This week I attended the 3rd annual World Conference for Personalized Medicine held at Microsoft’s Computer Science Museum in Mountain View, California. A museum for the history of computing seemed appropriate for a new industry which has been dramatically impacted by the accelerating pace of technological innovation. The most oft quoted theme in the conference: that the price of sequencing an entire genome has dropped at breathtaking speed over the last few years. Just four years ago, the price for a genome sequence hovered in the tens of millions of dollars. Now the price is below $10,000 and still dropping. This is hardly news. But how is this affecting the industry? It was ventured by Randy Scott, CEO of Genomic Health, that the price would go to zero dollars in time and that we as patients would all be part of a network and subscribe to a service, that of having one’s genome carefully scrutinized for genes that mean something of significance for our personal well being. This was quickly countered by Clifford Reid who held on to the idea that the price for the genome sequence would continue to drop but would end up more in the neighborhood of a few hundred dollars. Reid’s company, Complete Genomics, is a leading provider of complete genome sequences, having sequenced thousands of genomes for the NIH, academic hospitals, and private requests. Whether the price will be zero or the price of a cell phone, clearly the age of personalized medicine is upon us. What does this mean? How will this vast increase in technology affect medicine as we know it? How will this vast potential for such personalized data change the patient/doctor relationship? Will regulation keep pace or slow things down? These are some of the questions the conference, filled with speeches and presentations by the industry’s leaders--scientists, entrepreneurs, investors, IP lawyers, and government representatives--sought to address.
Lasting two very full days, the conference was split into four major categories: Targeting Cancer, New Business Models, Regulation, and Personal Wellness.
Speaking at the outset, Randy Scott contrasted the model of the healthcare world we’ve been living in with what it will be. For the past 30 years, he said, medicine has been “drug centric, where the patient has been fit to the drug.” Now we embark on an era away from the drug centric toward a diagnostic centered world. His example was breast cancer. $20 billion dollars has been spent in breast cancer drugs. Yet these drugs work only 50% of the time or less. Mr. Soctt and others after him affirmed that these numbers are not good enough with the availability of a person’s genomic data. There are certain things we know about breast cancer. With the patient’s genetic information we can give a score for low vs. high recurrence. We can target the therapy to the patient. “We are redefining the molecular biology of breast cancer,” claimed Scott whose company, Genomic Health, offers the OncoTypeDX, a diagnostic test for breast cancer patients to aid in treatment. The test can help indicate how likely it is that a woman’s cancer may return in the future (distant recurrence). Scott was the first to bring up the idea of targeted clinical trials, that is trials for a group of individuals with a homogenous genotype. According to Scott, such a targeted approach would bring the success rate up for clinical trials and shorten the length of the approval cycle.
Clifford Reid sought to dispel the myth that cancer grows fast. Instead, according to Reid, it takes 10 years from the original mutations to parental cancer cells. Mr. Reid talked about a cancer signature. With a few cells from a lung cancer tumor, his company has mapped the genome of the tumor, finding 50,000 mutations. He believes we will find signature genotypes for the lung cancer so that we can identify it in the body long before the tumor grows. Finding the cancer during the stage from mutation to the first parental cancer cells--and by beginning treatment at this stage, we can halt the cancer early. Reid also talked about a new subject which floated on the tongues of many in the audience during the breaks--that of circulating tumor cells. Many times patients hear that they are cancer free after surgery or other treatment only to find later that the cancer has come back. Tumor cells that are circulating free in the blood may be responsible. Potentially we will be able to insert “killer genes” in the cells and stop the cancer before another metastisis.
Martin LeBlanc of Caprion took the conference into the field of proteomics, or protein based biomarkers. There are many instances where tumor cells cannot be reached, such as lung cancer. In this case LeBlanc’s company has had success with tests that target certain proteins in the blood. They are collaborating with pharma companies on new drug target discoveries relating to prostate cancer, lung cancer, and TB, among others. LeBlanc was enthusiastic about his company’s approach, saying they had been profitable since 2006.
With such exciting discoveries and new tests, what is holding the industry back? Mara Aspinall is the Vice Chairman for the Personalized Medicine Coalition and CEO of On-Q-ity, a company focused on circulating tumor cell diagnostics. Mara spoke of a “battle to be waged. The question for her was whether big pharma and the insurers will play. She called for strong action and for the need for clarity with regulation and reimbursement reform. She is requesting that a new center at the FDA be dedicated to diagnostics. She proposed a pretty revolutionary drug reimbursement system based on performance. That if there was no response by a patient to a drug, that the patient shouldn’t pay. This would be an incentive for both big pharma and insurers to come to the diagnostics table.
The cancer session could best be summed up in the words of Alexis Borisy of Foundation Medicine who sat with several of his peers on a panel for questions. “Personalized medicine is no longer stories about the future, but about now.” Mr. Borisy spoke also to the need for a universal, comprehensive diagnostic test, one which includes perhaps hundreds of biomarkers.
After this first session, there was certainly a palpable excitement in the large ballroom. The show was designed with several long breaks for networking. In the audience, which overflowed out the front doors and into the entryway, many standing without a chair, one noticed employees from Genentech, investors, scientists from Stanford, doctors, all taking notes, emailing colleagues on the spot, or tweeting about the latest discoveries. Perhaps the only word of caution during the entire morning came from one of Mr. Borisy’s colleagues in the panel discussion, Hartmut Juhl of Invidumed in Germany. He spoke to the fact that although looking at genetic mutation is certainly a robust science, there are many other variables in the tumor cell which regulate its action. We typically study a tumor cell in the lab. Yet the same tissue analyzed in the lab can be very different than when it is in the body. When Hartmut said he was tired of hearing about new break throughs which didn’t take into account these variables he received an immediate applause from the audience.
New Business Models
The afternoon session afforded its highlights as well beginning with Kathy Hudson from NIH. She announced the creation of a new center within the NIH to be called NCATS or the National Center for Advanced Translational Sciences. Kathy was speaking to the vanguard in this field, she knew it, and she reached out and connected. (Unlike the FDA who made a presentation the next day.) She made sure she showed a slide with a picture of the new NIH website which the crowd acknowledged with applause. Later Kathy joined a panel with some industry heavy hitters: George Church, well known scientist from Harvard, Ralph Snyderman, Chancellor from Duke University, Lee Hood, who developed the five instruments which are the foundation for modern molecular biology, and Brook Byers, VC investor who has worked closely with and funded over fifty new companies.
More than anything, these experienced veterans in their field all sought to design new mental models for how the future will look. Church, who was involved in the first genome sequencing project and the first commercial sequencing, talked about the day when genetic information will be shared over a social network such as Facebook. He brought up Wikipedia as an example of users being involved, putting it out there that people are willing to take healthcare into their own hands. Church, honorary keynote speaker the next day, began the Personal Genome Project which recruits volunteers willing to share their genomic data with the research community and general public. Ralph Snyderman from Duke talked about a program he set up within the Duke Medical System that gets individuals more involved in their health, saying the results were dramatic. In this system, patients (employees of the University) are given a Health Risk Assessment that includes their genetic information. An individual is then given a healthcare coach and then sets their own health goals. Snyderman said that costs had gone down, emergency visits had declined and there was a conformity of care. When the panel was asked to predict what personalized medicine would look like in 2020, Hudson said she saw everyone being a participant in research. All of them talked of patients going outside the current system. Byers said it will be all about networks--on the social level and on the molecular level. The work now was to educate patients and physicians.