I think investors are really idiots. They do not understand the value of a big new like this. When a fool analist will explain the power of stemcells to stop in the 1st 2 years this terrible desease all the idiots will buy . Hope too late for them IMHO.
Sentiment: Strong Buy
One scientist like Dr Eva Feldman does not fly to Europe without having in hand positive news about trial IMHO
Sentiment: Strong Buy
The day after UM and Feldman happily announced Phase 2 trials would commence, loud and strident protesters showed up at UM to bloviate against the use of embryonic stem cells.
They were on TV, they were on the radio. Never mind that the embryo they were concerned about died 11 years ago and that this is a line of cells gathered legally in a process that obeyed all state and federal rules and is finally giving hope to people who no longer are sure their disease is a death sentence.
"I don't think the protestors understand," said Harada. "An embryo dying is a one-time tragedy, I know that. But this is a way to turn it into a gift. And it's a gift that's done so much good. That's proved to be life extending."
Harada was one of three patients who got two rounds of injections, the second last August. Researchers monitored all patients for side effects, the trials proved to be safe and last month, the U.S. Food and Drug Administration gave its blessing for Phase 2 trials, to begin later this year in Ann Arbor and at Emory.
Signs of Harada's ALS diminished noticeably after his first injection, and the improvement after his second injection was even more noticeable.
He hasn't used his canes in months, his strong grip has returned, he easily walks upstairs to kiss his kids goodnight. On Oct. 20, he was even able to do a 2.5-mile fundraising walk in Atlanta to fight ALS.
"If the walk had been in July, I wouldn't have attempted it," he said. "After a third of a mile I would have been done. I would have sat down and said, `Someone come pick me up in a car.' "
Harada still has ALS. He still knows the likely prognosis is death. But there's hope the prognosis of death won't always accompany the diagnosis, now that there's clearly some mechanism for improvement that researchers need to understand and refine.
"We've got to turn Lou Gehrig's disease into Lou Gehrig's chronic illness," he told me last summer.
Today, Harada told me nearly all the improvement that happened after his last injection is still evident. Wednesday, he underwent his usual round of post-injection testing at Emory. "I've been doing great and feeling great. Just now, the left leg showed a little bit of weakness returning, but I'm still so much better than I was before the surgeries. It's the first time, since August, they've noticed any slight weakness.
"It's clear from the data that the injections reversed my symptoms and slowed down the progression of the disease. I've received a blessing. I almost forget I have ALS. I don't have the constant reminder of having to use the canes. Now, I don't think about ALS every day. Every couple of days something happens and I think, `Oh, yeah, I have ALS.' "
When Feldman told me the good news in April that the FDA had given its blessing for Phase 2 trials, she said Harada would be welcome to apply for another round of injections.
And Boulis briefly told him the same thing in Atlanta. But he and Feldman had overlooked an important detail: The trial protocol calls for patients who have been diagnosed within a certain window of time. Harada had been recently diagnosed when he got his first injection, and the thought, based on how well he did, is that those more recently diagnosed will show more dramatic results.
Alas, the irony is that based on his success, the change in test criteria now excludes him from further participation.
"I'd be intellectually dishonest if I said I wasn't disappointed, but I'm still the biggest cheerleader for the trials," he said. "If they can get good results and get to market, I might still be able to take advantage
You can understand Ted Harada being more than a little cheesed off when knee-jerk protesters start whining about embryonic stem cells and how it's against God's wishes and all that is moral and right to use them in the name of science.
But Harada is too busy still reveling in the seemingly miraculous improvement in his symptoms of ALS to be mad at anyone. Nothing is going to wipe that smile off his face.
Here's the deal:
Harada, 40, is a former manager at FedEx who first noticed symptoms of ALS in 2009 while playing Marco Polo with his kids in the family swimming pool.
On March 9, 2011, he got an injection of 500,000 stem cells — the cells were derived by Rockville, Md.-based Neuralstem Inc. after a patient donated her fetus' spinal-cord tissue in 2002 — as part of an 18-operation, 15-patient trial that last 2 1/2 years.
Harada doesn't know if the tissue was from an embryo that was aborted or one that was miscarried or one that died as a result of an accident. The stem cells he got weren't from that embryo, they were from cells that begat cells that begat cells that begat cells during 11 years and many generations of cells.
The operations were conducted by Emory University Hospital physician Dr. Nicholas Boulis. The trial was designed, in part, by Dr. Eva Feldman, director of the A. Alfred Taubman Medical Research Institute at UM and director of the ALS clinic at the UM Health System. Boulis is a former colleague of hers at UM.
