|
A collaborative effort among family members, a stem cell scientist, and a manufacturer of cells for use in research, said on February 19 it plans to push a potential stem cell therapy for spinal muscular atrophy (SMA) toward human clinical trials.
As a first step, the partnership is planning animal studies to be conducted in accordance with FDA regulations to assess the safety of motor neuron progenitors derived from human stem cells after transplantation.
Participating in the effort are Families of Spinal Muscular Atrophy (FSMA), a stem cell scientist at the University of California, Irvine (UCI), and a private firm called California Stem Cell, Inc. (CSC).
SMA is a genetic disorder that is the leading genetic killer of children younger than two years of age.
SMA typically is marked by the degeneration of voluntary muscle movement, including the muscles that control crawling, walking, swallowing or breathing due to the dysfunction or death of motor neurons.
It is a debilitating and often fatal disease for which there is no treatment.
Safety studies are critical in advancing stem cell therapy into human trials for SMA.
High purity human motor neuron populations for use in transplant therapies were developed by CSC and have been used successfully in proof of concept efficacy and preliminary safety studies in the laboratory of Dr. Hans Keirstead at UCI with funding from FSMA.
CSC uses scalable manufacturing protocols to produce the large population of clinical-grade motor neuron progenitors required for the pivotal studies and future human clinical trials.
“This collaboration illustrates the breadth of skills that are required to take a potential treatment from the bench to the bedside,” said Keirstead. “I am confident that we have assembled the right team and the right plan to move this treatment forward with both diligence and speed.”
Studies are being conducted in parallel at both the Keirstead laboratory at UCI and the laboratory of Dr. Douglas Kerr at Johns Hopkins University, with funding from FSMA, to show the benefit of human motor neuron progenitor replacement in animal models of motor neuron disease.
According to the partnership, stem cell therapy for SMA has the potential to replace the motor neurons lost during the disease course.
While other types of therapies have the potential to slow disease progression and possibly increase strength, motor neuron replacement through the use of stem cells is the only means to replace motor neurons once they are gone.
This strategy may be useful for treating multiple disorders such as spinal cord injury, transverse myelitis, and amyotrophic lateral sclerosis (ALS) in addition to spinal muscular atrophy.
Families of SMA is dedicated to developing a treatment and cure for SMA by promoting and supporting research, helping families cope through informational programs and support, and educating the public and the medical community about SMA.
Contact: http://www.curesma.org
Contact: http://www.californiastemcell.com
...
Citation: (Article name, authors,
publication, DOI)
Article or Abstract: (link)
Contact: (link)
Start a free one-week trial
subscription to StemCellResearchNews.com to access
ALL premium
content on this Web site.
Read about research you won't hear about on the nightly news or
on Google.
Click here. (See below for information on why Premium Content is
really worth
it.)
Get the whole story: article citations, links
to further information, links to journal abstract pages, related
articles, researcher contact information, etc.
Or ...
Subscribe
to Stem Cell Lab World, Stem Cell Research News,
Stem Cell Business News for complete access PLUS biweekly
e-mail delivery of a PDF publication you can save on your
computer or print and store in a binder.
FAQ: Why do you charge to read Premium Content ($) articles?
For many years we have provided stem cell-related news and
analysis free of charge on this Web Site, supported by
advertising and subscription income. Unfortunately, in today’s
economy, advertising placements are at an all-time low. That has
forced us (and other publishers) to seek income elsewhere.
Hence, the subscription fees for premium content.
FAQ: What makes Premium Content articles worth the price?
There are seven key reasons:
1) You read about stem cell research you'll never hear about
in the general press, and you'll get the complete article, not just a snippet.
2) Important references are clickable links. (e.g., a
reference to a research institution
or researcher's lab site or more
information about a disease or disorder
are highlighted and clickable as Web links.)
3) Full citations are provided for all journal articles
so you can access the original research.
4) Links to article abstracts are provided.
5) Links to researcher e-mail addresses and lab Web sites
are provided whenever available.
6) Graphic images are provided when available.
7) Links to Related Articles in our database of more than
2,000 stem cell research articles.
Subscribe now!
Click here
to subscribe to a newsletter (including biweekly delivery of PDF
editions PLUS Web site Premium Content) or
click here to
subscribe to the Web site Premium Content only.
 |
