Speaker Spotlight: Marc Boutin, Executive Vice President & COO, National Health Council



Marc Boutin

The National Health Council brings together all segments of the healthcare community to provide a united voice for the more than 133 million people with chronic diseases and disabilities and their family caregivers.
Boutin has been actively involved in health advocacy, policy, and both federal and state legislation throughout his career. He has designed and directed advocacy strategies for legislative initiatives, which have included issues ranging from access to healthcare to cancer prevention. Before joining the Council, Boutin served as the vice president of government relations and advocacy at the American Cancer Society for New England. In addition, he was a faculty member at Tufts University Medical School, where he lectured on healthcare policy.

At the CHI 2011 Annual Meeting, to be held Nov. 3 in San Francisco, Boutin will discuss the Moddern Cures Solution, a proposal to update the U.S. regulatory system to remove barriers to innovation and provide greater predictability in the search for answers to the nation’s unmet medical needs. It is not too late to register.

Q: Let’s talk about the impetus for the National Health Council. What was the driving need behind its formation?
A: The National Health Council was formed in 1920, so we have a long history. We were formed by a group of about 20 patient organizations that was looking for a trade association.


Q: Were there any other large trade associations of its kind in existence?
A: No, and there currently is nothing out there. We still serve in that function, so half our work is really directed at member services. Our work includes anything from board management, governance, corporate structures, volunteers, income development and benchmarking studies. We still maintain what is called the standards of excellence. The standards are a set of nonprofit standards, essentially, that the patient groups have to meet in order to be in membership. They are actually the most strident nonprofit standards in the industry.


Q: How is your organization funded?
A: We are a dues-based organization so about half our income comes from dues and the other half comes from sponsorships.


Q: Can you give me an example of some of the leading patient advocacy groups that make up your membership?
A: Some of the original founders include American Cancer Society, American Heart, American Diabetes and we run the gamut from those very large groups such as Easter Seals to small groups that represent various disorders like Sjögren’s disease or Alpha-1.


Q: What unique function does your group serve?
A: We are able, through our process, to engage the community, create a common understanding and then a common platform from which to advocate on systemic issues. And when they come together, they tend to have a great deal of impact.


Q: What are some of those messages you are working to convey through this organized effort?
A: In terms of public policy, we really are focused on two different things. One would be access to care, so we’ve been heavily involved with the implementation of health reform in a variety of different initiatives right down to the level of helping craft part of the regulatory language, as well as the legislative language, that created the Affordable Care Act. So, for example, we worked on defining the essential health benefits the Secretary of Health and Human Services is working on. We did our own actuarial analysis of what it would mean to put certain benefits into the design. We did recommendations on regulatory language.


On the other hand, we are extremely interested in development of new treatments, and so there is the dual focus.


Q: Tell me a little about your latest effort, the Moddern Cures Solution, which stands for Modernizing Our Drug and Diagnostics Evaluation and Regulatory Network?
A: We are promoting legislative language that will do two things. It will, one, allow a company to bring a medicine to market without a patent, and that is significant. A lot of people don’t understand, but when somebody identifies a new potential medicine in the early pre-clinical phases, it typically will go to a company that could develop and commercialize that product and bring it to U.S. Food and Drug Administration approval. In 80 percent to 90 percent of those cases, new potential treatments are dismissed not because the science is not good, but because they have weak patents.


It just makes no sense from a patient perspective, and this legislation corrects that. It says if you’ve got good science, you can bring it to market, receive a return on your investment, and still allow a generic to enter at a predetermined time.


Q: Does this proposed change include medical devices?
A: That is the second half of the legislation. There are mechanisms that address specific issues such as creating the incentives to invest in the device/diagnostic arena.


Q: Is this modeled after any other country’s system?
A: No, and actually these initiatives were identified by the chief medical officers of the patient groups. In other words, they came to the council about five years ago and said, “We’ve doubled the investment in the development of new treatments, and we are not getting double the output of new treatments.” And they start to look at the lifecycles of the development of treatments and diagnostics and identify barriers.


And then asked us to look at possible solutions and we settled on these four major issues and developed solutions that we think could work, but are not actually modeled on existing solutions. We are, however, already seeing Europe and other countries look at this as a potential metric that they would copy.


Q: What has the reception been like so far from the policy side?
A: Extremely positive. It has been described by some as one of the few major health policies that has bipartisan support and the current Congress and administration could actually enact. It is exciting.


Q: What kind of dialogue do you hope to get out of the 2011 CHI Annual Meeting?
A: I am hoping, given the audience that will be at that meeting, that there will be a great deal of synergy on what we are doing on the development of new treatments and how it will positively impact the environment that your audience is working and living in.

I figured it is an incredible event, and I was really pleased to be invited to participate.

CHI-Advancing California biomedical research and innovation

Event Spotlight: CHI Co-Hosts Congressional Briefing on Infection Prevention

On Oct. 12, CHI and MassBio co-hosted a congressional briefing in Washington D.C. titled, “Infection Control: Detection, Prevention, and Treatment.” Participants were joined by Reps. Jackie Speier (D-CA) and Edward Markey (D-MA). The partnership was hailed as a powerful combination of two of the most significant state powers in the biotech industry. As a father of a child suffering from cystic fibrosis, MassBio President and CEO Bob Coughlin said passionately, “I don’t care if [cystic fibrosis] is cured in California or Massachusetts. All boats rise.”

