I originally shared this post as an announcement to the 15,000+ members of my Digital Health group on LinkedIn.
In this update:
- 15 Influencers Shaping Digital Health
- Happtique Mobile Health App Certification Program
- Making Sense of Sensors (Jane Sarasohn-Kahn on CHCF)
- X PRIZE’s Nokia Sensing X CHALLENGE – Application Deadline
- Upcoming Events: Future of Genomic Medicine VI Conference
15 INFLUENCERS SHAPING DIGITAL HEALTH
The list of 15 Influencers Shaping Digital Health (via BIONIC.LY) was compiled by London-based digital communications professional Stephen Davies, and is noteworthy because he accurately includes (as part of digital health) leaders in the field of genomics, e.g. Anne Wojcicki, Co-founder of 23andMe, and Jessica Richman, CEO at uBiome. I’m incredibly honored to be included in such esteemed company.
http://bit.ly/15ShapingDH
HAPPTIQUE CERTIFICATION PROGRAM FOR MOBILE APPS
In announcing the new “Happtique Health App Certification Program,” CEO Ben Chodor stated that “Healthcare professionals and consumers need third-party certification to verify that the app they are prescribing or downloading delivers credible content, contains safeguards for user data, and functions as described.” The new program contains standards to be used by partner organizations in assessing operability, privacy, security and content of health apps.
http://bit.ly/HapptiqueAppCert
MAKING SENSE OF SENSORS: HOW NEW TECHNOLOGIES CAN CHANGE PATIENT CARE
Well-known health economist Jane Sarasohn-Kahn (@HealthyThinker) created this excellent report for the California HealthCare Foundation (CHCF, @CHCFNews), a nonprofit grant-making philanthropy. Note the cool infographic on passive sensors.
http://bit.ly/CHCFSensors
X PRIZE’S NOKIA SENSING X CHALLENGE – APPLICATION DEADLINE APPROACHING
Along with the Qualcomm Tricorder X PRIZE (@TricorderXPRIZE) and the Archon Genomics X PRIZE (@AGXP), the Nokia Sensing X CHALLENGE (@NokiaXCHALLENGE) being administered by the X PRIZE Foundation (@XPRIZE) is fostering digital health innovation and catalyzing the creation of a collaborative global innovation ecosystem. The goal of the Nokia Sensing X CHALLENGE is to transform personal health with sensing. Innovation in sensing is an important component to creating a means for appealing, usable, smarter digital health solutions.
The CHALLENGE is comprised of two distinct competitions running through 2014, with total prize purses of $2.25 million. The application deadline for Challenge #1 is less than five weeks away.
http://bit.ly/SensingXChallenge
UPCOMING EVENTS: Future of Genomic Medicine VI Conference
Along with my Popper and Co. colleague Shane Climie, Ph.D., I’ll be attending the Future of Genomic Medicine VI conference in La Jolla/San Diego, CA (March 7-8). I’m excited to learn the very latest about opportunities for genomics to change medical practice and make healthcare more precise and individualized. I gather that the term is now “Precision Medicine.”
I’m particularly looking forward to hearing the following people speak:
- Gholson J. Lyon, MD, Ph.D.,
- Cinnamon S. Bloss, Ph.D.,
- Misha Angrist, Ph.D.,
- Daniel MacArthur, Ph.D.,
- George M. Church, Ph.D. (encoded 20 million copies of his book into DNA: http://bit.ly/ChurchColbert),
- Jonathan Eisen, Ph.D.,
- Atul Butte, M.D., Ph.D., and
- A.J. Jacobs, author of “Drop Dead Healthy”.
Note that all of the folks and organizations listed above are included in my Digital Health Twitter list: http://bit.ly/DHTwitterList. And you can access the comprehensive global digital health event list at http://bit.ly/DHEvents.
