Stem cell: Hunting a cure for diabetes

An eminent Harvard stem cell researcher who is searching for a cure for an affliction that plagues 29 million Americans stood on a San Francisco stage this week and spoke of “things we don’t understand.”

The scientist is Doug Melton, who is on a deeply personal quest for a cure for diabetes. Both of his children have the disease. And the state of California is helping out on his search with $5 million.

The $5 million from California is a grant made last March by the state’s $3 billion stem cell agency, formally known as the California Institute for Regenerative Medicine (CIRM).

The occasion for Melton’s remarks was the presentation of the Ogawa-Yamanaka Stem Cell Prize, a$150,000 award for his work in cellular reprogramming.

Here is how Hannah Robbins of the Harvard Gazette described the results of Melton’s research:

“As a pioneer in programming insulin-producing beta cells from stem cells, Melton’s lab can now generate therapeutic quantities of functional, stem cell-derived beta cells, which Melton hopes will someday soon replace the life-saving yet painful daily insulin injections for diabetics.”

The $5 million from California is a grant made last March by the state’s $3 billion stem cell agency, formally known as the California Institute for Regenerative Medicine (CIRM).

Doug Melton. (Photo courtesy of BD Colen.)

The cash is going to a firm in Cambridge, Ma., called Semma Therapeutics, Inc., which Melton co-founded and which is named after his two children, Sam and Emma. Melton now serves on the firm’s board of directors. The business is only two years old, but has raised roughly $50 million to translate Melton’s work into actual treatments.

His grant application to CIRM in March was titled simply “Personalized Cell Therapy for Diabetes.”The proposal (application number TRAN1-08561) received a score of 90 out of 100 from the agency’s scientific reviewers who are from out-of-state. A summary of the closed-door review said the proposal was “strong, well-­designed, feasible. and high impact.” The research “has excellent product development plans and great regulatory support,” reviewers said.

The stem cell agency is limited to funding only work that is done in California. Semma announced earlier this month that it has set up arrangements with UCLA, Cedars-Sinai and the City of Hope in the Los Angeles area to generate “suitable clinical grade” cells from patients and “to establish a path leading to the transplantation of these cells back into patients in a clinical trial.”

The financial backers are led by MPM, a venture capital firm with offices in South San Francisco and Cambridge, Mass. Robert Millman, formerly of MPM, is CEO of Semma.

Peter Butler, chief of Endocrinology, Diabetes and Hypertension at UCLA, will be dealing with patient selection. Dhruv Sareen of the Induced Pluripotent Stem Cell Core Facility at Cedars-Sinai will direct derivation and analysis of pluripotent stem cells from each patient’s blood. The cells will be transferred to the City of Hope for manufacture of products for clinical trials.

Overseeing the entire project is Felicia Pagliuca, principal investigator on the CIRM grant and chief scientific officer of Semma. She also was also a lead on the original research in Melton’s lab.

The financial backers are led by MPM, a venture capital firm with offices in South San Francisco and Cambridge, Mass. Robert Millman, formerly of MPM, is CEO of Semma. Other investors include Medtronic,Novartis, Fidelity Biosciences, Arch Venture Partners and theJuvenile Diabetes Foundation.

Semma is still hiring and has openings listed for five positions on its web site, ranging from scientists to a director of device development and manufacturing.

Here is a video recording of the award ceremony in which Melton discusses his research and “the things we don’t understand” in the science.
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Ed’s Note: David Jensen is a retired newsman who has followed the affairs of the $3 billion California stem cell agency since 2005 via his blog, the California Stem Cell Report, where this story first appeared.  He has published more than 4,000 items on California stem cell matters in the past 11 years.