Coughlin and CHI President and CEO, David L. Gollaher, Ph.D, made opening remarks and introduced Reps. Speier and Markey, who each made their own comments. Next, a panel of experts provided educational presentations on challenges and advances in combating healthcare-associated infections (HAIs). The program, held in the Gold Room of the Rayburn House Office Building, attracted more than 50 congressional staffers, patient advocates, and industry representatives.

Speakers outlined the scope and effect of HAIs. Estimates show that HAIs take the lives of 100,000 patients a year – patients who enter healthcare facilities in good health, or without such infections yet die from exposure in healthcare settings. As Speier noted, “That’s the equivalent of 333 [Boeing] 747 airplanes crashing every year.” This makes HAIs the 10th leading cause of death, and the No. 1 cause of death in non-cardiac intensive care units. In the U.S. alone, antibiotic-resistant infections are responsible for $20 billion in excess healthcare costs, $35 billion in societal costs and $8 million in additional hospital days.

Despite these numbers, HAIs remain a dangerously under-recognized threat. Continuing her airplane metaphor, Speier said, “If that many planes were crashing every year, we’d expect the FAA to do something about it.” Dr. Arnold Huang, Ph.D., of Thermo Fisher Scientific, later polled the audience to demonstrate the high degree of recognition of October as breast cancer awareness month. He juxtaposed those numbers with the audience’s lack of knowledge about sepsis – a condition that causes four to five times as many deaths each year.

Despite the high death rate and the continued appearance of new strains of antibiotic-resistant bacterium (such as MRSA), the number of new antibiotics in the development pipeline has been shrinking during the past several decades. Rick Winningham, CEO of Theravance, described the trends that have led to diminished approvals of new antibiotics. Overall, “antibiotic drug development is dying,” he said. He attributed this to market dynamics which have led large pharmaceutical firms to exit the market entirely, leaving small firms to overcome tremendous obstacles. These dynamics include: decreases in expected returns on investment; rising costs of clinical trials and the number of required patients; and regulatory challenges and delays from the FDA. Winningham laid responsibility for these on both Congress – for having taken the FDA to task over past high-profile safety issues – and industry itself, for problems in effectively pricing previous products.

Speakers ended the day by noting the promising introduction of the Generating Antibiotic Incentives Now Act (GAIN), and giving a call to action. The GAIN Act would give developers of select antibiotic products five or more years of additional data exclusivity and provide for priority review of their products by the FDA among other provisions. Despite this positive development, participants like Kathy Warye of Becton Dickinson stressed a fundamental challenge to inspiring advocacy to address HAIs: Because they strike and kill patients relatively quickly and are not long-term chronic diseases, it’s impossible to put a face on the problem in the way that advocates for other diseases and conditions have succeeded in doing. Further, the public fails to relate deaths from these sorts of HAIs to our lack of effective antibiotics. As Winningham said, “Leadership has to drive this change.”

CHI-Advancing California biomedical research and innovation

Guest Blog: Fish & Richardson Attorneys Explore European Patent System

 

Steven Carlson

Germany is a top country for patent enforcement. Knowing the German system is fundamental for technology companies that sell products in Europe, both for patentees and for companies pulled into German courts as defendants.

Alexander Harguth

In Germany, patent cases frequently take less than a year, at a fraction of the cost of U.S. cases.


A tremendous advantage to patentees is that infringement cases are tried separately from challenges to the validity of a patent. Likewise, many of the procedural “outs” that defendants enjoy in the United States (such as motions to dismiss, early claim construction rulings, and summary judgment proceedings) are unavailable in Germany — accordingly, the first time the parties appear in court is typically for the trial on the merits. Defendants, therefore, must be ready to act quickly to develop their case, to prepare their validity challenge, and to pursue other available defenses.

Customs actions provide another way to enforce patents. Customs authorities may seize goods upon the patentee’s request, after which the case may be referred to the trial courts. Therefore, potential infringers must be aware, for example, when presenting goods at a trade show, there could be a seizure action carried out on the floor of the exhibition.

German damages awards are typically lower than in the United States. Nonetheless, there may be nowhere better for winning an injunction if the patentee has strong patent rights. Although infringement relief will be limited to Germany’s borders, being enjoined from selling in Europe’s biggest market may be intolerable for many companies. A German enforcement strategy, or a parallel strategy of enforcement in Germany and the United States, may, therefore, result in a global settlement of disputes.

We have written a new book on the German IP system: “Patents in Germany and Europe: Procurement, Enforcement and Defense.” As native attorneys of Germany and the United States, we have tried to present Germany’s system to the U.S. audience in an accessible and authoritative handbook, including chapters on:

 

• Sources of law in Germany;

• The German court system;

• Procurement of IP rights in the German and European patent offices;

• Invalidity challenges through oppositions and nullity actions;

• Enforcement actions through infringement trials and customs proceedings; and

• Employee rights in inventions.

Moreover, key provisions of German and European law are provided in the appendices, as well as sample pleadings, ending with a list of key distinctions between U.S. and German/European law.

