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Of Starfish, Stem Cells and $70 Million: How California Therapies May Reach Patients Quicker

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Ever wonder why medicines cost so much? One reason is the incredible expense of testing them.

Before any new treatment can be sold on the market, the Federal Drug Administration (FDA) asks two crucial questions: Is it safe, and will it work? This is right and proper.

Unfortunately, answering those questions may take years of testing (clinical trials) and mountains of money. The costs of the testing are paid for privately -- with failure costs accumulating. If a company spends $100 million to develop a drug and fails, that cost is added to the next year's budget. The tests become staggeringly expensive.

According to Alan Trounson et al., "The average cost of delivery of a new biopharmaceutical drug into medical practice has been estimated to be [as much as] 3.9 billion, including capital costs and the costs of failed drugs."

We need the tests, of course. Without them, unscrupulous outfits might give injections of salt water and say it was stem cells, or worse, do actual harm to unsuspecting patients while bilking them of their life savings.

But if the price of testing continues to skyrocket, valuable new treatments may not even be tried. Chronically ill people will continue to suffer, with therapies just out of reach.

Example: The California stem cell program has developed 77 new stem cell methods, each one a potential treatment for as many as 38 diseases and crippling injuries.

Among those efforts might be a way to ease diabetes, reverse Alzheimer's, or loosen the clutch of paralysis, but how can we afford the increasingly expensive safety tests?

There may be a way to streamline the costs of clinical trials without compromising safety.

The Alpha Stem Cell Clinics Network is a concept under development by the California Institute for Regenerative Medicine (CIRM), created when voters approved Proposition 71 in 2004.

Alpha Clinics would be hospitals and laboratories carefully prepared to design, administer, or advise on safety tests for new stem cell research methods. When a new therapy is due to be tested, a center of excellence would be ready and waiting, with expert scientist/medico/clinicians available. Instead of endlessly reinventing the wheel, designing and redesigning tests, teams of experts could make clear to both research scientists and patients exactly what must be done to achieve success. Also, patients would have a place to go for reliable information.

Run by CIRM, there might be half a dozen Alpha Clinics up and down the state. Their location would be decided by competition, with potential sites bringing their best to the table: special equipment, good housing, special access to clinicians or patients, demonstrated experience and expertise.

Each site would focus on a different condition or group of ailments: perhaps heart disease, neurological disorders, diabetes, blindness, immune deficiency, and cancer.

Like the arms of a starfish, the sites would all connect to a central management center, which would collect and organize data.

Crucially, this data would be shared. In the past, information about clinical trials was often hidden, or not communicated effectively; with more open information (though patient privacy would be respected), we may avoid repeating mistakes of the past.

As it does with any major initiative, CIRM called a meeting to gather ideas on Nov. 14 and 15, 2012. Invited were scientists, patient advocates, doctors, clinicians, cell manufacturers, and biomedicine and pharmaceutical companies.

Parts of the meeting involved patented information and was therefore private, but a lengthy overview was open to the public.

It was amazing. I did not understand everything I heard, of course; my brain was swimming with new information.

But I felt that click and shock of recognition, the sense that here was something vital.

So many times I had heard scientists talk about the "Valley of Death," when potential new therapies got lost in the costs of testing. Here might be an answer to that problem.

Changes were so wide-ranging it seemed that medicine itself, not just stem cell research, could be forever changed, and for the better.

The price of setting up California's system was estimated at $70 million -- certainly a lot of money. But compared to the costs of endlessly maintaining our loved ones in their misery (roughly $2 trillion a year is spent on chronic disease, more than the annual national deficit), $70 million is a bargain. And, once in place, it is hoped the system will be self-sustaining, paid for by charging corporations a fee to use the facilities.

Here is a personal comment from Dr. Alan Trounson, President of CIRM, who understands the situation better than I ever will:

The Alpha Stem Cell Clinics Network Proposal ... [would be] a network of cell therapy clinics ... to provide for the rising number of clinical trials and recognized treatments....



The network can provide data and a learning base that will assist other studies to progress, provide the opportunity for sound clinical development in major medical centers, and provide support for businesses working in regenerative medicine....



We need to ensure the centers have the critical clinicians, facilities, equipment and
support staff to ensure the work we are supporting gets to the patients. We also want to support independent counselors to assist patients to find the best treatment options ... rather than seek unscientific and unregulated procedures that have no benefit and are very risky.



We will work together with the major Californian medical centers to create the network and a hub [to] help with quality control standards, data management, business development ... assistance with reimbursement, etc.

Should the Alpha Clinic Stem Cell Sites go forward?

On July 25, the governing board of the stem cell agency will meet to consider the proposal. (Location and details will be posted at www.cirm.ca.gov under "meetings.") You are invited. If you care to share an opinion, you will have three free minutes at the microphone.

For anyone concerned about the rising costs of medicine and the suffering caused by chronic disease, July 25 may well be the most important meeting of the year.