Join the 15,000+ member Digital Health group on LinkedIn to stay up-to-date on these and many of the other happenings in the digital health space. And, if you have a digital health idea you’d like to discuss, please contact me directly at sonnier@popperandco.com to learn how Popper and Co. can help guide your business development strategy.
Tags: digital health, digital heatlh events, genomic medicine, genomics, healthcare events
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The cost and risk associated with biopharmaceutical R&D is enormous and the rewards are increasingly elusive. For many young firms, the “valley of death” is a very real obstacle. What can startups (and larger companies) do? In the first blog post based on my interview with H3 Biomedicine CEO Markus Warmuth, we discussed H3’s strategy to propel innovative therapies into the market using a tighter time frame based upon a new research and clinical paradigm that is almost the reverse of traditional biopharmaceutical development. In this segment, Warmuth shares with us some specifics about H3 Biomedicine’s unique approach to drug development.
Q: What is the current approach to drug discovery at H3?
Warmuth: It is very target centric. Our discovery and development is focused heavily on target identification and validation. When systematic gene expression studies started to come out, it came with a lot of hope but then failed the industry. We found out that a change in the expression of a gene doesn’t necessarily mean a change or impact on the course of a disease. Instead, gene mutations have proven very powerful in predicting disease relevance. And, through public efforts like The Cancer Genome Atlas (TCGA) now we have more information at hand to draw conclusions quickly about the specific consequences of mutations. In some cases we find that alternative splicing can provide some insights, and this is an area we are very focused on. However, we have observed that mutations – and those are the majority – can inactivate or change the function of a gene or its product such that the mutated gene might no longer be a viable drug target. Under such circumstances we typically need to find targets that lie downstream or are synthetic lethal to the target – an area we are exploring very heavily.
Q: What is the best method for target validation in drug discovery?
Warmuth: There’s no simple answer. You have to go gene by gene and ask, “What are the key scientific questions that would support the nomination of a given gene or its product as the target for a discovery program?” The right method or technology is the one that gets you to the finish line with the most convincing data. Today, you can’t bank on one specific technology. We’re trying to diversify our methods to find the right answers related to different kinds of targets.
Q: How are you different from “big pharma”?
Warmuth: I’m not claiming that our approach is better, but it’s unique—it’s completely focused on our strategy. In a traditional development paradigm, there are a lot of clinical failures, especially in Phase 2, because the discovery process is very compound-centric. The only time most researchers in drug discovery think about the target is at the beginning of a project, prior to a screen. From then the process and milestones are very compound (or NCE) centric. They do a screen to find hits, they take the “hit” to a lead, keep optimizing the lead candidates, select, and continue to optimize the compound until they’ve arrived at a compound ready to put in the clinic. In this process, many times scientists forget to ask whether the original hypothesis around the target still holds up, because they are rewarded for bringing a compound forward. So it’s all compound-centric. We’re trying to support a target-centric approach. We challenge the target and our hypothesis at every step of the preclinical process, with different methods and compounds. Because of that, we will have by the time we enter the clinic so much confidence in the target that, we’re not trying to just “see what we get.”
Q: What is a druggable target?
Warmuth: That is a target that is amenable, and responsive to current drug discovery approaches. Some targets are considered to be “undruggable,” if their activity cannot be modulated using compounds that are found in typical libraries of drug – like molecules. But with the right library, you might be able to “drug” the target. There are two important considerations here:
- Chemical space; some compound libraries are geared to one class of target, but sometimes one still needs to switch to another class of target. Research consortia would be great; they would enable us to exchange our chemical libraries more easily, so we wouldn’t have to reinvent our own chemicals. At H3 we’re building a proprietary chemical library that’s suitable for certain classes of target, including those which have traditionally been considered to be undruggable.
- Have the right assay in place. Many organizations settle too quickly on an established assay. We’re trying to establish a screening platform that’s medium throughput (3,000-5,000 compounds at a time). We can then look at different versions of an assay to find the right one. We need to have the right components in an assay and make sure the assay allows the enzyme to be regulated the way it would be in a cell.
