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Tay-Sachs, Canavan, GM1 and Sandhoff diseases

Canavan Trial Updates

Update from Aspa


On Wednesday, the 28th of August, Canavan Foundation, Canavan Research Illinois and NTSAD met with Aspa Therapeutics to discuss updates on their gene therapy program.  Aspa reported they have been in discussions with FDA and that they remain on track for a gene therapy treatment trial to be open for enrollment in 2020.  Agreement has been reached on the preclinical requirements, and manufacturing is on schedule.  

Aspa commented that throughout the exchange with FDA, the importance of the natural history study was emphasized: Without strong natural history data, Aspa may be asked to run a placebo controlled study, which could result in a delay in getting the therapy to many patients.  Aspa also made the point that a strong study means not only rigorous data collection but also necessitates enrollment of enough patients to be considered a robust comparator group – Aspa estimated that a total of 30 enrolled patients or more may be required, depending on ages and amount of data available for each patient.  On the update call, Aspa referenced the recent approval of Zolgensma (a gene therapy approved for the treatment of patients with SMA) as a positive example of how a natural history study supported a clinical trial with no placebo group and a smooth path to approval.  These interactions with FDA have confirmed the importance of the natural history study to the Canavan community.

Aspa plans to give us further updates as preparations for the gene therapy clinical trial proceed.

To enroll in the natural history study, CANinform, a family will need their child’s medical record.  Aspa is providing a free service to families to collect the medical record.  15 families have signed up for record retrieval so far.   For those families who are able to travel to the clinical site, Aspa is providing travel support.  It is important for families to sign up soon as it can take some time to obtain all records.  Families who have lost a loved one are also encouraged to participate.  To sign up for this service, please call 1-833-764-2267 or This email address is being protected from spambots. You need JavaScript enabled to view it.

Canavan Updates

What is Canavan?

Canavan is a progressive neurological genetic disorder caused by the absence of a vital enzyme known as aspartoacylase (ASPA). ASPA breaks N-acetylaspartate acid (NAA) into building blocks essential for building myelin. Myelin is a fatty membrane (also known as white matter) that forms a proactive coating around each nerve ensuring the nerve functions properly.  It is one of over 50 genetically inherited disorders known as Leukodystrophies Diseases.

What about research?

There are currently two gene therapy programs in development - Aspa Therapeutics licensed Dr. Guangping Gao's Canavan gene therapy work from the University of Massachusetts Medical School in Worcester, Massachusetts. They are currently working on a natural history study to have an even deeper understanding of the progression of Canavan; which will lead to establishing better endpoints in the upcoming clinical trial. The other program is at Rowan University under the purview of Paola Leone, PhD where she is exploring gene therapy using a different approach. More information is pending.