Cure SMA to Put $2.5 Million Toward Research Efforts

Cure SMA, an organization dedicated to the treatment and cure of Spinal Muscular Atrophy (SMA), will provide $2.5 million over the next 12 months for research and to ensure the development of therapies for all ages, types, and stages of SMA. The announcement was made during the organization’s 2016 Annual SMA Conference.

SMA, characterized by progressive muscle weakness that eventually results in respiratory and mobility impairment, can occur in variable degrees of severity. Four SMA categories are named according to the age of the patient at initial onset of muscle weakness and related symptoms: Type I (Infantile), Type II (Intermediate), Type III (Juvenile) and Type IV (Adult onset).

Cure SMA recently conducted a systematic review on its research funding priorities. Through talks with scientific advisory groups, the Medicine and Science Committee and independent SMA specialists, the organization created a strategic research plan to identify the areas of greatest need. The areas include:

• Continued funding for basic research on the biology and causes of SMA, particularly focusing on the survival motor neuron (SMN) protein which is impaired in patients with the disease. A better understanding of the SMN protein and the identification of impaired systems, pathways and processes involved in the disease, provide the scientific basis for developing future SMA drugs. This knowledge could potentially be used in combination with other approaches to develop treatments for all types, ages, and stages of the disease.

• Increase funding for SMA clinical trials, address regulatory issues and involve patients in the process. Funding will be used in projects developing regulatory methods for patient-focused drug advance; to define clinical relevance as well as risk and benefit to the SMA community; to hold an official Voice of the Patient Meeting with the FDA; for the standardization and preparation for clinical studies procedures across clinical settings; and to engage and educate patients, caregivers and doctors. Many research projects will be conducted through a newly created collaborative industry consortium in which seven SMA drug development companies will share ideas, data and information and work together on projects to benefit the SMA community.

• Greater funding for patient care initiatives: Over the past several years, Cure SMA has been collecting information on what is it like to live with the disease. Earlier this year, the organization launched a survey tool for newly diagnosed people with SMA. A second survey targeting individuals and families not newly diagnosed, will be launched in coming months. In the coming year, Cure SMA will use funds to gather patients, families and SMA advocate responses into a database to demonstrate the impact of SMA over time. This information will help the research community create answers to common concerns and accelerate the development of treatments for SMA.

The newly announced funding will also be used to create SMA excellence centers.