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Prize4Life
recently attended the 8th Annual Neurotech Investing & Partnering Conference, Advances in Drugs, Devices & Diagnostics for the Brain and Nervous System hosted by the Neurotechnology Industry Organization (NIO) and NeuroInsights
held May 23-24, 2013 in San Francisco, California. While many of the
sessions explored the latest innovations in devices and treatments for nervous
system disorders, the “Movement Disorders: Parkinson’s, ALS, and more session”
was one of the most exciting sessions, covering some of the most novel
approaches to treating neurodegenerative diseases.
The chair of the session, Steve Mickel, Director of
Commercial Development at AbbVie,
opened the session with a provocative question; “Can devices pick up where
pharmaceuticals leave off?” The landscape is evolving. No longer are we focused
on finding one small molecule drug for one disease. Instead, academia,
biotechs, and pharma are turning their efforts towards the development of
devices and biologics as therapeutic opportunities. Each of the talks in this
session appropriately highlighted this shift in thinking, focusing on the use of
proteins, peptides, phage, and even devices for the treatment of movement
disorders including ALS and Parkinson’s disease.
Dr. RJ Tesi, CEO of FPRT Biosciences, led the session describing his company’s clinical stage
protein-based therapy, XPro1595, for the treatment of neurodegenerative
diseases including Alzheimer’s diseases, Parkinson’s disease, multiple
sclerosis, ALS, and Huntington’s disease. XPro1595 is a blood-brain penetrant
anti-inflammatory protein that promotes neuronal survival by treating a single
pathology common across many neurodegenerative diseases, excessive
neuroinflammation. How does the protein work? XPro1595 is a 17kD “kissing
cousin” of soluble TNFα (XPro1595 has six amino acid substitutions as compared
to the wild-type protein). The minor changes in sequence gives XPro1595 a
therapeutic advantage that in Tesi’s words is “unmatched”; XPro1595 has the
ability to selectively bind and neutralize soluble TNFα, while not affecting
transmembrane TNFα. XPro1595 sequesters soluble TNFα blocking downstream
receptor-ligand interactions that would normally activate an inflammatory
response. FPRT Biosciences just closed their Series A financing round and are
looking forward to beginning Phase Ia and Phase Ib trials in ALS, which will go
through 2015.
In the second talk of the session Jonathan Solomon, CEO
of NeuroPhage Pharmaceuticals, spoke about NeuroPhage’s exciting developments using
their General Amyloid Interaction Motif (GAIM) technology, which can broadly
target misfolded proteins species from oligomers to mature fibrils in protein
misfolding diseases such as Parkinson’s disease, Alzheimer’s disease,
Huntington’s disease, and ALS. More and more evidence suggests that
neurodegenerative diseases share a common underlying pathological mechanism
involving the propagation of toxic protein species across the brain via a
prion-like mechanism. NeuroPhage’s GAIM technology works to block this propagation via a multipronged
approach: 1. targeting multiple pre-existing protein pathologies, including
oligomers, and fibers, 2. preventing and blocking the assembly of misfolded
proteins, and 3. decreasing the cytotoxicity of misfolded proteins.
NeuroPhage has extensive biochemical and cell-based data
to support their GAIM technology, which appears able to robustly target
multiple pathogenic protein species in a number of neurodegenerative diseases.
Furthermore, NeuroPhage has shown that their approach translates into cognitive
behavioral improvements in animal models. NeuroPhage’s original NP002 technology
was based on the ability of the filamentous bacteriophage M13 to reduce
ß-amyloid in mice. NeuroPhage is currently developing second generation
Ig fusion-based product candidates with potentially superior pathogenic
protein-targeting properties based on the active motif in NP002.
