Buffalo Initiative

Buffalo Initiative Inc.

Of the estimated 10,000 rare diseases impacting approximately 30 million people in the United States and 400 million around the world, 80% are genetic, 70% start in childhood, 75% have neurological symptoms and 95% do not have a single FDA-approved treatment. Children living with these diseases have a significant and disproportionate impact on the resources and costs in the healthcare system, estimated at 10 times higher than common diseases. The financial and emotional burden on families is immense, and their tremendous unmet need for life-changing therapies cannot be overstated. 

Fortunately, remarkable advances in diagnostic tools, data collection technologies and cell and gene therapies make the development of curative treatments for rare pediatric brain diseases possible on a scale we have never seen before. However, these advancements are not translating into treatments quickly enough for patient communities living with the burdens of these diseases for three main reasons: 

(i) Market failure - The need for the biopharmaceutical industry to achieve an adequate rate of return is at odds with the small market size of these diseases, the high development costs of cell and gene therapies and a regulatory framework that was not designed for rare diseases. 

(ii) Lack of a patient-centric approach - Despite their expertise and sophistication, patient communities and their advocates have not been engaged as true partners in the traditional scientific-expert-driven research and development model. Research funding is primarily given to a narrow circle of credentialed scientists, often working in silos and not connected to the research agendas of patient communities. Sadly, current patient engagement efforts by the medical research and development enterprise remain largely extractive and tokenistic. 

(iii) Collaborative and open science principles have not been mainstreamed -  Many academic scientists are hesitant to share unpublished tools and data because of career impact concerns, which leads to fragmented efforts, slow progress and duplicated research.

While changing feeding tubes, stopping seizures, and witnessing children lose milestones, parents and caregivers cannot afford to wait for solutions so they are creating them. Parents and caregivers are increasingly orchestrating therapeutic development efforts through patient organizations because the return on their investment is their children’s lives. Their prioritized research agendas are guided by the experiences, relationships, data and intimate knowledge of the community. However, the scale and impact of their efforts are largely dependent on the financial resources available within their patient communities because they are not well supported by federal research dollars, institutional philanthropy or existing umbrella rare disease organizations. Furthermore, while therapeutic development for many childhood brain diseases share the same underlying challenges, most research and development efforts are not coordinated across diseases or the estimated 500 patient groups focused on childhood brain diseases and existing shared infrastructure is not adequate, necessitating a need for a new organizational model.

Buffalo Initiative is a collaborative therapeutics accelerator for rare pediatric brain diseases that will synthesize funding, efforts and knowledge across the ecosystem with a singular goal in mind – more treatments for more kids. It is comprised of three main programmatic activities:

1. Cure Chaser Fund

One of the key elements of the accelerator is the establishment of a centralized funding pool from philanthropic and private sector contributions that will enable patient organizations to align researchers and the biopharmaceutical industry towards the priorities of patient communities and to engage in the therapeutic development process as full partners. 

Patient leaders have a proven track record and competitive advantage in therapeutic development because of their lived experience, access to patient populations, ability to build collaborative and cross-institutional research teams and intrinsic motivation. But even the most motivated and resourced among them get stuck at a stage that is aptly named the “valley of death.” This term refers to the phase between safety and efficacy experiments in animals and treating patients. The Cure Chaser Fund will provide between $2M-$8M in funding per organization for either (i) cross-cutting disease research to address challenges unique to genetic strategies for treating pediatric brain diseases or (ii) pre-specified studies needed to get through the “valley of death” to first-in-human clinical trials.

2. Shared Infrastructure & Strategic Partnerships

A team of technical consultants who are specialized in translational research, therapeutic development, manufacturing and regulatory requirements will be established to streamline the development of new treatments across the organizations and their research networks. In addition, Buffalo Initiative will leverage its strategic partnerships with academic institutions, biopharmaceutical companies and regulatory agencies to provide access to advanced technologies, manufacturing platforms and clinical trial networks. Shared infrastructure and resources will reduce the financial burden on individual projects and expedite the development process. 

Finally, Buffalo Initiative plans to have a pipeline of commercial partners experienced in drug development who are committed to co-investing and sharing intellectual property rights with patient organizations taking translational proof-of-concepts to phase I/II clinical trials.

3. Knowledge Platforms

Buffalo Initiative will champion open science principles, requiring patient organizations and partners to share data, findings, failures and methodologies. By fostering a culture of transparency and collaboration, we can eliminate redundant efforts and accelerate progress. A secure, centralized data repository will be created where organizations and partners will deposit and access data, ensuring that valuable insights are not siloed. This approach will also facilitate meta-analyses and the identification of patterns across different studies and diseases.  

In conclusion, Buffalo Initiative is a new organizational model that fosters a unified, efficient and well-resourced approach for patient-centric therapeutic development. The ultimate goal of this collaborative therapeutics accelerator is to bring effective treatments to clinic faster, improving the lives of families affected by rare pediatric brain diseases. By synthesizing funding, efforts and knowledge, we can overcome the systemic issues that currently impede progress to effective genetic treatments.

