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Thread: Neuralstem Receives FDA Approval To Commence Phase II Stem Cell Trial In Amytrophic L

  1. #1

    Neuralstem Receives FDA Approval To Commence Phase II Stem Cell Trial In Amytrophic L

    I love this small company. It is getting good results, very positive news.


    Neuralstem Receives FDA Approval To Commence Phase II Stem Cell Trial In Amytrophic Lateral Sclerosis
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    Trial Expands to Two Centers; Approval Includes Significant Increase in Dosing

    ROCKVILLE, Md., April 17, 2013 /PRNewswire/ -- Neuralstem, Inc. (NYSE MKT: CUR) announced that it has received approval from the Food and Drug Administration (FDA) to commence a Phase II trial using NSI-566 spinal cord-derived human neural stem cells in the treatment of amyotrophic lateral sclerosis (ALS or Lou Gehrig 's disease). This Phase II dose escalation and safety trial will expand to two centers, Emory University Hospital in Atlanta, Georgia, where Phase I was recently completed, and ALS Clinic at theUniversity of Michigan Health System, in Ann Arbor, Michigan, subject to approval by the Institutional Review Board at each institution. The trial is designed to treat up to 15 patients, in five different dosing cohorts. All of the patients will be ambulatory and reside within close geographic proximity to the research center where they will participate. The first 12 patients will receive injections in the cervical region of the spinal cord only, where the stem cells could help preserve breathing function. The final three patients will receive both cervical and lumbar injections.

    "The aim of this Phase II trial is to obtain the maximum tolerated dose using the same route of administration as in Phase I, which was through direct injections into the gray matter of the spinal cord," said Karl Johe , PhD, Neuralstem Chairman and Chief Scientific Officer. "As a result of the excellent safety and tolerability demonstrated in Phase I, we will be able to proceed more aggressively in Phase II. In Phase I, we started with just five injection sites per patient, and advanced to a maximum of 15 injections of 100,000 cells each. In Phase II, we will advance up to a maximum of 40 injections, and 400,000 cells per injection based on safety.


    more...

    http://www.fiercebiotech.com/press-r...-fda-approval-

  2. #2
    Dr. Young, this would be a question for you (or anyone else who knows): what is the transferability of safety data for different conditions when the procedure itself is the same or similar? They say here they are trying to determine the maximum tolerable dose of cells and they're injecting the cells into the spinal cord; does it matter (I'm guessing it very well may) which condition they're treating? Do potential interactions between the condition and the treatment make applying safety data from one treatment in one condition insufficient in judging the safety of the same therapy treating another condition? Or is there simply not much precedent here? Thanks as always...

  3. #3
    ay2012,

    Joy Cavagnaro was answering some of these indepth questions about dose and safety on cellular therapies in her preparations for the CIRM grantees that are moving toward taking their work to the FDA. Her experience and insights were very interesting on figuring out dose for cellular therapy. Here's a link where you can watch them discuss how best to work out the solutions that the FDA would find acceptable and the various rational. Her video presentations are #3 and #4 if you're interested in watching her.
    http://spinalcordresearchandadvocacy...cirm-workshop/

  4. #4
    Quote Originally Posted by GRAMMY View Post
    ay2012,

    Joy Cavagnaro was answering some of these indepth questions about dose and safety on cellular therapies in her preparations for the CIRM grantees that are moving toward taking their work to the FDA. Her experience and insights were very interesting on figuring out dose for cellular therapy. Here's a link where you can watch them discuss how best to work out the solutions that the FDA would find acceptable and the various rational. Her video presentations are #3 and #4 if you're interested in watching her.
    http://spinalcordresearchandadvocacy...cirm-workshop/
    Thank you Grammy!

  5. #5
    Quote Originally Posted by ay2012 View Post
    Thank you Grammy!
    After listening to Joy's explanations...I'm sure you can see that in the Ph ll for ALS they're ramping the cell load to find efficacy and still using their special cannula for delivery. However, one key question in converting over to the SCI patient population would be if they intend for the delivery to be into the gray matter like they're doing for ALS or if they are moving to a different injection location and then they have to decide at what level. It's one of the many differences between "disease" models and sci "injury" models that's difficult to address because they're worlds apart in more ways than one would realize. In other words, does their pre-clinical animal data show better potential efficacy traits in the gray matter or moving to a different location for better regeneration in a sci injury model? They are putting the cells into the gray matter of the sci patients also the same as the ALS patients but the dosing for sci is running behind the ALS patients, but their treatment protocol looks to be about the same.

    If I'm not mistaken, I think Neuralstem may possibly be ready to give a progress report at W2W 2013 in Boston for spinal cord injury Ph 1 and be available to answer more questions as they arise. (Anyway, I'm hoping so.)

