Guest Commentator: David J. Chang, MD, MPH, FACR, Chief Medical Officer, Cabaletta Bio @Cabalettabio
Why is it important for cell and gene therapy companies to establish meaningful collaborations with patient advocacy organizations?
Cell and gene therapies represent novel treatment approaches with strong roots in the region. Several companies have been established based on the research conducted at Philadelphia institutions. To advance these therapies into clinical trials with patients, establishing close collaborations with patient advocacy organizations (PAOs) is important for several reasons.
First, because these therapies are relatively new, patients are largely unaware if they are even available for their specific disease. The source for information regarding the latest in therapies for many patients comes directly from PAOs, which often maintain a listing of ongoing clinical trials and new therapies.
Second, the process for patients to receive cell or gene therapy is more complicated than taking a pill. These therapies require injections, are administered in combination with other strong medications such as steroids or immunosuppressive agents, and may also involve the collection of patients’ cells to manufacture the product. PAOs serve an important function in explaining to patients, in lay terms, how these drugs work and are administered, thereby giving patients information to determine if they should consider participating in a clinical trial.
Finally, some of the targets for these therapies represent rare diseases or diseases without good treatment options. PAOs can thrive and make the greatest impact in these disease areas since these patients will gravitate towards organizations that provide resources for information on the disease and treatment, connection with peers, and contact with physicians and researchers. Through collaborations with these PAOs, cell and gene therapy companies can also benefit from these organizations because they spread the word about the companies’ research and clinical trials. For example, at Cabaletta Bio, the progression of our cell therapy treatments in two rare diseases, mucosal-dominant pemphigus vulgaris, and MuSK myasthenia gravis, into clinical trials have relied on partnerships with the PAOs, the International Pemphigus and Pemphigoid Foundation, and the Myasthenia Gravis Foundation of America. We were in early contact with these groups, discussed the goals of the relationship, and outlined how we would mutually achieve those goals. These organizations have provided us with the opportunity to present at patient information webinars, write articles for newsletters, and give talks at conferences.
Cell and gene therapies signify the latest in potential breakthrough treatments for certain diseases, but to develop these therapies into approved drugs, the support and collaboration of PAOs is invaluable.
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