Buffalo Initiative serves several key groups: children living with severe and life-threatening genetic brain diseases and their families, patient organizations focused on advancing treatment development for childhood brain disorders and the research community.

Buffalo Initiative aims to expedite the development of effective treatments for these children. Cell and gene therapy advancements are not translating into treatments quickly enough for patient communities living with the burdens of these diseases. By pooling resources and fostering collaboration in ways that do not currently happen, Buffalo Initiative will streamline individual research efforts, reduce redundancies and fast-track the development of new therapies. This means faster access to life-changing treatments, improved health outcomes and enhanced quality of life for these children and their families.  

By connecting patient organizations across diseases and investing in scientific enterprises driven by patient organizations and their research networks, Buffalo Initiative enhances each organization’s likelihood of success. These groups are de-risking science, identifying patients, organizing research networks, developing tools and data resources, empowering participation in clinical research, and defining the objectives and end points that are most meaningful to patients, but they are not being adequately supported by institution stakeholders. Despite their ability to accelerate research, patient organizations are not recipients of federal research dollars and with few exceptions, they are not well supported by institutional philanthropy or existing umbrella disease organizations.

Lastly, Buffalo Initiative will enhance research efficiency, improve access to patient-focused research assets, foster collaboration and reduce duplicative efforts, which will enable researchers to produce innovations more quickly. 

Buffalo Initiative is in the prototype stage because we have formed the organization and assembled the core team. We have also publicly presented the effort and met with numerous advisors, patient organization leaders and potential funders to lay out key milestones over the next three years. We are in active discussions with the founding funders and key opinion leaders in the philanthropic and biopharmaceutical space about launching the pilot stage of the work, which will entail funding the first set of pre-specified studies.

Buffalo Initiative is innovative for two key reasons. 

1. Buffalo Initiative is advancing a different approach to therapeutic development for childhood brain diseases. It is the first-ever initiative to scale efforts driven by patient organizations and their collaborative research networks (“community-led research efforts”) to bring de-risked therapies from pre-clinical to clinical trials in a patient-focused manner. At Buffalo Initiative, we believe that community-led research efforts can help deliver valuable, transformative and measurable breakthroughs that are not being attempted at traditional scientific institutions. Patient organizations have a proven track record and competitive advantage in driving therapeutic development because of their lived experience, connectivity to patient communities, and intrinsic motivation. No one has more at stake than parents of sick kids. Despite this, the vast majority of funding goes to research and researchers that are not connected to the research agendas of patient communities. As a result, remarkably little translational research has occurred to date for pediatric genetic brain diseases. 

   Despite expanding knowledge of the molecular genetics and clinical presentation of these diseases, lack of prospective clinical data, patient-derived biospecimen repositories, and inadequate communication across research groups and clinicians severely hampers progress. No other stakeholder in this ecosystem is better positioned to convene all relevant stakeholders in an impartial, patient-centric way than patient organizations.

2. Buffalo Initiative transforms current disparate and isolated patient organization efforts into a coordinated, efficient and impactful whole. To date, there have been no systematic, large-scale efforts to advance genetic strategies to treat pediatric brain diseases in the clinic even though there are common challenges and similarities. There has been an abundance of basic research, but true progress in understanding and treating these diseases is impeded by a lack of coordination. Buffalo Initiative is a unified platform for collaboration that acts as a central hub to align goals, streamline processes and ensure all efforts are directed towards the priorities of patient communities. A well-organized, patient-driven therapeutics accelerator would consolidate our current understanding of the disorders, identify patterns and knowledge gaps across diseases, centralize patient data and biospecimens, align stakeholders, and accelerate translational research towards improved diagnostics and effective treatments. Right now, one of the only ways to share discoveries is through research publications. By promoting open science, Buffalo Initiative aims to shape this endeavor to be collaborative rather than competitive, sharing rather than siloed. Buffalo Initiative ultimately enables breakthroughs for one brain disease to be leveraged to accelerate breakthroughs in other brain diseases.

We are applying to the Amgen Prize because of the validation and exposure it will bring to our work of redefining the role of patient communities in the therapeutic development ecosystem. This prestigious award can help us overcome barriers rooted in bias and skepticism that our patient communities face in the scientific and therapeutic development ecosystem. It will significantly elevate the visibility of our new patient-centric therapeutics accelerator and help us attract attention from key stakeholders across the philanthropic and clinical research sectors. As an award recipient, we would be invited to speak at high-profile events that will amplify a paradigm shift towards community-led efforts and make it easier to attract additional funding. This increased visibility will also help us build credibility and attract additional strategic partnerships and funding, creating a ripple effect that will amplify our impact.

