Between the uncertainties of science, the fickleness of markets, and really just everything related to the White House, the fate of science and medicine in 2018 seems nigh unpredictable to us. So we reached out to a bunch of people who would know better and asked for their crystal ball readings of what the new year will bring. Here’s what they said.
Get ready for better gene editing
This is a transparent ploy by STAT to get inside info, masquerading as “prediction.” Nevertheless, I’ll be a good sport and give you some options. We may see:
- Methods that eclipse CRISPR, able to make any DNA change precisely and efficiently.
- Reconsideration of the 749 rider of the 2016 Consolidated Appropriations Act to allow FDA to evaluate ways to reduce abortions via sperm therapy.
- Widespread push (analogous to the 1991 ISTE Act for airbags) for making human genome reading available to and understandable by all citizens — which might be 50 times less costly than current medical practices of pregnancy termination and orphan drugs.
— George Church, professor of genetics, Harvard Medical School
New technologies will reach a tipping point
Following the approval and launch of the first CAR-T therapies for cancer and a first gene therapy approval for hereditary blindness, we will see momentum accelerate further for these game-changing, potentially curative therapies. As the first-movers contend with launch and the complexities of commercializing these new precision medicine therapies, we’ll see a huge wave of additional players, disease indications, next-gen improvements, as well as mission-critical supply and patient management solutions. And transactions. History has shown us to expect this wave to build rapidly — with considerable activity likely taking place in 2018 for CAR-T and gene therapies — and ultimately that will be positive for our industry and patients.
— Nina Kjellson, general partner, Canaan Partners
M&A is finally coming back to biotech …
After years of relying on price increases and other forms of financial engineering, large companies are learning that revenue growth is no longer an entitlement. Witness Celgene’s reckoning to see how vulnerable they have become. As an investor, it almost makes you want to curl up in the fetal position. Luckily, our industry has great diversity and smaller companies are producing science-based innovation that has never been more exciting. Those with commercial products or evidence-based data suggesting a near-term approval will finally be acquired in a big way in 2018.
— Brad Loncar, biotech investor
… which will mean big names getting bought …
Within short order after the tax bill goes into law will come the long-awaited wave of megamergers, with Pfizer bidding for Bristol-Myers Squibb, followed in short order by Merck bidding for Biogen. This is followed in fairly short order by AbbVie, Amgen, and Celgene all decrying large megamergers as destroying value but agreeing that consolidation is inevitable and trumpeting the value of “tuck-in” deals, with Celgene buying Clovis Oncology and Bluebird Bio, AbbVie buying Incyte, and Amgen buying someone (Alexion Pharmaceuticals?).
— David Chan, managing director, Jennison Associates
… but that won’t be a panacea
While M&A will go up in the pharma and biotech sector, the majority of repatriated earnings will go to share buybacks. R&D productivity will continue its downward trend overall, but bright spots will be in the approval of breakthrough therapies in gene and RNAi therapies. FDA will continue its pursuit of the regulatory science to support novel endpoints for clinical trials and will stretch that beyond oncology — but it will take a while to pull through into approvals.
— Deborah Dunsire, CEO, XTuit Pharmaceuticals
Artificial intelligence is going to shake up drug development
AI will massively extend our ability to mine clinical data for new medical insights, beyond current capabilities of the human brain. The vision is that this will lead us to more breakthrough therapies, more quickly. We’ve also seen AI’s potential to automate steps of the clinical trial process, predicting and monitoring trial enrollment, cost and quality. In the new year, I anticipate AI’s integration into the pharma world will continue to accelerate, enhancing our ability to make data-driven decisions and develop new medicines in ways that are smarter, faster and more cost-effective than ever before.
— Vas Narasimhan, head of global drug development and incoming CEO, Novartis
And VR, too
I think in 2018, we will see continued activity in genetic medicine such as RNA therapies and gene editing with the first FDA approval of an siRNA therapy and continued advancement of mRNA therapies. I also think we will see better ways to improve medication adherence, new applications and tools for cellular therapies, and more advances in medical virtual reality and therapeutic digital medicine.
— Robert Langer, David H. Koch Institute professor, MIT
We’re going to peer inside the brain
Functional brain imaging has mostly been about looking at indirect measures of brain electrical activity — blood flow, in the case of functional MRI of human subjects, or calcium flux, in the case of optical calcium imaging in animals — rather than direct observation of the electrical pulses that brain cells make. This makes the interpretability of the data challenging. In 2018, I predict that direct imaging of the voltage of neurons will start to take off, at least in the neuroscience of living mammals. Contrast agents that turn neural voltage into something observable — by a camera, or by an MRI scanner — will start to appear and become widely used in neuroscience. Human translation may not begin right away, but will start to be considered, thanks to its noninvasiveness.
