Stem cell clinical trial- has this been posted yet?

[pocuita]

Can someone tell me if this is new info?




September 21, 2009
Neuralstem Receives FDA Approval To Begin First ALS Stem Cell Trial

Neuralstem, Inc. today announced that the U.S. Food and Drug Administration (FDA) has approved its Investigational New Drug (IND) application to commence a Phase I trial to treat Amyotrophic Lateral Sclerosis (ALS or Lou Gehrig’s disease) with its spinal cord stem cells.

The Rockville, MD-based Neuralstem is the first company to commence a stem cell trial to treat ALS. The trial will study the safety of Neuralstem’s cells and the surgical procedures and devices required for multiple injections of Neuralstem’s cells directly into the grey matter of the spinal cord. The FDA’s approval represents a significant step toward delivering regenerative medicine directly to damaged neural cells in humans. ALS affects roughly 30,000 people in the U.S., with about 5,600 new diagnoses per year. ALS is a progressive fatal neurodegenerative disease that affects nerve cells in the brain, leading to the degeneration and death of the motor neurons in the spinal cord that control muscle movement.

“This is an extremely important trial that will test the stem cell approach in ALS in a rigorous way,” said Dr. Lucie Bruijn, senior vice president, Research and Development at The ALS Association. “This is the first stem cell trial in ALS and it will be conducted initially to study its safety for use in humans.”

Neuralstem CEO and President, Richard Garr, stated, “The beginning of our clinical trial program is a major step towards achieving Neuralstem’s goal of treating ALS, a fatal neurodegenerative disease for which currently there is no effective treatment or cure. While this trial aims to primarily establish safety and feasibility data in treating ALS patients, we also hope to be able to measure a slowing down of the ALS degenerative process. This trial will be headed by Dr. Eva L. Feldman, M.D., Ph.D., director of the University of Michigan Health System ALS Clinic and the Program for Neurology Research & Discovery, and Dr. Jonathan Glass, director of the Emory Neuromuscular Laboratory and Director of the Emory ALS Center, world-renowned for their study and treatment of ALS patients. We believe that there is no better team to conduct this study for us,” said Garr. Their participation is subject to formal IRB approval by their institutions.

“We are very excited about this clinical trial,” Feldman said. “This is a major advancement in what still could be a long road to a new and improved treatment for ALS. Feldman also directs the A. Alfred Taubman Medical Research Institute. “In work with animals, these spinal cord stem cells both protected at-risk motor neurons and made connections to the neurons controlling muscles. We don’t want to raise expectations unduly, but we believe these stem cells could produce similar results in patients with ALS,” Dr. Feldman concluded.

The ALS patients will be treated through spinal injections of its patented human neural stem cells.

This first trial, which will primarily evaluate safety of the cells and the surgery procedure, will ultimately consist of 18 ALS patients with varying degrees of the disease. The FDA has approved the first stage of the trial, which consists of 12 patients who will receive five-to-ten stem cell injections in the lumbar area of the spinal cord. The patients will be examined at regular intervals post-surgery, with final review of the data to come about 24 months later.

Neuralstem expects to conduct the trial at Emory University with Dr. Jonathan Glass, M.D., Director of the Emory Neuromuscular Laboratory and Director of the Emory ALS Center, as site Principal Investigator (PI) and with Dr. Nicholas Boulis, M. D. performing the neurosurgery. The overall PI for the ALS trial program is Dr. Eva Feldman, M.D., Ph.D., Director of the University of Michigan Health System ALS Clinic and the Program for Neurology Research & Discovery.

Neuralstem's patented technology enables, for the first time, the ability to produce neural stem cells of the human brain and spinal cord in commercial quantities, and the ability to control the differentiation of these cells into mature, physiologically relevant human neurons and glia. The company is targeting major central nervous system diseases including: Ischemic Spastic Paraplegia, Traumatic Spinal Cord Injury, Huntington’s disease and ALS. Neuralstem plans to initiate a Phase I clinical trial to treat ALS with its stem cells.

