Sometimes the most powerful innovations come from the most personal places. This episode tells the extraordinary story of Danyelza (naxitamab), a life-saving immunotherapy for children with neuroblastoma, and how it earned a finalist spot in the AUTM Better World Project.

What makes this story truly remarkable isn't just the science, although that's impressive enough. It's the unlikely partnership between a researcher who refused to give up, a tech transfer team that believed in an "ultra-orphan" drug when no one else would, and a father who turned his desperation into determination by founding a company to bring this therapy to other children facing the same devastating diagnosis as his daughter.

Joining me are three key figures from Memorial Sloan Kettering Cancer Center who shepherded this breakthrough from lab bench to bedside: Dr. Nai-Kong Cheung, the Enid A. Haupt Chair in Pediatric Oncology and a world-renowned expert in antibody-based therapies for childhood cancers; Dr. Yashodhara Dash, Vice President of Entrepreneurship & Commercialization at MSK; and Dr. Imke Ehlers-Surur, Director of Technology Development & Licensing, who negotiated one of the most unconventional licensing deals in tech transfer.

We discuss the 25-year journey from early mouse antibodies to FDA approval, why pharma companies initially passed on this technology, how regulatory designations like the rare pediatric disease priority review voucher changed the business case, and what happened when MSK had to decide which patients would receive limited drug supplies a moment Dr. Cheung compares to Schindler's List.

Disclosure: MSK and Dr. Cheung have financial interests in Danyelza.

In This Episode:

[03:07] Dr. Cheung explains neuroblastoma is a devastating childhood cancer that spreads to bone, bone marrow, and other organs, making it one of the most difficult pediatric cancers to treat.

[04:31] The "aha moment" came in the mid-1990s with antibody 3F8, when the team saw how these antibodies could light up tumors and eliminate metastatic disease.

[07:00] The early mouse antibody got rejected by the body, so the team used protein engineering to create a humanized version that could arm the immune system without rejection.

[08:14] Funding was one of the toughest challenges working with small budgets meant relying heavily on internal grants and parent groups like the Band of Parents.

[09:31] Dr. Dash describes the commercial landscape as a "hard sell". It was an ultra-orphan market, and companies wanted small molecules instead of antibodies.

[10:22] Persistence and entrepreneurial mindset kept the project going internally, applying for FDA designations and advancing the technology before finding the right partner.

[11:01] Dr. Ehlers reveals Y-mAbs Therapeutics was founded by Thomas Gadd, the father of one of Dr. Cheung's patients, who built a company when other paths stalled.

[12:07] Working with a founder who had unparalleled motivation but limited drug development experience meant MSK developed a forward-looking commercialization strategy.

[14:28] The biggest difference in negotiating with a patient-family founder versus traditional VCs was making sure both sides were speaking the same language.

[17:34] While MSK's Technology Development Fund provided some gap funding, the real story was philanthropic support from groups like the Band of Parents.

[18:42] The conversation turns to FDA accelerated approval in November 2020, after running out of drugs and having to decide which patients would benefit a moment compared to Schindler's List.

[21:12] To avoid conflicts of interest when helping form Y-mAbs in 2015, stepping out of the clinic completely became necessary to focus on research full time.

[24:19] The regulatory designations were "transformative" orphan drugs given seven years of exclusivity, and the rare pediatric disease designation came with a priority review voucher worth $80-300 million.

[27:18] Without these regulatory incentives, the startup may not have been fundable at all, though Thomas Gadd is noted as a "force of nature."

[28:18] Y-mAbs' acquisition by CERB Pharmaceuticals means proceeds will flow back to MSK to fund future cancer research, a typical life cycle for early stage technologies.

[29:41] Finding a drug or cure for a child so they can reach their full potential is described as priceless, with reflections on the anguish parents face.

[30:58] A powerful story about a physician father whose daughter responded to the antibody but ultimately died from graft versus host disease, an experience that drives the mission.

[34:05] When there's a convergence of mission and purpose with many people participating, that flame will continue to burn and inspire other tech transfer offices.

[35:34] The conversation concludes with emphasis on the importance of philanthropy, the power of parents, and finding a mission-driven partner to achieve FDA approval.

Resources:

AUTM

Memorial Sloan Kettering Cancer Center

The Nai-Kong Cheung Lab

Dr. Nai-Kong Cheung

Dr. Yashodhara Dash

Dr. Yashodhara Dash - LinkedIn

Dr. Imke Ehlers-Surur

Dr. Imke Ehlers-Surur - LinkedIn

Danyelza

AUTM Better World Project

Band of Parents

Y-mAbs Therapeutics

SERB Pharmaceuticals