|Neuralstem Announces ALS Long-Term Follow-Up Phase I Data; Tekmira Establishes Regulatory Framework For Emergency Use Of TKM-Ebola|
|By William Kent|
|Monday, 22 September 2014 20:01|
Neuralstem, Inc. (NYSE MKT: CUR) announced Jonathan D. Glass, MD, site investigator at Emory University, presented long-term follow up data on the Phase I trial testing NSI-566 human neural stem cells in the treatment of amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease). The presentation, which occurred at the Annual Symposium on ALS of the Foundation Andre-Delambre, in Montreal, Canada, on Friday, and was not open to the public, covered data up to approximately 1200 days post the stem cell treatment.
Dr. Glass reported that patients in the last safety cohort (n=3), who received treatments in both the lumbar and the cervical region with the highest number of cells per injection, all showed significant slowing of the progression of the disease. One patient showed functional improvement from pre-treatment baseline, which is maintained to present day. The other two patients are maintaining the same level of functionality as they had at the baseline for over three years since the stem cell treatment.
"The long-term follow up data is very encouraging," said Karl Johe, PhD, Neuralstem's Chairman and Chief Scientific Officer. "In Phase I, patients 10, 11, and 12 each received 10 lumbar and five cervical injections, of 100,000 cells each, which was far below the safe maximal dose. Even so, the data shows a significant slowing of the disease progression for over three years. If replicated on a larger scale, this could represent meaningful improvement in quality of life, and lifespan, compared to untreated patients. In our Phase II dose escalation trial, we successfully reached the maximal dose planned, which consisted of 20 lumbar and 20 cervical injections of 400,000 cells each, more than ten times the number of stem cells delivered in the highest dose cohort of the Phase I trial."
"The progress in this trial is truly groundbreaking," said Dr. Glass, who is Director of the Emory ALS Center at Emory University, the first site in the trial. "It has provided data on the safety of multiple injections and multiple transplantation surgeries in ALS patients, as well as the long- term survival of the transplanted cells in the human spinal cord. This provides a strong foundation for moving ahead with more definitive trials focused on the potential therapeutic efficacy of spinal cord transplantation of neural stem cells for ALS."
Tekmira Pharmaceuticals Corporation (Nasdaq: TKMR) (TSX:TKM), a leading developer of RNA interference (RNAi) therapeutics, today announced that the FDA has authorized Tekmira to provide TKM-Ebola for treatment under expanded access protocols to subjects with confirmed or suspected Ebola virus infections.
"Tekmira is reporting that an appropriate regulatory and clinical framework is now in place to allow the use of TKM-Ebola in patients. We have worked with the FDA and Health Canada to establish this framework and a treatment protocol allowing us to do what we can to help these patients," said Dr. Mark J. Murray Tekmira's President and CEO.
"We have insisted on acting responsibly in the interest of patients and our stakeholders," added Dr. Murray. "Today we are reporting that, working closely with regulators in the United States and Canada, we have established a framework for TKM-Ebola use in multiple patients. In the US, the FDA has granted expanded access use of TKM-Ebola under our Investigational New Drug application (IND) and Health Canada has established a similar framework, both of which allow the use of our investigational therapeutic in more patients."
"We have already responded to requests for the use of our investigational agent in several patients under emergency protocols, in an effort to help these patients, a goal we share with the FDA and Health Canada. TKM-Ebola has been administered to a number of patients and the repeat infusions have been well tolerated. However, it must be kept in mind that any uses of the product under expanded access, does not constitute controlled clinical trials. These patients may be infected with a strain of Ebola virus which has emerged subsequent to the strain that our product is directed against, and physicians treating these patients may use more than one therapeutic intervention in an effort to achieve the best outcome," said Dr. Murray. "Our TKM-Ebola drug supplies are limited, but we will continue to help where we can, as we continue to focus on the other important objectives we have to advance therapies to meet the unmet needs of patients."
TKM-Ebola is an investigational therapeutic, being developed under an FDA approved IND, which is currently the subject of a partial clinical hold under which the FDA has allowed the potential use of TKM-Ebola in individuals with a confirmed or suspected Ebola virus infection
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