Pre-clinical work has shown Neuralstem’s cells to extend the life of rats with ALS (as reported the journal TRANSPLANTATION, October 16, 2006, in collaboration with Johns Hopkins University researchers), and also reversed paralysis in rats with Ischemic Spastic Paraplegia, (as reported in NEUROSCIENCE, June 29, 2007, in collaboration with researchers at University of California San Diego).

[pocuita]

My dad is being treated at uofm for ALS. Can he get in this trial?

This looks like promising news!

[psiep]

Looking forward to hearing more about this.

[glenn gavanaugh]

finally. awesome news. crossing my fingers as tightly as possible that this is going to prove fruitful. please God, anything at this point is a Success.

[Blackpool]

I would love to go on this trail.We should all be able to.lol

[rocmg]

go-go neuralstem! the only unfortunate part is the results will not be know for 2 years... but i believe Neuralstem are taking their operation to Taiwan also... so maybe people will have the option of going there for treatment? i can't help sounding naive when i'm trying my best to be positive about ALS!

[brooksea]

My husband and I are awaiting answer back from the research nurse re this trial at Emory. Will let y'all know the details and the requirements to participate.

[Big Mike]

This is very exciting! First Brainstorm and now Neuralstem! In response to what rocmg said, yes, the Neuralstem trials in Taiwan will start next year but will go much faster than here in the states. If all goes well with the trials, treatments will be available over there in a few years. I know that's still a ways off, but it's something to hang our hats on. Don't forget about the KNS drug as well going into stage 3 of the clincial trial process next year. Let's keep our fingers crossed!

[brooksea]

"On September 21st, 2009 Neuralstem , Inc (Stem Cell Research at Neuralstem (http://www.neuralstem.com)) announced FDA approval for a phase I trial of spinal cord derived stem cells as a therapeutic intervention for patients with ALS. Now that we have approval from the FDA, our proposal will be submitted to Emory’s Human Investigations Committee for approval. This process to ensure the safety for our patients will take several weeks or possibly months, and we will keep this site updated with our progress. At this time, we do not have a date when we plan to release details of the proposed trial, including selection of participants."


Jonathan D. Glass, MD Director, Emory ALS Center

[Blackpool]

Pals can't sit around for long please make haste.

[Erica]

To CJ:
CJ, Do you regularly go to Emory's ALS clinic, or just contacted them re: this trial?
THX.

[brooksea]

Erica,

Emory is where we go to clinic. We normally see Dr. Glass.

[brooksea]

This was posted at ALSTDI by steelgraham:

"There's an interesting piece about this announcement in Nature."

Here is an excerpt from the article:

However, Johe emphasized that the upcoming 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. Because the injected cells protect rather than replace motor neurons, these sicker patients are less likely to benefit from treatment, but they are 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 injections into the spinal cord. Only one patient will be injected each month, so that researchers can monitor for effects over a longer period. Eventually, Johe says, 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 keep motor neurons healthy.

I fail to see the logic in the above and if true, my husband would not qualify to participate.

[Erica]

CJ,
Thank you for your reply...
It's true that phase I trial only assess the safety; going in to phase 2 and 3 that where they looking at the efficacy.
I don't think it's necessary that phase I will be restricted to pts unable to move.
Lets hope and pray that your husband will get accepted. Erica

P.S. CJ, was your husband onset considered Limb, or bulbar? I wonder if trial Inclusion criteria might be restricted to limb onset.

[crystalkk]

CJ,

I agree about failing to see the logic also.... Hopefully this is not true and your hubby will get in the trial.
My fingers and toes are crossed.......

[rocmg]

a prayer will have been answered for whatever PALS is chosen to go first...

i suppose they will be choosing patients who are in the latter stages of disease -- if death is delayed for 2 years (length of study time?), then the researchers and doctors might be able to deduce that the stem cells have played a part in that..

