An increasing amount of research on drugs to treat spinal muscular atrophy (SMA) is successfully progressing as a consequence of clinical trials, and Cure SMA believes there is a need to inform and educate the community about the clinical trial process. To do so, the nonprofit organization has released a new online and open access booklet entitled Learning About Clinical Trials.
This is another one of the organization’s publications included in its care series booklets, which are being released to increase the knowledge and understanding on the chronic and disabling disease. In this edition, Cure SMA seeks to offer the community accurate and unbiased information on clinical trials, in order to address specific unmet needs.
The educational effort was developed by a panel of expert reviewers, including members of Cure SMA’s Medical Advisory Council. Learning About Clinical Trials introduces and explains the clinical trial process, while providing insights on the unique challenges and opportunities associated with conducting research for novel SMA treatments.
Cure SMA explains that the scientific investigation currently on course is based on a series of different therapeutic approaches. There are investigational therapies expected to target the underlying genetics,while others attempt to protect nerves or muscles to delay or stop disease progression. THis means that researchers need to test such approaches on patients with different ages, stages, and types of SMA, which can prove challenging when it comes to trial design.
SMA is classified as an orphan disease, which means it is rare with a patient population less than 200,000. As such, clinical trials can only gather a small number of patients when compared to trials for non-orphan diseases. On the bright side, these trials are usually eligible for special incentives from the U.S. Food and Drug Administration (FDA). When it comes to patient enrolment, one needs to remember that most SMA patients are very young, making it imperative that these trials pay special attention to additional indicators while testing infants or children.
Cure SMA care series booklets are available online and will be distributed during the organization’s Annual SMA Conference.