Category Archives: Stem Cells

FDA approves Phase II of stem cell trial for ALS led by U-M’s Dr. Eva Feldman

ANN ARBOR, Mich. — For nearly two years, University of Michigan neurologist Eva Feldman, M.D., Ph.D. has led the nation’s first clinical trial of stem cell injections in patients with the deadly degenerative disease known as amyotrophic lateral sclerosis, often called ALS or Lou Gehrig’s disease.

Now, a new approval from the U.S. Food and Drug Administration paves the way for U-M to become the second site in the trial, pending approval of the U-M Institutional Review Board. To date, the first phase of the trial has taken place at Emory University, with Feldman serving as principal investigator.

The FDA approval of a Phase II trial was announced today by Neuralstem, the company whose product the trial is testing. The Phase II trial will continue to evaluate the safety of the stem cell injections, delivered directly into patients’ spinal cords in escalating doses of up to 400,000 cells per injection, with a maximum of 40 injections. It will also assess any signs that the injections might be impacting patients’ ALS symptoms or progression.

Feldman serves as an unpaid consultant to the company, and has led the analysis of results from the Phase I trial. In data presented last year, spinal cord injections of up to 100,000 cells were delivered safely and tolerated well — with possible signs that in one subgroup of participants, ALS progression may have been interrupted.

“In Phase II, we’ll be injecting stem cells into the upper part of the spinal cord, and our goal is to continue to assess whether this approach is safe, and to look at whether this approach offers some benefit to our patients. We are very pleased at the potential to bring this trial to the University of Michigan, where the initial research behind this technology was done — as well as having it continue at Emory,” says Feldman, the Russell N. DeJong Professor of Neurology at the U-M Medical School, research director of U-M’s ALS Clinic, and director of U-M’s A. Alfred Taubman Medical Research Institute. The neurosurgeon for the trial is Parag Patil, M.D, Ph.D.

The approach uses injections of stem cells delivered during an operation performed by a neurosurgeon. The first phase of the trial involved 15 patients; specific plans for Phase II are still being made but information will be available at neuralstem.com.

If the U-M site team receives IRB approval to recruit local participants, more information will be available at uofmhealth.org. The study at U-M will be funded by the ALS Association, the National Institutes of Health and Neuralstem.

New method for mass-producing RPE cells paves way to treating age-related blindness

Durham, NC – Scientists have developed a new, simpler way to produce human pluripotent stem cells in quantities large enough that they can be used to develop treatments for age-related macular degeneration — the leading cause of irreversible blindness among the elderly. The results of this new study are published in the current issue of STEM CELLS Translational Medicine.

Age-related macular degeneration (AMD), which affects up to 50 million people worldwide, is associated with the dysfunction and death of retinal pigment epithelial (RPE) cells.

“As a result, there has been significant interest in developing RPE culture systems both to study AMD disease mechanisms and to provide substrate for possible cell-based therapies. Because of their indefinite self-renewal, human pluripotent stem cells (hPSCs) have the potential to provide an unlimited supply of RPE-like cells,” noted Donald Zack, M.D., Ph.D., who with Julien Maruotti, Ph.D., led the team of researchers from the Wilmer Institute, Johns Hopkins University School of Medicine in Baltimore, Md., and the Institute of Vision in Paris in conducting the study.

“However, most of the currently accepted methods in use for deriving RPE cells from hPSC involve time-and-labor-consuming  steps done by hand, and they don’t yield large enough amount of the differentiated cells – which has posed a problem when trying to use them to develop potential new therapies,” Dr. Maruotti added.

The Zack/Maruotti team simplified RPE cell production by modifying a standard protocol for isolating the cells from spontaneously differentiating hPSC monolayers. In the new method, hPSCs were amplified by clonal propagation and the RPE cells enriched by serial passage rather than mechanical picking.

“These modifications eliminate the need for the time- and labor consuming manual steps usually required to culture hPSCs and to purify the RPE population, and thereby provide a readily scalable approach to generate large numbers of high quality RPE cells — up to 36 times more than the best protocols previously reported during the same time interval,” Dr. Zack said.

“This improved process represents a step toward mass production of RPE and could prove useful for applications requiring large number of cells such as cell therapy, drug screening or disease modelling,” said Anthony Atala, M.D., Editor of STEM CELLS Translational Medicine and director of the Wake Forest Institute for Regenerative Medicine.