In the third talk, Winston Ko, CEO of Genervon Biopharmaceuticals,
presented data on their “master regulator” therapeutic, GM6, a peptide
derived from the naturally occurring protein involved in embryonic development,
motoneuronotrophic factor (MNTF). According to Genervon, GM6 has all the
advantages of a peptide-based drug: very high potency and activity, low
toxicity, minimum drug-to-drug interaction, low accumulation in tissues (i.e.,
a short half-life), extremely high specificity, and little to no unspecific
binding to molecular structures other than the desired targets. GM6 is small
enough that it does not have the disadvantages of large peptides in terms of
stability, solubility, mutagenicity, immunogenicity, or the high cost of
manufacturing through transgenic or recombinant methods.
According to Ko’s presentation, GM6 works through 12
interactive pathways, including axonal transport, RNA metabolism, neuronal
plasticity, and protein aggregation. Additionally, GM6 targets 22 biological
processes, including neurogenesis, neuronal development, and neuronal
signaling. GM6 targets over 80 ALS-related genes, suggesting that GM6 could be
an effective therapy in ALS. It looks like we will soon know the answer, as Ko
was excited to announce during his presentation that Genervon has recently
received FDA approval for testing GM6 in a Phase IIa clinical trial in
ALS. Genervon is currently recruiting patients for a Phase II stroke study
and a Phase II study in Parkinson’s has also been approved.
In the fourth and final talk, the focus of the session
moved from biologics to devices. Geoffrey Thrope, Founder and Managing Director
of the venture capital and commercialization firm NDI Medical, spoke about a new technology
for deep brain stimulation (DBS) systems developed by a new spinout revealed at
the conference, Deep Brain Innovations (DBI). DBI has developed a technology called Temporally
Optimized Patterned Stimulation (TOPS) which optimizes the efficiency and
enhances the performance DBS systems in Parkinson’s disease patients. TOPS uses
an algorithm to identify patterns that can be used to optimize energy and
efficiency of DBS devices allowing them to function longer, which in turn
minimizes the number of battery replacements required by a device, translating
to reduced health care costs and patient risk. A blinded market survey of
neurosurgeons showed that TOPS delivers what the market
demands.
During the question and answer part of the session, many
of the common themes heard throughout the conference were touched on. These
included the hesitation that the investment community has around “new” drugs
with novel mechanisms, such as FPRT Bio’s TFNα inhibitor and Genervon’s growth
factor based drug. Dr. Tesi suggested education is the key to derisking these
“new” drugs. At the end of the Q & A, Thrope argued that although the
investment community is cautious about investing in “new” drugs, the primary
challenge is the limited number of partners willing to invest and the time it
will take to see a return on their investment. Although this was somewhat
discouraging news, based on the topics discussed in this session, let’s hope
the continued successes of these four companies (as well as others) will help
to change the way that investors are thinking. - Jessica Goodman
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| Seth Cassel |
The Samuel H.
Abramson Memorial Research Fellowship (Abramson Fellowship) at Harvard
University was established in 1983 by Edward and Harriet Abramson in honor of
Edward’s father to support students conducting research on issues related to
Israel and Jewish studies. However, when
Edward (HC'57 and HLS'60) passed
away following a courageous battle with ALS in 2001, the focus of the
fellowship shifted to support students conducting stem cell research
particularly in the areas of ALS and neurodegenerative disease.
The Abramson
Fellowship is open to Harvard University students in their sophomore, junior,
or senior years. The Abramson Fellowship has been awarded to more than 250 of
Harvard’s best and brightest students.
Thank you to
Prize4Life supporter and ALS advocate Harriet Abramson, as well as Dr. Jeffrey
Macklis, Professor of Stem Cell and Regenerative Biology at Harvard University
and mentor to many of the students that receive the Abramson Fellowship, for
the opportunity to get to know some of the most
recent recipients of the
Abramson Fellowship.
