First FDA-Approved Mesenchymal Stromal Cell Therapy Ushers in a New Era of Regenerative Medicine

Transformative Scientific Breakthrough Pioneered by Spencer Trask & Co. Venture Osiris Therapeutics

NEW YORK, Jan. 16, 2025 /PRNewswire/ -- The Food and Drug Administration (FDA) approved the first mesenchymal stromal cell (MSC) therapy, RYONCIL^®, indicated for steroid-refractory acute graft-versus-host disease (SR-aGVHD) in children two months and older. This milestone in regenerative medicine was developed and patented by Osiris Therapeutics (Osris), a venture founded by Kevin Kimberlin and Dr. Arnold Caplan, and first backed by Spencer Trask & Co.

Kimberlin, who previously collaborated with Dr. Jonas Salk to develop the first FDA-approved cell-based immunotherapy, partnered with Dr. Caplan to conduct the first human clinical trials with mesenchymal stromal cells. This work was instrumental in establishing the safety and feasibility of MSC therapies, directly contributing to the development and eventual FDA approval of treatments like RYONCIL. To complete the necessary Phase 3 trials, Osiris licensed the intellectual property in exchange for milestone payments and royalties, and sold the product, originally called Prochymal, to Mesoblast Limited (Mesoblast), creating a powerhouse at the forefront of regenerative medicine.

"When we started with Dr. Caplan, we envisioned a future where this living cell would be the medicine," said Kimberlin, Spencer Trask & Co. Chairman. "This FDA action validates our pioneering work and sets the stage for MSCs to treat or cure some of the most challenging unmet needs."

Originally thought to be a stem cell, the MSC was characterized by Osiris scientists in a seminal paper published in Science that has been cited 29,925 times since 1999. There are over 1,700 filings at ClinicalTrials.gov to test mesenchymal stromal or mesenchymal stem cell treatments for more than 1300 different medical conditions. Today, the scientific consensus is that the MSC acts primarily by orchestrating the body's response to injury and many infections.

"MSCs are to healing what antibiotics are to bacterial infection," Kimberlin stated. "They augment our natural mechanisms to treat and heal once unhealable, untreatable conditions."

RYONCIL is the latest in a series of landmark MSC innovations by Osiris which include Osteocel^®, the first product marketed in the U.S. containing adult MSCs, and Grafix^®, the standard-of-care today for treating chronic diabetic foot ulcers, the leading cause of amputation. RYONCIL will be available to children in the immediate future at transplant centers and hospitals around the United States. Mesoblast is advancing late-stage clinical trials with MSCs for healing cardiovascular conditions and treating lower back pain.

About Spencer Trask & Co.
Spencer Trask & Co. is an advanced technology development firm that discovers big ideas that drive meaningful change. The firm and its principals have co-founded or first-funded world leaders in genomics, the Internet, mobile communications, healthcare, and artificial intelligence. By supporting smart entrepreneurs, engineers, scientists, and obsessive missionaries, the firm helps create one-of-a-kind opportunities to make the world a better place. www.spencertraskco.com.

About Kevin Kimberlin
Kimberlin is a tech and biotech entrepreneur and, since 1991, Chairman of Spencer Trask & Co. He co-founded or first funded several ventures that have changed the healthcare landscape and have saved tens of thousands of lives. In addition to Osiris Therapeutics, he co-founded the first genomics venture, Myriad Genetics, which discovered the breast cancer gene, BRCA1, and Ciena Corporation, the world leader in the optical networking systems that power the Internet and the infrastructure connecting AI platforms to their data and users.

References

1. Lazarus HM, Haynesworth SE, Gerson SL, Rosenthal NS, Caplan AI. Ex vivo expansion and subsequent infusion of human bone marrow-derived stromal progenitor cells (mesenchymal progenitor cells): implications for therapeutic use. Bone Marrow Transplant. 1995 Oct;16(4): 557–64.
2. Maxson S, Lopez EA, Yoo D, Danilkovitch-Miagkova A, LeRoux MA. Concise Review: Role of Mesenchymal Stem Cells in Wound Repair. Stem Cells Transl Med. 2012 Feb;1(2):142–9.
3. Pittenger MF, Mackay AM, Beck SC, Jaiswal RK, Douglas R, Mosca JD, et al. Multilineage Potential of Adult Human Mesenchymal Stem Cells. Science. 1999 Apr 2;284(5411):143–7.
4. Watson JD, Crick FHC. Molecular Structure of Nucleic Acids: A Structure for Deoxyribose Nucleic Acid. Nature. 1953 Apr;171(4356):737–8.
5. Pittenger et al. (1999) has been cited 29926 times. Watson and Crick (1953) has been cited 19694 times. Metrics retrieved from Google Scholar, December 19th, 2024.
6. Pittenger, M.F., Discher, D.E., Péault, B.M. et al. Mesenchymal stem cell perspective: cell biology to clinical progress. npj Regen Med 4, 22 (2019). https://doi.org/10.1038/s41536-019-0083-6
7. Stem cell use part of therapy. The Baltimore Sun. 2005 Sep 23 [cited 2024 Dec 19]; Available from: https://www.baltimoresun.com/2005/09/23/stem-cell-use-part-of-therapy/
8. DaVanzo J, Hartzman A, Surfield C, Dobson A. Cryopreserved Placental Membrane Allograft Reduces the Likelihood of Developing a New or Recurring Foot Ulcer and All-Cause Mortality in Diabetic Patients When Compared with Other Cellular- and Tissue-Based Products. Advances in Wound Care. 2023 Apr 1;12(4):169–76.
9. Fan XL, Zhang Y, Li X, Fu QL. Mechanisms underlying the protective effects of mesenchymal stem cell-based therapy. Cell Mol Life Sci. 2020 Jul;77(14):2771-2794. doi: 10.1007/s00018-020-03454-6. Epub 2020 Jan 21. PMID: 31965214; PMCID: PMC7223321.

SOURCE Spencer Trask & Co.