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Alzheimer’s Association Outlines Research Priorities & Treatment Horizons

This week, Real World Health Care brings you a conversation with Maria Carrillo, PhD, Alzheimer’s Association Chief Scientific Officer. Formed in 1980, the Alzheimer’s Association advances research to end Alzheimer’s and dementia while enhancing care for those living with the disease.

Real World Health Care: According to your literature, more than 5 million Americans suffer from Alzheimer’s disease. What do the majority of these patients most need in terms of treatments: treatments to address the symptoms of the disease, or treatments to stop or slow the progression of the disease? Why?

Maria Carrillo, Alzheimer's Association

Maria Carrillo, Alzheimer’s Association

Maria Carillo: What people need most is hope – hope that comes from significant progress in Alzheimer’s research that they can understand and that benefits their day to day life. Thanks to the amazing work of thousands of Alzheimer’s Association advocates, and many dedicated legislators, we have in place a U.S. National Plan to Address Alzheimer’s Disease. The Plan calls for real progress in treating and preventing Alzheimer’s disease by 2025. An expert workgroup convened by the Alzheimer’s Association recommended a federal investment of $2 billion per year for 10 years to make this achievement a real possibility. We are making strides toward that level of commitment – we’re over half way there – but we still have a way to go.

To more directly answer your question, both symptomatic and preventive therapies have a lot of value. People with Alzheimer’s, and those at high risk of getting it in the near-term, need something to help them now. Greatly reducing or eliminating dementia symptoms and/or pushing back the onset of dementia by ten, or even five, years, will act as something like prevention for the great majority of people – they will live most if not all of their lives free from debilitating symptoms.

Finally, we all look forward to the development of proven ways to reduce risk and prevent Alzheimer’s disease and other dementias – for the millions of lives saved, the billions of dollars saved, and the enormous suffering eliminated.

RWHC: What are some of the most promising areas of treatment research that the Alzheimer’s Association is funding?

MC: The Alzheimer’s Association is the largest private, nonprofit funder of Alzheimer’s research, awarding more than $350 million to more than 2,300 projects. Our goals are to (1) advance our understanding of Alzheimer’s, (2) help identify new treatments, (3) improve care, and (4) further our knowledge of brain health and Alzheimer’s prevention.

The Alzheimer’s Association has provided essential startup funding to, and/or funding to expand the scope of, three major Alzheimer’s disease prevention trials currently underway – the A4 Trial, DIAN TU, and API APOE4.

The Alzheimer’s Association grants program has funded – and continues to fund – some of the most important research threads in Alzheimer’s science. These topics and ideas move the field forward by contributing to knowledge about Alzheimer’s, refining research questions, and yielding clues to causes and treatments. Examples include:

  • Sex, gender and vulnerability to Alzheimer’s and other dementias
  • Commonalities between cancer and Alzheimer’s
  • Effects of oxidative stress and inflammation in Alzheimer’s
  • Vascular contributions to Alzheimer’s
  • Protein misfolding and Alzheimer’s
  • Tau toxicity in Alzheimer’s
  • Biomarkers for Alzheimer’s
  • Development of new scales for Alzheimer’s, including pain and clinical meaningfulness
  • Vaccines/Immunotherapies for Alzheimer’s
  • Insulin and insulin-degrading enzyme in Alzheimer’s
  • Down syndrome and Alzheimer’s
  • Patient ability to consent in Alzheimer’s
  • Differences in Alzheimer’s in minority communities
  • Blood pressure control and Alzheimer’s

RWHC: What are some of the biggest challenges or obstacles that Alzheimer’s researchers are facing in terms of developing new treatments? What can or should be done to overcome those challenges or obstacles, and how is the Alzheimer’s Association helping to overcome them?

MC: The two biggest issues/obstacles are insufficient funding for Alzheimer’s research and the need for more participants for clinical studies.