The book is available on Amazon.com. If your company has particular interest in the German system, we will be happy to provide a complimentary copy along with an in-person seminar where feasible. Please feel free to us contact directly, at Harguth@fr.com or Carlson@fr.com.

CHI-Advancing California biomedical research and innovation

Executive Spotlight: Mitchell Kronenberg, President and Chief Scientific Officer, La Jolla Institute for Allergy & Immunology

Kronenberg is an internationally recognized scientist and one of the most highly cited immunologists in the world. He was appointed president of La Jolla Institute in September 2003. In addition to his executive duties, he serves as chief scientific officer and conducts an active research program. He received his doctorate from the California Institute of Technology in 1983, and stayed on to complete his postdoctoral work before joining the faculty of the UCLA School of Medicine in 1986. At UCLA, he rose through the ranks to full professor. In 1997, he came to La Jolla Institute to head the division of developmental immunology, a position he held for 14 years, before stepping down recently to devote more time to his duties as president and chief scientific officer.

Over the years, Kronenberg has received many major awards, most recently a prestigious Merit Award for scientific achievement from the National Institutes of Health. He has also been a Burroughs Wellcome Fund Visiting Professor at Harvard University and recently was asked to deliver the Joseph S. Ingraham immunology lecture at the Indiana University School of Medicine.


Q: Tell me a little about the institute and its most significant milestones. How did it first get started?
A: The La Jolla Institute for Allergy and Immunology was established in 1988 by a coalition of leaders from academia and industry who envisioned a unique and dynamic partnership that would lead to breakthroughs in the understanding of the immune system and improvements in human health. We began our laboratory operations in 1989 with the arrival of two pioneering immunologists: Kimishige and Teruko Ishizaka. They were M.D./Ph.D.s from Johns Hopkins University renowned for their seminal discovery about the molecular origins of allergies. They started us down a path of scientific excellence that has included major discoveries on a number of immune-mediated diseases over the years. Among these are autoimmune diseases, like type 1 diabetes and Crohn’s disease, and cancer and heart disease, where the role of the immune system and inflammation is becoming increasingly appreciated. Of course, the immune system is highly protective, and we also have important findings related to vaccine development and infectious diseases such as H1N1 influenza.

Our significant progress is evidenced by the fact that we were recently ranked by Thomson Reuters among the top five organizations worldwide for research impact in immunology. In addition, five potential therapies, based on our institute’s discoveries, are currently in the pharmaceutical pipeline.


Q: Why was it important to dedicate an institute to exploring the body’s defense system?
A: The immune system goes to every part in your body to defend your body and we know you can’t live without your immune system. If you’re highly immunocompromised, you die rapidly from infection. And we also know that vaccination is probably the most effective public health measure we have ever found. So, immunology is very important in doing good in our bodies, and, yet, it also can do bad by causing inflammation leading to autoimmune diseases. There are about 83 known autoimmune diseases. There are probably other inflammatory conditions not classified as autoimmune, in which the immune system plays a role, including atherosclerosis. So, the immune systems does great things to keep us alive every day, and is a great target in terms of vaccinations for keeping us healthy, in other words, giving us more immunity.

However, sometimes the immune system is irrationally exuberant, if I can quote Alan Greenspan. It could begin attacking the pancreas, causing type 1 diabetes or early onset diabetes. It could be attacking the nerves and you get multiple sclerosis. It could be contributing to arthritis. I already mentioned atherosclerosis and many, many more diseases.

We think the immune system, aside from its inherent intellectual fascination, has the greatest implications for health and disease of people. If you’re going to form an institute and dedicate it to one area of biomedical research, it seems to me that immunology would be a very good one because of its wide implications for human health and disease.


Q: We are in a unique position in San Diego as one of the clusters of biomedical research, including early-stage academic research. Talk about some of your industry/academic collaborations on important science.
A: We have a very interesting, long-term partnership with a Japanese pharmaceutical corporation called Kyowa Hakko Kirin. Our partnership actually started with Kirin many years ago, a name that may sound familiar to you because they also make Kirin beer. The current organization was formed by the merger of Kyowa Hakko and Kirin Pharma in 2008, and it is a moderately large pharmaceutical company in Japan. They also have 45 people who work in the building here. Our census is about 350 for our institute. It’s like having an endowment in the sense that every lab, every year gets money to buy equipment and engage in projects of their choosing. In return, and within certain limitations, the company gets first rights to negotiate for our intellectual property and they also enjoy the ease of collaboration by co-locating with us. There are two projects that originated from this institute that are in clinical development with Kyowa Hakko Kirin and others that are in preclinical research.


Q: How else is the institute funded?
A: We’re dependent on NIH for about 75 percent of our annual revenues and every year we’ve had increases in our NIH funding. No. 2 is our contract research agreement with Kyowa Hakko Kirin, which gives us about 13 percent of our annual budget. Other sources include philanthropy contributions and also grants from other agencies such as the Juvenile Diabetes Research Foundation, Crohn’s & Colitis Foundation of America, American Cancer Society and other private agencies that support biomedical research, as well as our licensing income.


Q: Talk about your new Center for RNAi Research, and what you hope to accomplish.
A: We received a large NIH grant in 2010, in a very competitive process, to set up a center for RNA interference (RNAi) screening. We opened the center in August. RNAi is a young and very powerful technology that we will use to try and understand what each gene in our genome does. The center will incorporate robotics and high-throughput screening methods and will have particular expertise in the use of real-life experimental models of disease.

