These are incredibly exciting times in biomedical research. Though the biopharmaceutical industry is in flux, prospects for a greater understanding of the disease process, and the development of new treatments, has never been brighter. Gilead Sciences’s new hepatitis C drug sofosbuvir (Sovaldi) will actually cure most patients of their infections, and replaces treatments that were both less effective and marred by serious side effect profiles. A new combination of cancer drugs extended the lives of breast cancer patients not by weeks, but by 15.7 months compared to older treatments. Next generation sequencing technologies are enabling the rapid diagnosis of both infectious and metabolic diseases. Many rare disorders, which in years past might never have been accurately diagnosed, have given up the secrets encoded in their DNA.
In the case of cancer, many different tumor types have been sequenced and their molecular defects identified. These data will point the way towards treatments that are focused on these particular biomolecules. Not all of the genetic information will direct how the patients will be treated, but the knowledge gained may enable future therapies, or spare patients from existing modalities that won’t work. A new drug was just approved for treating melanoma, the sixth one granted by the FDA since 2011 to treat this deadly disease. Advances in cellular repair therapies like CRISPR may one day make it possible to correct genetic defects in cells, allowing for the treatment of literally hundreds, if not thousands, of monogenic diseases (those caused by a mutation in only a single gene), such as Duchenne muscular dystrophy and cystic fibrosis. And stem cells are being tested in thousands of different clinical trials for a wide variety of disorders.
Here in Seattle bright rays of light are penetrating the gloomy cycles of repeated layoffs. Adaptive Biotechnologies raised $105 million in April, and Novo Nordisk is offsetting the dissolution of their Seattle inflammation group by establishing a new obesity research team in Seattle. The most brilliant arc to shine through the region lately was the launch of cancer immunotherapy company Juno Therapeutics. Founded out of technologies coming from New York’s Memorial Sloan-Kettering Cancer Center, the Fred Hutchinson Cancer Research Center, and Seattle Children’s Research Institute, the company has raised an astounding amount of capital ($310 million) in its first year. The sum reflects the enthusiasm that many venture capitalists and institutional investors have for this approach. As a bonus, Juno has been able to hire local biotech veterans from immunology-focused companies including Immunex, Dendreon, and Xcyte Therapies. More good news: Celgene has committed to establishing an “Immuno-Oncology Center of Excellence” in Seattle, although details of its plan have not been made public.
Juno, though, is not your typical biotech company. Most biotechs don’t have the luxury of raising money after their technologies have already been at least partially validated by small-scale clinical trials. Even if Juno’s treatment regimens can be successfully expanded at scale, the cost associated with them is going to be uber-expensive for payers. That, in turn, is going to attract the attention of those Big Pharma companies who need to fill patent-expiration holes in their revenue streams (which in any given year is most of them). Then we’ll be left with the million-dollar question of whether a future acquirer wants to expand its presence here, or if it’ll be more layoffs as part of “The Never Ending Consolidation Story.”
The Bigger Picture: Biomedicine Faces Challenges In Multiple Areas
The loss of biopharma jobs is a significant concern here in Seattle. However, jobs are just one of the issues facing the biomedical industrial complex. Declines in pharmaceutical research productivity and the challenge of drug affordability continue to be vexing problems with no easy solutions. Funding for government sponsored biomedical science has endured serious cuts in recent years, especially for those supported by the National Institutes of Health. It’s driving many scientists to retire or change jobs. A recent paper, “Rescuing US biomedical research from its systemic flaws,” written by some of our nation’s most prominent research scientists, points to the need for fundamental changes within this system. They declare that the status quo is unsustainable. Whenever you hear about the need for science, technology, engineering, and mathematics (STEM) jobs, keep in mind that not all of these positions are created equally. While there is a strong need for more engineers, it appears that there are not enough jobs out there to employ the current crop of biologists as well as those presently earning their PhDs in this field. I’ve given career development talks at a number of area academic institutions in the past year, and I can tell you that many of the graduate students and postdocs are very concerned about their future job prospects.
If we’re going to talk about completely remaking our biomedical enterprise in the U.S., then we should also engage in some serious conversations as to whether or not our current system for coming up with new drugs is the best that we can imagine. Maybe it’s time to think about new ways of funding biomedical science. There are some non-profit organizations that work on drug development, and this model should be given a closer look. Roger Stein and his colleagues at MIT have suggested a new model for funding drug development by financing bonds to pay for it. While I’m not an economist, I like the approach because it illustrates creative thinking and it could work out financially. For those of you who haven’t been paying attention to drug costs, paying for new medicines, especially in oncology, is becoming increasingly problematic. It’s one thing to pay a lot for a curative drug that can save your life (e.g. Gilead’s hepatitis C drug sofosbuvir, priced at $84,000 in the U.S.), but some of the most expensive drugs in oncology only extend most patients lives by a few weeks.. These high costs led cancer doctors at Sloan-Kettering to write an editorial explaining why they were not going to prescribe a new drug called ziv-aflibercept (Zaltrap): its high cost outweighed its benefits. This resistance persuaded Sanofi, the developer of the new medicine, to cut the price in half.
Are Our Efforts to Encourage Biotech Growth Competitive With Other States?
As Seattle Seahawks quarterback Russell Wilson likes to point out, if you want to achieve success, “the separation is in the preparation.” A number of other states appear to be doing a better job of investing in their biomedical futures than we are. California launched its $3 billion stem cell initiative in 2004, an enormous commitment at the time. The University of California system just announced that it’s making a $250 million investment in venture funding to back the ideas of researchers at its member institutions. Massachusetts launched a $1 billion initiative (a mix of loans, grants, and tax credits) in 2008 to boost its biotechnology industry, although it was later scaled back as a result of the financial crisis. I’m sure that supporters and detractors will argue over the exact benefits of these investments for years, but what’s not debatable is that these two states are the industry leaders in biotechnology.
They’re also not alone in promoting biotechnology. New York City launched a bioscience initiative in 2011 as a public-private partnership, and followed that up in 2013 with a $100 million investment in early-stage life science companies. It also helped recruit Seattle’s Accelerator to establish a branch in Manhattan that will fund local startups.