A: The Jaime Leandro Foundation (JLF) is a private nonprofit created to help patients access personalized neoantigen peptide vaccines via the expanded access patient pay cost recovery program. The expanded access program gives individuals the right to try a medical treatment when no comparable alternative options are available. Serving as the hub, JLF brings the critical components together, partnering with expert academic and commercial institutions to use advanced genetic sequencing, identify the best patient-specific neoantigen targets, and manufacture, assemble, vial, and ship the personalized vaccine. The vaccines are administered by a treating physician and JLF partners monitor and report results for the advancement of knowledge overall.

A: There are many different types of cancer vaccines, including DNA, peptide, and mRNa vaccines. JLF helps patients access personalized neoantigen peptide vaccines. Personalized neoantigen peptide vaccines are being studied in clinical trials and are considered an experimental therapy.

A: A personalized neoantigen peptide vaccine is a type of immunotherapy that attempts to use small pieces of proteins, called peptides, to stimulate a specific component of the immune system (t-cells) to recognize cancer cells. Cancer cells often have proteins (neoantigens) on their surface that are different from those of healthy cells. Using whole exome sequencing and algorithms, the unique changes (mutations) predicted to be recognized by the immune system can be prioritized and included in the vaccine. When a person receives a personalized neoantigen peptide cancer vaccine, their immune system recognizes the peptides as foreign and mounts an immune response. Personalized neoantigen peptide vaccines are being studied in clinical trials and are considered an experimental therapy.

A: JLF leverages the FDA’s expanded access patient pay cost recovery program to bring personalized neoantigen peptide vaccines to eligible patients. Patient eligibility is determined on a case by case basis. However, generally speaking, the FDA requirements for compassionate access are that the patient has been diagnosed with a serious disease for which there are not curative therapies available and the potential benefits of a personalized neoantigen peptide vaccine outweigh the potential risks. If a patient meets this criteria, JLF will work with the physician to submit a single patient IND for review with the FDA or a patient can enroll in an open protocol. All protocols must be reviewed and approved by the FDA before the vaccine can be administered.

A: On average, it takes 4-5 months from receipt of whole exome sequencing to deliver the series of vaccines to the treating physician. A breakdown of the timeline is as follows:

1. Whole exome sequencing (if needed): 4 weeks

2. Vaccine neoantigen target identification & FDA filings: 6 weeks

3. Vaccine manufacturing: 10 - 12 weeks

A: Patients receive seven vaccines to be administered following the administration protocol. Additional doses could be appropriate, but are decided in partnership with the treating physician and involve additional cost.

A: JLF leverages the expanded access patient pay cost recovery program to help make personalized cancer vaccines available to qualifying patients. JLF fees include the cost to design, manufacture, and ship a series of vaccines for administration. The cost for a series of personalized neoantigen cancer vaccines is roughly $83,000. A breakdown of the costs are as follows:

1. Neoantigen target identification and vaccine design: $20,000

2. Vaccine manufacturing and vialing: $51,000

3. Shipping and immune monitoring / testing: $12,000

4. Additional administrative costs to store, prepare, and administer the vaccine may be charged by the treating physician. These fees are at the discretion of the physician and vary by physician, but can range from $15,000 - $30,000.

A: No, JLF is a nonprofit organization.

A: Every patient should make the decision to pursue vaccination through expanded access only if a loss of the financial resources will not cause a hardship for the family. Since this is a cost recovery model, the money is immediately transferred to the partner(s) responsible for designing, manufacturing, and/or assembling the vaccine. There is no way to refund money once design, manufacturing, or vialing has started. JLF splits payment into 3 invoices, which allows us to only collect for services being performed at that particular stage of vaccine development. Once payment is made, we incur costs that cannot be refunded.

A: Yes. JLF only serves as the facilitator in vaccine design and manufacturing. The process requires each patient to identify an appropriate physician to serve as the study investigator. Once approved, the investigator will be responsible for following study guidelines related to vaccine storage, preparation, administration, and updating Patient Drug Accountability Logs. JLF has resources available to help interested physicians better understand the requirements needed to serve as a vaccine administrator.

A: The side effects and risk of personalized neoantigen peptide vaccines are not known. The treatment protocol and consent form are available upon request and should be reviewed with the treating physician.

A: Because JLF leverages the compassionate access program, most patients with advanced cancers are candidates. The patient and treating physician have the discretion to use treatments they deem appropriate based on the latest science and any relevant personalization approaches identified.

A: Personalized neoantigen peptide vaccines are considered experimental therapies that come with no guaranteed outcome. Clinical trial data continues to be the best sources for researching efficacy of personalized cancer vaccines. In general, the effectiveness of personalized therapies may vary by cancer type, stage of disease, and the patient’s overall health.

A: JLF has worked with patients with a variety of cancer diagnoses, including breast, ovarian, glioblastoma, pancreatic, lung, and sarcoma. As of February 2023, a total of 13 patients have been treated and an additional 13 patients are in the queue for vaccine development or manufacturing.

A: No, not all clinical trials must be registered on ClinicalTrials.gov. If information is not included on about expanded access availability for a particular investigational drug, physicians or patients may wish to contact the sponsor (or manufacturer of the investigational drug, if different from the sponsor) about possible availability.

So, there are some things to be said. There is a nice presentation from Covington here.

https://www.fdli.org/wp-content/uploads/2017/05/annual17-thursday-Communicating-Emerging-Drug-Therapies.pdf

A: Information about the availability of expanded access to an investigational drug may be available on the website of the relevant drug manufacturer or distributor. The enactment of the 21st Century Cures Act added section 561A to the Federal Food, Drug, and Cosmetic Act (FD&C Act); that section requires a manufacturer or distributor of one or more investigational drugs for the diagnosis, monitoring, or treatment of one or more serious diseases or conditions to make its policy for evaluating and responding to expanded access requests submitted under section 561(b) of the FD&C Act both public and readily available, such as by posting the policy on a publicly available website.

Additionally, information on publicly and privately supported clinical trials on a wide range of diseases and conditions is available at ClinicalTrials.gov, a Web-based resource maintained by the National Library of Medicine for patients, their families, health care professionals, researchers, and the public.

JLF’s F&A rate is 11%.