Wednesday, June 26, 2013

CIRM’s first Google Hangout to focus on ALS

We'd like to welcome our journalism fellow Rina Shaikh-Lesko, who will be helping us out this summer writing about progress in the stem cell field. This is her inaugural blog entry.

We’re about to kick off our first ever Google Hangout on July 3 at noon. Google’s foray into online video conferencing makes interacting with fellow participants easier than traditional webinar formats. CIRM grantees and patient advocates will be talking about progress in finding a stem cell-based therapy for Amyotrophic Lateral Sclerosis, or ALS, a devastating neurodegenerative disease.

On hand to provide an update on stem cell research into ALS and answer questions about the disease will be CIRM grantees Lawrence Goldstein of the University of California, San Diego, and Clive Svendsen of the Cedar Sinai Medical Center in Los Angeles, as well as ALS patient advocate and CIRM governing board member, Diane Winokur. Have a question for our panel? Send it to or leave your question as a comment on this post. You can also post questions via Twitter to @cirmnews, or wait and ask during the Hangout.

ALS, sometimes called Lou Gehrig’s Disease, occurs when the nerve cells that control muscles, or motor neurons, die off for reasons scientists don’t entirely understand. The muscles they were connected to eventually wither away, leaving patients paralyzed. Most people who are diagnosed die within four years. According to the ALS Association, about 30,000 people in the U.S. have ALS. Stem cell researchers hope to develop an experimental treatment within a few years.

Goldstein leads a team working to mature stems cells into a kind of cell that normally lives near motor neurons, called astrocytes. They plan to transplant these astrocytes into people with the disease where the cells could protect neurons from further damage from ALS. This video explains his team’s research and how ALS has affected one family.


Svendson’s team is working on a way to modify neuronal stem cells so they excrete a protein that can protect neurons. Those modified stem cells can be transplanted into a person’s brain or spinal cord to slow down the progression of ALS.

Winokur has been a fierce advocate for families coping with ALS and other neurodegenerative diseases since her own family was affected nearly two decades ago. Her youngest son, Douglas, was diagnosed with ALS in 1995 and died two years later in 1997. Her oldest son, Hugh, was diagnosed in 2005 and died in 2010.

You can register for our inaugural CIRM Google Hangout at this link. Future Google Hangouts will spotlight other diseases CIRM researchers are working on. Watch this blog and our Google+ page for updates. In the meantime, put our first one, July 3 at noon, on your calendar and come join the discussion.



  1. Open letter to the FDA featured in yesterdays edition of POLITICO

    When you're facing a loaded gun, what's the difference?

  2. Doctor claims breakthrough in race for spinal cord injury ‘cure’

    A leading researcher into severe spinal cord injuries on Wednesday said trials for stem-cell therapy showed groundbreaking results in giving immobile patients the ability to walk again.

    After progress in a second round of tests using stem cells to regrow nerve fibres, the China Spinal Cord Injury Network (ChinaSCINet) has applied for regulatory approval in China for a third and final phase, which it hopes to start in the autumn.

    “This will convince the doctors of the world that they do not need to tell patients ‘you will never walk again’,” US-based doctor Wise Young, chief executive officer of ChinaSCINet, told AFP.

    The treatment involves injecting umbilical cord blood mononuclear cells into patients’ damaged spines to help regenerate nerves, while lithium is used to promote the growth of the nerve fibres.

    Each component of the combination therapy will be tested in the third phase, which Young said would involve 120 patients in China and another 120 across India, Norway and the United States.

    “If the phase three trial is successful, we should have achieved worldwide regulatory approval by the beginning to the middle of 2015,” he said.

    ChinaSCINet, a non-profit organisation that calls itself the world’s largest clinical trial network for spinal cord therapies, was established in Hong Kong in 2005.

    1. That story about Wise Young claiming regeneration was talked about by Sam Maddox on the CDRF web

  3. June 26, 2013

    FDA Granted Fast Track Designation for Genervon's Breakthrough Biotechnology Multi-Target ALS Drug GM604 Expediting Drug Approval Process

    FDA approved clinical trial with Fast Track designation for ALS increases hope for victims of this heart-wrenching disease. GM604 efficacy against ALS involves 12 pathways and over 80 ALS related genes.

    PASADENA, Calif.--(BUSINESS WIRE)--In a huge victory for ALS sufferers, FDA granted Genervon Biopharmaceuticals a fast track designation for its first in class highly effective multi-target master regulator biotechnology drug GM604 for ALS. FDA said it met the necessary criteria.

    Genervon has received many inquiries from ALS patients interested to participate in the FDA approved IND GM604 clinical trial for ALS ( NCT01854294). GM604's mechanisms of action against ALS involve 12 pathways and up to 22 biological processes. It modulates over 80 specific ALS related genes interactively, systemically and dynamically.

    In order to stimulate the diseased and compromised axonal transport system and delay ALS onset and death of neurons, GM604 regulates 25 genes in this pathway alone. Further scientific details and informative videos can be found at

    In the 1990s, Genervon hypothesized that neurological and neurodegenerative diseases involve the interplay of multiple targets and processes in an interactive dynamic mechanism/network. Most researchers/experts in the field have now confirmed that ALS is a complex, multiple targets and multi-system neurodegenerative disorder. That is one of the main reasons why single target drugs have uniformly failed in previous clinical trials.

    Genervon scientists using breakthrough proprietary biotechnologies discovered GM604 which is an endogenous, multiple target, master regulator signaling peptide. Highly expressed in week 9 of human embryonic/fetal gestation, GM604 plays a significant role in nervous system development, protection and correction.

    The impressive effects of GM604 are consistent with the role of a master regulator of the nervous system in embryonic development. Because ALS has many diverse forms with different pathogeneses, previous single target drug trials tried to restrict the enrollment to a small selective uniform segment of patients. Since GM604 is a multiple target biotechnology drug, able to control multiple ALS genes through multiple pathways, Genervon's FDA approved fast track clinical trial is broadly inclusive.

    FDA's fast track designation will expedite drug approval process to treat ALS.

    Genervon Biopharmaceuticals is an innovative, clinical stage biotechonolgy company providing breakthrough biological drugs for some of the world's critical unmet medical needs.

    1. Christopher & Dana Reeve foundations blogger Sam Maddox did a story on this. China SCI Study: Premature Mid-Trial Speculation