The inhibitor Zavesca ® (miglustat), approved in Europe and the United States for the treatment of Gaucher Type 1, is in clinical trials in persons affected with Niemann-Pick Type C, and in children with juvenile G_M2, Tay-Sachs and Sandhoff and in children under the age of 2 with Tay-Sachs and Sandhoff disease.

From the Lysosomal Diseases of New Zealand Newsletter

This is just a part of an article written by a conference attendee in 2006.  It summarizes a panel discussion on Zavesca (Miglustat)

NTSAD 28th Family Conference April 6-9 2006

Alexandria, Virginia USA

Gina Murray was supported by the Lottery Grants board to attend her first ever National Tay Sachs Conference. Gina’s report follows.

*Medical and Research Update:

(The NIH/NINDS Workshop: Glycosphingolipids in Health and Disease was held 5-6 April at the same venue and we were fortunate to have the following presenters from that workshop.)

* BruceA. Bunnell PhD, (overview of current research efforts):

Caloric restriction along with gene therapy or stem cell therapy shows a lot of promise in delaying progression in neurodegenerative diseases. Appears to target CNS inflammation associated with ganglioside disorders.

* Paula Gregory PhD, (ABC’s of Genetics)

* Edwin Kolodny MD, Professor of Neurology NYU School of Medicine,

* David Korosec, RN,

* Gustavo Maegawa MD,

* Cynthia Tiff MD, PhD.

This session primarily focused on an update of Tay-Sachs and Sandhoff clinical trials of Zavesca (Migulstat).

The end point for these trials was stabilization in muscle strength but there was progressive deterioration in all patients from both the New York and Cleveland trials for LOTS. This is not to say that there wasn’t improvement in other areas. Anecdotal evidence showed an improvement in speech and stabilization of psychiatric symptoms.

Neutral study results thus far don’t sufficiently support in adult patients with LOTS but might benefit Early Onset and/or Juvenile Onset Tay-Sachs.

This session allowed for plenty of questions and informal discussion from the floor.

*Navigating off-label use of Zavesca:

A panel of parents who have successfully navigated the system for off-label use of Zavesca for their child (all pre school age). All of these children have the infantile form of the disease. Although Zavesca is not available in New Zealand as yet  I believe it was important to hear what these parents had to go through in order to get this drug for their children.

Some of the children are still on Zavesca and some have been taken off after several weeks mainly due to side effects such as severe diahorrea (which was also a huge problem for a lot of the teenagers and adults using Zavesca both on and off label).

These parents were unsure how Zavesca was working for their children as the nature of the disease is one of deterioration and was it Zavesca that was perhaps slowing things up or was it just the way the disease was progressing. There is really no answer to this.

http://www.ldnz.org.nz/newsletters/gmurray_-_national_tay_sachs_06

**Rachaeli's parents were on this panel.  They were the only ones in favor of taking Zavesca

Back to top