Canavan Research Foundation, Inc.

Curing Brain Disease

aka C.R.F.   |   New Fairfield, CT   |  www.canavan.org

Mission

Canavan Disease is dedicated to eradicating neurodegenerative brain disease, including Canavan Disease, Taysachs Disease, MS, ALS, Alzheimer's, and Parkinson's.

Ruling year info

2001

Executive Director

Ms. Samantha Karlin

Main address

88 Route 37

New Fairfield, CT 06812 USA

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EIN

06-1579083

NTEE code info

Birth Defects, Genetic Diseases Research (H20)

Brain Disorders (H48)

Pediatrics Research (H98)

IRS filing requirement

This organization is required to file an IRS Form 990 or 990-EZ.

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Communication

Programs and results

What we aim to solve

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Our programs

SOURCE: Self-reported by organization

What are the organization's current programs, how do they measure success, and who do the programs serve?

Gene therapy

We are currently supporting a gene therapy trial to treat Canavan Children. This is the first vector that has the ability to treat the white matter in the brain, where the virus exists. It has the potential to cure children with this fatal disease.

Population(s) Served

The objective of this project is to define whether a nutritional supplement (anapleurotic therapy) capable of promoting energy metabolism can improve brain development and motor function in a mouse of model of Canavan Disease. Anaplerotic diet therapy is based on the concept that an energy deficit in certain diseases is improved by providing an alternative substrate for Krebs cycle and enhanced ATP production. Unlike other therapeutic approaches, which have a relatively long lead time, anapleurotic therapy may represent the shortest route to a disease modifying clinical therapy for Canavan disease. This study will generate safety and efficacy data to support the development of a Phase I/II study to assess the Safety/Benefit of Anapleurotic Therapy in patients affected by Canavan Disease.

Population(s) Served

In order to develop effective treatments for Canavan Disease, and to customize diets for individual Canavan Children, the lab must do a neurological evaluation on each child and also analyze the specific mutation of the Canavan Disease to provide the best level of care for that child.

Dr. Paola Leone is a well-recognized pioneer in gene and cell therapy of Canavan disease. In early 1998, Dr. Leone spearheaded the first clinical gene therapy trial of Canavan Disease in the world with liposome-mediated nonviral gene transfer as well as the first generation of recombinant Adeno-Associated Viral Vector therapeutics in Canavan patients in 2001. As a passionate and tireless advocate of Canavan disease research, Dr. Leone has dedicated her research primarily in developing different therapeutic approaches for treating Canavan disease including gene and stem cell therapy, and pharmacological interventions. The Cell and Gene Therapy Center (CGTC) was founded in 2001 at UMDNJ-School of Medicine, as a combined clinical and basic research facility. CGTC includes an international patient registry of 84 patients with confirmed diagnosis of Canavan Disease. The focus of the Center’s work is to provide clinical support to patients as well as counseling to families afflicted by newly diagnosed patients and provides services such us genetic mutation analyses for rare mutations as well as neuroradiologic assessments and recommendations for pharmacologic therapies. CGTC is a "translational" research facility, which means that discoveries from the laboratory bench are directed to applications in clinical settings. The CGTC also focuses on developing novel therapeutic approaches for the treatment of Canavan Disease. CGTC shares collaborative clinical subcontracts with the Department of Neuroradiology at CHOP (Philadelphia, PA), Department of Neurology, Capital Health Hospital, Pennington, NJ and the Department of Neurogenetics at NYU (NY, NY).

Population(s) Served

We propose to initiate preclinical development of a gene therapeutic targeted to Alzheimer’s
Disease. The World Health Organization’s and Alzheimer’s Disease International’s report on
Dementia (2012) recognizes that the global burden of Alzheimer Disease is forecast to worsen significantly with prevalence predicted to double every 20 years. Interventions that could provide a modest delay in progression would provide a significant reduction in the high level care required later in the disease. This Phase I proposal describes an Adeno-associated viral vector-based metabolic intervention strategy to combat age-related cognitive decline in the mouse model of familial Alzheimer’s disease.
This proposal seeks to test a novel gene-therapy strategy for the treatment of memory dysfunction in an animal model of Alzheimer’s disease (AD). Specifically, this strategy will employ an enzyme that liberates free acetate from an abundant amino acid found within neurons in the brain to supply fuel for energy-generating reactions that support memory circuits. This abundant neuronal molecule, N-acetyl-Laspartic acid (NAA) currently has no known function, bus it associated with neuronal dysfunction in many clinical contexts, including AD. By using engineered gene vectors, this proposal tests the feasibility of ectopically expressing an enzyme in neurons to provide acetate as a fuel for the tricarboxylic acid cycle. This approach offers the potential of a one-time treatment with multi-year protection of cognitive function via the provision of extra fuel to support neuronal network function. If shown to be feasible in this proposal this program will be advanced through definitive preclinical dose ranging and toxicity studies, prior to submission of an IND in support of a clinical trial.

Population(s) Served

Where we work

Awards

Young Investigator Award 1999

UNESCO

Woman of the Year Award 2006

Lions Club, Italy

Foundation of UMDNJ's Excellence in Research Award 2009

University of Medicine and Dentistry of New Jersey

International Rare Disease Day, Hall of Fame 2011

International Rare Diseases Research Consortium

Edward J. III Medical Foundation Award in Biomedical Research 2012

Edward J. III Excellence in Medicine Foundation

Neuro Film Festival Award 2013

American Academy of Neurology

Human Genome Exhibit 2001

Museum of Natural History, New York City

Goals & Strategy

SOURCE: Self-reported by organization

Learn about the organization's key goals, strategies, capabilities, and progress.

Charting impact

Four powerful questions that require reflection about what really matters - results.

Our goals:
1- To treat 20 Canavan children in clinical trials of stem cells, and develop the stem cell application to be translated and used across the neurodegenerative disease spectrum.
2- To treat all Canavan children with anapleurotic therapy. Unlike other therapeutic approaches, which have a relatively long lead time, anapleurotic therapy may represent the shortest route to a disease modifying clinical therapy for Canavan disease.
3- To initiate preclinical development of a gene therapeutic targeted to Alzheimer’s
Disease.

Financials

Canavan Research Foundation, Inc.
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Operations

The people, governance practices, and partners that make the organization tick.

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Canavan Research Foundation, Inc.

Board of directors
as of 3/27/2019
SOURCE: Self-reported by organization
Board co-chair

Dr. Roger Karlin


Board co-chair

Helene Karlin

Christopher Janson, MD

Roger Karlin, MD

Sean W.J. McPhee, PhD

Andrew Freese, MD

Jeremy S. Francis, PhD

Roscoe Brady, MD

Ruben Matalon, MD