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Posts tagged ‘Dr Todd Sherer’

Using CSF Biomarkers for Differential Diagnosis of Neurological Diseases

Drug development for neurodegenerative brain diseases such as Parkinson’s or dementia, of which Alzheimer’s is the most common form, needs to focus on patients early in the disease, not those where brain damage has already occurred.

Diagnosing and treating patients more effectively earlier will, even if you aren’t able to instigate a cure, offer the ability to modify the disease progression and slow or delay when brain damage occurs.  In the case of Alzheimer’s, once the amyloid plaques (tangles of misshapen proteins) have accumulated in nervous tissue, it has so far been impossible to untangle or remove them.

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Last year, I interviewed Dr Todd Sherer, (then the Chief Program Officer) and now the CEO of the Michael J. Fox Foundation, who told me that: “biomarkers are a real focus of the foundation.” Sherer went on to say that:

“Parkinson’s is a difficult disease to diagnose, there is no definitive diagnostic test, so it ends up a clinical diagnosis.  Getting a biomarker that could help better confirm the diagnosis would allow people to get the correct treatment earlier in their disease”

Which is why I was interested to see new research published earlier this week in the journal Archives of Neurology (online first, August 27, 2012), by Sara Hall and colleagues at Lund University, University of Gothenburg and Skåne University Hospital in Malmo, Sweden.

Hall and colleagues describe how a panel of five cerebrospinal fluid (CSF) biomarkers allowed the differential diagnosis of common dementia from Parkinsonian disorders:

  • Beta-amyloid 42
  • Total tau
  • Phosphorylated tau
  • Alpha-synuclein
  • Neurofilament light chain

Patients with early symptoms of neurodegenerative diseases can be hard to diagnose.  Misdiagnosis can occur, which means patients may not respond to treatment or they could be enrolled into a clinical trial, and end up skewing the results.

Ensuring that we have the right patients in clinical trials is important as we seek to alter disease progression.  In other words it’s important to see whether new drugs or treatments are impacting the disease course.  If you have a wrongly diagnosed patient in a trial, then the drug may show no effect, not because it’s not effective, but that patient’s disease is not responsive.

Multivariate analysis indicated that the panel of 5 CSF biomarkers could accurately differentiate Alzheimer’s disease (AD) from Parkinson disease with dementia (PDD), and dementia with Lewy bodies (DLB). The Neurofilament light chain biomarker alone could differentiate PD from atypical Parkinson disease, Hall and colleagues noted.

Whilst the panel was not able to distinguish all forms of dementia, in an accompanying editorial Richard J. Perrin MD, PhD from the University of Washington, stated that this research “represents a significant step forward.” Perrin concluded that:

“Implementation of CSF biomarker panels such as this one should improve the efficiency of clinical trials and accelerate the evaluation and discovery of new effective treatments for neurological diseases.”

Summary

Developing biomarkers that assist in the ability to diagnose Alzheimer’s, Parkinson and dementia patients correctly, and then be able to monitor their subsequent disease progression, should be a key focus of those biotechnology and pharmaceutical companies that want to do innovative and rational drug development.

References

ResearchBlogging.orgSara Hall, MD, Annika Ohrfelt, PhD, Radu Constantinescu, MD, Ulf Andreasson, PhD, Yulia Surova, MD, Fredrik Bostrom, MD, Christer Nilsson, MD, PhD, Hakan Widner, MD, PhD, Hilde Decraemer, Katarina Nagga, MD, PhD, Lennart Minthon, MD, PhD, Elisabet Londos, MD, PhD, Eugeen Vanmechelen, PhD, Bjorn Holmberg, MD, PhD, Henrik Zetterberg, MD, PhD, Kaj Blennow, MD, PhD, & Oskar Hansson, MD, PhD (2012). Accuracy of a Panel of 5 Cerebrospinal Fluid Biomarkers in the Differential Diagnosis of Patients With Dementia and/or Parkinsonian Disorders Arch Neurol. DOI: 10.1001/archneurol.2012.1654

Richard J. Perrin, MD, PhD (2012). Cerebrospinal Fluid Biomarkers for Clinical Trials Arch Neurol. (August 27 Online First) DOI: 10.1001/archneurol.2012.2353

Making a difference to Parkinson’s Disease Research, an interview with Dr Todd Sherer, Michael J Fox Foundation

Biotech Strategy blog recently had the privilege to do a phone interview with Dr Todd Sherer, the Chief Program Officer of the Michael J Fox Foundation.  In this two- part interview, Pieter Droppert asks what the MJFF approach to research funding is and what the future holds for Parkinson’s disease research?

