Scleroderma: Research Locally, Share Globally

Greater global cooperation and collaboration among scleroderma researchers and clinicians would be all that is required to fuel a drive toward important discoveries and optimize outcomes for patients with this chronic, progressive, and often debilitating autoimmune disease. And the organizations are in place to support such collaboration.

"Scleroderma is a multisystem disease, and we as a community, working together, can learn to understand it, treat it, and perhaps cure it," Dr. Daniel Furst said.

Currently, multiple research groups in multiple countries around the world strive in a largely independent way to combat scleroderma through experimental, translational, and clinical studies. These separate silos of researchers – and patients – could benefit from increased interaction and cooperation, Dr. Furst said in an interview.

"If the international community agrees to work together, we can move things forward much more quickly," said Dr. Furst, Carl M. Pearson chair of rheumatology and director of interventional therapeutics at University of California, Los Angeles Medical Center for Health Sciences.

"The scientific complexity and the clinical heterogeneity of scleroderma benefit from multiple collaborators each contributing a piece to the puzzle."

"The timing is right for this," said Dr. Furst, now that there is an organization, the World Scleroderma Foundation (WSF), to facilitate increased collaboration worldwide. He cofounded the foundation in 2009 and currently serves as treasurer.

"The main message is that we need to work together and share our experiences in order to better understand the disease, thus ameliorating our capacity to fight its evolution," said Dr. Marco Matucci-Cerinic, WSF chairman and professor of rheumatology at the University of Florence in Italy.

"Recently, we have worked all together to lift the cultural barrier differentiating the clinical behavior of the American and European physicians," Dr. Matucci-Cerinic said. "The challenge for the future is, therefore, to foster worldwide the awareness of the disease and teach the rheumatologist to have all the same approach to the disease in order to make an early diagnosis, obtain remission and [block] disease progression."

Increased collaboration also is a matter of economics, Dr. Furst said. "There isn’t enough money in any given place to do all the research that is needed."

The Switzerland-based WSF also aims to increase translational research worldwide or find ways to take scientific findings in scleroderma from the bench to the bedside. For this reason, the U.S.-based Scleroderma Research Foundation, with its focus on experimental rather than clinical research, could be an important ally in increasing the amount of translational research studies.

Promotion of greater patient awareness about scleroderma is a second major aim for the WSF. Greater education could foster earlier scleroderma diagnoses and greater participation in care, Dr. Furst said. Ten years ago, it took 1-2 years from the onset of symptoms until patients with scleroderma received the correct diagnosis. "Even today, in the United States, it’s still 6 to 9 months on average for time to diagnosis. So awareness is a major issue."

In terms of physician education, every American College of Rheumatology annual meeting includes a skin scoring session. The intention is for rheumatologists to learn how to recognize and score the skin changes in scleroderma. Dr. Furst said, "We are trying to teach rheumatologists how to recognize and score scleroderma, and we are trying to teach patients how to educate their rheumatologists."

"Not only rheumatologists, but internists as well, through listening to their patients and through accessing things like the WSF or the Scleroderma Foundation in the U.S., can make it easier for patients to understand their disease," Dr. Furst said.

Leading the charge in Europe is the EULAR Scleroderma Trials and Research (EUSTAR) group. This group "has significantly helped to raise the awareness of the disease among physicians and politicians and has pushed ahead the clinical and basic research on the disease," Dr. Matucci-Cerinic said.

"The main message is that we need to work together and share our experiences in order to better understand [scleroderma]."

Scleroderma affects people worldwide, even in regions without a strong research base. There are approximately 100,000 persons with scleroderma in the European Union and the United States, but as many as 2.5 million worldwide, said Dr. James R. Seibold, founder and principal member of Scleroderma Research Consultants in Avon, Conn. "The scientific complexity and the clinical heterogeneity of scleroderma benefit from multiple collaborators each contributing a piece to the puzzle."

There are smaller groups, including PANLAR in South America. There is also a scleroderma group in Australia, and there are national groups in Asia, "but they are not really well organized," Dr. Furst said.

"Large populations where scleroderma is both prevalent and high impact lack the infrastructure and financial resources," Dr. Seibold said. "I would include South Asia, China, and much of South America as currently underserved areas."

Even though findings will come from multiple research groups around the globe, Dr. Furst does not expect any significant hurdles in terms of protected data. "It just so happens that the scleroderma community is very collaborative. While there always will be some proprietary issues, partially because of governmental regulations, the overwhelming urge among scleroderma community researchers is to work together rather than to work apart."

The WSF is holding its second annual World Scleroderma Congress in Florence in February 2012. Dr. Furst said, "We already have over 500 registrants four or five months ahead. It really is very encouraging."

Dr. Furst disclosed that his scleroderma research is supported by the National Institutes of Health, Gilead, and Actelion. Dr. Matucci-Cerinic said the he had no relevant disclosures. Dr. Seibold said he has consultancy relationships with a number of companies considering drug development projects in scleroderma, including Actelion, Bristol Myers Squibb, Celgene, Genentech, Gilead, MedImmune, NexMed, Pfizer, Sanofi-Aventis, Sigma Tau, and United Therapeutics.

