OHSU

Stem Cell Discovery May Be Key to New Cures

Shoukhrat Mitalipov, Ph.D.

OHSU Extra, Summer 2013

A world-leading discovery by Shoukhrat Mitalipov, Ph.D., has brought new hope to patients with Parkinson’s disease, spinal cord injuries, multiple sclerosis, diabetes and cardiac disease. “As a Parkinson’s patient, I am absolutely thrilled by the potential of Mitalipov’s research, not only to treat PD but many other disorders as well,” said OHSU Foundation trustee Gregory Chaillé.

Mitalipov, a senior scientist in OHSU’s Oregon National Primate Research Center, worked in collaboration with OHSU reproductive endocrinologist Paula Amato, M.D., to become the first team in the world to convert human skin cells into embryonic stem cells. Stem cells can be converted into different cell types – such as nerve cells, liver cells or heart cells – that might one day be used to replace cells damaged by injury or illness. The potential is so great, and the discovery so revolutionary, the research made headlines around the world, sparking more than 2,000 news reports from Japan to South Africa.

Mitalipov and his team used a technique called somatic cell nuclear transfer (SCNT), which removes chromosomes from a human donor’s unfertilized egg and then replaces them with new DNA obtained from a patient’s skin cells. Using a specially formulated growth medium (one surprising ingredient is caffeine), the researchers prompted the modified egg to divide and eventually become stem cells that have the potential to grow into other kinds of cells. The team spurred the growth of human heart cells, which can be seen rhythmically beating, in unison, inside a petri dish. Mature cells created using this method will exactly match a patient’s own DNA. This may prove a significant advantage in regenerating cells or tissues that the body will not reject.

“The task is now to produce these different cells – whatever cells the patient needs – in the lab,” said Mitalipov. “Then we could transplant these mature cells back into the patient.” The technique holds promise for treating diseases such as Parkinson’s, which is caused by a dysfunction in one type of brain cell, or neuron, that produces a chemical the body needs to function properly. “In many patients, these neurons have died off. They’re gone,” says Mitalipov. “There are no other types of cell that can make this chemical. So now in the lab we can learn how to make these neurons and transplant them into patients.”

"If we don't fund promising projects in their early stages, then these opportunities to improve the human condition will be lost to all of us."

- Karen Hinsdale

Mitalipov’s latest breakthrough is the culmination of years of painstaking research, a combination of basic science discoveries made at the OHSU primate center and privately funded human cell studies. Researchers were able to pursue promising leads thanks to substantial institutional investment from OHSU, support from the Leducq Foundation, and early funding from donors in the OHSU Center for Women’s Health Circle of Giving.

“If we believe that solutions to serious health problems lie in scientific research, then it’s going to take the private sector to step forward and let the scientists pursue that research,” said Karen Hinsdale, who was chair of the Circle of Giving when it awarded research funding to Mitalipov in 2010. “If we don’t fund promising projects in their early stages, then these opportunities to improve the human condition will be lost to all of us.”

The worldwide media reaction touched off by Mitalipov’s recent findings included both kudos from prominent scientists around the globe – and some concerns. The SCNT method is considered a technique for cloning stem cells. Some worry that these therapeutic cloning techniques might lead others to attempt to clone human beings. Mitalipov, whose work is focused solely on the potential to cure human diseases, says that such concerns are understandable but in the case of SCNT, he believes they are unwarranted. “We don’t believe our findings could be used by others to advance the possibility of human reproductive cloning. We’ve been doing it for many years in monkeys, and none of the embryos could develop into live monkeys. We can assume that the same techniques, if applied to humans, would not be able to produce live clones.”

Mitalipov operates at the complicated intersection of scientific promise, political debate and regulatory constraint. On the one hand, the SCNT method is an alternative to the politically fraught use of stem cells derived from fertilized human embryos. On the other hand, because federal funding for stem cell research is restricted, Mitalipov’s work is conducted under tightly regulated conditions that mandate a separate lab space designated for human embryonic stem cell research and funded solely through private support.

“Only private investment will move this research forward,” said Daniel Dorsa, Ph.D., OHSU’s senior vice president for research. “Private funders need to step in where federal agencies cannot. This research will fuel the development of stem cell therapies to combat several diseases and conditions for which there are currently no treatments or cures.”

Through ongoing collaborative efforts, Mitalipov and his colleagues around the world will refine the innovation that has given new hope to so many.

For information on how you can support this research, contact Cathy Kemmerer at 503 412-6353 or kemmerer@ohsu.edu.

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