Focus on Neurology & CNS: July 2012

BIO recently published the July 2012 version of its quarterly therapeutic newsletters.  These six newsletters focus on the therapeutic areas of member companies and include updates from biotech stakeholders around Washington, including Congress, FDA, NIH, and patient organizations. 

The therapeutic areas covered by these newsletters are:


  • Cardiology, Pulmonology, & Blood
  • Nephrology, Endocrinology, Metabolism, & Gastroenterology
  • Oncology
  • Allergy, Infectious Disease, & Antiviral
  • Rheumatology, Inflammation, Anesthesia, & Pain
  • Neurology & CNS

The below is an excerpt from the “Focus on Neurology & CN” newsletter.  To download it in its entirety, please click here.  If you have any questions or would like to receive a specific set of therapeutic newsletters, please contact Charles Crain at ccrain(at)

NINDS Funding Announcements

PAR-12-183, NIA Analysis of Alzheimer’s Disease Genome Sequencing Project Data(U19) – September 8, 2013

PA-09-217, The Role of Apolipoprotein E, Lipoprotein Receptors and CNS Lipid Homeostasis in Brain Aging and Alzheimer’s Disease(R01) – September 8, 2012

PAR-09-263, Ancillary Studies in Clinical Trials of CNS/PNS Disorders NINDS Accelerated Awards Program(R01) – August 17, 2012

PA-09-193, Mechanisms, Measurement, and Management of Pain in Aging: from Molecular to Clinical(R01) – January 8, 2013

PA-11-014, HIV Infection of the Central Nervous System(R01) – January 8, 2014

PA-11-067, Focal Cognitive Deficits in CNS Disorders(R01) – January 8, 2014

FDA Advisory Committee News

On May 14, 2012, the FDA Peripheral and Central Nervous System Drugs Advisory Committee met to discuss new drug application (NDA) 202737 for tafamidis meglumine capsules, proposed trade name VYNDAQEL, submitted by FoldRx Pharmaceuticals, Inc. a subsidiary of Pfizer, Inc. The proposed indication is for the treatment of transthyretin (TTR) familial amyloid polyneuropathy (FAP).

FAP is caused by a mutation in the gene that codes for TTR. Currently, the treatment of choice in the United States is liver transplant, because TTR is primarily synthesized in the liver.

The sponsor’s assertion is that the tafamidis-induced stabilization of TTR can successfully treat the symptoms of FAP.

The materials and minutes from this meeting are available online, as well as a complete transcript.  For more information, please click here

NINDS Research Provides Clues to Late-Onset Alzheimer’s Disease

Researchers may have discovered a mechanism behind the largest known genetic risk factor for late-onset Alzheimer's disease. The finding suggests possible strategies for prevention as well as a potential new drug target.

A hallmark of Alzheimer’s is a protein fragment called beta-amyloid, which is thought to be toxic and forms clumps within the brain. Past studies have revealed genetic risk factors for Alzheimer's disease. A gene called APOE has shown the strongest connection to the late-onset form of the disease.

A research team led by Dr. Berislav Zlokovic of USC studyied several lines of genetically engineered mice. One lacks the mouse version of the APOE gene altogether, and 3 other lines produce only human APOE2, APOE3, or APOE4. The study was funded by NINDS and NIA.   The researchers discovered that mice whose bodies made only APOE4, or no APOE at all, had a leaky blood-brain barrier. With the barrier compromised, harmful proteins in the blood made their way into the mice's brains.

The researchers found that APOE2 and APOE3 help control the levels of an inflammatory molecule called cyclophilin A (CypA), but APOE4 does not. Levels of CypA were raised about 5-fold in the blood vessels of mice that produce only APOE4. The excess CypA activated an enzyme called MMP-9, which destroys parts of the blood-brain barrier.

Treating the mice with the immunosuppressant drug cyclosporine A, which inhibits CypA, preserved the integrity of the blood-brain barrier and lessened damage to the brain. An inhibitor of the MMP-9 enzyme had similar effects.

These findings identify CypA as a potential new drug target for Alzheimer's disease. They also suggest that one approach to preventing Alzheimer's disease among APOE4 carriers may be to work on improving vascular health.

To learn more about this research, please here.

BIO Announces FDASIA and JOBS Act Webinars

BIO would like to invite you to participate in our upcoming educational webinar series in September and October on key provisions contained in the Food and Drug Administration Safety and Innovation Act (FDASIA) and the Jumpstart Our Business Startups (JOBS) Act.

The FDASIA webinars, scheduled for September 13 and September 26, will provide information on the intent and goals of the provisions in FDASIA, including Enhanced Communications, NME Reviews, Expanded Accelerated Approval, and Breakthrough Therapies.  Industry experts will also discuss implementation issues and timelines.

The JOBS Act webinars, scheduled for September 18 and October 3, will offer companies information on the key facets of the law and offer expert analysis on how to navigate the new rules.  The JOBS Act contains exciting new fundraising methods for biotech companies, including the IPO On-Ramp, Crowdfunding, and changes to SEC Regulations A and D.

The webinars are free for all BIO R&D members and BIO state affiliates.  Non-member R&D companies are invited to join for $100.  For more information or to register for the webinars, please email Charles Crain at

NCATS Announces Institutional CTSA Program

The NCATS CTSA program supports disease- and condition-specific networks funded by other NIH Institutes and Centers.  NCATS has announced that its CTSA program will include Institutional CTSA Awards.  Institutional CTSAs are made to degree granting institutions or groups of institutions that receive significant funding from the NIH.  CTSAs require institutional commitment, the status of a major scientific and administrative entity within and across an applicant and partner institution(s), and a CTSA PD(s)/PI(s) with the authority and influence necessary to successfully create an institutional home for clinical and translational research.

To learn more about the NCATS Institutional CTSA Program (RFA-TR-12-006), please click here.  The letter of intent is due December 10, 2012 and the application is due January 8, 2013.

To download the full “Focus on Neurology & CNS” newsletter, please click here.