May 26, 2014 Issue
The Most Powerful Name In Corporate News and Information
Blood-based Tests for Early Detection of Neurodegeneration
DiamiR, LLC is a
privately held molecular diagnostic company focused on developing
noninvasive tests for early detection and monitoring of Mild Cognitive
Impairment, Alzheimer's disease, other neurodegenerative diseases, and
cancer. The proprietary technology is based on quantitative analysis of
circulating organ-enriched microRNAs in plasma and can be used for
screening, patient stratification as well as disease and treatment
11 Deer Park Drive, Suite 102G
Monmouth Junction, NJ 08852
Interview conducted by: Lynn Fosse, Senior Editor, CEOCFO Magazine, Published Ė May 26, 2014
CEOCFO: Dr. Sheinerman, what is the concept for DiamiR?
Dr. Sheinerman: The concept is to be able to diagnose neurodegeneration early on when intervention can be effective prior to massive cell death. That is the initial and basic concept that we operate on. We are trying to capture neurodegeneration at synaptic dysfunction stage that precedes neuronal death.
CEOCFO: Has this been tried in the past?
Dr. Sheinerman: I am guessing it has, but not very successfully. The brain is a very complex organ. Today we know that Alzheimerís disease for example begins 20+ years prior to clinical manifestation. Our brain has trillions of synapses and billions of neurons, and they compensate for each otherís functions until it is no longer possible to compensate for neuronal damage. When clinical manifestation occurs, this is already a sign of a more advanced condition. How do you diagnose something very early, pre-symptomatically? Recently, significant progress has been made with neuroimaging and analysis of cerebrospinal fluid biomarkers. These technologies are rather invasive and expensive. Insurance companies are not rushing to reimburse for these procedures. There is a strong need for a non-invasive or minimally invasive technology able to close this gap of early, preferably pre-symptomatic detection of neurodegeneration.
CEOCFO: What is your approach?
Dr. Sheinerman: Our approach is based on analysis of microRNA biomarker pairs in plasma. MicroRNAs are small non-coding RNAs that were discovered fairly recently; it is a quickly growing field. They are small, so they are about 22 nucleotides in length on average, and they are able to cross the blood brain barrier and other barriers in the human body. They are relatively stable in circulation, and most importantly for our approach, they can be specific to, or significantly enriched in certain organs, tissues, cell types and even cellular compartments. Speaking specifically about neurodegeneration, there are microRNAs that are enriched in the specific regions of the brain, such as the hippocampus or midbrain, certain types of neurons and even in synapses and neurites. This is the basis of our technology. We did an extensive data mining exercise analyzing the literature, databases and other sources available, and created our own internal database of organ-enriched microRNAs. To date, about 2,000 human microRNAs are known. We estimate that about 100 to 120 of them are brain enriched. For our purposes, microRNAs also need to be detectable in blood. To date, we analyzed about 50 brain-enriched microRNAs as potential biomarkers for neurodegenerative diseases, including Mild Cognitive Impairment, a condition at the root of many neurodegenerative diseases, such as Alzheimerís, Parkinsonís, Chronic Traumatic Encephalopathy and Frontotemporal dementia. Our current focus is on Mild Cognitive Impairment, Alzheimerís and Parkinsonís, and we are expanding our programs to other neurodegenerative diseases. A key to our approach is the use of microRNA biomarker pairs, consisting of microRNAs present in neurites and synapses of brain regions known to be affected during disease development and microRNAs enriched in other brain regions.
CEOCFO: Would you give us an example of what you are looking at, what you might find and how you evaluate?
Dr. Sheinerman: In case of Alzheimerís disease, we are looking at microRNAs that are present in synapses and neurites of the hippocampus. The hippocampus is known to be affected during the development of Alzheimerís disease, so the idea behind our approach is that dysfunction and destruction of synapses lead to higher secretion and excretion of microRNAs present in those synapses into extracellular space; these microRNAs ultimately appear in the blood stream, where we detect them. For other neurodegenerative diseases, we focus on microRNA biomarkers present in cells of those specific brain regions affected during the disease development. In the case of Parkinsonís disease we select a biomarker among microRNAs enriched in midbrain and frontal cortex, and a second microRNA in the pair among brain-enriched microRNAs not enriched in these brain regions. In the example of Alzheimerís disease, second microRNA in the pair would be brain-enriched microRNA not enriched in hippocampus. This normalization technique allows us to compensate for variable factors related to the brain, such as blood-brain barrier permeability, but not related to the disease.
