FDA green lights stem cell clinical trial for Lou Gehrig’s disease


Lou Gehrig’s Disease is a really nasty disease. The patient experiences a progressive degeneration of the nervous system that affects the brain and the spinal cord. The progressive degeneration destroys the nerves that help move muscles and causes a gradual loss of the ability to move. It eventually leads to the death of those neurons. Paralysis slowly sets in right before the patient’s eyes. Then other basic abilities slowly leave, like the ability to talk, void the bladder, control bowel movements and so on. It kills its victims slowly and horribly.

Neuralstem, a company in Rockville, Maryland, has received US Food and Drug Administration (FDA) permission to test spinal cord stem cells in twelve patients with Lou Gehrig’s disease (amyotrophic lateral sclerosis).  This approval comes approximately one month after the FDA placed Geron’s planned clinical trial on hold for a second time. Neuralstem’s trial had also previously been placed on hold by the FDA in February before it received the go-ahead in September.

Though both trials involve placing cells into the spinal cord, Neuralstem’s product is made of cultured neural stem cells derived from a single eight-week-old fetus, whereas Geron’s product, intended to treat spinal cord injury, is derived from embryonic stem cells that have been differentiated into precursors of neuron-support cells.

Lucie Bruijn, a scientist at the ALS Association stated that this is the first stem cell approach for Lou Gehrig Disease.  The chief science office at Neuralstem, Karl Johe, says tests of large animal models show that the transplanted neural stem cells are able to protect motor neurons, although, it’s not entirely clear how.  Neuralstem and their collaborators showed in a rat model of Lou Gehrig’s Disease that transplanted cells could develop into interneurons that formed connections with the rats” motor neurons.

Nevertheless, this approved trial will assess safety rather than efficacy. The first few patients selected for the procedure will be those who are no longer able to walk.  Since the injected cells protect rather than replace motor neurons, these sicker patients are less likely to benefit from treatment, but they are also less able to lose function if something goes wrong. Cells will be injected only on one side of the spinal cord in order to minimize the number of injection sites. Only one patient will be treated each month so that researchers can monitor effects over a longer period of time. According to Johe, the goal is to be able to inject cells in both lower and upper regions of the spinal cord in healthier patients and see if the injections can help motor neurons survive.

Other companies using neural cells include ReNeuron, which received permission from UK authorities this January to start clinical trials for stroke. Its cell product is made from genetically modified cultures of neural stem cells, also of fetal origin.

StemCells Inc. is conducting trials in Batten disease, a neurodegenerative disorder that strikes children, and recently received approval for a clinical trial in a similar disease. It also uses neural stem cells from material originally derived from fetuses and has recently published research showing that its cell product delayed some symptoms of the disease by about three weeks.

It is really a shame that fetuses had to die to give us these cells.  I know that people will argue that the mother’s decision to “terminate her pregnancy” had nothing to do with the use of this person’s cells for research, but the fact remains that a doctor probably killed this very young baby and now his or her neural stem cells are being used in clinics.  Is this the way to value the youngest and most valuable members of our society?  Forgive me, but I find this shameful.

There is another experimental treatment that does not need to use dead babies.  It uses mesenchymal stem cells derived from the patients who receive them.  Read more about it here.

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Published by

mburatov

Professor of Biochemistry at Spring Arbor University (SAU) in Spring Arbor, MI. Have been at SAU since 1999. Author of The Stem Cell Epistles. Before that I was a postdoctoral research fellow at the University of Pennsylvania in Philadelphia, PA (1997-1999), and Sussex University, Falmer, UK (1994-1997). I studied Cell and Developmental Biology at UC Irvine (PhD 1994), and Microbiology at UC Davis (MA 1986, BS 1984).