A new idea on how to involve patients into the goal-oriented research projects

1. Commentaries
REJUVENATION RESEARCH
Volume 16, Number 5, 2013
ª Mary Ann Liebert, Inc.
DOI: 10.1089/rej.2013.1471
From Personalized Medicine to Personalized Science:
Uniting Science and Medicine for Patient-Driven,
Goal-Oriented Research
Alex Zhavoronkov1–4 and Charles R. Cantor 3,5–7
Abstract
We developed a new model for initiating, coordinating, funding, and managing biomedical research projects.
The concept involves engaging the patients with chronic conditions with no known cures into goal-oriented
research activities. In this model, the patient seeks the help of a research organization to bring together a
multidisciplinary team of research scientists and physicians to initiate research projects using the patient’s grant
funding, samples, as well as the management expertise. This model may be of interest to other research institutions because it has many benefits, including new sources of private research funding, when government
funding is getting scarce, motivating scientists and physicians to work closely together on goal- and patientoriented research projects, and using patients’ management skills.
Introduction
T
he concept of personalized medical science is not
new. Egyptian Pharaohs, Islamic Caliphs, and European
monarchs supported research and encouraged medical
teams to focus on their own personal medical problems,
which incidentally contributed to many areas of science.
Many wealthy, influential individuals actively pursued
medical science to solve their own medical problems and
assembled interdisciplinary teams.1 In the modern world,
this practice of finding effective healthy solutions for a particular individual has evolved and separated into concierge
medicine, often referred to as ‘‘boutique medicine,’’2–4 where
medical doctors receive a generous retainer for their services
and contract research services5 and where a contract research
organization receives a task-based order for an individual.
The practice of concierge medicine is not yet very prevalent
and may not be in the best interest of the general public6
because its main goal is to provide faster and better-quality
medical service to the select few who are willing to pay for it.
Concierge medicine allows a wealthy patient access top
clinical talent to select the best diagnostic and therapeutic
options known to be potentially applicable to his case. An
example is the off-label use of cancer therapies based on gene
expression profiling and the particular somatic mutations
found in a tumor biopsy or even patient plasma.
What we propose here is a significant extension of this
process where a team of researchers is recruited to aid clinicians by using both experimental and bioinformatics tools
in an attempt to discover a novel disease management
strategy. Currently research and clinical practice in privately
funded health care are largely separate. This may be impeding progress and the adoption of cutting-edge methods
into mainstream clinical use. Many top-rated medical and
academic institutions are using the concepts of concierge
medicine to subsidize other areas7 without involving patients directly into the research projects. Many private individuals with medical problems donate to research or medical
organizations, without engaging in research activities.
Here we propose a new model in which the patient becomes directly involved with the research project to find new
solutions for unmet medical needs. In addition to paying for
the research and providing clinical samples, the patient helps
to coordinate the research activities. This may lead to improvements in the way the research is integrated directly into
clinical practice.
1
The Biogerontology Research Foundation, Reading, United Kingdom.
Center for Pediatric Hematology, Oncology and Immunology, Moscow, Russian Federation.
Moscow Institute of Physics and Technology, Moscow, Russian Federation.
4
First Open Institute for Regenerative Medicine for Young Scientists, Moscow, Russian Federation.
5
Department of Biomedical Engineering, Boston University, Boston, Massachusetts.
6
Department of Physiology and Biophysics, University of California Irvine, Irvine, California.
7
Sequenom, Inc., San Diego, California.
2
3
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2. NEW MODEL FOR FUNDING AND MANAGING RESEARCH PROJECTS
Involving Patients in Personalized Science to Fund
Research Projects
Many high-net-worth individuals with excellent management skills donate billions of dollars to research via
foundations and fellowships, without being directly involved with the research projects. These foundations are
typically set up to address a broad cause and usually do not
have a core goal-oriented project. A team of experts is hired
to supervise research activities and provide grants to scientists who are usually already funded by the government,
academia, or industry. Some high-net-worth individuals,
besides donating to basic research, would also like to solve
personal medical problems and steer some of the research
effort. These individuals often have excellent project management and organizational skills, but lack the scientific
background and confidence to start their own research
projects. In contrast, many young scientists and medical
doctors would like to apply their skills to conduct cuttingedge research and work with patients, but lack management expertise. Personalized science projects could bridge
this gap and link patients with management expertise with
teams of scientists and physicians who are interested in
pursuing goal-oriented science, the patient provides research grants, samples, and management expertise. The
team of physicians and research scientists executes research
projects that address the patient’s future needs and research
interests.