— Ed Boyden, associate professor at MIT Media Lab and McGovern Institute
The health care system will finally embrace addiction as a disease …
The opioid overdose epidemic has pushed the inadequacy of previous policies and practices for addressing addiction to the fore of our national consciousness. The time has arrived to mobilize and empower health care to tackle the worst public health crisis in decades, through this needed shift in mindset. Substance use disorders can and should be screened for in general health care settings and emergency rooms, and opioid addiction can be successfully managed with medications, the way we manage other chronic medical conditions.
— Dr. Nora Volkow, director, National Institute on Drug Abuse
… and industry will be there to supply new therapies
Chronic, serious mental illness, and addiction are human tragedies. They are also budget-busters. Affecting millions of patients and their families, these conditions have a huge economic impact, dominating spending in public health and criminal justice systems around the country. For more than a decade, the focus for many in our industry has been on creating new therapies for small patient populations: genetically-defined cancers, orphan and ultra-orphan diseases. Meanwhile, a smaller number of companies, ours included, have also made important progress in developing new medicines for some of the major psychiatric disorders of our time, including schizophrenia, treatment-resistant depression and addiction. This progress will become manifest in 2018 with important new FDA decisions and more proof-of-concept results in clinical trials.
— Richard Pops, CEO, Alkermes
There might finally be hope for sickle-cell disease
We may see the first examples of gene-editing technology leading to significant therapeutic benefit for inherited diseases in 2018. Treatments currently exist for less than 500 of the 7,000 diseases with a known molecular cause. Gene editing might be a way to cure most of them. Among the earliest potential success stories is sickle cell disease. Today, the only way SCD can be cured is by a bone marrow transplant, but most patients lack a well-matched donor. So, researchers are now seeking to use CRISPR gene-editing to fix the underlying defect in a patient’s own bone marrow stem cells. If this succeeds for SCD, just imagine what might be done for thousands of other genetic diseases awaiting a cure.
— Dr. Francis Collins, Director of the National Institutes of Health
The FDA will keep the pace in approving novel drugs
There will be a record, double-digit number of FDA approvals of innovative “advanced medicines,” including CAR-T, gene therapy, and RNA therapeutics, highlighting the transformative potential of these medicines for patients.
— John Maraganore, CEO, Alnylam Pharmaceuticals
Science will find promise in the body’s ‘underground transit system’
The lymphatic system will make news with emerging science that could impact a range of diseases from cancer to neurodegeneration. Its architecture creates a sort of underground transit system of the body and emerging research suggest it could be harnessed to directly access circulating immune cells. The human brain meninges harbor a lymphatic system and are enriched with monocytes and resident T-cells which may play a critical role in neuro-inflammation. The lymphatic system also has intriguing potential to traffic engineered cells to desired locations in the body to treat a range of immune related diseases including and beyond cancer.
— Daphne Zohar, CEO, PureTech Health
We’ll find out whether biotech’s boom can endure
2018 will be a big year for VC-backed biotech, especially around whether the private market’s buoyancy in 2017 will find a positive reception from the broader buy side — both the public markets and pharma. Major traditional catalysts for the public biotech sector — outperforming large caps and the pharma M&A “put” — aren’t showing signs of strength right now, yet the private VC-backed world is booming: 2017 was the largest year ever for VC funding. Investors and private biotech executives are actively preparing for a big wave of 2018 IPOs and M&A outcomes, so expectations are riding high. The 2018 bull case is that the public markets, as well as pharma buyers, will step up with enthusiasm and embrace a large cohort of these emerging private biotechs at robust valuations; the bear case is they don’t show up. I’m confidently hopeful it’s going to be the former, but I occasionally lose sleep on the latter.
— Bruce Booth, partner, Atlas Venture
And the world will figure out how to pay for gene therapy
Globally, the pipeline for investigational gene therapies is robust and reflects the fast-paced progress made in this exciting area of science and medicine. As potential one-time therapies that may provide a lifetime of benefit, gene therapies present a unique challenge for the current U.S. health care system, which is primarily set up to reimburse chronic treatments, rewarding volume not value. I anticipate patient groups, regulators, insurers, and manufacturers will collaborate to build new models that ensure access for patients and families to accommodate these potentially transformative medicines, bringing our health care system into the 21st century.
— Jeff Marrazzo, CEO, Spark Therapeutics