Seth Cassel, Class of 2013
Harvard
University senior and Abramson Fellow Seth Cassel was born and raised in
Baltimore, Maryland and attended McDonogh School in Owings Mills, Maryland. Seth became interested in research in seventh
grade when he wrote a speech on the power of embryonic stem cells. After
his presentation, he continued to read more about stem cell research and
decided that that he wanted to further pursue his growing interest at college. Seth chose to attend Harvard University because
of the available resources in stem cell biology which include Harvard’s
world-renowned Department of Stem Cell and Regenerative Biology and a unique
undergraduate major, Human Developmental and Regenerative Biology, which is
specifically dedicated to the study of cellular plasticity.
In addition to
his interest in stem cell research, Seth has been an active member of the Harvard
community having served as president of the Student Mental Health Liaisons and as
president and Editor-in-Chief of the Harvard Science Review. He also cofounded the Harvard chapter of DREAM
Youth Mentoring as well as cofounded a youth literacy website, LitPick.com.
We recently
asked Seth to share his thoughts about being awarded an Abramson Fellowship in
the summer of 2012 and how his research at Harvard will impact the
understanding of neurodegenerative diseases such as ALS.
Q: How did you become involved in the
Abramson Fellowship program?
A: When I came to college, I knew I wanted to
study stem cell biology. Since hearing Christopher
Reeve and Nancy Reagan advocate for stem cell research when I was in seventh
grade, I have been fascinated by the incredible therapeutic potential that stem
cells hold. In order to continue
exploring this interest, I decided to concentrate in Human Developmental and
Regenerative Biology at Harvard. During
my sophomore year in college, I joined Professor Kevin Eggan’s lab in Harvard’s
Department of Stem Cell and Regenerative Biology. Two years after joining the lab, I was
honored to be awarded the Abramson Fellowship.
The Abramson Fellowship supported my research in the months leading up
to the submission of my senior thesis,
“Old cells have new tricks: Nestin+ neural progenitor cells in SOD1G93A-Tg
disease model mice do not respond to neurodegeneration in the SVZ or in
adjacent brain regions but proliferate and differentiate down astrocyte and
oligodendrocyte lineages in the spinal cords of diseased mice.”
Q: What type of research are you currently
conducting?
A: In my sophomore year I took the “Stem Cells and Regeneration in the
Pathobiology and Treatment of Human Disease” course taught by Professor George
Daley which explores the intersection of science and medicine. In the class, I became interested in the
behavior of neural progenitor cells in the brain and spinal cord in the context
of ALS. These neural progenitor cells
have been reported to produce new mature cell types in response to spinal cord injury
and thus represent a potential source of cells to ameliorate cell death following
trauma. I hypothesized that neural
progenitor cells may behave in a similar proliferative fashion during ALS
disease progression. Therefore, my
project is aimed at studying these neural progenitor cells in ALS to better
inform efforts to modulate their behavior for therapeutic purposes during
disease progression.
Q: Why did you choose to focus on neurodegenerative
disease research?
A: About six years ago, my grandfather was diagnosed with dementia. I saw his condition steadily decline during
the time I have been in college. One of
the most frustrating aspects of the disease is that there is little doctors can
do to help him maintain his memory. This
experience has fueled my desire to study neurodegenerative diseases in my
classes and through scientific research. Given my interest in stem cells, I found that
the clinical challenges presented by neurodegenerative diseases offered me an
opportunity to use regenerative biology to work towards improving human health.
Thus, I was fortunate to discover
Professor Eggan’s laboratory in Harvard’s Department of Stem Cell and
Regenerative Biology which gives me a chance to participate in research at the
intersection of basic science and medicine.
Q: How has being awarded an Abramson
Fellowship motivated you to continue with your research?
A: During my time studying ALS, some of my most powerful and motivating
experiences have come as a result of being connected to the ALS community. I have had the opportunity to shadow
neurologists in an ALS clinic and meet patients affected by the disease. Additionally, the Abramson Fellowship has
further strengthened my connection with ALS and brought me closer to those who
are personally affected by the disease.
