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Thu, Jun 18 · Leave a Comment

From Hypothesis to Bedside

By Center for Regenerative Medicine

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Nathan Staff, M.D., Ph.D.

Medical research can be a challenging process, slowed by the complicated nature of translating research into treatment, but Mayo Clinic’s Nathan Staff, M.D., Ph.D., Assistant Professor of Neurology, has a plan to surmount these obstacles and streamline the research process. He and his colleagues detailed this plan in an issue of the journal Science Translational Medicine.

Take Alzheimer’s research for example. Even if the next major breakthrough in treating the condition is discovered, it may take up to "16 to 17 years" for that treatment to reach patients, according to Dr. Staff’s article. A study performed by the U.S. National Cancer Institute identified several “bottlenecks” causing this slowdown, some of which included: the hand-off from academia to industry, the transfer of manufacturing from research lab to good manufacturing practices (GMP), and difficulties in clinical trial regulation.

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Anthony Windebank, M.D.

Another study, by Anthony Windebank, M.D., and colleagues (Biomaterials 2000), found that a large percentage of successful studies using animal subjects were never advanced to human testing, essentially leaving the study findings in limbo. They found the causes of this were: the hand-off from academia to industry, early trial design not focused on GMP, and academic scientists not prepared for the regulatory approval process. One noted finding of this study was that several researchers expressed their ultimate goal as publishing study findings rather than developing those findings into a therapy, leaving that step to the industry.

Dr. Staff recognizes several of these hurdles in his discussion and proposes two main remedies. The first is to redirect funding towards studies aimed at producing clinical treatments. This means government funding agencies, nonprofits, and private investors must focus on approving studies that have a clear plan to translate their research for use in the field.

The second remedy is to educate researchers on the process involved in taking a therapy through the regulatory process. To facilitate this, he says, communication must be bolstered between the parties involved, the clinician-investigator and the regulating agency. Mayo Clinic provided an example of this cooperation in a recent study focused on Amyotrophic Lateral Sclerosis (ALS), often referred to as Lou Gehrig’s disease. At Mayo Clinic, the department of Neurology and the Center for Regenerative Medicine established a strong, two-way communication channel with the FDA Center for Biologics Evaluation and Research. By participating in this cooperative communication, Mayo Clinic was able to establish two studies using stem cell therapy to treat ALS in 2009 and 2011.

Dr. Staff points out that medical research is designed to bring new therapies to the community and by strengthening education and communication, those therapies can reach the community much faster.

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Tags: ALS, amyotrophic lateral sclerosis, Anthony Windebank, Lou Gehrig's Disease, Mayo Clinic, Nathan Staff, Regenerative Medicine, Research, stem cells


Tue, May 26 · Leave a Comment

Regenerative Medicine to Repair Larynx

By Center for Regenerative Medicine

David Lott, M.D. is working to establish Mayo Clinic as a leader in laryngeal transplantation utilizing regenerative medicine. He detailed his plan during a recent talk given at Mayo Clinic in Rochester.

The larynx is the rigid organ situated in the neck responsible for protecting the airway, swallowing, and producing speech. When the organ is damaged or removed due to disease, patients quickly realize how large a role it played in their everyday lives. After a laryngectomy, the procedure in which the larynx is removed, the patient must breathe through an opening in their neck. This disables their ability to smell, taste, swallow, and speak.

During his talk Dr. Lott explains that 50,000 laryngectomies are performed annually and this growing number of patients must learn to live without these basic qualities of life.

Traditional larynx transplantations using a donor organ are rare, but they have been performed. However, there are several challenges to the procedure. Many patients who would benefit from the operation have been diagnosed with cancer and therefore cannot receive the required immunosuppressive therapy, as their cancer would become metastatic. Another challenge is that the voice box is very specialized and small changes to the vocal fold itself will have a dramatic impact to the patient’s voice.

However, regenerative medicine offers promising new options. Dr. Lott proposes to engineer a patient-specific graft using stem cells from the patient and then implant the replacement graft at the time of the cancer surgery. This process will avoid the need for immunosuppressive therapies altogether.

