Clinical Trials and Cerebral Palsy: The Basics

Clinical Trials and Cerebral Palsy: The Basics

Clinical Trials and Cerebral Palsy
As with most medical conditions, cerebral palsy does not have just one treatment. Considered “incurable,” the condition of cerebral palsy results in a lifelong quest for improvements, not cures. Parents of children with cerebral palsy spend incredible amounts of time and energy tracking down the latest developments in scientific research, treatments, therapies and medical procedures. These new innovations are often deemed “alternative,” “experimental,” or even “radical” and just because the FDA approves something new as “safe,” doesn’t mean your insurance company, assuming you have one, will fall in line and jump to cover such expenses. And yes, new procedures, treatments and therapies are often prohibitively expensive, in part because they are not yet mass-produced or consumed and practitioners must receive extensive, ongoing training in its application and use. Before a new innovation finds itself on an insurance company’s radar, it must demonstrate long-term safety and overwhelmingly positive results.

All of which leads us to… clinical trials.

In short, clinical trials involve the participation of people in medical research at little or no cost to the participant. It is one of the many paths taken by parents in their quest for a higher quality of life for their children with CP. Here are some of the basics you need to know.

What is a Clinical Trial?

All FDA approved clinical trials are conducted in 4 phases and can last for many years.

  • Phase I: Screening for safety. A small number of patients and healthy volunteers (20-80) test a drug/procedure for safety, tolerance, dosage range and side effects.
  • Phase II: Screening for effectiveness. Also involving a small number of patients (100-300) to determine the short and long-term effect on a patient’s cerebral palsy.
  • Phase III: Comparative results. To determine how new treatments compare to the standard treatments already in place. These trials may be conducted on hundreds or thousands of patients on a much larger scale, sometimes even nation-wide.
  • Phase IV: Postmarketing studies. To further delineate risks, benefits and optimal use.

Clinical trials are conducted at medical centers, hospitals, universities and private facilities all over the world. The principal investigator in charge of the study must ensure that all clinical administrators, no matter where they are, are conducting the trial(s) in exactly the same manner.
Clinical trials are conducted at medical centers, hospitals, universities and private facilities all over the world. The principal investigator in charge of the study must ensure that all clinical administrators, no matter where they are, are conducting the trial(s) in exactly the same manner.

The Benefits of a Clinical Trial

Clinical trials are an essential element to the advancement of science, but the companies looking for a thumbs up to begin marketing new techniques, products or drugs aren’t the only ones that stand to benefit. Rest assured that if your child is chosen for a clinical trial, it means they are among the populace most likely to reap the benefits of the new treatment. If the new treatment is effective, your child’s wellbeing will almost certainly benefit. Clinical trials are administered by the highest quality experts in the field. Because of the nature of “a study,” your child’s overall health stands to be monitored like never before.

In most cases you will not have to pay for the medical attention your child receives while participating in the trial. In some cases, you may even be compensated.

The Risks and Downsides to a Clinical Trial

As with anything, clinical trials may involve risks. Because it is, by definition, a new treatment, it cannot be known ahead of time if it will work or what the side effects might be. That said, before a child is admitted into a clinical trial, the administrators are obligated to prepare a legal “informed consent” document detailing every possible risk and benefit they perceive, the researcher’s expectations of you and your child and your child’s rights as a patient. You must sign the document before your child can participate. Before a clinical trial begins, researchers must get approval from an Institutional Review Board (IRB), an advisory group that makes sure a trial is designed to protect your child’s safety. The IRB has the right to terminate a trial at any point if they feel harm might be done to the patients. Before participating, it is very important to understand your rights as a participant in a clinical trial.

There are many obvious reasons parents pin huge hopes on the notion of “the newest in medical technology and scientific advancement at no cost.” As I’ve learned from following numerous parenting blogs, there can be also be emotional and financial downsides to seeking and participating in clinical trials. They note that it is difficult not to buy into “false hopes” when chasing a clinical trial, not only regarding the desired outcome, but even getting accepted into one in the first place. Commonly, far more people apply for clinical trials than they could ever use and qualifying for one can be a lengthy and taxing string of events. Families sometimes go through months or years of applications and pre-determining medical and psychological testing, only to be rejected in the end.

Clinical trials require tremendous funding. Some trials are advertised and talked about for years, but never actually happen. Also, because clinical trials are somewhat “local” events, families have been known to travel great distances at great expense just to participate in the application process.

What Current Clinical Trials are Addressing CP?

In researching several sites for current clinical trials addressing cerebral palsy in the US, the three topics most prevalent were: stem cell therapy; Botox and phenol injections and the application of various orthotic or robotic devices.

In January 2010, Medical College of Georgia was granted the first FDA-approved clinical trial to determine whether an infusion of stem cells from umbilical cord blood can improve the quality of life for children with cerebral palsy. In March of the same year, the Robertson Foundation awarded Duke University $10.2 million to develop a state-of-the-art Transitional Cell Therapy Center. At present, these are the only two entities involved in FDA stem cell clinical trials within the US, however, there are dozens more outside US borders.

The popular term “Botox” is just one of many brand names for “botulinum toxin,” the acutely toxic substance responsible for botulism. These days it is most known for its uses in cosmetic surgery. However, scientists, such as those at Loyola University Health System, have found that injecting the drug into children with cerebral palsy can cause very tight and locked muscles to relax. Likewise, phenol, an anesthetic drug, is being injected into patients who suffer from painful spasticity. The phenol destroys the nerve pathways involved with spasticity resulting in an immediate relief that can last 6-36 months before the nerve pathways grow back and the pain returns. There are numerous ongoing clinical trials involving both of these treatments.

Orthotics refers to any externally applied device used to modify the structural and functional characteristics of the neuromuscular and skeletal system. Robotics is a form of orthotics, using computer controlled devices to help reduce impairment and facilitate the neuro-development of children with cerebral palsy. There are some clinical trials involving orthotic and robotic devices, but since they are non-narcotic and non-surgical, it is easier for developers to get FDA approval without performing lengthy, costly clinical trials.

Qualifying for a Clinical Trial

Most clinical studies have stringent eligibility criteria. This can include age, sex, type and stage of your child’s condition, as well as their treatment history. Prior to acceptance into a clinical trial, your child may have to undergo numerous medical and psychological tests. You may also be required to provide pertinent information regarding family stability. This will help determine the likelihood of a child remaining available for follow-up examinations for months or even years down the road. Long term information is key to a successful clinical trial.

How Do I Find Out about Clinical Trials in My Area?

In part 2 of this article, I address how and where to look for clinical trials in your area.


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