The road from paralysis research labs into human clinical testing

Over the years, advocates have provided a significant role in moving promising therapies forward. Legislators and scientists endorse advocate participation, but few people are willing to enter the complicated world of neuroscience. Those who do, provide a personal perspective in matters of funding and priorities in research initiatives. According to a Harris Poll taken in 2005 in a neurological disease patient population, it was estimated that only 1 in 10 people will want to participate in a clinical trial and 85% of the trials finish late due to difficulties enrolling participants. More alarming is the fact that nearly one-third of trials in the poll failed to recruit a single subject and could not begin. Sadly, only 1 in 12 therapies undergoing clinical trial testing actually make it to the bedside.

As potential therapies emerge and governments around the world approve new human clinical trials, the need for realistic expectations and scientific background information is needed in order for the patient to make an educated decision about participation in the trial. A clinical trial is a study involving human volunteers to evaluate new ways to prevent, diagnose, or treat diseases and conditions. Clinical trials determine if experimental treatments are safe and more effective than current available treatments. Participants should fully understand there are no guaranteed outcomes when they submit to human testing. The first phase of a human clinical trial tests for safety.

Before participating in a clinical trial, patients should take the time to explore where the research began and if the animal studies had strong efficacy to warrant the clinical trial. The proposed treatment should have positive results in animal studies without toxic side effects. The results of these studies should have been replicated by qualified labs and the results published in peer reviewed science and medical journals.

In the United States, before an experimental treatment can be tested in humans, it is carefully studied in the laboratory with relevant animal models to determine its safety and effectiveness. Clinical trials are reviewed at both the national level by the Food and Drug Administration (FDA) and at the local level by an institutional review board (IRB). Each hospital or clinic conducting a clinical trial has an IRB made up of health-care professionals, patient advocates, and community leaders who review the trial for safety and fairness. The trials are designed to collect valid data, but there are no guarantees the proposed therapy will provide functional recovery.

Safety and effectiveness are far less of a gamble, when the proper research protocols and approvals have been followed before a therapy is delivered to the bedside. Oftentimes, vulnerable patients harbor an unrealistic hope of personal benefit and dismiss the risks and uncertainties of the outcomes because the science is too complicated for them to understand. If considering participation in a clinical trial, a patient should discuss their participation with their doctor.

It is important to understand that some risks are inherent in clinical research. The specific risks associated with any research protocol are described in detail in the consent document, which patients are asked to sign before taking part in the trial. The risks of participation should be explained and any concerns discussed prior to signing the consent to participate. A patient must carefully weigh the potential risks against any possible benefits. The personal benefits of participating in a clinical trial are not guaranteed and as a result of participating in one clinical trial, the patient may be excluded from future clinical trials.

A patient should ask the following questions before deciding to participate:

* What is the purpose of the study?
* What is required of me?
* What is my role in the study?
* Will the study directly benefit me?
* Will the study benefit others?
* Is there risk? If so, what are the risks and what are the chances that they will occur?
* What discomforts are involved?
* What is the total time involved?
* Are there other inconveniences?
* Have I discussed participation in the study with those who are important to me, such as family and friends?
* Can my life expectancy, quality of life or level of neurological function be adversely affected by the experimental treatment?
* Did I investigate and understand all the animal research studies leading up to the human trial?
* Have these studies been published and peer-reviewed in top science journals?
* Was the animal research replicated by other scientists in an independent lab or the National Institute of Health to check for validity or data accuracy?
* Has this therapy or stem cell already been through human trials before in another country and then later abandoned for safety or lack of efficacy reasons?
* Do I wish to participate in this study?
* Have I read the ICCP Handbook and fully understand what is happening?

With so many new proposed human studies, it is important to stay informed and knowledgeable about the research being conducted. It is a priority for us in the SCI community to understand the various research projects and proposed trials being undertaken. In a tight economy the government research dollars are precious and funding for trials is difficult to obtain. We need to be vigilant about moving the most valuable and important pieces of a combination therapy forward. We must ask researchers to provide us with the maximum potential benefits and minimize the risks by obtaining the proper amount of evidence and scientific information prior to human experimentation.

Although research has always had improved patient outcomes as its goal, we can discourage government officials from funding research that satisfies scientific curiosity and professional goals but does little to bring recovery to the paralysis community. We must also be mindful of surgical interventions without any real benefits to the SCI community gaining wide acceptance and implementation due to lack of performance on clearly interpretable clinical trials. In such a case, thousands with chronic paralysis could subsequently be exposed to unnecessary surgical procedures that would lead to medical complications, potential functional losses or even death. Not all clinical trials are equal in scientific merit. Even though clinical investigators and ethic review committees may conclude that a therapy is acceptable, the ultimate responsibility lies with the patient to weigh the potential risks and benefits.

At U2FP we’re building teams of neuroscientists with solid data and providing financial and collaborative opportunities to bring combination therapies to clinical trials. We are spending the money smarter as we work to identify and chaperon the ideas that are most likely to have an impact on chronic spinal cord injury. Our goal is to prioritize research that can transform the lives of people living with chronic paralysis. Our team has the personal connection and expertise to make real in-roads. We not only understand the science, but the business of science. We are increasing the odds of success and making real progress.

Our world class Scientific Advisory Board and knowledgeable consumers on the Advocacy Research Committee work together, to identify and advance promising research. We can no longer accept complacency in this effort. Our personal connection brings a sense of urgency and deep understanding of the life threatening issues which spinal cord injuries bring. We must avoid wasting precious time and scarce funds on research which has failed to provide solid data. Supporting collaborative efforts with qualified data is a good investment which will provide us the best opportunities to move therapies to the bedside.

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