Greater global cooperation and collaboration among scleroderma researchers and clinicians would be all that is required to fuel a drive toward important discoveries and optimize outcomes for patients with this chronic, progressive, and often debilitating autoimmune disease. And the organizations are in place to support such collaboration.

"Scleroderma is a multisystem disease, and we as a community, working together, can learn to understand it, treat it, and perhaps cure it," Dr. Daniel Furst said.

Currently, multiple research groups in multiple countries around the world strive in a largely independent way to combat scleroderma through experimental, translational, and clinical studies. These separate silos of researchers – and patients – could benefit from increased interaction and cooperation, Dr. Furst said in an interview.

"If the international community agrees to work together, we can move things forward much more quickly," said Dr. Furst, Carl M. Pearson chair of rheumatology and director of interventional therapeutics at University of California, Los Angeles Medical Center for Health Sciences.

"The scientific complexity and the clinical heterogeneity of scleroderma benefit from multiple collaborators each contributing a piece to the puzzle."

"The timing is right for this," said Dr. Furst, now that there is an organization, the World Scleroderma Foundation (WSF), to facilitate increased collaboration worldwide. He cofounded the foundation in 2009 and currently serves as treasurer.

"The main message is that we need to work together and share our experiences in order to better understand the disease, thus ameliorating our capacity to fight its evolution," said Dr. Marco Matucci-Cerinic, WSF chairman and professor of rheumatology at the University of Florence in Italy.

"Recently, we have worked all together to lift the cultural barrier differentiating the clinical behavior of the American and European physicians," Dr. Matucci-Cerinic said. "The challenge for the future is, therefore, to foster worldwide the awareness of the disease and teach the rheumatologist to have all the same approach to the disease in order to make an early diagnosis, obtain remission and [block] disease progression."

Increased collaboration also is a matter of economics, Dr. Furst said. "There isn’t enough money in any given place to do all the research that is needed."

The Switzerland-based WSF also aims to increase translational research worldwide or find ways to take scientific findings in scleroderma from the bench to the bedside. For this reason, the U.S.-based Scleroderma Research Foundation, with its focus on experimental rather than clinical research, could be an important ally in increasing the amount of translational research studies.

Promotion of greater patient awareness about scleroderma is a second major aim for the WSF. Greater education could foster earlier scleroderma diagnoses and greater participation in care, Dr. Furst said. Ten years ago, it took 1-2 years from the onset of symptoms until patients with scleroderma received the correct diagnosis. "Even today, in the United States, it’s still 6 to 9 months on average for time to diagnosis. So awareness is a major issue."

In terms of physician education, every American College of Rheumatology annual meeting includes a skin scoring session. The intention is for rheumatologists to learn how to recognize and score the skin changes in scleroderma. Dr. Furst said, "We are trying to teach rheumatologists how to recognize and score scleroderma, and we are trying to teach patients how to educate their rheumatologists."

"Not only rheumatologists, but internists as well, through listening to their patients and through accessing things like the WSF or the Scleroderma Foundation in the U.S., can make it easier for patients to understand their disease," Dr. Furst said.

Leading the charge in Europe is the EULAR Scleroderma Trials and Research (EUSTAR) group. This group "has significantly helped to raise the awareness of the disease among physicians and politicians and has pushed ahead the clinical and basic research on the disease," Dr. Matucci-Cerinic said.

"The main message is that we need to work together and share our experiences in order to better understand [scleroderma]."

Scleroderma affects people worldwide, even in regions without a strong research base. There are approximately 100,000 persons with scleroderma in the European Union and the United States, but as many as 2.5 million worldwide, said Dr. James R. Seibold, founder and principal member of Scleroderma Research Consultants in Avon, Conn. "The scientific complexity and the clinical heterogeneity of scleroderma benefit from multiple collaborators each contributing a piece to the puzzle."

There are smaller groups, including PANLAR in South America. There is also a scleroderma group in Australia, and there are national groups in Asia, "but they are not really well organized," Dr. Furst said.

"Large populations where scleroderma is both prevalent and high impact lack the infrastructure and financial resources," Dr. Seibold said. "I would include South Asia, China, and much of South America as currently underserved areas."

Even though findings will come from multiple research groups around the globe, Dr. Furst does not expect any significant hurdles in terms of protected data. "It just so happens that the scleroderma community is very collaborative. While there always will be some proprietary issues, partially because of governmental regulations, the overwhelming urge among scleroderma community researchers is to work together rather than to work apart."

The WSF is holding its second annual World Scleroderma Congress in Florence in February 2012. Dr. Furst said, "We already have over 500 registrants four or five months ahead. It really is very encouraging."

Dr. Furst disclosed that his scleroderma research is supported by the National Institutes of Health, Gilead, and Actelion. Dr. Matucci-Cerinic said the he had no relevant disclosures. Dr. Seibold said he has consultancy relationships with a number of companies considering drug development projects in scleroderma, including Actelion, Bristol Myers Squibb, Celgene, Genentech, Gilead, MedImmune, NexMed, Pfizer, Sanofi-Aventis, Sigma Tau, and United Therapeutics.

