In its Friday press release, Geron, a California-based biotech, announced the FDA’s clearance to proceed with its embryonic stem cell trial for spinal cord injury. This will be the first clinical trial to test the use of embryonic stem cells in human patients, and it will have widespread implications for many other common and rare diseases, including Canavan disease.
“We are pleased with the FDA’s decision to allow our planned clinical trial of GRNOPC1 in spinal cord injury to proceed,” said Thomas B. Okarma, Ph.D., M.D., Geron’s president and CEO. “Our goals for the application of GRNOPC1 in subacute spinal cord injury are unchanged — to achieve restoration of spinal cord function by the injection of hESC-derived oligodendrocyte progenitor cells directly into the lesion site of the patient’s injured spinal cord. Additionally, we are now formally exploring the utility of GRNOPC1 in other degenerative CNS disorders including Alzheimer’s, multiple sclerosis and Canavan disease.”
Yes, you read that correctly. Canavan disease. Geron has already provided their stem cell line to lead Canavan researcher Dr. Paola Leone, and she is currently in the process of testing the cells on a Canavan mouse model. Furthermore, the press release goes on to talk specifically about Canavan and Dr. Leone.

Canavan Disease: Canavan disease is a fatal neurological disorder that belongs to a group of genetic disorders called leukodystrophies, characterized by the abnormal development or degeneration of myelin. Symptoms of Canavan disease present in the first six months of life and death usually occurs at 3 — 10 years of age. GRNOPC1 is being tested in a rodent model of Canavan disease in collaboration with Dr. Paola Leone, Director of the Cell and Gene Therapy Center, at the University of Medicine and Dentistry of New Jersey.

This is tremendous for our community, both because of the attention it is helping garner for Canavan, a rare disease which is listed in this article right next to more commonly known diseases, such as MS and Alzheimers, and because of the formal recognition of Canavan as a good model on which to test therapies for other white matter disease. We are all hopeful for this upcoming trial and excited that the years of fighting to have the stem cell ban lifted are at an end. If this trial is successful, the possibilities are endless.
For example, here is an explanation and visualization of the results of the animal testing:

And here are videos of the actual rats in the lab:

While these circumstances were highly controlled and we should not necessarily expect the same results in humans, we can certainly hope! I think that is the key word of this new development. Hope. When we sent this press release out to all our families and sister organizations on Friday, hope was the word which came up repeatedly in almost every reply we received. What a tremendous distance we have come in the past fourteen years since Jacob was diagnosed.
So, thank you, Geron, for fueling our hope that one day we will have truly effective treatments for these devastating disease.
(This article was originally posted at the Jacob’s Cure blog: A Blog Against Canavan Disease.)