Personalized Medicine for the Masses
How new ways of creating, developing, and funding drugs can speed the age of individualized medicines.
Imagine being pregnant, awaiting the results of a genetic test to determine if your baby is healthy. The test comes back and it indicates cancer cells are circulating in your blood. The cancer is not in your baby’s cells, it’s in yours.
That situation has occurred to a number of women during the past few years as prenatal DNA tests rise in popularity and decrease in price. While frightening, it has demonstrated that detecting cancer before signs of malignancy become apparent is possible—and it has set off a race by companies to create an early detection cancer blood test, or liquid biopsy.
Advances in gene therapy have also brought incredible results. For the first time, a child with sickle cell disease was cured by the technique, for instance, offering hope in a disease that affects millions globally.
“We envision a world without disease,” said Dr. William N. Hait, Global Head of Research & Development of the Janssen Pharmaceutical Companies of Johnson & Johnson. “To realize this, we have developed a compelling vision for changing the trajectory of health care based on approaches designed to eliminate disease through prevention, interception and cures.”
Progress towards that aspirational goal can seem slow, however. In virtually every industry, from banking to manufacturing, new technologies have boosted productivity and cut costs. But there’s one glaring exception: healthcare. Pharmaceutical and biotech companies have experienced almost a reverse Moore’s Law, with the cost of developing new therapies more than doubling in the past 10 years to $2.6 billion, according to PhRMA.
These challenges, however, are being met with innovative solutions.
One trend is the increasing involvement of individuals and patient groups in research and drug development. Billionaires and family foundations are funding medical research and even clinical trials in areas ranging from psychiatric disorders and Parkinson’s disease to prostate cancer and spinal cord injury. Meanwhile, the Cystic Fibrosis Foundation has pioneered a new model for patient groups, working with companies to successfully develop new treatments in addition to providing help for those with the disease.
Other patients and advocates are using social media campaigns to compel pharmaceutical companies to allow compassionate use for experimental medicines or push for approval when regulators are skeptical.
“If you can start thinking about customized printing of medicines based on patient’s information, all of a sudden the traditional estimates of cost go out the window.”
Oliver Fetzer, CEO of Synthetic Genomics
For instance, people living with diabetes formed a “Do It Yourself Pancreas System,” modifying an FDA-approved medical device to improve the alerts from glucose monitors. The group’s work has led to new innovations, such as real-time processing of blood glucose and predictive alerts for future high- or low-blood glucose states, hours in advance. The group was ultimately able to devise a closed-loop artificial pancreas.
The power of the crowd also can be seen in sites such as PatientsLikeMe.com. These sites connect people with similar conditions, collect their stories, and work with academic institutions and medical companies to use the information to improve research efforts.
Meanwhile, pharmaceutical companies are trying to utilize new technologies, creating apps to connect with customers and potential clinical trial volunteers. According to a survey by Tufts Center for the Study of Drug Development, drugmakers used social media to recruit patients in 11 percent of clinical trials, with most of the companies surveyed predicting that percentage to increase.
The rapidly declining costs of gene sequencing may also bring a long-expected payoff: smarter drug prescribing and better clinical trials. Testing for the variants of some genes, for example, reveals who will experience strong or weak effects from specific medicines. That enables doses to be tailored to each individual for maximum effectiveness. Inexpensive whole-genome sequencing would allow this pharmacogenetics approach to be used for virtually any drug, reducing the tremendous current toll from side effects and drug-drug interactions.
In addition, gene sequencing makes it possible to spot those most likely to respond—or not to respond—to experimental drugs. Select only the good responders for clinical trials, and trials should be shorter, cheaper, and more conclusive.
The rapid increases in the understanding of the underlying mechanisms of disease are also stepping up the pace of drug discovery. New drugs that inhibit the programmed T-cell death protein (PD-1) have proven to be remarkably effective in treating melanoma, and are now being tested against a range of other malignancies, including ovarian and lung cancer.
Companies are also exploring other new targets, such as the RAS signaling pathway, and developing new approaches. Examples include linking cancer-killing drugs to tumor-targeting peptides to create what is being called Pentarins™, or creating large numbers of live T cells engineered to become ferocious cancer killers.
In the clinic now are new classes of drugs to fight migraines, multiple sclerosis, and Parkinson’s, along with vaccines against the Zika and Ebola viruses, among many others. In fact, the number of clinical trials registered on ClinicalTrials.gov is now three times higher than in 2010—and nearly 60 times higher than in 2000.
Finally, advances in technology are bringing us closer to truly personalized medicine. Currently, engineering patients’ own cells to fight their unique cancer, or creating other individualized treatments, is prohibitively expensive. But what if we can “write” DNA at a low cost? In fact, we can, says Oliver Fetzer, CEO of Synthetic Genomics: “If you can start thinking about customized printing of medicines based on patients’ information, all of a sudden the traditional estimates of cost go out the window.”
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