Consistently ranking among the nation’s top 20 medical schools, its medical specialties ranked amid the nation’s best, and six of those specialties ranked highest in Illinois by US News & World Report magazine, Northwestern University is an exemplary academic medical center. Encompassing over 3 million square feet of medical building space, approximately 2,000 clinical research studies or clinical trials took place at Northwestern in 2009.
FOCUS ON ACADEMIC EXCELLENCE
With a faculty of 3,173 and 2,500 medical and graduate students, residents and fellows, Northwestern University’s Feinberg School of Medicine is able to conduct medical and scientific research creating one of the most complete academically based Clinical Study centers throughout the U.S. and around the world.
Below are three clinical research studies, currently taking place and changing lives.
CLINICAL RESEARCH STUDIES AND THEIR IMPACT ON MEDICINE
According to ClinicalTrials.gov, clinical research studies are generally considered to be biomedical or health-related research studies in human beings that follow a pre-defined protocol. ClinicalTrials.gov includes both interventional and observational types of studies. Interventional studies are those in which the research subjects are assigned by the investigator to a treatment or other intervention, and their outcomes are measured.
Observational studies are those in which individuals are observed and their outcomes are measured by the investigators. The clinical trial process depends on the kind of trial being conducted. The clinical trial team includes doctors and nurses as well as social workers and other health care professionals. They check the health of the participant at the beginning of the trial, give specific instructions for participating in the trial, monitor the participant carefully during the trial, and stay in touch after the trial is completed.
WHAT ARE THE BENEFITS AND RISKS OF PARTICIPATING IN A CLINICAL RESEARCH STUDY?
Benefits
Clinical trials that are well-designed and well-executed are the best approach for eligible participants to:
- Play an active role in their own health care.
- Gain access to new research treatments before they are widely available.
- Obtain expert medical care at leading health care facilities during the trial.
- Help others by contributing to medical research.
Risks
There are risks to clinical trials.
- There may be unpleasant, serious or even life-threatening side effects to experimental treatment.
- The experimental treatment may not be effective for the participant.
- The protocol may require more of their time and attention than would a non-protocol treatment, including trips to the study site, more treatments, hospital stays or complex dosage requirements.
CLINICAL RESEARCH STUDY 1
Geron Trial ~ Human Embryonic Stem Cells
Human embryonic stem cells (hESCs) are nature’s master stem cells. They are a self renewing source for the scalable manufacturing of functional replacement cells for every tissue and organ in the body. The hESCs with which Geron works were derived from surplus in vitro fertilized embryos originally created as part of an in vitro fertilization (IVF) procedure.
The embryos, which would otherwise have been destroyed, were donated for research by the parental donors under informed consent. The hESC line that is used to produce GRNOPC1 is the H1 line. Studies using this line qualify for U.S. federal research funding, although no federal funding was received for the development of the product or to support the clinical trial.
“We are very pleased to be participating in the world’s first human embryonic stem cell clinical trial for spinal cord injury,” said lead national investigator Richard Fessler, M.D., professor of neurological surgery at Northwestern University Feinberg School of Medicine and a surgeon at Northwestern Memorial Hospital.
hESCs have two characteristics that make them different from other naturally occurring stem cells. First, they are immortal – they express the enzyme telomerase that enables the cells to divide endlessly in tissue culture. This allows scalable manufacturing of therapeutic cells derived from a master cell bank. Second, hESCs have the ability to differentiate into any of the more than 200 functional, specialized cells that make up the tissues and organs of the human body.
Geron scientists have learned how to grow undifferentiated hESCs under carefully defined conditions, enabling them to be numerically expanded to form large cell banks (hundreds of vials of frozen undifferentiated hESCs) that serve as uniform starting material for manufacturing procedures that convert the undifferentiated hESCs into functional therapeutic cells.
The FDA-approved clinical study is a Phase I multi-center trial designed to assess the safety and tolerability of GRNOPC1 in patients with complete ASIA (American Spinal Injury Association) Impairment Scale grade A thoracic spinal cord injuries. Spinal cord injuries are graded in severity according to the ASIA Impairment Scale (AIS) – grade A being the most severe with complete loss of locomotor and sensory activity below the site of the injury.