In addition to funding my research, the Abramson Fellowship gave me the
opportunity to attend two conferences – one in Japan and one in Boston – in
order to learn more about the field of ALS research, which has influenced the
direction of my own work. Through these
experiences, I was also able to meet ALS patients and hear their moving stories
of living with the disease.
Q: What are your plans for the future?
A: I am fascinated by the ability of basic science to impact human health. This year I am applying to MD/PhD programs. I see an MD/PhD education as an incredibly
important step in furthering my understanding of both basic science and
clinical medicine. With this foundation,
I hope to serve as a clinician-scientist, using clinical observations to direct
basic science research to maximize our impact on human health.
Q: What is your message to ALS community?
A: One of the most powerful experiences for me has been hearing the
stories and seeing the enthusiasm that comes from those who are personally
affected by the disease. Your bravery
and willingness to publically share your experiences puts a human face on the
disease, giving incredible meaning to the work we are doing. Also, during my time working in Professor
Eggan’s lab, I have been exposed to an impressive array of ALS researchers worldwide. ALS is a complex disease, but exciting
progress is being made. The level of
collaboration between labs is inspiring as each draws upon its own area of
expertise to form a powerful network of researchers trying to understand and
treat this awful disease. I want to
thank those in the ALS community and the supporters of the Abramson
Fellowship. The opportunity to be a part
of the ALS research effort has a profound effect on me personally. It has helped me realize that I want to work
to bring science into the clinic to help develop better therapeutics for
patients.
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Janet Song
|
The Samuel H. Abramson Memorial Research Fellowship
(Abramson Fellowship) at Harvard University was established in 1983 by Edward
and Harriet Abramson in honor of Edward’s father to support students conducting
research on issues related to Israel and Jewish studies. However, when Edward passed away following a
courageous battle with ALS in 2001, the focus of the fellowship shifted to
support students conducting stem cell research particularly in the areas of ALS
and neurodegenerative disease.
The Abramson Fellowship is open to Harvard University
students in their sophomore, junior, or senior years. The Abramson Fellowship
has been awarded to more than 250 of Harvard’s best and brightest students.
Thank you to Prize4Life supporter and ALS advocate
Harriet Abramson, as well as Professor Jeffrey Macklis, Professor of Stem Cell
and Regenerative Biology at Harvard University and mentor to many of the
students that receive the Abramson Fellowship, for the opportunity to get to
know some of the most recent recipients of the Abramson Fellowship.
Janet Song, Class
of 2013
Harvard University senior and Abramson Fellow Janet Song
grew up just outside of Philadelphia in Audubon, Pennsylvania. Janet’s interest
in science began when, as a sixth grader, she started participating in science
fairs. Her passion for science continued to develop throughout middle school, and
as a freshman in high school, Janet was inspired to conduct cancer research at Drexel
University College of Medicine under the guidance of Dr. Timothy Block. Janet
made significant progress on her research project, which was to develop a urine-based
test that could be used for the early detection of cancer, qualifying her as a
finalist in the 2009 Intel Science Talent
Search. She went on to become her high school’s valedictorian before
entering Harvard in the fall of 2009.
In addition to her impressive academic work, Janet is an
accomplished musician; in 2004 she won the World Piano Competition and she has
also performed at Carnegie Hall. When she is not in lab, Janet is an avid
Philadelphia Eagles fan and enjoys playing video games.
We recently asked Janet to share her thoughts about being
awarded an Abramson Fellowship in the summer of 2012 and how her research at
Harvard will impact the understanding of neurodegenerative diseases such as
ALS.
Q: How did you
become involved in the Abramson Fellowship program?
