Crowdsourcing A Cure For Scleroderma PDF Print E-mail
Thursday, 18 April 2013 06:38
The University of Michigan Health System is widely known for medical innovation — from the research laboratory to the patient’s bedside — but in partnership with dedicated donors, the institution is also innovating the way it raises money to accelerate groundbreaking research.

With the help of local philanthropists Jon and Lisa Rye, whose close family member was diagnosed with the debilitating skin disease Scleroderma, UMHS is reaching out to the Scleroderma community to help fund promising new research underway in the U-M Division of Rheumatology.

“We are extremely optimistic that the research being done at the U-M will lead us closer to a cure for Scleroderma and other devastating fibrotic diseases,” says Jon Rye, chairman of Greenfield Partners in Birmingham, Mich., who has given more than $2.5 million to the U-M Scleroderma Program. “Given the current climate for science funding, we are asking other families whose lives have been touched by this deadly disease to partner with us in hopes of getting a cure for everyone who is suffering as quickly as possible.”

Scleroderma is a crippling autoimmune disorder that affects more than 300,000 people in the U.S. alone. It is characterized by a thickening and hardening of the skin that is so severe, it steals away the use of patients’ fingers, hands and limbs. As it advances across their bodies, the uncontrolled growth of fibrous tissue can also damage patients’ hearts, lungs and other organs, often leading to a prolonged and painful death.

“As we watched our loved one’s extremities harden, his fingers curve and his hands lose all strength, we lived a nightmare, and we could only imagine how painful it was for him,” says Lisa Rye. Doctors at the U-M were able to determine that the illness was actually a rare condition in the Scleroderma family of diseases — generalized subcutaneous morphea — and with an accurate diagnosis, a better path for him finally could begin.

But there are many others for whom effective treatment remains elusive. New compounds developed at the U-M hold promise for shutting down the progression of Scleroderma by targeting the molecular mechanisms driving it.

“As a clinician, the promise of this research makes me very hopeful,” says Dinesh Khanna, M.D., M.S., the Marvin and Betty Danto Research Professor of Internal Medicine and director of the U-M Scleroderma Program. “If successful, it could mean a huge improvement in the quality of life and function for patients here at U-M and around the world.”

The discovery originated in the lab of Rick Neubig, M.D., Ph.D., professor of pharmacology and associate professor of internal medicine in the U-M Medical School. Neubig also heads the U-M Center for the Discovery of New Medicines.

What Neubig and his team did was develop a new approach to stopping the reaction that leads to the disease in the first place. “Most of the ways researchers have tried to treat Scleroderma in the past have revolved around blocking the initial inflammation,” says Neubig. “But just cutting off the inflammation does not stop the progression of the associated fibrosis. Our compounds target a genetic switch that controls the formation of myofibroblasts — which are the cells that produce too much collagen leading to the thickening of the skin and damage to other organs.”

The next steps will be to expand testing in patient cells, continue to improve and refine the compounds, and most importantly demonstrate success in rodent models.

“Given the difficult environment for federal research funding, we are seeking philanthropic support from the community to accelerate the completion of the laboratory research and, hopefully, speed a treatment to patients,” says David Fox, M.D., who heads U-M’s Division of Rheumatology.

To support fundraising efforts, U-M has created a secure online giving page for donations to support the next stage of research and development. The message will also be shared through the crowdfunding website CrowdRise.com.

“More than $500,000 will be required to take our research to the next level,” says Fox. “We’re hoping that individuals who have been impacted by this terrible disease will rally in support of our research.”

Fox cautions that even working quickly, advancing to human trials would still take time. “A few years may sound like a long time,” he says, “but when you’re talking about something that has the potential to dramatically improve the quality of life for hundreds of thousands of patients who may suffer and die because of Scleroderma, it’s important to take the time to get the science right.”

Source: University of Michigan Health System (2013), "UMHS partners with local philanthropists to crowdfund the search for scleroderma’s cure";
 
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