Q: Are you developing companion diagnostics? How do they fit within your drug development strategy?
Warmuth: This is absolutely key to our strategy, which is patient-centric. Every project we’re working on starts with a hypothesis derived from real patients (or, more specifically, patient samples). Then, we always want to explore the effects of a drug candidate in a population related to the population we started from with our original genomic data (we would not study the same patients, but a similar population with same mutation). But we need to identify them with companion diagnostics. This area will get more complicated as patient populations get smaller and smaller. Today, target-centric developers are looking at something that happens in 5 to 10 percent of a certain cancer type, which makes recruiting for clinical trials challenging. As an example, our lead project is on a mutation found in a gene called SF3B1. We know this mutation occurs in multiple different cancer types, but in some it is very rare, in the order of 3 to 5 percent. It will be crucial to have the right companion diagnostic in place to allow selection of those patients once we are in a clinical trial.
Q: Where do you believe H3 will be in five years?
Warmuth: We want to be at the stage where we’re very close to clinical proof of concept. We’ve validated a new discovery paradigm that is focused on human biology, the human cancer genome and new metrics of drug discovery. It would be nice at that time to be able to reflect on the results of the strategy that we have discussed today; to have access to more models, to more closely incorporate patients as part of the discovery process, and to be able to access patients more easily for hypothesis testing.
What do you think of H3’s model? How have advances in genomics affected your product development strategies? Is there a way to digitize this new biological knowledge, or even create tools to help uncover other biological trends? Please share your thoughts with us here.
Tags: biological trends, biomedicine, biopharmaceutical, drug development, drug therapies, genomics, H3 Biomedicine
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Digital health is propelled by many different drivers: genomics research that allows for inexpensive, accurate sequencing (and biomarker discovery), smart phone and internet technology that provides consumers with access, and the increased power of consumers to demand remote healthcare services. Perhaps as a symptom of this increased demand (or at least awareness), we’ve observed digital health as a topic on two popular television shows.
Recently, Dr. Eric Topol, author of The Creative Destruction of Medicine and an advocate of these drivers who provides much commentary on digital health, appeared on the television show The Doctors. What was significant about this appearance?
- He reached a large, mostly consumer audience with the message of digital health and the innovations that are happening vis-à-vis empowerment provided to patients by mobile devices.
- He introduced biomarkers for certain diseases and the sensing technologies for them, and showed how powerful the combination can be in disease prediction and prevention.
- By illustrating the power of a smart-phone attached electrocardiogram, he underscored the notion that consumers/patients will be connected to their doctors remotely.
As consumers become more informed and empowered, they will continue to drive the disruption of healthcare delivery in the U.S. and elsewhere, helping to bend the cost curve downward, states Dr. Topol. This will happen as more healthcare is handled outside of hospitals, ensuring healthier consumers who are less likely to need care, and moving us into the realm of disease prevention.
As these changes are rapidly altering the healthcare landscape, it’s important to note how we got here. Former President Bill Clinton noted recently on The Daily Show that it was the collaboration of government, non-profits and the private sector that gave us both digital technology and the genomics advances behind digital health (he specified how San Diego, California, was transformed from a Naval center into a genomics/digital hub, thanks to companies like Qualcomm, the San Diego mayor’s office, the University of California, San Diego, and the non-profit J. Craig Venter Institute). Genomics is an important component of the digital revolution we’ve seen mostly in the academic and research worlds thus far, but which will be a big part of medicine and healthcare going forward.
What did you think of Dr. Topol’s appearance on The Doctors? How close are we to the “disease prevention” model of modern healthcare? Is the San Diego formula of private/public/non-profit partnership the only successful one? What do you make of a former U.S. president spending part of a TV interview speaking about digital health? We’d love to hear your thoughts.
