Scientists convert adult skin cells into cardiomyocytes using direct reprogramming strategy

Scripps Research Institute scientists have converted adult skin cells directly into beating heart cells efficiently without having to first go through the laborious process of generating embryonic-like stem cells. The powerful general technology platform could lead to new treatments for a range of diseases and injuries involving cell loss or damage, such as heart disease, Parkinson’s, and Alzheimer’s disease.

"We are truly grateful for Douglas’s support in this effort, and impressed by his knowledge of Alzheimer’s disease therapeutic development," said Bredesen. "The funding gap between basic discovery and drug development is commonly called ‘the valley of death,’ because that’s where most discoveries end up," he said. "This valley, along with a lack of understanding of the basic processes at work in the disease, represent the main reasons that no one has yet developed a significant treatment for the disease. Douglas is taking us through this valley, allowing a completely new approach to enter clinical trials, which, if his goal for support is reached, should begin in the next two to three years."

Rosenberg established Sponsored Research Holdings, LLC (SRH) as the vehicle for funding the new therapeutics, an arrangement that could yield profits for his foundation and other venture philanthropists or investors joining his effort. Ideally, SRH will raise and invest up to $10 million to perform research and human proof of concept studies. SRH and Buck will participate together in any royalties and milestones that occur as a result of any licensing and/or commercialization created during the effort.

"Douglas’s involvement is unique in the realm of biomedical research," said Brian Kennedy, PhD, the Buck Institute’s Chief Executive Officer. "I know of no other individual who has committed to fund lead and pre-clinical drug development with the expressed intent of moving it specifically to human testing. His commitment and his business approach to this urgent need provide a new model for the research industry."

"I believe in what Dale is doing and will be extremely active in meeting the $10 million goal," said Rosenberg. "I’ve established different investment vehicles for this partnership to facilitate involvement from any organization or individual who’s interested in supporting this work. A foundation can either make a grant or make a program related investment (PRI); an individual can make a donation and take the tax deduction or they can invest as if it were a straightforward venture deal. Either way we should have enough different buckets to attract the requisite funding."

Rosenberg says his father, who set high standards for how to give back to the world, has been a role model for this effort. "I recognize the odds are stacked against us, but as my dad always said, it’s important to try. If I can inspire others to commit their dollars and time too, then I will have done a job to be proud of," said Rosenberg. "Best case scenario is we get a successful treatment for Alzheimer’s in my lifetime. At the very least we can lead the research effort in a desperately needed new direction."

Source: Buck Institute for Age Research

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