The Phase II trial is designed to treat up to 15 ambulatory ALS patients, in five different dosing cohorts, advancing up to a maximum of 40 direct injections and 400,000 cells per injection, based on safety. This compares to a maximum of 15 injections of 100,000 cells each, directly into the gray matter of the spinal cord, in the completed Phase I trial. The first 12 Phase II 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.

• Crawford Research Institute at the Shepard Center in Atlanta. Former Reeve Foundation International Research Consortium on Spinal Cord Injury Associate Keith Tansey, M.D., Ph.D. is listed as principal investigator for the study. (keith_tansey@shepard.org).
• University of Miami
• Thomas Jefferson University Hospital, Philadelphia
• Medical College of Wisconsin, Milwaukee
• Karl Johe, the chairman and chief medical officer of Neuralstem, announced this week that UCSD Medical School would also be one of the trial centers. For more about this site, contact Adrienne Rebollo, arebollo@ucsd.edu.

This open-label, multi-site study will enroll 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 post injury. These patients exhibit no motor or sensory function in the relevant segments at and below the injury, and are considered to be in complete paralysis. Study patients 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, 200,000 cells per injection. All NSI-566 SCI patients will receive post-surgery physical therapy, 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 NSI-566 for the treatment of paralysis and related symptoms due to SCI. The secondary objectives are to evaluate graft survival in the transplant site by MRI, as well as the effectiveness of transient immunosuppression.

… the Italian government is on the brink of giving free rein to stem cell quacks, but it’s worse than that. This order, as the scientists point out [see below], in essence allows government-funded facilities to collaborate with stem cell quacks and facilitate their treating patients.

…the Italian case is the first in which unproven ‘stem cell therapies’ may be de facto made legal, rather than being stopped by regulatory bodies and the government. Thus, this is the first case in which unproven stem cell treatments are officially recognized as a bona fide treatment, without having been tested in rigorous clinical trials, and based on flimsy and highly debated preclinical evidence, to be made part of a publicly funded, public health care system.

The protection of patients from potential fraud was the main reason why drug regulation first arose (first enacted with the Pure Food and Drug Act in 1906, applied to the historical case of ‘snake oil’ (USDA, 1917), and later evolved into the FDA). The Italian case is the first instance in the western world in which this vital regulatory barrier might be breached.
 

The argument was offered in the Italian case that safety is not a concern in the face of severely ill children or adults, for whom there are no therapeutic alternatives. However, the terminally ill need extra safety and protection, not less. Exposing the weakest people to unknown risks is ethically unacceptable. Recourse to unproven and unsafe therapy is said to be ‘compassion’, or to fall into an arbitrary category of ‘compassionate treatment’. This is not the case at all. Compassion only applies when one offers a safe and potentially effective remedy. That a remedy is effective must be supported by published clinical data. If such data are not available, there is no legitimate assumption of effectiveness in the individual patient, and therefore no ‘compassion’.

One point that the scientists didn’t really nail, and that’s the issue of informed consent. One notes that Vannoni’s stem cell quackery has no evidence for it published in the peer-reviewed biomedical literature nor any compelling clinical trial results. Consequently, if claims are being made for this treatment it is impossible to give informed consent because there is no evidence upon which to base even a rough estimate of the chances for success weighed against the risks of the treatment. Even worse, we don’t even know that these are really stem cells. Seriously. As the scientists point out, there is no transparency, and if there’s an area of clinical research where transparency is essential, it’s stem cell research.
 

The journal Nature, which is following the story, reported today that the Stamina therapy may be reeled in, and only made available in the context of clinical trials. A final decision is expected May 25. Meanwhile, Stamina head Vannoni says he will not comply with the requirement to provide the therapy for trials according to good manufacturing practice.