The grant pays for four projects that relate to the immune system, but we don’t want the facility to be confined only to immune system work. We intend to explore genetic questions that will be important in disease processes of all kinds. In addition to our own research, we want it to be open to the academic groups on the Torrey Pines Mesa and to the universities. There will be a fee-for-service arrangement. We hope to have all kinds of projects. We even hope to expand the size and scope of this facility.


Q: When you aren’t spending your time at work, what are you doing?
A: I like to spend time with my family. My wife is also a busy scientist here and we have three sons, two of whom are grown and attending universities and one who lives with us still. I lift weights one day a week and run a couple days a week and I read compulsively. I was recently reading a book called “The Women” by T.C. Boyle and it’s actually a biography of Frank Lloyd Wright. I have been reading another book that’s called “Let the Great World Spin” by Colum McCann and it won the Man Booker Prize. It’s a wonderful novel about intersecting lives, Irish immigrants, people in the ghetto in New York City. It is a fantastic story.






CHI-Advancing California biomedical research and innovation

Executive Spotlight: Daniel Laser, CEO and President, Wave 80 Biosciences

Daniel Laser

Wave 80 Biosciences Inc. develops next-generation molecular diagnostics for infectious disease, cancer, autoimmune disease and other human health conditions. The company began operations in 2003, with a small team of engineers, biologists, and chemists working out of warehouse space in San Francisco’s Dogpatch neighborhood to develop high-performance, cost-effective technologies for processing complex fluid samples.

In 2007, the company initiated a development program to address emerging needs for viral load monitoring to accompany expanding access to antiretroviral therapy among the estimated 30 million people living with HIV in low- and middle-income countries. The first versions of the EOSCAPE-HIV system are scheduled to enter beta testing in clinics in Kenya and South Africa in 2012.

President and CEO Daniel Laser spoke with the CHI Blog to explain how Wave 80, a name born of an anagram, has contributed to improving tests that treat for both infectious and noninfectious diseases, including hepatitis C, hepatitis B, cardiovascular disease, autoimmune disease and cancer.

Q: How did the company get a name like Wave 80 Biosciences?

A: My wife and I came up with it sitting at the Top of the Mark in 2003 while applying for the first grant application that got the company started. It is actually an anagram that includes the name of the chemist whose discovery of electro-osmosis led to one of our seminal patented technologies, an architect whose work inspired certain structural aspects of our product’s design, and the word “silicon,” which is the basic element of our key technology components. My wife is an English major so she probably deserves the credit.


Q: How did you decide to start this company, and what were you looking to accomplish at the time?

A: The company has had a couple of major phases of its development. The current phase started in 2007 when we began focusing on HIV.

In the earlier phase of the company, we were more of a research and development shop doing specialized work for the U.S. military. There were some challenges that the military felt were on the horizon in the wake of 9/11, relating to new chemical and biological weapons and battlefield medicine more generally. I had become aware of those military R&D interests through involvement with Professor William Perry at Stanford, who had previously served as the U.S. Secretary of Defense. Through my work with Perry’s group, the Center for International Security and Cooperation, I saw an overlap with some of the work I had been involved with as a graduate student. My graduate work was focused on the semiconductor industry, but I saw the opportunity to port the technology into healthcare and became intrigued.

It was not clear that it could solve the issues that the U.S. military was confronting, but it just seemed like it might, and it was pretty clear that the only way that work was going to get done would be if I made it happen. There was no one else who was likely to both be aware of the technology and also be aware of the military-specific needs.

Q: So what brought Wave 80 into HIV?

A: We were doing foundational work with the U.S. military regarding how to move proteins around inside miniaturized assay cartridges, initially unrelated to HIV. However, we established a link with the head of research and development at a group called the International AIDS Vaccine Initiative (IAVI). The head of R&D at IAVI, Wayne Koff, found out about Wave 80’s work for the military and was acutely aware of unmet needs in HIV in the developing world. Wayne had the foresight to see how the military technology could be paired with a specific assay method and that it might be fruitful, which is exactly what how it turned out.

There is now a fairly extensive and well-developed infrastructure for HIV patient care and clinical research in sub-Saharan Africa as well as Southeast Asia, the Caribbean and Latin America. I think maybe more remarkable, at least to me, is the extent to which this relatively new HIV patient care infrastructure is serving as the basis for improving treatment of both infectious and noninfectious diseases such as tuberculosis, hepatitis, cardiac disease, diabetes and many others.

A positive outcome of the great tragedy of the HIV epidemic is this significant improvement in the healthcare infrastructure of countries around the world. Wave 80 is building HIV and other products to increase these clinics’ capabilities in caring for the local populations.

Q: Talk about your partnership with the San Francisco Public Health Laboratory and San Francisco General Hospital to pilot the quicker, point-of-care test.