The Alzheimer’s Association and its nationwide constituency is the leading advocate for increased research funding at the federal level, and also the leading nonprofit funder of Alzheimer’s research. That said, many more voices need to be heard – every day and through every vehicle – to ensure that the federal government understands exactly how high a priority this issue is, and allocates the needed funds.

Recruiting and retaining clinical trial participants is now the greatest obstacle, other than funding, to developing new and better treatments for Alzheimer’s disease. Before any drug or therapy can be used in medical practice, it must be rigorously tested to certify that it is safe and effective.

To address the growing need for clinical trial participants, the Alzheimer’s Association launched Alzheimer’s Association TrialMatch – a free clinical studies matching service. More than 100,000 individuals have registered to search for Alzheimer’s clinical trials using TrialMatch.

Alzheimer’s Association TrialMatch provides users with comprehensive clinical trial information and a customized list of studies they may seek to enroll in. TrialMatch is open to everyone, including people with Alzheimer’s or other dementias, their caregivers, family members, and anyone who wants to get involved in the fight against Alzheimer’s.

There are several other registries also working to identify and marshal individuals for Alzheimer’s disease research. The primary need is for increased awareness that these services exist, and that participating in Alzheimer’s research is one of the greatest gifts that one can give to current and future generations.

RWHC: Is a preventative or curative treatment on the horizon? Do you think this is a disease that can ultimately be prevented before it starts or cured after it starts?

MC: Similar to the way we address heart disease, the preferred strategy for Alzheimer’s would include:

  • Early detection methods that identify those most at risk. Preferably these would be simple, inexpensive and non-invasive, such as a blood test.
  • Effective methods to track the biological course of the disease, and the impact of drugs and other interventions.
  • Preventive and risk reduction strategies – lifestyle and pharmacological – that can be engaged in by everyone throughout life.
  • Multiple effective treatments – that address the disease in multiple ways – for those who, despite the points above, suffer from dementia symptoms.

The Alzheimer’s Association believes that all these things are possible in the relative near-term with the appropriate investment in research.

We have made great strides in treating and preventing many diseases – even major killers such as cancer, heart disease, and HIV/AIDS – when we have made the issue a high priority and made the resources available for research. Now is the time to do the same for Alzheimer’s disease.

RWHC: What sort of initiatives or activities can we expect to see from the Alzheimer’s Association over the next few years?

MC: We still do not completely understand the causes of Alzheimer’s disease, and many questions remain about exactly how it progresses. The Alzheimer’s Association plans to redouble its efforts to support and spotlight in the basic science of the disease, for example at the Alzheimer’s Association International Conference. Increasing our knowledge in these areas should uncover multiple opportunities for therapeutic targets.

At the same time, the Association is deeply involved in clinical trials aiming to prevent Alzheimer’s disease. In addition to the funding initiatives mentioned earlier, we collaborate with Fidelity Biosciences Research Initiative (FBRI) and the principal investigators of all the major prevention studies in the Collaboration for Alzheimer’s Prevention (CAP) seeking to facilitate the sharing of information, resources and expertise that may speed the discovery of new preventive treatments. CAP was first convened in 2011 to help researchers learn from and support each other’s work; share data; harmonize data gathering and trial outcomes to allow for comparability across studies; and hold open, informal dialogue with regulators.

Inflammation is a factor that contributes to the cause and progression of many diseases and conditions. This is emerging as a significant area now in Alzheimer’s research. The Alzheimer’s Association’s Part the Cloud initiative, for example, is making the development of new therapies in this area a target of multi-million dollars in research funding.

Newly launched is Alzheimer’s Combination Therapy Opportunities (ACTO), which aims to provide pilot funding to explore combination therapy opportunities in Alzheimer’s disease. This program will support a biomarker-based clinical trial testing of repurposed drug combinations through Phase II proof of concept.

The Alzheimer’s Association also continues to prioritize issues such as the impact of sex/gender, race/ethnicity, education, and socioeconomic status on the development and progression of Alzheimer’s and other dementias.