Most such patients do not recover function or respond significantly to physical therapy. The first subjects to receive GRNOPC1 under the clinical protocol will be AIS grade A injured patients with a thoracic injury resulting in a neurological level of T3 to T10. The therapeutic protocol is also limited to subjects with subacute injuries – injuries that can be treated with GRNOPC1 within seven to 14 days after the injury.
Animal studies have demonstrated that GRNOPC1 injections are ineffective if administered more than three months after the injury due to the scarring that occurs in the injured cord as part of the inflammatory response to spinal cord injury.
“Injection of oligodendrocyte progenitor cells directly into the spinal cord lesion is a rational way to attempt to arrest or reverse the structural damage in the spinal cord caused by severe trauma,” Fessler said. “We are eager to begin evaluating the effects of these cells in subjects with severe spinal cord injuries.”
The primary endpoint of the study is safety. Standardized physical examinations and neurological testing will be administered before and after the injection of GRNOPC1 at specified time points for one year after the injection to monitor safety parameters. The secondary endpoint of efficacy will use similar testing for evidence of any return of sensory function or lower extremity motor function for one year after injection of GRNOPC1.
Subjects will be immune-suppressed from the time of injection with low-dose tacrolimus for 46 days, at which time the immune suppression will be tapered and withdrawn at 60 days. Subjects will be monitored for a total of 15 years after administration of GRNOPC1.
“The first recipient receiving the injection of oligodendrocyte progenitor cells more than six months ago has not experienced any serious adverse events attributed to the stem cell transplant to date,” said Fessler. “It remains too early in the trial to determine improvement in neuromuscular control or sensation.”
CLINICAL RESEARCH STUDY 2
Tongue Piercing for Paralysis
A new assistive neuro-technology (ANT), called the Tongue Drive System (TDS), enables individuals with severe disability access their environment with nothing but their tongue motion. The human tongue is inherently capable of sophisticated control and manipulation tasks with many degrees of freedom.
It can move rapidly and accurately within the mouth such that the tip can touch every single tooth. The direct connection between the brain and the tongue generally allows it to escape damage even in severe spinal cord injuries (SCI). Unlike the brain, the tongue is accessible, and its location inside the mouth affords a degree of privacy.
TDS consists of a magnetic tracer, the size of a lentil, attached to the tongue by gluing, implantation, or piercing. The tracer generates a magnetic field inside and around the mouth that is detected by an array of magnetic sensors mounted on a wireless headset.
Tongue-movement-induced changes in the magnetic field are sent wirelessly to an ultra-mobile computer or smartphone, carried by the user, which processes and translates every tongue motion to a particular user-defined function.
Once an individual with disability is “enabled” to access a computing device, he/she can nearly do everything that an able-bodied individual can do with that device. This includes communicating, education, training, entertainment, and controlling other devices such as powered wheelchairs (PWC), assistive robotic manipulators, and other home/office appliances on a local area network (LAN). Even the individual’s own natural or prosthetic limbs can be manipulated to move by functional electrical stimulation (FES).
This trial is intended to evaluate the TDS by the ultimate intended users, individuals with severe disabilities, who are the best experts for indicating the benefits and possible shortcomings of any new ANT. The goal is to assess the acceptability and usability of the TDS for various tasks that are important in daily life such as computer access, wheeled mobility, and environmental control.
Patient Martin Mireles, 37, was shot in the neck nearly two decades ago, leaving him with a severe spinal cord injury. Mr. Mireles is one of the participants of the Evaluation of a Tongue Operated Assistive Technology for Individuals with Severe Paralysis (TDS-1) clinical research study. Researchers at the Northwestern University School of Medicine are examining the technology aspect the study, being financed with almost $1 million in federal stimulus funds.
To operate the system, the user wears a headset with sensors that pick up magnetic signals from the tongue ring. Moving the tongue to the mouth’s upper left corner, for instance, moves the wheelchair forward. (The researchers hope that in the future, touching each tooth could signal a different command, from turning on the television to answering the phone to opening a door.)
Researchers decided to use the tongue because they wanted to take advantage of some of the functions a severely disabled person still had. The tongue does not tire easily, they said, and it is not usually affected by a spinal cord injury because it is directly connected to the brain through a cranial nerve.
Initially, they just glued the magnet to the user’s tongue, but it often fell off after a few hours. The idea for the piercing came from Dr. Anne Laumann, an associate professor of dermatology at Northwestern who had studied body art.
