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PPMI and Biomarkers FAQs

PPMI

Biomarkers

PPMI

Am I eligible to participate in PPMI?
Your eligibility will be determined by staff at the study site during your screening visit. We are seeking 400 PD participants — recently diagnosed Parkinson’s patients who are not currently taking standard medications for Parkinson’s disease — and 200 control participants — adults who do not have PD who are at least 30 years old and who do not have a first-degree relative (parent, child or sibling) with PD. Interested volunteers should contact the study call center at (877)-525-PPMI (7764).

How long will the study last?
PPMI will be conducted over the course of about five years. For individual participants, the duration will range from three to five years depending on their particular study entry date during an initial two-year enrollment period.

What will happen at the first visit?
The first, or screening, visit will be conducted by the study coordinator and investigator.  The screening visit is expected to last three to four hours and includes two parts:

  1. First there will be a comprehensive review of study details and consent form. The consent form is a document you will be asked to sign if you are interested in participating in the study. This review is an opportunity to ask questions and address any concerns about participating. You may want to take the consent form home to review it and come back for the screening activities once you decide to join PPMI
  2. Once you provide consent, evaluations and assessments will be conducted to determine your eligibility to participate in PPMI. This includes a medical history, neurological exam, physical exam, assessment of your vital signs, blood collection and cognitive assessments, medication review and DaTSCANTM. Consult the PPMI Imaging Procedures and Blood Sampling Overview/FAQs for more information about these procedures.

How often will study visits take place?
After the first (screening) visit, you will visit the site upon enrolling and again at 3, 6, 9 and 12 months. Visits will then occur every 6 months until the study is over.

How long will each visit take?
The study has been designed with a schedule of shorter and longer visits. Shorter visits are expected to take 1–3 hours and will include assessment of vital signs, medication review, motor testing and blood collection. Longer visits include a more comprehensive set of assessments and sampling, including motor, neuropsychiatric and cognitive assessments; DaTSCANTM and MRI imaging; and blood, spinal fluid, urine and DNA sampling. These visits are expected to take 6–8 hours, and can be spread over 2 days if more convenient. Please consult the Schedule of Study Activities for Year 1 to learn more about the procedures required at each visit.

The nearest PPMI site is far from where I live. Can I be part of the study but do my visits at a medical facility closer to my home?
Unfortunately, no. Every PPMI site has been carefully selected for its ability to adhere to strict processes and procedures. This is because sample acquisition, handling and storage must be standardized so that results from all sites can be compared — critical for maximizing what researchers are able to learn from this and other studies.

Will I be paid for my time?
Participants will be compensated upon completion of each visit — $50/€38 for each short visit and $200/€150 for each long visit.

Can I choose to do only some of the study activities?
As a PPMI participant, you will be expected to complete all study activities and assessments. Parkinson’s disease involves multiple systems and processes in the body, and researchers have designed the study for the greatest chances of success.

Will my personal information and medical data be kept confidential?
Your privacy is very important. All data collected in PPMI will remain anonymous. Significant measures have been taken in designing the study to ensure that your identity remains completely private. Additional information on privacy policies and procedures can be found in the Informed Consent Form. 

What will happen to the biological samples you collect?
The samples collected in PPMI will be “de-identified” (stripped of your personal information to ensure your privacy) and sent to a central storage facility. De-identified samples will be analyzed for study-relevant characteristics using state-of-the-art lab procedures. The data from this analysis will be entered in a database maintained by the PPMI bioinformatics core at the Laboratory of NeuroImaging (LONI) at the University of California, Los Angeles. Samples will be banked in a central repository. The data, and the samples themselves, will be available to qualified Parkinson’s researchers on request for use in other studies.

Can I participate in other trials while I am enrolled in PPMI?
Yes, but we ask that you wait one year. After you have been enrolled in the study for a year, you may enroll in any other study you wish at the same time as you are participating in PPMI.

If I enroll, do I have to stop seeing my current physician?
No, you are encouraged to maintain your relationship with your doctor. When you come for study visits as a PPMI participant you will be evaluated by an experienced clinical research team. All other decisions about your medical care outside of the study are up to you. PD patients who enroll in PPMI may elect to have the PPMI study site share test results and clinically relevant findings with their personal physician.

What if my doctor suggests that I take medication for my Parkinson's after I enroll?
Your health is of the utmost importance. While our hope is that participants will not begin taking Parkinson’s disease medications for the first 6 to 12 months, physicians and patients should make the decision to begin a new medication regimen independent of participation in PPMI.

What if a PPMI test reveals that I have an unexpected illness or medical problem?
The PPMI clinical team will report any unexpected results of this nature to you and, if given your permission, share this information with your physician.

Where is PPMI being conducted?
PPMI is occurring at 18 sites across the United States and in Europe. Find a site near you.

Who is sponsoring this research?
PPMI is sponsored by The Michael J. Fox Foundation for Parkinson’s Research.

I don't qualify for PPMI, but I still want to help. What can I do?
Please help us spread the word to people who might be interested in participating. If you know someone recently diagnosed with PD, or someone who does not have PD and is not a first-degree relative (parent, child, sibling) of a PD patient, please refer them to http://www.michaeljfox.org/PPMI. You should also contact your nearest study site as they very likely are conducting other trials for which you may be eligible.

Biomarkers

What is a biomarker?

