for. Importantly, it is not just the outright costs of the research that burden drug developers, but it is the loss sustained because a majority of drugs developed never make it to the market. According to one science writer, at least 90 percent of medicines that start being tested on people don't reach the market because they are unsafe or ineffective. The $2.7 billion figure includes the cost not only of these failures but also of not putting the money spent on them into something that would give a more reliable return.