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Throughout the ALS research community, you’ll find brilliant scientists working tirelessly on promising breakthroughs. In our Under the Microscope series, we are shining a light on some of these scientists leading collaborative projects supported by Target ALS in partnership with The Association for Frontotemporal Degeneration (AFTD) — work that could someday change the outlook for ALS and FTD patients worldwide.

Meet Dan Elbaum, Ph.D.

Meet Dr. Dan Elbaum, Chief Scientific Officer at the biotech company QurAlis. Together with researchers at Harvard University and the University of Massachusetts Medical School, Elbaum is working on new research focused on measuring changes in a protein, STATHMIN-2, to inform a new model that will help with patient selection and earlier evaluation of drug targets.

Name: Dan Elbaum, Ph.D.
Professional title: Chief Scientific Officer, QurAlis
Project: Antisense Oligonucleotides to Restore Expression of Full Length Stathmin 2 in Sporadic ALS

Please tell us about your project for which Target ALS has provided funding.

Our project is designed to develop tools, models, and assays to advance a therapy into the clinic as rapidly as possible. The QurAlis portion of the effort is focused on developing an assay that can measure changes in STATHMIN-2 levels in cerebrospinal fluid or other biofluid. This assay will help with patient selection and an early evaluation of target engagement (is the compound doing what it should) during clinical studies.

How would you explain this research to someone without a background in science or medicine?

Our research is specifically geared toward determining the level of STATHMIN-2. We know from the literature that STATHMIN-2 is decreased inpatient spinal cord tissue from deceased patients. We are working on developing an assay to measure the level in patients while they are still alive.

Have you encountered any barriers in ALS research? How have you overcome them or what would be helpful to you in addressing them?

The biggest barrier in the research that Target ALS has helped to fund is finding cerebrospinal fluid samples from ALS patients. To overcome this challenge, the Northeast ALS consortium (NEALS), the National ALS Biorepository at the CDC, and commercial sources have been helpful.

Targeting the discovery of biomarkers has become a big focus in ALS research, including through the Target ALS Diagnosis Initiative. How might you describe a biomarker to someone who isn’t a scientist? Once biomarkers are discovered, how/why will they be important to the field?

A biomarker allows one to assess what a drug or drug candidate is doing before it is apparent that there is what the FDA calls “clinical benefit.” For example, if you wanted to know if a drug that is expected to lower blood pressure prevents strokes in patients with high blood pressure, you would need a large number of subjects and a long period of time to show that the incidence of stroke was lower in the treated group compared to the placebo group. However, to ensure that the drug was doing what was expected, you could measure blood pressure after a short period of time. If there is no effect on blood pressure then you might stop the trial early, as the drug was not doing what it was expected. That is, it was not engaging its target in a productive manner. Blood pressure in this case would be a biomarker. For ALS, an early read on target engagement would allow drug candidates that were not likely to be successful to be culled early so that the teams can focus on more promising therapeutics.

 

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