Clinical trials. There are lots of them and unfortunately many struggle to enroll enough participants. Knowing this, I enrolled with Fox trial finder to see what might come up. Well I didn’t have to wait long before I got an e-mail asking if I would be interested in participating in a phase 1 trial. Phase one means that it is primarily about safety with a secondary outcome being efficacy. I responded immediately and asked what was involved. She explained that if I passed all the entry criteria it would involve implanting a viral vector with gene’s that would improve my brains ability to process dopamine. Real cutting edge stuff.
I talked it over with the family and responded the next day with a “Let’s do it”. Then things got really fun. Below are the eligibility requirements (I think getting into the Navy SEAL’s would be easier!):
- Diagnosed with idiopathic Parkinson’s disease and on dopaminergic therapy for 5 or more years, with at least the last 3 years including levodopa.
- Modified Hoehn and Yahr Staging of III or IV off medication.
- Candidate for surgical intervention because of disabling motor complications.
- UPDRS Part III (total motor) score ≥ 25 and a maximum of 60 in the OFF state.
- Unequivocal responsiveness to dopaminergic therapy.
- Stable Parkinson’s symptoms and medication regimen for at least 4 weeks prior to screening examination.
- Ability to comprehend and sign the informed consent.
- Normal Laboratory values prior to surgery.
- Neutralizing AAV2 antibody titer ≤ 1:1200
- Ability to travel to study visits alone or able to designate a caregiver.
- Subject agrees to defer any neurological surgery, including deep brain stimulation, until after completing the 12 month study visit (unless recommended by study neurologist).
- Subject agrees to not participate in any other therapeutic intervention study for 12 months after surgery.
- Subject agrees to not have any vaccinations within 30 days of surgery.
- Atypical or secondary parkinsonism, including but not limited to symptoms believed to be due to trauma, brain tumor, infection, cerebrovascular disease, other neurological disease, or to drugs, chemicals or toxins.
- Presence of dementia as defined by a Mattis Dementia Rating Scale-Second Edition (MDRS-2) of less than 130 at screening.
- Presence or history of psychosis, with the exception of mild, benign hallucinations believed in the judgment of the investigators to be related to Parkinson’s medications.
- Presence of severe depression as measured by Beck Depression Inventory II (BDI-II) > 28 or a history of a major affective disorder within 5 years of screening examination.
- Current suicidal ideation or suicide attempt within 5 years of screening examination.
- History of substance abuse within 2 years of screening examination.
- Brain imaging abnormalities in the striatum or other regions that would substantially increase risk of surgery.
- Contraindication to MRI and/or gadoteridol.
- Coagulopathy or inability to temporarily stop any anticoagulation or antiplatelet prior to surgery.
- Prior brain surgery including deep brain stimulation, infusion therapies or any other brain surgery.
- Prior gene transfer.
- History of stroke, poorly controlled or significant cardiovascular disease, diabetes or any other acute or chronic medical condition.
- History of malignancy other than treated carcinoma in situ within three years of screening evaluation.
- Clinically apparent or laboratory-detected infection.
- Prior or current treatment with any investigational agent within 2 months of screening evaluation.
- Chronic immunosuppressive therapy, including chronic steroids, immunotherapy, cytotoxic therapy and chemotherapy.
- Pregnant and lactating women.
- Subject with reproductive capacity who is unwilling to use barrier contraception.
- Any factors, medical or social, which would likely cause the subject to be unable to follow the study protocol, including geographical inaccessibility.
- Ongoing treatments such as, neuroleptic medications, apomorphine, or levodopa infusion therapy (Duodopa®).
I passed. There were MRI’s, PET scans, and off med visits. My personal favorite was any visit that required me to be off medication as that is my nightmare. My muscles would start cramping and seizing (it is really fun to see your pinky start moving outward and your facial muscles tightening to the point where you can barely speak). My brother accompanied me on one of these visits and thought I was having a stroke! To make things more interesting, the PET scan which involves getting into a tube inches from your face, was for over an hour. All these were pre-surgery and would be repeated all over again after the surgery. I was a pin cushion with all the IV’s and blood tests. Good thing my veins are bigger than an LA freeway. The surgery was scheduled for Jan 12th, but before that, I would have to make a trip to Oregon…