clinical trials Archives

Early encouraging UC Davis trial data on cell therapy for spina bifida

March 6, 2026 / Professor Paul Knoepfler, Ph.D. / 1 Comment

cell therapy for spina bifida, Diana Farmer

A Lancet paper is sparking excitement about an investigational cell therapy for spina bifida. Here’s the study: Feasibility and safety of cellular therapy for in-utero repair of myelomeningocele (CuRe Trial): a first-in-human, phase 1, single-arm study. Myelomeningocele is the most severe manifestation of spina bifida The work was led by my UC Davis colleagues Diana Farmer and Aijun Wang. The trial is […]

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Could Capricor’s report of strong data on deramiocel for Duchenne spark FDA approval?

December 4, 2025 / Professor Paul Knoepfler, Ph.D. / Leave a Comment

The cell therapy biotech Capricor just released new Phase 3 data from its Duchenne Muscular Dystrophy (DMD) program The firm is trialing its stromal cell product deramiocel for DMD. These new results are very encouraging and stronger than past data. Why am I excited here? These newest deramiocel results released in a PR show statistically

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Hoping for approval, Mesoblast says FDA wants new remestemcel-L trial

August 3, 2023 / Professor Paul Knoepfler, Ph.D. / 1 Comment

Australian biotech Mesoblast has had a rollercoaster ride working toward FDA approval of its mesenchymal cell therapy product remestemcel-L. They are seeking approval for use in patients with graft-versus-host disease (GvHD). More specifically, it is intended for pediatric patients with steroid-refractory acute graft versus host disease, or SR-aGVHD. This is a serious condition and new treatment

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Mayo Clinic charges patients just to talk about clinical trials?

February 15, 2022 / Professor Paul Knoepfler, Ph.D. / 7 Comments

What if some legit place like The Mayo Clinic charged people just for the opportunity to talk to a doctor about possibly enrolling in a clinical trial? Imagine this kind of conversation. “Hey, Doc, I have this medical problem and heard you have a relevant clinical trial. Tell me about it.” Then the doctor fills

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COVID-19 stem cell trials pile up during novel coronavirus outbreak

February 19, 2020 / Professor Paul Knoepfler, Ph.D. / 15 Comments

Could stem cells help patients with the novel coronavirus disease COVID-19? It’s hard to say at this point. Is it worth doing trials during the outbreak to find out? Three new clinical studies have popped up on Clinicaltrials.gov investigating the potential of stem cells to help patients infected with the new coronavirus 2019-nCoV. Update, the

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Hope on Parkinson’s front: Japan IPSC trial 1st patient

November 18, 2018 / Professor Paul Knoepfler, Ph.D. / 2 Comments

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In Parkinson’s Disease patients develop neurological dysfunction as they lose a special kind of brain cell called dopaminergic (or dopamine) neurons. While a number of different approaches to this disease have been studied for decades, nothing has proven particularly successful in slowing its progression. As a result there has been a big need for novel thinking

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Huge clinical trial fees allowed by FDA at times, details often secret

May 22, 2016 / Professor Paul Knoepfler, Ph.D. / 9 Comments

Why can there be huge clinical trial fees for participants? I am often critical of for-profit stem cell clinics on this blog for numerous reasons. For instance, one thing that concerns me greatly about these clinics is that they charge patients to get experimental “treatments” that have not been proven to be safe or effective. But

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Pay-for-play clinical trials? 70% negative polling

April 8, 2016 / Professor Paul Knoepfler, Ph.D. / 2 Comments

We’ve heard of payphones, pay toilets, and other services for which people have to pay for access, but what about pay-for-play clinical trials? In a pay clinical trial, patients have to pay anywhere from $10K to over $100K to get in the door. I asked readers of this blog about how they feel about patients

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Poll: How do you feel about pay-to-play clinical trials?

March 30, 2016 / Professor Paul Knoepfler, Ph.D. / 15 Comments

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There’s a growing trend whereby patients have to pay to be in a clinical trial, which are called pay-to-play clinical trials. Proponents of this idea point toward potential benefits in the form of patients gaining access to innovative therapies. They also argue that this approach provides a much-needed funding mechanism for the trials and that

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Interview with Clinicaltrials.gov Director Deborah Zarin

September 24, 2014 / Professor Paul Knoepfler, Ph.D. / 4 Comments

This post is the first in a series about the Clinicaltrials.gov website. This piece is an interview the Director, Dr. Deborah Zarin. I want to thank her for taking the time to answer my questions. Later, I will post Part 2 in which I discuss my concerns about the trend of for-profit stem cell clinic trials

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