CEOCFO: What have you found so far that has surprised you?
Dr. Sheinerman: I am happy to report that so far our experimental results are in line with our expectations. We conducted a number of studies, including in collaboration with the Roskamp Institute of Sarasota, FL, that have confirmed that the brain-enriched biomarker pairs that we detect in plasma with a quantitative RT-PCR are able to successfully differentiate Mild Cognitive Impairment, Alzheimerís disease, Parkinsonís disease, and age-matched control from each other. For example, Mild Cognitive Impairment is differentiated from age-matched control with 96% accuracy. Next, we would like to be able to identify Mild Cognitive Impairment patients who would progress to Alzheimerís disease.
CEOCFO: Would you tell us about your recent patent?
Dr. Sheinerman: The US PTO granted a patent to us in February of this year. This patent covers early detection and monitoring of neurodegenerative diseases using our approach. The overall situation in medical diagnostic IP is currently not favorable, so we are quite happy the US PTO found our application contained a number of innovations for the patent to be granted.
CEOCFO: Is the medical community or the portion that should be aware of what you are doing knowledgeable about DiamiR or is it too early?
Dr. Sheinerman: We have started to talk about our work about a year and a half ago. We now have three peer reviewed publications reporting our results; we have some feature articles and we also published a review focused on the role of microRNAs as biomarkers for neurodegenerative and neurological disorders. Now, I think we are gaining quite a bit of interest for our work from the biomedical community. We presented a poster at the Alzheimerís Association meeting last year, and we are planning to present again at the Alzheimerís Association meeting in July of this year. We have established several collaborations, because obviously we are relatively early stage and we need to further validate our technology. We need to analyze more samples from heterogeneous cohorts, including from longitudinal studies, with our biomarker signatures. Right now, I can say that we have access to samples to be analyzed and there is interest from academia in working with us.
CEOCFO: Where are you with funding? I know you have a background in investment and merchant banking. How does that help?
Dr. Sheinerman: Up until now, the company has been funded with founderís capital as well as grants. We were fortunate to receive some highly competitive grants. We will continue to pursue such non-dilutive funding. Research in Alzheimerís disease is gaining momentum and many people are paying attention. This being said, a biotech company can do things much faster with additional capital; there is always a bare minimum and there is a wish list of what we could accomplish. I think right now we are getting to the point where we have enough critical mass to start raising capital. The programs are prioritized, we published peer reviewed papers, we have received our patent, we have established collaborations and we have a full-time CEO. I co-founded this company in 2009, but I joined full time last summer. All of this is coming together nicely and I think we should be ready to start raising capital soon. We are a small company, and we can manage capital very efficiently. We are considering different funding scenarios, including private and public offerings.
CEOCFO: How do you deal with the frustration of knowing it is such a slow and arduous process?
Dr. Sheinerman: This is a fair question. I am coming from the investment banking world, which is often about a sense of urgency. The more you work, the faster you can close a transaction. This experience does not translate directly to research and development of commercial products, which is where we are heading with our first assay. I am learning that one has to be patient. The only thing that we can do is to facilitate and streamline the process. A factor to take into account is that Alzheimerís is a slowly developing chronic disease, and clinical studies take a long time. This is why we are trying to maximize use of well-characterized retrospective samples as much as possible. At the same time, we are starting to be involved in some prospective studies as well.
CEOCFO: Put it all together for our readers. There are many companies to look at in your arena. Why DiamiR?
What we are
working on is delivering a brain signature detectable in peripheral system,
i.e. blood. There is a great unmet need in non-invasive, accurate,
cost-effective tests for early detection and monitoring of neurodegenerative
diseases. There are attempts to develop blood-based tests using proteins,
antibodies and lipids as potential biomarkers. We respect and closely follow
the research of others, at the same time I believe we are in the lead as far
as microRNA biomarkers are concerned, and this class of molecules holds a
great promise. With our approach we detect changes in the brain with
microRNA biomarker signatures in blood, and thus we hope to be able to
monitor brain health with a simple non-invasive test. Importantly, we
already completed a small longitudinal study demonstrating power of our
approach: in the study we detected Mild Cognitive Impairment in the majority
of patients one to five years prior to clinical diagnosis. Finally, we are
applying similar approach based on organ-enriched microRNA pairs to early
detection of gastrointestinal and pulmonary system diseases Ė we recently
published data from the first study, which appears highly promising.
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