Title: Neuralstem ALS Trial Data Presented At The American Association Of Neurological Surgeons Annual Meeting
Date(s): 29-Apr-2013 11:15 AM
Sentiment: Strong Buy
The company said, "2013 promises to be a transformative year for the company, with five NSI-566 cell therapy trials planned. The two new U.S. trials will be the ALS Phase II, and the recently FDA-approved Phase I in chronic spinal cord injury. We hope to have agreements with multiple sites for the Phase I chronic spinal cord injury trial in place by the end of the 2nd Quarter and then begin the transplantations. Internationally, our ischemic stroke trial is expected to commence in Beijing at world-class BaYi Brain Hospital in the coming weeks, through our subsidiary, Neuralstem China. A planned ALS combined Phase I/II is expected to take place in Mexico City, pending finalization of a partnership agreement. Later this month, we expect to file an IND for an acute spinal cord injury trial in Seoul, South Korea, which we anticipate conducting with our partner, CJ CheilJedang."
Sentiment: Strong Buy
Well done IMHO. Hope we'll win, Als patients will win
Sentiment: Strong Buy
"You never want a serious crisis to go to waste"
- Rahm Emanuel (attribution)
Posted: February 27th, 2013
As callous and cold hearted as this sounds, it is a political truism. There are some systems, some bureaucracies that are so gridlocked, or so moribund that only the urgency that comes from dealing with a crisis can create enough momentum to make progress. The FDA held a hearing on Monday to allow the ALS community to voice their opinions on how to proceed with ALS research. While it is unclear exactly what precipitated the calling of this hearing, the failure of the Phase III Biogen “dex” trial to treat ALS patients has perhaps, finally created a “crisis” mode at the FDA. The audience was able to assure the FDA that they are already in crisis mode.
In emotional and chilling testimonies, patients and their families spoke of living with and dying from ALS. Parents wept for their children, dead and alive. Speaker after speaker, from patients to care givers to clinicians to researchers, echoed the same themes. The FDA needs to redefine “risk” for this patient population; using their existing model is not “protecting” these people from anything, it is killing them. The FDA needs to allow broader access to drugs for this population if these drugs can improve the quality of life for patients, regardless of whether or not it can impact survival. And finally, the FDA needs to dramatically speed up the entire process; no one has a moment to wait. And, in what I confess was a moment of immense pride personally, Ted Harada (patient 15 of in ALS trial) stood up and excoriated the FDA for slowing down a trial and a therapy that held enormous potential for ALS patients. “Just look at me” he said, in more than words. In his testimony, Ted proposed that it is time that the FDA did the right thing and treat this process with the same urgency that the ALS community feels. He spoke with passion, elegance and conviction.
But to me, the most chilling and important testimony came from our site principal investigator at Emory, Dr. Jonathan Glass. Dr. Glass is a clinician with one of the largest ALS practices in the U.S. who has seen over a thousand ALS patients in his career. He is also a world class scientist and researcher who knows first-hand what our experience with the FDA has been like in the Neuralstem trial. In short, Jon speaks here with more experience and authority than anyone else in the field, in the world, literally. Add to that, the fact that outside of our trial, Jon will have to go before the FDA many more times throughout his career and you can understand that criticizing them over “one” experience could have lasting reverberations. But such is his conviction and dedication to his patients, and to science, that he felt compelled to speak truth to power. We should all be thankful.
What Jon said was basically this; Neuralstem has completed 18 surgeries over an artificially long, but agreed upon timeframe to demonstrate that both our cells and the intraspinal surgery route of administration are safe. We did just that beyond any question. Ten experts at NIH have reviewed the data from the first trial, and our proposed Phase II protocol, and found it worthy of millions of dollars of NIH funding. Yet the FDA, without the expertise or experience in the field that the NIH can harness, continues to place arbitrary hurdles in our way, and demand an inappropriately slow time frame for the dosing of patients in the next trial.
The problem is easy to fix, Dr. Glass added. The FDA merely needs to admit when they don’t have the expertise to deal with a subject, and borrow it from another government agency that does; in this case the NIH. They should rely on the NIH scientific and medical vetting at a level the FDA doesn’t have the resources to produce. This is no time for “turf” wars or false egos, people are dying. Furthermore, all the FDA needs to do to speed up the process is talk to us. As Jon said, we are constantly “guessing” where they will be on any particular issue, and waiting the statutory 30 days for responses to every question.
In a recent presentation at the New York Stem Cell meeting, I said that the FDA was not the enemy. They are not. They are under resourced, and in our particular case, without experience or expertise. However, more to the point, they are trying to work from a model which simply isn’t applicable to this disease. At the pace they are currently suggesting we proceed, even if we are right and our therapy works; almost every ALS patient alive today will be dead before it is approved. The ALS community deserves better. We all deserve better.
Had I said these things first, it would have sounded like just another CEO whining about the FDA being “tough.” We all owe Dr. Glass a debt of gratitude for not just his service to the ALS community, but his willingness to speak up. None of these problems are insurmountable. Let us hope that the FDA takes the opportunity that this crisis presents to change the way they approach our trial, and any other novel therapy that has the potential to help this population. I will conclude as Jon started, reminding everyone that we are all on the same team here.
Sentiment: Strong Buy