Stem Cell Therapies The Game-changing Path to FDA Approval

Stem cell therapies, and the field of regenerative medicine to which they belong, represent some of the most exciting and promising scientific advancements and medical breakthroughs of the last 20 years.

Recently, interest has escalated amongst researchers, physicians, and the general public in the potential of adult stem cells to treat a wide variety of conditions that could allow those with macular degeneration to see again, those with spinal cord injuries to regain greater mobility, those with arthritis to live with dramatically less pain, those with severe burns to heal without scarring, those with diabetes to become independent of insulin, and those with many other medical issues to receive life-extending and life-enhancing treatment.

Stem cells are unique among cells in that they replicate for extended periods through cell division and, within specifically controlled conditions, can be incubated into mature cells with specialized functions for treating different diseases and conditions.

There are four types of stem cells: totipotent stem cells that develop into cells comprising the cells in an embryo and fetus; multipotent stem cells that, as their name suggests, can give rise to multiple types of cells within particular tissues, organs, or bodily systems; pluripotent stem cells, or “blank slate” stem cells that carry with them the potential to become any type of cell; unipotent stem cells can both regenerate and give rise to a single mature cell type.

Because stem cell therapies represent the frontier of regenerative as well as personalized medicine and have acquired a reputation among the general public as capable of curing otherwise untreatable diseases by regenerating tissue of all types, private investment and private interests have been quick to monetize market demand by offering untested and unapproved stem cell interventions.

Paul Knoepfler, professor of cell biology and human anatomy at the University of California, Davis School of Medicine, reported on this phenomenon in the journal Cell Stem Cell in which he documented 570 clinics across the U.S. where significant gaps existed between the stem cell interventions these clinics offered and regulatory oversight and approval.

A long-standing tension has existed between the innovation efforts of private biotech companies and research institutions and the need for governmental regulatory standards to protect consumers. The challenge has been to strike a balance between innovation and safety, and recent FDA regulation seems to have taken a positive step toward achieving this goal.

The newest FDA guidelines state that “stem cell-derived products that are minimally altered and are used for the same purpose in both donor and host do not need premarket approval and are subject only to regulations that prevent the transmission of communicable diseases.”

Essentially, the FDA is giving a nod to stem cell therapies that pose extremely low thresholds of risk while encouraging biotech firms (both big and small) and research institutions who are attempting to innovate more complex (and potentially risky) advances in stem cell therapies to engage with them early in their research efforts. Thus, allowing the FDA to offer support with a trial so long as it falls within regulatory guidelines.

The release of this framework is a gamechanger for stem cell research and stem cell therapies because it clears the way for legitimate research and clinical trials to freely move forward that can lead to FDA approval and consumer access to these promising treatments.

The FDA is currently conducting its own regenerative medicine clinical trials with adult (non-embryo derived) mesenchymal stem cells (MSC’s). The MSC Consortium is researching eight distinct cell lines, each obtained through commercial sources from eight adult donors. (Males and females age 22 to 47 donated bone marrow stem cells.)

The Consortium is studying multipotent cells: “They can differentiate (mature into) at least three cell types: bone, fat and cartilage, primarily,” explains Steven R. Bauer, Ph.D., chief of the Cellular and Tissue Therapy Branch in FDA’s Office of Cellular Tissue and Gene Therapies. “They can also differentiate into nerve cells, liver cells and a kind of cell called ‘stroma’ that is in the bone marrow and supports blood forming cells,” he continues. “Then, for investigational clinical uses, they’ve been used for repairing hearts, repairing bone and repairing cartilage.”

Not all obstacles to patients receiving stem cell therapies or bringing them to market are regulatory. Some are structural.

Though certain approved and available stem cell therapies currently exist, both doctors and patients have been challenged by the multi-month time frame required to create enough cells to administer multiple treatments.

Further, even world-class research institutions (such as the Mayo Clinic) that have been prepared to conduct large-scale regenerative medicine clinical trials have been thwarted in their efforts by the difficulty of sourcing adequate amounts of stem cells.

This, however, is about the change. In response to their own needs, scientists at the Mayo Clinic’s Center for Regenerative Medicine have developed an FDA-approved automated bioreactor. The bioreactor can manufacture billions of stem cells within a short period, producing in a matter of days what used to take months, thus solving a key piece of the structural puzzle.

“With this new technology, we now can develop phase II trials enrolling larger numbers of patients to fully test the efficacy of cell-based therapies,” said Abba Zubair, Mayo Clinic’s medical director for transfusion medicine and the Human Cell Therapy Laboratory.

After decades of fits and starts in private sector and institutional research attempting to bring safe, effective stem cell therapies to market, the path for the advancement and availability of regenerative and personalized medicine is finally becoming clear.

As regulatory gaps are closed and structural obstacles overcome, the stem cell therapies of the future are closer to being the realities of today.

At Lifecycle Biotechnologies, in our role of providing leading-edge tools and services to these cellular therapy companies and the life science industry as a whole, we’re committed to keeping abreast of the latest scientific discoveries and breakthroughs. In doing so, we aim to better understand and respond to the needs of the rapidly evolving industry and support our diverse client population across multiple disciplinary domains and market divisions.

If you’d like to see how our innovative tools and services can help your company scale for the research methodologies of the future, contact us today and see what we can do for you.