Dr. Tim Townes has been researching cures for sickle cell disease since 1985. One hot summer day in 2006, 21 years later, Dr. Townes and his lab were finally able to cure sickle cell disease – but not in humans. They cured the disease in mice; mice that had been genetically engineered to have the faulty human genes responsible for blood cell sickling. Dr. Townes cured these mice by using factors discovered by Dr. Shinya Yamanaka that could reprogram mature cells to pluripotent cells (a stem cell-like state), giving them regenerative properties that most mature cells lack. Townes took skin cells from the mice, turned them into pluripotent cells, corrected the sickle mutation, and transplanted the cells back into the mice. This treatment cured the mice.
I was an undergraduate in 2010 when Dr. Townes came to a nearby university to give a lecture on his lab’s achievement. He said that in five years, medicine would be changed forever. People would go to the doctor and have biopsies of cells taken. Those cells would be turned into pluripotent cells and then banked, creating a supply that could be used for regenerative therapies if patients ever needed them.
This prediction has not come to pass. As with many other promising technologies, complications arose almost immediately, and as of June 2016 there is only one active clinical trial using pluripotent stem cells. Why did it take the Townes lab 21 years to cure sickle cell disease in mice when it only took eight years for the Apollo program to put a man on the moon? Why have stem cells not yet fulfilled their potential to revolutionize medicine?
Scientific discovery is a long, arduous process. Gregor Mendel finished a study on the inheritance of traits like seed shape and flower color in pea plants in 1863. His work implied the existence of genes, a means to pass on those traits from parents to offspring. It was not until 1915, 52 years later, that Thomas Morgan and his fruit fly lab revealed that genes reside on chromosomes, and that chromosomes were physically passed from one generation to the next.
1915 was a long time ago, in a time without computers and when Thomas Morgan housed his flies in milk bottles from the Columbia University cafeteria (and possibly stolen from New York City stoops). But even with modern research funding and advances in technology, years often pass before a lab or field of research (e.g., Alzheimer’s, breast cancer) identifies a potential therapeutic target. After scientists point out a biological target to attack or aid, drugs will be screened for the desired effect. Once a likely drug candidate is discovered, it takes an average of ten years and $2.6 billion to bring that drug to market.
The slow speed of research is a serious concern. It is a concern for people who have diseases like Huntington’s, for which current treatments can alleviate its symptoms but not its deadly progression. It is also a societal concern as the population of the world increases and grows older. The percentage of people over age 60 in developed countries is on the rise, and since age is the largest risk factor for a host of disorders there will be a higher incidence of debilitating diseases like cancer and dementia. The world’s population is projected to reach 8.5 billion by 2030, an increase of 1.2 billion from 2015. As world population growth accelerates, problems caused by people also accelerate – most notably climate change, but also phenomena like air pollution that can aggravate heart disease and other ailments that have a greater impact on the elderly.
While many problems can be addressed through policy changes and activism, genetic disorders can only be remedied with biological research. Here at the Buck Institute for Research on Aging, our mission is to increase the healthy years of life, primarily through basic research on the causes of aging and it associated diseases. Speeding up research would go a long way towards accomplishing this mission. In the following three articles, I will discuss what I think are the three main components of increasing scientific output – time, money, and people.
(Featured image credit: Pedro Ribeiro Simões)