Understanding
Drug Development
ALSP is a rare and newly-characterized disease, making any additional insight extremely valuable to the teams working on potential treatments. Members of the ALSP community can actively contribute to - and even accelerate - the drug development process by sharing experiences and perspectives.
There are currently no FDA-approved therapies for ALSP. Existing care and management practices focus on symptom relief and maintaining quality of life. Commonly prescribed treatments do not target the cause or slow the progression of ALSP.
Therapies that are not yet approved by regulatory agencies like the FDA are called investigational. There are five stages of drug development, and the results of clinical studies will determine whether an investigational drug will be approved and can be prescribed to patients by their doctor.
“My first experience with ALSP started back with my father who was diagnosed with it. At the time I had zero interest in getting tested. Four years ago, I came to the decision that regardless of whether I have the mutated gene or not, there could be information I could provide that might help with research that hopefully helps my generation but also the next generation.”
Adam, living with ALSP
As experts in what it is like to live with and care for someone with the condition, patients and caregivers are uniquely positioned to inform the understanding of the therapeutic context for drug development and evaluation. There are many reasons and ways to work together with companies and patient advocacy organizations to form and foster productive, lasting relationships. Collaboration is the foundation for achieving patient-focused therapeutic development.
Sharing your experience is critical at each phase of development and allows stakeholders such as drug developers and regulatory agencies to better understand the unmet needs of the ALSP Community.
Are you living with ALSP or have you been diagnosed with a variant in the CSF1R gene?
Learn about opportunities to participate in research and ongoing clinical trials for ALSP at www.alspstudy.com and www.clinicaltrials.gov.