 

Collaboration among Patient Advocacy Groups, Scientists & Clinicians Moves Porphyria Research Ahead

Editors Note: Recognizing National Porphyria Awareness Week this week, we invited Desiree Lyon, the Executive Director of the American Porphyria Foundation (APF), to share how her organization is supporting research that improves treatment and ultimately seeks a cure for this rare disease.  Patient advocacy groups addressing rare diseases play a particularly important role in fostering research into their disease states.  Because rare diseases affect small numbers of patients, finding enough people for research studies can be difficult. The number of experts studying the illness is small. The largest sources of medical research support government and industry put far more money into studying widespread diseases because of the potential to help so many patients live longer and enjoy a higher quality of life.  APF is an example of a rare disease community that is working in many ways to overcome these challenges and provide hope for patients and families.

Desiree Lyon, American Porphyria Foundation

Desiree Lyon, American Porphyria Foundation

The American Porphyria Foundation (APF) has been instrumental in facilitating porphyria research for over thirty years. Porphyria is a group of at least eight disorders in which normal body chemicals accumulate abnormally, with symptoms that arise mostly from effects on either the nervous system or the skin. Porphyria affect fewer than 200,000 people in the United States, making it a rare disease.

As with other rare diseases, only a few doctors and medical centers have expertise on porphyria, making collaboration among patients, clinicians, researchers, patient advocacy groups and caregivers even more important than with other diseases.

The collaborative relationship with our Scientific Advisory Board of porphyria experts is vital to the success of our research projects. This relationship became even more productive when the APF and the experts formed the Porphyria Research Consortium, which was funded by the NIH Rare Disease Clinical Network.

Together, the APF and our Porphyria Research Consortium formed the National Porphyria Research Registry. The APF prepares patients who may be considering participation in a clinical trial by educating them on the value and process for trials, introducing them to the investigators, and encouraging them to join the Registry. We reduce patients’ fears and provide reassurance about the Registry, clinical trials, the expertise of the researchers and the merit of the research by publishing articles on the APF website, in newsletters, internet E-news and social media, including Facebook, Twitter, and blogs.

Another unique and successful collaboration to locate and register research participants occurs at APF patient education meetings held around the country. Porphyria experts volunteer for these gatherings to make presentations, answer questions, register patients for their research and often collect DNA samples.

The Porphyria Research Consortium and the APF are collaborating on eight clinical trials, including one on a powerful treatment for the acute porphyrias cleared by the FDA thirty years ago as the first Orphan Drug. Since the APF is aware of most people who are using this treatment, we have been instrumental in locating participants for the clinical trials and advancing the research.

Also important to our research efforts are the young physicians, who participate in the Protect the Future (PTF) program, an APF supported mentor project to train the next generation of porphyria experts and scientists so they are well versed in both the clinical and basic science of the porphyrias. PTF physicians serve as clinicians at APF-funded “satellite clinics” where patients are given the opportunity of visiting a physician with porphyria expertise, while the PTF physicians gain a new pool of research patients.

The HealthWell Foundation is proud to partner with APF to promote the availability of copay assistance for patients receiving treatment for porphyrias.  If you or someone you know could benefit from financial assistance related to porphyrias treatment, please contact the HealthWell Foundation to learn more and determine eligibility.

Foundations like the APF are critical to enhancing awareness of rare diseases. Are you involved with a rare disease Foundation like the APF? If yes, let us know what you’re doing to raise awareness in your disease community by leaving a comment below.

 

Research America Wants YOU to Support Public and Private Sector Investment in Research!

We’ll be live blogging today from the National Health Research Forum: Straight Talk about the Future of Medical and Health Research at Newseum.  Sponsored by Celgene, Johnson & Johnson, Onyx Pharmaceuticals, TEVA, Genentech, University of Maryland School of Medicine, and HudsonAlpha Institute for Biotechnology, we’re pleased to bring you updates from inside the room!

{Note from Editor: our updates below paraphrase speaker comments so that we can share the points being made in real time without the benefit of a recording to verify or use a transcript}

The first panel is titled, “Where will medical research be in 2023?”

