We're just at the dawn of molecular medicine. And it's going to change everything.
Take cancer treatment, for instance. Next generation therapies for cancer - including new molecular-targeted therapies, nanotechnology enhanced chemotherapy, gene therapy, and cancer immunotherapy - have the potential to "disrupt" tradiational cancer treatment paradigms, radically improving outcomes and (in the long run) sharply lowering the costs of treatment.
Researchers and the media have been talking about the revolution in "personalized medicine" for more than a decade now, but that just means that the most promising therapies are just beginning to reach the clinic now, with even more powerful therapies in the pipeline behind them.
As these treatments reach the mainstream, they will make many of our current health care debates obsolete. Every few years, we wring our hands about the cost of new drugs, and ask how pharmaceutical companies can charge so much for treatments that only extend life by a few weeks or months.
Of course, incremental innovations are better than no innovation at all, and new some cancer therapies, like Gleevec, are truly "game changers", and for a handful of other types of cancer (like breast cancer and testicular cancer) survival rates have skyrocketed as companies and researchers have substantially improved both diagnostics and treatments. But for most solid tumors, and some blood cancers, the prognosis is still unremittingly grim and the treatment costs are very high.
That prognosis, however, is likely to change, as both the effectiveness of new treatments rises and their cost plummets as new technologies mature. For instance, the New York Times this week chronicled how researchers at the Children's Hospital of Philadelphia genetically re-engineered leukemia patient Emma Whitehead's own T-cells - using a deactivated version of the virus that causes AIDS, no less - to attack her cancer, acute lymphoblastic leukemia. This was a last ditch experimental treatment, because Emma's cancer had resisted every other treatment her doctors had tried. The Times explains:
To perform the treatment, doctors remove millions of the patient's T-cells - a type of white blood cell - and insert new genes that enable the T-cells to kill cancer cells. The technique employs a disabled form of H.I.V. because it is very good at carrying genetic material into T-cells. The new genes program the T-cells to attack B-cells, a normal part of the immune system that turn malignant in leukemia.
The altered T-cells - called chimeric antigen receptor cells - are then dripped back into the patient's veins, and if all goes well they multiply and start destroying the cancer.
The T-cells home in on a protein called CD-19 that is found on the surface of most B-cells, whether they are healthy or malignant.
What is even more remarkable is that when Emma developed a life-threatening complication from the immunotherapy, her doctors were quickly able to run a battery of diagnostic tests to isolate the specific immune response that was causing the problem (an overproduction of interleukin-6). They then used another drug (off-label, normally used for rheumatoid arthritis) to save her life. The treatment has since been used successfully in several other patients who developed the same complication.
Researchers might have to administer another dose or two of the therapy later, or might not - they can easily track her cancerous B-cells to make sure the disease remains in check. Her genetically altered T-cells, however, will remain in the body, roaming hunter-killers seeking out signs of cancer. (Although, as the Times nnotes, the engineered T-cells attack all of Emma's B-cells, cancerous or not, since they both express the same cell surface protein. However, if researchers can identify a more specific cancer protein signature they can spare the healthy cells by making the engineered T-cells even more precise.)
The work at CHOPs is a stunning advance for cancer immunotherapy and personalized medicine, since the T-cells must be tailored for each patient, rather than brewed in enormous vats, like traditional drugs. The drug company Novartis is backing the commercial development of the technology, so it can eventually be scaled up for far more cancer patients - and eventually applied to other cancers, including solid tumors. (The first successful application of cancer immunotherapy, although it appears to be less successful as a therapeutic, is Provenge for advanced prostate cancer.)
As we suggested earlier, another bright spot in this story is the cost of the modified cell therapy, about $20,000 per patient, according to the Times. Compare that to the cost of chemotherapy. One drug, Clolar, can cost $68,000 for two weeks of treatment for relapsed pediatric ALL. Bone marrow transplants, another ALL treatment option, can cost hundreds of thousands of dollars and long hospital stays.
Another advantage of tailored immunotherapies (like other targeted therapies) is that they can show efficacy rapidly in smaller clinical trials, lowering the cost of development and allowing companies to press FDA regulators for rapid marketing approval in light of the high benefit-risk ratio for patients who've run out of other options. Doctors will then - as Emma's did in her case - fine tune them on the fly as diagnostic and treatment options improve around them.
CHOPs and Novartis are helping to pioneer a completely different model of drug development, and drug approval that can help de-risk the entire industry and enable rapid follow on innovations. While industry is going through the doldrums now, Emma's saga is a welcome sign that the future of the industry - and the science underlying it - is bright.
Of course, for Emma Whitehead and her parents, just having a future to look forward to is enough. The next time you hear someone worry about the cost of new cancer treatments, you might want to mention her story to them.