As a regulatory lawyer long-steeped in the world of stem cells and regenerative medicine, Im bound to latch onto the title of a recent Nature piece with particular interest. Cells or drugs? Its a great question from a regulatory point of view.
You cant place any medicine on the market, whether its a small molecule, a gene, or the fanciest of engineered cells, without a marketing authorisation. However, getting an MA can be pretty demanding, and especially when it comes to an advanced therapy product (ATMP) such as a population of cells. Avoiding cells has some obvious attractions.
The article reminds us that cell therapy is less about cells than their fate. Of course, manufacturers of such cells are obsessively concerned about their fate from a product point of view.
However, the bigger question is about what happens to the cells once theyre implanted. At least as important, what happens to the cells that were already at the implant site? The more we learn about how damaged tissues behave and what implanted cells do in their new home, the more it comes down to an understanding of the conversation between cells that determines their phenotype. If we assume that the conversation is mediated by molecules, then we only need cells because we dont yet know what those molecules are or because cells happen to be the best delivery device for them. If the cellular conversation is about growing new cells to replace those lost to ischaemia, for example, the proliferation of any cardiomyocytes surviving implantation may be of less importance than getting the wound site to grow new cardiomyocytes.
An alternative approach would be to mimic that molecular environment to administer well-characterised molecules at known doses. They wouldnt be ATMPs at all, so the extra ATMP burdens that fall on such products under regulations such as the ATMP Regulation would not apply; just those for a conventional biological medicinal products.
Regulatory reflection
Anyone familiar with the manufacturing requirements for somatic cell or tissue engineered products will know that the logistical and quality burdens on those seeking an MA for an ATMP far surpass even those for non-biological medicinal products. It is perfectly true that, under the EUs 2007 Advanced Therapy Medicinal Products (ATMP) Regulation there are only three varieties of ATMP: medicinal products comprising somatic cells or genes, and cells which have been substantially manipulated or used to fulfil a different function in the recipient than in the donor.
But why shouldnt an advanced therapy be delivered the old-fashioned way, as a drug?
The European Commission is reviewing EU blood, cell and tissue (BCT) quality and safety regulation (more on this soon), but has excluded the ATMP Regulation from its review. The Nature article does not touch upon regulation, but to me it suggests another reason for revisiting the ATMP Regulation in the light of changes in scientific understanding. The problem is that, although the ATMP Regulation shoe-horned cell and gene medicinal products into the well-worn boot of European medicines regulation, the foot doesnt entirely fit.
If you recall the heady days of the early 2000s in which the Regulation was developed, the future was one in which bags of embryonically-derived cells for fixing hearts and spines were distributed around the world like blister packs of ibuprofen. The future unfolded rather differently. Within months of the Regulation coming into effect, an invention for inducing pluripotent stem cell induction was published, reducing the necessity for embryonic cells (with the development of stem-cell based embryo models making even embryos unnecessary). More fundamentally, the future turned out to be dominated, not by packages of cells derived from other peoples or embryos, but by the use of the patients own cells; autologous cells.
However, the word autologous doesnt appear in the ATMP Regulation at all. One perfectly logical reason for their non-appearance is that its questionable as to whether a product intended solely for one person can be truly said to be placed on a market at all. If so, its at least odd to talk about authorising such a product to be placed on one. Sure, we legal our way around this so that autologous products do come bearing MAs, but this regulatory chafing of the cellular heel implies that the paradigm is wonky. The EUs BCT regulations already address quality and safety framework for cells, so perhaps the autologous cell therapy market is better regulated as a service than on the basis of a marketable product?
You get another idea about how awkwardly cell products fit into the regime for marketing medicines when you ask questions such as, what is the mechanism of action? and what is the dose? Again, we can get around irritating questions of this sort, but we should certainly pause for thought, and the Nature piece rather sharpens our appreciation.
Javaria Tehzeeb, author of a 2019 literature review on stem cell heart treatments thats cited in the Nature piece, wonders how things are likely to pan out. Perhaps stem-cell therapies will be approved first, but then be overtaken by drugs once the science has caught up? When we get to the end of the line with molecules, then maybe we can say stem cells are a thing of the past, says Tehzeeb. The manufacturing logistics and regulatory path to market would certainly be more straightforward, and there would be real control over the nature and amount of substances applied, in contrast to the informed-wing-and-prayer approach of cell injection. But were not in that future yet. As Tehzeeb says, until then, we should continue to pursue their potential.
