At the 2016 Annual SMA Conference, Cure SMA announced $2.5 million in new planned 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.
As the SMA research landscape has developed and the drug pipeline has grown, we recently undertook a systematic review of our research funding priorities. Through conversations with independent SMA experts, our scientific advisory groups, and the newly formed Medicine and Science Committee in our Board of Directors, Cure SMA has created a strategic research plan to guide us into this next phase of SMA research. This strategic research plan identifies the areas of greatest need and where we are best positioned to make a significant difference.
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.
Greater Funding for Clinical and Regulatory Research
As more SMA drug programs progress through clinical trials, there is an increasing need for us to address clinical and regulatory issues and bring the patient voice into the process. Funding for this area will be directed toward several critical projects:
- Developing regulatory approaches to patient-focused drug development
- Defining clinical meaningfulness and risk/benefit in ways significant to our community
- Holding a Voice of the Patient Meeting with the FDA
- Standardizing and training for clinical trial protocols across sites
- Educating and engaging physicians, caregivers and patients
Many of these projects will be carried out as part of a new a collaborative industry consortium. Through this group, seven companies working in SMA drug development will share information, ideas, and data, working together on projects that will benefit our community.
Greater Funding for Patient Care Initiatives
Cure SMA has been working for the past several years to collect data and information on the experiences of living with SMA. Earlier this year, we expanded our ongoing data collection efforts by launching a survey tool for those newly diagnosed with SMA. In the next several months, we will announce a second survey targeted toward individuals and families not newly diagnosed.
A portion of our research funding for the coming year will be used to gather these responses and create a database that will demonstrate the impact of SMA over time. This information will help the scientific and research communities create answers that address these real-world concerns, and accelerate therapy development for SMA.
The increased funding will also be used to help develop centers of excellence for SMA. Information from multiple SMA care centers will be used to create evidence regarding the best care for all those affected by SMA.
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 approval—everyone has a role to play. Thank you to everyone for their hard work and dedication.