A: Performing cancer research at Drexel University was really
fun -- and while working there I caught the “research bug.” I knew that I
wanted to continue to do scientific research, and potentially one day obtain a
Ph.D. in science. Harvard is one of the best research institutes in the world,
which made it a very attractive choice for college. Since arriving at Harvard
in 2009, I have been involved with the Harvard College Undergraduate Research
Association (HCURA), and was the co-president of HCURA in 2011. I have had an incredible number of opportunities as a
HCURA board member. I worked with fellow HCURA students to launch the first
ever National Collegiate Research Conference at Harvard in 2012. This conference
drew over 150 participants and included an impressive list of speakers from a
variety of disciplines. In addition to my participation in organizations
focused on scientific research, I have also been directly involved with basic
research since my freshman year. I was lucky to have been assigned Professor
Jeffrey Macklis as my freshman academic advisor. During the course of my
freshman year, Professor Macklis and I discussed which lab would be the best
fit for my research interests, and we mutually decided that joining his lab
would be a great fit. In the spring of my freshman year I became an official
member of the Macklis laboratory in the Harvard Department of Stem Cell and
Regenerative Biology. Two years later, in the summer of 2012, I had the honor
of receiving the Abramson Fellowship to support my research efforts. The
research I conducted with the support of the Abramson Fellowship culminated in
my senior thesis, “Investigation of candidate molecular controls over segmental
specificity of corticospinal motor neurons during development.”
Q: What type of
research are you currently conducting?
A: I currently work with postdoctoral fellow Dr. Vibhu
Sahni to characterize the role of a particular gene in corticospinal motor
neuron (CSMN) development. We are specifically looking at how this gene
regulates CSMN axon outgrowth. This research has direct applications for ALS. The
information that we are learning from these studies may allow us to identify
ways to keep motor neurons connected to the muscles they support and control. In
addition, my current work may also help people with spinal cord injuries. My
research is giving us clues about how we could potentially regenerate CSMN
after spinal cord injury.
Q: Why did you
choose to focus your research on neuroscience?
A: I have always been very interested in neuroscience. As
a junior in high school, I had the opportunity to work in Dr. Gordon Harris’
laboratory at Massachusetts General Hospital. In Dr. Harris’ lab, I took a
macro approach to neuroscience research -- I used magnetic resonance imaging (MRI) to study
the effects of alcohol on the size of the brain. As a high school senior, I decided
to attend Harvard University for college because I knew it would be a rich
environment filled with experts working in molecular biology and genetics,
especially in the field of neuroscience. After joining Harvard as a freshman, I
knew I wanted to understand how the brain works at the molecular level. I decided
to work in the Macklis laboratory where I am now using a variety of molecular
biology techniques to understand how neuronal growth is regulated.
Q: How has being
awarded an Abramson Fellowship motivated you to continue with your research?
A: Working with Professor Macklis and the other members
of the Macklis lab, including fellow Abramson Fellowship awardee Chris Devine,
solidified my decision to pursue a career in science. While I do not focus too extensively on the
potential clinical applications of my research, I am motivated by knowing that my
work may someday help people that are living with neurodegenerative diseases
such as ALS. ALS is a terrible disease that often affects people in the prime
of their lives. With so little known about this disease, I am inspired to do
what I can to help patients live longer, happier, and more productive lives.
Q: What are your
plans for the future?
A: Working in Professor Macklis’ lab has allowed me to
learn more about designing experiments and how to think about scientific
problems. I have learned the value of perseverance and consistent hard work. The
fact is most experiments do not work, but you have to keep trying. Even though
99% of the time my experiments fail, it is the one time the experiment works
which motivates me to continue my research. Working in Professor Macklis’ lab
and learning these extremely valuable lessons have made me realize that I want
to pursue a career in scientific research. After I graduate from Harvard in May,
I will attend Stanford where I will pursue a Ph.D. in genetics. After that, I
hope to hold a position as a postdoctoral fellow where I will prepare for an
academic research position.
Q: What is your
message to ALS community?