Tags: digital health, digital revolution, disease prevention, disruption of healthcare, Dr. Eric Topol, genomics, The Doctors
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On the heels of our team’s attendance at some key conferences over the last several days, I had a chance to talk with my colleagues. Shane Climie attended the JP Morgan Healthcare Conference in San Francisco, Ken Walz participated in OneMedForum (across the street from the JP Morgan conference), and both Ken and Paul Sonnier were at the Digital Health Summit in Las Vegas. Following are the highlights from my post-conference discussions with the team:
CP: Paul, tell us what happened in Vegas that shouldn’t stay in Vegas?
PS: There were two themes that will—and should—escape Vegas and transform our health. The first theme, digital health, was underscored by Eric Topol’s keynote address and hardcover book release. This was the most cogent expression I’ve seen of the convergence of consumer health, clinical care, research, and life sciences. Greg Lucier, CEO of Life Technologies, illustrated the second theme with his announcement of the company’s new $1,000 genome sequencer, which has the ability to be used in a doctor’s office. Lucier explained that technology is moving medicine into the “genetics age.”
CP: Ken, what did you take away from the OneMedForum and the Digital Health Summit?
KW: Generally, the panelists at OneMedForum (who represented private companies) were optimistic about the impact of innovation on healthcare quality, but were less optimistic about investment in these innovations. They also cited the usual impediments to progress – regulatory hurdles and reimbursement issues.
There were also some standouts among diagnostics and device companies, including:
- Invuity, a late-stage medtech firm, which has developed a device to deliver direct illumination, without heat, to assist surgeons.
- BrainScope, another late-stage firm, which has developed a non-invasive way to rapidly diagnose and assess traumatic brain injury.
- Ridge Diagnostics, which is developing diagnostics for neuropsychiatric disorders, including clinical depression.
At the Digital Health Summit, John Sculley, the former CEO of Apple who is becoming familiar to the many people reading the recently published biography of Steve Jobs, compared the current state of the digital health industry segment to that of the PC business in the early-1980s. We can only hope that some of the young companies in this space grow to play as important a role in our lives and our economy as Apple, Microsoft and IBM have.
CP: Shane, what were some key observations from the JPM conference?
SC: There was a bit less optimism here among instrument and tool companies, as the presenting companies from this sector seem to be struggling to maintain growth rates. Reduced (or flat) funding within the academic market is having an impact on sales, even though academic markets constitute no more than 20 percent of those companies’ revenues.
Future sales growth and manufacturing of product may depend on the success of these companies in getting into markets in China, India, South Korea, and Brazil (though North America and Europe will continue to be important).
As Paul mentioned, the announcement of large-scale, low-cost genome-scanning technologies shows the remarkable progress in next-gen DNA sequencing. At the same time, we’re seeing more software tools to support data analysis and cloud-based tools that make this technology accessible to more labs.
Clinical markets for sequencing could expand to $24 million in the next few years, while cancer and newborn sequencing markets could explode, perhaps up to $300 million. But the biggest market of all? Consumer sequencing, says Illumina CEO Jay Flatley, now that the technology is easier to use, and more affordable.
Next up? Paul has helped to organize an event focused on the integration of life science technology developments into digital health. “Personalized Medicine: How Genomics and Wireless Technologies are Making Healthcare More Predictive, Preventive, Personalized and Participatory” will take place in San Diego on January 24. Stay tuned for more learnings around medical and life sciences innovation, as we’ll report them in this blog.
Did you attend JPM12, OneMedForum or the Digital Health Summit? Do you have takeaways to share with our readers and us? Do you agree that this is the year we will see the power of the consumer in digital health? We look forward to hearing from you.
Tags: clinical health care, Consumer Electronics Show, consumer health, Digital Health Summit, genomics, JP Morgan Healthcare Conference, life science, OneMedForum
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To kick off the New Year, we’re attending three key conferences next week. Ken Walz and I will be in San Francisco, so we can tap into both the JP Morgan Healthcare Conference and OneMedForum, and Popper and Co. guest blogger Paul Sonnier will be at the Digital Health Summit, which is held along with the Consumer Electronics Show in Las Vegas. Following are some of our thoughts as we prepare to take off.