415
To test this personalized science concept, we organized a
group of young scientists and physicians with over 500
active members and conducted a series of 30 weekly lectures on regenerative medicine. In parallel, we used social
media to create multidisciplinary teams of biophysicists,
biologists, geneticists, and physicians to work on 25 research projects with topics spanning from metastatic mineralization of the connective tissue to bioinformatics
approaches to personalized medicine in oncology. Using
this pool of scientists and physicians, it became possible to
launch research-personalized science projects to address
both patient research interests and needs. Whenever a highnet-worth individual who has medical problems or would
like to prevent possible future medical problems becomes
interested in pursuing a research project, the project is announced to the community to form a core team that is
funded and co-managed by that individual. These small,
goal-oriented teams dedicated to solving specific patient
problems may be more effective than large foundations or
industries (Fig. 1).
Process
The high-net-worth individual creates a description of a
medical problem to be addressed and proposes the research
budget and a set of milestones (Fig. 2). The research organization then describes and announces the project to the
community of scientists and physicians that can submit
FIG. 1. Power to the patient. Patients provide grant funding to a research organization. The organization provides funding
and research infrastructure to young researchers. The research team then interacts with the patient and leading experts in the
field to provide care.

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ZHAVORONKOV AND CANTOR
FIG. 2. Process description. Teams of patients, MDs, and PhDs work together to determine the best course of action for
personalized medical care.
applications to join the research team. This creates a situation
in which candidates are not writing proposals based on extensions of the research they have already done, but instead
they address, a health question that has already been elaborated. The research organization, together with the initiator
of the project, can then select a qualified team from a pool of
interested candidates. The patient helps the effort by coordinating the project, setting targets, providing samples, and
providing the funding. The physicians perform a comprehensive clinical review of the medical problem, engaging top
experts in the field whenever possible. The physicians decide
what factors are likely to be relevant to the target problem
and form collaborations with the scientists. The scientists
perform various bioinformatics analyses of the factors and
target problem, focusing on the most promising of the therapeutic approaches. The physicians focus on performing
patient-specific studies, such as various ‘‘omics’’ analyses,
imaging, and any other additional tests as indicated by the
medical problem. Together, the physicians and scientists
develop a working treatment hypothesis. At this point, the
physicians and scientists design a series of experiments to
test the hypothesis. Finally, the team provides the managing
patient with a research report and review of the current information. They list current and possible new treatment
options for the patient. The team can also propose further
investigation to extend the project and possibly lead it to
commercialization.
Examples
A 40-year-old patient suffering from endometriosis was
the owner of a successful international manufacturing and
marketing business and decided to support research in
endometriosis. She contacted a large research and clinical
center running a voluntary organization in regenerative
medicine for young scientists. With guidance from the supervisors of the practicum, she interviewed young physicians, scientists, and students and provided research
fellowships to those interested in endometriosis. She also
decided that if the research project showed promise, she
would fund the project on a large scale. She selected physicians (ObGyn/embryology) and scientists (bioinformatics/systems biology) to work with her on the project.
The physicians consulted several leading experts in the field
internationally using the patient’s diagnostic data, performed a variety of tests, and prepared a report on the
factors involved in endometriosis and patient-specific diagnostic data. Together with the scientists and the patient,
the team developed screening and diagnostic routines that
went far beyond traditional practice to monitor hormonal
levels and screen for potential biomarkers in blood, saliva,
stool, and urine. Also, as suggested by the patient, the team
collected samples of endometrial cells, performed transcriptome analysis and signaling pathway analysis, identified perspective drug targets, and shortlisted a number of

4. NEW MODEL FOR FUNDING AND MANAGING RESEARCH PROJECTS
potential candidate drugs and therapies. In the meantime,
the patient stabilized her condition under the supervision
of the leading experts in the region, who became interested
in the science component of the project.
