News from the 9th Annual Meeting of the American Society for Experimental Neurotherapeutics, Washington, DC, March 8-10, 2007

April 1, 2007

Challenges related to the mechanisms of care, trust, and personal belief systems keep ethnic patients with Parkinson disease (PD) from participation in clinical trials, according to a study by researchers affiliated with National Institutes of Neurological Disorders and Stroke (NINDS) from the Medical University of South Carolina in Charleston. These researchers highlighted findings from 2 futility trials conducted by researchers from NINDS revealing that of 413 patients recruited, 91% were white persons and only 3% each were African American, Asian, and Hispanic persons.

LACK OF DIVERSITY IN PD CLINICAL TRIALS DEFINED.Challenges related to the mechanisms of care, trust,and personal belief systems keep ethnic patients withParkinson disease (PD) from participation in clinicaltrials, according to a study by researchers affiliatedwith National Institutes of Neurological Disordersand Stroke (NINDS) from the Medical University ofSouth Carolina in Charleston. These researchers highlightedfindings from 2 futility trials conducted by researchersfrom NINDS revealing that of 413 patientsrecruited, 91% were white persons and only 3% eachwere African American, Asian, and Hispanic persons.

To get a handle on why ethnic persons are scarce inclinical trials of PD, the researchers conducted focusgroups that included patients with PD and their intimates.Eleven focus groups with patients from minoritypopulations, 8 with intimates of this cohort, and 6with white patients of European descent were conducted.Face-to-face interviews with healthy AfricanAmerican and Hispanic persons from the communityalso were conducted.

The researchers learned that although patients usuallygo through the same pathways to end up at universitycare facilities, ethnic patients experience morediagnostic "detours" and some arrive because of serendipitousassociations with persons working at thefacility and not because of physician referrral.

Focus group members said that incentives for participatingin clinical trials include making a differencein how future patients are cared for, access to newmedications, and the opportunity to be closely monitored.Reported barriers to care include fear of the unknown,awareness of recent drug recalls, some recollectionof the infamous Tuskegee Syphilis Study (inwhich African American men with syphilis infectionwere intentionally provided misinformation and monitoredto examine disease progression), disturbance of"stable" condition status, and interference with regulartherapies. Community interviews revealed that althoughpersons appreciate the need for clinical trials,they are unfamiliar with the term "clinical trials."

Results of a physician survey revealed that physicianshave concerns about patient safety in clinical trialenrollment. Furthermore, trust of clinical trials islower among physicians whose patient populationmostly consists of persons from minority groups, regardlessof the physicians' own race or ethnicity.

APOMORPHINE BRINGS TRAUMATICALLY BRAIN-INJURED"BACK TO LIFE." Asmall, open-label study showed thatapomorphine could revive patients who became vegetativeor minimally conscious as a consequence oftraumatic brain injury (TBI). The study, which wasfunded by NeuroHealing Pharmaceuticals of Newton,Massachusetts, included researchers from the FLENIHospital in Buenos Aires; the Loewenstein RehabilitationHospital in Raanana, Israel; and the University ofPittsburgh Medical Center.

Eight patients who had been in a vegetative or minimallyconscious state for about 1 to 4 months as a consequenceof motor vehicle accident-associated TBIwere given continuous subcutaneous apomorphinevia external pump for 12 to 16 hours per day for 84days. The dosage was progressively increased from 1mg/h up to 8 mg/h. The patients had failed to respondto stimulant drugs, such as methylphenidate,bromocriptine, amantadine, and levodopa.

Elkan R. Gamzu, PhD, chairman and acting head ofclinical trials at NeuroHealing Pharmaceuticals, pointedout to Applied Neurology during the poster presentationsession where the findings were displayed thatalthough a study in the New England Journal of Medicinepublished in 1994 (Medical aspects of the persistentvegetative state [2]. The Multi-Society Task Forceon PVS. N Engl J Med. 1994;330:1572-1579) put theprobability of good recovery/moderate disability forsuch patients at 24%, 4 (57%) of 7 surviving patients inthe apomorphine study achieved good recovery/moderate disability status.

Response to therapy was seen within 2 to 4 weeks,and improvements were maintained after apomorphinewas discontinued, Gamzu reported. He relatedthat 1 of the 8 patients died of TBI, 1 remained in avegetative state, and 1 regained consciousness but wasdeemed severely disabled.

To drive home that promising results were seenwith the therapy, Gamzu pointed to the photograph ofa sailboat emblazoned on the poster, remarking that apatient who was designated as "near coma" at baselineachieved such a dramatic recovery that at 1 yearpostinjury, he was driving and sailing in the picturedboat. The research team concluded that their findingsjustify initiation of a double-blind, placebo-controlledtrial, which Gamzu said will be pursued once fundingbecomes available.