As in life science, so in life science regulation its a work in progress
See the rest here:
Cell therapies without cells? - Lexology
- SUM Hospital conducts Odishas first stem cell transplantation - The Times of India - April 24th, 2024
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- Man Paralyzed In Surfing Accident Regains Ability to Walk After Stem Cell Treatment - The Inertia - April 20th, 2024
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- UM, CUHK jointly hold symposium on stem cells and regenerative medicine - gcs.gov.mo - April 20th, 2024
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- The ISSCR announces 2024 election results - EurekAlert - March 28th, 2024
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- Opinion: The revolutionary sickle cell therapies - MSN - March 22nd, 2024
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- BioCardia and StemCardia Announce Biotherapeutic Delivery Partnership - Diagnostic and Interventional Cardiology - March 17th, 2024
- Cynata Therapeutics' breakthrough in regenerative medicine - Finance News Network - March 17th, 2024
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- This Swedish startup wants to reduce the cost, and controversy, around stem cell production - TechCrunch - March 6th, 2024
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- Stem Cell Therapy Market to Boost USD 137.5 Billion by 2034 and Projected to grow at 32.10% CAGR with increasing ... - Yahoo Finance - March 1st, 2024
- Studies expand use of stem cell therapies - WPLG Local 10 - March 1st, 2024
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- Pelage Pharmaceuticals Announces $16.75M Series A Financing led by GV to Revolutionize Regenerative Medicine ... - Yahoo Finance - February 27th, 2024
- Say Her Name - Henrietta Lacks, The Real Mother Of Stem - Her Campus - February 27th, 2024
- California Man Free of HIV And Cancer in Astonishing Medical Recovery - ScienceAlert - February 25th, 2024
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- Stem Cells in Regenerative Medicine - PMC - National Center for ... - February 24th, 2024
- The Promise and Reality of Stem Cell Therapy: What You Need to Know - Corporate Wellness Magazine - February 22nd, 2024
- Stem Cell Therapy: A Beacon of Hope for Patients with Untreatable Conditions - Corporate Wellness Magazine - February 22nd, 2024
- Leading Experts in Stem Cell Therapy: A Global Overview - Corporate Wellness Magazine - February 22nd, 2024
- Can Stem Cells Help Reverse the Effects of Aging? - Corporate Wellness Magazine - February 22nd, 2024
- Stem Cell Therapy for Cancer: Hope on the Horizon? - Corporate Wellness Magazine - February 22nd, 2024
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- Turning Back the Clock: The Science of Stem Cells in Anti-Aging - Corporate Wellness Magazine - February 22nd, 2024
- Insights into cellular therapies for cancer treatment - Drug Target Review - February 22nd, 2024
- The Role of Stem Cells in Rheumatoid Arthritis Recovery - Corporate Wellness Magazine - February 22nd, 2024
- Dr Ernesto Romeros vision transforms immunotherapy regenerative medicine into a global leader in stem cell treatment - Khaleej Times - February 22nd, 2024
- World-Renowned Doctors Leading the Way in Stem Cell Research and Treatment - Medical Tourism Magazine - February 20th, 2024
- Choosing the Best: A Guide to America's Premier Stem Cell Therapy Providers - Medical Tourism Magazine - February 20th, 2024
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- Global Leaders in Stem Cell Therapy: Where Excellence Meets Innovation - Medical Tourism Magazine - February 20th, 2024
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- Top Stem Cell Clinics in the USA: Pioneering Treatments for Chronic Conditions - Medical Tourism Magazine - February 20th, 2024
- Stem Cells and Autism: Understanding the Potential for Innovative Treatments - Medical Tourism Magazine - February 20th, 2024
- Stopping the awakening of sleeping Acute Myeloid Leukaemia stem cells to prevent disease relapse - University of Birmingham - February 20th, 2024
- City of Hope Achieves Ground Breaking Treatment for Blood Cancer and HIV - Medriva - February 18th, 2024
- Regenerative Medicine Market Size to Worth USD 95 Billion by 2032 - InvestorsObserver - February 18th, 2024
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- Chinese team tests lung treatment that may be first to reverse COPD damage - South China Morning Post - February 15th, 2024
- Why leukemic stem cells not harmed by chemotherapy begin to grow and produce AML cells after treatment - Medical Xpress - February 15th, 2024
- Regenerative Medicine Market is Expected to Reach $49.0 Billion | MarketsandMarkets - Yahoo Finance - February 15th, 2024
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