When Alexander Fleming took a close look at a strange mold growing in his lab, he discovered penicillin—and transformed the treatment of bacterial infections. But victory over treating or curing most diseases requires a number of smaller advances rather than a single eureka moment.
The treatment of the human immunodeficiency virus (HIV), the virus that causes acquired immunodeficiency syndrome (AIDS), is a prime example of how a series of incremental improvements can turn a deadly disease into a chronic condition that people can live with for decades. The scientific progress made by HIV researchers over the past 30 years shows how medical innovation can impact patient outcomes.
HIV thrives by invading cells and coercing them into building new copies of the virus. The first treatment to combat HIV, zidovudine (better known as AZT), disrupts one step in this viral invasion.
AZT first reached patients in 1987, six years after doctors initially described AIDS cases in the United States. But AZT isn’t a cure; it only slows the virus. Even after its introduction, the death rate from HIV infections in the United States continued to climb. In 1994, AIDS became the leading cause of death for Americans between the ages of 25 and 44, killing more than 40,000 people that year.
Then in 1995, a new kind of treatment entered the anti-HIV arsenal—protease inhibitors, which prevent the construction of new viruses inside infected cells. The combination of AZT and protease inhibitors made a dramatic impact. In 1996, for the first time since the beginning of the AIDS epidemic, the number of people dying from the disease declined.
HIV researchers have continued to develop new treatments, and doctors now can choose from more than 30 options. Some of these therapies work differently than AZT and protease inhibitors, by blocking HIV’s ability to enter cells.
Today it’s also easier to stick to treatment, thanks to innovations in therapy delivery. In the mid-1990s, patients might have swallowed more than 20 tablets every day, on a rigid schedule. But now, some patients can take an all-in-one pill just once a day.
These discoveries have transformed the prognosis for millions of people who are infected with HIV. Using a combination of available medicines, patients can often hold the virus in check for decades. As a result, the death rate from HIV infections has plunged more than 80 percent in the United States since the mid-1990s. Worldwide, the death toll from AIDS-related causes fell by more than 500,000 between 2005 and 2011.
The development of four HIV therapies contributed to a 90 percent decline in deaths from the disease. In addition to more than three million life years saved for patients with HIV, approximately $1.3 trillion was gained in economic value.
Researchers in other arenas have taken inspiration from the HIV success story. Now, meaningful improvements are transforming the treatments of some cancers, including acute lymphoblastic leukemia (ALL), the most common type of cancer in children. A child diagnosed with this disease in the 1960s had less than a 10 percent chance of surviving five years. But around that time researchers discovered certain oral treatments, such as methotrexate, could temporarily halt the disease. When they began to test combinations of these and other therapies, they obtained even better results. This progress has continued to this day, and now, today’s list of treatments includes more than 20 drugs. As a result, children with ALL now have more than a 90 percent chance of surviving at least five years.
For patients with chronic myeloid leukemia (CML), three new targeted therapies created a 68 percent decline in deaths. This decrease resulted in $143 billion in economic growth from the 700,000 life-years gained.
Taking risks and not fearing difficulties are essential methods for moving medical research forward, in order to better serve patients. “At Celgene, one of the most powerful demonstrated capabilities of our company has been to look at innovation as a way to change the paradigm of the treatment of serious diseases like hematologic cancers,” Robert Hariri, Founder, Chairman and Chief Scientific Officer and former Chief Executive Officer told the the Wall Street Journal, in their Startup of the Year Tech Innovators Week series. “And we’re not afraid of chasing very challenging problems in terms of drug discovery.”
With the evolution of therapies for diseases such as HIV and leukemia, many patients’ lives have been dramatically improved and, in some cases, extended. For cancer researchers, this serves as inspiration to create a similar success story. Already, progress in diseases such as ALL, as well as myeloma and breast cancer, among others, suggests such success is within reach, as long as we continue to support medical innovation through pro-patient and pro-innovation policies.