We posted the story below about the promising future of personalized medicine in mid-August, 2015. Sixteen months later that future is no longer just a dream. On Dec. 13, 2o16, Democrats and Republicans united to pass the 21st Century Cures Act almost unanimously, and President Obama signed the legislation into law.
The act provides $6.3 billion to build a research infrastructure necessary to act on the many changes occurring in healthcare: precision medicine; ending cancer as we know it, through the newly named “Beau Biden Cancer Moonshot;” unlocking cures for brain diseases like Alzheimer’s; and help for people with opioid addiction.
Streamlined regulations in the way FDA approves drugs will benefit patients with rare diseases that have few or no treatments. The few opponents to the Act are concerned that some regulatory changes may lead to drug safety issues. But in the main, there is scarcely an American family who will not benefit from the downstream effects of this law.
REMEMBER THOSE OLD Star Trek episodes where Dr. McCoy used some thingamajig to instantly diagnose a patient’s illness? Although we may not be at that point yet, the new thinking in medicine will affect your health care, if not tomorrow or next year, then in the not-too-distant future.
Precision medicine is personalized medicine; it analyzes individual differences in genes, environments and lifestyles to help prevent diseases to which people are predisposed and treat diseases they currently face.
In the next year, millions will be diagnosed with diseases including diabetes, Alzheimer’s, Parkinson’s and cancer. For the last century, doctors have treated patients with the same diagnosis the same way, using one or two drugs for everyone who suffers from the same disease.
But we are not the same. Two decades ago, the human genome’s three billion lines of code were sequenced. Since then scientists have been able to distinguish discrepancies from one person to the next. We always understood that each person was different; now we can see why. It’s all right there in your genes, and today you can even have your genome sequenced overnight.
The stories about cancer patients whose chemotherapy was unsuccessful are legion. Today’s cancer patients have a less than 35 percent chance of getting the right chemo the first time. A drug can gain FDA approval with only a 50 percent success rate. What about the other 50 percent?
Drug manufacturers design medicines for the average patient—not the individual patient. Now doctors have the tools to better understand the mechanisms underlying each patient’s condition. Our genetic roadmap was always there, it just took science time to figure out how to read it.
Imagine a repository that holds your genetic information and medical and family history. If you have cancer, pieces of your tumor will be stored there. Then scientists can implant pieces of that tumor into genetically modified mice. A small army of these mice will act as your avatar, and scientists can test potential treatments on them to see which treatment would work best for you. No more chemo treatments that don’t work. Your doctor will know exactly which treatment is tailor-made for your individual genetic mutation. This is no longer the stuff of science fiction. Individualized medical care is already here.
Similar to the AIDS fight of the ‘80s or the War on Cancer, public policy now is turning its spotlight on how to better predict a patient’s future disease to develop prevention strategies and then to choose which treatments will be most effective if the patient develops that disease. In Washington—even in these highly partisan times—there’s a bi-partisan effort at a major infrastructure upgrade that will enable us to move from a one-size-fits-all approach to one that targets specific genetic mutations that drive disease.
President Obama’s Precision Medicine Initiative calls for $215 million. In Congress, the 21st Century Cures legislation provides the National Institutes of Health $1.75 billion per year for five years, and $110 million for each of five years to the FDA. This legislation easily passed the House and is moving now to the Senate.
— Jodie Klein
Jodie Klein, principal of Klein On Point, has worked in healthcare communications for 30 years.