A: One of the challenges that comes with HIV is identifying individuals who are HIV-positive as early in the infection cycle as possible. Standard HIV diagnostics that one sees in an HIV clinic or in a primary care setting are based on what is called a serologic response. This is when HIV antibodies appear in the patients’ blood, but these antibody-based diagnostics only turn positive several weeks after infection.
Something that we are at the forefront of now, along with UCSF, SF General and the SF Public Health Lab, is identifying HIV-infected patients during what is called the acute phase before the antibodies appear. It turns out that transmission risk is highest in the acute phase, so, if you can diagnose HIV in its first few weeks, then the prospects for reducing transmission go up greatly.

I think we can all look forward to, if not the end of the epidemic, a significant decrease in the number of new infections as these universal “test and treat” measures take hold.

Q: What do you see as some of your biggest goals in the next few years?

A: We have one overarching goal which is to move the products fully into the clinical setting. We are entering the validation process right now. If it were a U.S. product, this would essentially mean preparing the FDA submission. We are bringing this product through the FDA, but for the global market it is not really desirable to hold up the product while waiting for U.S. regulatory approval.

Instead, what we do is we work through a very rigorous set of validation procedures. The U.S. Centers for Disease Control and Prevention (CDC) will oversee the bulk of early validation. The World Health Organization and the Ministries of Health of the countries into which the machines are going to be placed will also be involved. We gradually work through approvals and demonstrations of our diagnostic specs and capabilities in the settings where our products will be deployed.

Alongside that, we have the goal of using Wave 80’s HIV diagnostic platform in HIV vaccine clinical trials. This is actually a major driver of the funding package that we have from the U.S. government, in order to help differentiate between someone who is HIV positive versus a vaccine trial participant who is HIV negative, but whose blood may carry antibodies from exposure to a potential vaccine.







CHI-Advancing California biomedical research and innovation

Speaker Spotlight: Naser Partovi, Founder and CEO, Sanitas Inc.

Naser Partovi

Partovi has over 25 years of management, corporate development and operating experience. Last year, he established Sanitas Inc., a mobile health technology company that helps patients with chronic conditions manage their treatment. This year, Sanitas is testing its Wellaho product in heart failure patients.

Prior to starting Sanitas, Partovi was president and CEO of SKY MobileMedia, a leading provider of multimedia applications framework for cellular handsets and new generation of connected multi-media devices. Before that, Partovi was a managing director at Enterprise Partners Venture Capital based in San Diego, where he managed a portfolio of companies in the communications industry, including Ascendant Systems, DragonWave, Inc., Quorum Systems and ReliOn, Inc. Before joining Enterprise, Naser served as vice president of strategy and business development for Nortel's optical networks business.
Partovi graduated from Canada’s McGill University with a master’s degree in electrical engineering.

Q: Tell me a little about your background.
A: I am an engineer by training. I graduated from McGill University. Then, I was working at a company called Nortel, a telecommunication company. I was the vice president of business development and strategy at Nortel when I left in 2000. I was recruited from there and joined Enterprise Partners, which was the largest venture capital firm in Southern California. I was a general partner there until 2006, when I left to run one of the companies I had invested in based in San Diego, SKY MobileMedia. We sold SKY in 2008, and, then last year, I started this new company, Sanitas.

Q: Describe, simply, the product that Sanitas is offering.
A: Wellaho is a product to help patients with chronic conditions to manage their treatment. The way we differentiate ourselves from what is out there on the market is what we call the three pillars of our treatment plan. One is education. One is monitoring of progress. And one is getting support from your friends and family and your doctors. There are other companies who are providing bits and pieces of these services. To the best of our knowledge, Wellaho is the only one that’s providing all the three services combined as a continuity of care system.

Q: Monitoring of progress … is that where mobile health technology comes in?
A: Yes. When you or your loved one is diagnosed with a chronic condition, you can do a Google search and you will hit 1 million sites and you need to figure out what is reliable information and what is not. We look up the patients’ own health record. We find out exactly what their diagnosis is, and what their treatment plan is, and we dynamically build a tailored education plan for the patient. Either your primary care doctor, or, in the case of heart failure, your cardiologist, will decide the signs and symptoms they want you to monitor. They set up the parameters.

For example, if I have heart failure and I’m pretty healthy otherwise, they might say, “Okay, your resting shortness of breath should be at this level, and, if you have walked a mile, it should be at this level.” Patients fill in information about themselves using a cell phone or they can do it on their iPad or iPhone or Android application. We bring up the information and say, “Okay, what is your level of shortness of breath?” or “How far did you walk?” “These are your medications. Which one did you take today?”

We tailor monitoring to each patient’s needs based on what their cardiologist wants to know. And the cardiologist can see this information immediately or they can see it the next time they log in, which could be a week. We’re not a real-time monitoring system.

Q: Besides heart health, does this application apply to other conditions, such as cancer?
A: The first module we introduced and the one that we are conducting the field trials on is heart failure. We will announce other modules as they become available. The reason we are introducing different condition modules in a sequence is because we go very deep inside each condition. For example, in heart failure we have over 350 modules. A lot of other applications targeting the conditions go an inch deep, miles wide. We are at the other end of the spectrum. We are going an inch wide and miles deep because we believe that for each condition you need very detailed information on how to treat, what diagnostics options are available and so on and so forth. We have modules scheduled for the next three years to come.

