Five years ago this month, Michigan voters opened the door for a full range of stem cell research in the state. Today, that effort is well underway at U-M, and yielding results that are expanding knowledge of a wide range of diseases.
The Michigan ballot initiative approved in 2008 amended the state constitution and allowed for the first time the production of new human embryonic stem cell (hESC) lines in Michigan.
Since that approval, U-M founded what is now known as the MStem Cell Laboratories, based in the Medical School, to derive hESCs using donated embryos that would otherwise have been discarded by couples undergoing fertility treatment.
U-M also established a framework and oversight panel to guide this work under the appropriate state and federal statutes and regulations.
In less than three years, the research has flourished, and researchers from U-M and other institutions are able to use hESCs derived at U-M in their work.
At the same time, a broad range of other stem cell research continues at U-M, including research on “adult” and induced pluripotent stem cells, cancer stem cells, and treatments and clinical trials based on delivering stem cells into the body. This includes a Phase II clinical trial investigating the use of stem cells in patients with Lou Gehrig’s disease.
“We’ve been working hard to do what the people of Michigan asked us to do, and we’re starting to see benefits in terms of scientific discoveries being made,” says Gary Smith, who directs the MStem Cell lab and is a professor of obstetrics and gynecology, molecular and integrative physiology, and urology.
He notes that hESC work and other types of stem cell work complement one another.
Key facts about stem cell research at U-M since the 2008 vote
• Each line of hESCs derived at U-M has been grown in a slow, meticulous process that takes months.
• Once there are enough cells in a line, the team freezes them and submits extensive information about them to the National Institutes of Health for consideration for including the line in the NIH hESC registry.
• The U-M team derived its first hESC line in October 2010 — and in April 2011 announced its first line containing a genetic defect that causes disease.
• In early 2012, the first line derived at U-M was listed on the national registry, making the cells available for use by researchers who request them, sign an agreement to guide their use, and pay $800-$2,200 to cover costs.
• 12 lines derived at U-M have been approved and listed on the registry. Five more are waiting for approval. Requests for the cells come in regularly.
• The 12 approved lines include ones containing genetic mutations for:
- Charcot-Marie-Tooth disease, a crippling nerve disorder.
- Hypertrophic cardiomyopathy, which thickens the heart muscle and is a silent killer of young athletes and others.
- Hemophilia B, a deadly blood clotting disorder.
- Huntington’s disease, a lethal nerve disease that affects both the brain and muscle control.
- Aniridia, a disorder that blinds from birth.
- Hydroxysteroid dehydrogenase deficiency, which profoundly affects hormone production by causing a condition called congenital adrenal hyperplasia.
- Multiple endocrine neoplasia 2a, a disorder where tumors form in the body’s network of hormone-producing glands.
• The MStem Cell lab moved to a new, expanded location early in 2013, giving the team more room to work.
• Funding for all equipment, salaries, supplies, utilities and other support for MStem Cell hESC derivation must come from non-federal sources under U.S. law.
• The MStem Cell lab’s funding originally came from a group of donors, who supported the founding of what was then called U-M’s Consortium for Stem Cell Therapies in 2009. Recently, it received support from the U-M President’s Office, the Medical School Dean’s Office, the A. Alfred Taubman Medical Research Institute, and the Department of Obstetrics and Gynecology.
• The lab now accepts monetary donations online and also receives some foundation support.
• The lab continues to accept embryo donations under strict guidelines. Due to lab capacity, only embryos that have been tested through preimplantation genetic diagnosis and found to have single-gene disease mutations, translocations and aneuploidy (chromosome number issues) are being accepted at this time.