CHICAGO, Oct. 26, 2018 /PRNewswire/ -- At the recently concluded 2018 ACR/ARHP Annual Meeting, SRF-funded researcher Michael Whitfield, PhD, Professor of Biomedical Data Science, Geisel School of Medicine, Dartmouth College announced that he has developed a classifying tool that may predict which scleroderma patients are most likely to benefit from stem cell transplants.
The stem cell transplant process is lengthy and burdensome for scleroderma patients and–despite continuous improvements to the protocol–still has the potential for treatment-related mortality. Additionally, not all patients respond to the treatment; a method for identifying those most likely to respond to this therapy would therefore be a significant advance for patients considering a stem cell transplant.
Groundbreaking research led by Dr. Whitfield seems to have found such an identifier. Dr. Whitfield's study is a collaborative project with researchers from the Scleroderma: Cyclophosphamide or Transplantation (SCOT) trial at Duke University and other sites around the U.S., which compared the potential benefits of stem cell transplant with high-dose monthly cyclophosphamide (Cytoxan) for the treatment of scleroderma. Whitfield's team analyzed gene expression data from blood samples collected from 67 SCOT trial participants. Using a proprietary classifying algorithm, which the Whitfield lab developed and refined over years of effort, patients were sorted into three groups based on their blood cells' gene expression signature prior to treatment. As reported by Dr. Whitfield this week, their classification algorithm was able to identify which groups of patients were most and least likely to respond to stem cell transplant.
"The results indicated that for one group of participants, called the 'normal-like group,' event-free survival did not differ between patients receiving transplant and patients receiving cyclophosphamide. This suggests that patients who fall into this group are unlikely to benefit from stem cell transplant," explains Dr. Whitfield. "In contrast, for participants in another group, called the 'fibroproliferative group,' the data showed a statistically significant improvement in event-free survival in patients receiving transplant as compared to patients receiving cyclophosphamide. This suggests that patients who fall into this group are more likely to benefit from stem cell transplant. This was also an important finding because patients who fall into the fibroproliferative group tend not to respond to immunosuppressive therapy."
Whitfield says the next step is to validate the results and develop his classifier into a diagnostic tool that clinicians can use. "This is an important result and potentially a significant step for scleroderma research," comments Deann Wright, whose experience with scleroderma both as a patient family-member and board member at the SRF give her a unique perspective on the field, "If this classifying tool can be validated and developed for clinical use, then it can help guide patients and their clinicians who are considering a stem cell transplant. This would be a big step toward personalized medicine for scleroderma patients."
This study informs future research that could change the face of scleroderma treatment; it brings research one step closer to a cure, and gives hope to thousands of patients.
The Scleroderma Research Foundation's mission is to fund the most promising medical research aimed at improving therapies and a cure. We are recognized by Charity Navigator as one of the Top 10 Medical Research Foundations in the country.
For more information, contact the Scleroderma Research Foundation directly at 415-834-9444 or email us at [email protected]. Our office mailing address is: 220 Montgomery Street, Suite 484 San Francisco, CA 94104.