Q: Immunotherapy is considered a huge game-changer and holds a lot of promises. Is the hype around immunotherapy justified? What are some of the exciting developments in the recent years?

A: I do believe in the promise of immunotherapy to significantly help people living with cancer.

But it’s important to find the right people to give it to, as it’s certainly not a one-size-fits-all approach. The immune system is incredibly complex, and each person’s immune system may respond differently. Some of the most exciting recent developments are related to improving our ability to identify both responders and non-responders to immunotherapy – and for the non-responders, determining why.

As my colleague Dan Chen noted last year, one of the most common biomarkers for immunotherapy is measuring the levels of a protein called PD-L1, but there are some issues with consistency and reliability with this approach. The identification of new, quantitative, independent biomarkers will likely be key to realizing the promise of immunotherapy. One such genomic biomarker, tumor mutational burden (TMB), has been shown to help predict response immunotherapy across a range of tumor types, including lung, bladder and melanoma, and the list is growing.

Technological advances like liquid biopsies are also helping. The ability to measure these genomic biomarkers from pieces of tumor DNA from the blood can help expand the promise of immunotherapy to patients for whom tissue biopsies are not feasible, such as the estimated 1 in 3 patients with advanced lung cancer who cannot undergo a tissue biopsy. This is why we have a new Phase 3 trial that investigates TMB measured from the blood as a non-invasive biomarker of response to first-line immunotherapy in advanced lung cancer patients.

Another exciting development is the incorporation of “real world” clinical and genomic data to our trial design. At Genentech we’re investing in technologies and companies that are experts in the organization of vast amounts of healthcare data, including tumor genetics, treatments, lifestyle, clinical endpoints and patient-reported outcomes for thousands or even hundreds of thousands of people. This “big data” approach can help us better understand patterns of who responds or doesn’t respond to immunotherapy. Importantly, for those that don’t respond, we have a wealth of information to use to generate new testable hypotheses about why, and we can take those insights from the clinic straight back to the lab and develop new approaches that match the clinical and genomic profiles of those individuals.

Q: Will immunotherapy over time become the standard-of-care for most cancers? What are some of the main challenges we need to overcome to make immunotherapy applicable to more cancers? What are some innovative approaches we need to take to move the needle?

A: Immunotherapy certainly has the potential to become the standard of care for many cancers. But it may be in combination with other types of therapies that involve manipulating the immune system in different ways to recognize or attack a specific type of cancer. Navigating the complexity of each person’s immune system is one of our main challenges, but fortunately the science is moving quickly.

One of the first major roadmaps for teasing apart the biology was the cancer-immunity cycle. The cycle described the diverse ways immune cells can detect, locate, infiltrate and destroy a cancer. Each of these steps in the cycle could be targeted to enhance an immune response, and this paved the way for designing “rational combinations” that fine-tune this response based on the type of cancer is present. Depending on the cancer, a given immunotherapy may be most effective with a chemotherapy, targeted therapy or another immunotherapy, and at Genentech we have dozens of ongoing clinical trials testing each of these possibilities guided by unique biomarkers.

We’ve also learned that the cycle alone can’t explain why some people respond but others don’t. There are many factors beyond the cycle that are involved. Biomarkers that characterize not only the tumor, but also the patient’s immune system can help to tailor cancer immunotherapies in a personalized way.

Understanding the state of a person’s immune system, or their cancer-immune set point, could therefore inform what approach is best for each individual. For example, some tumors don’t express any mutated proteins that can be recognized by immune cells, while in other cases immune cells may be blocked from entering the tumor by physical barriers like blood vessels. Combining this knowledge with tumor biology could bridge scientific and clinical insights and inform the most effective rational combination strategy.

But it will certainly take a village to achieve this vision, and one innovate approach we are taking is to “crowdsource” the collective wisdom of the scientific research community to inform and evolve this framework. We are now sponsoring an online, interactive version of the set point framework that allows scientists to add, edit or remove factors based on their own validated findings. Tapping into the knowledge of the entire community, in real-time, could help rapidly advance the field of personalized immunotherapy.

Q: Cancer vaccines is a growing, exciting area. What new and exciting developments make this a promising solution?