 

Part 1:  Research Funding

Research funding is key to science. Without it there would be no translational medicine that takes basic research and turns it into clinical applications that benefit humans.  One organization that is making a difference and bridging the gap between patients and research is the Michael J Fox Foundation (MJFF).

Biotech Strategy Blog: What does a Chief Program Officer do?

Dr Sherer: My role as Chief Program officer is to lead the research efforts at the Michael J Fox Foundation. My background is as a neuroscientist so I have a PhD in neuroscience, and my lab work was in Parkinson’s disease before I joined the foundation.  At the foundation we have a number of additional scientists, seven in total. They work closely with people with business backgrounds in a collaborative way.  What we are trying to do is apply business principles to scientific research.  So, select the best scientific projects and then design them in a way where there are deliverables, milestones and goal directed research with the ultimate aim to improve treatment for Parkinson’s patients.

Biotech Strategy Blog: MJFF receives 800 grant applications a year, what is your approval process, is it similar to the National Institutes of Health?

Dr Sherer: It is modeled after the NIH process with the one main difference being that our internal scientific staff has a proactive role in making the final funding decisions.  We take the peer review panel to help us evaluate, but the ultimate decision lies with our internal staff.  We do a rapid turn around of those reviews so from receiving an application to funding is usually for us 3 to 4 months total.

Biotech Strategy Blog: What business principles do you use in managing the research that you fund?

Dr Sherer: One of the main things we do with every grant we make is laying out from the beginning: what is actually the goal of that grant. This probably seems logical, but actually a lot of scientific research is very open ended – it is kind of “let me see what I discover”. But we are really trying to define from the beginning: what are we trying to accomplish? Then what we can do is set milestones along the way to that goal to make sure during the course of the project, we are making the progress we need to achieve that goal.

Biotech Strategy Blog: Are grant payments linked to performance?

Dr Sherer: The payments for the grants are all tied to the milestones, and I would say that we are realistic in that we are experienced in how research works.  This is not manufacturing, and we know that you can have the best plan, but it is all based on a hypothesis and things can come up in the course of the research. Our scientific staff here will always work closely with those grantees to make the rational adjustments in the plan based on what we are learning along the way.

Biotech Strategy Blog: In addition to the grant applications that you receive, does the Michael J Fox Foundation initiate its own projects?

Dr Sherer: Yes. We have identified specific priority topics within Parkinson’s disease, whether they be specific therapeutic targets, or something like a biomarker, where we are more proactively working with the research community to develop projects to address critical challenges in those areas.  We also do conduct some research using a virtual research model where the scientists here may outsource some work with contract research organizations.  We have been particularly doing that around research tools so that we can make those tools widely available to our researchers — things like antibodies, plasmids, vectors —  we want to quickly get these made without any licensing or intellectual property restrictions and then make them broadly available to the research community.

Biotech Strategy Blog: Why does MJFF fund research by commercial entities e.g. biotechnology & biopharmaceutical companies?

Dr Sherer: The goal of the foundation, the goal of all the research that we are supporting is to develop new therapies for Parkinson’s disease patients.  We understand that will involve all players in the research space, so it involves academic researchers.  But really to convert those discoveries into therapies for people, we want to proactively engage and interact with the biotech and biopharma community because they are the ones who in the end make the therapies for patients.  About 5 or 6 years ago we decided to more proactively outreach to the biopharma community to determine: if we provided funding, could we induce more companies to work in Parkinson’s disease or accelerate their work in Parkinson’s disease.

Biotech Strategy Blog: What types of biotech & biopharma companies do you fund?

Dr Sherer: There are some companies that have worked in related diseases, like another neurodegenerative disease, and our  funding allows them to apply that technology to Parkinson’s disease.  Another category would be a platform type of company that we have supported, where they could really go to any disease, but the Fox funding allows creates a rationale to choose Parkinson’s.  Then we have funded some companies who are interested in Parkinson’s but for budgetary reasons have made some trade-offs in the design of that study. With additional funding from the foundation they can increase the sample size, hone the end points or what have you, to really make that study more robust.  We have seen a lot more companies working in Parkinson’s as a result of this outreach.

Biotech Strategy Blog: Do you take any rights in any commercial research you fund?

Dr Sherer: In most of our projects we do not have any stipulation like that, particularly in the case where we are funding some preclinical testing. That said, in our largest, multi-million dollar grants, particularly those in late-stage clinical development, we usually have an agreement that there is a return payment after some amount of sales.  That is more the exception than the rule.

Biotech Strategy Blog: Thank you

Part 2 of this interview with Dr. Todd Sherer, Chief Program Officer of the Michael J. Fox Foundation, will provide insight on the current status of research into Parkinson’s disease, and what we can expect the future to hold?

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