Patients with less severe forms of the disease - an autoimmune disorder that causes a variety of skin lesions - may need no more than topical creams. But about a third of cases are more severe, resulting in treatments with “systemic” medicines, such as pills, that send medicines throughout the body. It’s in the systemics category that prices are really going up, according to researchers Vivianne Beyer, M.D., and Stephen E. Wolverton, M.D. They reported their findings in this month’s Archives of Dermatology.

There’s a broad range in those systemic prices as well. Dr. Beyer, an IUSM grad doing her first year of residency at St. Vincent Hospital, and Dr. Wolverton, Theodore Arlook Professor of Dermatology, put together a cost model and found that annual treatment costs ranged from $1,197 for methotrexate, a traditional systemic therapy, to $27,577 for two 12-week courses of alefacept.  From 2000 to 2008, they found, the wholesale prices of brand-name psoriasis therapies rose an average of 66 percent, compared with  30.1 percent for all prescription drugs.

The bottom line: “In addition to considerations of safety, efficacy and patient convenience and preference, health care professionals should be aware of current costs and anticipate future costs when making therapeutic decisions.”

Q: In your comments to the New York Times, you called the recent developments “exciting.” Do these recent trials reflect a resurgence of interest by researchers in gene therapy?

A: Gene therapy has always been there, and if you look at the estimated accruals (of patients into gene therapy trials) over the past decade – at least reviewing data from the Office of Biotechnology Activities, which oversees gene transfer research at the National Institutes of Health – patient accrual has not changed significantly – or has increased. Some re-tooling has been done as we learned what approaches are likely to work or not work.

Q: What is it about gene therapy that has made successes so difficult to achieve?

A: Considering how long scientists have been working on gene therapy, I think we are doing extremely well. We are seeing success in less time than it took to successfully perform bone marrow transplants. Gene therapy is moving at the expected pace for any new therapeutic.

Clearly we have the proof of principle to treat disease, and as was reported in the New England Journal of Medicine earlier this year (in a trial to treat children withsevere combined immunodeficiency) we have patients with long term benefits – with one patient now out over eight years.

Q: How does the IU School of Medicine benefit from the presence of the IU Vector Production Facility and the National Gene Vector Biorepository?

A: The recent Science article shows the arrest of disease progression in ALD utilized lentiviral vectors which are the type of vectors produced at the IUSM. We are one of the few centers in the world capable of making these vectors for clinical use. While we were not involved in that trial, we are involved with a number of trials that are just starting and we will continue to be involved as these trials move into the clinic.

So what do we need from you? Just this: Suggestions for a new name, something different than “IUSM Research News,” and something better than “IUSM Research Blog.”

So leave a comment or send me an email.

Thanks.

Simon Conway, PhD, professor of pediatrics, co-authored a paper with Christoph Lepper and Chen-Ming Fan (PDF) of the Carnegie Institution in Baltimore reporting that adult muscle stem cells do not have the same genetic requirements as embryonic and newborn muscle stem cells. Among the implications: Scientists shouldn’t make assumptions about the biology of adult stem cells based on research into their embryonic progenitors, and should carefully consider the potential impacts of the age of stem cells used in transplantation-based therapies. Dr. Conway suggests that “these remarkable and unexpected findings will stimulate thinking in the entire field of myogenesis” — the process of muscle tissue formation.

The editors at Nature felt the study was so important they rushed it into print online as a “near final version.”

On the print side of the the publication, Wells Center Director Mervin Yoder, MD, and researcher Momoko Yoshimoto contributed to a study with Harvard University scientists that provides a better understanding of how the first hematopoietic stem cells are formed in embryonic development. Hematopoietic stem cells are the rare but critically important cells in bone marrow that enable the creation of red and white blood cells — billions of them every day. The Nature report found that the biomechanical forces created by the initial flow of blood in the embryonic aorta are necessary to cause the first hematopoietic stem cells to appear — they bud out from the interior walls of the aorta. The implication: To replicate these early stem cell processes for potential use in treating disease, it may be necessary to re-create those biomechanical forces.

• Aaron Carroll, MD, associate professor of pediatrics and a health services researcher, received $500,855 to develop a clinical decision support system to help primary care physicians optimally diagnose and treat attention-deficit/hyperactivity disorder (ADHD).
• Loren Field, PhD, professor of medicine and an investigator in the Herman B Wells Center for Pediatric Research, received $192,500 for work screening therapeutic agents in heart tissue that promote growth and repair.
• Ruben Vidal, PhD, associate professor of pathology and laboratory medicine, received $189,125 for research to develop new animal models to study abnormal iron metabolism in different organ systems and in association with a variety of neurodegenerative diseases.

IUSM has submitted more than 200 proposals for grants available in the stimulus package in a broad range of categories.

Elsewhere, this piece on proteomics work at IUSM is just one of many excellent articles in the latest issue of Research and Creative Activity, this time with the theme of “networks.” Note that the magazine, produced by the IU Office of the Vice Provost for Research in Bloomington, has unveiled a cool new website too.