A progression biomarker (or progression marker) can be any objectively measurable characteristic that changes over time in a way that can be correlated to the progression of disease. This type of marker is a critically needed tool for successful clinical trials, particularly trials of potential disease-modifying treatments. A diagnostic biomarker (or diagnostic or risk marker) can be any objectively measurable physical characteristic associated with the presence of disease. Here are two examples from other health conditions:

  • White blood cell count is objectively measurable and a progression marker of leukemia. A count that rises to abnormal levels indicates that the condition is getting worse, while a count that returns to normal indicates improvement.
  • Cholesterol is objectively measurable and, when high, acts as a risk marker of potential heart disease.

Progression markers could help transform the development of next-generation PD treatments. Imagine that researchers conducting Parkinson’s clinical trials could run a straightforward, standardized laboratory test to understand how or if a possible new treatment were successfully slowing the disease process, helping to speed effective therapies toward pharmacy shelves. This is the kind of acceleration that a progression marker could make possible.

Diagnostic markers could help transform diagnosis and treatment for people living with PD today. Imagine that your doctor could simply draw some blood and instantly confirm a PD diagnosis, know how far the disease had already progressed, and make an informed prediction about the rate and nature of your disease progression going forward. While it may not ultimately come in the form of a blood test, this is the kind of simplicity and objectivity that a diagnostic marker for PD could make possible.

What are the biomarkers of Parkinson’s disease?

Unfortunately, no practical, definitive biomarkers of Parkinson’s have yet been identified. There are a few markers used in advanced neuroimaging techniques (approved for clinical use in Europe but not yet in the United States) that can help clinicians diagnose PD in its earliest stages. But no widely and easily applicable, affordable diagnostic markers, and no progression markers whatsoever, have been conclusively validated.

What are some specific ways that Parkinson’s patients are affected by the lack of biomarkers?

  • There is no way to identify people at risk for PD or to establish strategies for PD prevention. It is an often-stated reality that, by the time the first symptoms of Parkinson’s become evident, as many as 60-70 percent of an individual’s dopamine neurons may already have died. A diagnostic marker would allow us to identify people at risk for PD earlier.Diagnosis is subjective, based on observing symptoms and rating them on a clinical scale. This translates to a high rate of misdiagnosis, especially among general practice physicians and neurologists who do not specialize in movement disorders.
  • PD clinical trials are frequently and frustratingly inconclusive. Why?
    • With no diagnostic marker, an estimated 10 percent of PD trial enrollees unwittingly do not have Parkinson’s disease, confounding results.
    • With no progression marker to track the disease, there is no way to objectively measure treatment effects.
    These factors make PD trials highly risky for drugmakers and prolong the wait for next-generation PD therapies.

I thought a biomarker was basically a gene — and don’t we know the genes that play a role in PD?

No — in fact, a biomarker is not the same thing as a gene or genetic mutation. Remember, it’s possible to have one or more genetic irregularities linked to PD but never get the disease. Genetic research leads scientists to certain biomarker candidates, but many other physical and cellular characteristics are valid as possible biomarkers if they are measurable and provide an accurate window into disease presence or progression. Here are the major avenues of pursuit today:

  • Imaging-based markers (such as PET/SPECT) can provide indirect measures of dopamine neuron function, but do not have sufficient resolution to measure actual cell numbers. More sensitive neuroimaging markers for PD, capable of measuring dopamine function in the brain, are also under development. These include DATscan (already approved for clinical use in Europe) and other ways to measure dopamine activity.  These dopamine-based markers may provide a way to monitor disease in its earliest stages. MRI and ultrasound markers are also in development.
  • Biological markers in blood, urine, cerebral spinal fluid or tissue biopsies can serve as ‘signatures’ of the disease. These can include genetic, protein or other chemical and molecular signposts. Leading biological marker candidates include measures of antioxidants such as urate, or measures of proteins produced by PD-implicated genes such as alpha-synuclein and DJ-1.  
  • Clinical measures — such as tests of motor ability, or the presence of disease-associated symptoms such as loss of the sense of smell, sleep disorders, constipation or early speech problems — can be good markers, though not necessarily Parkinson’s-specific, may still provide a means for detecting early stages of PD or tracking disease progression. The downside is that clinical measures are subjective, variable over time, and very sensitive to symptom-masking effects of drugs, limiting their utility to measure disease-modifying effects sought in neuroprotective trials.

Overall, it is likely that we need to develop a combination of markers (imaging, biologics and clinical) and measure them in the same individuals over time to identify useful biomarkers for PD. MJFF’s PPMI study is designed to do just this.

In the absence of a PD biomarker, how have there been clinical trials of disease-modifying treatments at all?

Most trials use some form of clinical measure, such as the Unified Parkinson’s Disease Rating Scale (UPDRS), to determine whether a treatment is working. However, given the subjectivity and variance in clinical measures, and because symptoms of PD are affected by the medications and therapies patients have already used, it is difficult or impossible to measure disease-modifying effects in this way. This may have contributed to a history of inconclusive results from trials of disease-modifying treatments in particular.

Do other diseases have biomarkers?

Some do, but most don’t, and the lack of biomarkers is a particular problem for neurodegenerative diseases (such as Alzheimer’s and ALS in addition to PD). You may have heard of a study called the Alzheimer’s Disease Neuroimaging Initiative (ADNI). Launched in 2004, ADNI has made important strides toward discovering biomarkers of Alzheimer’s disease and has reinvigorated research focused on disease-modifying treatments for AD. We anticipate similar outcomes for Parkinson’s from PPMI.

Has The Michael J. Fox Foundation worked on biomarkers before?

Yes. With investments of about $25 million to date, The Michael J. Fox Foundation has spent years on the search for a PD biomarker. As we embark on PPMI, we believe more strongly than ever that the discovery of PD biomarkers is a high-impact use of our intellectual and financial resources — and that this study will pay dividends toward better treatments and a cure.

August 15, 2011

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