A: The challenge with cancer vaccines is that cancer cells very closely resemble healthy cells, and historically it’s been very hard to get the immune system to recognize the cancer as “non-self”. Finding cancer-specific mutations called neoantigens has been like finding a needle in a haystack.

Fortunately, there have been rapid improvements in next-generation sequencing technology, and today we can sequence the whole genome of a tumor in just days. This also allows us to identify neoantigens in individual tumor samples, and develop cancer vaccines tailor-made for each patient. It’s the ultimate form of personalized medicine.

What’s particularly exciting is how quickly we can now create these personalized cancer vaccines. We can go from sequencing to neoantigen identification to treatment administration all in a matter of weeks – crucial efficiency in what can often be a race against time.

This approach is very promising, but it is also likely that these cancer vaccines will be most effective when combined with other cancer immunotherapies, like checkpoint inhibitors. Rational combinations guided by the cancer-immunity cycle become important here. You can imagine the “one-two punch” when combined with a personalized cancer vaccine: the vaccine “primes” the immune system to recognize the disease, and the checkpoint inhibitor “removes the brakes” to enable the immune system to attack the cancer.

Q: Immunotherapy has potential for dangerous side effects (e.g. autoimmune diseases): how advanced is our knowledge in understanding to fine tune the potency of these new medicines in order to identify and stay within a safe therapeutic window?

A: I think it’s important to note that as a monotherapy, most responses to immunotherapy have generally been durable. As they become more widely available we’ll be able to understand more about their long-term safety and efficacy profile from real world data.

But given that combinations may be the future of cancer immunotherapy, here is where we’ll have to pay special attention to safety. Combining checkpoint inhibitors (e.g., anti-CTLA4 and anti-PD1) has indeed proven to be quite toxic and should only be used for certain patients.

This is a relatively new field, and we only have a few major classes of checkpoint inhibitor (anti-CTLA4, PD1, PD-L1) and cell based immunotherapies (CAR-T). In the short-term, our approach will be based on adjusting the dosing of specific combinations to make different regimens as tolerable as possible. In the long-term, we will hopefully have a larger repertoire of validated targets to combine that will be tailored to the characteristics of each individual and their cancer.

Q: Cancer immunotherapy is a relatively young field: what do we know already on the long-term cure rates, relapses, efficacy profile in relapse patients, etc.?

A: Due to cancer immunotherapy’s relative youth we don’t yet have concrete or reliable long-term data for most checkpoint inhibitors, particularly around cure rates and relapse rates. But what we do know is that for these types of immunotherapies the overall response rate across all tumors studied so far is around 10-30%.

We now have the opportunity to significantly increase those percentages through the approaches I’ve described above. Using a multiple biomarker strategy combined with a deep understanding of the state of each person’s immune system can help us determine why a given tumor might be “escaping” an immune response, and provide us with the tools to prevent that escape.

For example, one hypothesis is that immunotherapies only work in people whose immune system has previously recognized cancer cells. Biomarkers that reflect pre-existing immunity can help us find those people.

In clinical trials we often study large populations, but as individuals we are all unique. With a robust approach to personalized cancer immunotherapy, we can convert that 10-30% average response rate into a 100% response rate for a specific individual.

To learn more about Ira Mellman and Dan Chen’s work and their crowdsourcing initiative, visit A Community Map of Cancer Immunity.

Interview with Gabriel Bien-Willner of Palmetto GBA

Q: What does your role entail as the director of the MolDX program at Palmetto GBA?

A: The job directing MolDX is multifaceted; first and foremost the MolDX program is responsible for assessing molecular diagnostic tests on the market and makes coverage and pricing determinations for such tests and technology. This is usually done through local coverage determination policies or technical assessments.

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Interview with Peter Marks of FDA

Q: The CBER’s Regenerative Medicine Advanced Therapy Designation program has been very successful, with about 100 requests for designation in the two years of its existence. Can you please tell us about the program and how it was put together?

A: The Regenerative Medicine Advanced Therapy (RMAT) Designation program came into being as part of the 21st Century Cures Act that was signed into law on December 13, 2016.

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Interview with Calum MacRae of Harvard Medical School

Q: What patient data do we need to better understand the underlying cause of disease and how to prevent it?

A: Medicine at present is highly underdetermined and data poor. To be precise, one must be comprehensive, so medicine (with our consent) will use not only what we currently conceive of as biomedical information, but also data from across our lives.