Greater global cooperation and collaboration among scleroderma researchers and clinicians would be all that is required to fuel a drive toward important discoveries and optimize outcomes for patients with this chronic, progressive, and often debilitating autoimmune disease. And the organizations are in place to support such collaboration.

"Scleroderma is a multisystem disease, and we as a community, working together, can learn to understand it, treat it, and perhaps cure it," Dr. Daniel Furst said.

Currently, multiple research groups in multiple countries around the world strive in a largely independent way to combat scleroderma through experimental, translational, and clinical studies. These separate silos of researchers – and patients – could benefit from increased interaction and cooperation, Dr. Furst said in an interview.

"If the international community agrees to work together, we can move things forward much more quickly," said Dr. Furst, Carl M. Pearson chair of rheumatology and director of interventional therapeutics at University of California, Los Angeles Medical Center for Health Sciences.

"The scientific complexity and the clinical heterogeneity of scleroderma benefit from multiple collaborators each contributing a piece to the puzzle."

"The timing is right for this," said Dr. Furst, now that there is an organization, the World Scleroderma Foundation (WSF), to facilitate increased collaboration worldwide. He cofounded the foundation in 2009 and currently serves as treasurer.

"The main message is that we need to work together and share our experiences in order to better understand the disease, thus ameliorating our capacity to fight its evolution," said Dr. Marco Matucci-Cerinic, WSF chairman and professor of rheumatology at the University of Florence in Italy.

"Recently, we have worked all together to lift the cultural barrier differentiating the clinical behavior of the American and European physicians," Dr. Matucci-Cerinic said. "The challenge for the future is, therefore, to foster worldwide the awareness of the disease and teach the rheumatologist to have all the same approach to the disease in order to make an early diagnosis, obtain remission and [block] disease progression."

Increased collaboration also is a matter of economics, Dr. Furst said. "There isn’t enough money in any given place to do all the research that is needed."

The Switzerland-based WSF also aims to increase translational research worldwide or find ways to take scientific findings in scleroderma from the bench to the bedside. For this reason, the U.S.-based Scleroderma Research Foundation, with its focus on experimental rather than clinical research, could be an important ally in increasing the amount of translational research studies.

Promotion of greater patient awareness about scleroderma is a second major aim for the WSF. Greater education could foster earlier scleroderma diagnoses and greater participation in care, Dr. Furst said. Ten years ago, it took 1-2 years from the onset of symptoms until patients with scleroderma received the correct diagnosis. "Even today, in the United States, it’s still 6 to 9 months on average for time to diagnosis. So awareness is a major issue."

In terms of physician education, every American College of Rheumatology annual meeting includes a skin scoring session. The intention is for rheumatologists to learn how to recognize and score the skin changes in scleroderma. Dr. Furst said, "We are trying to teach rheumatologists how to recognize and score scleroderma, and we are trying to teach patients how to educate their rheumatologists."

"Not only rheumatologists, but internists as well, through listening to their patients and through accessing things like the WSF or the Scleroderma Foundation in the U.S., can make it easier for patients to understand their disease," Dr. Furst said.

Leading the charge in Europe is the EULAR Scleroderma Trials and Research (EUSTAR) group. This group "has significantly helped to raise the awareness of the disease among physicians and politicians and has pushed ahead the clinical and basic research on the disease," Dr. Matucci-Cerinic said.

"The main message is that we need to work together and share our experiences in order to better understand [scleroderma]."

Scleroderma affects people worldwide, even in regions without a strong research base. There are approximately 100,000 persons with scleroderma in the European Union and the United States, but as many as 2.5 million worldwide, said Dr. James R. Seibold, founder and principal member of Scleroderma Research Consultants in Avon, Conn. "The scientific complexity and the clinical heterogeneity of scleroderma benefit from multiple collaborators each contributing a piece to the puzzle."

There are smaller groups, including PANLAR in South America. There is also a scleroderma group in Australia, and there are national groups in Asia, "but they are not really well organized," Dr. Furst said.

"Large populations where scleroderma is both prevalent and high impact lack the infrastructure and financial resources," Dr. Seibold said. "I would include South Asia, China, and much of South America as currently underserved areas."

Even though findings will come from multiple research groups around the globe, Dr. Furst does not expect any significant hurdles in terms of protected data. "It just so happens that the scleroderma community is very collaborative. While there always will be some proprietary issues, partially because of governmental regulations, the overwhelming urge among scleroderma community researchers is to work together rather than to work apart."

The WSF is holding its second annual World Scleroderma Congress in Florence in February 2012. Dr. Furst said, "We already have over 500 registrants four or five months ahead. It really is very encouraging."

Dr. Furst disclosed that his scleroderma research is supported by the National Institutes of Health, Gilead, and Actelion. Dr. Matucci-Cerinic said the he had no relevant disclosures. Dr. Seibold said he has consultancy relationships with a number of companies considering drug development projects in scleroderma, including Actelion, Bristol Myers Squibb, Celgene, Genentech, Gilead, MedImmune, NexMed, Pfizer, Sanofi-Aventis, Sigma Tau, and United Therapeutics.