Stories about: blood stem cells

Seeding medical innovation: The Technology Development Fund

Monique Yoakim Turk Technology Development FundMonique Yoakim-Turk, PhD, is a partner of the Technology Development Fund and associate director of the Technology and Innovation Development Office at Boston Children’s Hospital

Since 2009, Boston Children’s Hospital has committed $6.2 million to support 58 hospital innovations ranging from therapeutics, diagnostics, medical devices and vaccines to regenerative medicine and healthcare IT projects. What a difference six years makes.

The Technology Development Fund (TDF) was proposed to Boston Children’s senior leadership in 2008 after months of research. As a catalyst fund, the TDF is designed to transform seed-stage academic technologies at the hospital into independently validated, later-stage, high-impact opportunities sought by licensees and investors. In addition to funds, investigators get access to mentors, product development experts and technical support through a network of contract research organizations and development partners. TDF also provides assistance with strategic planning, intellectual property protection, regulatory requirements and business models.

Seeking some “metrics of success” beyond licensing numbers and royalties (which can come a decade or so after a license), I asked recipients of past TDF awards to report back any successes that owed at least in part to data generated with TDF funds. While we expected some of the results, we would have never anticipated such a large impact.

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Can blood cells be rebooted into blood stem cells?

Hematopoietic hierarchy blood development stem cells
The classic hematopoietic hierarchy. What if we could turn those arrows around?

Think, for a moment, of a cell as a computer, with its genome as its software, working to give cells particular functions. One set of genetic programs turns a cell into a heart cell, another set creates a neuron, still another a lymphocyte and so on.

The job of controlling which programs get booted up, and when, falls in part to transcription factors—genes that act like molecular switches to turn other genes on and off.

Derrick Rossi, PhD, spends a lot of his time thinking about transcription factors. A stem cell and blood development researcher in Boston Children’s Hospital’s Program in Cellular and Molecular Medicine, Rossi believes that transcription factors hold the power to achieve one of the most sought-after goals in regenerative medicine: producing, from other cell types, transplantable hematopoietic stem cells (HSCs).

“There are about 50,000 HSC transplants every year,” Rossi explains, noting that the success of a transplant is highly dependent on the number of cells a patient receives from her donor. “But HSCs only comprise about one in every 20,000 cells in the bone marrow.

“If we could generate autologous HSCs from a patient’s other cells,” he continues, “it could be transformative for transplant medicine and for our ability to model diseases of blood development.”

As they reported April 24 in Cell, Rossi and his collaborators have taken a significant step toward that goal: Using a cocktail of eight transcription factors, they reprogrammed mature mouse blood cells into what they have dubbed induced HSCs (iHSCs).

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