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Headlines from PMWC 2019 Silicon Valley

A big ‘Thank You’ to all of our presenters and attendees for celebrating 10 years of precision medicine progress with us! PMWC 2019 Silicon Valley was attended by 2000 participants from 35 countries, which included over 400 speakers in 5 parallel tracks!

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Interview with Ken Bloom of Ambry Genetics

Q: Tell us more about your organization/company. What patient population are you serving and which services are you specializing in?

A: Ambry Genetics is a recognized leader in high quality complex genetic testing. We seek to find the genomic cause or contributors to rare diseases, abnormal phenotypes and hereditary disorders.

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Interview with Lee Pierce of Sirius Computer Solutions

Q: What is the state of big data and analytics in healthcare, and how to best use the reams of data available?

A: More than ever, Healthcare organizations are achieving measurable value through use of their data and analytics assets. There is more raw material available than ever to create value. This raw material is the data flowing from internal systems and applications and also from devices and systems external to healthcare organizations.

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Interview with Anita Nelsen of PAREXEL

Q: There are various new, emerging technologies that bring us closer towards a cure for life-threatening disorders such as cancer, HIV, or Huntington’s disease. Prominent examples include the popular gene editing tool CRISPR or new and improved cell and gene therapies. By when can we expect these new technologies being part of routine clinical care?

A: Today’s emerging technologies are making the promise of individualized treatment a reality.

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Interview with Ilan Kirsch of Adaptive Biotechnologies

Q: The Nobel Prize in Medicine was awarded recently to James Allison and Tasuku Honjo for their work on unleashing the body’s immune system to attack cancer, a breakthrough that has led to an entirely new class of drugs and brought lasting remissions to many patients who had run out of options. The Nobel committee hailed their accomplishments as establishing “an entirely new principle for cancer therapy.” What is your first-hand experience the impact that those new drugs had on patients?

A: For decades cancer was viewed as solely a cell-autonomous condition.

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BMS buys Celgene | Lilly buys Loxo Oncology – Does this Signal a Return to Strong Deal-Making Activities in 2019?

Bristol-Myers Squibb’s blockbuster $74B deal to buy Celgene creates an oncology powerhouse amid industrywide excitement about the rapidly evolving science and explosive growth of the sector. The agreement could signal a return to deal-making for the pharmaceutical industry in the $133B global oncology therapeutics market.

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Interview with Gini Deshpande of NuMedii

Q: What need is NuMedii addressing?

A: NuMedii, has been pioneering the use of Big Data, artificial intelligence (AI) and systems biology since 2010 to accelerate the discovery of precision therapies to address high unmet medical needs. Artificial Intelligence approaches are a natural fit to harness Big Data as they provide a framework to ‘train’ computers to recognize patterns and sift through vast amounts of new and existing genomic

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Interview with Minnie Sarwal of UCSF

Q: Genomic medicine is entering more hospitals and bringing with it non-invasive technology that can be used to better target and treat diseases. What are some key milestones that contributed to this trend?

A: Completion of complete sequence data from the human genome project, and the advances in proteomic, microRNA and epigenetic assays added a layer of pathway biology to the understanding of human diseases.

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Interview with Shidong Jia of Predicine

Q: Once sequencing has been validated as a clinical solution via trusted workflows, and coinciding with the technological developments driving costs lower, we can expect accelerated human genome profiling for clinical Dx. How soon, do you think, will we see accelerated growth and what can we expect?

A: We will see accelerated human genome profiling for clinical Dx in 2019 and the coming years as more biomarker-based cancer drugs are gaining approval.

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Interview with Iya Khalil of GNS Healthcare

Q: Artificial intelligence (AI) techniques have sent vast waves across healthcare, even fueling an active discussion of whether AI doctors will eventually replace human physicians in the future. Do you believe that human physicians will be replaced by machines in the foreseeable future? What are your thoughts?

A: I think that there’s a lot of speculation and uncertainty around AI, but I don’t foresee a time when we won’t need physicians.

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Interview with Ilya Michael Rachman of Immix Biopharma Inc.