“There is no treatment. Just go home and love her.” This was the advice I received from a pediatric neurogeneticist after diagnosing my daughter with a rare brain disease caused by a change in a single gene in her body. It is the same advice given over and over again to parents and caregivers as they are hurled into the rare disease community. When your child is diagnosed with a disease only known as a series of letters and numbers that do not exactly roll off anyone’s tongue, it becomes your diagnosis too. You feel like you’ve been back into a corner, and you must and you will do anything you can to solve this for your child. 

From the very beginning of the diagnostic odyssey to the day of diagnosis, you are constantly reminded that no one is going to solve this for you. No one has more at stake than us. Each of us faces a ticking clock. We must find something to save our child or we need to get them healthy enough so that their siblings do not have to face a massive caregiving burden after we are gone. These are our realities. 

The questions we are asking aren’t being answered, and the solutions we seek aren’t being worked on. So you can only imagine why there has been a groundswell of parents across the rare disease community over the last decade who have taken it upon themselves to roll up their sleeves and enter the world of drug development after their child receives a similar diagnosis as I did. 

The Buffalo Initiative was founded when a group of patient leaders with decades of combined experience in finance, law, and biotech and in running their own sophisticated disease foundations came together to create a viable path forward for developing therapies for genetic brain disease. We are each parents of children with different genetic brain diseases whose commitment to this work is unwavering because the return on our investment is our children’s lives. As parents and patient leaders that are trusted members of the communities we aim to serve, we have been able to identify and prioritize the unmet needs of our communities that are overlooked by other stakeholders in the therapeutic development system. We have each been focused on efforts that will have the greatest impact on children’s lives, and to date, we have been braving the path of drug discovery and development alone. Only to realize, later on, that by combining forces, we could push not only our individual disease areas forward faster and more efficiently but also create a sustainable funding model for the most promising therapeutics for childhood brain diseases. 

2024-2025 Impact Goals:

• Adopt key principles and definitions of “differential impact” with governing board
• Issue impact survey and publish white paper on community-led therapeutic discovery
• Secure funding for Cure Chaser Fund and develop RFP process
• Formalize partnerships with biopharmaceutical partners, philanthropic advisors and influencer platforms

2025-2030 Impact Goals:

• Stakeholders see "differential impact"
• Board meetings are used to engage, educate and influence each other and the field
• Board provides platform for intelligence gathering about emerging trends
• Build out evaluation metrics and data sharing platform
• Deploy $80M by year 5
• Strategic partnerships are effectively advancing programmatic goals
• Successful lead generation and advisor-influenced fundraising from strategic partnerships
• Disseminate data & evaluation from first 5-years (publications, speaking engagements)
• Present model at influential convenings (e.g., Milken, Davos, SXSW, Aspen Ideas)
• Identify potential commercialization approaches based on data and evaluation

Our theory of change revolves around the central belief that having patient communities lead at every stage of the therapeutic development process will lead to more effective, efficient and patient-centric treatments. This approach transforms the traditional development process by prioritizing patient needs, experiences and insights, thus addressing the unique challenges families face when their child or loved one is diagnosed with a rare brain disease with no existing treatment options.

Inputs

1. Patient Advocacy Groups:

• Bring firsthand knowledge and a deep understanding of patient needs.
• Mobilize the patient community and provide valuable data on disease impact.

• Include researchers, clinicians, industry partners, and regulatory bodies.
• Foster an environment of open communication and resource sharing.

• Secure financial support from philanthropic organizations, grants, and donations.
• Utilize resources for patient engagement, research, and development activities.

Outputs

1. Increased Patient Involvement:

• More patients and families actively participating in research and development processes.
• Higher quality and relevance of patient-reported data influencing research directions.

• Accelerated discovery of therapeutic targets due to collaborative data analysis.
• Reduction in duplicated efforts and more efficient use of resources.

• Therapies that better address the specific needs and preferences of patients with rare brain diseases.
• Improved clinical trial designs that are more inclusive and representative of the patient population.

In addition the Founder, our team is made up of four founding advisors and three strategic advisors. We do not currently have any paid team members.

Th concept of Buffalo Initiative was first created in October 2023. The organization was officially incorporated in March 2024.

Currently, our team is 88% women, geographically and racially diverse, with 50% of the team reflecting members of Asian descent, and 60% are representatives of the patient community. As we continue to grow our team and bring on paid staff, we will be intentional in our outreach and recruitment to increase our team's diversity. Buffalo Initiative plans to put in frameworks that enhance aspects of diversity, equity, and inclusion (DEI) within our organization. 

We are in advanced discussions with foundational funders who are interested in providing the funding for the first three years of operational expenses and leveraging their influence to bring other science and research funders to the collaborative, especially to seed the Cure Chaser Fund. Longer term, Buffalo Initiative will have processes and team members in place to file patents to protect IP, identify IP licenses and negotiate license terms for the projects it funds. Buffalo Initiative plans to use revenue from licensing fees and royalties to sustain our work.