Researchers have more training opportunities

NIH wants next research generation trained

When the National Institutes of Health (NIH) established the Rare Diseases Clinical Research Network (RDCRN) in 2003 with $51 million in grant funding over five years, one of the NIH's stated goals was to train the next generation of investigators.

Investigators working within the RDCRN have found these goals attainable, largely because of the network's ability to improve research infrastructure and the additional funding available for hiring young physicians/investigators.

"Everyone wants young investigators in their field," says Charlie Strange, MD, professor of pulmonary and critical care medicine at the Medical University of South Carolina (MUSC) in Charleston.

Strange has a rare disease fellow who is working on a project involving Alpha-1 Antitrypsin Deficiency, under Strange's guidance.

One of the big focuses of the neurologic channelopathies consortium is training junior neurologists who are neophytes in the study of rare diseases, says Richard J. Barohn, MD, chair and professor in the department of neurology at the University of Kansas Medical Center in Kansas City, KS.

"We hope this experience will stimulate them to stay in the field and do research into rare neuropathies," Barohn says. "At each site we have a young neurologist and a senior investigator."

A big portion of the consortium's grant has been designated for training this next generation of young neurologists, he notes.

"At monthly conference calls, we try to have young neurologists lead the discussion and give data from their site," Barohn says. "I think our project is the only one that is doing that actively."

Barohn is training his second young neurologist since the network was formed, and some of the sites have as many as three young neurologists, he notes.

Some of the young neurologists come up with their own independent grant funding, and this helps to keep them active in the field, Barohn adds.

The young neurologists are post-fellowship doctors, and some of them are junior assistant professors in academics. They can spend half of their time on the RDCRN project, which gives them time to interact with patients and families, doing detailed physical exams, and participating in monthly network conferences, Barohn explains.

RDCRN sites, such as MUSC's, which are part of the lung disease consortium, are finding funds from either NIH grants or foundations to start projects with the purpose of developing pilot data for young investigators, Strange explains.

"We want to launch them into a career that has lots of investigator support," Strange says. "The grant actually pays for independent fellows to train in rare disease research, to establish these [rare diseases] foundations, and to provide for some small clinical trials in each of the clinical areas."

Bright young physicians have lots of career paths open to them, so the hope is that RDCRN sites will obtain enough funding to train them in rare disease research, which will keep them in the business of being good clinical researchers rather than have them steered toward the more lucrative private practice medicine, Strange says.

"Based on philanthropic funds, we've been able to support specific fellowships within the network," says Brendan Lee, MD, PhD, an investigator with the Howard Hughes Medical Institute and a professor in the department of molecular and human genetics at Baylor College of Medicine in Houston, TX. Lee studies urea cycle disorders as part of the network.

"It's a strength of our network — the ability to supplement NIH funds," Lee adds. "One of the fellows we funded here is leaving and taking a faculty position to be a rare disease physician, so I know this process works."

One of the investigators involved in the RDCRN says he personally has benefited from the training and mentoring opportunities provided by the network.

"I've been more involved in clinical evaluations and not so much in research," says Steve Skinner, MD, a senior clinical geneticist and associate director and director of clinical services at the Greenwood Genetic Center in Greenwood, SC.

"I've spent a good bit of time with Rett Syndrome research, being trained by investigators in Alabama and Houston," Skinner says.

"This is an ideal model," Skinner says. "I have not actually been involved in this extensive degree of clinical research before, so I have learned to walk through the IRB process with institutions, and I'm learning data analysis and how to conduct clinical trials because all of that is included in our training."