A: If we look back many decades, before recent medical
advancements, many common diseases and ailments were potentially fatal. What we
have learned over the past few decades is that there is hope for those
afflicted with conditions that were once deemed incurable. Just thirty years
ago, those diagnosed with HIV/AIDS were given a death sentence. In a relatively
short period of time, HIV/AIDS has become a manageable disease and patients are
given the chance to live long and fulfilling lives after diagnosis. It is my
hope this will soon be the case for ALS. Although we do not currently have
effective treatments or a cure for ALS, I am optimistic that we will continue
to make scientific discoveries that will lead to the discovery of these effective
treatments and cures, giving hope to ALS patients around the world that are battling
this debilitating disease.
Prize4Life is pleased to bring you coverage of the 7th Annual Drug Discovery for Neurodegeneration Conference, a conference presented by the Alzheimer’s Drug Discovery Foundation (ADDF). The conference, held February 10-12, 2013 in sunny San Francisco, California, brought together attendees from a variety of different sectors including academia, industry, biotech, government, and non-profit organizations. The goal of the meeting was to delve into the details of the drug discovery process -- covering topics from high throughput screening, to identifying and optimizing a lead compound, to preclinical studies – all in the context of the important considerations that need to be addressed when developing drugs for neurodegenerative diseases such as Alzheimer’s disease and ALS.
Dr. Howard Fillit, ADDF Executive Director and Chief Science Officer, opened the conference with a broad overview of the current state of the field. In his talk, Fillit suggested that the drug development landscape is changing. There is much more collaboration between industry and academia then even five years ago, and such collaboration is necessary for successful drug development. Yet, although we are making progress and these collaborations are helping advance discoveries, scientists in the business of developing drugs for neurodegenerative diseases still face tremendous challenges. Three of these challenges will be covered in today’s post: 1. the changing and evolving use of animal disease models in translational research, 2. the rising importance of biomarkers, and 3. the inherent difficulties in targeting protein-protein interactions (particularly in the brain).
The first plenary speaker Dr. Piet van der Graaf, Senior Director of Clinical Pharmacology/Pharmacometrics at Pfizer, didn’t shy away from one of the most controversial topics discussed at the meeting -- the reliability of using animal models of disease for preclinical testing. In his presentation “Transforming Drug Discovery for Complex Diseases: A Systems Biology Approach” van der Graaf suggested that all too often animal disease models have little predictive value. His comments echoed the recent findings published in Proceedings of the National Academy of Sciences that showed some mouse models of disease may not recapitulate the immune response observed in human disease. The discussions at the conference emphasized the growing concern in the drug development community of whether mice are an appropriate model for human disease, or if we need to find new disease models. Computational and iPS cell derived models are two alternatives that were discussed in detail at the conference.
But should we really leave our mice behind? Dr. Katya Tsaioun of Pharma Launcher challenged van der Graaf’s thinking about animal models. She argued that we need to closely examine the black/white thinking that animal models “don’t predict” -- perhaps we just need to be more aware of the limitations of our animal models. Both Tsaioun, during her talk “Absorption, Distribution, Metabolism, Excretion and Toxicology (ADME-Tox) in Compound Refinement”, and Fillit in his opening remarks, suggested that animal models are highly useful for collecting multiple supporting pieces of data critical for successful drug development. Effective and well-understood animal models of disease allow us to look at disease mechanisms and investigate what is going on at the molecular level. Using animal models to collect these types of data might be the best way to harness these models in a productive way. Although the jury is still out on the utility of animal models of neurodegeneration -- this is clearly a perennially hot topic that was in hot debate at the conference!
The importance of identifying and developing biomarkers, was another underlying theme highlighted by several speakers at the conference. Dr. Kalpana M. Merchant, Chief Scientific Officer for Translational Science at Eli Lilly and Company, in her talk “Improving Clinical Success through Optimal Target Validation” emphasized that biomarkers are essential for monitoring whether therapies have engaged their target. How do you successfully choose the right biomarker? Make sure that you know your therapy’s target and then make sure you have a disease-associated biomarker that you can use to monitor target engagement. Tsaioun also pointed out that once you have a druggable target, biomarkers can help with correlating your in vitro results with your in vivo results. Further to her point about changing the way we use our animal models, Tsaioun suggested that animal-based disease models can be useful for collecting biomarker data.
