Research News
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By Peggy Willocks |
The world of Parkinson's research took on a special significance to me. While attending there, most of the unpublished reports were presented in person at the Parkinson's Action Network (PAN) Forum 2002 in Washington, D.C. in May. Leaders in the research field gave us updated trials and even speculative reports that all point to positive treatments and even a possible cure for Parkinson's.
BIOMARKER STUDIES FOR PD
At the PAN Forum, Dr. John Marler, Asso. Dir. for Clinical Trials at the NINDS, reported on the largest PD study ever. This study includes 3 key areas:
1) Broad participation
2) Communication & Education
3) Neuroprotection
Better statistical gathering and biomarkers (such as more focused tests for diagnosing PD, consistency in ratings of measurements, etc.) will be used from this study to allow research scientists easier access to information, and help eliminate subjective information in clinical trial results.
DBS Approval by Medicare Promising (AMA/Pub-Med)
Patients with considerable disability despite optimal medical therapy may benefit from neurosurgical intervention in the form of thalamotomy or thalamic DBS. One study compared the 2 approaches and showed both procedures to be equally effective in reducing tremor. Patients treated with DBS improved in their ability to perform activities of daily life, whereas patients who underwent thalamotomy did not. Another advantage of DBS is that bilateral intervention is possible, whereas bilateral thalamotomy is not recommended because of residual speech and gait dysfunction.JAMA.
This study confirms the great value of subthalamic nucleus stimulation in the treatment of intractable PD. Pub MedAbstract
Off medication, the UPDRS part III score was reduced by 55 % during on stimulation. In the on medication state, no difference was noted between the preoperative and the postoperative off stimulation conditions. The severity of motor fluctuations and dyskinesias assessed by UPDRS IV was reduced by 76 % at 6 months. Off meds (the UPDRS II or ADL score was reduced by 52.8 % during on stimulation . The daily dose of antiparkinsonian treatment was diminished. These results remained stable at 12 months for the 14 patients studied
RASAGILINE – PRESTO
The Parkinson Study Group (PSG), in collaboration with Teva Clinical Research, Inc., is conducting a study for patients with moderate to advanced Parkinson’s Disease. This 26-week study will examine the safety, effectiveness and tolerability of an investigational drug in patients who do not experience full benefit from their L-dopa dosage .
Ragasiline N-Propargyl-1(R)-aminoindan (rasagiline) is now under phase III clinical trials for Parkinson's disease (PD), and it rescues dopamine neurons from cell death in animal and cellular models of PD. Recently, we proved that rasagiline protected dopaminergic cells against apoptosis induced by a dopaminergic neurotoxin and provides suppression of death signal transduction in mitochondria. Intial reports indicate the ability of rasagiline to adjust the apoptotic threshold and protect degenerating neurons in PD.
RETINAL PIGMENTED EPITHELIAL CELL IMPLANTATION
article 436
Results from a 12-month, open-label, six-subject phase I/II trial indicate that Spheramine improves motor function in subjects with advanced Parkinson's disease. Subjects were evaluated using the Unified Parkinson's Disease Rating Scale (UPDRS) pre- and post-treatment, both 'on' and 'off' their normal medication. According to the trial data, subjects experienced an almost 50% improvement in motor function with Spheramine treatment. An average 43% improvement was observed in total UPDRS score, in addition to improvements in quality of life and activities of daily living. Spheramine is being developed by Titan and Schering AG.
NEUROPROTECTION
Results from a multicenter, double-blind phase II trial suggest that Guilford Pharmaceuticals' GPI-1485 may delay the loss of dopamine transporters in subjects with mild-to-moderate Parkinson's disease. Three hundred subjects were randomized to receive placebo, low-dose GPI-1485 or high-dose GPI-1485 administered orally for 24 weeks. Change in dopamine transporter density was measured utilizing Dopascan Injection and single photon emission computed tomography (SPECT). SPECT brain scans were obtained for 105 subjects at baseline and six months after randomization. According to a second evaluation of the SPECT scores, the mean percent change from baseline in the density of dopamine nerve terminals was -2.4% in the placebo group, -3.5% in the low-dose group and +1.6% in the high-dose group in a placebo-controlled trial of 16 patients with advanced PD, KW-6002 was shown to potentiate the antiparkinsonian effects of levodopa and prolong its efficacy, but without increasing the dyskinetic
effect
article 439
SHOULD I ENROLL IN A CLINICAL STUDY??
Before a pharmaceutical company can initiate testing in humans, it must conduct extensive preclinical or laboratory research. This research typically involves years of experiments in animal and human cells. The compounds are also extensively tested in animals. If this stage of testing is successful, a pharmaceutical company provides this data to the Food and Drug Administration (FDA), requesting approval to begin testing the drug in humans. This is called an Investigational New Drug application (IND).
The clinical testing of experimental drugs is normally done in three phases, each successive phaseinvolving a larger number of people. Once the FDA has granted a New Drug Approval (NDA), pharmaceutical companies also conduct post marketing or late phase three/phase four studies
centerwatch.com
People participate in clinical research for a variety of reasons. People who volunteer for phase II and phase III trials can gain access to promising drugs long before these compounds are approved for the marketplace. They typically will get excellent care from the physicians during the course of the study. This care also may be free.
The patient's rights and safety are protected in two important ways. First, any physician awarded a research grant by a pharmaceutical company or the NIH must obtain approval to conduct the study from an Institutional Review Board. The review board, which is usually composed of physicians and lay people, is charged with examining the study's protocol to ensure that the patient's rights are protected, and that the study does not present an undue or unnecessary risk to the patient. Second, anyone participating in a clinical trial in the United States is required to sign an "informed consent" form. This form details the nature of the study, the risks involved, and what may happen to a patient in the study. The informed consent tells patients that they have a right to leave the study at any time.
Patients considering participating in clinical research should talk about it with their physicians and medical caregivers. They also should seek to understand the credentials and experience of the individuals and the facility involved in conducting the study.
Other questions to ask include:
How long will the trial last?
Where is the trial being conducted?
What treatments will be used and how?
What is the main purpose of the trial?
How will patient safety be monitored?
Are there any risks involved?
What are the possible benefits?
What are the alternative treatments besides the one being tested in the trial?
Who is sponsoring the trial?
Do I have to pay for any part of the trial?
What happens if I am harmed by the trial?
Can I opt to remain on this treatment, even after termination of the trial?
By Peg Willocks