StemCell Therapy

Scientific advances with stem cells and gene editing have raised the prospect that many diseases, traumatic injuries, and chronic conditions could one day be treatedeven curedwith products derived from human biological materials. Most cell- and gene-based regenerative therapies remain in clinical trials or earlier stages of development; a handful have gained Food and Drug Administration approval as new drugs. However, hundreds of businesses in the United States have seized on public interest in this promising field to sell unproven and potentially dangerous stem cell treatments directly to consumers. Patients receiving such products have experienced bacterial infections, permanent vision loss, strokes, and other harms.

Knowingly or not, many clinics offering cell-based therapies may be violating federal law and regulations governing the approval of new drugs, medical devices, and biologic products. FDA has taken important steps to prompt these businesses to comply with appropriate requirements, and the agency should continue enforcement activities to reduce and ultimately eliminate the marketing of unapproved stem cell products.

As of May 2017, at least 716 clinics, spread across 45 states and the District of Columbia, offered unapproved stem cell products.1 (See Figure 1.) The number of new stem cell businesses with websites doubled on average every year between 2009 and 2014, suggesting that the total number operating today could be significantly larger.2

Stem cell businesses promote their products as treatments for a wide range of conditions, from orthopedic pain and arthritis to multiple sclerosis and Parkinsons disease. Little to no evidence exists to support most claims, but several of these treatments have adversely affected patients. For example:

Such cases underscore the public health risks when businesses fail to demonstrate a products safety and effectiveness to FDA prior to marketing and do not adhere to the agencys rules for good manufacturing practices.

Broader enforcement activitiesbacked by sufficient resources from Congressare needed to protect Americans from the risks that unapproved stem cell products pose. The agency should maintain pressure on businesses offering these products to ensure that this promising medical field develops into one that clinicians and patients can trust and safely access.

FDA established a regulatory framework for regenerative therapies in 2005 and issued four guidance documents in November 2017 to clarify the characteristics and approval requirements associated with low-, middle-, and high-risk products. High-risk therapies include those in which stem cells undergo more than minimal manipulation or are intended to perform a different function in the recipient than they do in the donor; this is referred to as nonhomologous use.7 Such products must receive FDA approval as new drugs, devices, or biologics before they enter the market.

These materials laid important groundwork for broader enforcement efforts. Agency leaders have said they will target enforcement resources at products that pose the greatest risks to patients until November 2020. FDA should build on this foundation for effective oversight by using its full array of enforcement tools, including:

Read more:
FDA Enforcement Will Foster Development of Safe, Effective Regenerative Therapies - The Pew Charitable Trusts

This entry was posted on November 2, 2019 at 12:41 am and is filed under Fat Stem Cells. You can follow any responses to this entry through the RSS 2.0 feed. Both comments and pings are currently closed. |