[brooksea]

It seems they may be looking for those pALS that have rapidly progressed with their disease and therefore if the experiment goes wrong, "no harm done." My husband and I spoke about this and he still wants to participate. I, however would like to wait for further trials.

To answer your question Erica: My husband started with bulbar and hand symptoms simultaneously.

[pocuita]

a prayer will have been answered for whatever PALS is chosen to go first...

i suppose they will be choosing patients who are in the latter stages of disease -- if death is delayed for 2 years (length of study time?), then the researchers and doctors might be able to deduce that the stem cells have played a part in that..

I am praying this works!!

My Father was dx 4/09 he went from walking to needing a feeding tube:sad:
I pray they stop the progression!

[Big Mike]

a prayer will have been answered for whatever PALS is chosen to go first...

i suppose they will be choosing patients who are in the latter stages of disease -- if death is delayed for 2 years (length of study time?), then the researchers and doctors might be able to deduce that the stem cells have played a part in that..

Yes, but if some of the participants are on a trache and vent, then time to death will be more indefinite.

[pocuita]

My father had a visit at UofM yesterday and they said he would be a perfect candidate for this trial. They are considering him. They are accepting 12 patients.

They also talked about potential risks. My mom did not have time to tell me exactly what they are, just that it could be bad.

I will keep you updated.


Take care
Jen

[hopingforcure]

I will be very interested to hear more about your father and the trial. Thank you for the post, this forum allows us so much avaliability to share such important and helpful information.

[Blackpool]

Good luck to your Dad, keep us updated.Maybe this is the break through.

[flyingaseagles]

I hope they do come through with stem cell trail. That would be so wonderful !!!

[gramma]

My father had a visit at UofM yesterday and they said he would be a perfect candidate for this trial. They are considering him. They are accepting 12 patients.

They also talked about potential risks. My mom did not have time to tell me exactly what they are, just that it could be bad.

I will keep you updated.


Take care
Jen

Hi,

May I ask who would be the best person to contact at U of M regarding the trial?

Thanks.

[rocmg]

My father had a visit at UofM yesterday and they said he would be a perfect candidate for this trial. They are considering him. They are accepting 12 patients.

They also talked about potential risks. My mom did not have time to tell me exactly what they are, just that it could be bad.

I will keep you updated.


Take care
Jen


Jen,

Please keep us updated as to the requirements of the study and if your dad is successful in being accepted. I hope he is.

Please God, something good will come of this. we all -- PALS and CALS -- desperately need it.

R.

[pocuita]

Hi,

May I ask who would be the best person to contact at U of M regarding the trial?

Thanks.

I will ask my mom. I know they talk to Anne Marie and my dad had scheduled an appointment with the clinic, I am not sure who they spoke with.

[Cassandra]

The following is from the Emory site:



This trial will deliver neural progenitor cells to the spinal cords of ALS patients. These cells have been demonstrated to differentiate into neurons and glial cells. Their therapeutic impact in animal models is attributed to the ability of the neuronal progeny to secrete inhibitory neurotransmitters, and the ability of the glial cells to secrete trophic factors and scavenge excitatory amino acids.

The trial will initially recruit nonambulatory patients to undergo lumbar spinal cord transplants. If these transplants prove safe, ambulatory patients will be recruited for lumbar transplants. Finally, if these patients have an acceptable level of complications, we will recruit ambulatory patients for cervical spinal cord transplants, with the goal of preserving respiratory and upper extremity function.

[KinzaDAF]

Is it going to be two years then, until this phase is over and ambulatory patients will be considered? Sigh. HURRY!

Debbie

[pocuita]

I spoke with my mom about this again. She is being told it could be a year before they even start the treatment!! Sadly, I think it will be too late for my dad to be apart of it, because he is progressing very fast.

It is so frustrating to be so close but yet so far!!

Praying the speed this trial up for everyone!!

