Tuesday, June 26, 2012

Texas makes the case for why science should take time

A few weeks ago, David Lesher wrote in the Sacramento Bee that science takes time if you want to make sure the new therapies being developed are safe. He was writing in particular about a CIRM-funded approach to treating HIV/AIDS.
None of the research under way will reach patients until long after the 10 years of funding by the ballot measure runs out. With the HIV project, researchers hope to be in human trials by 2014, but it is likely to be at least 10 years before they can show it might work in humans. And in the case of a stem cell cure for AIDS, it would be many years after that before a treatment is widely available.

That's partly due to the caution of dealing with new and powerful techniques, and the reality that each scientific step takes time to assess. Even with rapid scientific advance, it also shows how much is still unknown.
 A great example of that concern is being reported in the Houston Chronicle, which gained access to a Food and Drug Administration review of the Texas stem cell company Celltex Therapeutics Corp. This is the company that treated Texas Gov. Rick Perry last year. The Chronicle reported:
The Sugar Land company involved in Gov. Rick Perry's unlicensed adult stem-cell procedure is rife with basic manufacturing problems, according to the U.S. Food and Drug Administration.

In a report one expert called a blow to the entire adult stem-cell industry, the FDA found that Celltex Therapeutics Corp. cannot guarantee the sterility, uniformity and integrity of stem cells it takes from people and then stores and grows for eventual therapeutic reinjection.

A former FDA official who asked not to be identified, however, said the deficiencies - 79 in all, from incorrectly labeled products to failed sterility tests - are so serious that Celltex risks being shut down if it does not remedy the problems quickly.

Adult stem cells are cells in the body that multiply to replenish dying cells. Long used to treat leukemia and other cancers, they have shown promise for tissue repair in many other diseases in the last decade, although most scientists in the field consider them not ready for mainstream use.
 They also quote from the report itself:
"You have not performed a validation of your banking and thawing process to assure viability" of the stem cells, reads the April 27 report, meaning that the company cannot verify the cells are alive.
 This story makes clear why it’s so important for CIRM to work closely with grantees and with regulators like the FDA. It’s true that therapies take time to develop, in part because we want to be sure each step of the way that the therapy being developed will be safe as well as effective. Once therapies reach patients we want to be absolutely sure those patients will be helped instead of harmed.

Here’s more information about our efforts to work with regulatory agencies, from our 2011 annual report.


1 comment:

  1. Great piece.
    I think while science takes time measured in years if not decades and I believe it requires rigorous oversight, we can work together with regulatory agencies creatively to find legitimate ways to get treatments and cures to patients faster than in the past. However, taking shortcuts or avoiding regulatory agencies is a recipe for trouble.
    I blog about my own involvement in and take on the Texas situation here: http://www.ipscell.com