Unravelling undiagnosed diseases. Finding a cure for type 1 diabetes. How about a vaccine for dengue fever? 2014 could go down in history as the year that saw major advancements in all three areas and led to great improvements in the health of mankind.
Undiagnosed Diseases Program Brings Patients Closer to Treatment
For the estimated 30 million Americans who suffer from a rare disease, the National Institutes of Health’s Undiagnosed Diseases Program (UDP) offers a new sense of hope. What started as a pilot program in 2008 was expanded to eight of the nation’s top research hospitals.
The UDP’s approach is to bring patients in to meet with an array of specialists and to perform rigorous testing to find the cause of their potentially debilitating ailments.
The program aims to help people to finally get a proper diagnosis and, perhaps, a treatment.
Two of Samantha Anastasia’s three children lost their ability to speak in infancy. While other doctors were stumped for the next 20 years, UDP specialists were able to diagnose their disease as Aicardi-Goutieres 2B, a rare genetic mutation that affects just 450 people worldwide.
“I had never met anybody with two kids with something as severe as my kids. When they told me what they had, and there were other families, some like mine, some with three kids affected, and worldwide, I was overwhelmed with emotion. I just started to cry. I wasn't happy or sad; it was just relief,” Anastasia told Healthline in July.
Photo courtesy the Anastasia family.
Harvard Researchers Make Inroads on Path to a Diabetes Cure
Stem cell researchers at Harvard University made a breakthrough that may cure type 1 diabetes, which affects three million people in the United States alone.
Lead researcher Doug Melton, Ph.D., dedicated his career to finding a cure for type 1 diabetes after his infant son was diagnosed with the disease 23 years ago.
In October, Melton’s team published research showing they were capable of mass-producing insulin-producing beta cells from human embryonic stem cells. Using this approach, doctors could transplant new beta cells into a patient’s pancreas to restore the body’s insulin production.
“It was gratifying to know that we could do something that we always thought was possible, but many people felt it wouldn’t work,” Melton said. “If we had shown this was not possible, then I would have had to give up on this whole approach. Now I’m really energized.”
Dengue Virus Vaccine on the Horizon
One-third of the world’s population is at risk of being bitten by a mosquito that carries the dengue virus. With up to 400 million people infected with the dengue virus annually, it is the leading cause of illness and death in the tropics and subtropics. Outbreaks have also occurred in Florida, Puerto Rico, and the U.S. Virgin Islands.
Until now, there were no effective vaccines against the virus. The difficulty has been finding vaccines that can treat the four related strains of the virus simultaneously.
The latest findings from a Latin America study show that a new vaccine candidate has reached 95 percent efficacy in clinical trials.
The Cleveland Clinic has listed this dengue vaccine as a promising medical innovation for 2015.
“The world’s first vaccine has been developed and tested, and is expected to be submitted to regulatory groups in 2015, with commercialization expected later that year,” said The Cleveland Clinic, in a press statement.