One of the biggest questions on everyone’s mind throughout the conference was: why don’t we have more drugs for neurodegenerative diseases? One obvious answer is that it is hard to get drugs across the blood-brain barrier. Dr. Bruce Morimoto of Allon Therapeutics Inc. in his talk “Peptide Therapeutics and Mechanisms for Intranasal Delivery” suggested that one solution to this problem is to use intranasal delivery, which allows drugs to bypass the blood-brain barrier entirely. Another answer for why there are so few drugs for CNS diseases is that many neurodegenerative diseases are protein-aggregation diseases, and protein-protein interactions are notoriously difficult to target. Dr. Jim Wells, Professor in the Department of Pharmaceutical Chemistry at the University of California, San Francisco, discussed two key limitations to targeting protein-protein interactions in his talk entitled “Challenges of Protein-Protein/Protein-Peptide Targets.” First, proteins often associate with one another using large interfaces that make preventing these interactions difficult. Second, proteins often form tight complexes with one another that need to be dissociated in order to restore function and/or remove dysfunctional proteins from the cell. Wells mentioned that one way to target unwanted protein-protein interactions is to use alanine-scanning mutagenesis to identify hot spots of binding and then you could potentially find ways to target these hot spots with drugs. Dr. Ryan Watts, Associate Director and Head of Neurodegeneration Labs in the Department of Neuroscience at Genentech, Inc., suggested in his talk “Developing Therapeutic Antibodies for Neurodegenerative Disease” that antibodies are the best way to target protein-protein interactions. However, despite the promise antibodies hold for targeting and disrupting protein-protein interactions, they do have some limitations including the challenge of getting the antibodies across the blood-brain barrier (bringing things full circle!).
This summary is only a small taste of the numerous useful topics discussed at ADDF’s 7th Annual Drug Discovery for Neurodegeneration Conference. The conference was a great success -- the attendees received some hearty "food for thought" about our current system of drug development, especially in the complex and challenging neurodegenerative disease space. For those of you who weren’t able to make it to California for the conference (maybe you happened to be snowed in by a blizzard) but want to learn more, the entire 2013 Drug Discovery Conference webcast is now available. Click here to watch it for free. - Jessica Goodman
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| Christopher Devine |
The Samuel H. Abramson Memorial Research Fellowship (Abramson
Fellowship) at Harvard University was established in 1983 by Edward and Harriet
Abramson in honor of Edward’s father to support students conducting research on
issues related to Israel and Jewish studies.
However, when Edward passed away in 2001 after a courageous battle with
ALS, the focus of the fellowship shifted to support students conducting stem
cell research particularly in the areas of ALS and neurodegenerative
disease.
The Abramson Fellowship is open to Harvard University
students in their sophomore, junior, or senior years. The Abramson Fellowship
has been awarded to more than 250 of Harvard’s best and brightest students.
Thank you to Prize4Life supporter and ALS advocate Harriet
Abramson, as well as Dr. Jeffrey Macklis, Professor of Stem Cell and
Regenerative Biology at Harvard University and mentor to many of the students
that receive the Abramson Fellowship, for the opportunity to get to know some
of the most recent recipients of the Abramson Fellowship.
Christopher Devine,
Class of 2013
Harvard University senior and Abramson Fellowship awardee
Christopher Devine grew up just outside of New York City in Ramsey, New
Jersey. Before beginning his studies at
Harvard in the fall of 2009, Christopher attended high school at Bergen County
Academies (BCA) where he chose to specifically focus on medical science
technology. While at BCA, Christopher
honed his leadership and communication skills.
These skills have matured and continue to serve him well with his
studies and scientific research efforts at Harvard.
We recently asked Christopher to share his thoughts about
being awarded an Abramson Fellowship in the summer of 2012 and how his research
at Harvard may impact the understanding of neurodegenerative diseases such as
ALS.
