Lymphoma Researchers Receive LRF Grants to Investigate Potential New Treatments

Last week the Lymphoma Research Foundation (LRF) announced the awarding of $1.62 million in funding for lymphoma research and lymphoma related training grants. Among the awardees were two Lymphoma Program research collaborators, Dr. Leandro Cerchietti and Dr. Pilar Dominguez Rodriguez.

Leandro Cerchietti
Leandro Cerchietti, M.D.

Dr. Cerchietti is an Assistant Professor of Medicine and Raymond and Beverly Sackler Research Scholar at Weill Cornell Medicine. He received a grant from the LRF for his work in predicting follicular lymphoma transformation without biopsy. For follicular lymphoma (FL) patients their slow growing tumor can turn into a much more aggressive follicular lymphoma that limits their treatment options. The mechanisms behind these transformations are poorly understood, but researchers are trying to better understand the mechanism of transformation. Currently invasive and expensive biopsies are the only way to determine whether a patient is at risk for follicular transformation. Based on his previous research Dr. Cerchietti has determined that FL cells release certain products into a patient’s body, and that these products in the bloodstream can be used to anticipate FL transformation. Dr. Cerchietti plans to build on his previous research and potentially develop new non-chemotherapy treatments for follicular lymphoma.

mariadelpilar
Pilar Dominguez Rodriguez, Ph.D.

Dr. Dominguez Rodriguez is a Post-Doctoral Associate, who specialized in cancer biology in Dr. Ari Melnick’s lab at Weill Cornell Medicine. Her current research focuses on the ten eleven translocation 2 (TET2) gene. TET2 is associated with DNA methylation, a process involved in the regulation of certain genes. Previously in patients with diffuse large B-cell lymphomas (DLBCL) the deregulation of DNA methylation has been identified as a source of DLBCL cell growth. However, researchers are still searching for answers as to why the methylation mechanisms malfunction. TET2 could potentially be a link due to its role in DNA methylation and the fact that is frequently mutated in lymphomas. Dr. Dominguez Rodriguez project seeks to discover whether there is a relationship between TET2 and DNA methylation in B-cells, and then identify how TET2 affects the development of DLBCL. If this relationship can be established the findings have the potential to identify new treatment targets for patients with B-cell lymphomas.

Dr. John Leonard on Treating Patients with Relapsed Follicular Lymphoma

In this video from OncLive, Lymphoma Program Director, Dr. John Leonard discusses treatment considerations for patients with relapsed follicular lymphoma.

The FDA Breakthrough Therapy Designation: A Primer

 

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By Peter Martin, M.D.

The Breakthrough Therapy Designation was introduced as part of the 2012 Food and Drug Administration Safety and Innovation Act and is designed to expedite the development of new treatments for serious conditions like lymphoma. If preliminary evidence from clinical trials demonstrates that a new drug represents a significant improvement over currently available therapies, the drug developer (also called the Sponsor) may request a Breakthrough Therapy Designation.

If the FDA agrees with the Sponsor and grants the designation, they will subsequently commit to providing additional resources to the development and review process.  Practically this means  more frequent meetings and communications with senior FDA officials, and aid in designing more efficient clinical trials. Although the Breakthrough Therapy Designation does not make a new drug available, early experience with the program suggests that it can shave years off of the typical development process.

Since 2012, the Sponsors of 342 treatments have applied for this status, with 111 receiving the designation. Of the treatments that have received the designation 42 have received full approval. In 2016 pembrolizumab received the designation for the treatment of patients with relapsed or refractory classical Hodgkin lymphoma and venetoclax received it in combination with rituximab to treat patients with relapsed/refractory chronic lymphocytic leukemia. Previously nivolumab, idelalisib, ofatumumab, obintuzumab, and ibrutinib all received the Breakthrough Therapy Designation for different lymphoma indications.

Despite the success of the program there are several points to keep in mind. The FDA’s definition of “breakthrough” is very different from its interpretation by lay persons and the media. The FDA’s definition refers to a drug that in the early stages of development has shown the potential for an improvement in patient care. It is not a guarantee of the approval or long-term success of the treatment.

As the FDA and Sponsors gain additional experience with the program we are likely to see some changes, including the number of applications and the cost of requesting a Breakthrough Therapy Designation. We are also likely to witness some unintended consequences, including investments by venture capitalists and opaque marketing strategies. When in doubt a patient should consult a physician about the usefulness of any treatment.

At the Weill Cornell Lymphoma Program, we are interested in any program that helps make promising new therapies available to patients as quickly as possible, and so we continue to follow this and other related programs closely. In the next post in the series we will discuss the Fast Track Designation.

Previous Entries in the Primer Series

The FDA Approval Process