A Harvard Medical School professor and scientist, Sabrina Paganoni, has won the 2019 American Association of Neuromuscular and Electrodiagnostic Medicine (AANEM) Scientific Impact Award for her work in a Phase 2a trial testing fingolimod as a potential treatment for amyotrophic lateral sclerosis (ALS).
Fingolimod is marketed as Gilenya (by Novartis), an oral treatment for multiple sclerosis. It works by blocking signaling from the S1P receptor, which traps immune cells in lymph nodes, and prevents them from reaching the nervous system and causing inflammation. In a mouse model of ALS, fingolimod was seen to significantly extend survival and ease neurological deficits.
Trial (NCT01786174) findings, “Phase IIa trial of fingolimod for amyotrophic lateral sclerosis demonstrates acceptable acute safety and tolerability,” were published in the journal Muscle & Nerve.
The safety study — sponsored by the Massachusetts General Hospital with collaboration from the ALS Therapy Development Institute — included 30 ALS patients (mean age, 55.9); 60.7% were on riluzole (marketed as Rilutek by Sanofi, and Tiglutik by ITF Pharma). They were assigned to either 0.5 mg fingolimod or a placebo daily for four weeks.
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Results showed no serious side effects. Adverse events were similar between the 18 patients given fingolimod and the 10 on placebo, with no clinically meaningful changes in blood counts, urinary analysis, and liver function. Two people, both on fingolimod, discontinued treatment.
Changes in lung function, as assessed by forced expiratory volume in 1 second (FEV1) and FEV1/slow vital capacity were also similar comparing the two groups. But the number of circulating lymphocytes and the expression of nine immune‐related genes – including FOXP3 and CD40LG – were significantly lower in patients on fingolimod.
“I am incredibly honored to be the recipient of the AANEM Scientific Impact [physical medicine and rehabilitation] Award,” Paganoni, MD, PhD, said in a press release. “I focused the first years of my career as a physician scientist on developing expertise in the development of innovative trial designs that can accelerate the path to effective treatments for ALS. My career goals today stay true to that mission.”
Paganoni attributes her interest in ALS research to her mentor, the late physician scientist Lisa Krivickas, who had the disease. Paganoni is the co-principal investigator of the HEALEY ALS Platform Trial, which is “poised to transform the way we test investigational products for ALS,” she said.
This strategy allows researchers to evaluate multiple potential therapies in a single trial, which may allow for their faster development.
AANEM is is a nonprofit aiming to improve knowledge, technology, and education in neuromuscular, musculoskeletal, and electrodiagnostic medicine. The organization’s annual Achievement Awards are given to highlight its members in recognition of work they have done to advance these fields.
Paganoni has been a member of AANEM since 2010.