We’re underway with our first panel.  Dr Anthony Fauci, Director, National Institute of Allergy and Infectious Diseases, is told that he does a good job of advocating for his division. Has the pendulum shifted away from investment in research?

Dr. Fauci: When you have even a modest increase in a budget, you can preserve the fundamental research activities. But the problem is the budget has been flat for 10 years and inflation takes a bigger toll. As a nation we have to make a commitment that fundamental research should not be part of a discretionary pool.  So any cutting of a budget hits the discretionary pool.  Some people get it and some don’t when we tell this story that basic research is what it is we’ve done for you.  Five years from now we’re not going to be able to tell anyone if we continue to stay flat and not even account for inflation.

John Seffrin, PhD, CEO American Cancer Society: Research America has doubled the NIH budget in a period of five years.  But we haven’t made the case to the public to explain the need for long term investment.  The tsunami of non-communicable diseases coming down the road – we need to convince the public of the need to invest.

France Cordova, PhD, Director, National Science Foundation: Industry gets the need to invest in basic research and advocacy for it, but it seems to be number 10 on the priority list.

Dr. Fauci: I can assure you that no one will stop me and say, with regard to the ebola outbreak, why didn’t we invest more in basic research years ago?  They’ll say: why don’t you have a vaccine now?

Dr. Cordova: We need to make science more accessible, to what I like to call “K to gray.”  With this science is accessible to all ages.

Panel 2: Code Red Again: Can We End the Assault on Public Health Research and Practice

Tom Frieden, MD, MPH, Director, Centers for Disease Control and Prevention: We save $3 for every $1 invested in vaccines, in the health sector alone, and $10 overall…..

Only for this event would I take an hour off from ebola.  The ebola epidemic is worse than is recognized. Reported cases are a small fraction of the total.  Despite maximum efforts – 100 field officers – the largest response in CDC history.  If we had  invested in systems that would find, respond, prevent we could have prevented this disaster…..

Public health is a “best buy.” It keeps us safe and prevents disaster. Every dollar investment in public health pays off.  Ebola, drug resistance – ongoing threats – it will take a funding commitment and partnerships. We at CDC have a partnership with CMS that never existed before.  Public health is the governments responsibility but the government can’t do it alone…..

Handwashing in hospital is not up to par, neither is blood pressure maintenance. We have room to grow in health quality.  Hospital acquired infections are too common…..

The average person can have a big impact on the government. One person is able to change policy. One person made government improve and keep open TB clinics in NY….

With regard to health disparities, high blood pressure and heart attacks is the single largest cause of differences among the races.  The second big issue is teen pregnancy.  These are two areas where intervening makes a huge difference.

Georges Benjamin, MD, Exec Director, American Public Health Association.  The public thinks we have a better protection system than we do….

Seeing your doctor 2-4 times a year is a system where patients think they are staying healthy, but many times this is not effective.  We need to create a better system to keep patients healthy.  Prevention Research should be funded more…..

Saying that you support research, and actually voting to increase or keep funding for research is different.  We need to hold policy makers accountable. Meet with local or state elected officials, with out an agenda, so when you do have an agenda they are more willing to help….

Lynn Goldman, MD, MS, MPH, Dean, Milken Institute School of Public Health, George Washington University:  In the US we feel comfortable that we have systems in place, however it would be possible for a slightly different pathogen to arise that we could not protect ourselves from. We’re doing very little about antibiotic resistant pathogens. We’re not going to be able to control something like that…..

The pay off for the American tax payer is tremendous for increasing vaccinations…..

Richard Kronick, PhD, Director, Agency for Healthcare Research and Quality, HHS: Is there a perception problem? Yes. The public can see that price and accessibility are not perfect, but most people see safety and quality as a non-issue, when it is, especially in less developed countries…..

Where are where failing? What can your organization do better? and in what time frame? This should have been done yesterday.  We need to not only produce evidence, but make sure that this evidence is used.  We have funded work to show how low-income children are overly prescribed anti-psychotics, but we are not able to implement any rules or policies to change this. ….