Ken:
Every year we look forward to the JP Morgan Healthcare Conference as an opportunity to renew industry acquaintances, look for business opportunities on behalf of our clients and to do a pulse-check on the outlook for the coming year. The overall sentiment at JPM is often cited as an industry barometer and by Tuesday or Wednesday we’ll all be reading and hearing about “the mood at JP Morgan.” Hopefully, this year that mood will be “optimistic.”
Recently, other conferences have taken advantage of the swarm of life science professionals descending on San Francisco by scheduling events that complement rather than compete with JPM. For example, the OneMedForum takes place across the street from JPM’s venue with a focus on emerging companies and panel discussions; this forum is distinctly different from JPM, with its packed agenda of (mostly) public company presentations.
Paul:
Could this be the year we see tangible traction vis-a-vis the convergence of genomics, consumer health, and clinical health care? I’ll find out how close we are at the Digital Health Summit at CES, the Consumer Electronics Show. I’m looking forward to hearing Greg Lucier, CEO of Life Technologies, talk about his company’s intentions for the digital health space (one of Life’s goals is to drive the cost of a complete genome analysis below $1,000).
Also, world-famous cardiologist and digital health visionary Dr. Eric Topol will release the hardcover (non-digital) version of his book, The Creative Destruction of Medicine: How the Digital Revolution Will Create Better Health Care. I’m also hoping to see a demonstration of Qualcomm Life’s 2Net Platform. Paul Jacobs, the CEO of Qualcomm – the parent of Qualcomm Life, which is its wholly owned subsidiary – will discuss the platform’s ability to catalyze the ecosystem of digital health innovators.
We’ll bring back our impressions and views of these exciting meetings and what they mean for you – our life science clients, but feel free in the mean time to let us know how you view our industry as you face the year ahead. Please post your comments below.
Tags: clinical health care, Consumer Electronics Show, consumer health, Digital Health Summit, genomics, JP Morgan Healthcare Conference, life science, OneMedForum
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Genomic research is accelerating at a rapid pace and improvements in technology are fueling these advances (as has previously been addressed within the Popper and Co. blog). We’re now entering a phase of evaluating how to incorporate translated genomic information into clinical testing. With this comes a critical need to verify how and when to use a test, how these tests can modify clinical care, and how this process translates into improved outcomes for patients.
In April, Margaret Piper, Ph.D., M.P.H., presented at the Personalized Medicine Partnerships Conference outside of Washington, DC. Dr. Piper is director, genomic resources, at the Technology Evaluation Center of the Blue Cross and Blue Shield (BCBS) Association. Her presentation, “Assessing the Evidence for Genomics: Focus on the Patient,” centered on the impact of genomics on administrative processes and the adoption of new technologies into clinical care. As Dr. Piper noted, “We’re generating a lot of information that relates genomics to disease, but we’re only just starting to gather information on how to translate this into treatments and medical decision making.”
Standing on the cusp of this rising tide of genomics data raises challenges for health providers (including medical school students) as well as for insurance administrators and health technology companies. The BCBS Technology Evaluation Center seeks to provide healthcare decision makers with patient-centered assessments of new technology based on evidence (or essentially, on published data). The center conducts systematic reviews, issues special reports, assesses clinical evidence, and uses a panel of independent medical experts to provide assessments to aid in creation of guidelines and practice patterns. Most importantly though, the center strives to evaluate outcomes that patients can appreciate.
After hearing Dr. Piper, I directed some questions to Caroline Popper of Popper and Co. to glean her insights. The first question involves the Center’s criteria for issuing basic guidance only on “published” outcomes.
JLM: Given that the Center is only providing guidance based on published evidence, what might this mean for timing in terms of adoption of new technologies into the marketplace?