[FindlayPALS]

My husband has been in contact with U of M and Emory regarding this trial. He is, of course, very interested. He was told it will probably be several months before necessary approvals are obtained to begin. He was also told they are most interested in candidates living in the Atlanta area, since frequent visits and close monitoring will be required. Emory will be deciding who participates. Further criteria was given as "significant leg weakness but with good breathing (a forced vital capacity of greater than 60%)." They're expecting it to take 2 years+ for completion of this first phase.

As a side note, my husband is thinking about the ceftriaxone trial and he was told by both parties that would not affect his ability to participate in the stem-cell trial at a later date.

Hope this info is helpful.

[brooksea]

Neuralstem Receives Approval To Commence First ALS Stem Cell Trial At Emory ALS Center (http://www.medicalnewstoday.com/articles/174522.php)

We were told by the research team that you had to be in advanced stages and unable to walk in order to participate in the trial. This is because the trial is so risky and they do not want to cause further damage if something goes wrong. Emory (as of Friday) has one enrolled in the trial thus far.

[*ENV*]

While it would take over a year to reinnervate the toes, the thigh muscles could probably be reached in a few months. If some resumption of function was observed and side effects mild, I would expect to see the schedule accelerated.

[CRF8]

I thought they were doing a quicker paced trial in another country as well.

[*ENV*]

I think Taiwan, but that was for another condition (spinal cord injury).

[Ratman55]

I've been watching/reading the thread with great interest recalling my older sister's participation in a clinical trial some 20 years ago. Her MS was slowed significantly and she eventually rec the meds when the study concluded. I know this is not the same; stem cell is hot, hot pharmacologically and politically. But hope is always good! No scripture from any religion has ever told us to give up hope! I just retired, and with my progression slow for now, the letters (to fund stemcell, etc) are flying! I say, "Let's Roll!"
G_d Bless all PALS!

JK

[Al]

We are allowed to say God Bless all Pals JK.

AL.

[pmbenb83]

CJ,
Thanks for sharing this article. I haven't felt this hopeful in a long time. I pray that the phase I patients have success and no harm comes to them. I know it is a gamble and I appreciate them taking the risk not only for themselves but for everyone who has been stricken with this disease.

Thanks again.
Pam B in Va

[_Saj_]

I think Taiwan, but that was for another condition (spinal cord injury).

I think they are investigating ALS in Taiwan too

[Big Mike]

I think they are investigating ALS in Taiwan too

From what I understand, Neuralstem will conduct ALS trials at a Chinese hospital in Taiwan after the stroke trials start using their product (haven't heard anything about spinal cord injury trials) and some safety data comes in from the phase I study at Emory University. Richard Garr, CEO of Neuralstem, communicated by email to me that he could not say with any certainty when that will be. My guess would be sometime in 2011. However, if all goes well, the trials will take only about 18-24 months to complete in Taiwan.

[glenn gavanaugh]

Great to see that this trial is finally underway.

[Big Mike]

I believe the Emory trial starts January 20th. Finally!

[carrlos]

Neuralstem Announces First Patient Treated in ALS Stem Cell Trial

Download image ROCKVILLE, Md., Jan. 21 /PRNewswire-FirstCall/ --
Neuralstem, Inc. (NYSE Amex: CUR) announced that the first ALS patient
was treated with its spinal cord stem cells yesterday at the Emory ALS
Center at Emory University, in Atlanta, GA. A total of up to 18
patients is planned to be treated in this first U.S. clinical trial to
evaluate human neural stem cells for the treatment of ALS (Amyotrophic
Lateral Sclerosis, or Lou Gehrig's disease). ALS affects roughly
30,000 people in the U.S., with about 5,600 new diagnoses per year,
according to the ALS Association.

"The treatment of the first patient with our spinal cord stem cells
represents a significant milestone for the Company," said Neuralstem
President and CEO, Richard Garr.

"Our scientists have worked tirelessly to bring our discovery of
adherent neural stem cells from the bench to the bedside," said Dr.
Karl Johe, Chairman of the Board and Chief Scientific Officer at
Neuralstem, and the inventor of the technology. "This trial aims to
establish the safety and feasibility of using our cells to treat ALS.
For now, we are focused on the safe and speedy recovery of the first
patient, and we wish to thank him and his family for their courageous
participation in the trial."