Jack Watters, MD, VP, External Medical Affairs, Pfizer: The happiest news is that we are all living longer – a cause for celebration. One of the best thing that’s happening is that healthy younger people are living to be healthier older people…..

I see far more appetite for public/private partnerships, in research, in delivering public health. We should recognize that we are all in this together and we must partner more – I welcome the increased appetite……

Seeing a doctor a couple of times per year is better than not at all, but we need to increase contact between all health professionals and patients (nurses and pharmacists)….

We are seeing an improvement in the private sector.  There is a shift in the appreciation to public health by the pharmaceutical industry. Some money is being used for research, but it is harder to convince policy makers for more.  Public health problems are not as “sexy” as proactive research in medicine for uncured/treated conditions…..

There are simple things we can do to increase public health, for example with depression, just asking a person could make a significant difference.

Panel 3: What’s Right – and Wrong – with the Research Ecosystem?

Moderator Margot Sanger-Katz, Health Care Correspondent, The New York Times:

Pablo Cagnoni, MD, President, Onyx Pharmaceuticals: What do you think of the US Research system? We are not moving as quickly as necessary, we could be moving more quickly. From lab to market, the timing is too long.  That being said, we still have the best ecosystem in the world.

The competitive system keeps priorities in the wrong place.

We have to extend Dr Woodcock’s good work to CBER and get companion diagnostic testing approved rapidly.

There are 2 areas where “big data” is playing out. One is in research, more importantly another is in utilization.  Are we utilizing the right drugs with the right patients?

Kathy Giusti, MBA, Founder and CEO, Multiple Myeloma Research Foundation (was unable to make it to the event)

Robert Hugin, Chairman, CEO, Celgene: What do you think of the US Research system? I think that it is a fact that we have the best research system in the world.  Yes we still need to improve, but we always need to. We have a very competitive system, that makes for a great spirit in the science community, and we are always improving. Transational medicine (bringing research to the patients) does not get the visibility, but it is important and appreciated.

We need to provide more economic incentives to collaborate, to avoid redundancies between different research centers. More transparency would help this problem.

Ways to improve- Prevention research by asking for congressional help, more investments.

At $1.5 billion per drug who can sustain this? Something has to change. It’s not sustainable. We must review the system.

With regard to the increasing costs of drugs: I think we look at this in an inappropriate way, the only way we can capture costs is through price. If someone could create a drug that can cure a cancer (and avoid downstream costs), we have to remember that.  The overall impact is very positive. We do a better job than Europe with access.  We should never be embarrassed to talk about it.  Taking cancer for example. In 1970, the cost was 1 percent of spending for oncology drug spending, now it’s at .5 percent.  Remember, the generics didn’t discover those drugs – they wouldn’t have them without our research. Exchange programs discriminate against the working poor – at Fred Hutch, Memorial Sloan Kettering, MD Anderson, people are kept out of those excellent care facilities….CBO says when Rx costs rise, other healthcare costs go down.  We want to talk about Lindsay Lohan and rehab instead of better access to care.  We have this discussion backwards. (Applause)

We are finally able to bring technology to the research because of the high costs. Ten years from now, our lives will be fundamentally different, because of (advances)…

E. Albert Reece, MD, PhD, MBA, Dean, University of Maryland, School of Medicine: What do you think of the US Research system? Our system is very rigorous, and it works very well, but it is not perfect.  Looking at other countries, our research infrastructure is more rigorous, and that is our strength. Our weakness is that we are not sustainable.

Boom or Busts in research, training young people takes a long time, so there is a constant roller coaster in the amount of researchers that we have. The amount of researchers that we needs is constantly changing also.

I agree with Bob, there have to be improvements and legislation to increase efficiencies.

Janet Woodcock, MD, Director, Center for Drug Evaluation and Research, FDA: What do you think of the US Research system? We don’t have the infrastructure necessary to carry the information, that we find through our research, to the American public, and to implement new findings.