Q: You have a unique perspective as a mobile tech investor. What can you tell me is most influencing investments in the mobile healthcare space?
A: In mobile health, there are a lot of really good companies coming up with a lot of products. My biggest worry about this is who is going to pay for it. The reimbursement mechanism is not out there and insurance companies are not paying and the government is not paying for it. There are a lot of issues around liabilities because, if this is real-time monitoring and the doctor misses that information, what happens? So, I think there are a lot of elements of this mobile health area that need to be figured out.

These things take a lot of time in the healthcare. You need to have a lot of clinical trials, prove the efficacy of the system, go back to the insurance companies, and go back to CMS to convince them that this is improving outcomes. We have tried to take the low-tech route to helping patients because, for example, with heart failure, if we can even improve 1 percent of readmissions to the hospitals by a combination of education, monitoring and support, then that is lots of money saved by hospitals and insurance companies.

Q: What are you looking forward to hearing about at the CHI mobile health event “Take this Pill and Tweet Me in the Morning?”
A: What I would like to learn from other speakers as well as the audience is their experience in dealing with patients, patient adaption, how often patients actually stick with it. How do we convince the insurance companies and the government that this is as important as medication and surgery? So, this is a big cultural change that needs to happen.

CHI-Advancing California biomedical research and innovation

Guest Column: Patenting Genes and the Future of Personalized Medicine

By Ashwin Mudaliar, Stanford University

Patents pertaining to the use and appropriation of biological information have been in the news prominently over the last year. The majority of attention has been paid to the case of Association for Molecular Pathology v. U.S. Patent and Trademark Office, which challenges the legality of Myriad Genetics' patent of the breast cancer genes BRCA1 and BRCA2, and more broadly, the patentability of human genes in the United States. However, Prometheus Laboratories Inc. v. Mayo Collaborative Services, which considers the patentability of medical methods, may have just as broad an impact over the greater landscape of biotechnology intellectual property.

The Patent Act of 1952 specifies four categories upon which an invention can be judged patentable: proper subject matter, novelty, non-obviousness and utility. It further specifies that a patentable invention be any “process, machine, manufacture, composition of matter, or improvement thereof.” If a prospective invention does not meet all four of these categories, it is not considered germane in view of the law and is thus not eligible for patent protections, as determined by the United States Patent and Trademark Office (USPTO). Laws of nature, physical phenomena, and abstract ideas are explicitly barred from patentability on the basis of not being proper subject matter.

Method patents, which are patents on processes of performing a set of steps (methods) to obtain certain results, have traditionally been awarded as long as a substantive transformation through the method was demonstrated and if the method in question was tied to a specific apparatus. However, the scope of method patents was brought into question in the case of Bilski v. Kappos (In re Bilski), which stems directly from USPTO’s rejection of Bernard Bilski and Rand Warsaw's patent application for a method of hedging commodities risk, whose claims fell under the unofficial category of business method claims. The examiner at the USPTO rejected the claims of the application because the method was not tied to a specific apparatus and that it "merely manipulate[d]" abstract concepts to solve a mathematic question without a substantive transformation. In further appeals through the legal system, the United States Court of Appeals for the Federal Circuit, which is tasked with handling all patent related matters, affirmed the initial rejections of the Bilski patent and went further by significantly modifying the standards for resolving whether any invention is statutory subject matter under the Patent Act through its promulgation of a new test, termed the machine-or-transformation test, to determine patent eligibility for any process claim. This test aims to determine if a claimed process or method is tied to a specific apparatus and/or if the same claim transforms a specific article into a different state. If either stipulation is met, then the claim is deemed statutory assuming it meets the other categories of patentability (novelty, usefulness, etc.). The plaintiffs appealed to the Supreme Court to reconsider this decision, which obliged by granting certiorari to hear the case.

In Prometheus v. Mayo, Prometheus Laboratories, a diagnostics and therapeutics company from San Diego, Calif., sued Mayo Medical Laboratories for patent infringement on two methods for determining the optimal drug therapy for a gastrointestinal autoimmune disease when Mayo Medical announced that it would sell its own diagnostic test based on similar methods. The original patent specified a method for measuring the metabolites 6-methyl-mercaptopurine (6-MMP) and 6-thioguanine (6-TG) following the administration of a thiopurine drug for autoimmune diseases. Based on the level of both, drug dosage would be altered for maximal efficacy and minimal toxicity. In March of 2008, a district court in California invalidated two patents held by Prometheus on the grounds that the methods were not patentable subject matter. However, in September of 2009, the United States Court of Appeals for the Federal Circuit overturned this ruling and claimed that the methods described in the patents satisfied the machine-or-transformation test, and more specifically that the administration and determination steps in the original claim brought about sufficient transformation. The lawyers for Mayo Medical argue that observed correlations between blood test results and health are simply “basic, natural biological relationships,” and thus non-patentable, but the Federal Court ruled that any kind of treatment that alters the body in a therapeutic manner could be patentable.

The court argued that the claims in the plaintiff’s patent did not simply cover natural correlations (metabolite levels) or data-gathering (measurement) because the, “asserted claims are in effect claims to methods of treatment, which are always transformative when a defined group of drugs is administered to the body to ameliorate the effects of an undesired condition (emphasis added).” The court also ruled that measurement that goes beyond “mere inspection” is transformative. Mayo Medical Laboratories quickly filed an appeal to the Supreme Court.