About the Trial

This Phase I trial, which will primarily evaluate safety of the cells
and the surgery procedure, is designed to enroll up to 18 ALS
patients, at varying stages of the disease progression. They will
receive spinal injections of Neuralstem's patented human neural stem
cells as a one-time treatment. The FDA has approved the first stage of
the trial, which consists of 12 patients who will receive five-to-ten
stem cell injections in the lumbar area of the spinal cord. The
patients will be examined at regular intervals post-surgery, with
final review of the data to come about 24 months later.

The overall Principal Investigator (PI) for the Neuralstem ALS trial
program is Dr. Eva Feldman, M.D., Ph.D., Director of the University of
Michigan Health System ALS Clinic and the Program for Neurology
Research & Discovery.

The Emory ALS Center has posted the relevant trial information for
patients on its website at
Emory ALS Center: ALS Research (http://www.neurology.emory.edu/ALS/Stem%20Cell.html).

About Neuralstem, Inc.

Neuralstem's patented technology enables, for the first time, the
ability to produce neural stem cells of the human brain and spinal
cord in commercial quantities, and the ability to control the
differentiation of these cells into mature, physiologically relevant
human neurons and glia. The company is targeting major central nervous
system diseases including: Ischemic Spastic Paraplegia, Traumatic
Spinal Cord Injury, Huntington's disease and Amyotrophic Lateral
Sclerosis (ALS), often referred to as Lou Gehrig's disease. ALS is a
progressive fatal neurodegenerative disease that affects nerve cells
in the brain, leading to the degeneration and death of the motor
neurons in the spinal cord that control muscle movement. Pre-clinical
work has shown that Neuralstem's cells extended the life of rats with
ALS (as reported in the journal TRANSPLANTATION, October 16, 2006, in
collaboration with Johns Hopkins University researchers), and also
reversed paralysis in rats with Ischemic Spastic Paraplegia, (as
reported in NEUROSCIENCE, June 29, 2007, in collaboration with
researchers at University of California San Diego).

Cautionary Statement Regarding Forward Looking Information

This news release may contain forward-looking statements made pursuant
to the "safe harbor" provisions of the Private Securities Litigation
Reform Act of 1995. Investors are cautioned that such forward-looking
statements in this press release regarding potential applications of
Neuralstem's technologies constitute forward-looking statements that
involve risks and uncertainties, including, without limitation, risks
inherent in the development and commercialization of potential
products, uncertainty of clinical trial results or regulatory
approvals or clearances, need for future capital, dependence upon
collaborators and maintenance of our intellectual property rights.
Actual results may differ materially from the results anticipated in
these forward-looking statements. Additional information on potential
factors that could affect our results and other risks and
uncertainties are detailed from time to time in Neuralstem's periodic
reports, including the annual report on Form 10-K for the year ended
December 31, 2008 and the quarterly report on form 10-Q for the period
ended September 30, 2009.


SOURCE Neuralstem, Inc.

[*ENV*]

First U.S. stem cells transplanted into spinal cord - CNN.com (http://www.cnn.com/2010/HEALTH/01/21/stem.cell.spine/)

[easbah1]

this is amazing. does anyone know any of the participants so we can keep up w/ the results?

[carrlos]

this is amazing. does anyone know any of the participants so we can keep up w/ the results?

I think there has been only 1 person so far, they are still recruiting. This will be a fairly small group (18 persons, if i remember correctly)

I agree, this would be nice to see if the procedure has any effect on the person(s). But I guess they want to make sure patients cannot be identified, as these persons would be flooded with requests from PALS (which is understandable) but also from financial speculators, as Neuralstem is publicly traded company.

[_Saj_]

If the target of the trial was testing the security of the procedure they'll just publish about the security of the procedure... I don't think they will go too long about the general state of the patient...

(sorry for my english)