The first step of the process is to build a scaffolding for the replacement larynx. This can be done by using either a 3D printing technique or by using a donor organ that has been decellularized. The scaffolding is then put inside a bioreactor, a machine used to promote cellular growth, and the patient’s stem cells attach to the scaffolding. After the new larynx section is complete it will be transplanted into the patient.

Even in these early stages of the research patients have expressed desire for the procedure. Seventy-five percent of those who were asked said they would sign up for the reconstruction operation if it were an option.

Mayo Clinic is currently building the first laryngeal transplantation and research center under the directorship of Dr. Lott.

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Tags: bioprinter, clinical trials, david lott, graft, Laryngeal transplantation, larynx, Mayo Clinic, Regenerative Medicine


Sat, Apr 11 · Leave a Comment

Regenerative Medicine Lecture Series – Rochester, Minn.

By Center for Regenerative Medicine

The Mayo Medical School and Center for Regenerative Medicine will host a regenerative medicine lecture series April 13-17. The lectures are part of the Mayo Medical School Regenerative Medicine Selective course, but are open to anyone who is interested in learning more about this exciting field.

For more information, view the Lecture Series Announcement or contact Katherine Campbell  or Saranya Wyles. No registration is necessary.

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Fri, Apr 10 · Leave a Comment

Research Information Center to Host “Meet the Researcher” April 16

By Center for Regenerative Medicine

David Lott M D (3)David Lott, M.D. will present “Harnessing the Power of Regenerative Medicine for Head and Neck Reconstruction” in the Research Information Center in Rochester, Minn. (Gonda Building, Lobby Level) Thursday, April 16, at 2 p.m.

Dr. Lott is dedicated to investigating disorders of the larynx (voice box) and airway. This includes bioengineering of laryngeal and tracheal tissues, laryngeal transplantation techniques and immunotherapy, laryngeal cancer, and functional outcomes of various laryngeal surgical procedures.

Join Dr. Lott to learn about the need for novel larynx and trachea reconstruction techniques and the important role regenerative medicine plays in this process. These novel approaches promise to restore breathing, swallowing, and voice to millions of suffering people.

The presentation is open to the public.

To learn more about the Mayo Clinic Research Information Center and to view past “Meet the Researchers,” please visit: http://www.mayo.edu/research/about/information-center.

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Tags: david lott, larynx, Mayo Clinic, Regenerative Medicine, research information center, stem cells, trachea


Wed, Jan 21 · Leave a Comment

Seeing Hope: A Girl and Her Family Glimpse a Better Future

By Center for Regenerative Medicine

From the pages of Mayo Clinic Magazine.

The first thing the Arndts want you to know about them is their strong faith in God. They say it’s gotten them through some very tough times — mother Tara’s two bouts of cancer (one melanoma, one Hodgkin lymphoma); daughter Kelly’s head wound that sent her to the emergency room in an ambulance; and daughter Madison’s fight with H1N1 flu, which had killed several children in their home state of Minnesota that year.

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Madison and Tara Arndt

They knew their faith would see them through another trial two years ago, but Mom and Dad were worried. Doctors at Mayo Clinic noticed lesions on Madison’s eye, and the 11-year-old’s vision was getting worse. Her sight had never been great — she’d gotten her first eyeglasses at age 4, starting with a +3 prescription. Then at age 6, local doctors saw scarring on both eyes and referred her to Mayo.

In the years between 6 and 11, Mayo Clinic worked with Madison and her parents, and Madison’s vision remained fairly stable. But these new lesions worried Mayo Clinic ophthalmologist Jose S. Pulido, M.D., as did the inflammation in her eyes.

Mayo Clinic doctors placed a steroid implant, but soon Madison had headaches and blurry vision. Her eye pressures were extremely high at 43 (normal eye pressure ranges from 12 to 22), which Dr. Pulido relieved through a prescription of four eyedrops and a pill. And even though Dr. Pulido was doing everything he and colleagues could to control Madison’s symptoms, the future frightened her family.

“We worried about the unknown,” Tara says. “That was the scary part — is she going to go blind? As a parent, you just want to know what’s wrong. Let’s get this fixed.”

Read the rest of this entry »

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Tags: alan marmorstein, best disease, biotrust, Eye disease, Jose Pulido, Madison Arndt, Matthew Clark PhD, Regenerative Medicine, stem cells, Tara Arndt


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