Carla (pseudonym) was in desperate need of something different. After retiring from a long career as a schoolteacher, routine testing ordered by her family physician revealed a sky-high white blood cell count. Further workup led to a diagnosis of acute lymphoblastic leukemia (ALL). Despite initial response to treatment, her disease returned 18 months later and was now unshaken by salvage chemotherapy regimens.
While on an elective oncology rotation during my third year of medical school, Carla was the first patient I was assigned to monitor. Her case also provided the opportunity to learn about an innovative new medicine for patients with this difficult-to-treat blood cancer. Just moments after the nurse let me start an IV, the precisely engineered antibody began to course through Carla's veins. At the time, Blinatumomab (Blincyto), had a narrow indication for a subset of those suffering from relapsed ALL. When it was officially released to the market, one year of therapy was priced at $178,000 per patient.
Now, I find myself on the other end of the medical-economic spectrum. After the inspiring rotation, I grew motivated by the need to do better for those with leukemia. I took a sabbatical from medical school and am currently in the midst of a yearlong research fellowship. Our laboratory works to characterize the forces behind leukemic relapse. We also execute clinical trials with millions of dollars worth of experimental treatments provided for free to our courageous participants.
Blinatumomab is a member of a new modality in our anticancer arsenal: immunotherapy. A variety of immunotherapies are currently available to patients. Sipuleucel-T, a personalized vaccine, extends the survival of men with metastatic prostate cancer. Some patients will be treated with antibodies to target specific proteins on cancer cells. A different flavor of antibody therapies known as checkpoint inhibitors do not target the cancer directly but instead ramp up antitumor immunity. Finally, much excitement is currently focused on cellular therapies. In this approach, a patient's T-cells are removed and genetically manipulated in the laboratory. Upon infusion, they go to work as biologic homing devices, orchestrating a molecular onslaught against the tumor.
There is growing recognition that the promise of immunotherapy is becoming eclipsed by competition to be at its helm. In fact, most of the leading academic scientists researching T-cell treatments have co-founded their own pharmaceutical companies with investments from venture capital. Efforts toward the commercialization of cellular therapies have come with legal battles over patent infringement filed by these major players against one another. Recent news has been more sobering, with reports of several fatal toxicities in patients enrolled on T-cell trials. Pharma giant Novartis subsequently announced their plan to take a step back.
On September 9th, the major television networks, movie stars, musicians and Vice President Biden joined together for the 2016 Stand Up To Cancer Telethon. Since 2008, more than $370 million has been pledged to support the organization's strategy for research that is both visionary and cooperative. Instead of engaging in clashes over drug ownership, they assemble top physicians and scientists into one room to brainstorm new projects together. Generous funding is predicated on the development of collaborative research plans. The "Dream Teams" work to investigate the genetic and molecular underpinnings of cancer, in addition to bringing the most promising therapies and biomarker tests out of the lab and into the clinic.
This tactic is actually quite novel. Our current research infrastructure provides grant funding to scientists based on the credibility of their ideas and a track record of excellence. The enterprise was designed at a time when biomedical research was growing at a seemingly limitless pace. But as four authoritative scientists have pointed out, "Today too many people are chasing too little money to support increasingly expensive research." We of course root for our colleagues to succeed. But in reality, resources are limited and a jaded sense of rivalry has emerged between people with the same passion. The situation in cancer immunotherapy is just one example of tension from a system under strain.
Thanks to years of dedicated work, we now have the ability to gaze into the depths of cancer like never before. However, these investigations have untangled the true diversity of cancer at the genetic level. We have known for some time that the disease is not the same in everyone. But now, we are humbled by findings showing that it may not be the same in anyone.
If we are to turn the tide on cancer, our approach to research and therapeutic development needs to be increasingly robust. In my mind, this means putting patients ahead of egos and collaboration ahead of competition. Battles over intellectual property pale in comparison to the battles Carla and other cancer patients are fighting. Much can be accomplished by focusing more energy on the goal and less on who gets the credit. Through "team science," we can position our resources to deliver the best results possible. Our patients depend on it, and on us. This is the true incentive and reward.