A Rational Approach to Assessing SMA Therapies: Julie Parsons, MD

Video

The primary investigator of the phase 4 RESPOND study discussed the role that postmarketing studies can play in assessing treatments for SMA.

“Nusinersen was approved just 4 years ago. The fact that this changing landscape has just continued to rapidly evolve, and we have more questions now than we did at the beginning when we had no therapies is absolutely fascinating.”

Just this month, Biogen announced that the first patient was treated in its global phase 4 RESPOND study (NCT04488133) of nusinersen (Spinraza) in infants and children with spinal muscular atrophy (SMA). The trial will evaluate the gene therapy’s performance in those who have unmet clinical needs despite treatment with another FDA-approved medicine, Zolgensma (onasemnogene abeparvovec; Avexis).

RESPOND primary investigator Julie Parsons, MD, Haberfeld Family Endowed Chair in Pediatric Neuromuscular Disorders, professor of clinical pediatrics and neurology, at Children’s Hospital Colorado and the University of Colorado School of Medicine, told NeurologyLive that this trial will feature a number of new outcome measures, including assessments of biomarker levels, such as neurofilament.

Parsons shared her thoughts on what information these evaluations will provide for patients with SMA and their physicians, and how they can impact clinical care. As well, she discussed the role that postmarketing studies can play in the attempts to recognize patients who may respond poorly to treatment as the field of medicine continues to strive for precision, personalized treatment.

REFERENCE
Biogen announces first patient treated in respond study evaluating benefit of spinraza® (Nusinersen) In patients treated with zolgensma® (Onasemnogene abeparvovec). January 8, 2021. Accessed January 26, 2021. https://investors.biogen.com/news-releases/news-release-details/biogen-announces-first-patient-treated-respond-study-evaluating
Recent Videos
Ben Samelson-Jones, MD, PhD, assistant professor pediatric hematology, Perelman School of Medicine, University of Pennsylvania and Associate Director, Clinical In Vivo Gene Therapy, Children’s Hospital of Philadelphia
Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado
Steven W. Pipe, MD, a professor of pediatric hematology/oncology at CS Mott Children’s Hospital
Haydar Frangoul, MD, the medical director of pediatric hematology/oncology at Sarah Cannon Research Institute and Pediatric Transplant and Cellular Therapy Program at TriStar Centennial
David Barrett, JD, the chief executive officer of ASGCT
Georg Schett, MD, vice president research and chair of internal medicine at the University of Erlangen – Nuremberg
David Barrett, JD, the chief executive officer of ASGCT
Bhagirathbhai R. Dholaria, MD, an associate professor of medicine in malignant hematology & stem cell transplantation at Vanderbilt University Medical Center
Caroline Diorio, MD, FRCPC, FAAP, an attending physician at the Cancer Center at Children's Hospital of Philadelphia
© 2024 MJH Life Sciences

All rights reserved.