Is money being spent in the right way on the right research?  A problem that we have is that a big amount of research is not able to produce the same findings more than once. We need to be more intentional with research. We are trying to change the way that clinical evaluation is done. It is too expensive and not sustainable. We should not have a brand new clinical trial for every single experimental drug, we need to come up with a system that can be reused, and is therefore more sustainable.

How does the regulatory system change the research ecosystem? It creates a lot of challenges, we’re seeing a shift in pharmaceutical research to drugs that depend on genome, more precise medicine. Because this is so new, there are a lot of uncertainties.

Unless we change the cost and drug development process, then we will not keep improving.  We don’t have the right science to actually make the right drugs, only 2/10 drugs even make it to trials. Efficiency needs to change, and this can change through changing the drug manufacturing process.

Our healthcare system is poorly designed when it comes to non-drug interventions. The final translation into practice – we’re trying to look at more patient centered measures to look for ways to benefit through proven, unconventional benefits.

 

Categories: General

KIDS: Providing Children and Families a Voice in Medicine, Research, and Innovation

The active involvement of patients in health care choices, diseases, research, and innovation is an area of recent focus for many public and private entities (e.g., FDA’s Patient-Focused Drug Development initiative).  As an innovative method to engage children, the KIDS (Kids and Families Impacting Disease Through Science) project was launched as an advisory group of children, adolescents, and families focused on understanding, communicating about, and improving medicine, research, and innovation for children. KIDS is a unique collaboration between the American Academy of Pediatrics (AAP) Section on Advances in Therapeutics and Technology (SOATT), local AAP Chapters, children’s hospitals, local schools, and other partners.

The objectives for the KIDS project are as follows:

  • Learn, teach, and advocate for medicine, research, and innovation that improves the health and well-being of children;
  • Engage in the process through projects and consultation activities with hospitals, researchers, and other partners in the public and private sectors;
  • Provide input on research ideas, innovative solutions, unmet pediatric needs, and priorities;
  • Contribute to the design and implementation of clinical studies for children (e.g., assent, monitoring tools, schedules, etc.);
  • Serve as a critical voice for children and families in the medical, research, and innovation processes.

KIDS launched as a pilot program in Connecticut in September 2013 and will be expanding to other states in the US (e.g., Utah, New Jersey). The KIDS Connecticut Team has participated in meetings at Connecticut Children’s Medical Center (Hartford, CT) and Yale-New Haven Children’s Hospital, a Research Summit at Pfizer’s Connecticut Laboratories, and an advisory session with Mr. David Tabatsky, author of Write for Life.

In addition, the team attended the AAP’s Healthy Children Conference & Expo in Chicago in March, at which they staffed an exhibit booth highlighting their work and the importance of research and innovation for children. They also conducted survey-based research by collecting more than 300 responses with a focus on participants’ opinions of the importance of research in their lives and the role of children in research. Three KIDS Team Members delivered an invited Learning Zone presentation for conference attendees discussing the importance of research, the work of the KIDS Team, and the vision for future expansion. Finally, the KIDS interacted with numerous AAP leaders and staff members throughout the weekend. Overall, the KIDS involvement in the conference was a resounding success as each Team Member was articulate and passionate about their work and the importance of medicine and research. Feedback from attendees, exhibitors, and AAP leadership/staff was overwhelmingly positive. The Team will also be attending the Pediatric Academic Societies meeting in Vancouver in early May and will be collaborating with a similar children’s advisory group located in that city.

In addition to a KIDS expansion in the US, SOATT is working with existing young person advisory groups and other partners to develop an international network of children advisors. The children, families, leaders, and partners are very excited about the potential opportunities for these teams and the future network to make a significant impact on the health and well-being of children worldwide.

Do you think it is important for children to be involved in shaping pediatric medical research? Have you had experience with kids getting involved in their health care? What was the outcome? Share your thoughts in the comments section.

If you are interested in participating in this project or would like more information, please contact Charlie Thompson (charles.a.thompson@pfizer.com).