CP: Making data-driven decisions and evaluating costs vs. benefits makes sense. However, there are other market forces at play that will drive adoption before rigorous assessment is complete—such as basic consumerism and the Internet. Over time though, a balance between sound data-driven decision making and what I call “irrational exuberance” will be found. Clearly, organizations like the Technology Center have a big role to play. Most importantly, they should make sure there’s no perception that simple cost-control is the only driver for adoption of technology into clinical practice.
The second issue that struck me was around coding for reimbursement since Dr. Piper explained how healthcare plans are still in the dark about how to code molecular testing, for example. She noted that BCBS is working along with various insurance plans to address this issue, and that coding changes that may be implemented in 2012 may help. Still, the issue of immediate processing bears some further thought.
JLM: What can be done today to help insurance plans have a clear path for covering molecular testing? Can you shed some light on pending coding changes?
CP: Dr. Piper may have been referring to the implementation of codes by Centers for Medicare and Medicaid Services that have more to do with the value of the information generated then the work units that generate the information. This process will reimburse tests that are very useful and improve quality while saving cost, even though the tests may be simple to perform. It will eliminate or reduce the common default practice of code stacking—a process that piles up as providers use as many existing codes for every component of a test as possible in order to achieve a higher reimbursement.
And lastly, I wanted Caroline’s insight on a part of the presentation centering on how device or diagnostics companies bring their technologies to Dr. Piper’s Tech Center for assessment. According to Dr. Piper, if a device or diagnostics company has a new technology, it would bring it to the attention of one of the 39 independent BCBS entities and then the entity would feed it up to the Tech Center. The Tech Center can’t talk directly to a company about all that is needed for a positive assessment, but the Center staff might schedule an hour to talk through some suggestions.
JLM: What advice would you give to a diagnostics company getting ready to schedule an hour with the Tech Center?
CP: Have a clear idea of what the test will claim, whether it goes to the FDA or if it’s a LDT (lab-developed test). Make sure you know how the test fits into the standard care paradigm, i.e., how the physician will use the information from the test in managing the care of the patient.
JLM: What questions should company representatives be prepared to ask and what should they have on hand for the meeting?
CP: They should ask what the Tech Center considers good measurable endpoints and whether it is feasible to collect these points during the validation process. They should come with the pilot results about the analytical performance of the test, a good understanding of the competing alternatives, and a reasonably comprehensive view of all the elements of an episode of care in which the test plays a role.
Do you have other questions for Caroline on this topic? Do you have experiences with how new diagnostic technologies factor into medical decision-making? Let us know your thoughts.
Tags: bcbs, genetics, genomic research, genomics, health providers, health tech, insurance
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“Translating the knowledge we are gaining from gene discoveries into practical clinical and public health applications will be critical for realizing the potential of personalized health care and improving the health of the nation.”
Muin J. Khoury, M.D., Ph.D., Director, Office of Public Health Genomics, Centers for Disease Control & Prevention
There are several interesting and exciting developments in personalized health that extend well beyond clinical medicine and into areas of science, ethics, government policy and regulation, patient advocacy, and business. In January of this year, the Ewing Marion Kauffman Foundation published The Personalized Health Project—Identifying the gaps between discovery and application in the life sciences, and proposed solutions. This report provides insights from key thought leaders as to how far we have come, where there are gaps and barriers, and how far away we are from reaching the goal of implementing personalized health care solutions.
It is incredible to think that we are already 20 years into the Human Genome Project and so much has been gained in terms of huge data repositories of genomic knowledge. More and more we see genetic tests being utilized to prevent, predict, diagnose and treat. Some of these tests used in clinical medicine are becoming incorporated into clinical guidelines and standard of care. Additionally the cost of genome sequencing has decreased tremendously in the past 10 years, eclipsing Moore’s Law, which is typically an accurate way to conduct long-term planning for technological innovation.