Hopes are high that this evolution in medicine will give those that would have once lost all mobility, the ability to understand their condition and how this device will enable them to have more independence and improved quality of life.
CLINICAL RESEARCH STUDY 3
Breast Cancer ~ Tamoxifen Citrate as Possible Treatment
The rationale behind the study is that estrogen can cause the growth of breast cancer cells. Hormone therapy using tamoxifen may fight breast cancer by blocking the use of estrogen by the tumor cells. It is not yet known whether topical tamoxifen causes less damage to normal tissue than systemic tamoxifen in treating patients with ductal carcinoma in situ.
This study is being conducted to compare the effects of tamoxifen taken by mouth to the effects of a gel preparation 4-hydroxytamoxifen (4-OHT) which can be applied directly to the skin of the breast. The purpose of the study is to evaluate 4-hydroxytamoxifen to see how well it works compared with tamoxifen citrate in treating women with newly diagnosed ductal breast carcinoma in situ.
Tamoxifen is an anti-estrogen pill, which is part of the usual treatment for duct carcinoma in situ (DCIS) of the breast. It is recommended to women with hormone sensitive DCIS after the surgical treatment. (Hormone sensitivity is present when the tumor cells make a protein called estrogen receptor or ER.
This can be tested for in needle biopsy samples and your doctor will be able to tell you whether your DCIS is hormone sensitive or not) When tamoxifen is taken by mouth, it is broken down by the liver into its active form (4-hydroxytamoxifen or 4-OHT). Application of the 4-OHT gel directly to the skin may help women avoid some of the side effects of the tamoxifen pill. Unlike the pill, the gel is concentrated in the breasts and therefore very little will be circulating throughout the blood
stream and the body. Also, tamoxifen is not as effective in women whose enzymes do not convert tamoxifen into its active form and therefore their benefit from the tamoxifen pill may be less. It is possible that the use of the 4-OHT gel will be more effective than the tamoxifen pill in these women who have these enzymes.
Participating in this study will require three (3) visits to the Lynn Sage Comprehensive Breast Center – a screening visit, the pre-surgical visit and a follow-up visit. During the screening and pre-surgical visits study participants will have a physical exam and blood work, and will be asked to complete a health questionnaire. In addition, nipple aspirate fluid (NAF) will be collected from the healthy breast.
For this, the breast will be warmed with a hot pack, massaged briefly, and a small suction device will be applied, so that if any fluid is present in the ducts of the nipple, it will be drawn out (It is normal for most women have some fluid sitting in the ducts of the breast.) Any fluid that is produced will be collected for analysis, to see how much 4-OHT is accumulating in the breast tissue.
Study participants found to be eligible for the study will be randomized to treatment with either oral tamoxifen (pill) or transdermal 4-OHT (gel). Randomization means that participants are put into a group by chance and neither participants nor the doctor will know which treatment she is receiving while in the study. Participants in the oral tamoxifen group will take one capsule containing tamoxifen and apply placebo gel to their breasts each day.
Participants in the transdermal 4-OHT group will take one placebo capsule and apply 4-OHT gel each day to their breasts. In either case, participants will take one capsule and apply a gel each day to their breasts. All eligible participants will be on the study medication for 6-10 weeks before their standard surgical procedure for DCIS.
Northwestern Memorial Hospital Featured Site Visit ~ 4th Annual World Medical Tourism & Global Healthcare Congress
As a premier center of excellence, Northwestern Memorial Hospital has been selected as one of our featured site visits for registered attendees of our upcoming 4th Annual World Medical Tourism & Global Healthcare Congress, being held on Tuesday October 25th. For more information on registering to participate in the visit, please email info@MedicalTourismCongress.com.
References:
www.geron.com/GRNOPC1Trial
About the Author
Gabriella Vicuña serves as a Global Program Coordinator for Medical Tourism Association in areas which span events, membership, and publications. As an Associate Editor of the Medical Tourism Magazine and Health Tourism Magazine, Ms. Vicuña also provides editorial leadership for the MTA’s publications. Ms. Vicuña holds a degree in Business Administration and Tourism from the University of Florida. Prior to joining MTA, she was involved in patient services within an academic setting and in the private sector. Ms. Vicuña can be reached at Gaby@medicaltourismassociation.com