On June 28, 2010, the Supreme Court issued its ruling on the In re Bilski appeal, which affirmed the Federal Circuit’s ruling that Bilski process was not patentable subject matter. However, the Court also ruled that the machine-or-transformation test was not the exclusive test to evaluate method patents but rather just a “useful and important tool” amongst others. It did not offer further or more specific guidance. The Supreme Court then vacated the initial federal circuit court decision in Prometheus and ordered that this court reconsider its decision in light of the new ruling on Bilski. In its reconsideration of Prometheus on Dec. 17, 2010, the federal circuit court reconfirmed that method claimed by Prometheus constituted patentable subject matter and stated that the Bilski decision “did not undermine [its] preemption analysis of Prometheus’s claims.” As such, its ruling was substantively unchanged. Mayo Medical appealed again to the Supreme Court on May 17, and was granted a writ of certiorari on June 20 for consideration. A definitive ruling is expected next summer.

The issues at stake in Prometheus case are very important to the future of commercial diagnostic tests and, more broadly, the commercialization of personalized medicine. Very often, diagnostic methods similar to those described in the Prometheus patents are used to deliver safer and more effective treatments to patients who might otherwise react adversely to a therapeutic intervention. If the Supreme Court were to side with the defendants and invalidate the Prometheus patents in a broad decision, it could potentially call into the question the validity of a number of previously issued diagnostic patents that companies rely on to protect their investments. Considering that many of these sorts of diagnostic tests are created by smaller biotech companies, invalidation of the “method of medical treatment” system could hinder innovation and prevent early-stage biotechs from attracting investments. This is especially critical as we enter an age of increasingly personalized medicine, where therapeutic regimes will be closely tailored to the specific physiological states of individual patients. Without adequate protection for the research and development of such diagnostic methods, we could see a marked reduction in the number of private companies and research institutions that engage in the type of research that leads to these interventions as well as the quality of that research. Given these considerations, the Supreme Court’s ruling next year will have an enormous impact on the future of life sciences and medical technology.

CHI-Advancing California biomedical research and innovation

Event Spotlight: CHI Co-Hosts Congressional Briefing on Alzheimer’s Disease

On July 14, CHI and the Healthcare Institute of New Jersey (HINJ) co-hosted a congressional briefing in Washington, D.C. focused on Alzheimer’s disease. Congresswoman Linda Sanchez of California delivered particularly poignant remarks. Sanchez’s father suffers from Alzheimer’s disease, giving her and her siblings, including fellow Congresswoman Loretta Sanchez, personal experience in caring for a parent suffering from this mind-robbing disease. Despite this challenge, Linda Sanchez considers herself fortunate to have the support of her six siblings, asking, “I wonder what it would have been like to be an only child?”

CHI President and CEO David L. Gollaher, Ph.D., and Dean Paranicas, who serves as president and CEO of HINJ, delivered opening remarks, followed by presentations on cutting-edge research and scientific advances achieved by CHI and HINJ members. The event wrapped up with closing remarks from Reps. Chris Smith (R-NJ) and Linda Sanchez (D-CA). The program, held in the Capitol Visitor Center, attracted more than 60 congressional staffers, patient advocates and industry representatives.

The speakers on this day made clear that the experience of the Sanchez family is sadly common, and growing at a rate that will be both emotionally and economically devastating. As Gollaher noted, “The Alzheimer’s crisis is of great import because of its personal and economic effects” which will have “great implications for this country and the rest of the world as populations get older.” To illustrate the scope of the crisis, he noted that 5.4 million people are living with Alzheimer’s disease, and someone develops the disease every 69 seconds. Further, in 2010, family members and friends provided 17 billion hours of unpaid care to those with Alzheimer’s and other dementias — care valued at approximately $202 billion. The effect of the disease in California alone is particularly acute with more than 500,000 Californians suffering from the disease today and estimates that the number will double to 1 million by 2040.

Yet, participants and presenters also made clear that there is hope. California leads the nation in National Institutes of Health funding, making Alzheimer’s disease one of the largest concentrations in the California R&D pipeline, Gollaher noted. A panel of top researchers then offered their unique approaches to treating and potentially curing Alzheimer’s disease. Dr. Stuart Lipton, director of the Del E. Webb Neuroscience, Aging and Stem Cell Research Center at Sanford-Burnham Medical Research Institute, described his ongoing work with NitroMemantine– a combination of an existing Alzheimer’s therapy called memantine and nitroglycerin. As both drugs are already approved for other indications, the compound’s potential speed to market is dramatically shorter than that of a new drug.

Joseph Hammang, Ph.D., the senior director of worldwide science policy for Pfizer, noted that improved diagnostic tools and personalized approaches to treating the disease that rely on a person’s unique genetic make-up are potentially critical advances in treating Alzheimer’s, which actually includes a number of distinct diseases. He said Pfizer has committed $100 million over five years on collaborations with public institutions, demonstrating its continued commitment to work with academic partners in the U.S. and around the world.

Kimberly Scearce-Levie, Ph.D., head of in vivo neurobiology for Genentech, detailed her team’s recent advances in methods to block a protein that composes the plaque thought to be responsible for cognitive impairment in AD sufferers. The Genentech team has developed a way to hijack the body’s own mechanism for passing iron from blood into the brain, essentially tricking the body into transporting a critical blocking agent through its own defenses..