Q: How did you become
involved in the Abramson Fellowship program?
A: Since arriving
at Harvard, I have been actively involved with a number of activities including
peer advising and tutoring, and have held leadership roles in Student
Government and on the Student Life Committee. However, my most personally and
professionally defining experience at Harvard has been being a member of Dr.
Jeffrey Macklis’ laboratory in the Harvard Department of Stem Cell and
Regenerative Biology. I joined the lab
in the spring of my freshman year as an undergraduate concentrating in
Neurobiology. Two years later, in the
summer of 2012, I had the honor of receiving the Abramson Fellowship to support
my research efforts. The research I conducted with the support of the Abramson
Fellowship culminated in my senior thesis, “Investigating the Fidelity of
Axolotl Forebrain Regeneration.”
Q: What type of
research are you currently conducting?
A: Research in
the Macklis lab is at the forefront of developmental and regenerative biology.
I am currently working with postdoctoral fellow Dr. Hari Padmanabhan to better
understand neuronal development and regeneration. More specifically, I work
with a unique species of salamander that can naturally regenerate its body
parts, including the spinal cord and brain. If we are able to better understand
the mechanisms involved with brain regeneration and repair, it will bring us
one step closer to understanding the complexity of neurodegenerative diseases
like ALS.
Q: Why did you choose
to focus your research on understanding neuronal regeneration and
neurodegenerative diseases?
A: Of all known
medical conditions, neurodegenerative diseases are by far the least
understood. The fact that so little is
known about neurodegenerative diseases, and in particular ALS, has served as a
powerful motivator for me to engage in basic scientific research focused on the
development and regeneration of the central nervous system. It is important for
me know that my research, no matter how small the contribution is, may in fact
contribute to the fight against ALS and possibly provide hope to those who are
currently battling the disease or will be diagnosed in the future.
Q: How has being
awarded an Abramson Fellowship motivated you to continue with your research?
A: Being awarded
an Abramson Fellowship has empowered me to become involved with understanding
how to translate basic research findings into discoveries that can one day help
treat patients with neurodegenerative diseases. I have shadowed neurologists
and have seen how debilitating neurodegenerative diseases such as ALS and
Parkinson’s disease can be to patients and their loved-ones. These diseases can
strike anyone regardless of who you are, where you come from, or what you have
done with your life. Not only is the
research I am conducting intellectually stimulating, it reminds me that I am
making a difference for ALS patients. I
am truly lucky to be participating in cutting edge research that will
contribute to a better understanding of the cellular mechanisms that drive
neuron development and repair.
Q: What are your
plans for the future?
A: My long term
goal is to help those who suffer from neurodegenerative diseases as a
physician-scientist. This will allow me to engage in both medical practice and
in translational research. I am confident that the foundation I have built at
Harvard will help me to achieve this goal.
Following graduation this May, I will be attending the University of
Cambridge in the United Kingdom to pursue my MPhil in Translational Medicine
and Therapeutics as well as continue to develop my skills in translational
research. After that, I plan to return to Harvard Medical School and then
eventually practice medicine with the hope of using translational research to
take potential therapies from the bench to the bedside.
Q: What is your
message to ALS community?
A: Conducting
innovative research and working together is what I believe will lead to
effective treatments and a cure for ALS and other neurodegenerative
diseases. And it is not just the work I
am conducting in the Macklis lab as an Abramson Fellowship awardee; it is the
work of other Abramson Fellowship awardees, undergrads, graduate students, post
docs, and researchers around the world as well who are studying basic biology
and making new scientific discoveries to advance our understanding of
neurodegeneration. I would like to express my appreciation to the ALS community
for being so supportive and passionate.
This is incredibly meaningful to students like myself and keeps us
motivated to continue pursuing scientific research that contributes to the
fight against ALS. I want to say to the
ALS community and the supporters of the Abramson Fellowship, thank you for
allowing young researchers like me to be involved in such an important mission.
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