Help Simmaron AMPlify therapies for M.E.!

Apr 22

Written By

Simmaron is fundraising for its one-of-a-kind study to analyze data from ME/CFS patients who respond to treatments like Ampligen®, IVIG, and cidofovir. The goal of the study is to build a scientific roadmap for getting a first medication approved by the FDA for our disease.

Which patient subset responds to immune-based treatments, like Ampligen and IVIG?

Or anti-viral treatments like cidofovir?

Which subset responds to saline or amino acid infusions?

What measures demonstrate response?

These therapies are currently in use at Sierra Internal Medicine in Incline Village under individualized treatment plans by Dr. Daniel Peterson, and Simmaron’s role is to compare the results of 3 different treatment groups in a prospective analysis that can give us clues to measuring treatment response vigorously.

Collaborators

Simmaron is collaborating with Maureen Hanson, PhD of Cornell, who will lead scientific research on patient samples; the US Centers for Disease Control, which will lead statistical analysis; and Sierra Internal Medicine, where patients receive the treatments.

Fundraising

The funds we raise will pay for data extraction, analysis, and sample preparation to study who responds, what markers track improvement in how patients feel, and what characteristics can be used to refine patient enrollment in double-blind trials. The funds are not covering individuals’ costs for the treatment itself.The study has a budget of $200,000, and some of the elements have secured funding from generous donors so far. We have a goal of raising $20,000 during this fundraising effort!

The Data Study

The Responder Study takes advantage of an unfortunate year-long hiatus in availability of Ampligen, meaning that the patients are being tracked from a baseline of not having that treatment for more than a year and the functional decline that came with the hiatus.The study will include 13 patients who resume Ampligen, 13 who receive other intravenous therapies, and 13 patients who receive no infusion-based treatments, matched for age and sex.Our research team will track the following measures before, during, and at 52 weeks for the different treatment arms:

NK cell (natural killer cell) function
Cytokine expression
Multiple symptom surveys
Exercise tolerance test
Cornell-designed research protocols

Gunnar Gottschalk, Simmaron Research Fellow, describes the urgency of this study, "It is imperative that we publish findings that track an individual’s response to therapy in order to understand this patient population more completely."

NIH Presentation

During NIH’s recent workshop for Young ME/CFS Investigators, Gottschalk presented on the clinical experience of 25 patients treated with Ampligen from 2011 to 2017 at Sierra Internal Medicine. The presentation showed “…significant and sustained clinical reduction in both the physical and neurological symptoms following Rintatolimod (Ampligen®)”, including improvement in physical functioning, pain and energy after 6 months of treatment.

Informing A Pathway to Approval

We know first-hand that patients need well-designed treatment trials to achieve FDA approval for a first ME medication. Rigorous data analysis, starting with this study, can provide a building block to designing rigorous placebo-controlled trials that have a stronger chance of approval, and ultimately attract pharmaceutical companies to the disease.Simmaron is leading the effort to turn science into treatments for patients, and your support powers our work. Help us reach our goal of raising $20,000 in the next month to fund an important part of this study!