Bob Nelson, Ph.D., research fellow at Lundbeck Research USA, continued the conversation by saying he believes that the story is likely to be more complicated. Lundbeck Research has begun to challenge conventional wisdom about the disease by asking slightly different questions. Are the identified plaque compounds associated with AD strictly bad substances? Might blocking them also have harmful effects? Might the abundance of plaque-causing proteins be caused by victim’s inability to remove or reabsorb the protein rather than due to a problem of overproduction?

Lastly, Wayne Poon, Ph.D., director of the UCI MIND Brain Bank and Tissue Repository, described his approach to solving a basic problem in Alzheimer’s disease diagnosis. The disease, he said, is only diagnosable when a patient has died and his or her brain can be examined. Because of this, up to 15 percent to 20 percent of patients thought to have AD are later found to have been misdiagnosed.

In closing, Rep. Smith of New Jersey, chairman of the Alzheimer’s Disease Caucus for 11 years, thanked the panel for its efforts. The U.S. and the world are facing a figurative pandemic, he said, which is on the verge of a breakout with the arrival of a “demographic winter.”

“We have a crisis and funding is flat-lined,” he said. “We simply aren’t going to have the people to take care of these [AD sufferers].”

Rep. Sanchez concluded, “I wish I was here to celebrate a cure…Yet, with the talent and work of many, we will be able to celebrate that day in the future.”

CHI-Advancing California biomedical research and innovation

Speaker Spotlight: Anthony Costello, COO Mytrus

Anthony Costello

Mytrus is testing the boundaries of traditional clinical trial research, with the first all-electronic, home-based clinical trial to gain U.S. Food and Drug Administration approval. As co-founder and chief operating officer of San Francisco-based Mytrus, Costello is leading the charge. Costello is a leader in the areas of technology innovation and clinical operations. After beginning his research career at Genentech, Costello went on to co-found several technology start-up companies including Nextrials, a clinical research firm where he worked for 10 years as the vice president of product development. He is a frequent presenter on topics related to the efficient use of technology and clinical research.

Q: What brought you into the clinical research industry?

A: I got into the clinical research industry in the ’90s when I started working at Genentech. At the time, I was in data management and spent pretty much my whole career at Genentech in data management. I ended up overseeing the data management group for their oncology center and working mostly on the Herceptin trials and the early trials for what is now Avastin.

Back in those days, everybody was doing clinical research on paper. We did all of the international Phase 3 trials for Herceptin on three-part no-carbon-required paper, mailing it around the world and then data entering everything. Although I loved Genentech and it has certainly launched my career, I wanted to develop a technology that was faster and more effective. I left Genentech in 1999 to co-found a company that makes electronic data capture systems for clinical trials, called Nextrials.

Q: What happened next?

A: I left Nextrials in 2008, after almost 10 years there, and I met Dr. Steve Cummings (Mytrus CEO and chairman) and got excited by his idea of building more of a consumer-focused clinical trial that would allow patients to participate from home. I joined an advisory board that Steve was putting together to sort of vet out this idea, and we ended up deciding to form Mytrus around that. I joined as a co-founder and as the vice president of product development and commercialization and I now serve as chief operating officer.

Q: Mytrus is hoping to validate a new kind of study model. If this kind of remote results monitoring works, what might the future look like for pharmaceutical companies and their trials?

A: We hope that it’s going to make a big difference. Our hypothesis that is staring to play out is that there are some kinds of trials – we estimate about 15 percent to 20 percent – conducted in the United States annually that can be done using this method. They do not require specialized equipment at a site. They do not require specialized training, and, if a patient is willing and able to correspond via the web and provide their own updates about what is happening to them during the study, then those trials are suitable to use this method. It really changes once you free your mind, as Steve likes to say, from the idea that you have to have a bricks-and-mortar site for trials.

You do not have to have as much monitoring because the patients are the direct source for data and you do not have data being transcribed from the patient to a chart and then from a chart to an electronic data capture system and then from an EDC system to an alpha system. All those data transcription events are the reason monitoring was effective — to make sure nothing got missed or skipped or inadvertently entered. So, you could get rid of a lot of that. You don’t have to go through the contracting and start-up and hierarchy approval with hundreds of clinics around the country.

Q: What are the technology requirements for participation?

A: If you do not have an Internet connection, you cannot do it. In fact, depending on the trial, there are some studies where it is really going to be critical to have an Internet connection in the home. There are other studies where it would be perfectly suitable to have a smart phone or an iPad or even a shared Internet connection in some sort of public place if the patient is comfortable using that and it is private enough that someone can enter their own data. Patients are much more apt these days to use the Internet for their own medical research and they are much more comfortable communicating with doctors over email or over the Internet.

Q: What are you looking forward to hearing about at the CHI mobile health event “Take this Pill and Tweet Me in the Morning?”

A: There is just a lot more focus on health 2.0 technologies these days and so we’re excited about being involved. It seems like the lineup of speakers are people on that leading edge of moving trials or studies or health in a new direction so it is a perfect kind of conference for us.


Hear more from Anthony Costello at “Take This Pill and Tweet Me in the Morning,” happening Aug. 4 at the Salk Institute in San Diego.



CHI-Advancing California biomedical research and innovation