The progress and outcomes of new treatments are important to us all, but arguably no one is more invested in the discovery of new therapies than patient advocate groups. Made up of passionate individuals who voluntarily organize to advocate on behalf of those with a particular disease or disorder, these ‘disease advocates’ dedicate their lives to bringing positive changes for others, sometimes including their families and themselves.
The personal experience of living with a disease or caring for someone who has one, brings a unique perspective that, together with a motivation for change, can lead to valuable contributions to the scientific process. These contributions can include advocating for more resources or relevant legislation from governmental agencies, forming action groups to raise awareness, or establishing up support groups to help others afflicted with the disease.
This influence can also extend into the laboratory, when patient groups partner with scientists to provide research samples, financial support and the motivation to drive discovery. At the ISSCR 2016 meeting in San Francisco, attendees heard two inspirational stories from Kristin Macdonald and Adrienne Bell-Cors of what it’s like to live with diseases such as retinitis pigmentosa (RP), and sickle cell disease, respectively.
The impact that an advocate can have is evident by viewing the U.S. Food and Drug Administration’s (FDA) Patient-Focused Drug Development Initiative. This impressive library called “The Voice of the Patient Reports,” chronicles patient perspectives on more than 20 different diseases. These reports are critical in the FDA’s regulatory decision-making process.
Additional ways that patient experiences can help advance potential therapies is modeled by a group called ALSUntangled [link out]. This consortium of researchers, clinicians, and patients, has “systematically investigated and graded” evidence for over 35 “alternative” treatments for Amyotrophic Lateral Sclerosis (ALS, aka Lou Gehrig’s disease), a fatal disease that destroys the nerve cells that control voluntary muscle movement. This group talks to patients, and reviews scientific and clinical claims, medical notes and online reports, and other data, to establish an evaluation of each potential treatment. Their analysis can be helpful to scientists and others looking for pathways to effective treatment as well as patients seeking answers to whether these alternative therapies really work.
This model could help provide some clarity for patients, scientists and clinicians regarding the treatments that patients are obtaining at clinics offering unproven stem cell therapies. These clinics are popping up all over the world, offering purported stem cell treatments for a variety of medical conditions. Hopeful patients, many viewing these clinics as a last chance to treat an intractable disease or chronic injury, are seeking out these treatments, while many others, including the medical community, are seeking answers to the question of “do they work?” The ability to pool and objectively evaluate patient outcomes could provide some much-needed clarity to evaluate these interventions.
The ALSUntangled model doesn’t provide the same level of review that would come from a regulated clinical trial, but it can help. By considering patient experiences and feedback, researchers and advocates alike can learn more about the safety and efficacy of potential stem cell therapies.
Patients who participate in testing experimental stem cell therapies, whether in regulated clinical trials or from clinics offering unproven therapies, are pioneers. They are taking a risk for themselves and others, and their experiences, if properly utilized, can help move the scientific process forward.
Disease advocates and the medical community all want the same thing when it comes to cellular therapies – safe and effective treatments. As the stem cell field works toward developing treatments for RP, sickle cell and other diseases, the pursuit is strengthened and the process enhanced by the partnership of patients and disease advocates.