Julie Parsons, MD

“We’re not done yet. Just because we have 3 drugs that are available [for patients with spinal muscular atrophy], we’re not complacent about this space.”

The spinal muscular atrophy (SMA) treatment paradigm has shifted drastically in the past few years with the approval of 3 treatments: nusinersen (Spinraza; Biogen), onasemnogene abeparvovec (Zolgensma; Avexis), and risdiplam (Evrysdi; Genentech/Roche). Despite this recent string of successes, though, those who are treating patients and researching therapeutic targets have yet to feel satisfied, according to Julie Parsons, MD.

Parsons, who serves as the primary investigator for the ongoing phase 4 RESPOND trial of nusinersen, is also the Haberfeld Family Endowed Chair in Pediatric Neuromuscular Disorders, and professor of clinical pediatrics and neurology at Children’s Hospital Colorado and the University of Colorado School of Medicine, spoke with 

 about the current state of the science. She noted the importance of considering rational therapeutic approaches and the collection of more high-quality data as more studies are conducted. She explained just how these goals go hand in hand, as this additional data can be valuable in developing those approaches.

In this conversation, Parsons also explored some of the obstacles that remain in treating the disease and shared her outlook on how the field is expected to continue to advance over the next few years.

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The Endowed Chair in Pediatric Neuromuscular Disorders and professor of clinical pediatrics and neurology, at Children’s Hospital Colorado discussed the future of SMA treatment.

Looking to the Future of SMA Treatment: Julie Parsons, MD

“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 

 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

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

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

“I think this really is mimicking real-life experience, where we do have patients who have not really responded clinically to a single therapy for SMA. I do think that it’s really, really important to have clinical trial data to be able to look at outcomes.”

Biogen recently announced that the first patient was treated in the global phase 4 RESPOND study (NCT04488133) of nusinersen (Spinraza) in infants and children with spinal muscular atrophy (SMA). The trial’s goal is to assess the safety and efficacy of the gene therapy in patients who have unmet clinical needs despite treatment with another FDA-approved medicine, Zolgensma (onasemnogene abeparvovec; Avexis).

 spoke with 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. She offered insight into what motivated the group to conduct the trial, and what she and her coinvestigators are hoping to observe. Particularly, she noted, they’re seeking the opportunity to confirm the utility of a different mechanism of action to improve clinical outcomes in patients with SMA.

In addition to detailing the measures of the study—including a number of relatively new outcomes—Parsons shared her insight into why clinical trials like this are so vitally important for this patient population.

The primary investigator of RESPOND discussed the trial’s goals and measures, as well as insight into why clinical trials like it are important in spinal muscular atrophy.

Julie Parsons, MD

Articles

Looking to the Future of SMA Treatment: Julie Parsons, MD

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

Phase 4 RESPOND Trial and the Need for Similar Trials in SMA: Julie Parsons, MD