Monday, June 3, 2013

Highlights from Neurotech 2013: Movement Disorders: Parkinson’s, ALS, and More

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

Thursday, May 9, 2013

Meet Seth Cassel, a 2012 Recipient of the Abramson Fellowship at Harvard University


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.

Wednesday, April 17, 2013

Meet Another Recipient of the Abramson Fellowship at Harvard University


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. 

Friday, March 29, 2013

Prize4Life's Coverage of the 7th Annual Drug Discovery for Neurodegeneration Conference


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


Monday, March 25, 2013

Meet One of the Recipients of the Abramson Fellowship at Harvard University

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.







Monday, December 17, 2012

Torsten Hothorn, PhD, Second Place Winner of The DREAM-Phil Bowen ALS Prediction Prize4Life Challenge Profiled on InnoCentive Blog

Dr. Torsten Hothorn has been on quite a run lately working on Prodigy “Big Data” Challenges. Recently, he won the $30,000 Cleveland Clinic Challenge, Build an Efficient Pipeline to Find the Most Powerful Predictors, and he earned a $10,000 award for his second place finish in The DREAM-Phil Bowen ALS Prediction Prize4Life Challenge.  He was recently profiled as a guest blogger for InnoCentive's Perspectives on Innovation blog.  To read his post, click here.


Wednesday, December 5, 2012

Prize4Life is proud to announce the launch of the PRO-ACT database.


The PRO-ACT database is the largest ALS clinical trial database ever created containing patient data from completed clinical trials.  It is also the first ever open-access ALS database to be made available to researchers around the world who know how to use “Big Data” to mine for valuable information.

The PRO-ACT database contains data that could help to better classify newly diagnosed ALS patients.  This will assist in helping to better define the stages of ALS, as well as address whether there are different types of ALS.  Additionally, it can help to better design and conduct clinical trials which will save time and money.

Learn more at the PRO-ACT website.