March 2015 Progress Update

We want to sincerely thank our subjects who have participated in our studies. Without your participation, we would not have the knowledge we have today.

Research Findings

  • In our last few letters, we informed you about the progress we are making in understanding how natural killer T cells in the blood show evidence of "exhaustion" in their ability to make inflammatory products. We are continuing to study these cells by looking at whether they migrate to the lung in sarcoidosis patients compared to control subjects. Since there are medications that can turn on these natural killer T cells it is essential that we figure out if they are "good" or "bad" in making sarcoidosis inflammation before using the medications.
  • We also told you about an exciting finding relating to a blood marker that predicted which subjects showed decline in their lung function and which did not. Well to get something like this into the clinic, we MUST validate it in another NEW group of patients. So we are continuing our study and enrolling additional subjects to participate. IN ADDITION, because we, at UCSF, are the NIH site for recruitment into the GRADS study, we will be able to use samples collected as part of the GRADS study, which some of you may have participated in. All this is very exciting and necessary steps to get closer to a blood marker that we can use in the clinic.
  • We are working on applying for additional funding to push this finding further, understand the biology of the blood marker, called CXCL9, and our goal is by doing so, we may find new "targets" in sarcoidosis therapy.

Finally, the GRADS study will be ending on June 30th, 2015. We will be working with the NIH to analyze the information and samples collected, so this year is looking very fruitful!

What is new?

Exciting news on the clinical trials front! We were awarded funds to perform a clinical trial looking at a medication called Acthar. This medication is FDA-approved for sarcoidosis, and the goal is to figure out which patients will best benefit from it. We anticipate that we will have approval to start enrolling in early 2016.

What can we do with this information?

These discoveries are really important for many reasons:

  • Sarcoidosis needs non-invasive diagnostic tests. Work using blood to identify unique "signatures" is one way we think that we can find a way to get closer to a "blood test" for sarcoidosis.
  • Discoveries such as CXCL9 represent important biological tools for pharmaceutical companies as they perform clinical trials and can use these biomarkers to assess the patient response to a therapy. This is the goal with the industry-related clinical trial mentioned above.
  • Discovering the importance of CXCL9 in the disease course of sarcoidosis also opens up an entire area of study which may identify additionally important biomarkers that also could be targets for new therapies.
  • These findings give us hope that in the future we will have several biomarkers to use in clinical practice and new drugs focused on these new biomarkers with the goal of decreasing side effects of therapy.
  • These findings can help educate doctors about the symptoms that patients with sarcoidosis suffer from so they may be better in finding ways to alleviate the suffering.

The goal we are all working for is to find a cure and stop the suffering!

Where are we going from here?

  • We have reached a big milestone in our study and have a better understanding about the visit time points that appear to be very useful. We have found that the most critical time points for the blood samples are at the first visit, year 1 and year 2. We do not yet have enough study participant visits at the 5 year time point so we would like to continue this time point and hope that participants stay involved.
  • Also we think another very important study is the relapse or flare study. So little is known about why sarcoidosis symptoms suddenly worsen that this work really needs to be done to understand and have any hope to have better treatments.

We hope to grow our clinical trials program once we have the experience from this first trial. There are many biologic medications out there and our research is showing rationale to use them in sarcoidosis.

As a team, we are making great strides in pushing the field forward. Thank you for your time and dedication to this research effort!