ALS United Mid-Atlantic Funds Two Major ALS Projects at the Jefferson Weinberg ALS Center

The Jefferson Weinberg ALS Center in Philadelphia, PA, is a nationally recognized leader in ALS research and care. In 2024, ALS United Mid-Atlantic committed to funding two significant projects at the Jefferson Weinberg ALS Center to better understand neurofilament levels in ALS patients and develop cell-based therapies for ALS and FTD (Front-temporal dementia.)

These studies combine to benefit global ALS research and will lead to future studies based on the results of these projects.

“ALS United Mid-Atlantic is proud to partner with the Jefferson Weinberg ALS Center to advance ALS research,” said Jeff Cline, Executive Director of ALS United Mid-Atlantic. The experts at Thomas Jefferson University are contributing to our universal knowledge about ALS, which is crucial to finding new treatments and a cure for this disease.”

Project 1 – Routine Evaluation of Neurofilament L Levels in Clinical Care of ALS Patients
This project aims to monitor disease progression and therapy efficacy through routine measurement of Neurofilament (NfL) levels in ALS patients. Neurofilament L (NfL) is a marker of neurodegeneration and in the last few years it has been established as a relevant biomarker of disease with particularly high levels of NfL in ALS and ALS/FTD patients, including in the prodromal phase. NfL has been used successfully for the approval of Tofersen/Qalsody as a predictor of efficacy of the drug. Thus, tracking changes in NfL levels, starting at diagnosis and over the course of the disease and in patients participating in clinical trials, can be useful.

Project 2 – Development of Novel Cell-Based Therapy for ALS/FTD
This project aims to create an innovative cell-based therapy using chimeric antigen receptor (CAR)-T cells to target neuroinflammatory microglia in ALS and ALS-FTD.