By: Sondra Simpson, Project Manager, MedSource
April 11th is World Parkinson’s Day. This is a day set aside to center public attention on the more than 10 million worldwide who are living with this disease. Each year approximately 60,000 Americans are diagnosed with Parkinson’s disease (PD), and it is estimated that by the year 2020, close to one million people will be living with the disease in the United States alone.
Parkinson’s disease is a progressive neurodegenerative disease, the second most common disorder of this type after Alzheimer’s. The disease is named for Dr. James Parkinson who published an essay on the “Shaking Palsy” over 200 years ago which described the signs and symptoms of PD. When small clusters of dopaminergic neurons in the midbrain begin to die, the result is a reduction of the critical neurotransmitter called dopamine. Dopamine is responsible for transmitting messages to parts of the brain which coordinate muscle movement.
PD is a costly disease, both physically and financially. The combined (direct and indirect) cost is estimated to be nearly $25 billion per year in the United States alone. Medications cost an average of $2500 a year, and therapeutic surgery can cost as much as $100,000 per person.
While there are treatment options available which can improve quality of life, there are no therapies available for slowing down the progression of the disease or for preventing PD, which is the ultimate goal. A therapy that could alter or prevent Parkinson’s prior to symptom onset would impact millions of patients worldwide.
The past few years have been hopeful due to new therapies being developed at a fast pace making it a critical time to focus on advancing clinical research and the development of new treatments for Parkinson’s disease. Preclinical studies suggest that a new drug, Exenatide, plays a role in inhibiting cell death, reducing oxidative stress, enhancing mitochondria function, as well as promoting neuronal functioning. As research continues with other drugs, Levodopa, Amantadine, Opicapone, and Istradefylline, additional support may be provided to patients by way of compassionate use exemption. Providing early access to drugs that are already commercially available outside of the US allows participating subjects to continue receiving the investigational product until the drug is approved or until it is determined that the drug will no longer be pursued for marketability in the US.
There is reason for excitement today in the field of PD, and this excitement should be shared with patients being diagnosed and treated with the disease. To continue making progress, the focus must remain on the collaboration of neuroscience and physician scientists as well as the advancement of clinical research for the development of new therapies. As new drugs, devices and techniques continue to be developed, efforts must be made concentrating on individualized treatment to each patient’s symptoms and needs.
During this pivotal time of research for PD, patients and caretakers alike should be equipped with the necessary resources to help shape the future. The drug development process can be enhanced with the engagement of patients and their caregivers by:
- Getting connected with local or regional groups, such as the Michael J. Fox Foundation
- Joining state supervised PD registries to be alerted to new trials starting in the patient’s respective areas
- Attending regional registries’ regular support meetings and workshops for both patient and caretaker
- Utilizing valuable resources provided by registries or groups such as patient and healthcare provider connections specific for PD outside of the movement disorder spectrum, literature about the disease, and information on ongoing studies
- Promoting regular awareness campaigns and public education
It is also important for researchers to continue to support national and international collaboration through the US and World Parkinson’s Congress empowering the research to move forward. Clinical Research Organizations and professionals should also be well-versed in the laws and regulations around drug development, specific to PD. Clinical research associates and regulatory teams can be an asset to site teams by helping reduce the administrative burden, preventing or correcting deviations, and ensuring that sites are audit ready when participating in PD clinical trials. The teamwork between clinical research associates and the site staff can make all the difference in alleviating the burden of research and finally identifying a cure for this disease and others like it.
Setting aside a day to recognize Parkinson’s disease keeps the spotlight on both the past and the future of drug development, as well as the importance of patient and caregiver engagement. Join us in acknowledging April 11th as World Parkinson’s Day to raise awareness for this disease and the future of drug development for a cure.
Sources:
https://www.parkinson.org/Understanding-Parkinsons/Statistics
https://www.niehs.nih.gov/health/topics/conditions/parkinson/index.cfm
https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm547852.htm