Q: The Nobel Price in Medicine was awarded recently to James Allison and Tasuku for their work on unleashing the body’s immune system to attack cancer, a breakthrough that has led to an entirely new class of drugs and brought lasting remissions to many patients who had run out of options. The Nobel committee hailed their accomplishments as establishing “an entirely new principle for cancer therapy.” Besides CAR T-cell therapy what do you think next generation immunotherapies will look like to successfully combat cancer?

A: The next generation of immunotherapies will build on the insights discovered by immunologists like James Allison and Tasuku Honjo and extend them to modify the body’s response to tumors.

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Join me to Kick off PMWC Silicon Valley in the Santa Clara Convention Center, Focusing on Every Element of Precision Medicine

My team worked in collaboration with Bill Dalton, Kim Blackwell, Atul Butte / India Hook Barnard, Nancy Davidson and Sharon Terry to create a program that touches every component of precision medicine while bringing together all of its key stakeholders. Leading participating institutions including Stanford Health Care, UCSF, Duke Health, Duke University, John Hopkins University, University of Michigan and more will share their learnings and experiences and their successes and challenges, as they make precision medicine the new standard of care for all.

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Johns Hopkins
University Of Michigan

The Precision Medicine World Conference (PMWC), in its 17th installment, will take place in the Santa Clara Convention Center (Silicon Valley) on January 21-24, 2020. The program will traverse innovative technologies, thriving initiatives, and clinical case studies that enable the translation of precision medicine into direct improvements in health care. Conference attendees will have an opportunity to learn first-hand about the latest developments and advancements in precision medicine and cutting-edge new strategies and solutions that are changing how patients are treated.

See 2019 Agenda highlights:

  • Five tracks will showcase sessions on the latest advancements in precision medicine which include, but are not limited to:
    • AI & Data Science Showcase
    • Clinical & Research Tools Showcase
    • Clinical Dx Showcase
    • Creating Clinical Value with Liquid Biopsy ctDNA, etc.
    • Digital Health/Health and Wellness
    • Digital Phenotyping
    • Diversity in Precision Medicine
    • Drug Development (PPPs)
    • Early Days of Life Sequencing
    • Emerging Technologies in PM
    • Emerging Therapeutic Showcase
    • FDA Efforts to Accelerate PM
    • Gene Editing
    • Genomic Profiling Showcase
    • Immunotherapy Sessions & Showcase
    • Implementation into Health Care Delivery
    • Large Scale Bio-data Resources to Support Drug Development (PPPs)
    • Microbial Profiling Showcase
    • Microbiome
    • Neoantigens
    • Next-Gen. Workforce of PM
    • Non-Clinical Services Showcase
    • Pharmacogenomics
    • Point-of Care Dx Platform
    • Precision Public Health
    • Rare Disease Diagnosis
    • Resilience
    • Robust Clinical Decision Support Tools
    • Wellness and Aging Showcase

See 2019 Agenda highlights:

    • Five tracks will showcase sessions on the latest advancements in precision medicine which include, but are not limited to:
      • AI & Data Science Showcase
      • Clinical & Research Tools Showcase
      • Clinical Dx Showcase
      • Creating Clinical Value with Liquid Biopsy ctDNA, etc.
      • Digital Health/Health and Wellness
      • Digital Phenotyping
      • Diversity in Precision Medicine
      • Drug Development (PPPs)
      • Early Days of Life Sequencing
      • Emerging Technologies in PM
      • Emerging Therapeutic Showcase
      • FDA Efforts to Accelerate PM
      • Gene Editing / CRISPR
      • Genomic Profiling Showcase
      • Immunotherapy Sessions & Showcase
      • Implementation into Health Care Delivery
      • Large Scale Bio-data Resources to Support Drug Development (PPPs)
      • Microbial Profiling Showcase
      • Microbiome
      • Neoantigens
      • Next-Gen. Workforce of PM
      • Non-Clinical Services Showcase
      • Pharmacogenomics
      • Point-of Care Dx Platform
      • Precision Public Health
      • Rare Disease Diagnosis
      • Resilience
      • Robust Clinical Decision Support Tools
      • Wellness and Aging Showcase
  • Luminary and Pioneer Awards, honoring individuals who contributed, and continue to contribute, to the field of Precision Medicine
  • 2000+ multidisciplinary attendees, from across the entire spectrum of healthcare, representing different types of companies, technologies, and medical centers with leadership roles in precision medicine
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