Another example involved a high-net-worth individual
interested in aging research, who briefly described the condition of one of his colleagues, which involved accelerated
mineralization of connective tissue. On the basis of this
suggestion, six young physicians and scientists formed a
multidisciplinary team to study the condition. The team
worked for 6 months using the research organization’s
infrastructure to perform a detailed analysis of the factors
involved in mineralization, literature review, and bioinformatics analysis and proposed a series of experiments to
confirm their hypotheses. The patient did not fully engage in
the project, but it resulted result in closer collaboration between the scientists and medical doctors working together to
address the patient-specific problem.
Benefits of Personalized Science
From the patient’s perspective, there are many benefits of
personalized science. Patients can achieve a better understanding of their medical problem. Due to their direct involvement with the funding aspects and the research team,
they have the ability to direct research, set objectives, and
apply management talents. Their own samples and information can be used for research purposes, creating the ultimate in personalized medicine. Receiving informed consent
from the patient becomes exponentially easier, because they
have a better comprehension of the medical issues and the
treatment plan. While their personalized care is being developed by researchers, the physicians can use their networks to put their patient in contact with leading experts in
the field, so that the patient can receive better treatment in
the future. Finally, the patient has the potential prospect of
turning the research project into a business and generating
more revenue.
Researchers benefit from this new funding paradigm in a
number of ways. The process creates new avenues for
funding young scientists. These scientists will spend less
time on grant writing and reporting and more time on the
actual research problem. By using goal-oriented research
with a specific patient in mind, it may be easier to determine
the best course of action and experimentation. Teams with
physicians and scientists working together will pool the assets from both fields. The scientists have the ability to contact
authorities in the field of study and possibly publish a review
or a case report at the end. Finally, researchers may be able to
commercialize the research findings with an educated seed
investor.
The public will benefit from personalized science, because
patients will assume the cost of fundamental research. This
process also creates a new bottom-up approach to solving
global medical problems. The public will have faster access
to cutting-edge medicine and science. Finally, it may be able
to reduce health care costs by identifying optimal targetoriented therapies aimed to cure specific medical issues.
Legal and Ethical Issues of Personalized Science
Although there are clearly many benefits to personalized science, it raises some serious legal and ethical issues.
417
The concierge medicine concept is increasingly drawing
ethical criticism,8 because today’s research scientists rarely
engage in behavior classified as misconduct that involves
direct contact and working directly with the patients.9
Direct patient involvement may prompt unnecessary and
unregistered screening and diagnostic procedures, misuse
of patient records, and self-experimentation, especially
considering cases of off-label use of registered drugs. A
patient making tax-deductible donations to non-profit organizations performing research using patient’s material
may be deriving personal benefits that are not legal from
the taxation standpoint. Unavoidably, wealthier patients
engaged in personalized science may get an unfair advantage compared to regular patients. A wealthy patient
could also conceivably become disillusioned by the results
or have unacceptably high expectations. However, all of
these issues can be addressed and potentially resolved on
a case-by-case basis. For example, the patient may decide
that benefiting from personalized science outweighs a tax
break for donating funds and forgo receiving the tax
break. If wealthier patients allow other patients to come
into the study at a reduced cost, less wealthy patients may
benefit as well. The public benefits from this new paradigm may help compensate for these potential legal and
ethical issues.
Summary
The new paradigm of personalized science yields funding, patient samples, data, and skills for goal-oriented research in the current resource-constrained environment.
Young researchers working together in small teams with
expert management from the patient may find new ways to
treat patients. Funding personalized science will create a
new wave of opportunities for researchers and provide
high-net-worth patients with the ability to engage in solving their personal medical problems while subsidizing and
directly contributing to research activities. These opportunities are excellent starters for new business ventures and
treatment options for the public at large. Overall, personalized science is a fascinating new possibility for science.
Author Disclosure Statement
No competing financial interests exist.
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ZHAVORONKOV AND CANTOR
Address correspondence to:
Alex Zhavoronkov
The Biogerontology Research Foundation
4 Hill Street
London, W1J 5NE
United Kingdom
E-mail: alex@biogerontology.org