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Regenerative Medicine: The hope and the Hype

December 1, 2015

By Angela Woodall, Healthcare and Environmental Reporter.

 

A paralyzed child learns to walk. A mother diagnosed with cancer is tumor free within a year.  And a man suffering from a degenerative eye condition watches his grandchildren graduate from college, the first time he has seen their face since they were born.

Researchers believe regenerative medicine holds the key to making these scenarios a reality. The technology, they believe, has the potential to develop therapies for previously untreatable diseases and conditions — diabetes,  heart disease, renal failure, osteoporosis and spinal cord injuries.

Indeed, early trials are feeding a 2005 prediction by the U.S. Department of Health and Human Services that regenerative medicine “will revolutionize health care treatment.”

But a decade later, the distance between prediction and reality is still wide.

The field is full of promise and that is exciting, said Rosemarie Hunziker of the National Institutes of Health. As program director of the Tissue Engineering/Regenerative Medicine, Biomaterials, she is involved with multiple aspects of regenerative medicine.

The excitement over the promise is warranted, she said. But, she added, “you have to understand what promising means.”

 

Regenerative Medicine: A Definition

Some of the misunderstanding starts with the expanding scope of what is considered regenerative medicine.

It sounds like a straightforward concept. But like anything involved in human biology, pinning down what regenerative medicine is (as opposed to what it is not) can be tricky.

For starters, regenerative medicine is a “truly interdisciplinary field” that utilizes and brings together nearly every field in science — from cell/development biology, materials science, bioengineering and computational modeling, to name a few — according to the Department of Health and Human Services. That makes it hard to narrow down.

Regenerative medicine investigators Chris Mason and Peter Dunnill offer this description in their 2014 paper, “A brief definition of regenerative medicine:” The goal is to replace or regenerate human cells, tissue or organs, to restore or establish normal function.

In other words, whatever method is needed to replace or regenerate cells, tissue or organs to restore or reestablish normal function, as Mason put it.
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Tissue Engineering

Bone, cartilage, blood vessels, the bladder, organs — these are structural tissues that can be repaired or replaced using cells.

Researchers at Tufts University reported they are developing  artificial brain tissue that can model the way neurons on a football player’s or soldier’s brain would respond to impact.

And research teams with support from the NIH, DARPA, FDA are developing “tissue chips” that resemble human cells in order to test experimental medications for safety or efficacy.

Regenerative medicine also overlaps with includes stem cell-based therapies — one of the oldest being bone marrow transplants.

 

Stem Cell Therapy

Hunziker said much of the early work in understanding stem cells (some going back a hundred years or more) predates any notion of cell-based therapies, and many applications of stem cell science do not involve cell transplantation.  But some of the most exciting work does indeed hold promise for using a patient’s own stem cells to help heal their damaged tissues and organs.

They are the supporting actor whose performance deserves an Oscar award, but they are not the star.

For example, scientists are learning how to create an environment in which the stem cells can develop into replacement tissue. The goal is to use them to replace damaged heart tissue and to rebuild muscle. In 2013, Anthony Atala of the Wake Forest Institute for Regenerative Medicine  used a 3-D printer to create a life-size kidney made of human cells. And cells are being used to regrow muscles, tendons and organs to treat the wounds of soldiers.

One company is in Phase II of a study using a novel form of therapy in which reprogrammed stem cells are injected directly into a the spinal cord of patients who have spinal cord injuries. Other studies further along include stem cell therapies for heart attack victims and the a leading cause of blindness, dry macular degeneration.

But whether the therapy is stem cell-based or tissue engineering, the border of regenerative medicine once stopped at the concept of “tissue for life,” meaning that whether tissue was repaired or replaced, it stayed with a person and became a part of their body.

Now the demarcation is morphing into what the research community want it to include, Hunzicker said. “It’s a moving target.”

 

Gene Therapy

One big change has been the inclusion of gene therapy: treatments involving altering the genes inside the body’s cells to stop disease.

These treatments were seldom included under the umbrella of regenerative medicine until researchers found they could create killing machines able to attack cancerous tumors using a patient’s T-cells.

In the case of cancer, T-cells are separated from a sample of a patient’s blood, infused with a modified gene known to fight tumors, and injected back into the patient’s blood stream. The cells circulate through the body until they came across the tumor. Then they attack.

The therapy is not a classic form of regenerative medicine because the T-cells are going to die off eventually instead of staying in a patient’s body in the same way the skin graft or organ transplant would. But the therapy capitalizes on regenerative medicine skills and techniques so the umbrella got bigger.

In multiple early trials in the United States some trial participants recruited from among terminally ill patients showed no response. But others have emerged from the therapy tumor-free, sparking hopes that the therapy could have long-lasting effects that will effectively cure different types of cancer.

The result has been heated excitement in labs as well as among investors.

Regenerative medicine and advanced therapy companies raised more than $9.3 billion in the first three quarters of 2015, up 163 percent when compared to the same time period last year, according to Alliance for Regenerative Medicine, a Washington, D.C.-based advocacy organization. Also, 571 clinical trials are underway worldwide, the organization reported recently.

But no gene therapies have been approved in the United States and the only way to receive gene therapy is to be enrolled in a clinical trial.

“There are many companies running gene therapy trials, Brookside Capital Director Daniel Krizek said in the ARM report. But, he continued, “the financing window, at least partially, will depend on outcomes of those trials.”

Yet words like disruptive and transformative are thrown around by non-scientists. The optimism has prompted a rush to use stem cell products in the clinical realm, according to Matthew Li, co-author of a recent analysis of clinical trials involving stem cell-based treatments worldwide.

The Federal Drug Administration, which oversees clinical trials, finds itself wedged between the need to move forward cautiously with innovations in regenerative medicine, in order to safeguard patients, and the urgent demands of many of the very patients the agency is trying to protect.

In contrast, the patients and their families are racing against the clock. They are desperate for cures now, not years in the future.

“The promise is amazing but we have to move slowly to keep people safe,” Hunziker said.

 

Angela Woodall | Healthcare and Environmental Reporter

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