(www.genome.gov/sequencingcosts)
While progress has occurred, there still exists a need to understand, refine, translate and validate the knowledge gained in the last 20 years and to apply it to personalized health. For example, understanding the complexity of the information generated from a genetic test is a key component to consider integrating into personal health. How realistic is it to assume a physician will be able to interpret genetic tests? For physicians and patients, and in the case of direct-to-consumer tests, it will be imperative to communicate clear results to the end-user. (See Point/Counterpoint: On FDA Regulation of DTC Genetic Tests.)
With the daily deluge of information written on the topic of personalized health, one source of information that I find to be particularly useful is on the CDC Genomics and Health Impact Update whereby the CDC provides weekly updates on genomics and public health along with important and useful links.
Industry conferences are another source of current information. Popper and Co. will attend the 3rd Annual Personalized Medicine Partnerships Conference from April 11-12 in Washington, DC. It will be interesting to hear firsthand how different businesses have developed successful models for bringing to fruition their strategic approaches to personalized health.
Where do you see gaps in the translation of genomic data into useful personalized health strategies? Are there “go-to” sources for information on personalized health topics and trends that you’d like to share? Please respond below. We look forward to learning more.
Tags: genomics, human genome project, life sciences, moore's law, personal genomics, personalized health, personalized health care, personalized health project
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On March 9, 2011, the U.S. Food & Drug Administration approved Benlysta® (benlimumab) for the treatment of systemic lupus erythematosus. Benlysta is the first new drug approved for the treatment of lupus in more than 50 years: a triumph of genomic medicine. The drug was born of a partnership, dating back some 18 years, between biotech Human Genome Sciences and multinational pharma GSK. The Benlysta story is an interesting example of how molecular biology and genomic technologies can be applied to create new treatments for challenging diseases, even though the timeline extended well beyond what was anticipated by the lofty projections made in the earliest days of genomic medicine some 20 years ago.
Benlysta is a monoclonal antibody that was developed by HGS and GSK from antibodies that were provided by Cambridge Antibody Technology (which was acquired by AstraZeneca and is now part of AZ’s biologics arm, MedImmune). It acts against B-lymphocyte stimulator (BLyS), which is believed to increase the production of white cells that attack healthy tissue in lupus patients.
There are at least a couple of key breakthroughs that ultimately paid off with the approval of Benlysta.
- First, researchers at HGS – well known as a pioneer in genomic medicine – applied tremendous effort to clone and characterize many human genes in the hopes of identifying drug targets and, eventually, protein-based therapeutics. BLyS, the target of Benlysta, was identified by HGS researchers as a member of the TNF family, which at that time was well recognized as a member of a family of cytokine proteins that can cause cell death and are useful as cancer therapeutics. By looking under the spotlight provided by the earlier discovery of TNF and other cytokines, HGS was able to isolate the BLyS gene (and many others). The expression profile of BLyS and functional studies (using purified, recombinant BLyS) revealed that inhibition of this protein might be an effective treatment for conditions such as lupus, which are B-lymphocyte mediated. Genetic sleuthing had laid the foundation for an assault on lupus.
- Second, BLyS was among the first generation of protein targets that were tackled via phage display, ultimately leading to the construction of Benlysta, a human monoclonal antibody that was assembled from components that were encoded within recombinant libraries of antibody-fragments. A series of regulatory hurdles were ultimately overcome, paving the way for the approval of this drug. All told, it took about 12 years from the cloning and characterization of the BLyS gene to getting Benlysta out the regulatory door. This is an interesting example of the power of genomic technologies, broadly, and genetic engineering, in particular.
Although armed with powerful tools and solid rationale, drug discovery and development generally continues to be a lengthy and extremely windy road with numerous potholes along the way.
Do you think that current technologies are speeding up the process? Or, do they only raise further questions that might slow things down in the long run? I’m interested to see how quickly companion diagnostics and other technologies will enhance and improve the drug development process. What technologies are you watching in this space? Please share your thoughts below.
Tags: astrazeneca, benlysta, genomics, genomics medicine, GSK, HGS, lupus, lupus medicine, medimmune
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