    In their Phase 1 SCI trial they said all patients in the study will receive six injections in, or around, the injury site. The first four patients will receive 100,000 cells per injection, the second four patients will receive 200,000 cells per injection. Basically, they're ramping up the dosages in the SCI patients too. All patients will also receive physical therapy post surgery, as well as immunosuppressive therapy, which will be for three months, as tolerated. The trial study period will end six months post-surgery for each patient. The primary objective of the study is to determine the safety and toxicity of human spinal stem cell transplantation for the treatment of paralysis and related symptoms due to chronic spinal cord injury (SCI). The secondary objectives of the study are to evaluate graft survival in the transplant site by MRI, as well as the effectiveness of transient immunosuppression. Additionally, the study will look at exploratory objectives to evaluate the ability of human spinal cord stem cell (HSSC) transplantation to positively affect AIS level, ISNC SCI motor and sensory index scores, bowel and bladder function, pain, UAB IMR scores, SCIM scores, evoked sensory and motor potentials, and electromyogram (EMG).
    Last edited by GRAMMY; 04-19-2013 at 12:39 AM.

  6. #6
    Quote Originally Posted by GRAMMY View Post
    However, one key question in converting over to the SCI patient population would be if they intend for the delivery to be into the gray matter like they're doing for ALS or if they are moving to a different injection location and then they have to decide at what level.
    Yea, that's precisely why I was wondering. At the very least, the location of injection will be different (cervical vs. thoracic spinal cord)....

  7. #7
    This open-label, multi-site study, enrolled up to eight patients with thoracic spinal cord injuries (T2-T12), who have an American Spinal Injury Association (AIS) A level of impairment, between one and two years after injury. AIS A impairment refers to a patient with no motor or sensory function in the relevant segments at and below the injury, and is considered to be complete paralysis. In China they're doing stroke with the NSI-566, and they were preparing for a trial with NSI-566 to treat acute spinal cord injury patients in Korea this last summer. As they begin to create proof-of-principle data in multiple indications like chronic sci, ALS and also stroke with this cell line, they are also creating additional NSI-556 safety data across indications and borders. We'll surely know more as they move into their Ph 2 for SCI since they are treating other indications with the NSI-556.

    In addition, Neuralstem received approval from the FDA to move into the cervical (upper back) stage of the trial in the fall of 2011 for ALS. The first of six patients in the cervical cohorts to receive stem cells was treated on November 18, 2011, which marked the first FDA-approved intraspinal surgical transplantation of stem cells into the cervical region. The trial then advanced to the final cervical cohort of three patients. The FDA approved the return of three patients from earlier cohorts to receive cervical transplants, making them the first to receive stem cell transplantation in both the lower and upper parts of their spinal cord. The first of these was treated in June 2012, and received five stem cell injections into the cervical region of the back, for a total of 15 injections, including the ten lower-back injections previously received. The last patient in the Phase I trial was treated in August 2012.

    In my opinion, this company is working aggressively to bring the NSI-556 line to the bedside for probably both cervical and thoracic injury. We seem to be following the same protocol just behind ALS patients. The ALS cervical trial probably cleared the path for us. Prior to that, nobody wanted to inject the cervical region for fear of damage and breathing being involved for sci.
    Last edited by GRAMMY; 04-19-2013 at 12:50 AM.

  8. #8
    Quote Originally Posted by GRAMMY View Post
    In my opinion, this company is working aggressively to bring the NSI-556 line to the bedside for probably both cervical and thoracic injury. We seem to be following the same protocol just behind ALS patients. The ALS cervical trial probably cleared the path for us. Prior to that, nobody wanted to inject the cervical region for fear of damage and breathing being involved for sci.
    They are running rapidly and from what is rumored are already talking with facilities.
    "2012 saw the company achieve success in all of its Phase I clinical trial objectives," said Karl Johe, Ph.D., Neuralstem's Chairman of the Board and Chief Scientific Officer. "We have been able to demonstrate the safety and tolerability of Neuralstem's novel core technologies, from intraspinal transplantation procedures, to the cells themselves in ALS patients, as well as our NSI-189 neurogenic small molecule drug in healthy volunteers. Additionally, we believe we have seen evidence of a treatment effect in some NSI-566 cell therapy patients over a sustained period of time, as measured by levels of functional recovery and a slowdown in the progression of ALS. In spinal cord injury, a leading peer-reviewed scientific journal, 'CELL,' published compelling evidence that NSI-566 cells can 'bridge the gap' in a severed spinal cord animal model and return functionality. We have recently been approved by the FDA to commence a trial treating chronic spinal cord injury patients.
    http://investor.neuralstem.com/phoen...cle&id=1796715

  9. #9
    Thanks c473s. Here's the link off the clinical trial chart.

    http://www.prnewswire.com/news-relea...186768041.html

  10. #10
    Quote Originally Posted by GRAMMY View Post
    Thanks c473s. Here's the link off the clinical trial chart.

    http://www.prnewswire.com/news-relea...186768041.html
    Thanks !
    And it will still take several years at best to advance after following patients post injection and moving through P2, P3 assuming the data supports advancement from one phase to another.

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