RASCALS Note: The following article was written for Investment Underground, an online market and trends analysis publication. While we are in no way advising our readers to invest in Neuralstem, we did want to share information on the trial contained therein.
by Ramu Iyer
A few days ago, local Fox TV stations aired a story about a medical trial by Neuralstem (CUR), which they described as “one of the most powerful stories we’ve ever reported.” Ted Harada is the eighteenth and final patient to receive human stem cell transplant treatment for amyotrophic lateral sclerosis (ALS) patients as part of a clinical phase 1 trial being conducted at Emory. He is also one of the first patients to ever experience a partial recovery from the dreaded disease. It is also called Lou Gehrig’s disease (the famous sporting star Lou Gehrig died of the disease), and sometimes motor neuron disease, because it is a gradual degeneration of the nerves that control motor functions. It progressively incapacitates the body’s motor functions to the point where the patient cannot breathe by himself or herself. Up until this point, it has been invariably fatal, with no current treatment or cure. A new stem cell treatment from Neuralstem could change all that.
Neuralstem announced the completion of the phase 1 trial of its NSI-566 spinal cord neural stem cells for the treatment of amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease), with the treatment on Harada. Harada was the third patient to return for an additional set of injections, and phase 1 will conclude six months after this last treatment. He had his first treatment seventeen months ago when stem cells were transplanted. Soon he began to get better, and could even walk around the neighborhood with the help of a cane. But recently, the weakness started to come back and Ted volunteered for the second round of treatment.
Neuralstem found out that the fatal nerve damage caused by ALS could be slowed down, and even reversed, by supporting healthy cells in the spinal column with the integration of targeted stem cells. The stem cells have been cultured and multiplied in their laboratories, and treatment consists of both injecting cells into the spinal cord and exposing the spinal cord to introduce the cells. The stem cells have the capability of growing into nerve cells that support the existing nerves. You can appreciate the delicacy of the process when you understand that the spinal cord controls every breath you take and every muscle in your body. After completing preliminary studies in animals, the company received FDA approval for human treatment as well as the highly coveted orphan designation. The company developed a patented device for intra-spinal cord surgery, which was used for the stem cell treatment in the eighteen patients. Neuralstem also devised procedures to freeze and preserve the stem cells until they are used.
The purpose of the phase 1 trial was only to assess the safety of the treatment, not to prove the efficacy of benefits. The phase 1 trial commenced in January 2010. The first twelve patients received the treatment in the lumbar or lower back region of the spine. The treatment was then advanced to the cervical or upper back region. The last three patients received treatment in both regions and the FDA approved the inclusion of previously treated patients in this last batch. The motor neurons that control breathing and the ones that patients need to survive are in the upper spinal cord, primarily in the neck. It is also important to remember that the dosages used in the trial, including the treatment of Ted Harada, were approximately one third of the dosages that will be eventually used. The trial also meant that patients had to agree to undergo the spinal procedure and to take immuno-suppressant drugs for the rest of their lives in order to prevent rejection of the stem cells being infused.
“There have been many firsts in this trial, including the first lumbar intraspinal injections, the first cervical region intraspinal injections, and the first cohort of patients to receive both,” commented Jonathan D. Glass, MD, Director of the Emory ALS Center. Eva Feldman, MD, PhD, Director of the A. Alfred Taubman Medical Research Institute and Director of Research of the ALS Clinic at the University of Michigan Health System, is the principal investigator on the trial, and an unpaid consultant to Neuralstem. She noted, “We have found the procedure to be extremely safe. In some patients, it appears that the disease is no longer progressing, but it is too early to know if the result from that small number of patients is meaningful.”
Just a few days earlier, Neuralstem announced that it had received a notice of issuance for patent number 12/710,097, titled “Transplantation of Human Neural Cells for Treatment of Neurodegenerative Conditions.” This patent covers both the culturing of central nervous system cells as well as transplanting them into spinal cord tissue to treat neurodegenerative conditions including ALS. This is an important addition to its intellectual capital, because it covers every stage and facet of the treatment, and the patent is valid up to the year 2030.
In addition to ALS, the company is also targeting other major central nervous system ailments with its treatment such as spinal cord injury and ischemic spastic paraplegia, and has submitted an IND (Investigational New Drug) application to the FDA for a phase 1 safety trial in chronic spinal cord injury. The company is conducting a phase 1b to evaluate the safety of NSI-189, its first neurogenic small molecule compound, for the treatment of major depressive disorder (MDD).
This is a promising beginning, but there is still a long way to go before the treatment can be brought to market. This is shaping up to be a promising treatment for a disease previously considered as fatal and untreatable that is going to provide a ray of hope for many patients who may have given up on their condition altogether. While it is still too early to judge whether the treatment is going to be a blockbuster or not, the company is showing great promise. If you are interested in gaining exposure in the biotechnology sector, you should watch this stock carefully for further favorable developments.
Mr. Iyer’s Transparency/Disclosure: I am not a registered investment advisor and do not provide specific investment advice. The information contained herein is for informational purposes only. Nothing in this article should be taken as a solicitation to purchase or sell securities. Before buying or selling any stock you should do your own research. I am a consultant to a third-party and have received two hundred fifty dollars for independent research. Always discuss investments with a licensed professional advisor before making any financial decisions. Statements made herein are often “forward-looking statements” as stipulated under Section 27A of the Securities Act of 1933, Section 21E of the Securities Act of 1934, and the Private Securities Litigation Reform Act of 1995. While I have researched this company thoroughly, my due diligence is not a substitute for your own.
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