The Scope of Academic and Industry Sponsorship in Shaping the Future of AL Amyloidosis Research

Background:

Systemic Immunoglobulin light chain (AL) amyloidosis is a rare plasma cell dyscrasia characterized by the deposition of AL chains in various organs. The prevalence of AL amyloidosis has increased in recent decades due to improved diagnostic techniques. Despite the critical role of funding agencies in guiding research efforts, the relationship between the types of funders and specific health conditions, such as amyloidosis, remains underexplored in current literature. Both the industry and academic sectors have a shared goal of broadening treatment options and improving outcomes for patients. The objective of this study is to identify the primary sources of funding and assess their impact on research in the field of AL amyloidosis. It examines the pivotal role of academic, industrial, and National Institutes of Health (NIH) sponsorships in advancing AL amyloidosis research and influencing clinical trial outcomes. Based on our results we anticipate potential shifts in funding patterns in the future.

Method:

We conducted a comprehensive search of amyloidosis clinical trials in the United States on ClinicalTrials.gov from January 1980 to February 2024 yielded 470 trials. 117 trials were included for analysis after excluding observational studies, studies on types of amyloidosis other than AL amyloidosis, and studies involving children. Of these 36 studies were automatically categorized, while the remaining 81 were manually categorized. The trials were divided into academic, industry, and NIH sponsored groups.

Result:

In our analysis of 117 trials, we found that 74 (63%) trials were sponsored by academic institutions, 27 (23%) by Industry partners, and 16 (14%) received funding from the NIH. Phase 2 trials were the majority, totaling 51 (44%) of all trials, while the Phase 4 trials were the least common, comprising only 4 (3%) of the total. Additionally, 9 (8%) trials were categorized as N/A phase, typically involving trials without a defined phase, such as those focused on devices or behavioral interventions. Looking specifically at phase 2 trials, 33 (64%) were sponsored by academic institutions, 9 (18%) by industry, and 9 (18%) by the NIH.

Our analysis revealed a decline in the number of academically sponsored trials. When comparing the periods 2021-2024 and 2011-2015, the percentage of industry- sponsored clinical trials increased from 12% to 40%, while the percentage of academically sponsored trials decreased from 71% to 57% of all trials. In contrast, NIH sponsored trials have maintained a steady pace since their inception in 1996.

In terms of patient enrollment, a total of 6441 patients were enrolled in trials from 1996 and 2024. Among these, 3099 (48%) patients were enrolled in academically sponsored trials, 2703 (42%) in industry-sponsored trials, and 639 (10%) were part of NIH-sponsored trials. Notably, there has been a significant surge in patient enrollment, particularly between 2021 and 2024, with 2176 (34%) patients enrolled in trials, particularly within the academically and industry-sponsored categories.

Upon reviewing the trial statuses, we found that 53 trials (45%) have been completed, 22 trials (19%) are actively recruiting, 21 trials (18%) have been terminated, and 18 trials (15%) are active but not recruiting. It is noteworthy that most of the completed trials, 38 (72%), were academically funded. However, our analysis did not yield any substantial correlation between the nature of funding and the probability of clinical trial completion (P-value= 0.069).

Conclusion:

At present, academic institutions are sponsoring most AL amyloidosis trials. However, recent trends suggest that industry-sponsored trials are changing the standard of care and projected to surpass academia in the next decade, reflecting a notable rise in industry-sponsored clinical trial activity with AL amyloidosis. The benefits derived should be viewed as complementary and mutually reinforcing rather than competitive.

Ahmed:Bristol Myers Squibb: Consultancy; Kite, a Gilead Company: Research Funding. Mahmoudjafari:Janssen: Consultancy; Sanofi: Consultancy. Mushtaq:Iovance Biotherapeutics: Research Funding. Lutfi:ADC Therapeutics: Membership on an entity's Board of Directors or advisory committees. McGuirk:Kite: Consultancy; CRISPR therapeutics: Consultancy; NEKTAR therapeutics: Consultancy; Caribou bio: Consultancy; Sana technologies: Consultancy; Legend biotech: Consultancy; Novartis: Consultancy; Allo Vir: Consultancy; Envision: Consultancy; Autolus: Consultancy; BMS: Consultancy. Anwer:BMS: Consultancy. Bhutani:Janssen: Research Funding; BMS: Research Funding; Takeda: Research Funding; Caribou Biosciences: Research Funding; Amgen: Research Funding; Abvvie: Research Funding. Atrash:Karyopharm: Research Funding; Janssen: Honoraria; Amgen: Research Funding; GSK: Research Funding.