Cure SMA is pleased to announce $5 million in new research funding over the next 12 months. This funding will be used strategically to help accelerate research and ensure we are developing treatments for all types, ages and stages of SMA. This funding will also be used to increase patient access to treatments and improved optimal clinical care.
In total $2.7 million of this funding—over half of the $5 million total—will go directly to local support and care, while the remainder will fund basic research, newborn screening, and clinical and regulatory initiatives.
As the SMA research landscape has developed and the drug pipeline has grown to include the first-ever FDA approved treatment, the needs for SMA research have also grown and developed. This $5 million investment will guide us into the next phase of SMA research and care, by funding the areas of greatest need, and where we are best positioned to make a significant difference.
$900,000 in Continued Funding for Basic Research
Basic research investigates the causes and biology of SMA, often revealing more effective ways of making SMA drugs. Continued funding in basic research will help address questions about survival motor neuron (SMN) protein, which is not produced properly in the bodies of those with SMA, and help us identify other systems, pathways and processes that are affected in SMA.
This research could then lead to the development of combination therapies for SMA, using both our knowledge of SMN protein and our knowledge of these other systems, pathways and processes. Approaches that work on these other areas could be used in combination with approaches that work on SMN levels, allowing us to develop treatments for all types, ages, and stages of SMA.
$1 Million in Continued Funding for Clinical and Regulatory Research
As the SMA drug pipeline continues to grow in depth and diversity, so does our need to address clinical and regulatory issues and continue to bring the patient voice into the process. Funding for this area will be directed toward several critical initiatives:
- Engagement with the FDA and other regulatory agencies
- Increased education and awareness to parents, families and the medical community to reduce diagnostic delays
- Expanding capacity for new clinical trial sites
$2.2 Million for Clinical Care Centers
Now that a treatment option is available, it is imperative to improve patient care and quality of life. With this in mind, Cure SMA is launching a network of Cure SMA care centers.
These centers will electronically submit information to the newly created Cure SMA clinical data registry. The registry will be used to answer questions about best health care for SMA by examining care practices across centers. Centers will receive center-specific feedback to improve care at their site. The Network will begin with 10 centers and then rapidly expand. Our goal is to improve the standard of health care for everyone with SMA.
$100,000 to Train Healthcare Providers to Provide Quality Care for SMA
Cure SMA is also funding a new Therapist Mentoring Program in order to improve care in the local community for those affected. The purpose of this program is to:
- Provide information, experiences and resources for physical therapists and occupational therapists who are new to SMA, or are seeking additional experience
- Increase access to therapists and pulmonologists with the expertise to evaluate individuals with SMA
$400,000 to Improve Access to Treatments and Care
In order to increase capacity to follow, treat and evaluate those affected by SMA, Cure SMA will award $50,000 grants to nine administration sites. These grants will help sites reduce some of the practical barrier that create delays for our community in accessing treatments and care . These barriers include pre-authorization and insurance approvals, scheduling, and clinic support.
$400,000 for SMA Newborn Screening Initiatives
With a treatment option now available and data indicating that pre-symptomatic treatment improves clinical outcomes, it is vitally important that SMA be added to state newborn screening panels as quickly as possible. Resources are also needed for state public health labs in order to implement newborn screening for SMA. Adding SMA to the Recommended Uniform Screening Panel (RUSP) is a critical step toward our goal of having SMA added to newborn screening programs across the US. In May, the RUSP application was unanimously accepted into the evidence review stage, with a decision expected in six to nine months.
$100,000 for Data Projects to Support Increased Access and Approvals
For the past several years, Cure SMA has been collecting data and information on the experiences of living with SMA. Earlier this year, we released a survey for all individuals and families that have reached out over the past decade to gather how SMA has impacted their lives. In the coming months, we will release a similar survey to track any changes in the burden of SMA over the past year, now that an approved SMA treatment is available.
The dedicated support of our community has made all of this possible. Whether families raising funds and awareness, researchers investigating new potential treatments, or pharmaceutical and regulatory partners helping us take the next steps toward future approvals – everyone has a role to play. Thank you to everyone for their hard work and dedication.